FINAL PROGRAM American Society of Gene & Cell Therapy

13th Annual Meeting | Washington, DC USA May 19-22, 2010 Marriott Wardman Park

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Jointly sponsored by Indiana University School of Medicine and the American Society of Gene & Cell Therapy

www.asgct.org

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13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Table of Contents

ASGCT Officers and Board of Directors

A Welcome from Kenneth Cornetta, MD . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2 Committee Meeting Schedule . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 3 Committee Rosters. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4 Planning Committee and Group Coordinating Reviewers. . . . . . . . . . . . . . . . . . . . . . . . . . . 8 Annual Meeting Supporters. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 9 Annual Meeting Exhibitors. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10 Abstract Reviewers. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11 Excellence in Research Award and Travel Grant Recipients. . . . . . . . . . . . . . . . . . . . . . . . 13 Outstanding New Investigator & Outstanding Achievement Award Recipients. . . . . . . . . . . 14 Faculty Listing. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 15

President (09/10) Kenneth Cornetta, MD President-Elect (09/10) Barrie J. Carter, PhD Vice President (09/10) R. Jude Samulski, PhD Secretary (10) John F. Engelhardt, PhD Treasurer (11) Beverly L. Davidson, PhD Board Members

Christopher Baum, MD Michele P. Calos, PhD

General Meeting Information. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19

Cynthia Dunbar, MD

Marriott Wardman Park Hotel Floor Plans . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 23

Helen E. Heslop, MD

Schedule-at-a-Glance. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25

Harry L. Malech, MD

Detailed Program Schedule. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 27

Stephen J. Russell, MD, PhD

Exhibit Hall Floor Plan. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 76

Brian P. Sorrentino, MD

Exhibitor Descriptions. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 77 Exhibitor Information. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 84 Washington, DC Fun Facts. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85 Housing Information and Map of Hotel Area.. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85 Abstract Directory. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 86 Abstract Author Index. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 143 ASGCT Executive Office

555 East Wells Street, Suite 1100 Milwaukee, WI 53202 Phone: 414.278.1341 Fax: 414.276.3349 [email protected] www.asgct.org

Mary Dean – Executive Director Erin Hankey – Administrative Assistant Ken Janowski – Program Manager Roseann Marotz – Meetings Manager David Wood – Senior Meetings & Membership Manager Tara Withington, CAE – Consulting Partner

Samuel C. Wadsworth, PhD Matthew D. Weitzman, PhD John J. Rossi, PhD Chair, Advisory Council (ex-officio) Malcolm K. Brenner, MD, PhD Editor-in-Chief, Molecular Therapy (ex-officio)

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Welcome Dear Colleagues, On behalf of the American Society of Gene & Cell Therapy, welcome to the Society’s 13th Annual Meeting in Washington, DC. Since our last Annual Meeting, the field has continued to make strides. We are seeing clinical results reported in Science, Lancet, and The New England Journal of Medicine that show the continued promise and progress of our field. The Annual Meeting program developed by the ASGCT Program Committee, the scientific and standing committees, and the abstract review groups, highlights many of the developments in the field over the past year and gives a glimpse into the potential advances to come over the next year. The 2010 program features 24 scientific symposia, 10 education sessions, 28 oral abstract sessions, 3 poster sessions, and 4 meet-the-investigator lunch sessions. The Plenary Symposia this year will highlight a few of the most exciting developments in our field. Dr. Hal E. Broxmeyer will present the keynote lecture during the George Stamatoyannopoulos Lecture on stem and progenitor cell function. During the Presidential Symposium, Dr. Jean Bennett will present on gene therapy for congenital blindness and Dr. David Williams, the immediate past Editor-in-Chief of Molecular Therapy, will present on gene therapy for Fanconi anemia. Lastly, Dr. Katherine High, the 2010 Outstanding Achievement Award winner, will give a retrospective of her contributions to the field. A thank you is owed to the many exhibitors, non-profit organizations and industry supporters for their participation and generosity. Please be sure to take time to visit the vendors in the exhibit hall this year. Each exhibitor and supporter is vital to the success of the Annual Meeting. Gratitude is also due to the Annual Meeting faculty for sharing their expertise and time to make this a truly unique event. Furthermore, our membership is indebted to Mary Dean and the ASGCT Staff who work tirelessly at the Annual Meeting and throughout the year for our Society. And finally, thank you, the Annual Meeting participants, for attending the Society’s Annual Meeting. Welcome to Washington, DC and the 13th Annual Meeting! Sincerely,

Ken Cornetta, MD President, ASGCT

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13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Committee Meeting Schedule Committee terms technically commence and conclude at the Annual Meeting. Incoming chair and committee member terms officially go into effect on May 22, 2010 at the Business Meeting. All meetings will be held at the Marriott Wardman Park Hotel unless otherwise noted. WEDNESDAY, MAY 19th

11:45 am – 12:45 pm

Membership Committee Meeting

Harding Room

International Committee Meeting

Hoover Room

Education Committee Meeting

Park Tower 8216

Publications Committee Meeting

Park Tower 8212

12:30 pm – 1:30 pm

Scientific Committee Meetings: Cancer Gene & Cell Therapy; Cardiovascular Gene & Cell Therapy; Embryonic/Somatic Stem Cell and Tissue Engineering; Gene & Cell Therapy of Genetic Diseases; Genetic Vaccines; Hemopoietic Cell Gene Therapy; Immunology of Gene & Cell Therapy; Infectious Diseases; Musculo-Skeletal Disorders Gene Therapy; Neural Disorders Gene Therapy; Oligonucleotide Based Therapies; Physical Gene & Cell Therapy and Vectorology; Respiratory Tract Gene & Cell Therapy; Tissue Engineering; Viral Gene Transfer Vectors

Thurgood Marshall North and East

12:45 pm – 1:45 pm

Advisory Council Meeting

Park Tower 8222

Clinical & Regulatory Affairs Committee Meeting

Park Tower 8228

Ethics Committee Meeting

Congressional Room

Translational Science & Product Development Meeting

Park Tower 8219

Molecular Therapy Editorial Board Meeting

Maryland Suite

8:00 am – 8:30 am

Society Annual Business Meeting

Delaware Suite

1:15 pm – 2:15 pm

Bio-Industry Liaison Committee Meeting

Harding Room

THURSDAY, MAY 20th

12:45 pm – 1:45 pm

FRIDAY, MAY 21st

8:30 pm – 10:00 pm SATURDAY, MAY 22nd

Society Annual Business Meeting The ASGCT Business Meeting will be conducted on Saturday, May 22, 8:00 am – 8:30 am. A continental breakfast will be served at 7:45 am in the Delaware Suite. All ASGCT members are welcome to attend. Election results will be announced, new officers will be installed and the Society’s finances and membership statistics will be reviewed. We welcome your input and participation!

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Committee Rosters Advisory Council

Chair Mark A. Kay, MD, PhD (2010 - 2011) John J. Rossi, PhD (2009 - 2010) Members David M. Bodine, PhD (2009 - 2014) Kenneth Cornetta, MD (2010 - 2015) Theodore Friedmann, MD (2007 - 2012) Katherine A. High, MD (2005 - 2010) Leaf Huang, PhD (2006 - 2011) Mark A. Kay, MD, PhD (2006 - 2011) Andra E. Miller, PhD (2007 - 2012) Luigi M. Naldini, MD, PhD (2008 - 2013) Arthur W. Nienhuis, MD (2008 - 2013) Katherine P. Ponder, MD (2009 - 2014) John J. Rossi, PhD (2005 - 2010) Bio-Industry Liaison Committee

Chair Seng H. Cheng, PhD (2010 - 2011) Thomas Tillett (2008 - 2010) Vice Chairman Douglas J. Jolly, PhD (2008 - 2010) Members Estuardo Aguilar-Cordova, PhD (2008 - 2011) Joseph T. Bruder, PhD (2010 - 2013) Anthony T. Cheung, PhD (2008 - 2011) Robert L. Engler, MD (2008 - 2011) Michael Fons, PhD (2007 - 2013) Joyce L. Frey-Vasconcells, PhD (2009 - 2012) James E. Hagstrom, PhD (2010 - 2013) William F. Kaemmerer, PhD (2010 - 2013) Karen Kozarsky, PhD (2007 - 2013) Edward Lanphier (2008 - 2011) Jeffrey M. Ostrove, PhD (2008 - 2011) Ralph W. Paul, PhD (2004 - 2010) Vladimir Slepushkin, MD, PhD (2006 - 2012) Sean M. Sullivan, PhD (2010 - 2013) Geoff Symonds, PhD (2006 - 2012) Gabor Veres, PhD (2007 - 2010) Samuel C. Wadsworth, PhD (2004 - 2010)

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Cancer Gene & Cell Therapy Committee

Chemical Gene & Cell Therapy Committee

Chair Laurence JN Cooper, MD, PhD (2009 - 2011) Members Chiara Bonini, MD (2009 - 2012) Renier J. Brentjens, MD, PhD (2010 - 2013) Stephen Gottschalk, MD (2007 - 2013) John G. Hay, MD (2007 - 2010) Akseli E. Hemminki, MD, PhD (2007 - 2010) Maciej S. Lesniak, MD, FACS (2009 - 2012) Richard A. Morgan, PhD (2009 - 2012) John J. Nemunaitis, MD (2008 - 2011) Daniel H. Sterman, MD (2007 - 2013) Savio L.C. Woo, PhD (2010 - 2013) Lily Wu, MD, PhD (2010 - 2013) Masato Yamamoto, MD, PhD (2004 - 2010)

Chair Todd Giorgio, PhD (2009 - 2010) Theresa M. Reineke, PhD (2010 - 2011) Members Roger C. Adami, PhD (2007 - 2013) Todd Giorgio, PhD (2010 - 2011) Hideyoshi Harashima, PhD (2007 - 2010) James G. Hecker, MD, PhD (2006 - 2012) Kam W. Leong, PhD (2004 - 2010) Ian MacLachlan, PhD (2010 - 2013) Yong Serk Park, PhD (2007 - 2010) Suzie H. Pun, PhD (2007 - 2013) Theresa M. Reineke, PhD (2007 - 2010) Kevin G. Rice, PhD (2010 - 2013) Cyrus R. Safinya, PhD (2010 - 2013) Millicent O. Sullivan, PhD (2010 - 2013) Ernst Wagner, PhD (2007 - 2010) David B. Weiner, PhD (2010 - 2013)

Cardiovascular Gene & Cell Therapy Committee

Chair Barry J. Byrne, MD, PhD (2010 - 2011) H. Kirk Hammond, MD (2008 - 2010) Members David A. Dean, PhD (2007 - 2010) David A. Dichek, MD (2010 - 2013) J. Kevin Donahue, MD (2004 - 2010) Michael P. Murphy, MD (2009 - 2012) Timothy O’Brien, MD PhD (2009 - 2012) Gabor M. Rubanyi, MD, PhD (2010 - 2013) Sonia I. Skarlatos, PhD (2004 - 2010) Matthew L. Springer, PhD (2007 - 2013) Karen A. Vincent, PhD (2008 - 2011)

Clinical & Regulatory Affairs Committee

Chair Carl H. June, MD (2009 - 2011) Members Dale Ando, MD (2010 - 2013) Joy Cavagnaro, PhD, DABT, RAC (2009 - 2010) Jacqueline Corrigan-Curay, MD, JD (2008 - 2011) Elizabeth Kang, MD (2009 - 2012) Thomas M. Miller, PhD (2008 - 2011) Glenn F. Pierce, MD, PhD (2004 - 2010) Daniel M. Takefman, PhD (2009 - 2012) Peter K. Working, PhD, DABT (2010 - 2013) J. Fraser Wright, PhD (2009 - 2012)

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Committee Rosters Education Committee

Co-Chairs Linda B. Couto, PhD (2010 - 2011) Roland W. Herzog, PhD (2008 - 2010) Fulvio Mavilio, PhD (2009 - 2011) Members Linda B. Couto, PhD (2008 - 2011) William F. Goins, PhD (2007 - 2013) Scott Q. Harper, PhD (2010 - 2013) Karin Jooss, PhD (2007 - 2010) Tal Kafri, PhD (2007 - 2013) Harry L. Malech, MD (2010 - 2013) Robin J. Parks, PhD (2007 - 2010) Suzie H. Pun, PhD (2009 - 2012) Luk Vandenberghe, PhD (2010 - 2013) Embryonic/Somatic Stem Cell and Tissue Engineering Committee

Chair Gay Crooks, MD (2009 - 2011) Members Karen S. Aboody, MD (2009 - 2012) Fernando D. Camargo, PhD (2010 - 2013) Gay Crooks, MD (2008 - 2011) James Ellis, PhD (2009 - 2012) Jane Lebkowski, PhD (2010 - 2013) Jan A. Nolta, PhD (2009 - 2010) Kazuo Ohashi, MD, PhD (2006 - 2012) Evan Y. Snyder, MD, PhD, FAAP (2006 - 2012) Alice F. Tarantal, PhD (2009 - 2012) Mark E. Westhusin, PhD (2009 - 2010) Ethics Committee

Chair Jonathan Kimmelman, PhD (2008 - 2010) Nelson Wivel, MD (2010 - 2011) Members Roger H. Bertolotti, PhD (2009 - 2012) Theodore Friedmann, MD (2008 - 2011) Edward I. Ginns, MD, PhD (2009 - 2012) Dieter C. Gruenert, PhD (2010 - 2013) Diane Wara, MD (2007 - 2010) Nelson Wivel, MD (2009 - 2012)

Gene & Cell Therapy of Genetic Diseases Committee

Hemopoietic Cell Gene Therapy Committee

Chair Valder Arruda, MD, PhD (2010 - 2011) Fabio Candotti, MD (2009 - 2010) Members Valder Arruda, MD, PhD (2007 - 2010) Fatima Bosch, PhD (2010 - 2013) Fabio Candotti, MD (2010 - 2011) Lawrence C. Chan, MD (2010 - 2013) Andrew Davidoff, MD (2010 - 2013) Jim Hu, PhD (2007 - 2010) Stefan Karlsson, MD, PhD (2007 - 2010) Nuria Morral, PhD (2009 - 2012) Amit Nathwani, MD, PhD (2007 - 2010) Mark S. Sands, PhD (2009 - 2010) Thierry C. VandenDriessche, PhD (2009 - 2012) Charles P. Venditti, MD, PhD (2008 - 2011) Robin Ziegler, MS (2010 - 2013)

Chair Punam Malik, MD (2009 - 2010) Michel Sadelain, MD, PhD (2010 - 2011) Members Alessandro Aiuti, MD, PhD (2008 - 2011) Gianpietro Dotti, MD (2010 - 2013) David W. Emery, PhD (2006 - 2012) Karin Gaensler, MD (2006 - 2012) Manuel Grez, PhD (2009 - 2012) R. Keith Humphries, MD, PhD (2007 - 2010) Harry L. Malech, MD (2009 - 2010) Derek A. Persons, MD, PhD (2007 - 2013) Karen E. Pollok, PhD (2006 - 2012) Barbara Savoldo, MD, PhD (2010 - 2013)

Genetic Vaccines Committee

Chair Helen E. Heslop, MD (2009 - 2011) Members Ronald Alvarez, MD (2006 - 2012) John A. Barrett, MD (2010 - 2013) Si-Yi Chen, MD, PhD (2010 - 2013) K. Reed Clark, PhD (2004 - 2010) Ruxandra Draghia-Akli, MD, PhD (2007 - 2010) Mary Lynne Hedley, PhD (2006 - 2012) Jeffrey J. Molldrem, MD (2010 - 2013) Sattva S. Neelapu, MD (2010 - 2013) David B. Weiner, PhD (2003 - 2010)

Immunology of Gene & Cell Therapy Committee

Chair Adrian J. Thrasher, MD, PhD (2009 - 2011) Members Andrea Amalfitano, DO, PhD (2010 - 2013) Andrew Byrnes, PhD (2010 - 2013) Youhai H. Chen, MD, PhD (2007 - 2010) Gianpietro Dotti, MD (2009 - 2010) Soosan Ghazizadeh, PhD (2007 - 2010) Roland W. Herzog, PhD (2010 - 2013) Gerald S. Lipshutz, MD (2010 - 2013) John D. Mountz, MD, PhD (2007 - 2013) Daniel A. Muruve, MD (2007 - 2010) Maria-Grazia Roncarolo, MD, PhD (2004 - 2010) Denise Sabatino, PhD (2008 - 2011) Yiping Yang, MD, PhD (2010 - 2013) Infectious Diseases Committee

Chair Roberto Cattaneo, PhD (2009 - 2011) Members Ronald G. Crystal, MD (2009 - 2010) Wayne A. Marasco, MD, PhD (2006 - 2012) Steven Polyak, MD (2010 - 2013) Bruce E. Torbett, PhD, MSPH (2009 - 2012) Jeffrey B. Ulmer, PhD (2006 - 2012) James M. Wilson, MD, PhD (2007 - 2013) Stefan Worgall, MD, PhD (2006 - 2012)

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Committee Rosters International Committee

Chair John Rasko, MBBS, PhD (2009 - 2011) Members Alberto Auricchio, MD (2010 - 2013) Christopher Baum, MD (2010 - 2013) Olivier Danos, PhD (2009 - 2010) Guangping Gao, PhD (2009 - 2012) Bobby Gaspar, MD, PhD (2008 - 2011) Salima Hacein-Bey Abina, PhD (2008 - 2011) Takashi Shimada, MD, PhD (2009 - 2012) Sander Van Deventer, MD, PhD (2007 - 2013) Thierry C. VandenDriessche, PhD (2010 - 2013) Seppo Ylä-Herttuala, MD, PhD, FESC (2005 2011) Membership Committee

Chair Dexi Liu, PhD (2010 - 2011) Savio L.C. Woo, PhD (2008 - 2010) Members Margaret Black, PhD (2010 - 2013) Michele P. Calos, PhD (2010 - 2013) Larry A. Couture, PhD (2009 - 2010) Dexi Liu, PhD (2006 - 2012) Toya Ohashi, MD (2008 - 2011) Isabelle Rivière, PhD (2004 - 2010) Maria-Grazia Roncarolo, MD, PhD (2005 - 2011) John J. Rossi, PhD (2005 - 2011) Savio L.C. Woo, PhD (2010 - 2011) Ex-Officio Robert M. Frederickson, PhD (2005 - 2011)

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Musculo-Skeletal Disorders Gene & Cell Therapy Committee

Chair Paula Clemens, MD (2008 - 2010) Jerry R. Mendell, MD (2010 - 2011) Members Jeffrey Chamberlain, PhD (2010 - 2013) Alan R. Davis, PhD (2007 - 2013) Dongsheng Duan, PhD (2004 - 2010) Dan Gazit, DMD, PhD (2004 - 2010) Richard J. Gregory, PhD (2010 - 2013) Dwight Koeberl, MD, PhD (2010 - 2013) Alan J. Nixon, DVM, MS (2007 - 2013) Shin’ichi Takeda, MD, PhD (2010 - 2013) Bing Wang, MD, PhD (2010 - 2013) Neural Disorders Gene & Cell Therapy Committee

Chair Maria G. Castro, PhD (2009 - 2011) Members Robin R. Ali, PhD (2010 - 2013) William J. Bowers, PhD (2009 - 2012) Xandra O. Breakefield, PhD (2009 - 2012) Mary Bartlett Bunge, PhD (2007 - 2010) Maria G. Castro, PhD (2007 - 2010) Beverly L. Davidson, PhD (2008 - 2011) Nicole Deglon, PhD (2004 - 2010) David J. Fink, MD (2010 - 2013) John Forsayeth, PhD (2004 - 2010) Bill Hauswirth, PhD (2010 - 2013) Jeffrey H. Kordower, PhD (2007 - 2010) Anne Messer, PhD (2010 - 2013) Deborah J. Watson, PhD (2009 - 2012) John H. Wolfe, VMD, PhD (2010 - 2013)

Nominating Committee

Chair Malcolm K. Brenner, MD, PhD (2008 - 2010) Members Terence R. Flotte, MD (2009 - 2010) Luigi M. Naldini, MD, PhD (2009 - 2010) Jan A. Nolta, PhD (2009 - 2010) Samuel C. Wadsworth, PhD (2009 - 2011) Oligonucleotide Based Therapies Committee

Chair Bruce Sullenger, PhD (2009 - 2011) Members C. Frank Bennett, PhD (2010 - 2013) Brian D. Brown, PhD (2010 - 2013) Dirk Grimm, PhD (2010 - 2013) Dieter C. Gruenert, PhD (2004 - 2010) Mark A. Kay, MD, PhD (2009 - 2010) Anton P. McCaffrey, PhD (2006 - 2012) Paul B. McCray, Jr., MD (2008 - 2011) Kevin V. Morris, PhD (2010 - 2013) Matthew H. Porteus, MD, PhD (2006 - 2010) Bruce Sullenger, PhD (2004 - 2010) Marco Weinberg, PhD (2010 - 2013) Jon A. Wolff, MD (2004 - 2010) Physical Gene & Cell Therapy and Vectorology Committee

Chair Shulin Li, PhD (2009 - 2011) Members Michele P. Calos, PhD (2007 - 2013) Michael Fons, PhD (2009 - 2012) Richard Heller, PhD (2009 - 2012) R. Scott McIvor, PhD (2009 - 2012) Carol H. Miao, PhD (2009 - 2012) Lluis M. Mir, PhD, DSc (2009 - 2012) Matthew H. Porteus, MD, PhD (2010 - 2012) Sean M. Sullivan, PhD (2010 - 2013)

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Committee Rosters Program Committee

Publications Committee

Co-Chairs Kenneth Cornetta, MD (2010 - 2011) Ronald G. Crystal, MD (2010 - 2011) Stanton L. Gerson, MD (2008 - 2010) John J. Rossi, PhD (2009 - 2011) Members Valder Arruda, MD, PhD (2010 - 2011) Barry J. Byrne, MD, PhD (2010 - 2011) Bruce A. Bunnell, PhD (2009 - 2011) Fabio Candotti, MD (2009 - 2010) Maria G. Castro, PhD (2009 - 2011) Roberto Cattaneo, PhD (2007 - 2011) Seng H. Cheng, PhD (2010 - 2011) Paula Clemens, MD (2008 - 2010) Laurence JN Cooper, MD, PhD (2009 - 2011) Larry A. Couture, PhD (2010 - 2011) Gay Crooks, MD (2009 - 2011) Todd Giorgio, PhD (2009 - 2010) H. Kirk Hammond, MD (2008 - 2010) Helen E. Heslop, MD (2009 - 2011) Carl H. June, MD (2009 - 2010) Jonathan Kimmelman, PhD (2008 - 2010) Donald B. Kohn, MD (2009 - 2010) Shulin Li, PhD (2009 - 2011) Punam Malik, MD (2009 - 2010) Fulvio Mavilio, PhD (2009 - 2010) Jerry R. Mendell (2010 - 2011) Luigi M. Naldini (2010 - 2011) John Rasko, MBBS, PhD (2009 - 2011) Theresa M. Reinke, PhD (2010 - 2011) Michel Sadelain, MD, PhD (2010 - 2011) Daniel R. Salomon, MD (2010 - 2011) Bruce Sullenger, PhD (2009 - 2011) Adrian J. Thrasher, MD, PhD (2008 - 2011) Thomas Tillett (2008 - 2010) Savio L.C. Woo, PhD (2008 - 2010)

Chair Donald B. Kohn, MD (2009 - 2011) Members Hans-Peter Kiem, MD (2007 - 2013) Arthur W. Nienhuis, MD (2010 - 2013) Michel Sadelain, MD, PhD (2007 - 2013) Rachel Salzman, DVM (2008 - 2011) Brian P. Sorrentino, MD (2007 - 2010) Francis C. Szoka, Jr., PhD (2008 - 2011) Respiratory Tract Gene & Cell Therapy Committee

Chair Bruce A. Bunnell, PhD (2009 - 2011) Members David A. Dean, PhD (2010 - 2013) John F. Engelhardt, PhD (2010 - 2013) Terence Flotte, MD (2010 - 2013) Deborah Gill, PhD (2008 - 2011) Uta Griesenbach, PhD (2007 - 2010) Larry G. Johnson, MD (2009 - 2010) Jay K. Kolls, MD (2007 - 2013) Carolyn Lutzko, PhD (2007 - 2010) David W. Parsons, PhD (2007 - 2013) Tissue Engineering Committee

Chair Daniel R. Salomon, MD (2010 - 2011) Members Dan Gazit, DMD, PhD (2010 - 2013) William Giannobile, DDS, DMedSc (2010 - 2013) Bruce Levine, PhD (2010 - 2013) Paul Y. Liu, MD, FACS (2010 - 2013) Tatiana Segura, PhD (2010 - 2013) Stephanie Simek, PhD (2010 - 2013)

Translational Science & Product Development Committee

Chair Larry A. Couture, PhD (2010 - 2011) Members Joy Cavagnaro, PhD, DABT, RAC (2010 - 2013) Kenneth Cornetta, MD (2010 - 2013) Derek Hei, PhD (2010 - 2013) Doug J. Jolly, PhD (2010 - 2013) Ian MacLachlan, PhD (2010 - 2013) Alain Rolland, PhD (2010 - 2013) Richard Snyder, PhD (2010 - 2013) Viral Gene Transfer Vectors Committee

Chair Luigi M. Naldini, MD, PhD (2010 - 2011) R. Jude Samulski, PhD (2009 - 2010) Members Mavis Agbandje-McKenna (2010 - 2013) Frederic D. Bushman, PhD (2007 - 2010) John A. Chiorini, PhD (2010 - 2013) John T. Gray, PhD (2007 - 2013) Brendan Lee, MD, PhD (2008 - 2011) Luigi M. Naldini, MD, PhD (2008 - 2011) Philip Ng, PhD (2009 - 2012) Samuel D. Rabkin, PhD (2009 - 2012) Stephen J. Russell, MD, PhD (2010 - 2013) Dmitry M. Shayakhmetov, PhD (2009 - 2012) Richard O. Snyder, PhD (2007 - 2010) David Strayer, MD, PhD (2009 - 2012)

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ASGCT Abstract Planning Committee Plenary & Clinical Sessions

Barrie Carter, PhD Carter Bio Consulting Seattle, WA Kenneth Cornetta, MD Indiana University School of Medicine Indianapolis, IN John F. Engelhardt, PhD University of Iowa College of Medicine Iowa City, IA Guangping Gao, PhD University of Massachusetts Medical School Worcester, MA Stanton L. Gerson, MD Case Western Reserve University Cleveland, OH Matthew H. Porteus, MD, PhD University of Texas Southwestern Medical School Dallas, TX John J. Rossi, PhD Beckman Research Institute City of Hope Duarte, CA

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13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

ASGCT 13th Annual Meeting Supporters (as of April 2010) The ASGCT Annual Meeting is honored to have confirmed support for the 2010 Annual Meeting from the gene and cell therapy partners listed below. Partner Level Support

Printing of the Abstract Supplement

Registration Bags Contributor Level Support

Supported in part by March of Dimes Foundation Grant No. 4-FY09-5464220

Evening Foundation Symposium & Outstanding New Investigator Award (1)

Travel Grants

Patron Level Support

Marketing Support

Conference Grant Support

Outstanding New Investigator Award (1)

Evening Foundation Symposium

Excellence in Research Award (1)

A biopharmaceutical company committed to bettering human life

Excellence in Research Awards (1)

Excellence in Research Awards (1)

Excellence in Research Awards (1)

Conference Grant Support

Outstanding New Investigator Award (1)

Evening Foundation Symposium

Travel Grant (1)

Evening Foundation Symposium

General Meeting Support

Travel Grants (2)

Travel Grants (2)

Excellence in Research Awards (2)

Travel Grants (4)

Evening Foundation Symposium

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ASGCT 13th Annual Meeting Exhibitors The American Society of Gene & Cell Therapy would like to acknowledge the following exhibitors that are exhibiting at the ASGCT 13th Annual Meeting. A complete description of the products, services, and publications offered by these exhibitors is available on page 77. Aldevron

NHLBI Gene Therapy Resource Program

American Society of Gene & Cell Therapy

NIH Office of Biotechnology Activities

Biologics Consulting Group, Inc.

NIH-RAID

BioSpherix, Ltd.

Omnia Biologics, Inc.

Caliper Life Sciences

Penn Vector Core

Center for Cellular and Molecular Therapeutics at the Children’s Hospital of Philadelphia

Philips Research

Data Unlimited International, Inc.

Polyplus-transfection Puresyn, Inc.

Elsevier – Academic Press

ReGenX Biosciences

Exemplar Genetics

Sartorius Stedim North America Inc.

Institutional Biosafety Committee Services (IBC Services) Lovelace Respiratory Research Institute Mary Ann Liebert, Inc.

Seattle's Convention and Visitors Bureau St. Jude Children’s Research Hospital Techulon, Inc.

Meridian Life Science, Inc.

Thermo Scientific Genomics

Molmed S.p.A.

TriLink BioTechnologies, Inc.

Nanosight

Vandalia Research

National Gene Vector Biorepository Nature Publishing Group

VGXI, Inc. Waisman Clinical Biomanufacturing Facility

Nature Technology Corporation

Wiley-Blackwell

Nexcelom Bioscience

Make time each day to peruse the products, services and publications offered by the exhibiting companies. Stop by the ASGCT Booth to pick up your abstract disc and education sessions disc. Network and meet new colleagues in a relaxed atmosphere. Stop by the Job Exchange Area.

Thank you 13th Annual Meeting Exhibitors!

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13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Reviewers Committee Chair John F. Engelhardt, PhD RNA Virus Vectors

Coordinating Reviewer Arthur W. Nienhuis, MD Reviewers Christopher Baum, MD Fabio Candotti, MD Robert G. Hawley, PhD David W. Russell, MD, PhD AAV Vectors

Coordinating Reviewer John A. Chiorini, PhD Reviewers Douglas M. McCarty, PhD David V. Schaffer, PhD Matthew D. Weitzman, PhD Xiao Xiao, PhD Adenovirus and Other DNA Virus Vectors

Coordinating Reviewer Philip Ng, PhD Reviewers Anja Ehrhardt, PhD Joseph C. Glorioso, III, PhD Florian Kreppel, PhD Robin J. Parks, PhD

DNA Vectorology & Gene Targeting

Coordinating Reviewer Michele P. Calos, PhD Reviewers Deborah Gill, PhD Perry B. Hackett, PhD Jakob Giehm Mikkelsen, PhD Matthew H. Porteus, MD, PhD

Physical Methods of Delivery

Coordinating Reviewer Nancy Smyth Templeton, PhD Reviewers Michael A. Barry, PhD Jeffrey Hughes, PharmD, PhD Christian Plank, PhD Chun Wang, PhD

Chemical and Molecular Conjugates

Infectious Diseases and Vaccines

Genetic and MeTABolic Diseases Gene & Cell Therapy

Cancer - Immunotherapy

Coordinating Reviewer Cary Harding, MD Reviewers Dwight Koeberl, MD, PhD Brendan Lee, MD, PhD Jon A. Wolff, MD

Coordinating Reviewer Helen E. Heslop, MD Reviewers Ronald Alvarez, MD Jeffrey J. Molldrem, MD James J. Mulé, PhD David B. Weiner, PhD

Cardiovascular Gene & Cell Therapy

Cancer – Oncolytic Viruses

Coordinating Reviewer Suzie H. Pun, PhD Reviewers Hideyoshi Harashima, PhD Theresa M. Reineke, PhD Tatiana Segura, PhD Millicent Sullivan, PhD

Coordinating Reviewer Barry J. Byrne, MD, PhD Reviewers David A. Dichek, MD Roger J. Hajjar, MD Cathryn S. Mah, PhD Christina A. Pacak, PhD

Neurologic & Ophthalmic Gene & Cell Therapy

Coordinating Reviewer Michael Kaplitt, MD, PhD Reviewers Krystof Bankiewicz, MD, PhD Nicholas Boulis, MD Miguel Sena-Esteves, PhD Deborah J. Watson, PhD

Musculo-skeletal Gene & Cell Therapy

Coordinating Reviewer Alan R. Davis, PhD Reviewers Jeffrey Chamberlain, PhD Paula Clemens, MD Scott Q. Harper, PhD Terri A. Zachos, DVM, PhD

Coordinating Reviewer John A. Zaia, MD Reviewers Ann M. Leen, PhD Bruce E. Torbett, PhD, MSPH Grant D. Trobridge, PhD Dorothee von Laer, MD

Coordinating Reviewer Ronald Rodriguez, MD, PhD Reviewers Winald Gerritsen, MD, PhD Chinghai Kao, PhD David H. Kirn, MD Masato Yamamoto, MD, PhD

Cancer - Targeted Gene & Cell Therapy

Coordinating Reviewer William S. M. Wold, PhD Reviewers Karen S. Aboody, MD Frank Marini, PhD Stephen J. Russell, MD, PhD Richard G. Vile, PhD

Hematologic and Immunologic Gene & Cell Therapy

Coordinating Reviewer Michel Sadelain, MD, PhD Reviewers Gay Crooks, MD David W. Emery, PhD David Klatzmann, MD, PhD Adrian J. Thrasher, MD, PhD

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Abstract Reviewers Lung and Respiratory Disease Gene & Cell Therapy

Coordinating Reviewer Terence R. Flotte, MD Reviewers Dongsheng Duan, PhD Larry G. Johnson, MD Maria Limberis, PhD Patrick L. Sinn, PhD

Immunologic & Host Responses in Gene & Cell Therapy

Coordinating Reviewer Pedro Lowenstein, MD, PhD Reviewers Renier J. Brentjens, MD, PhD Evanthia Galanis, MD, DSc Maciej Lesniak, MD, FACS Lily Wu, MD, PhD

Gene Regulation

Stem Cell Therapies

Oligonucleotide & RNAi Therapeutics

Cell Processing and Vector Production

Coordinating Reviewer Olivier Danos, PhD Reviewers Michael N. Antoniou, PhD Giuliana Ferrari, PhD Dirk M. Nettelbeck, PhD Paul D. Robbins, PhD Coordinating Reviewer Peter Glazer, MD, PhD Reviewers Alan Gewirtz, MD Paloma H. Giangrande, PhD Dieter C. Gruenert, PhD Mark A. Kay, MD, PhD

Coordinating Reviewer Donald B. Kohn, MD Reviewers Emanuela M. Bruscia, PhD R. Keith Humphries, MD, PhD Carolyn Lutzko, PhD Alice F. Tarantal, PhD

Coordinating Reviewer Isabelle Rivière, PhD Reviewers Adrian P. Gee, PhD Stephen M. Kaminsky, PhD Bruce L. Levine, PhD J. Fraser Wright, PhD

Special thanks to the abstract reviewers for their time and expertise.

EXHIBITOR PROSPECTUS final program

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Excellence in Research Awards – For Students and Postdoctoral Fellows Jennifer E. Adair Fred Hutchinson Cancer Research Center Efficient Engraftment of MGMTP140K Gene-Modified CD34+ Cells Following Nonmyeloablative BCNU Conditioning in Patients with Glioblastoma

Eirini P. Papapetrou Memorial Sloan-Kettering Cancer Center Therapeutic Transgene Expression in Patient-Specific iPS Cells after Selection of “Safe Harbors” for Lentiviral Vector Integration

Melissa A. Badding University of Rochester School of Medicine Transcription Factor Binding by Plasmid DNA Is Needed for Interaction with Microtubules and Trafficking in Cells

Marco Ranzani San Raffaele Telethon Istitute for Gene Therapy Sensitive In Vivo Models To Assess the Risk of Insertional Mutagenesis in the Liver upon Vector Systemic Delivery

Richard Gabriel German Cancer Research Center Determining the Specificity of Zinc Finger Nucleases by Analyzing Insertion Site Distribution of Integrase Defective Lentiviral Vectors (IDLV) Trapped into DNA Double Strand Breaks

Axel Schambach Hannover Medical School A Novel Lentiviral Expression System Reveals Patchwork Colony Formation during Reprogramming to Induced Pluripotent Stem Cells

Travel Awards – For Students and Postdoctoral Fellows Luca Biasco Troy Brady Philippe Campeau Alessio Cantore Marcela Cataldi Daniela Cesana Maya Desai Biao Dong Masayuki Endo Darin Falk Christian Fernandez Jonathan Finn Pietro Genovese Bernhard Gentner

Lia Gracey Bo He Nathalia Holt Xuan Jiang Adam Johnson Kenya Kamimura Claudia Kettlun Hiromi Kinoh Matthias Kron Hojun Li Chin-Yu Lin Leszek Lisowski Angelo Lombardo Shannon Macauley-Rambach

Casey Maguire Claudio Mussolino Shruthi Naik Anton Neschadim Ali Nowrouzi Guy Odom Anna Paruzynski Elena Provasi Ferdinando Pucci Sylvie Rival-Gervier Dyana Saenz Erica Schleifman Sergey Seregin Laura Smith

Masataka Suzuki JaBaris Swain Tayaramma Thatava Kristina Thiel Ilaria Visigalli Yuhua Wang Korashon Watts Daniel Wolf Phonphimon Wongthida Jun Xie Jane Zhang Jiehua Zhou

The Excellence in Research Awards and Travel Grants were made possible through grant support from:

A biopharmaceutical company committed to bettering human life

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Outstanding New Investigator & Outstanding Achievement Award Recipients Outstanding new investigator award winners The following Outstanding New Investigator Award winners were selected by the Advisory Council based on merit and their contributions to the field of gene and cell therapy. The recipients will present their work at the special Outstanding New Investigator Symposium on Thursday, May 20 at 4:30 pm in the Marriott Ballroom. Alessandra Biffi, MD San Raffaele Telethon Institute for Gene Therapy (HSR - TIGET) Milano, Italy Rendering Hematopoietic Stem Cell Transplantation Efficacious for the Treatment of Lysosomal Disorders Kevin V. Morris, PhD Scripps Research Institute La Jolla, California Utilizing the Endogenous Long Non-Coding RNA Pathway in Human Cells to Transcriptionally Modulate Gene Expression

Bakhos A. Tannous, PhD Massachusetts General Hospital Charlestown, Massachusetts Ex-Vivo Monitoring of In Vivo Gene and Cell Therapy Charles P. Venditti, MD, PhD National Human Genome Research Institute Bethesda, Maryland Gene Therapy for Methylmalonic Acidemia (MMA)

The Outstanding New Investigator Awards were made possible through grant support from:

Outstanding Achievement Award winner The Outstanding Achievement Award winner was selected by the ASGCT Advisory Council as an example of an ASGCT member who has achieved a pioneering research success through a lifetime of significant scientific contributions to the field of gene and cell therapy. The Outstanding Achievement Award winner will present her work during the Presidential Symposium on Friday, May 21 at 4:30 pm in the Marriott Ballroom.

Katherine A. High, MD The Children’s Hospital of Philadelphia Philadelphia, Pennsylvania

EXHIBITOR PROSPECTUS final program

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Faculty List Alessandro Aiuti, MD, PhD Fondazione Centro San Raffaele Del Monte Tabor Milano, Italy Daniel G. Anderson, PhD Massachusetts Institute of Technology Cambridge, MA Valder Arruda, MD, PhD University of Penn School of Medicine Philadelphia, PA Michael A. Barry, PhD Mayo Clinic Rochester, MN Christopher Baum, MD Hannover Medical School Hannover, Germany C. Frank Bennett, PhD ISIS Pharmaceuticals Carlsbad, CA Jean Bennett, MD, PhD University of Pennsylvania Scheie Eye Institute Philadelphia, PA Jeffrey Bergelson, MD The Children’s Hospital of Philadelphia Philadelphia, PA Roger H. Bertolotti, PhD CNRS/Gene Therapy and Regulation Research Nice, France Carmen Bertoni, PhD University of California Los Angeles Los Angeles, CA Lilia Bi, PhD Center for Biologics Evaluation and Research Rockville, MD Leslie G. Biesecker, MD Genetic Disease Research Branch Bethesda, MD Alessandra Biffi, MD San Raffaele Telethon Institute for Gene Therapy (HSR - TIGET) Milano, Italy C. Anthony Blau, MD University of Washington Seattle, WA

Robert Blelloch, MD, PhD University of California, San Francisco San Francisco, CA Chiara Bonini, MD Fondazione Centro S. Raffaele Del Monte Tabor Milan, Italy Ryan L. Boudreau, PhD University of Iowa Iowa City, IA Charles R. Bridges, MD, ScD University of Pennsylvania Medical Center Philadelphia, PA Kate E. Broderick, PhD Inovio Biomedical Corporation San Diego, CA Brian D. Brown, PhD Mt. Sinai School of Medicine New York, NY Hal E. Broxmeyer, PhD Indiana University School of Medicine Indianapolis, IN Bruce A. Bunnell, PhD Tulane University New Orleans, LA Frederic D. Bushman, PhD University of Pennsylvania School of Medicine Philadelphia, PA Barry J. Byrne, MD, PhD University of Florida School of Medicine Gainesville, FL Peter A. Campochiaro, MD Johns Hopkins Hospital School of Medicine Baltimore, MD Michael Cancro, PhD University of Pennsylvania Philadelphia, PA Barrie J. Carter, PhD Carter BioConsulting Seattle, WA Nathalie Cartier-Lacave, MD INSERM Paris, France

Maria G. Castro, PhD Gene Therapeutics Research Institute Los Angeles, CA Roberto Cattaneo, PhD Mayo Clinic Rochester, MN Joy Cavagnaro, PhD, DABT Access BIO Boyce, VA Jeffrey Chamberlain, PhD University of Washington School of Medicine Seattle, WA Joel R. Chamberlain, PhD University of Washington School of Medicine Seattle, WA Al Charest, PhD Tufts Medical Center Boston, MA Si-Yi Chen, MD, PhD University of Southern California Los Angeles, CA Linzhao Cheng, PhD Johns Hopkins University School of Medicine Baltimore, MD Paula Clemens, MD University of Pittsburgh Pittsburgh, PA Laurence JN Cooper, MD, PhD University of Texas M.D. Anderson Cancer Center Houston, TX Kenneth Cornetta, MD Indiana University Indianapolis, IN Jacqueline Corrigan-Curay, MD, JD National Institutes of Health Bethesda, MD Timothy P. Cripe, MD, PhD Cincinnati Children’s Hospital Medical Center Cincinnati, OH Joseph DeSimone, PhD University of North Carolina Chapel Hill, NC

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Faculty List Mary C. Dinauer, MD, PhD Indiana University School of Medicine Indianapolis, IN J. Kevin Donahue, MD Case Western Reserve University School of Medicine Cleveland, OH Gianpietro Dotti, MD Baylor College of Medicine Houston, TX Steven F. Dowdy, PhD University of California San Diego La Jolla, CA Boro Dropulic, PhD Lentigen Corporation Gaithersburg, MD Gary C. du Moulin, PhD, MPH Genzyme Cambridge, MA Dongsheng Duan, PhD University of Missouri School of Medicine Columbia, MO James Ellis, PhD Hospital for Sick Children Toronto, Canada John F. Engelhardt, PhD University of Iowa College of Medicine Iowa City, IA Lynn W. Enquist, PhD Princeton University Princeton, NJ Mitchell Finer, PhD Novocell Inc San Diego, CA John G. Flannery, PhD University of California Berkeley Berkeley, CA William A. Gahl, MD, PhD National Human Genome Research Institute Bethesda, MD G. Ian Gallicano, PhD Georgetown Lombardi Comprehensive Cancer Center Washington, DC

EXHIBITOR PROSPECTUS final program

Bernhard Rudolf Gentner, MD San Raffaele Telethon Institute for Gene Therapy (HSR - TIGET) Milano, Italy Steven C. Ghivizzani, PhD University of Florida Gainesville, FL Peter M. Glazer, MD, PhD Yale University School of Medicine New Haven, CT William F. Goins, PhD University of Pittsburgh School of Medicine Pittsburgh, PA Stephen Gottschalk, MD Baylor College of Medicine Houston, TX Eric D. Green, MD, PhD National Human Genome Research Institute Bethesda, MD Philip D. Gregory, PhD Sangamo BioSciences, Inc. Richmond, CA Manuel Grez, PhD Georg-Speyer-Haus Frankfurt, Germany Markus Grompe, MD Oregon Health & Science University Portland, OR Dieter C. Gruenert, PhD California Pacific Medical Center Research Institute San Francisco, CA Roger J. Hajjar, MD The Zena and Michael A. Wiener Cardiovascular Institute New York, NY Bill Hauswirth, PhD University of Florida Gainesville, FL Roland W. Herzog, PhD University of Florida Gainesville, FL Helen E. Heslop, MD Baylor College of Medicine Houston, TX

Katherine A. High, MD The Children’s Hospital of Philadelphia Philadelphia, PA Matthew Hirsch, PhD University of North Carolina at Chapel Hill Chapel Hill, NC Edwin M. Horwitz, MD, PhD The Children’s Hospital of Philadelphia Philadelphia, PA Leaf Huang, PhD University of North Carolina at Chapel Hill Chapel Hill, NC Deborah A. Hursh, PhD Food and Drug Administration Rockville, MD Insoo Hyun, PhD Case Western Reserve University School of Medicine Cleveland, OH Pamela Itkin-Ansari, PhD University of California, San Diego La Jolla, CA Peter Johnston, MD, FACC Johns Hopkins University Baltimore, MD Douglas J. Jolly, PhD Tocagen, Inc. San Diego, CA J. Keith Joung, MD, PhD Massachusetts General Hospital Charlestown, MA Carl H. June, MD University of Pennsylvania Merion Station, PA Tal Kafri, PhD University of North Carolina at Chapel Hill Chapel Hill, NC Brian K. Kaspar, PhD The Research Institute at Nationwide Children’s Hospital Columbus, OH Petra Kaufman, MD NINDS/NIH Rockville, MD

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Faculty List Mark A. Kay, MD, PhD Stanford University School of Medicine Stanford, CA Jonathan Kimmelman, PhD McGill University Montreal, Canada Christoph Klein, MD Hannover Medical School Hannover, Germany Anna M. Krichevsky, PhD Brigham & Women’s Hospital Boston, MA Sean E. Lawler, PhD Ohio State University Columbus, OH Jane S. Lebkowski, PhD Geron Corp Portola Valley, CA Peter I. Lelkes, PhD Drexel University Philadelphia, PA Alfred Lewin, PhD University of Florida Gainesville, FL Shulin Li, PhD Louisiana State University School of Veterinary Medicine Baton Rouge, LA William W. Li, MD The Angiogenesis Foundation Cambridge, MA Christian L. Lorson, PhD University of Missouri Columbia, MO Douglas W. Losordo, MD Northwestern University Feinberg School of Medicine Chicago, IL Michael T. Lotze, MD McGowan Institute for Regenerative Medicine Pittsburgh, PA

Carolyn Lutzko, PhD Childrens Hospital Los Angeles Los Angeles, CA David M. Lynn, PhD University of Wisconsin Madison Madison, WI Ian MacLachlan, PhD Tekmira Pharmaceuticals Corp. Burnaby, Canada Harry L. Malech, MD NIH/NIAID Laboratory of Host Defenses Bethesda, MD Fulvio Mavilio, PhD San Raffaele Scientific Institute Milan, Italy Paul B. McCray, Jr., MD University of Iowa College of Medicine Iowa City, IA Ronald D.G. McKay, PhD National Institute of Neurological Disorders and Stroke Bethesda, MD Joshua Mendell, MD, PhD Johns Hopkins University Baltimore, MD Ko Mitani, PhD Saitama Medical University Hidaka, Japan Jonathan D. Moreno, PhD University of Pennsylvania School of Medicine Philadelphia, PA Richard A. Morgan, PhD National Cancer Institute Columbia, MD Ryuichi Morishita, MD, PhD Osaka University Graduate School of Medicine Suita, Japan Bernard Moss, MD, PhD National Institute of Allergy and Infectious Diseases Bethesda, MD Patrick Most, MD Jefferson Medical College Philadelphia, PA

M. Maral Mouradian, MD UMDNJ - Robert Wood Johnson Medical School New Brunswick, NJ Luigi M. Naldini, MD, PhD University Vita-Salute San Raffaele Milano, Italy Yasutomo Nasu, MD, PhD Okayama University Hospital Okayama, Japan Sattva S. Neelapu, MD The University of Texas M.D. Anderson Cancer Center Houston, TX John J. Nemunaitis, MD Mary Crowley Medical Research Center Dallas, TX Alan J. Nixon, DVM, MS Cornell University Ithaca, NY Roger J. Packer, MD Children’s National Medical Center Washington, DC Dao Pan, PhD Cincinnati Children’s Medical Center Cincinnati, OH Frederic Paques, PhD Cellectis SA Romainville, France Marco Passini, PhD Genzyme Corporation Framingham, MA Madhusudan V. Peshwa, PhD MaxCyte Rockville, MD Christian Plank, PhD Technical University Munich Munich, Germany Matthew H. Porteus, MD, PhD UT Southwestern Medical School Dallas, TX Samuel D. Rabkin, PhD Massachusetts General Hospital Boston, MA

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Faculty List John Rasko, MBBS, PhD Royal Prince Alfred Hospital, Sydney Sydney, Australia Steven A. Rosenberg, MD, PhD National Cancer Institute Bethesda, MD John J. Rossi, PhD Beckman Research Institute City of Hope Duarte, CA Blair Roszell, MS University of Connecticut Health Center Farmington, CT Carl Bart Rountree, MD Penn State College of Medicine Milton S. Hershey Medical Center Hershey, PA David W. Russell, MD, PhD University of Washington Seattle, WA Michel Sadelain, MD, PhD Memorial Sloan-Kettering Cancer Center New York, NY Aliasger K. Salem, PhD University of Iowa Iowa City, IA R. Jude Samulski, PhD University of North Carolina at Chapel Hill Chapel Hill, NC Andrew M. Scharenberg, MD University of Washington Seattle Children’s Research Institute Seattle, WA Jeffrey Schlom, PhD National Cancer Institute Bethesda, MD Edward M. Schwarz, PhD University of Rochester Rochester, NY Lonnie D. Shea, PhD Northwestern University Evanston, IL

EXHIBITOR PROSPECTUS final program

Robert Sims, MD Dendreon Corporation Seattle, WA Evan Y. Snyder, MD, PhD, FAAP Burnham Institute for Medical Research La Jolla, CA Declan M. Soden, PhD University College Cork, BioSciences Institute Cork, Ireland Robert D. Steiner, MD Oregon Health & Science University Portland, OR David F. Stroncek, MD NIH Clinical Center Bethesda, MD Jeremy Sugarman, MD, MPH, MA Berman Institute of Bioethics and Department of Medicine Baltimore, MD Bruce Sullenger, PhD Duke University Medical Center Durham, NC Daniel M. Takefman, PhD Food and Drug Administration Rockville, MD Adrian J. Thrasher, MD, PhD Institute of Child Health London, United Kingdom Thomas Tillett TillettConsulting, LLC Ambler, PA David L. Urdal, PhD Dendreon Corporation Seattle, WA Sander van Deventer, MD, PhD Amsterdam Molecular Therapeutics Haarlem, Netherlands Luk H. Vandenberghe, PhD University of Pennsylvania Philadelphia, PA Charles P. Venditti, MD, PhD National Human Genome Research Institute Bethesda, MD

Inder M. Verma, PhD Salk Institute La Jolla, CA Christof von Kalle, MD PhD NCT Heidelberg Heidelberg, Germany Ernst Wagner, PhD University of Munich Munich, Germany David B. Weiner, PhD University of Pennsylvania Philadelphia, PA Daniel Weiss, MD, PhD University of Vermont College of Medicine Burlington, VT David A. Williams, MD Children’s Hospital Boston Boston, MA Michelle Williams, PhD Osiris Therapeutics Columbia, MD Yiping Yang, MD, PhD Duke University Durham, NC Seppo Ylä-Herttuala, MD, PhD, FESC University of Kuopio Kuopio, Finland Mervin C. Yoder, MD Cancer Research Institute Indianapolis, IN De-Chao Michael Yu, PhD State Key Laboratory of Biotherapy West China Hospital Sichuan University Chengdu Kanghong Biotech, Inc. Chengdu, Peoples Republic of China Chae-Ok Yun, PhD Yonsei University College of Medicine Seoul, Korea Elias T. Zambidis, MD, PhD Johns Hopkins University Baltimore, MD Ming Zhan, PhD National Institute on Aging Baltimore, MD

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

General Meeting Information AMERICAN SOCIETY OF GENE & CELL THERAPY Mission and Goals

The American Society of Gene & Cell Therapy (ASGCT) is a professional non-profit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. ASGCT is the largest medical professional organization representing researchers and scientists dedicated to discovering new gene and cell therapies. ASGCT was established in 1996, and has grown to 2,000 members from around the world. The Society’s goals are to:

• Promote Research, Development and Application of Genetic and Cellular Therapies to a Wide Variety of Human Diseases • Exchange Information and Promote Education Among Professionals and the Public Through the Society’s Annual Meetings, the ASGCT Journal, Molecular Therapy, and Interactions with Scientific, Medical, and Regulatory Communities The ASGCT 13th Annual Meeting will serve as a comprehensive forum to achieve these goals. Abstract Volume – Molecular Therapy

All abstracts, except Late Abstract Submissions, accepted for presentation at the ASGCT 13th Annual Meeting have been published in the May supplement of Molecular Therapy. Each attendee will receive one copy of the supplement along with their registration materials. Late Abstract Submissions accepted for presentation have been printed in a booklet that will be distributed to each attendee. Admission

Official name badges will be required for admission to all ASGCT sessions. All Annual Meeting attendees receive a name badge with their registration bag. Name badges should be worn at all times inside the hotel, as badges will be used to control access to sessions and activities. Attendees are cautioned against wearing their name badges while away from the hotel as badges may draw unwanted attention to your status as visitors to Washington, DC. Admission by Ticket Only

Some sessions at the Annual Meeting will require an additional ticket to control access and capacity. Tickets for these sessions are not included in the meeting’s regular registration fees. Tickets will be collected at the door by ushers. There are still a limited number of tickets available for the Meet-the-Investigator sessions, which may be obtained at the Registration Desk. Meet-the-Investigator tickets are $30.

Business Center (FedEx Office)

There is a FedEx Office located in the Marriott Wardman Park Hotel. It is open Monday – Friday from 7:00 am to 7:00 pm and closed on Saturday and Sunday. Business Meeting

Join the Society’s leadership for a business meeting and continental breakfast. Saturday, May 22, 2010 8:00 am – 8:30 am (A light continental breakfast will be offered at 7:45 am) Room Delaware Suite All members are welcome. See page 3 for more details. Camera/Recording Policy

Due to privacy issues, with the exception of a hired professional photographer, it is the policy of ASGCT that no cameras, camera phones, or video recorders are permitted in the meeting sessions, exhibit hall, or poster sessions. Please refrain from taking any photos in those locations. Audio taping is also prohibited. Cell Phones & PDAs

For the courtesy of all presenters and attendees, please ensure that cell phone and PDA ringers are turned off or silenced during all sessions as they may interfere with session audio. Committee Meetings

Most standing and scientific committees will meet during the Annual Meeting. See page 3 for Committee Meeting information. Concierge Desk

Washington D.C. and the Woodley Park neighborhood offers a wide variety of activities and restaurants to meet all tastes and budgets. A Concierge Desk is located in the Lobby of the hotel that can help you with your city needs and reservations. Concierge Desk Hours

Sunday through Saturday

7:00 am – 7:00 pm

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General Meeting Information Continuing Medical Education Accreditation/Credit Designation

This activity has been planned and implemented in accordance with the Essential Areas and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint sponsorship of Indiana University School of Medicine and the American Society of Gene & Cell Therapy. Indiana University School of Medicine is accredited by the ACCME to provide continuing medical education for physicians. NOTE: Sessions marked with an asterisk (*) are not part of the CME program. Designation Statement

Indiana University School of Medicine designates this educational activity for a maximum of 37.5 AMA PRA Category 1 Credits™. Physicians should only claim credit commensurate with the extent of their participation in the activity. CME Certificates

Participants requiring CME certificates must go to the Registration Desk and complete a CME form. CME certificates will be mailed to participants after the meeting. There is a CME fee of $50 USD for the 13th Annual Meeting.

Educational Objectives

At the conclusion of the activity, the participant should be able to: Provide advice to patients who inquire about the potential of gene and cell therapy or the availability of open clinical trials, based on their exposure to the current clinical trials in gene and cell therapies. Better instruct their students in medical school and other health venues using the state-of-the-art basic science and clinical trials data presented at the meeting. Use the latest advances in gene and cell therapy to enhance their research mission, as physician scientists conducting basic and clinical research. Demonstrate improved regulatory compliance in conducting gene and cell therapy clinical trials, through exposure to NIH and FDA faculty during the educational program. Evaluation Method

Evaluation by questionnaire will address program content, presentation, and possible bias.

Disclosure

Needs

In accordance with the Accreditation Council for Continuing Medical Education (ACCME) Standards for Commercial Support, educational programs sponsored by Indiana University School of Medicine (IUSM) must demonstrate balance, independence, objectivity, and scientific rigor. All faculty, authors, editors, and planning committee members participating in an IUSM-sponsored activity are required to disclose any relevant financial interest or other relationship with the manufacturer(s) of any commercial product(s) and/or provider(s) of commercial services that are discussed in an educational activity.

Clinical gene transfer has become more and more complex due to ongoing developments in the fields of gene and cell therapy itself, together with bioethics, research integrity, and financial conflicts, as well as federal mandates, regulations and guidelines. Oligonucleotide Therapies, Novel Vector Development, Host-Vector Interactions and Vaccine Therapies will be discussed as well as many other scientific topics. This meeting will provide an educational forum for scientists and clinicians to expand their knowledge about the broad developments in these fields.

Note

Target Audience

While it offers CME credits, this activity is not intended to provide extensive training or certification in the field. Educational Methods and Materials

Lectures, Case Presentations, Panel Discussions, Question and Answer Sessions, Audio/Video Presentations, Abstracts, Posters

EXHIBITOR PROSPECTUS final program

The target audience includes basic science and translational researchers, clinical investigators, physicians, postdoctoral fellows, graduate students, employees of federal government and regulatory agencies, and other healthcare professionals with an interest in the latest advancements in the field of gene and cell therapy.

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

General Meeting Information Exhibits

Exhibits will be located in Exhibit Hall C, of the Marriott Wardman Park Hotel. The companies, organizations and institutions exhibiting at the ASGCT Annual Meeting will be providing you with the latest information on products and services directly related to your professional needs, as well as recruiting for open positions. An Exhibit Hall Social will be held on Wednesday evening and a coffee social will be offered on Thursday in the exhibit hall. All participants are urged to allow adequate time in your daily schedule to visit the exhibits, as they are an integral part of the success of the meeting. Exhibit Hall Hours

Exhibits will be open Wednesday, May 19 through Friday, May 21, 2010. Wednesday, May 19 Thursday, May 20 Thursday, May 20 Friday, May 21

7:00 pm – 9:00 pm 9:15 am – 10:30 am 6:15 pm – 8:15 pm 6:30 pm – 8:30 pm

Guest Attendance

ASGCT asks registered attendees to refrain from taking children, spouses, or guests to any educational session or functions offered at the 13th Annual Meeting. Internet Access & Policy

Internet access will be available at the Marriott Wardman Park Hotel to all meeting attendees. Limited computer stations will be provided. When others are waiting at the internet kiosks, please limit your Internet use to 15 minutes at a time so other attendees can access the internet as well. If you have a laptop, the Marriott Wardman Park offers wireless internet access at certain locations within the facility. For guests staying at the Marriott Wardman Park in the ASGCT room block, free internet access is available in their hotel rooms as well as the hotel’s lobby.

Job Bank

All openings posted as of April 23, 2010 on the ASGCT Job Bank have been included in the Jobs Available Booklet found in the registration bags. Contact information is included in the booklet. Job postings for open positions and résumés may be posted on the Job Posting boards in Exhibit Hall C. You are responsible for providing multiple copies of your posting. A number of tables and chairs will also be available in the job exchange center for interview use. Lead Retrieval

A lead retrieval system has been made available to all exhibitors of the ASGCT 13th Annual Meeting. Exhibitors may ask to scan attendee name badges with a hand held scanner in order to obtain attendee contact information. Materials Distribution

Please refrain from distributing promotional materials throughout the meeting; these may only be distributed from an exhibit booth in Exhibit Hall C. Media

Members of the working media may register for the 13th Annual Meeting in the Press Room – Buchanan Room. Interview space, telephones, and internet access are available for the convenience of media representatives covering the meeting. Press must register, provide credentials, and wear their press badge for admittance to ASGCT sessions. Assistance will be provided to members of the media that would like to schedule interviews. No Smoking

Smoking is prohibited at all 13th Annual Meeting sessions and events. The Marriott Wardman Park is a non-smoking hotel.

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General Meeting Information Posters

Abstract Posters will be on display in Halls B-South and C. Thursday, May 20 Abstract Poster Session I

10:00 am – 12:00 pm

Poster Setup by Authors

12:30 pm – 8:15 pm

Poster Viewing (authors present from 6:15 pm – 8:15 pm)

6:15 pm – 8:15 pm

Exhibit Hall Open

6:15 pm – 8:15 pm

Welcome Reception, Exhibits, and Poster Session

8:15 pm – 8:45 pm

Authors Remove Posters

Registration Desk

Name badges, final programs, and abstract supplements will be distributed at the registration desk located outside of Exhibit Hall C in the atrium, exhibition level of the Marriott Wardman Park Hotel. Registration Desk Hours

Tuesday, May 18 5:00 pm – 8:00 pm Wednesday, May 19 7:00 am – 8:00 pm Thursday, May 20 7:00 am – 5:00 pm Friday, May 21 7:30 am – 5:00 pm Saturday, May 22 7:30 am – 5:00 pm Shuttle Bus

ASGCT will not be providing shuttle transportation for the 13th Annual Meeting. Speaker Ready Room

Friday, May 21 Abstract Poster Session II

7:00 am – 12:00 pm

Poster Setup by Authors

12:00 pm – 8:30 pm

Poster Viewing (authors present from 6:30 pm – 8:30 pm)

6:30 pm – 8:30 pm

Exhibit Hall Open

6:30 pm – 8:30 pm

Networking Reception, Exhibits, and Poster Session

8:30 pm – 9:00 pm

Authors Remove Posters

Saturday, May 22 Abstract Poster Session III

7:00 am – 12:00 pm

Poster Setup by Authors

12:00 pm – 8:00 pm

Poster Viewing (authors present from 6:00 pm – 8:00 pm)

5:00 pm – 6:00 pm

Poster Hall Social in Atrium

6:00 pm – 8:00 pm

Poster Session

8:00 pm – 8:30 pm

Authors Remove Posters

Press Room

The Press Room is located in the Marriott Wardman Park Hotel, Buchanan Room. Press Room Hours

Wednesday, May 19 3:00 pm – 6:00 pm Thursday, May 20 8:00 am – 5:00 pm Friday, May 21 8:00 am – 5:00 pm Saturday, May 22 8:00 am – 5:00 pm

EXHIBITOR PROSPECTUS final program

The Speaker Ready Room is located behind the registration desk located outside of Exhibit Hall C in the atrium of the Marriott Wardman Park Hotel. All faculty must deliver their presentations to the Speaker Ready Room the day before their session or at least four hours prior to their presentations. Equipment is available for faculty to review their materials. Audiovisual personnel will be available for assistance. Please mark your materials (your name, session and speaker order) so the materials can be returned to you. The Society strongly encourages faculty to pre-load presentations in the Speaker Ready Room; those faculty who load presentations in the meeting rooms during the sessions will have that time deducted from their presentation time by the Chair. ASGCT strongly encourages faculty presenting on Wednesday of the Annual Meeting to check-in at the Speaker Ready Room on Tuesday, May 18 to avoid congestion. Avoid delays and check-in early! Speaker Ready Desk/Room Hours

Tuesday, May 18 5:00 pm – 8:00 pm Wednesday, May 19 6:30 am – 9:00 pm Thursday, May 20 6:30 am – 7:00 pm Friday, May 21 7:00 am – 7:00 pm Saturday, May 22 7:00 am – 3:00 pm Specialty Needs

If you require special accommodations in order to fully participate in the meeting, please visit the Registration Desk; ASGCT staff will be happy to assist you with your specific needs.

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Hotel Floor Plans eXHIBIT hALL

• Exhibit Hall • Internet Kosks • Job Exchange • Poster Hall • Registration • Speaker Ready Room

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Hotel Floor Plans Lobby level

8209

8210

• Plenary Lecture Hall • Session Rooms (Education, Oral Abstract, Scientific)

8211 8212

8216 8217 8218 8219

8222 8223 8224 8226

8228 8229

mezzanine level

• Committee Meetings • Press Room – Buchanan • Session Rooms

EXHIBITOR PROSPECTUS final program

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Schedule-at-a-Glance (This program is subject to updates/changes) tuesday, may 18 5:00 pm – 8:00 pm

Registration Open

Wednesday, may 19 7:00 am – 8:00 pm

Registration Open

8:30 am – 12:15 pm

ASGCT Board of Directors Meeting

8:30 am – 11:30 pm

Scientific Symposia Session I: Cell Therapy BioProcessing Symposium and International Symposium: A Global Gene Therapy Renaissance

1:00 pm – 3:00 pm

Scientific Symposia Session II

3:30 pm – 5:00 pm

Education Session I

5:30 pm – 7:00 pm

Education Session II

7:00 pm – 9:00 pm

Exhibit Hall Welcome Reception

Thursday, may 20 7:00 am– 5:00 pm

Registration Open

8:00 am– 9:15 am

George Stamatoyannopoulos Lecture – Keynote: Hal E. Broxmeyer, PhD: Understanding and Enhancing Stem/Progenitor Cell Function and Engraftment

9:15 am– 10:30 am

Exhibit Hall Coffee Social

10:30 am– 12:30 pm

Scientific Symposia Session III

12:30 pm– 1:45 pm

Meet-the-Investigator Sessions

12:30 pm– 8:15 pm

Poster Viewing (Authors present from 6:15 pm – 8:15 pm)

2:00 pm– 4:00 pm

Oral Abstract Session I

4:30 pm– 6:15 pm

Outstanding New Investigator Symposium

6:15 pm– 8:15 pm

Exhibit Hall Open & Networking Reception Poster Session I

7:00 pm– 10:00 pm

Foundation Supported Symposium: Expanding Frontiers in Spinal Muscular Atrophy Gene Therapy

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Schedule-at-a-Glance (This program is subject to updates/changes) friday, may 21 7:30 am– 5:00 pm

Registration Open

8:00 am– 10:00 am

Plenary Session: Top Oral Abstract Presentations

10:30 am– 12:30 pm

Scientific Symposia Session IV

12:30 pm– 8:30 pm

Poster Viewing (Authors present from 6:30 pm – 8:30 pm)

2:00 pm– 4:00 pm

Oral Abstract Session II

4:30 pm– 6:30 pm

Presidential Symposium – Keynote Speakers: Katherine A. High, MD: Outstanding Achievement Award Lecture Jean Bennett, MD, PhD: An Aye for Gene Therapy: Lessons from a Clinical Trial for Congenital Blindness David A. Williams, MD: New Approaches to Gene Therapy for Fanconi Anemia

6:30 pm– 8:30 pm

Exhibit Hall Open & Networking Reception Poster Session II

7:00 pm– 10:00 pm

Foundation Supported Symposium: Progress and Prospects for Gene Therapy for CGD and Other Primary Immunodeficiencies

SATURDAY, MAY 22 7:30 am– 5:00 pm

Registration Open

8:00 am– 8:30 am

ASGCT Business Meeting (with continental breakfast)

8:30 am– 10:30 am

Scientific Symposia Session V

11:00 am– 1:00 pm

Oral Abstract Sessions III

1:00 pm– 8:00 pm

Poster Viewing (Authors present from 6:00 pm – 8:00 pm)

1:00 pm– 2:15 pm

Meet-the-Investigator Sessions

1:00 pm– 2:45 pm

ASGCT Board of Directors Meeting

3:00 pm– 5:00 pm

Oral Abstract Sessions IV

5:00 pm– 6:00 pm

Poster Hall Social in Atrium

6:00 pm– 8:00 pm

Poster Session III

8:00 pm

13th Annual Meeting Concludes

EXHIBITOR PROSPECTUS final program

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

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Program Schedule

Scientific Symposium 100

8:30 am - 11:30 am Room: Maryland Suite

Cell Therapy BioProcessing Symposium Co-Chairs Boro Dropulic, PhD & Thomas Tillett Speakers David L. Urdal, PhD

Manufacturing of Sipuleucel-T for the Treatment of Men with Metastatic, Castrate Resistant Prostate Cancer Sipuleucel-T is an autologous active cellular immunotherapy for men with late stage prostate cancer. It is comprised of a patient’s peripheral blood mononuclear cells, including antigen presenting cells that have been cultured with a recombinant antigen delivery cassette composed of prostatic acid phosphatase and granulocyte-macrophage colony stimulating factor. The development of sipuleucel-T will be discussed and the manufacture of this novel product candidate will be described. Gary C. du Moulin, PhD, MPH Incorporating the Concepts of Modern Quality Systems into Cell-based Product Development Well understood manufacturing processes that identify critical sources of variability can lower risk and lead to safe and efficacious treatment modalities that consistently deliver the intended therapeutic performance. The evolution of pharmaceutical quality systems and its focus on patient safety brings the application of a scientific approach and quality risk assessment into practice. This presentation introduces developers to the principles of risk management and quality by design, concepts that can assist in insuring that quality attributes are identified early in the product design phase and incorporated into the control of critical processes for cell and gene therapy products. Jane S. Lebkowski, PhD

Development of Human Embryonic Stem Cells for Therapeutic Applications HESC-based regenerative cell therapies require 1) evidence for reliable production and quality control of product manufacturing, 2) rigorous safety testing in preclinical models, and 3) the design of clinical trial protocols that assess the safety and benefit of the therapy in appropriate patient populations. GRNOPC1 is a population of allogeneic cells containing oligodendrocyte progenitors derived from characterized, dedicated, human embryonic stem cell banks. GRNOPC1 induces myelination of axons in rats with spinal cord injuries and in Shiverer mice, which lack compact myelin, and also produces numerous neurotrophic factors such as midkine, BDNF, and activin. Extensive preclinical studies were performed to determine the distribution of GRNOPC1 as well as any potential toxicities after injection near the thoracic injury epicenter. Based on this IND application, the FDA has granted clearance for Geron to initiate a human clinical trial to assess the safety of GRNOPC1 in patients with subacute, complete ASIA A, thoracic injuries whose last fully preserved neurological level is T3 to T10. Madhusudan V. Peshwa, PhD

Use of Flow Electroporation to Load Human Cells to Deliver Clinically Relevant Genes for Cell Therapeutics The ability to specifically enhance/augment biological function of cells using safe, non-gene modifying technologies with ability to deliver the product at clinical/commercial scale using automated, regulatory-compliant, cost-effective processes overcomes the key hurdle in developing “effective” cellular therapies.  This presentation provides an overview of our experiences in development and characterization of cellular products using immune cells and stem cells that have been engineered to deliver clinically relevant potency/biological activity.  Specific case studies discussed will include clinical/commercial delivery of a (1) a patient-specific cellular vaccine marketed as an Oncology therapy in Japan and (2) an engineered endothelial stem cell product for treatment of pulmonary arterial hypertension (PAH) in phase I/II trials in Canada.  Mitchell Finer, PhD Challenges in the Manufacturing Scale-Up of hES Cell Derived Therapeutics Human Embryonic Stem (hES) Cells have been traditionally derived and passaged as adherent clumps of cells on mouse or human fibroblast feeder layers. In order to produce the quantities of cells sufficient for preclinical development and human clinical trials at an acceptable quality standard, methodology must be developed to enable xeno product free/serum free culture of hES cells as single cells in the absence of feeder lines. Further, while growth on plastic can support limited preclinical and clinical development in some indications, achieving the scale of hES cell amplification necessary to treat diseases such as Type 1 Diabetes that require significant cell numbers (~100 billion differentiated cells), the ability to expand ES cells in suspension in the absence of differentiation, and the ability to synchronously differentiate large numbers of cells is required for product development in Type I Diabetes. Data describing the generation of feeder-free cGMP derived hES cell banks, expansion of large scale hES cell cultures in the undifferentiated state and scaled differentiation in suspension of hES-cells into pancreatic b-cell progenitors will be presented.

Wednesday, May 19th

Wednesday, May 19

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Wednesday, May 19th

Program Schedule Michelle Williams, PhD Development of Prochymal, an Adult MSC Therapy Osiris has been developing stem cell therapies to treat unaddressed medical needs since 1992.  An important part of this process has been navigating the regulatory pathway, particularly through work in the lead indication, graft-versus-host-disease.  In 2005, Osiris secured the first ever Fast Track and Orphan Drug designations from FDA for a stem cell product, followed by Orphan drug designation in Europe as well.  In 2008, FDA and Health Canada granted approval for a pediatric expanded access program, providing all children with severe GvHD access to our stem cell drug, Prochymal.  The late stage clinical development process is an instructive case study of development of a novel therapy for a orphan indication. Scientific Symposium 101

8:30 am - 11:30 am Room: Marriott Ballroom Salon 1

A Global Gene Therapy Renaissance Chair John Rasko, MBBS, PhD Speakers De-Chao Michael Yu, PhD

Clinical Development of KH901, a Conditionally Replicating Oncolytic Adenovirus Encoding Human GM-CSF, in Patients with Recurrent Head and Neck Cancers KH901 is a conditionally replicating oncolytic adenovirus, in which the viral E1 genes are under the control of a modified telomerase promoter and a cDNA gene of granulocyte macrophage colony-stimulating factor (GM-CSF) is inserted in the E3 region. Preclinical studies have demonstrated that KH901 selectively replicates in telomeraseupregulated and E2F-positive tumor cells and has strong anti-tumor activity. A total of 110 patients with recurrent head and neck cancer have been enrolled into clinical trials in which KH901 was given intratumorally while chemotherapy was the standard of care. KH901 was found to be well tolerated and safe while a significant immune responses and pro-longed progression-free survival were observed when compared to the chemotherapy group. Christoph Klein, MD Gene Therapy for Wiskott-Aldrich Syndrome Wiskott-Aldrich syndrome (WAS) is a primary immunodeficiency disorder associated with thrombocytopenia, eczema and autoimmunity. We treated ten WAS patients by transfusion of autologous, genetically modified hematopoietic stem cells (HSC) between 2006 and 2009. We demonstrate sustained WAS protein (WASP) expression in HSC, lymphoid and myeloid cells, and platelets after gene therapy. T and B lymphocytes, natural killer (NK) cells, and monocytes were functionally corrected. The patients´ clinical condition markedly improved with respect to hemorrhagic diathesis, eczema, autoimmunity, and predisposition to severe infections. Genome-wide insertion site analysis demonstrated vector integration that targeted multiple genes controlling growth development and immunological responses in a persistently polyclonal hematopoiesis. Yasutomo Nasu, MD, PhD Prostate Cancer Gene Therapy in Japan - from HSV-tk to REIC/Dkk-3 Intraprostatic therapeutic gene transduction (in situ gene therapy) is one of the potent therapeutic option for prostate cancer gene therapy aiming at antimetastatic benefits through the generation of immune cell-mediated cytotoxic activities that affect not only the primary tumor but also metastatic lesion. Many clinical trials with Herpes Simplex Virus Thymidine Kinase (HSV-tk) gene therapy have been conducted in many institutes showing clinical safety and efficacy including Japanese trial (Molecular Therapy 15:834, 2007). Also immunomodulatory in situ gene therapy using adenoviral Interleukin-12 gene is under way in Japan. As a preclinical study, new therapeutic gene REIC/Dkk3 has been proved to be potential therapeutic gene for prostate cancer as it has induces local apoptosis and systemic immune activation. (Cancer Research 65:9617, 2005, Cancer Gene Therapy 14:765,2007, Int J Oncol 34:657,2009, J Bio Chem 2009 online). In this symposium ongoing development of in situ gene therapy for prostate cancer will be reported with regard to systemic immune activation.

EXHIBITOR PROSPECTUS final program

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Program Schedule

Seppo Ylä-Herttuala, MD, PhD, FESC AdVEGF-D Local Vascular Gene Transfer Program - To Reduce Graft Stenosis in Dialysis Patients Oncolytic virotherapy is the use of genetically engineered viruses that specifically target and destroy tumor cells via their cytolytic replication cycle. An important issue in replicating virus-based cancer gene therapy that has received little attention is the limited distribution such viruses have been able to achieve in solid tumors. With the aim of improving viral distribution and tumor penetration for cancer gene therapy, we have developed a novel relaxin-expressing oncolytic adenovirus, DWP418. We carried out a phase I study of DWP418 in patients with recurrent solid tumors. The preliminary results show that intratumoral administration of DWP418 is feasible, well tolerated, and associated with biological activity. Alessandro Aiuti, MD, PhD Gene Therapy for Primary Immunodeficiencies Gene therapy with hematopoietic stem cells is an attractive therapeutic strategy for several forms of primary immunodeficiencies. Results of the ADA-SCID and SCID-X1 trials have shown long-term restoration of immune competence and clinical benefit. The use of a reduced-dose conditioning was crucial to allow substantial engraftment of multipotent gene-corrected hematopoietic stem cells. Self-inactivating lentiviral vectors have recently entered into the clinic and should provide significant advantages over gammaretroviral vectors.

Lunch Break 11:30 am – 1:00 pm Scientific Symposium 110

1:00 pm - 3:00 pm Room: Thurgood Marshall W

Gene and Cell Therapy Translational Studies Chair J. Kevin Donahue, MD Speakers J. Kevin Donahue, MD

Gene Therapy for Atrial Fibrillation Atrial fibrillation affects 2-5 million people in the US and millions more around the world. Mechanisms known to sustain the arrhythmia include structural and electrical remodeling. We developed a novel method for atrial gene transfer that paints the vector on the atrial epicardium. Using this method with adenovirus vectors and a transgene encoding a dominant negative potassium channel mutation, we show elimination of atrial fibrillation. Douglas W. Losordo, MD Autologous CD34 + Cell Therapy for Ischemic Tissue Repair Preclinical studies documented the potential therapeutic potency of endothelial progenitor cells, both as cultured and freshly isolated CD34+ cells. Phase I and II clinical trials using autologous, GCSF mobilized autologous CD34+ cells have been completed in patients with refractory angina and critical limb ischemia. These studies provide data supporting feasibility, safety and bioactivity. Importantly, the early evidence suggests the efficacy of CD34+ cells and provides important data regarding endpoints and power calculations for later phase studies. Accordingly, the goal of ischemic tissue repair appears feasible and is being approached in human clinical trials.

Wednesday, May 19th

Chae-Ok Yun, PhD A Phase I Study of Relaxin-Expressing Oncolytic Adenovirus Oncolytic virotherapy is the use of genetically engineered viruses that specifically target and destroy tumor cells via their cytolytic replication cycle. An important issue in replicating virus-based cancer gene therapy that has received little attention is the limited distribution such viruses have been able to achieve in solid tumors. With the aim of improving viral distribution and tumor penetration for cancer gene therapy, we have developed a novel relaxin-expressing oncolytic adenovirus, DWP418. We carried out a phase I study of DWP418 in patients with recurrent solid tumors. The preliminary results show that intratumoral administration of DWP418 is feasible, well tolerated, and associated with biological activity.

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Wednesday, May 19th

Program Schedule Barry J. Byrne, MD, PhD Therapeutic Strategies for Pompe Disease: Fact or Fiction Early presentation of cardiac dysfunction and the diagnosis of primary cardiomyopathy in infancy and childhood is most often associated with a genetic etiology. One example of early cardiomyopathy is Pompe disease due to striated muscle glycogen storage. In order to develop a specific treatment based gene transfer, the presentation will focus on systemic and cardiac gene transfer as an approach to disease management. Additional insight into therapeutic strategies is gained from the ongoing study of a cohort of patients who have received enzyme replacement therapy. The rational for the first clinical gene transfer trial in Pompe disease will be presented. Patrick Most, MD S100A1: A Novel Molecular Therapeutic Advance for Heart Failure Advanced understanding of the functional networks controlling cardiomyocyte performance together with the evolution of safe and efficient gene transfer technologies have provided the foundation for the development of an innovative and novel genetically-targeted HF therapy resulting from the unique molecular profile of the cardiomyocyte Ca2+ sensor protein S100A1. Here we show profound long-term therapeutic effectiveness and a favorable safety profile for AAV9-S100A1 gene therapy in a preclinical HF model employing cardiac-targeted gene delivery techniques and a gene dosage in pigs that are directly applicable to humans.

Scientific Symposium 111 1:00 pm - 3:00 pm Room: Wilson ABC

Ethical Issues with Embryonic Stem Cells and Induced Pluripotent Stem Cells Chair Jonathan Kimmelman, PhD Speakers Jonathan D. Moreno, PhD

Update on Ethical Issues in Stem Cell Research In this talk I review the history of the embryonic stem cell debate and describe the current policy situation in the US and abroad. I then characterize the significance of work on induced pluripotent stem cell (iPSC) technology for the ongoing debate. In brief, with iPSC’s the controversy is likely to shift from the origins of the cell lines in human embryos to their fate as human regenerative and even reproductive materials. Insoo Hyun, PhD Medical Innovation Outside of Clinical Trials: Proceed with Caution Recently the U.S. Food and Drug Administration inaugurated a new policy allowing expanded access to investigational interventions by clinicians attempting to treat patients outside the research context.  For those interested in creating reliable platforms for a whole new generation of medical tools through genomics, gene transfer, and stem cell research, the FDA’s new policy holds many hazards.  New models for local oversight of evidence-based medical innovation will be needed in order to avoid the uncontrolled and premature clinical exploitation of these platform technologies and still translate them successfully to the clinic. Jeremy Sugarman, MD, MPH, MA Ethical Issues in Translational Research Involving Human Pluripotent Stem Cells The clinical translation of human pluripotent stem cells may take several different paths including clinical translational research, clinical innovation, and the use of untested or unproven interventions. Each of these translational pathways raises a unique set of ethical issues. Furthermore, there are distinct standards of practice and procedural mechanisms for addressing the ethical issues inherent to each pathway. As such, it is essential that these differences be recognized to best protect not only the well-being of patients, but also of the scientific enterprise as it seeks to understand the true value of stem-cell based interventions. Panel Roger H. Bertolotti, PhD James Ellis, PhD Deborah A. Hursh, PhD

EXHIBITOR PROSPECTUS final program

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

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Program Schedule 1:00 pm - 3:00 pm Room: Marriott Ballroom Salon 1

Barriers to HSC Gene Therapy Co-Chairs Manuel Grez, PhD & Harry L. Malech, MD Speakers Mary C. Dinauer, MD, PhD

New Approaches for Marrow Conditioning in HSC Gene Therapy In many genetic blood disorders, there is no selective advantage for hematopoietic stem cells following gene correction.  Hence, in addition to expression of adequate levels of the therapeutic protein, successful use of HSC gene therapy in these disorders will require efficient transduction, homing and engraftment of transduced HSC, as well as cytoreduction of uncorrected HSC. It is highly desirable to develop effective conditioning regimens that reduce marrow content of uncorrected HSC yet minimize exposure of patients to genotoxic agents and acute and long-term multiorgan toxicities. This talk will present new approaches for marrow conditioning for transplantation in HSC gene therapy, including more selective targeting of HSC in their niche. Luigi M. Naldini, MD, PhD Exploiting and Antagonizing miRNA in Gene Therapy C. Anthony Blau, MD Pharmacologically Regulated Cell Therapy Hematopoietic stem cells (HSC) compete for the opportunity to contribute to various mature cell lineages in the blood, however little is known about the rules that govern this competition. Eight years ago, two dogs were transplanted with autologous CD34+ cells that had been transduced with an gammaretrovirus vector encoding green fluorescent protein (GFP) and a conditionally activatable derivative of the thrombopoietin receptor (F36VMpl). Responses to F36VMpl signaling, elicited through administration of a chemical inducer of dimerization (CID), were reported previously. Now long-term monitoring shows no spontaneous rises in GFP+ cells over time, suggesting that F36VMpl is inert in the absence of CID. Stably higher frequencies of GFP+ B and T cells relative to other lineages suggests that CID administration within the first 18 months post-transplantation generates stable increases in transduced lymphocytes, whereas only myeloid lineages responded when later courses of CID were administered in one animal. Clonal tracking identified functionally distinct categories of hematopoietic stem cells: those that persisted and remained CID responsive, those that persisted but were CID unresponsive, and those that did not persist. Christopher Baum, MD Approaches to Avoid Adverse Events in the Genetic Modification of Hematopoietic Cells Adverse events related to insertional mutagenesis or ectopic transgene expression may compromise the therapeutic prospects of gene therapy in the hematopoietic system. Over the past years, major progress has been made in the understanding of the underlying mechanisms, opening rational approaches to increase the therapeutic index. We have established sensitive nonclinical models, in transplanted C57BL/6J mice or using cultured cells, to reveal the transforming capacity of insertional activation of cellular proto-oncogenes such as Evi1 or Prdm16, and developed integrating gene vectors designed to reduce the risk of insertional transformation by altering the insertion pattern and the expression cassette of the transgene. In a murine model of Mpl deficiency, major adverse events related to ectopic protein expression could be controlled by transcriptionally targeted vectors. Two important principles emerge to ensure undisturbed long-term hematopoiesis from gene-modified cells. The first is the establishment of polyclonal hematopoiesis using vectors with an untargeted integration profile and “physiological” transgene cassettes. The second is the generation of clonally defined hematopoiesis derived from induced pluripotent cells with genetic modification in bona fide safe harbours.

Wednesday, May 19th

Scientific Symposium 112

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Wednesday, May 19th

Program Schedule Scientific Symposium 113

1:00 pm - 3:00 pm Room: Maryland Suite

Delivery of Oligonucleotide Therapeutics Chair Bruce Sullenger, PhD Speakers Leaf Huang, PhD

Deliver siRNA for Cancer Therapy siRNA can not fulfill its promise to become the next generation pharmaceutics unless it is efficiently delivered to the cytoplasm of the target cells. Membrane/core type nanoparticles with appropriate surface modification evade the reticuloendothelial system and enable high level accumulation of siRNA in the tumor cells. Recent effort focuses on de-assembly of the nanoparticles and cargo release from the endosome. Specific examples of siRNA mediated therapy in animal models will be presented. Joseph DeSimone, PhD Micro- and Nano-Fabricated Particles for Therapeutics and Vaccines We are employing top-down particle fabrication technologies adapted from the microelectronics industry to make highly uniform shape specific particles of controlled composition for use as therapeutics and vaccines.  Strategies will be discussed for the intracellular release of various cargos and for selective targeting of particles. Steven F. Dowdy, PhD siRNA Delivery: Tackling the 800 Pound Gorilla siRNA induced RNAi responses have great potential to treat human disease, especially cancer and viral infections. However, siRNAs are negatively charged, 14,000 Dalton macromolecules with no bioavailability to enter cells and siRNA cellular delivery remains a significant rate-limiting step for development of RNAi therapeutics. To address the siRNA delivery problem, we developed a Peptide Transduction Domain-dsRNA Binding Domain (PTD-DRBD) fusion protein. DRBDs bind siRNAs in a sequence-independent manner that masks the negative charge, allowing for PTD-mediated siRNA delivery in vivo. PTD-DRBD fusion proteins begin to address the siRNA delivery problem with a high efficiency and low toxicity, biodegradable platform. Joshua Mendell, MD, PhD MicroRNA Replacement Therapies MicroRNAs (miRNAs) are ~21-23 nucleotide RNAs that regulate the stability and translation of target messenger RNAs. We and others have demonstrated that dysregulated expression of miRNAs is a ubiquitous feature of cancer cells and specific miRNAs can act as oncogenes and tumor suppressors. More recently, we showed that due to their ability to strongly inhibit tumorigenesis, select miRNAs represent potent and non-toxic anti-cancer therapeutic agents when delivered systemically. I will present our latest findings regarding the roles of miRNAs in cancer and other diseases and the therapeutic potential of miRNA delivery.

Break 3:00 pm – 3:30 pm EDUCATION SESSIONS

The goal of the ASGCT Education Program is to provide participants with a broad background and introduction to topics relevant to the field of gene and cell therapy. Education Sessions will be subdivided into two headings. “Topical Review” sessions will provide a broad perspective of an area of importance to the gene and cell therapy community such as major vector systems, specific target organs or diseases, and funding/regulatory issues. “Emerging Field Review” sessions will provide a broad perspective of a technological or scientific discipline that has recently become of great significance to ASGCT members.

EXHIBITOR PROSPECTUS final program

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Program Schedule 3:30 pm - 5:00 pm Room: Virginia Suite

Emerging Field Review: Genomic Basis of Human Disease This program will be presented by three established investigators at the NIH National Human Genome Research Institute and will highlight active research programs of direct relevance to the field of gene and cell based therapies. The topics to be covered include: 1) The Genomics Landscape Circa 2010, 2) The ClinSeq Project: Piloting Genome Sequencing for Research in Genomic, and 3) The NIH Undiagnosed Diseases Program. Chair Paul B. McCray, Jr., MD Speakers Eric D. Green, MD, PhD

The Genomics Landscape Circa 2010 The Human Genome Project’s completion of the human genome sequence in 2003 was a scientific achievement of historic proportions. It also signified a critical transition, as this new foundation of genetic information started to be used in powerful ways by researchers and clinicians to tackle increasingly complex problems in biomedicine. Current efforts in genomics research are focused on using genomic data and technologies to acquire a deeper understanding of biology and to uncover the genetic basis of human disease. Together, these pursuits are moving us down an exciting path towards genomic medicine and fulfilling the promise of a sequenced human genome. Leslie G. Biesecker, MD Large Scale Sequencing and Hypothesis-Generating Clinical Research To date, high-throughput DNA sequencing has been used to elucidate the complete sequence of various species’ genomes. In contrast, its application to clinically directed research has been minimal. It is postulated that this technology will be applied to clinical care in the future – such as for pre-symptomatic diagnoses of medical conditions, customization of preventive strategies, and determination of individuals’ pharmacogenetic profiles. The National Human Genome Research Institute (NHGRI) has a longterm goal of developing the technology and infrastructure for performing large-scale medical sequencing (LSMS) for health research and, eventually, routine clinical care. Thus, at this time pilot studies are needed to start addressing some of the issues surrounding the development and implementation of LSMS in the clinical research setting.

We have launched a pilot project, called ClinSeq™, within the NHGRI intramural program. The objective of ClinSeq™ is to pilot approaches to the technical, medical, and genetic counseling issues related to LSMS by generating a large amount of sequence data from a set of participants. Unlike other projects that maintain anonymized samples, ClinSeq™ is being designed to maintain identified samples to allow us to implement an iterative clinical research strategy that over time will lead to return of individual results to study participants. With this design we hope not only to contribute to the knowledge about susceptibility genes, but also to understand how to integrate this technology into clinical practice. William A. Gahl, MD, PhD The NIH Undiagnosed Diseases Program The NIH Undiagnosed Diseases Program (UDP) has as its goals to provide answers to patients with mysterious conditions that have long eluded diagnosis and to advance medical knowledge about rare and common diseases. In the past two years, the UDP has received 2700 inquiries and 1100 medical records. Approximately 240 patients have been accepted; a diagnosis has been obtained for 10-15% of patients seen. The application of state-of-the-art genetic analysis makes this program a model for the use of genetics in the investigation of diagnostic dilemmas. One new disease has been discovered, along with unusual variants of known diseases. The Program also provides insights into the current practice of medicine in the United States.

Wednesday, May 19th

Education Session 120

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Wednesday, May 19th

Program Schedule Education Session 121

3:30 pm - 5:00 pm Room: Marriott Ballroom Salon 1

Emerging Field Review: Regulatory RNAs About a decade ago, it became clear that RNA has a widespread, general role in gene regulation. At least part of this regulation is mediated through a mechanism termed RNA interference (RNAi). The first talk will discuss the basic principles of RNAi and the biology of the small RNA species which mediate RNAi. The discovery of RNAi has opened up new avenues in genetic engineering which will be covered by the subsequent talks: Delivery of a small interfering RNA (siRNA) or an artificial microRNA (miRNA) with a specific sequence directed against an endogenous mRNA can result in potent knockdown of any gene of interest. Conversely, endogenous regulatory RNAs can be exploited as an additional layer of regulation to restrict the expression of an exogenously introduced transgene to cells of a specific tissue origin, lineage or differentiation state. Finally, as miRNAs are increasingly being implicated in human disease, technologies that allow their manipulation (knockdown and overexpression) become relevant. Co-Chairs Bernhard Rudolf Gentner, MD & Alfred Lewin, PhD Speakers Brian D. Brown, PhD

The Biology and Application of Regulatory RNAs Over the past few years major advances have been made in understanding the action and mechanism of regulatory RNAs.  These advances have helped to identify some of the important factors that influence microRNA and target interaction, which, in turn, is facilitating the improvement of technologies that utilize or target the microRNA pathway, including RNA interference (RNAi).  Here we will discuss some of the recent findings in regulatory RNA biology and how these findings are being applied. Ryan L. Boudreau, PhD RNAi-based Strategies for Silencing Disease With a better understanding of endogenous miRNA biogenesis and gene silencing processes, scientists have devised strategies to co-opt the cellular RNAi machinery in order to specifically silence various genes of interest. In this way, RNAi serves as a powerful molecular tool used to study gene function in biological processes and provides a novel strategy to treat a variety of diseases (e.g. dominant genetic disorders, cancer and viral invasion, among others). The application of RNAi as a biological or therapeutic tool is primarily limited by our ability to introduce inhibitory RNAs into target cells or tissues. Inhibitory RNAs can be designed to mimic primary miRNA stemloops (artificial miRNAs), processed pre-miRNAs (short-hairpin RNAs or shRNAs) or mature miRNAs with perfect complementarity to their targets (small interfering RNAs or siRNAs). This presentation will overview these RNAi vectors, delivery strategies, and highlight important results regarding RNAi vector safety. Bernhard Rudolf Gentner, MD How to Study and Exploit microRNAs for Gene Therapy Chromatin state and the presence of a particular set of transcription factors binding to the promoter sequence determines the transcription of a gene into mRNA. More recently, it has become clear that the mRNAoma is extensively subjected to negative regulation by a system involving hundreds of small RNA molecules, the so called “microRNAs”. Similar to the transcription factor complement, the “microRNAoma” is specific for tissues, lineages as well as cellular differentiation and activation states, offering an opportunity to exploit this endogenous layer of regulation to tune the expression of engineered DNA. microRNA regulation has proved a powerful tool to shut down expression in entire tissues like hematopoietic cells, hepatocytes, muscle, and nervous tissue, and thus control transgene immune responses, limit off-target transgene toxicity and even change the tropism of oncolytic viruses. Another emerging application for microRNA regulated expression cassettes is stem cell research and therapy. microRNAs allow to dissect different stages of differentiation, from the bona fide stem cell down to the progeny of different lineages. Applications for iPS cells and hematopoietic stem cells will be discussed. A potential side effect of exploiting miRNA regulation is the saturation of miRNA activity and thus deregulation of natural miRNA targets. Guidelines how to avoid miRNA saturation will be presented.

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Program Schedule 3:30 pm - 5:00 pm Room: Wilson ABC

Topical Review: Cell Therapy Chair Edwin M. Horwitz, MD, PhD Speakers Edwin M. Horwitz, MD, PhD

Fundamentals of Cell Therapy Peter Johnston, MD, FACC Stem Cell Therapy for the Heart Nearly 6 million people in the United States suffer from heart failure. This burden of disease has driven interest in stem cell therapy for the heart in the hope that dysfunctional myocytes and vasculature might be repaired or replaced. Clinical trials to date have explored the use of various skeletal, bone marrow-derived, endothelial, and endogenous cardiac precursor cells for cardiac repair. Along with much promise, there remain many questions about not only the optimal type of cell, but also the timing and delivery method necessary to achieve the maximal therapeutic benefit. Dao Pan, PhD Cell and Gene Therapy for CNS Abnormalities in Lysosomal Storage Disorders Lysosomal storage diseases (LSD) are a group of ~40 inherited metabolic disorders with a combined incidence of 1 in 1500 to 7000 live births. More than 70% of all LSD affects the central nervous system (CNS). Bone marrow stem cell transplantation has been shown to ameliorate many visceral symptoms in some LSD patients; however CNS disease associated with LSD remains as a major challenge. Here we will discuss experimental and preclinical studies toward new therapeutic strategies, including stem cell-based brain transplantation therapies, hematopoietic stem cell gene therapies, and CNS-targeted gene therapies. Issues that are pertinent to their potential translational applications will also be addressed. Education Session 123

3:30 pm - 5:00 pm Room: Thurgood Marshall W

Topical Review: Gene Therapy for Eye Diseases Recent successes in viral vector-based gene therapy clinical trials underscore the potential of gene therapy as a means to provide definitive solutions for currently incurable ocular diseases. This session will focus on the basic biology and vector development for ocular diseases. In addition, it will provide recent updates on novel strategies in vector design, as well as  the status and prospects of gene therapy clinical trials for ocular diseases. Co-Chairs Peter A. Campochiaro, MD & Tal Kafri, PhD Speakers Bill Hauswirth, PhD

AAV Vector Considerations for the Retina Considerations for AAV vectors intended for transducing cells of the retina include 1) the anatomical site of vector injection, 2) the vector serotype, 3) the size of the vector genome, 4) the strandedness of the vector genome and 5) the ability of novel AAV vectors to penetrate retinal tissue.  Each issue will be discussed in the context of gene-based therapies for degenerative retinal diseases. John G. Flannery, PhD Evolution of AAV Vectors for Ocular Gene Therapy

Wednesday, May 19th

Education Session 122

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American Society of Gene & Cell Therapy

Wednesday, May 19th

Program Schedule Peter A. Campochiaro, MD Overview of Gene Therapy for Ocular Diseases The potential of ocular gene therapy has been demonstrated in animal models and early clinical trials. Proof-of-concept has been provided for gene replacement of mutated genes in inherited retinal degenerations by 3 clinical trials demonstrating improved visual function by replacement of the RPE65 gene in patients with Leber’s congenital amaurosis. Studies are being considered for X-linked retinoschisis and Stargardt’s disease. A trial in which an antiangiogenic protein was delivered with an adenoviral vector for neovascular age-related macular degeneration (AMD) has shown good safety and some suggestion of biological activity. A trial in which a vascular endothelial growth factor binding protein is delivered with an AAV vector in patients with neovascular AMD has recently started. Preclinical studies suggest that gene delivery of neurotrophic factors or antioxidant proteins may be considered for retinal degenerations. Thus early clinical trials suggest several promising avenues for ocular gene therapy. Education Session 124

3:30 pm - 5:00 pm Room: Maryland Suite

Topical Review: Stem Cell Basics In order for stem cells to fulfill their potential as viable cell transplant/gene transfer therapeutics, it will be necessary to understand the process of stem cell self-renewal to obtain sufficient cell numbers to be applicable for transplant. Additionally, the genes/factors involved in differentiating these pluripotent cells down specific lineage pathways will also be crucial in the development of a transplantable therapeutic. The speakers in this session will address these important issues that not only provide further insight into organ and tissue development but also open up therapeutic avenues of practical application of these cells in regenerative medicine. Chair William F. Goins, PhD Speakers G. Ian Gallicano, PhD

Stem Cells: Past, Present, Future The idea of stem cell based therapies is particularly prevalent in the fields of diabetes, cardiac and neurological disorders. However, while the field of stem cell therapies may be relatively new, the road for stem cell research has been long and winding. Understanding hoe the evolution of the stem cell field has led to its current state is not only interesting, it can serve as a powerful tool for predicting its future. Consequently, this presentation will focus on the basic evolution of stem cells and their use in therapies. David F. Stroncek, MD Gene and MicroRNA Expression in Human Embryonic Stem Cells Global gene expression analysis has been used to identify factors responsible for the pluripotency of human embryonic stem (hES) cells. Most genes expressed by hES are involved with RNA post-transcriptional modification, DNA replication, recombination and repair, RNA Damage repair, cell cycle, and cell growth and proliferation. Transcript factors are among the most highly expressed hES genes. Studies focusing on specific genes found that the transcription factors OCT4, NANOG and SOX2 are important in hES development and pluripotency. Transcription factors likely interact with chromatin remodeling factors and histone modifying enzymes to modulate chromatin conformation. The knockout of dicer in hES, a nuclease critical in microRNA generation, compromises hES cell proliferation and differentiation and demonstrates an important role for microRNA in hES cells. MicroRNA in the miR-302 and miR-520 clusters are expressed in hES cells and may contribute to chromatin structure modifications. Ming Zhan, PhD Computational Biology Studies to Decipher Molecular Mechanisms Controlling Embryonic Stem Cell Pluripotency Computational biology has played a significant role in investigation of embryonic stem cells (ESCs). In this presentation, I will describe comparative genomics and comparative transcriptomics studies conducted on ESCs and genetic regulatory network analysis of ESCs through mathematical modeling. All the studies contribute to a systems understanding of molecular mechanisms controlling ESC self-renewal and differentiation.

Break 5:00 pm – 5:30 pm

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Program Schedule 5:30 pm - 7:00 pm Room: Marriott Ballroom Salon 1

Emerging Field Review: Progress Towards Using ES/iPS Cells for Regenerative Medicine The excitement over embryonic stem cells and induced pluripotent stem cells is that they are a potential source of cells for tissues in which tissue specific stem cells have either not been identified, not easily isolated, or not easily expanded.  That pluripotent stem cells can now be generated from somatic cells, without the destruction of an embryo, removes a major ethical barrier to their use.  Nonetheless, a continued barrier to the translation of pluripotent stem cells to clinical use is to expand and differentiate them in  both a safe and quantitatively sufficient manner that they can be used for tissue specific regenerative medicine applications.  This educational session will focus on the progress that has been made in generating sufficient numbers of safe tissue specific cells towards this end. Chair Matthew H. Porteus, MD, PhD Speakers Ronald D.G. McKay, PhD

Elias T. Zambidis, MD, PhD Generation of HSC and Red Blood Cells from Normal and Hemoglobinopathic iPSC Evan Y. Snyder, MD, PhD, FAAP Cross-Talk and Developmental Programs in Stem Cell Biology – A Key to Regenerative Medicine Education Session 131

5:30 pm - 7:00 pm Room: Maryland Suite

Emerging Field Review: Targeted Integration Uncontrolled insertion of foreign DNA into the human genome may have deleterious consequences, and is a serious safety concern in the clinical application of gene therapy. New molecular tools are being developed to target transgene integration to specific sites in the genome and increase the frequency of homologous recombination, with the purpose of achieving gene correction or replacement in the original genomic environment. These advances include the use of Zn-finger nucleases and homing endonucleases, and the delivery of homologous recombination cassettes by viral and non-viral vectors. They will be discussed in the context of developing better and safer gene transfer technology. Chair Fulvio Mavilio, PhD Speakers J. Keith Joung, MD PhD

Engineered Zinc Finger Nucleases for Targeted Genome Modification Engineered zinc finger nucleases (ZFNs) form the basis of a broadly applicable approach for inducing targeted alterations with high efficiency in human and other mammalian cells. This session will provide an introduction to this important technology by: (1) discussing methods for engineering customized ZFNs including Oligomerized Pool ENgineering (OPEN) and modular assembly; (2) describing how ZFNs have been used to introduce knockout mutations, insertions and other specific alterations via non-homologous end-joining and homologous recombination; and (3) considering future experiments and goals for advancing the use of ZFNs for therapeutic and research applications. Andrew M. Scharenberg, MD Homing Endonucleases as Tools for Targeted Integration The idiosyncratic nature of side effects resulting from random integration of promoter/gene cassettes in the context of viral vector-mediated gene therapy has led to increasing interest in targeted integration as an alternative approach. A promising methodology for targeted integration involves application of rare cleaving nucleases to induce site specific homologous recombination. Homing endonucleases are an important class of rare cutting nucleases whose potential applications in targeted integration are only now emerging. Homing endonuclease biology, properties, engineering, and applications in targeted integration will be reviewed.

Wednesday, May 19th

Education Session 130

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American Society of Gene & Cell Therapy

Wednesday, May 19th

Program Schedule David W. Russell, MD, PhD AAV-Mediated Gene Targeting AAV vectors efficiently recombine with homologous chromosomal sequences in mammalian cells. Up to 1% of the entire cell population exposed to vector particles undergoes gene targeting under optimal conditions. Insertions, deletions and substitutions can all be introduced at precise chromosomal locations with high fidelity. The technique works well in many cell types, including transformed cell lines, stem cells, and even in vivo in murine hepatocytes. Importantly, the gene targeting occurs without genotoxicity and endonucleases are not required to obtain high targeting frequencies. Education Session 132

5:30 pm - 7:00 pm Room: Thurgood Marshall W

Topical Review: Gene Delivery to the Heart Heart disease is one of the leading causes of morbidity and mortality worldwide.  While molecular causes are known for many heart diseases, it requires effective heart gene delivery methods to translate these findings into therapy.  Because of the dynamic nature of the heart physiology and the unique anatomic location, cardiac gene transfer has been a great challenge.  Significant progress has been made over the last few years.  In this session, a historical overview of heart gene transfer will be provided.  Considering the importance of the large animal model in translational medicine, special emphasis will be given to cardiac gene delivery in the canine and porcine models. Chair Dongsheng Duan, PhD Speakers Dongsheng Duan, PhD

Overview of Heart Gene Transfer in Mice Efficient cardiac gene transfer holds great promise in treating heart diseases, a major health threat worldwide. Over the last decades, tremendous progresses have been made in delivering the marker and/or therapeutic genes to the mouse heart. This presentation will provide a historic review of the literature and an update of the current developments. Roger J. Hajjar, MD Gene Therapy for Heart Failure: New Targets and New Vectors Despite the proliferation of therapies, congestive heart failure remains a progressive disease where a desperate need for innovative rather than incremental therapies to reverse the course of ventricular dysfunction. Recent advances in understanding the molecular basis of myocardial dysfunction, together with the evolution of increasingly efficient gene transfer technology; have placed heart failure within reach of gene-based therapies. One of the key abnormalities in both human and experimental HF is a defect in sarcoplasmic reticulum (SR) function, which is responsible for abnormal intracellular Ca2+ handling. Restoring SERCA2a levels has been shown to improve function, metabolism and/or survival in a number of experimental models of heart failure. Over the last ten years we have undertaken a program of targeting important calcium cycling proteins in experimental models of heart by somatic gene transfer. This has led to the completion of a first-in-man phase 1 clinical trial of gene therapy for heart failure using adeno-associated vector (AAV) type 1 carrying SERCA2a. In this Phase I trial, there was evidence of clinically meaningful improvements in functional status and/or cardiac function which were observed in the majority of Neutralizing antibody negative patients at various time points. The safety profile of AAV gene therapy along with the positive biological signals obtained from this phase 1 trial has led to the recent completion of a phase 2 trial of AAV1.SERCA2a in NYHA class III/ IV patients. Charles R. Bridges, MD, ScD Methods for Cardiac Gene Delivery This presentation will review the available methods of cardiac vector mediated gene delivery including those in use in current clinical trials. Methods considered will include intracoronary injection, intramuscular injection, pressurized coronary venous retroinfusion, and cardiopulmonary bypass-mediated techniques.  The relative strengths and weaknesses of these delivery methods will be discussed with reference to efficiency of transfection, cardiac versus collateral organ specificity and relative propensity to induce a T-cell mediated immune response. 

EXHIBITOR PROSPECTUS final program

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

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Program Schedule 5:30 pm - 7:00 pm Room: Virginia Suite

Topical Review: Immunotherapy of Cancer The immune system can be properly modified to eradicate cancer. Currently, effector molecules, such as monoclonal antibodies and cells such as tumor-reactive lymphocytes, are able to selectively target and eliminate tumors, may be isolated and/or engineered. The potency of cellular adoptive immunotherapy has been revealed by persistent and complete clinical responses obtained with by the transfer of allogeneic T lymphocytes in the context of allogeneic hemopoietic cell transplantation (alloHSCT) and by the transfer of autologous tumor specific cytotoxic T lymphocytes (CTLs) in cancer patients. Major hurdles limiting adoptive T cell therapy relate to toxicity (ie: graft-versus-host disease-GvHD in allo-HSCT), feasibility and efficacy (ie: difficulty in expanding rare, high-avidity tumor-specific CTLs). Gene transfer technologies may allow to overcome all these hurdles. The transfer of tumor-specific TCR or chimeric antigen receptors (CARs), confers tumor-specificity and activity to autologous T cells. The transfer of a suicide gene into allogeneic lymphocytes allows to tame toxicity of allogeneic lymphocytes. In this session, we will discuss clinical results, challenges and advances recently made to improve the potency and specificity of adoptive immunotherapy with genetically modified lymphocytes to treat cancer. Chair Chiara Bonini, MD Speakers Richard A. Morgan, PhD

Using Engineered T Cells for the Treatment of Cancer Adoptive cell transfer (ACT) using anti-tumor antigen reactive T cells has proven to be a useful strategy for the treatment of metastatic melanoma, with objective response rates of up to 72%, with 16% of patients rendered disease free. These studies involved the extraction and ex-vivo expansion of naturally occurring tumor infiltrating lymphocytes (TIL). Only one-half of patients are able to receive this therapy due to lack of harvestable tumors, inability to grow TIL, or a lack of cellular reactivity. Recent strategies aimed at broadening ACT involve the use of chimeric antigen receptors (CAR) or T-cell receptor (TCR) genes specific for tumor associated antigens and the use of viral vectors to transduce these genes into peripheral blood lymphocytes. TCR gene therapy has been applied with some success in recent trials targeting MART-1, gp100 and NY-ESO-1. CAR gene therapy trials are also showing promising results in protocols targeting CD19+ lymphomas. Efforts aimed at improving the response rate and minimizing on-target toxicity are in progress. Gianpietro Dotti, MD Car-Based Gene Transfer in T Lymphocytes Expression of a transgenic “T-body” or “chimeric antigen receptor” (CAR) in human T lymphocytes combines the specificity of an antibody with the homing and target-cell destruction capabilities of T cells. This technology was first described 15 years ago, and after significant improvements in gene transfer technologies and better understanding of immunological pathways is now reaching a critical phase of clinical evaluation. In this presentation we will summarize the concept of CAR-based immunotherapy, describe the steps accomplished, and outline the future progress we need to make if this approach is truly to improve cancer immunotherapy. Chiara Bonini, MD T-Cell Based Gene Therapy in Hematopoietic Stem Cell Transplantation The potency of cellular adoptive immunotherapy for the treatment of cancer has been clearly revealed by the persistent and complete clinical responses observed in patients undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) followed by the adoptive transfer of donor T lymphocytes. However, several hurdles remain to be overcome before adoptive immunotherapy can become a broadly applicable therapeutic approach to treat cancer, and gene transfer approaches represent ideal solutions to overcome these hurdles. In allo-HSCT, most of the antileukemic potential resides in alloreactivity towards patient-specific antigens, such as minor and major histocompatibility antigens, by donor lymphocytes. Unfortunately, alloreactivity is also responsible for the most serious and frequent complication of allo-HSCT: the Graft-versus-Host-Disease (GvHD). The risk of GvHD increases with the level of HLA disparity between host and donor, and leads to impaired quality of life and reduced survival expectancy, particularly in patients transplanted from HLA-mismatched donors. Gene transfer technologies are promising tools to manipulate donor T cell immunity to enforce the graft-versus-tumor (GvT) effect, to promote a functional post-transplant immune reconstitution (graft-versus-infection-GvI) and to prevent or control GvHD. To this purpose, several cell and gene transfer approaches, have been investigated at pre-clinical level, and are being implemented in clinical trials. The majority of these approaches, based on negative or positive selection of specific T cell subpopulations, aim at reducing the repertoire of donor lymphocytes infused with or after the graft, so to prevent alloreactivity. In contrast, suicide gene therapy aims at a selective control of GvHD in treated patients, thus permitting the full exploitation of alloreactivity against cancer cells. In a phase I-II clinical trial enrolling 54 pts undergoing haploidentical HSCT, we showed that the transfer of a suicide gene into donor lymphocytes, allows to provide rapid and effective immune reconstitution, and control of GvHD. By taming the toxicity of allo-HSCT the suicide gene approach renders haploidentical HSCT a feasible, safe and more effective option for cancer patients of all ages. Cell and gene therapy approaches currently implemented for a safe exploitation of donor immunity against cancer, in the context of allo-HSCT will be discussed.

Wednesday, May 19th

Education Session 133

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American Society of Gene & Cell Therapy

Wednesday, May 19th

Program Schedule Education Session 134

5:30 pm - 7:00 pm Room: Wilson ABC

Topical Review: Pre-existing Immunity to Gene Transfer Vectors Many viral gene therapy vectors are derived from parent viruses that are present in the human population. Pre-existing immunity to such vectors in form of antibodies and memory B and T cell responses can severely hamper gene transfer in humans. This session will review basic aspects of B cell biology, prevalence of antibodies to DNA viruses (AAV and adenovirus) in the human population, and CD8+ T cell responses to AAV capsid in humans. Chair Roland W. Herzog, PhD Speakers Michael Cancro, PhD

The Persistent Problem of B Cell Memory Primary and memory/effector B cell subsets occupy distinct anatomic and homeostatic niches. Despite growing understanding of the factors governing pre-immune B cell lifespan and homeostasis, the factors regulating antigen-experienced pools – including memory B cells and plasma cells – remain less clear. These likely include members of the BLyS family of cytokines and receptors, as well as molecular interactions between B lineage cells and survival-promoting stromal elements. Luk H. Vandenberghe, PhD B-cell Immunity to Viral Vectors Many of the commonly used vectors for gene therapy application are based on viruses that are endemic in the human population. Therefore, pre-existing immunity may alter the safety and efficacy of gene therapy strategies. In addition, repeated administration of the vector may be required for certain therapeutic applications which will also need to overcome the hurdle of pre-existing immunity. Here, we will focus on the breadth and implication for gene transfer of the presence and prevalence of antibodies to Adeno and Adeno-Associated viruses in humans and animal models. Katherine A. High, MD Human T Cell Responses to AAV Capsid Studies in healthy donors have shown antigen-specific memory CD8+ T cells to AAV capsid in a substantial portion of the human population. We have hypothesized that these cells may expand on re-exposure to AAV capsid through administration of AAV vectors. Studies in several AAV gene transfer trials have shown that vector administration is followed by development of positive responses on IFN-g ELISPot assays. We have shown that capsid-specific CD8+ T cells can lyse AAV-transduced hepatocytes in an in vitro assay, but the clinical and biological correlates of a positive IFN-g ELISPOt response are still under investigation. A clear understanding of capsid antigen processing and presentation, and of T cell responses to AAV, will be key to optimal application of the vector.

Exhibit Hall Open & Welcome Reception 7:00 pm – 9:00 pm Room: Exhibit Hall C

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13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

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Program Schedule Thursday, May 20 George Stamatoyannopoulos Lecture 200 8:00 am - 9:15 am Room: Marriott Ballroom Salons 1, 2 & 3 George Stamatoyannopoulos Lecture Chair

Hal E. Broxmeyer, PhD

Understanding and Enhancing Stem/Progenitor Cell Function and Engraftment There are a number of cytokines, cytokine receptors, cell surface proteins, and intracellular molecules that regulate hematopoietic stem (HSC) and progenitor cell (HPC) function.  Amongst these are the chemokine, stromal cell derived factor-1 (SDF-1/CXCL12), its receptor, CXCR4, the cell surface protein CD26/ Dipeptidylpeptidase IV, which truncates and inactivates specific cytokines, the Rapamycin sensitive m-TOR pathway, and the sirtuin family member, SIRT1.  Efforts are underway to modulate these proteins/molecules for enhanced engraftment of HSC/HPC and accelerated hematopoiesis. Exhibit Hall Coffee Social 9:15 am – 10:30 am Room: Exhibit Hall C

Thursday, May 20th

Barrie J. Carter, PhD Speaker

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American Society of Gene & Cell Therapy

Program Schedule Scientific Symposium 210

10:30 am - 12:30 pm Room: Marriott Ballroom Salons 2 & 3

Clinical Trials

Thursday, May 20th

Chair R. Jude Samulski, PhD Speakers Steven A. Rosenberg, MD, PhD

Cancer Immunotherapy Using Genetically Modified T Cells Patients with advanced cancer have been treated by the adoptive transfer of autologous T cells transduced with retroviral vectors encoding antitumor T cell receptors. Objective cancer regressions have been seen in patients with metastatic melanoma receiving cells targeting melanoma/melanocyte differentiation antigens and in patients with synovial cell sarcoma who received cells targeting the NY-ESO-1 cancer-testes antigen. Clinical trials have also begun targeting CD19 on lymphomas and targeting carcinoembryonic antigen on colorectal cancers. Ian MacLachlan, PhD A Phase I Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Lipid Nanoparticle ApoB siRNA (ApoB-SNALP) in Hypercholesterolemic Subjects Stabilized nucleic acid lipid particles (SNALP) are a modular lipid nanoparticle delivery platform developed for the delivery of nucleic acid based drugs. Two SNALP product candidates, containing siRNA payloads, are currently being evaluated in clinical trials in oncology and metabolic indications. By the end of 2010 we expect that as many as five SNALP based product candidates will be in clinical trials. SNALP containing siRNA that potently silence the conventionally non-druggable target, apolipoprotein B, have been evaluated in a Phase 1 clinical trial in hypercholesterolemic subjects. Early results, including the elucidation of previously cryptic immune stimulatory activity associated with the chemically modified siRNA payload, will be presented. Bill Hauswirth, PhD Leber Congenital Amaurosis (LCA) Gene Therapy Clinical Trial Results and implications of a recently initiated AAV vector clinical trial for LCA2 (recessive RPE65 mutations) will be discussed.  LCA2 patients received 150ul (5.9 exp10 vector genomes) of GMP grade AAV2-CBA-hRPE65 vector subretinally and their visual function then followed periodically over the next 18 months.  No adverse events were noted for any patient, all demonstrated substantial and significant improvement in light sensitivity, up to 63,000 times better that pretreatment baselines, and some developed a new preferred locus of visual fixation centered on the area of treated retina.  R. Jude Samulski, PhD Designing AAV Vectors for Translational Studies: Update on DMD Phase 1 Trial Initial AAV vectors for clinical studies have been derived from natural isolates. We have engineered and tested in Phase 1 DMD study chimeric vector derived from type 1 and type 2. Outcome of these studies and next generation of DMD viral vectors suited for efficient gene delivery will be described and pre-clinical data presented. Scientific Symposium 211

10:30 am - 12:30 pm Room: Marriott Ballroom Salon 1

Tissue Specific Stem Cells Chair Carolyn Lutzko, PhD Speakers Pamela Itkin-Ansari, PhD

Development of Cell Based Therapies for Diabetes The presentation will address a range of issues regarding development of pancreas specific stem cell based therapies. First, evidence for stem cells in the adult pancreas and suggested methods for improved identification of such populations will be discussed. Strategies for the recruitment and differentiation of tissue specific stem cell populations in vitro and in vivo will also be addressed. Finally, because insulin producing β-cells are cell autonomous (in contrast to many cell targets, e.g. neurons and cardiomyocytes), they are amenable to novel transplantation and regeneration approaches which will be presented.

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13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

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Program Schedule Markus Grompe, MD Hepatobiliary Stem/Progenitor Cells Carolyn Lutzko, PhD Developing Stem Cell Based Therapies for Lung Disease: Bringing Genes to Lung Stem Cells and Stem Cells to the Lung This presentation will focus on different strategies for developing stem cell based therapies for lung diseases. One strategy that will be discussed is the genetic modification of endogenous lung progenitor cells during development. This presentation will also discuss progress on the differentiation of lung progenitor cells from pluripotent stem cells in vitro.   Mervin C. Yoder, MD Are Endothelial Progenitor Cells Derived from the Endothelial Lining of Blood Vessels? Circulating endothelial progenitor cells (EPC) have been reported to play important roles in vascular repair and regeneration and are altered in concentration in patients with arthritis, diabetes, cancer, and cardiovascular disease. No unique marker exists to identify this cell. Indeed, evidence suggests that numerous cell types participate in neoangiogenesis under the term EPC. We will review recent literature that suggests the local endothelial lining to be a major site of vascular repair.

10:30 am - 12:30 pm Room: Virginia Suite

Cell and Gene Therapy Approaches for Genetic Diseases: Progress and Challenges Chair Charles P. Venditti, MD, PhD Speakers Nathalie Cartier-Lacave, MD

Gene Therapy for Adrenoleukodystrophy We report results of the first clinical trial using an HIV vector to correct hematopoietic stem cells (HSC) in adrenoleukodystrophy, a severe degenerative disease of the central nervous system. Three children received autologous corrected HSC after full myeloablative conditionning. Up to 36 months after treatment, stable expression was demonstrated in peripheral blood leukocytes and bone marrow CD34+ cells, with polyclonal integration of the lentiviral vector. Neurological outcome was comparable to that observed after allogeneic transplantation. These results demonstrate that lentiviral vectors are promising tools for gene transfer into hematopoietic stem cells in the absence of selective advantage. Alessandra Biffi, MD HSC Gene Therapy for Metachromatic and Globoid Leukodystrophies: The Next Trials for Inherited Neurological Disorders We are implementing a gene therapy approach based on the transplantation of gene corrected hematopoietic stem cells (HSC) for the treatment of severe forms of Lysosomal Storage Disorders (LSD) lacking efficacious and safe alternative therapeutic opportunities. To this goal, we exploit the unique features of lentiviral vectors (LV), which are prime candidates for HSC gene transfer. Indeed, by using LV for HSC gene correction, we proved the therapeutic potential and safety of HSC gene therapy in the murine model of metachromatic leukodystrophy (MLD), a severe dysmyelinating LSD. According to these promising results, HSC gene therapy for MLD has now reached clinical testing. Similarly, an advanced LV design allowed rendering HSC gene therapy a feasible and efficacious approach to be further developed also for globoid cell leukodystrophy, on which we obtained very promising evidences in the disease model. Ronald D.G. McKay, PhD Robert D. Steiner, MD CNS Transplantation of Neural Stem Cells in Neuronal Ceroid Lipofuscinoses: Phase I Trial Results Infantile and late-infantile neuronal ceroid lipofuscinosis (INCL and LINCL) are universally fatal neurodegenerative lysosomal storage disorders caused by palmitoyl protein thioesterase and tripeptidyl peptidase deficiency, respectively. A Phase I trial of human central nervous system stem cells (HuCNS-SC®) was conducted in subjects with INCL and LINCL. This study represents the first US FDA authorized use of human neural stem cells for clinical testing. The primary goal of the trial was to evaluate the safety of direct CNS delivery of HuCNS-SC, the immunosuppression regimen, and the surgical technique. Six children underwent HuCNS-SC transplantation;. the study results to be presented will include adverse events as well as the neurological, seizure, EEG, radiological, neuropsychological, functional, and quality-of-life measures assessed at frequent intervals post-transplant, as well as evidence of engraftment.

Thursday, May 20th

Scientific Symposium 212

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American Society of Gene & Cell Therapy

Program Schedule Scientific Symposium 213

10:30 am - 12:30 pm Room: Delaware Suite

How Viruses Cause Disease: Implications for Vector and Vaccine Development

Thursday, May 20th

Chair Roberto Cattaneo, PhD Speakers Lynn W. Enquist, PhD

Response of PNS Neurons to Herpesvirus Infection Most alpha herpesviruses are pantropic, neuroinvasive pathogens that establish a reactivateable, latent infection in the peripheral nervous system of their natural hosts. Various manifestations of herpes disease rely on extent and direction of the spread of infection between the surface epithelia and the nervous system components that innervate that surface. One aspect of such controlled spread of infection is the capacity for synaptically defined, transneuronal spread, a property that makes alpha herpesviruses useful tools for determining the connectivity of neural circuits and gene delivery. Spread of infection among neurons involves long distance travel of virion component in axons. I will discuss our recent work on the response of cultured PNS neurons to infection. Jeffrey Bergelson, MD Cell Biology of Virus Entry Viruses need cells, and they have evolved to manipulate cellular processes at many stages of their life cycle. In a number of cases, virus-induced signals are needed to prepare the cell for virus entry and infection. I will discuss several examples of viruses— including adenoviruses and picornaviruses— that use receptor-mediated signals to initiate the entry process. Bernard Moss, MD, PhD Poxvirus Replication and Pathogenesis: Implications for Vector and Vaccine Development The ability of vaccinia virus to infect most cells provides an important advantage for their extensive use as an expression vector and as an oncolytic agent. We have shown that an unprecedented number of viral proteins are required for the membrane fusion step, which can occur either at the plasma membrane or in endosomes. These 12 proteins are associated in a complex and conserved in all members of the poxvirus family. Roberto Cattaneo, PhD The Force Behind Targeting: How the Receptor-Triggered Membrane Fusion Mechanism of Measles Virus Works Measles is one of the most infectious viruses, and its portable entry system has become the paradigm for targeting oncolysis and therapeutic gene delivery. While this system is strictly regulated, the mechanism triggering fusion of the viral envelope with the plasma membrane was unknown. We show that re-alignment of the subunits of the viral attachment protein (H) is the trigger, and that force exerted at an appropriate location on the H-dimer unlocks the trigger at the plasma membrane, just as low pH or proteases trigger endosomal membrane fusion in other viral entry systems. Scientific Symposium 214

10:30 am - 12:30 pm Room: Maryland Suite

Gene and Cell Therapeutic Approaches for Musculo-Skeletal Diseases Co-Chairs Paula Clemens, MD & Alan J. Nixon, DVM, MS Speakers Edward M. Schwarz, PhD

Revitalizing Structural Allografts To the end of a tissue-engineering solution for large segmental defects in bone caused by trauma or tumors, we proposed rAAV-coated allografts, which introduce angiogenic, osteogenic and osteoclastogenic factors into the healing environment to stimulate vascular ingrowth, bone formation and remodeling of the necrotic tissue.  Here I will describe the molecular biology of this technology, its methodology and application in mice, and the translational outcome measures that have been developed to prove efficacy in large animals and humans in which torsional biomechanical strength is the primary outcome measure.  

EXHIBITOR PROSPECTUS final program

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

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Program Schedule Steven C. Ghivizzani, PhD Gene Therapy for Achondroplasia Achondroplasia is the most common form of short-limbed dwarfism and is caused by a specific mutation in the transmembrane portion of fibroblast growth factor receptor 3 (FGFR3), a negative regulator of growth and maturation in growth plate chondrocytes. This “gain of function” mutation causes enhanced signaling from FGFR3, leading to reduced growth in bones that grow by endochondral ossification. Using gene delivery, we have been exploring the utility of systemic delivery of a secreted FGFR3 variant as a means to titrate circulating FGFR3 ligands and, thereby, reduce aberrant FGFR3 signaling to rescue bone growth. Our results thus far in explant culture and in a mouse model of achondroplasia have been highly encouraging, and indicate that a related post-natal treatment approach may be viable in humans.

Carmen Bertoni, PhD Enhanced Cell-Mediated Regenerative Approaches to DMD Using RNA Interference Many of the diseases affecting muscle are distinguished by the impaired ability of muscle progenitor cells to fuse and form new fibers and the inability of the existing fibers to maintain normal homeostasis. Among those, Duchenne muscular dystrophy (DMD) is the most severe and is caused by lack of dystrophin expression. The loss of muscle mass and strength typically seen in DMD patients is thought to be attributed to the progressive atrophy and loss of individual muscle fibers, which is concomitant to the accumulation of fibrotic and fat tissues in skeletal muscles. The mdx mouse has been instrumental in determining the potential beneficial effects of some of the therapeutic approaches currently under development for DMD. We have used High Throughput Screen (HTS) to identify modulators of muscle progenitor cells fusion in vitro and screened several siRNA libraries in order to identify genes that play active role during terminal differentiation. Secondary and tertiary screens were used to confirm the effects of RNAi on wild type or mdx muscle cultures and to select for candidate hits that significantly enhance myotube size.Transplantation of wild type muscle progenitor cells undergoing silencing of the genes identified by HTS enhanced the ability of those cells to repair skeletal muscles of mdx/nude mice and resulted in a significant increase in dystrophin expression. Direct intramuscular injection of a lentiviral vector expressing shRNAs of those genes was also shown to dramatically improve regeneration of injected muscles. These studies advance cell-mediated regenerative approaches to DMD and may enable us to identify treatments capable of slowing down progressive muscle wasting characteristic of many neuromuscular disorders. Scientific Symposium 215

10:30 am - 12:30 pm Room: Wilson ABC

Novel Cell and Gene Therapy Interventions for the Lung Chair Bruce A. Bunnell, PhD Speakers Daniel Weiss, MD, PhD

Recent Advances in Gene and Cell Therapies for Lung Diseases Gene and cell therapy approaches for lung diseases continue to rapidly develop. Progress has been made in vector development and several notable advancements have been made over the past year with use of gene transfer approaches for lung cancer. In parallel, cell therapy approaches for lung diseases have broadened and include studies of ex vivo lung regeneration using state of the art bioengineering approaches as well as increasing appreciation of immunomodulatory effects of adult mesenchymal stem cells in the lung.  Most notably, progress has been made in clinical trials of cell therapies for both pulmonary hypertension and for chronic obstructive pulmonary disease.  The introductory comments and subsequent presentations in this session will highlight these advances. 

Thursday, May 20th

Joel R. Chamberlain, PhD Systemic Delivery of RNAi Therapeutics for Treatment of Myotonic Dystrophy Dominant genetic diseases pose a particular challenge for therapy since the disease-causing cell product must be eliminated. We have chosen an RNA interference-based approach to target toxic mRNA that causes myotonic dystrophy. We show that disease pathology in the transgenic HSALR mouse model of DM1 is mitigated by systemic application of AAV6 carrying an HSA-targeted RNAi expression cassette. Aspects of systemic delivery and endogenous RNAi pathway effects will be presented in the context of therapeutic development. These studies highlight the potential of RNAi as a therapy for dominant muscle disease and for other diseases where body-wide therapeutic RNAi distribution is required.

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American Society of Gene & Cell Therapy

Program Schedule Paul B. McCray, Jr., MD Lentiviral Vector Development for Prevention and Treatment of Cystic Fibrosis Lung Disease This talk will provide an overview of recent developments in lentiviral vector technology and their applications for lung disease treatment.  Additional data will be presented regarding animal models of CF lung disease and their potential utility in pre-clinical studies of efficiency and efficacy of gene transfer.

Thursday, May 20th

Blair Roszell, MS Two Step Differentiation of Mouse Embryonic Stem Cells to Definitive Endoderm and Lung Epithelial Cells Cell-based therapy is a novel and promising treatment option for lung disease, but deriving large quantities of lung epithelial cells from embryonic stem (ES) cells is still inefficient. In my talk, I will compare growth factor-based and small molecule-based methods for deriving definitive endoderm. After an additional lung induction step, activin/wnt and IDE2 treated cells express a similar profile of lung-specific markers. Furthermore, in fetal pulmonary cell co-cultures, activin/wnt treated cells can integrate into alveolar forming units; whereas IDE2 treated cells express SP-C, but do not integrate into lung-like structures. Finally, ES-derived lung cells were endotracheally injected into preterm mice with evidence of AEII distribution within the lung parenchyma. These studies concludes that a recapitulation of development may enhance derivation of an enriched population of lung-like cells for use in cell based-therapy. Peter I. Lelkes, PhD Biomedical Engineering in the Lung The goal of this invited lecture is to present an overview of current tissue engineering and regenerative medicine -based approaches to repair dysfunctional lungs, with focus on neonatal and adult pulmonary diseases. Specifically, we will present our developmental biology-based studies to create vascularized alveolar structures in vitro and in vivo. In addition, we will discuss attempts by us and others to optimize the directed differentiation of embryonic stem cells towards pulmonary epithelial and other lung-specific cell types for distal lung tissue engineering and regeneration. John F. Engelhardt, PhD New Developments in Animal Models of Cystic Fibrosis Cystic fibrosis (CF) is a recessive disease that affects multiple organs and is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Animal modeling of this disease has been challenging, with species- and strain-specific differences in organ biology and CFTR function influencing the emergence of disease pathology. CF mice have been an invaluable system for dissecting the biology of CFTR function in certain organs, however, they generally lack pancreatic and spontaneous lung disease seen in humans with CF. Recently, CF pig and ferret models have been generated. Comparative analysis of these CF models may help to understand how the pleiotropic functions of CFTR in multiple organs influence disease progression. Special NIH Program Session 216

10:30 am to 12:30 pm Room: Thurgood Marshall West NIH Resources for Cell and Gene Therapy Investigators Moderator Cheryl L. McDonald, MD Speakers Daniel Rosenblum, MDP The National Gene Vector Biorepository and Coordinating Center (NGVBCC) The NGVBCC provides an array of support services for NIH-supported investigators engaged in research on Gene Therapy. The services include archiving of biological materials, insertional site analysis, pharmacology and toxicology resources, and a reagent repository. It has an open database and educational resource. The archiving and analytical services are limited to investigators at academic and other not for profit institutions. We will review the scope of the services, methods for accessing them, ways of meeting eligibility for their use, website link information, specimen submission and electronic access, and other relevant information pertinent to use of the facility. How the Clinical and Translational Science Awards (CTSA) Program can Support Cell and Gene Therapy Investigators The CTSA program replaced the GCRC (General Clinical Research Center Program) with a much broader support for novel product and team science within and between academic institutions and other related research support such as the NIH and industrial sponsors. The aim is to streamline project development, review, and implementation of translational and clinical research by providing support from a variety of services such as biostatistics, research design, ethics, clinical research management, informatics, and regulatory knowledge (including FDA). It also facilitates the creation of networks between academic centers and the recruitment of trial participants from the community. The presentation will review pertinent summary information about the CTSA program including web access to program information, access to services, potential support from CTSA-related key functions for development of clinical trials, and a brief description of concierge service (a team of experts who can provide support in the creation and approval of a clinical trial).

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Program Schedule Catherine McKeon, PhDP The NIDDK Molecular Therapy Centers and Cell Therapy Program This presentation will cover the NIDDK Molecular Therapy Centers and the Cell Therapy Program, as well as some other programs that offer resources to investigators.

Roy S. Wu, PhD The NCI Experimental Therapeutics Program (NExT) and the Chemical Biology Consortium (CBC) The NCI has recently consolidated their drug discovery and development efforts into a single pipeline for increased timeliness and efficiency and with an emphasis on unmet clinical needs. Researchers in academia, government or industry, either nationally or internationally, may apply. NCI is committed to moving high-priority discovery and development projects through to proof-of-concept clinical trials in a partnership relationship. NExT is not a grant or contract mechanism of support. Rather, projects will be evaluated and ranked by a peer committee followed by internal committees that will assess clinical need, develop project budgets and milestones with go/no go decisions, and provide access to the NCI’s drug discovery and preclinical and clinical development resources at no cost to the applicant. The resources include activities such as production of pharmaceutical-grade material, development of clinical formulations, and first-in-human studies. The discovery arm of NExT, known as the NCI Chemical Biology Consortium or CBC, is a collaborative network of 12 Centers that provide access to high-throughput screens and chemistry to optimize compounds for human use. Applications for potential NExT projects are accepted online four times a year. Visit http://next.cancer.gov/ for application instructions and more information. Danilo A. Tagle, PhD The NINDS Translational Research Program The NINDS Translational Research Program is specifically focused on pre-clinical therapy development necessary to begin clinical testing of therapeutic candidates for neurological disorders. This program supports the pre-clinical development of drugs, biologics, and devices including activities that lead to Investigational New Drug (IND) or Investigational Device Exemptions (IDE) applications to the Food and Drug Administration (FDA). Supported projects can include lead optimization, pre-clinical efficacy testing, predictive ADMET (absorption, distribution, metabolism, excretion, and toxicology) testing, formulation, manufacture, pharmacology, toxicology, and IND or IDE submission. For entry to the program, projects must have one or more identified therapeutic leads and convincing proof-of-principle on efficacy demonstrated in credible models of the disease or against defined disease targets. Lisbeth Welniak, PhD Production Assistance for Cellular Therapies (PACT) The PACT Group is an NHLBI-funded initiative that is committed to the advancement of effective cell therapies. This program was renewed in January, 2010 for a second five-year period of funding. PACT supports the development of novel somatic cell therapy products by providing production assistance to the cell therapy community, as well as educational training via web seminars and at meetings. Production assistance is provided through the funded contract manufacturing of therapeutic cell products at PACT processing facilities. These facilities offer scale-up capability and expertise in the translational development needed to produce cell therapy products under cGMP and cGTP regulations. PACT’s educational training focuses on three general areas: translational development/scale-up and manufacture of cell therapy products; and quality assurance and regulatory issues. The objective of the PACT program is to continue to advance cellular therapy research in the areas of regeneration of damaged/diseased tissues, organs, and biologic systems, and targeted treatments for serious diseases without effective therapies. The cell processing facilities will support translational research in novel cell therapies to advance the field of cellular therapy and to enable scientifically meritorious basic science research to reach the clinical trial stage. Please visit the PACT website at www.pactgroup.net for more information regarding the PACT program.

Thursday, May 20th

David G. Badman, PhD The NIH Rapid Access to Interventional Development Program (NIH-RAID) The NIH Roadmap has established the NIH-RAID Program (Rapid Access to Interventional Development), to make available, on a competitive basis, critical resources needed for development of new small molecule or natural product-derived therapeutic agents. This program, part of the Translational Research component of Reengineering the Clinical Research Enterprise, uses resources of the National Cancer Institute’s (NCI) Developmental Therapeutics Program and the National Heart Lung and Blood Institute’s (NHLBI) Gene Therapy Resource Program. Services provided depend upon the project and strength of the preliminary data. Services potentially available include: bulk supply, GMP manufacturing, formulation, assay development suitable for pharmacokinetic testing, and animal toxicology. Assistance also can be provided in the regulatory process. Currently, animal efficacy studies are not supported; neither are syntheses nor formulation of recombinant proteins or monoclonal antibodies. However, the NIH-RAID Pilot will consider requests for services to support later-stage preclinical development of these entities. Manufacture of certain gene vectors (non-GMP viral and non-viral gene vectors and GMP-grade adeno-associated virus and lentivirus vectors) can be requested, and preclinical studies of vectors may be supported. Additionally, the NIH-RAID Pilot will now consider requests for manufacture of small molecule or natural product material for any clinical study. Proposals must originate from academic or non-profit investigators, as well as companies eligible for Small Business Innovation Research Program (SBIR). Collaboration with larger industry partners is encouraged.

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Program Schedule Sonia Skarlatos, PhD The Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases Another unique resource that NHLBI offers to gene therapy researchers is the Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases at the University of California, Davis (http://www.cfmgt.ucdavis.edu/). Established in 2001, this program provides NHLBI-funded investigators with essential expertise, services, and resources to evaluate the safety and efficacy of gene transfer strategies as they emerge.

Thursday, May 20th

The NHLBI Gene Therapy Resource Program (GTRP) The NHLBI Gene Therapy Resource Program (GTRP) was initiated in March 2007 and since that time it has become a very active and valuable program. The program offers several services at no cost to investigators researching gene therapy for heart, lung, and blood disorders. The GTRP includes four core labs to assist researchers with preclinical vector production, pharmacology and toxicology testing, and production of clinical-grade AAV and lentiviral vectors. Additionally, there is a Clinical Coordinating Center to assist both investigators and the NHLBI in all aspects of the program. The Science Moving Towards Research Translation and Therapy (SMARTT) Program The purpose of the SMARTT program is to assist translation to the clinic of novel synthetic, natural, or biologic therapeutic interventions arising in the scientific community for the treatment of heart, lung, and blood diseases. SMARTT will provide resources in the areas of preclinical and clinical-grade production and testing for biologics, nonbiologics and small molecules in accordance with Good Manufacturing Practices (GMP), and in the conduct of pharmacology/toxicology studies. In addition, SMARTT will provide regulatory assistance to investigators wishing to conduct early stage clinical trials. Ying Huang, PhD Communication with FDA/CBER/OCTGT (Office of Cellular, Tissue and Gene Therapies) Open Microphone Question and Answer Period This talk will introduce the audience to the pathways that are currently available in CBER/OCTGT to facilitate communication between CBER and the sponsor of an investigational cellular or gene therapy product to enable the conduct of safe clinical trials. This talk will briefly touch upon the various disciplines involved in the development program of these novel products, with a focus towards supporting translation from bench to bedside.

Open Microphone Question and Answer Period Lunch Break 12:30 pm – 2:00 pm Meet-the-Investigator Lunch 220

12:30 pm - 1:45 pm Room: Harding

Musculo-Skeletal Speakers Jeffrey Chamberlain, PhD & Paula Clemens, MD Meet-the-Investigator Lunch 221

12:30 pm - 1:45 pm Room: Hoover

Cancer Stem Cells Speakers Timothy P. Cripe, MD, PhD & Samuel D. Rabkin, PhD

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Program Schedule Oral Abstract Session 230

2:00 pm - 4:00 pm Room: Marriott Ballroom Salons 2 & 3

AAV Vectors: Vector Trafficking Co-Chairs David V. Schaffer, PhD & Xiao Xiao, PhD (Abstracts 1-8; see page 86) Oral Abstract Session 231

2:00 pm - 4:00 pm Room: Maryland Suite Co-Chairs Perry B. Hackett, PhD & Matthew H. Porteus, MD, PhD (Abstracts 9-16; see page 86) Oral Abstract Session 232

2:00 pm - 4:00 pm Room: Wilson ABC

Neurologic & Ophthalmic Gene & Cell Therapy Co-Chairs Michael Kaplitt, MD, PhD & Deborah J. Watson, PhD (Abstracts 17-24; see page 87) Oral Abstract Session 233

2:00 pm - 4:00 pm Room: Thurgood Marshall W

Cancer – Immunotherapy T Cell Studies Co-Chairs Chiara Bonini, MD & Dean A. Lee, MD, PhD (Abstracts 25-32; see pages 87-88) Oral Abstract Session 234

2:00 pm - 4:00 pm Room: Marriott Ballroom Salon 1

Cancer-Oncolytic Viruses I Co-Chairs Winald Gerritsen, MD PhD & David H. Kirn, MD (Abstracts 33-40; see page 88)

Thursday, May 20th

DNA Vectorology & Gene Targeting

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Program Schedule Oral Abstract Session 235

2:00 pm - 4:00 pm Room: Virginia Suite

Immunologic & Host Responses in Gene & Cell Therapy Co-Chairs Evanthia Galanis, MD, DSc & Maciej S. Lesniak, MD, FACS (Abstracts 41-48; see pages 88-89) Oral Abstract Session 236

Thursday, May 20th

2:00 pm - 4:00 pm Room: Delaware Suite

Oligonucleotide & RNAi Therapeutics I Co-Chairs Dieter C. Gruenert, PhD & Faye A. Rogers, PhD (Abstracts 49-56; see page 89) Outstanding New Investigator Symposium 240

4:30 pm - 6:15 pm Room: Marriott Ballroom Salons 1, 2 & 3

Outstanding New Investigator Symposium Chair John J. Rossi, PhD Speakers

Alessandra Biffi, MD Rendering Hematopoietic Stem Cell Transplantation Efficacious for the Treatment of Lysosomal Disorders Transplantation of hematopoietic stem cells (HSC) from healthy donors has been employed in Lysosomal Storage Disorders (LSD) for delivering a functional hydrolase to the affected tissues. However, likely due to the slow kinetics of infiltration of the nervous system, the efficacy of the transplant is poor in LSD with severe neurologic manifestations. The limited efficacy of the allogeneic procedure may be implemented by: i) understanding the mechanisms of myeloid infiltration into affected tissues, and in particular into the brain, in order to favour and accelerate this phenomenon; ii) rendering the transplanted HSC and or their progeny more effective than normal donors’ cells in enzyme delivery by means of gene transfer to enhance the metabolic correction of the affected tissue. We have addressed these issues attempting to develop an effective and safe therapeutic approach for different LSD with CNS involvement, for one of which we recently started clinical testing.

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Program Schedule

Bakhos A. Tannous, PhD Ex-Vivo Monitoring of In Vivo Gene and Cell Therapy Secreted reporters are useful tools for the detection and non-invasive monitoring of different biological processes in experimental models. We have characterized the naturally secreted Gaussia luciferase (Gluc) as a highly sensitive reporter for quantitative assessment of cells in vivo by measuring its activity in few microliters of blood. The Gluc blood level is linear with respect to cell number and therefore is a sensitive tool for monitoring tumor growth and response to therapy, gene transfer, viral infection and replication, transcription factor activation, as well as circulating cells survival and proliferation. We also applied the Gluc assay to monitor the effect of novel gene therapy based on the constitutively active mutant human brain sodium channel as well as tumor-specific nuclear factor kappa B inducible system driving the expression of a suicidal gene. The Gluc blood assay complements in vivo bioluminescence imaging which has the ability to localize the signal and provides a multifaceted assessment of biological processes in experimental disease and therapy models.

Charles P. Venditti, MD, PhD Gene Therapy for Methylmalonic Acidemia (MMA) The hereditary methylmalonic acidemias are inborn errors of metabolism and are excellent candidates for gene and cell therapy. We have generated new insights from mouse models and patient investigations to instruct successful pre-clinical experiments. To facilitate the implementation of clinical gene therapy for MMA, we have developed and validated a sensitive isotope ratio mass spectrometry technique to non-invasively monitor whole-body metabolic effects after gene delivery.

Thursday, May 20th

Kevin V. Morris, PhD Utilizing the Endogenous Long Non-Coding RNA Pathway in Human Cells to Transcriptionally Modulate Gene Expression Observations over the last few years have demonstrated that non-coding RNAs can transcriptionally modulate gene expression in human cells. Recent data has inferred long non-coding RNAs as endogenous effector molecules regulating gene transcription in human cells. Importantly, this non-coding RNA based mechanism can be taken advantage of to either transcriptionally silence gene expression in a specific long-term manner or activate gene transcription by the targeted degradation of gene specific regulatory long antisense non-coding RNAs.

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Program Schedule Exhibit Hall Open & Networking Reception 6:15 pm – 8:15 pm Room: Exhibit Hall C Poster Session I

Thursday, May 20th

6:15 pm – 8:15 pm Room: Exhibit Hall B South, C

AAV Vectors I (Abstracts 57 through 72; see pages 89-90) Adenovirus and Other DNA Virus Vectors I (Abstracts 73 through 86; see pages 90-91) Targeting Gene Modification and Integration (Abstracts 87 through 99; see pages 91-92) Physical Methods of Delivery (Abstracts 100 through 116; see page 92) Chemical and Molecular Conjugates I (Abstracts 117 through 132; see page 93) Genetic and Metabolic Diseases Gene & Cell Therapy I (Abstracts 133 through 147; see pages 93-94) Musculo-skeletal Gene & Cell Therapy I (Abstracts 148 through 160; see pages 94-95) Infectious Diseases and Vaccines: Vaccine Related Gene Transfer Research (Abstracts 161 through 174; see pages 95-96) Cancer – Immunotherapy I (Abstracts 175 through 190; see pages 96-97) Cancer-Oncolytic Viruses I (Abstracts 192 through 204; Abstract 191 moved to Oral Session 413; see pages 97-98) Cancer-Targeted Gene & Cell Therapy I (Abstracts 205 through 219; see pages 98-99) Hematologic and Immunologic Gene & Cell Therapy I (Abstracts 220 through 233 and Abstract 869 which has been moved from Poster Session III; see page 99) Gene Regulation (Abstracts 234 through 244; see page 100) Oligonucleotide & RNAi Therapeutics I (Abstracts 245 through 259; see pages 100-101) Stem Cell Biology (Abstracts 260 through 273; see pages 101-102) Cell Processing and Vector Production I (Abstracts 274 through 282; see pages 102) Late Breaking Science I (Abstracts 922 through 932; Abstract 926 withdrawn; see pages 102-103)

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Program Schedule Foundation Symposium 250

7:00 pm - 10:00 pm Room: Thurgood Marshall West

Expanding Frontiers in Spinal Muscular Atrophy Gene Therapy Chair Christian L. Lorson, PhD Speakers Christian L. Lorson, PhD

Introduction: Perspectives on SMA Therapeutics

Marco Passini, PhD Gene and Antisense Therapies in SMA Mice Brian K. Kaspar, PhD Rescue of a Spinal Muscular Atrophy Mouse Model by Early Postnatal Delivery of SMN with scAAV9 and Clinical Development Plans Petra Kaufman, MD SMA Clinical Trials – NINDS Perspective

Foundation Symposium 250 is supported by the following ASGCT partners:

Thursday, May 20th

C. Frank Bennett, PhD RNA Targeting Therapeutics for the Treatment of Motor Neuron Diseases

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Program Schedule Friday, May 21 Plenary Abstract Session 300

8:00 am - 10:00 am Room: Marriott Ballroom Salons 1, 2 & 3

Top Oral Abstracts Plenary Session

Friday, May 21st

Co-Chairs Stanton L. Gerson, MD & John J. Rossi, PhD Speakers Mervin C. Yoder, MD Introduces Eirini P. Papapetrou, MD, PhD

Therapeutic Transgene Expression in Patient-Specific iPS Cells after Selection of “Safe Harbors” for Lentiviral Vector Integration James Ellis, PhD Introduces Axel Schambach, MD PhD A Novel Lentiviral Expression System Reveals Patchwork Colony Formation during Reprogramming to Induced Pluripotent Stem Cells Dieter C. Gruenert, PhD Introduces Erica B. Schleifman, BS Targeted Genomic Modification and Inactivation of the CCR5 Gene Using PNA-Induced Homologous Recombination Matthew H. Porteus, MD, PhD Introduces Richard Gabriel, Dipl.-Biol. Lentiviral Microarrays for High-Throughput and Real-Time Monitoring of Gene Expression Dynamics Bruce Sullenger, PhD Introduces Jiehua Zhou, PhD Systemic Administration of Sendrimer- and Aptamer-siRNA Conjugates Efficiently Suppresses HIV-1 Infection Chiara Bonini, MD Introduces Antonio Di Stasi, MD CASPALLO: Phase I Clinical Trial of Allodepleted T Cells Transduced with Inducible Caspase 9 Suicide Gene after Haploidentical Stem Cell Transplantation Break 10:00 am – 10:30 am Scientific Symposium 310

10:30 am - 12:30 pm Room: Virginia Suite

Personalized Cell and Gene Therapy Chair John J. Nemunaitis, MD Speakers John J. Nemunaitis, MD

Success of Gene Therapy - A Personalized and Personal Approach Clinical use of molecular science is coming of age! Numerous targeted therapies, many related to gene based technology, in particular involving RNA interference platforms, are undergoing clinical track development. Mechanisms for cancer specific plasmid delivery are unfolding in the clinical arena. Furthermore, gene based vaccines are showing improved promise. These dynamics combined with more efficient and economic quantitative proteogenomic technology development will enable a personalized approach to cancer therapy. Results of personalized gene and cell based therapies will be discussed.

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Program Schedule Lilia Bi, PhD Recovery and Reduction Costs for Cell-based Gene Therapy Products This presentation will discuss the recovery and reduction costs for cell-based gene therapy products from following aspects: What is the purpose of cost recovery and where does it apply? Cost recovery regulations and rules What can be charged for under cost recovery? Process for requesting cost recovery and what information sponsors need to provide Potential cost reduction by following the step-wise approach to implementing cGMP’s and product characterization Carl H. June, MD Genetically Engineered T Cells: Can We Afford Not To Do This? A key concern with adoptive cell transfer therapy is a financial issue related to the potential costs of a lymphocyte infusions.  Factors that relate to the eventual pricing of a personalized therapy consisting of autologous T cells are numerous and include issues such as the magnitude of efficacy, durability of responses, safety and tolerability. In this regard, it is instructive to consider allogeneic stem cell transplantation procedures that have potent efficacy and considerable toxicity, and whether adoptive T cell transfer therapy could supplant and extend this procedure.  Jeffrey Schlom, PhD Recombinant Cancer Vaccines as Monotherapy and in Combination Therapy Recombinant poxviral vaccines have been developed that contain the transgenes for one or more tumor associated antigens and three T-cell costimulation molecules (TRICOM). Preclinical studies have shown that these vaccines have the ability to break tolerance and induce therapeutic activity in several models. Randomized clinical trials in patients with prostate cancer have shown statistical improvements in survival. Preclinical studies have shown that radiation and or chemotherapy can alter the phenotype of tumor cells and render them more susceptible to T-cell lysis; combination therapy studies with vaccine are ongoing. Recently results have shown that molecules involved in the Epithelial to Mesenchymal Transition (EMT) metastatic process can be targeted by vaccine therapy. Scientific Symposium 311

Stem Cells and Malignancy Chair Evan Y. Snyder, MD, PhD, FAAP Speakers Inder M. Verma, PhD

Cancer Stem Cells: Lessons from Glioblastomas We have generated novel mouse models of human gliomas using lentiviral vectors containing oncogenes or shRNA to suppressor genes.  The resulting tumors had stem cell properties.  We will discuss the implications of these results. Roger J. Packer, MD Childhood Brain Tumors: Opportunities for Biologic-Based Therapies Brain tumors are the most common solid malignancy of childhood and are the leading cancer related cause of morbidity and mortality in the pediatric-aged patients. Progress has been made in the management of childhood brain tumors, but lack a clear understanding of the molecular pathogenesis of these tumors and ways to exploit this molecular pathogenesis for treatment has slowed progress. Over the past decade, there have been tremendous advances in the understanding of the molecular underpinnings of a variety of different childhood brain tumors including the most common malignant brain tumor, the medulloblastoma, and the most common benign tumor, the low grade-glioma. Molecular targets have been identified and a host of biologic agents including antiangiogenesis drugs, tyrosine kinase growth inhibitors, and agents interfering with intracellular signaling are in clinical trials. To date, their use has not been associated with improvements in survival but there is great enthusiasm in continuing their study with the hope of developing more effective and safer therapies.

Friday, May 21st

10:30 am - 12:30 pm Room: Marriott Ballroom Salon 1

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Program Schedule Evan Y. Snyder, MD, PhD, FAAP “Lineage Mapping” The Development of a Brain Tumor Carl Bart Rountree, MD The Role of Stem Cells and Cancer Stem Cells in Liver Cancer Hepatocellular carcinoma (HCC) ranks as the third most frequent cause of cancer-related mortality in the world; and over the last four decades, traditional cytotoxic chemotherapy has failed to improve patient survival in HCC. Typically, patients develop HCC after many years of chronic liver injury, during which time, there is a significant expansion of the liver stem cell pool. Increasing evidence indicates that liver cancer stem cells (CSC) are the origin of a subset of HCC, and patients with a CSC phenotype HCC have a significantly worse prognosis. Additionally, using stem cell surface markers such as CD133, purified liver CSC populations have been identified within human HCC cell lines and mouse models that mimic human chronic liver injury and HCC development. These liver CSCs are resistant to traditional chemotherapy agents and ligand-receptor mediated apoptosis, form tumors at very limited dilution, and represent a critical therapeutic target in future HCC treatments. Scientific Symposium 312

10:30 am - 12:30 pm Room: Thurgood Marshall West

Current Status of Genetic Vaccines Chair Stephen Gottschalk, MD Speakers Si-Yi Chen, MD, PhD

Friday, May 21st

DC-based Tumor Vaccine by Combining SOCS1 Silencing and TLR Signaling Michael A. Barry, PhD Gene-based Vaccines Against Mucosal Pathogens The vast majority of pathogens enter the body at mucosal surface. Yet, most vaccine strategies are delivered into the systemic immune system where induced immunological memory may not efficiently “cross-over” to mucosal surfaces to provide barrier protection against these pathogens. To maximize protection, we will discuss comparisons of replication-competent and replication-defective first generation and helper-dependent adenoviral vaccines for their utility in systemic and mucosal infections by SHIV, influenza, and methicillin-resistant Staphylococcus Aureus (MRSA). We will also discuss “stealth” strategies during vaccination to evade pre-existing and vector-induced immunity against adenovirus including serotype-switching and PEGylation. Sattva S. Neelapu, MD Targeting Chemokine Receptors with Fusion DNA Vaccines Therapeutic vaccination with patient-specific tumor-derived idiotype protein conjugated to a carrier molecule, keyhole limpet hemocyanin and administered with granulocyte-monocyte colony-stimulating factor as an adjuvant was shown to induce tumor-specific immune responses and molecular remissions in follicular lymphoma patients in phase II clinical trials. Results from a recently completed randomized controlled double-blind phase III trial suggested that administration of idiotype protein vaccine in the setting of minimal residual disease improves disease-free survival in patients with follicular lymphoma as compared with control. However, a major limitation of this strategy is the requirement to generate a custom-made protein vaccine for each patient, by a process that is expensive, laborious, and time-consuming. DNA vaccines may be produced more rapidly and provide an alternative to protein vaccines. Strategies to enhance the efficacy of DNA vaccines by fusing tumor antigens to chemokines and using novel adjuvants will be discussed. Stephen Gottschalk, MD Adenoviral Vaccines for Enhancing T-cell Therapies for Cancer Although the benefits of T-cell therapy for cancer can be increased by prior lymphodpeletion of the host, this process has usually required chemotherapy or radiation. Vaccination with tumor associated antigens to which the transferred T cells respond should be a less toxic means of promoting antitumor activity, but to date this has proved ineffective, favoring instead the transition to a non-cytotoxic immune response. We have developed a new adenoviral based vaccine that contains antigen, a toll-like receptor 5 activator and an inhibitor of the antigen presenting attenuator A20. Administration of this vaccine prior to T-cell transfer induces a strong Th1-polarizing environment leading to potent antitumor effects even in the absence of lymphodepletion and even when vaccination or T-cell transfer alone are ineffectual. Such Th1polarizing vaccines may be of value when enhancement and maintenance of a Th1 response is desirable.

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Program Schedule Scientific Symposium 313

10:30 am - 12:30 pm Room: Maryland Suite

Precise Genetic and Post-transcriptional Modification of Stem Cells Chair Dieter C. Gruenert, PhD Speakers Peter M. Glazer, MD, PhD

Targeted Gene Modification in Hematopoietic Stem Cells via Triple Helix Formation Triple helix-forming peptide nucleic acids (PNAs) can bind to polypurine regions in DNA in a sequence-specific manner. The resulting triplexes constitute an altered DNA structure that can stimulate DNA repair and recombination in a site-specific manner in human cells. We have found that transfection of human hematopoietic stem cells with triplex-forming PNAs plus short single-stranded donor DNAs can produce targeted modification of disease related genes. Site-directed modification of two key human genes has been achieved: (1) the human beta-globin gene, mutations in which cause thalassemia and sickle cell anemia; and (2) the CCR5 gene, which encodes a co-receptor for HIV-1 entry into human cells. Triplex-modified hematopoietic stem cells have been transplanted into immune deficient mice, with full engraftment, normal patterns of differentiation, and persistence of the targeted gene modification for up to four months. Linzhao Cheng, PhD Gene Targeting in Human Pluripotent Stem Cells Human induced pluripotent stem (iPS) cells reprogrammed from adult somatic cells resemble embryonic stem (ES) cells that can proliferate indefinitely in culture while maintain their full differentiation potential (pluripotency), therefore providing an unlimited resource for disease research and therapy. These self-renewable stem cells also make it feasible to achieve gene targeting (to correct or create DNA mutation) in the genome of normal human cells by homologous recombination (HR) that is natural but inefficient process for genome repair. To enhance HR-mediated gene targeting efficiency we also incorporate the strategy utilizing zinc finger nuclease (ZFN), which is designed as a fusion protein with multiple zinc-finger DNA binding and the FokI endonuclease domains. Upon dimer formation, two ZFNs (co-delivered with a donor DNA template) can create a sequence-specific DNA double strand break to stimulate HR near the cut site. We found that ZFNs enhanced HR by >100-1,000 fold in either creating or correcting a mutation in human iPS and ES cells.

Robert Blelloch, MD, PhD MicroRNA Regulation of Embryonic Stem Cell Self-Renewal and Differentiation

Friday, May 21st

Dieter C. Gruenert, PhD Modification of the CF and Sickle Cell Anemia iPS cells by SFHR Induced pluripotent stem (iPS) cells have the potential of significantly advancing the development of stem cell-based therapies, especially as they pertain to inherited diseases. The potential to generate autologous pluripotent stem cells to repair and regenerate tissues and organs damaged by disease pathologies is a cornerstone of patient-specific therapy. Cystic fibrosis (CF), the most common inherited disease in the Caucasian population, and sickle cell anemia (SCA), the most prevalent hemoglobinopathy commonly found in individuals of African, Middle Eastern, Mediterranean, and Southeast Asian decent, have significant multiorgan damage that will benefit from cellular repair. In this context, somatic cells from a CF patient with a ∆F508/∆F508 genotype and a SCA patient have been reprogrammed, characterized, and corrected by small fragment homologous replacement (SFHR).

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Program Schedule Scientific Symposium 314

10:30 am - 12:30 pm Room: Delaware Suite

New Physical Gene Delivery System and Vectorology Chair Shulin Li, PhD Speakers Christian Plank, PhD

Nanomagnetic Nucleic Acid Delivery Physical force has been used with great success to enable, enhance or localize nucleic acid delivery. In magnetofection, which is one such method, vectors for nucleic acid delivery are associated with magnetic nanoparticles to accumulate or retain them at target sites with gradient magnetic fields. This concept of magnetic drug targeting enhances the efficiency of the delivery process over several orders of magnitude. This we have exploited to generate an integrated method of magnetic cell separation and genetic modification, to boost the efficacy of oncolytic adenovirus and for establishing a tumor vaccine to treat feline fibrosarcoma. In other recent approaches we focus on extending the concept of magnetofection with thermo-sensitive magnetic liposomes, magnetic microbubbles and magnetic aerosols for magnetically guided drug delivery.

Friday, May 21st

Declan M. Soden, PhD Endoscopic Electroporation: Targeted Gene Delivery to Intraluminal Tissues The minimally invasive control of primary tumours is a critically important part in the treatment process as it relieves symptoms, forestalls complications, facilitates responsiveness to systemic therapies and is often curative. We have developed a device, which allows for an endoscopic cancer treatment, specifically targeting colorectal, gastric and oesophageal cancers by applying a brief electric pulse to the tumour. This greatly enhances tissue permeability maximizing the local/tumor absorption of therapeutic agents (drugs or DNA). Absorption occurs only in the area that has been treated with the electrical field and therefore is targeted to the tumor, leaving surrounding healthy tissues unaffected. Our research has examined the efficacy of the device for targeted gene delivery to intraluminal tissues in large animals. We have compared the transfection efficacy of a standard CMV driven reporter gene (b-galactosidase) in both murine and porcine tissues. In addition we have examined the efficacy of a DNA vector coding for a viral RNA polymerase encoded from the Semliki Forest Virus in comparison to the CMV driven vector.

The treatment of spontaneous canine colorectal cancers has also been conducted using the device to facilitate local drug (bleomycin) absorption after tumor tissue electroporation. The results have demonstrated the treatment to be effective and safe with complete tumor ablation noted in the two inoperable cases treated to date. All procedures were conducted as a simple colonoscopy type procedure with no adverse side effects recorded. A phase I clinical study with the device is due to commence early 2010 to treat patients with inoperable rectal cancer and electrochemotherapy. Kate E. Broderick, PhD Dermal Electroporation Devices for Prophylactic DNA Vaccine Delivery During the presentation, Dr. Broderick will discuss novel prototype dermal devices developed by Inovio and the pre-clinical Immunogenicity data generated from these devices in a number of animal models. Laurence JN Cooper, MD, PhD Non-viral Gene Transfer into Human T Cells Non-viral approaches can be used to genetically modify T cells for therapy. We have combined electroporation with the Sleeping Beauty (SB) transposase/transposon DNA plasmid system to introduce desired transgenes. By improving the efficiency of electroporation with designer devices and improving the efficiency of integration by adapting the SB system for human translation, we seek to implement physical gene transfer systems for clinical trials.  

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Program Schedule Scientific Symposium 315

10:30 am - 12:30 pm Room: Marriott Ballroom Salons 2 & 3

Genetic Modification of Stem Cells for Therapeutic Applications Chair Frederic D. Bushman, PhD Speakers Christof von Kalle, MD PhD

The Clonal Distribution of Insertion Site Repertoires Retroviral vectors have been successfully used for the correction of inherited immune diseases in clinical trials. However, qualitative and quantitative in vivo monitoring of gene modified hematopoiesis in several clinical gene therapy trials revealed that vector induced side effects, ranging from subtle and not clinically obvious effects to clonal dominance and, rarely, leukemogenesis, have to be considered. Clonality data on oncoretroviral and lentiviral clinical gene therapy trials (i.e. CGD, SCID, WAS, ALD) show that integration site distribution is non-random and may influence cellular gene expression, thus inducing changes in the clonality of the hematopoietic repopulation. Incorporation of next generation sequencing technologies substantially improve in depth analyses and monitoring of individual and global clonal contribution in a hitherto unexpected resolution. Prospective monitoring of vector integration sites in clinical gene therapy studies is feasible, can detect side effects of gene therapy at the clonal level, and may help to gain new insights into the basic mechanisms leading to specific clonal behaviour in vivo.  Genome wide insertion studies will allow to improve the safety and performance of viral and non-viral integrating vector systems.

Frederic D. Bushman, PhD HIV DNA Integration: Mechanism and Consequences The lecture will cover integration by HIV and HIV-based vectors.  Recent results from gene therapy trials will be presented.  The lecture will also cover development of improved new methods for integration site isolation and analysis. Ko Mitani, PhD Chromosomal Manipulation of Human Pluripotent Stem Cells using Adenoviral Vectors In order to fully utilize human induced pluripotent stem (iPS) cells and embryonic stem (ES) cells for basic research and clinical applications, it is essential to develop efficient methods for homologous recombination (HR) in these cells. We have shown that, by delivering gene-targeting cassettes using helper-dependent adenoviral vectors (HDAdVs) into human ES/iPS cells, gene knock-in/-out has been efficiently achieved at both transcriptionally active and inactive loci. On average, one out of 2 - 20 x 105 infected cells was homologous recombinant. The efficiencies of HR per chromosomal integration were 10 - 50% (30 - 90% with negative selection), which were ~100-fold higher than those obtained by other DNA delivery methods. These findings suggest that HDAdV-mediated HR is not only a highly efficient strategy for chromosomal manipulation of pluripotent stem cells but also a viable option for ex vivo gene repair therapy using iPS cells derived from patients of inherited diseases, which would overcome the risk of insertional mutagenesis.

Lunch Break 12:30 pm – 2:00 pm

Friday, May 21st

Matthew H. Porteus, MD, PhD Gene Targeting with Zinc Finger Nucleases in Primary Cells Gene targeting by homologous recombination has the potential to be both a safer and more efficacious approach to gene therapy.  Therapeutically relevant rates of gene targeting have been achieved by using zinc finger nucleases in a variety of tissue culture cell lines and some primary human cells. We now demonstrate that therapeutically relevant frequencies of gene targeting by homologous recombination can be achieved in primary cells and that these modified cells can be successfully transplanted back into a recipient.  The demonstration that primary cells can be isolated, precisely modified ex vivo and then transplanted back into a recipient mimics the strategy we are working towards in humans as a cure for patients with monogenic diseases.

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Program Schedule Oral Abstract Session 320

2:00 pm - 4:00 pm Room: Marriott Ballroom Salons 2 & 3

Clinical Gene & Cell Therapy Co-Chairs Guangping Gao, PhD & Matthew H. Porteus, MD, PhD (Abstracts 289-296; see pages 104-105) Oral Abstract Session 321

2:00 pm - 4:00 pm Room: Virginia Suite

RNA Virus Vectors Co-Chairs Fabio Candotti, MD & Robert G. Hawley, PhD (Abstracts 297-304; see page 105) Oral Abstract Session 322

2:00 pm - 4:00 pm Room: Marriott Ballroom Salon 1

AAV Vectors: Vector Biology

Friday, May 21st

Co-Chairs Douglas M. McCarty, PhD & Matthew D. Weitzman, PhD (Abstracts 305-312; see pages 105-106) Oral Abstract Session 323

2:00 pm - 4:00 pm Room: Delaware Suite

Gene Therapy for Lysosomal Storage Diseases and Other Genetic Disorders Co-Chairs Cary Harding, MD & Chester B. Whitley, PhD MD (Abstracts 313-320; see page 106) Oral Abstract Session 324

2:00 pm - 4:00 pm Room: Wilson ABC

Physical Methods of Delivery Co-Chairs Jeffrey Hughes, PharmD, PhD & Nancy Smyth Templeton, PhD (Abstracts 321-328; see pages 106-107)

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Program Schedule Oral Abstract Session 325

2:00 pm - 4:00 pm Room: Thurgood Marshall W

Cancer-Targeted Gene & Cell Therapy Co-Chairs Frank Marini, PhD & Richard G. Vile, PhD (Abstracts 329-336; see page 107) Oral Abstract Session 326

2:00 pm - 4:00 pm Room: Maryland Suite

Hematologic/Immunologic: Basic Co-Chairs David Klatzmann, MD, PhD & John F. Tisdale, MD (Abstracts 337-344; see pages 107-108)

Break 4:00 pm – 4:30 pm

Friday, May 21st

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Program Schedule Presidential Symposium 330

4:30 pm - 6:30 pm Room: Marriott Ballroom Salons 1, 2 & 3

Presidential Symposium Chair

Kenneth Cornetta, MD

Friday, May 21st

Speakers

John J. Rossi, PhD Introduces Katherine A. High, MD

Katherine A. High, MD Outstanding Achievement Award Lecture

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Program Schedule Presidential Symposium 330 — continued

Jean Bennett, MD, PhD An Aye for Gene Therapy: Lessons from a Clinical Trial for Congenital Blindness Gene therapy has the potential to reverse disease or prevent further deterioration of vision in patients with incurable inherited retinal degeneration. The recent demonstration of safe and stable recovery of retinal/visual function in 12 children and adults with Leber’s congenital amaurosis (LCA) due to RPE65 mutations in a trial being carried out at The Children’s Hospital of Philadelphia (CHOP), and encouraging results from two other clinical trials for LCA-RPE65 provide great hope for people with other more common blinding diseases. This presentation will describe the latest safety and efficacy results in the LCA-RPE65 clinical trial being held at CHOP. This study is now >2.5 years past initiation. In addition, it will describe some of the challenges presented by the nature of the targeted disease itself, hurdles that have been navigated in order to conduct the study, and issues of importance for eventual approval of gene augmentation as a therapy for LCA and other blinding diseases.

*This presentation involves contributions from the following additional individuals (listed alphabetically): Carmen Acerra, Defne Amado, Alberto Auricchio, Sandro Banfi, Jeannette Bennicelli, Erin Bote, Yifeng Chen, Daniel Chung, Ann Fulton, Jeffrey Golden, Rebecca Grant, Bernd Hauck, Daniel Hui, Katherine A. High, Bart LeRoy, Arkady Lyubarsky, Albert M. Maguire, Maureen G. Maguire, Kathleen Marshall, Federico Mingozzi, Jessica Morgan, Kristina Narfstrom, Stephen Orlin, Eric A. Pierce, Settimio Rossi, Kenneth S. Shindler, Francesca Simonelli, Enrico M. Surace, Edwin Stone, Junwei Sun, Nasreen Syed, Francesco Testa, Nicholas J. Volpe, Jennifer Wellman, J. Fraser Wright, Gui-shuang Ying, Olga Zelenaia, Xiaosong (Sonia) Zhu

Friday, May 21st

David A. Williams, MD New Approaches to Gene Therapy for Fanconi Anemia FA is a rare recessive syndrome characterized by bone marrow failure, congenital anomalies and a predisposition to malignancy. FA cells have a defect in DNA repair that leads to increased spontaneous chromosomal breakage. Transgenic expression of FA genes in vitro corrects the increased chromosomal breakage and other cellular phenotypes of the disease. In a recent gene therapy trial, we have demonstrated that gene transfer per se is no longer a limitation to the effectiveness of gene therapy in this disease. Rather our clinical studies show that even early in the disease process there are significant deficiencies in the number and/or function of hematopoietic stem cells (HSCs) that can be collected and utilized as targets in gene transfer. One approach that could address this deficiency is the derivation of HSC from induced pluripotent stem (iPS) cells reprogrammed from non-blood somatic tissues. Important advantages of iPS cells for application of gene therapy are the apparent unlimited proliferative capacity of such cells (allowing production of large numbers of derived HSC) and the ability to clone such cells allowing both single cell correction of genetic mutations and prospective molecular characterization of manipulated cells prior to therapeutic applications.

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Program Schedule Exhibit Hall Open & Networking Reception 6:30 pm – 8:30 pm Room: Exhibit Hall C Poster Session II

6:30 pm – 8:30 pm Room: Exhibit Hall B South, C

RNA Virus Vectors I (Abstracts 345 through 361; see pages 108-109) AAV Vectors II (Abstracts 362 through 377; see pages 109-110) Adenovirus and Other DNA Virus Vectors II (Abstracts 378 through 391; see page 110) Chemical and Molecular Conjugates II (Abstracts 392 through 407; see pages 110-111) Genetic and Metabolic Diseases Gene & Cell Therapy II (Abstracts 408 through 422 and Abstract 764 which has been moved from Poster Session III; see pages 111-112) Cardiovascular Gene & Cell Therapy (Abstracts 423 through 440; see pages 112-113)

Friday, May 21st

Ophthalmic Disorders and Technological Advances (Abstracts 441 through 453; see pages 113-114) Musculo-skeletal Gene & Cell Therapy II (Abstracts 454 through 468; see pages 114-115) Cancer – Immunotherapy II (Abstracts 469 through 487; see pages 115-116) Cancer-Oncolytic Viruses II (Abstracts 488 through 506; see pages 116-117) Cancer-Targeted Gene & Cell Therapy II (Abstracts 507 through 520; Abstract 519 withdrawn; see page 117) Hematologic and Immunologic Gene & Cell Therapy II (Abstracts 521 through 534; see page 118) Immunologic & Host Responses in Gene & Cell Therapy (Abstracts 535 through 554; Abstract 541 withdrawn; see pages 118-119) Oligonucleotide & RNAi Therapeutics II (Abstracts 555 through 569; see pages 119-120) Cell Processing and Vector Production II (Abstracts 570 through 579; see pages 120-121) Late Breaking Science II (Abstracts 933 through 937; see page 121)

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Program Schedule Foundation Symposium 340

7:00 pm - 10:00 pm Room: Marriott Ballroom Salons 2 & 3

Progress and Prospects for Gene Therapy for CGD and other Primary Immunodeficiencies Speakers Mary C. Dinauer, MD, PhD

Gene Therapy for CGD - From Basic Discoveries to the Start of Clinical Trials Alessandro Aiuti, MD, PhD Conditioning Regimens for Haematopoietic Gene Therapy Harry L. Malech, MD Update on Gene Therapy Trials for CGD Manuel Grez, PhD The European Experience on GT for CGD Adrian J. Thrasher, MD, PhD Update on Clinical Trials for XC1-SCID, ADA and ALD Foundation Symposium 340 is supported by the following ASGCT partner:

Friday, May 21st

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Program Schedule Saturday, May 22 ASGCT Business Meeting 8:00 am - 8:30 am Room: Delaware Suite A light continental breakfast will be served at 7:45 am. Scientific Symposium 400

8:30 am - 10:30 am Room: Maryland Suite

Late Stage Industry Clinical Trials Co-Chairs Douglas J. Jolly, PhD & Thomas Tillett Speakers William W. Li, MD

Gene Transfer of FGF1 for Critical Limb Ischemia: A Phase III Clinical Trial Robert Sims, MD Progress in the Development of Sipuleucel-T for the Treatment of Men with Metastatic, Castrate Resistant Prostate Cancer Sipuleucel-T is an autologous cellular immunotherapy designed to elicit an antitumor response against prostate cancer. Sipuleucel-T is manufactured by incubating antigen presenting cell precursors with a fusion protein composed of prostatic acid phosphatase linked to GM-CSF. Sipuleucel-T is then reinfused into the patient and the process is repeated every 2 weeks for a total of 3 infusions. It has been evaluated in 3 randomized placebo-controlled Phase 3 studies in men with metastatic castrate-resistant prostate cancer, which have demonstrated clear evidence of prolongation of survival. The presentation will include an update on the regulatory status of sipuleucel-T current clinical trials, as well as planned future trials. Ryuichi Morishita, MD, PhD Development of HGF Gene Therapy Drug (Collategen) As HGF is a most potent mitogen to stimulate angiogenesis, a multi-center randomized, placebo-controlled, double blind study for critical limb ischemia (CLI) using HGF plasmid DNA was conducted, based on phase I/IIa open label study at Osaka University. Improvement rate was 70.4 % in HGF and 30.8% in placebo in Fontaine III/ Rutherford 4 patients (P=0.014). HGF gene therapy achieved a significantly higher improvement rate of ischemic ulcers compared with placebo (100% vs. 40%, P=0.018). No major safety problems were observed. Based on phase III data, NDA was submitted to Japan Regulatory Agency (MHWL) on 2008. In USA, phase III study using HGF gene therapy will be started in 2010, since phase III USA study obtained SPA from FDA.

Saturday, May 22nd

Sander van Deventer, MD, PhD Glybera for Lipoprotein Lipase Deficiency

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Program Schedule Scientific Symposium 401

8:30 am - 10:30 am Room: Virginia Suite

Local Delivery of Chemical Gene Therapy – Mechanisms, Quantitative Understanding and Applications of Biodegradable Carriers Chair Ernst Wagner, PhD Speakers Lonnie D. Shea, PhD

Localized Gene Delivery by Biomaterial Immobilization Biomaterials are combined with gene delivery to provide a structure that guides cell growth, while also inducing the expression of inductive factors that drives the cellular processes leading to functional tissues. We have investigated immobilization of gene therapy vectors to biomaterials as a means to maintain the vectors locally, co-localize the vectors to the surface for cell adhesion, and stabilize the vectors against degradation. The design principles we have developed for hydrogels and microporous scaffolds will be presented, as well as their use with in vitro tissue models and in vivo regeneration. These biomaterial scaffolds provide opportunities to enhance or control gene transfer relative to more traditional delivery methods (e.g., injection), and provide an enabling technology to molecularly dissect tissue formation or to promote functional tissue regeneration. Aliasger K. Salem, PhD Pulsatile Release of Biomolecules from Polydimethylsiloxane Chips Cytosine-phosphorothioate-guanine oligodeoxynucleotides (CpG ODN) is a recent class of immunostimulatory adjuvants that includes unmethylated CpG dinucleotide sequences similar to those commonly found in bacterial DNA. CpG ODN specifically triggers toll like receptor 9 (TLR9), which is found within phagoendosomes of antigen presenting cells (APCs) such as dendritic cells (DCs). CpG ODN triggers activation and maturation of DCs and helps to increase expression of antigens. CpG ODN can be used to induce polarized Th1 type immune responses. We have shown that antigens and CpG ODN must be co-delivered to generate the most potent therapeutic antigenspecific immune responses. We have developed a series of delivery vehicles to ensure co-delivery of antigens and CpG ODN to the same APCs and to significantly increase uptake by APCs. These strategies can result in antigen-specific immune responses that are 5 to 500-fold greater than administration of antigen alone. The approaches we have developed to achieve this include the utilization of conjugate molecules, multicomponent nanorods, biodegradable microparticles, cell-microparticle hybrids and finally polydimethylsiloxane based pulsatile release chips.

Daniel G. Anderson, PhD Combinatorial Development of Biomaterials for Gene Delivery High throughput, combinatorial approaches have revolutionized small molecule drug discovery.  Here we describe our work on high throughput methods for developing and characterizing biomaterials for genetic therapy and cell therapy.  We have developed automated methods allowing for rapid synthesis of 1000’s of biomaterials, as well as the testing of their chemical, material, and biological properties.  These methods have also been applied towards the development of new methods to control stem cell behavior, as well as vehicles for drug delivery.  In particular, these combinatorial libraries of different biomaterials have enabled new methods for non-viral gene therapy and siRNA delivery. 

Saturday, May 22nd

David M. Lynn, PhD A ‘Multilayered’ Approach to the Delivery of DNA: Exploiting the Structure of Polyelectrolyte Multilayers to Promote Surface-Mediated Transfection and Tunable Multi-Agent Delivery Methods for the layer-by-layer deposition of oppositely charged polymers on surfaces can be used to fabricate ultrathin multilayered films (or ‘polyelectrolyte multilayers’) using a broad range of natural and synthetic materials, including DNA. If the individual components of these assemblies are designed appropriately, these methods also provide opportunities to design thin films that provide control over the release of DNA and other agents from surfaces. This talk will describe recent work from our laboratory on the design of new polymers and the fabrication of thin films and coatings that provide spatial and temporal control over the release of DNA from surfaces in vitro and in vivo. These methods provide tunable control over the surface-mediated release of DNA over periods ranging from several hours to several days, weeks, or months, and can also be used to fabricate nanostructured films with hierarchical structures that permit control over the release of two or more DNA constructs with separate, distinct, and predictable release profiles (e.g., rapid release of one DNA construct followed by the slower, sustained release of a second DNA construct). With further development, these approaches could contribute to the design of thin films and coatings capable of delivering precise and well-defined quantities of multiple different DNA constructs (or combinations of DNA and other therapeutic agents) in a broad range of fundamental and applied contexts.

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Program Schedule Scientific Symposium 402

8:30 am - 10:30 am Room: Delaware Suite

Engineered T Cells in the Clinic: Emerging Clinical and Regulatory Issues Chair Jacqueline Corrigan-Curay, MD, JD Speakers Daniel M. Takefman, PhD

Gene Modified Cell Based Therapies: FDA Perspectives The purpose of this presentation is to provide FDA perspectives on gene modified cell based therapies. This presentation will outline some key regulatory considerations for the clinical use of patient specific gene therapies, such as engineered T-cells. The emphasis with be on manufacturing considerations as unique issues arise when manufacturing these types of products for clinical use. Joy Cavagnaro, PhD, DABT Limitations of Preclinical Studies for Predicting Safety of Uniquely Human Specific Therapies Carl H. June, MD Prolonged Engraftment and Decade Long Safety Record of Engineered T Cells Increasing data from pre-clinical experiments and pilot clinical trials illustrate the potential of engineered T cells for therapy of cancer, chronic infection and autoimmunity. A key issue facing the field has been limited persistence of engineered T cells in most trials. Our clinical data indicates that T cells engineered to express a chimeric antigen receptors can exhibit long term survival, and that in HIV infected patients, engineered T cells have a considerable safety record. Panel Helen E. Heslop, MD Michel Sadelain, MD, PhD Steven A. Rosenberg, MD, PhD Scientific Symposium 403

8:30 am - 10:30 am Room: Marriott Ballroom Salon 1

Mechanisms of Innate Immunity Influencing Gene Therapy

Saturday, May 22nd

Chair David B. Weiner, PhD Speakers Valder Arruda, MD, PhD

Gene Therapy for Tolerance Induction in Large Animal Models The formation of neutralizing antibodies (inhibitors) to clotting Factor VIII or FIX is a major safety concern on the development of novel therapies for hemophilia. Inhibitor formation renders the protein replacement ineffective resulting in both high morbidity and mortality. Therefore the prevention or eradication of inhibitors is of fundamental importance. To eliminate the presence of inhibitors in hemophilia, an immune tolerance induction strategy consisting of large amounts of FVIII/FIX injected on a daily basis for long periods of time (months to years) results in variable success rates. We will review gene-based strategies to prevent inhibitor formation in inhibitor-prone dogs. Notably, recent evidence demonstrates the possibility of the use of gene-based therapy for the eradication of preexisting inhibitors to FVIII from studies in hemophilia A dogs. Emerging evidence on the role of T regulatory cells in modulating immune responses to the therapeutic protein now endogenously expressed by gene therapy opens a novel potential application of gene therapy, not only to prevent, but also to eradicate inhibitors in hemophilia and other diseases.

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Program Schedule Yiping Yang, MD, PhD Innate Immunity in AAV-mediated Gene Therapy Recombinant adeno-associated viruses (AAVs) have been used widely for in vivo gene therapy. However, adaptive immune responses to AAV have posed a significant hurdle in clinical application of AAV vectors. Recent advances have suggested a crucial role for innate immunity in shaping adaptive immune responses. Here I will discuss how AAV activates the innate immune system and the potential implications for improving the outcome of AAV-mediated gene therapy. David B. Weiner, PhD High Immune Potency of Highly Optimized EP DNA Vaccines in Animal Models and Humans DNA vaccines are an important emerging vaccine technology, however the immune responses induced by past generations of DNA vaccines were poor in larger animals including nonhuman primates and importantly humans. We have recently utilized a combination of technologies for plasmid optimization, combined with enhanced EP delivery to dramatically improve the immune potency of this platform. These new EP systems also offer improved tolerability and effectiveness. We will present data in animal models and humans illustrating immune aspects of the technology for application to human infectious diseases. Michael T. Lotze, MD MicroRNAs in Immune Regulation-Opportunities for Cancer Gene Therapy Initially considered as means to regulate tissue differentiation and disordered in cancer, miRNAs [miR] have now been identified that are induced in response to hypoxia, oncogenic signaling, pathogens and tissue damage or injury. Each miR can regulate the translation of up to 100 separate mRNAs. Means to either express or knockdown miRNA expression [so-called antagomiRs] have been operationalized in murine models and represent exciting novel targets for modifying immunity. Scientific Symposium 404

8:30 am - 10:30 am Room: Wilson ABC

MicroRNA and siRNA Roles in Disease Progression and Development of Novel Therapeutic Targets Chair Maria G. Castro, PhD Speakers Anna M. Krichevsky, PhD

MicroRNAs as Targets for Glioma Therapy MicroRNA (miRNA) is a class of small RNA molecules involved in the regulation of expression of at least 30% of human genes and, therefore, it controls all principal cellular processes, including cell division and cell death. In my talk, I will describe our approach to reveal key miRNA regulators in human diseases and focus on miRNA functions in the development of human brain tumors. Our recent work presents proof-of-principles that specific inhibition of a single oncogenic miRNA could provide a novel therapeutic approach for physiological modulation of multiple proteins whose expression and activity is de-regulated in cancer. It also suggests a diagnostic potential for this novel class of molecules.

Sean E. Lawler, PhD MicroRNA Alterations and Functions in Glioblastoma MicroRNAs have recently emerged as important regulators of gene expression with potential therapeutic applications in many human diseases. We have identified extensive alterations in microRNA expression in the malignant brain tumor glioblastoma multiforme. We have shown that altered microRNAs play roles in proliferation, stem cell-like behavior, and glioma cell migration.  This talk will focus on the underlying mechanisms of microRNA action in glioblastoma, and their therapeutic potential.

Saturday, May 22nd

M. Maral Mouradian, MD MicroRNAs in Parkinson’s Disease and Other Neurodegenerative Disorders alpha-Synuclein is a key protein that accumulates in the brains of patients with Parkinson’s disease and related neurodegenerative disorders. Evidence from clinical genetic studies, animal models and cell biologic experiments indicate that elevated levels of this protein are toxic. This presentation will discuss down-regulation of alpha-synuclein expression by microRNA leading to neuroprotection.

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Program Schedule Al Charest, PhD Nanoparticle-Mediated Delivery of siRNA in a Pre-Clinical Mouse Model of Glioblastoma Multiforme Small interfering RNAs mediate cleavage of specific, complementary mRNA sequences and thus regulate gene expression. Not surprisingly, their use for treatment of diseases that are rooted in aberrant gene expression, such as cancer, has become a paradigm that has gained wide interest. We utilized dendrimer-conjugated magnetofluorescent nanoparticles to deliver anti-EGFR siRNA in vivo in an EGFR-driven transgenic model of glioblastoma. These nanoparticles were well tolerated in the CNS and demonstrated specific and significant suppression of EGFR expression by convection-enhanced delivery to the mouse brain. Our data demonstrate the feasibility of siRNA delivery in animals using a nanoplatform to efficiently knockdown target proteins. Scientific Symposium 405

8:30 am - 10:30 am Room: Marriott Ballroom Salons 2 & 3

Special Considerations with Using Viral Vectors for Gene Modification of Stem Cells Chair Philip D. Gregory, PhD Speakers Matthew Hirsch, PhD

The AAV Induced DNA Damage Response in Human Pluripotent Cells The ability to genetically engineer stem cells ex vivo, then re-introduce them into a host for therapeutic purposes is an attractive approach for a variety of human genetic diseases. Critical in this process is the ability to efficiently deliver transgenic DNA into the nucleus of stem cells in a manner that is well tolerated by the cell and without extensive host chromosome integration. A concern at the cellular level is the characterization of the cell type modified, and in particular, understanding the relationship between “stemness” and oncogenesis. For example, the DNA damage response of pluripotent human embryonic stem cells (hESCs), in general, differs from that of differentiated cells in that checkpoint functions are impaired and that these cells appear primed for rapid apoptosis. In this work, incidentally, we demonstrate that the adeno-associated virus (AAV) origin of replication, found ubiquitous in nature and currently used in human clinical trials for DNA delivery, induces a rapid, specific, and complete apoptotic response following transduction of non-differentiated hESCs. Cell cycle analysis by flow cytometry suggested that AAV-infected hESCs fail to induce a G1 checkpoint response and display an accumulation of cells in early S phase followed by apoptosis, suggesting that the AAV ITRs mimic stalled replication forks and/or perhaps uncapped telomeres. In contrast to reports of AAV-induced apoptosis in p53-deficient cancers, hESCs depleted of p53 survived significantly longer following AAV infection than the parental control. Collectively, these data demonstrate that the AAV origin of replication (present on all AAV vectors) induces a lethal signaling cascade during S-phase in hESCs and a subsequent p53-dependent apoptotic response. This unexpected phenomenon is the first report of AAV vector toxicity in a wild type human cell and is reminiscent of previous reports of toxicity in p53-deficient cancers cells following AAV transduction. Understanding such unique DNA damage responses in stem cells and cancers following DNA delivery is necessary for optimization of conditions for efficient genetic engineering and safety in cell replacement therapies.

Saturday, May 22nd

Philip D. Gregory, hD Stem Cell Modification with Zinc Finger Nucleases Frederic Paques, PhD Meganucleases and Viral Vectors: Alternative or Complementary Approaches Targeted approaches have emerged today as an alternative to current random insertion strategies for gene therapy. Meganucleases, the most specific natural endonucleases represent ideal tools for targeted genome editing, or “genome surgery”. Natural as well as engineered can be used to induce up to 20% of gene insertion into chosen human genes. The introduction of meganucleases into different cell types can be achieved by various means, including viral and non viral vectorization. Andrew M. Scharenberg, MD Gene Editing Using Lentiviral Expression of Homing Endonucleases Gene editing and targeted integration approaches to gene therapy offer the potential to avoid idiosyncratic side effects associated with the random integration of promoter/gene cassettes in traditional gene therapy. The Northwest Genome Engineering Consortium is exploring the development of lentiviral vector-mediated expression of homing endonucleases for gene editing and targeted integration in hematopoietic cells. Preliminary data on the efficacy of this approach in cultured and primary hematopoietic cell models will be presented, and challenges and future directions in the field will be discussed.

Break 10:30 am – 11:00 am

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Program Schedule Oral Abstract Session 410

11:00 am - 1:00 pm Room: Delaware Suite

Musculo-skeletal Gene & Cell Therapy Co-Chairs Jeffrey Chamberlain, PhD & Paula Clemens, MD (Abstracts 580-587; see page 122) Oral Abstract Session 411

11:00 am - 1:00 pm Room: Virginia Suite

Chemical and Molecular Conjugates Co-Chairs Hideyoshi Harashima, PhD & Millicent O. Sullivan, PhD (Abstracts 588-595; see page 122) Oral Abstract Session 412

11:00 am - 1:00 pm Room: Wilson ABC

Cardiovascular Gene & Cell Therapy Co-Chairs Roger J. Hajjar, MD & Xiao Xiao, PhD (Abstracts 596-603; see page 123) Oral Abstract Session 413

11:00 am - 1:00 pm Room: Marriott Ballroom Salons 2 & 3

Cancer-Oncolytic Viruses II Co-Chairs Ronald Rodriguez, MD, PhD & Masato Yamamoto, MD, PhD (Abstracts 604-611; Abstract 605 withdrawn; Abstract 191 moved from Poster Session I; see page 123) Oral Abstract Session 414

11:00 am - 1:00 pm Room: Maryland Suite Co-Chairs Gay Crooks, MD & Adrian J. Thrasher, MD, PhD (Abstracts 612-619; see page 124)

Saturday, May 22nd

Hematologic/Immunologic: Clinical, Preclinical and Safety

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Program Schedule Oral Abstract Session 415

11:00 am - 12:00 pm Room: Thurgood Marshall W

Lung and Respiratory Disease Gene & Cell Therapy Co-Chairs Terence R. Flotte, MD & Maria Limberis, PhD (Abstracts 620-623; see page 124) Oral Abstract Session 416

12:15 pm - 1:15 pm Room: Thurgood Marshall W

Gene Regulation Co-Chairs Olivier Danos, PhD & Giuliana Ferrari, PhD (Abstracts 624-627; see pages 124-125) Oral Abstract Session 417

11:00 am - 1:00 pm Room: Marriott Ballroom Salon 1

Stem Cell Therapies Co-Chairs Carolyn Lutzko, PhD & Alice F. Tarantal, PhD (Abstracts 628-635; see page 125)

Lunch Break 1:00 pm – 3:00 pm Meet-the-Investigator Lunch 420

1:00 pm - 2:15 pm Room: Balcony A

Stem Cells Speakers James Ellis, PhD & Carolyn Lutzko, PhD

Saturday, May 22nd

Meet-the-Investigator Lunch 421

1:00 pm - 2:15 pm Room: Balcony B

Roles of miRNAs Speakers Mark A. Kay, MD, PhD & Luigi M. Naldini, MD, PhD

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Program Schedule Oral Abstract Session 430

3:00 pm - 5:00 pm Room: Maryland Suite

Adenovirus and Other DNA Virus Vectors Co-Chairs Anja Ehrhardt, PhD & Florian Kreppel, PhD (Abstracts 636-643; see pages 125-126) Oral Abstract Session 431

3:00 pm - 5:00 pm Room: Delaware Suite

Infectious Diseases and Vaccines Co-Chairs Ann Leen, PhD & Bruce E. Torbett, PhD, MSPH (Abstracts 644-651; see page 126) Oral Abstract Session 432

3:00 pm - 5:00 pm Room: Marriott Ballroom Salons 2 & 3

Cancer – Immunotherapy II Co-Chairs Stephen Gottschalk, MD & David B. Weiner, PhD (Abstracts 652-659; see pages 126-127 ) Oral Abstract Session 433

3:00 pm - 5:00 pm Room: Thurgood Marshall W

Oligonucleotide & RNAi Therapeutics II Co-Chairs Paloma H. Giangrande, PhD & Peter M. Glazer, MD, PhD (Abstracts 660-687; see page 127) Oral Abstract Session 434

3:00 pm - 5:00 pm Room: Marriott Ballroom Salon 1 Co-Chairs Stephen M. Kaminsky, PhD & J. Fraser Wright, PhD (Abstracts 668-675; see pages 127-128)

Saturday, May 22nd

Cell Processing and Vector Production

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Program Schedule Oral Abstract Session 435

3:00 pm - 5:00 pm Room: Wilson ABC

Gene Therapy for Inborn Errors of Metabolism Co-Chairs Dwight Koeberl, MD, PhD & Brendan Lee, MD, PhD (Abstracts 676-683; see page 128) Oral Abstract Session 436

3:00 pm - 5:00 pm Room: Virginia Suite

Late Breaking Session Co-Chairs Guangping Gao, PhD & Stanton L. Gerson, MD (Abstracts 914-921; see pages 128-129)

Poster Hall Social (use your drink ticket at tonight's social) 5:00 pm – 6:00 pm Room: Atrium Poster Session III

6:00 pm – 8:00 pm Room: Exhibit Hall C

RNA Virus Vectors II (Abstracts 684 through 696; see page 129) AAV Vectors III (Abstracts 697 through 711; see page 130) Adenovirus and Other DNA Virus Vectors III (Abstracts 712 through 725; see pages 130-131) DNA Vectorology & Gene Targeting (Abstracts 726 through 741; see pages 131-132) Chemical and Molecular Conjugates III (Abstracts 742 through 757; see pages 132-133)

Saturday, May 22nd

Genetic and Metabolic Diseases Gene & Cell Therapy III (Abstracts 758 through 772; Abstract 764 has been moved to Poster Session II after Abstract 422; see page 133) Neurodegenerative Disorders, Spinal Cord and Trauma (Abstracts 773 through 783; see page 134) Musculo-skeletal Gene & Cell Therapy III (Abstracts 784 through 796; see pages 134-135) Infectious Diseases and Vaccines: Non-Vaccine Related Gene Transfer Research (Abstracts 797 through 808; see pages 135-136) Cancer – Immunotherapy III (Abstracts 809 through 827; see pages 136-137)

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13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

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Program Schedule Cancer-Oncolytic Viruses III (Abstracts 828 through 842; Abstract 838 withdrawn; see page 137) Cancer-Targeted Gene & Cell Therapy III (Abstracts 843 through 856; see pages 137-138) Hematologic and Immunologic Gene & Cell Therapy III (Abstracts 857 through 871; Abstract 869 has been move to Poster Session I after Abstract 233; see pages 138-139) Lung and Respiratory Disease Gene & Cell Therapy (Abstracts 872 through 881; see pages 139-140) Oligonucleotide & RNAi Therapeutics III (Abstracts 882 through 897; see pages 140-141) Stem Cell Therapies (Abstracts 898 through 913; see page 141) Late Breaking Science III (Abstracts 938 through 947; see page 142) Annual Meeting Concludes

Saturday, May 22nd

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Exhibit Hall Floor Plan

ASGCT 2010 Annual Meeting May 19-22, 2010 • Marriott Wardman Park Hotel • Washington, DC

Speaker Ready Room

Registration

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13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Exhibitor Descriptions Aldevron

BioSpherix, Ltd.

3233 15 Street, South Fargo, ND 58104 Telephone: (701) 297-9256 Fax: (701) 280-1642 E-mail: [email protected] Website: www.aldevron.com

19 Demott St. Lacona, NY 13083 Telephone: (315) 387-3414 Fax: (315) 387-3415 E-mail: [email protected] Website: www.biospherix.com

Booth Number: 506

Booth Number: 210

Aldevron is a leading contract manufacturing organization specializing in plasmid DNA, protein, antibody, DNA vaccine screening and genetic immunization (GIA™) services. We manufacture biologics for research, clinical, and diagnostic applications including ready-to-use AAV helper, cytokine, and reporter plasmids. Aldevron’s capacity and throughput capabilities support any project in bacterial, insect, and mammalian cell systems. By combining innovative technologies with best-in-class project management, our team ensures that we consistently exceed your expectations.  

Closed-incubation-system for clinical cell cultures. Multiple, independent, high performance, O2/CO2 incubators open into aseptic isolation modules with identical controlled atmospheres for uninterrupted conditions during cell handling. Can be a cGMP compliant mini-facility, offering cell and gene therapists an economical and practical alternative to expensive brick-and-mortar walk-in clean rooms.

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555 East Wells Street Suite 1100 Milwaukee, WI 53202 Telephone: (414) 278-1341 Fax: (414) 276-3349 E-mail: [email protected] Website: www.asgct.org

Booth Number: 609 & 611

Stop by the ASGCT membership booth to pick-up your Abstracts-on-Disc and the Education Session CD. Find out where the 2011 Annual Meeting will be held and other Society information. Biologics Consulting Group, Inc.

1317 King Street Alexandria, VA 22314 Telephone: (703) 739-5695 Fax: (703) 548-7457 E-mail: [email protected] Website: www.bcg-usa.com Booth Number: 401

Biologics Consulting Group, Inc., is an international firm providing regulatory and product development advice on the development and commercialization of biologicals, drugs, devices, diagnostics, and combination products. Since 1993, we have helped hundreds of products reach the marketplace. Our consultants come from the executive/senior tiers of FDA and industry.

Caliper Life Sciences

68 Elm Street Hopkinton, MA 01748 Telephone: (508) 435-9500 Fax: (508) 435-3439 Website: www.caliperls.com Booth Number: 514

Caliper Life Sciences is a leading supplier of tools and services for life science research including IVIS in vivo imaging systems.  IVIS is the most published platform for sensitive fluorescence and bioluminescence imaging in both 2D and 3D providing the insight to view complex biological interactions such as gene expression or stem cell engraftment in longitudinal studies of whole animals.  Center for Cellular and Molecular Therapeutics at the Children’s Hospital of Philadelphia

Room 5000 Colket Translational Research Building 3501 Civic Center Blvd. Philadelphia, PA 19104 Telephone: (267) 426-5256 Fax: (215) 590-3660 E-mail: [email protected] Booth Number: 313

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Exhibitor Descriptions Data Unlimited International, Inc.

15881-B Crabbs Branch Way Rockville, MD 20855 Telephone: (240) 631-7933 Fax: (240) 631-7937 E-mail: [email protected] Website: www.duii.com Booth Number: 211

Institutional Biosafety Committee Services (IBC Services)

2103 Harrison Av NW PMB 2436 Olympia, WA 98502-2607 Telephone: (360) 252-2850 Fax: (360) 252-2820 E-mail: [email protected] Website: www.ibcservicepoint.com

Starfruit Technologies® is software/database/computer/barcode for DNA diagnostic, genome/cancer research, immunogenetic labs – enables sample, patient, biorepository, billing databases; mutation, variant, disease, genotype databases, SNP databank; instrument interface, barcode, electronic signature, QA/QC; worksheet, workflow, inventory, protocol, image; epidemiological frequency, trend, geographical distribution, disease/disorder susceptibility, reporting; comply with 21CFR11, CAP, CLIA, HIPPA, PHI.

Booth Number: 505

Elsevier - Academic Press

15807 Quince Orchard Road N. Potomac, MD 20878 Telephone: (240) 477-8564 Fax: (240) 477-8571 E-mail: [email protected]

2425 Ridgecrest Dr. SE Albuquerque, NM 87108 Telephone: (505) 348-9457/9557 Fax: (505) 348-4983 E-mail: [email protected] Website: www.lrri.org

Booth Number: 406

Booth Number: 307

As the world’s leading publisher of science and health information, Elsevier serves more than 30 million scientists, students, and health and information professionals worldwide. Visit Elsevier for the latest clinical trial resources, such as: Clinical and Translational Science: Principles of Human Research by David Robertson and Gordon H. Williams.

The Lovelace Respiratory Research Institute is a private not-for-profit biomedical research organization. Lovelace offers animal models of disease, evaluation of efficacy of potential new therapeutics, and pre-clinical assessments including evaluation of pharmacokinetics/biodistribution in large and small animal models. Studies are routinely performed under GLP guidelines.

Exemplar Genetics

Mary Ann Liebert, Inc.

958 N. Main Sioux Center, IA 51250 Telephone: (712) 722-2767 Fax: (712) 722-2126 E-mail: [email protected] Website: www.exemplargenetics.com

IBC Services establishes and provides administrative support for local IBCs reviewing gene transfer clinical trials and related rDNA research. We have served medical practices, hospitals and health systems, and VA and university settings in 46 states and abroad. Broad experience from first-in-human and single-subject protocols to multicenter late-phase trials. Lovelace Respiratory Research Institute

140 Huguenot Street New Rochelle, NY 10801 Telephone: (914) 740-2100 Fax: (914) 740-2101 E-mail: [email protected] Website: www.liebertpub.com

Booth Number: 408

Booth Number: 510

Exemplar Genetics is enabling discovery through the creation of large animal models of human disease.  Through proprietary techniques and licensed processes, we have been successful in creating the only commercially available large animal model that replicates human disease.  These techniques can be utilized to create models of interest in any field. 

Mary Ann Liebert, Inc., publishes authoritative peer-reviewed journals in new and promising areas of science and biomedical research including Human Gene Therapy, (www.liebertpub.com/hum), named one of the 100 most influential Journals in Biology & Medicine over the last 100 years! Visit us @ booth #510.

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13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Exhibitor Descriptions Meridian Life Science, Inc.

National Gene Vector Biorepository

Booth Number: 208

Booth Number: 512

MLS provides contract R&D, process development, and clinical cGMP biomanufacturing for vaccines, viral challenge materials, vectored gene therapies, VLPs, and r-proteins from BEVS. With over 25 yrs. of cell culture, virus production, protein purification, assay development and regulatory experience, MLS is your trusted partner for preclinical to Phase I/II manufacturing.

The National Gene Vector Biorepository provides support for gene therapy research by offering several services. Check out the reagent repository comprising cell lines, plasmids and other tools used in gene therapy research. There are archiving services to meet FDA requirements. The NGVB will store clinical post-trial samples, final product reserves, and master cell banks as well as pharmacology/toxicology samples. A pharm/tox database containing gene therapy tox studies is a valuable resource. We offer assistance with insertional mutagenesis analysis through the SeqMap Bioinformatic Tool as well as LAM-PCR and other testing services. Go to https://www.ngvbcc.org or come and see us in Booth 512.

3471 River Hills Drive Cincinnati, OH 45244 Telephone: (513) 271-3700 Fax: (513) 271-3762 E-mail: [email protected] Website: www.meridianlifescience.com

Molmed S.p.A

Via Olgettina, 58 Milan, Italy 20132 Telephone: +39 0221277257 Fax: +39 0221277325 E-mail: [email protected] Website: www.molmed.com Booth Number: 209

MolMed is a medical biotechnology company with a primary focus on developing novel cell/gene therapies for treating cancer. The in-house GMP facility allowed MolMed to be involved in projects as GMP manufacturer. We are glad to offer you our solutions for GMP cell/gene therapy, from early stage to advanced clinical development. Nanosight

6660 N. High Street, Suite 2A Worthington, OH 43085 Telephone: (614) 888-0023 Fax: (614) 987-0045 E-mail: [email protected] Website: www.nanosight.com Booth Number: 405

Nanosight develops and manufactures instruments for Counting, Sizing and Visualizing Nanoparticles from 10 nm to 2.0 µm. Our unique Nanoparticle Tracking Analysis (NTA) technology allows for the measurement of Diffusion Coefficients of individual nanoparticles or viruses and determines the Count, Size & Concentration of the particle dispersions. Polydispersed and multi-modal samples are quickly identified and resolved. Direct Visualization of the dispersion being measured is displayed and a video of the particles being measured is created and stored along with the size distribution data. Options are available for fluorescence measurements.

980 West Walnut Street, R3 C602 Indianapolis, IN 46202 Telephone: (317) 274-4519 Fax: (317) 278-3327 E-mail: [email protected] Website: https://www.ngvbcc.org

Nature Publishing Group

75 Varick Street, 9th floor New York, NY 10013 Telephone: (212) 726-9200 Fax: (212) 696-9006 E-mail: [email protected] Website: www.nature.com Booth Number: 206

Nature Publishing Group brings leading scientific and medical research to your desk top. The NPG portfolio combines the continued excellence of Nature, its associated research and review journals, and over 45 leading academic and society journals in the life, physical and clinical sciences. Visit Booth Number 206 for free sample copies. Nature Technology Corporation

4701 Innovation Dr. Lincoln, NE 68521 Telephone: (402) 472-6530 Fax: (402) 472-6532 E-mail: [email protected] Website: www.natx.com Booth Number: 400

Nature Technology Corporation is a discovery-based bioengineering technology company, providing industry solutions for DNA-based gene therapeutics and DNA vaccines development, including: design, manufacturing, testing, and tech transfer of best-in-class vectors for in vivo gene expression.

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Exhibitor Descriptions Nexcelom Bioscience

360 Merrimack Street Bldg 9 Lawrence, MA 01843 Telephone: (978) 327-5340 Fax: (978) 327-5341 E-mail: [email protected] Website: www.nexcelom.com Booth Number: 414

Nexcelom’s Cellometer line of simple-to-use cell counters automate manual cell counting procedures by obtaining accurate counts, viability, and cell sizes in less than 30 seconds & only 20uL of sample. Fluorescence detection capabilities enable fast & simple determination of GFP transfection rates, PI-viability, & direct counting of WBCs without lysing. NHLBI Gene Therapy Resource Program

National Heart, Lung, and Blood Institute, NIH 2 Rockledge Center – 6701 Rockledge Drive Bethesda, MD 20892-7940 Telephone: (301) 435-0477 Fax: (301) 480-7971 E-mail: [email protected] Website: www.gtrp.org Booth Number: 311

The NHLBI Gene Therapy Resource Program (GTRP) facilitates the translation of gene therapy research into clinical applications primarily in heart, lung, and blood diseases. Services provided by the program include preclinical and clinicalgrade vector production, pharmacology/toxicology testing, immunology testing, regulatory support, and clinical trial funding assistance. NIH Office of Biotechnology Activities

6705 Rockledge Drive Suite 750 Bethesda, MD 20892 Telephone: (301) 496-9838 Fax: (301) 496-9839 E-mail: [email protected] Website: http:\\oba.od.nih.gov Booth Number: 508

The NIH Office of Biotechnology Activities (OBA) promotes science, safety, and ethics in biotechnology through advancement of knowledge, enhancement of public understanding, and development of sound public policies. OBA accomplishes its mission through analysis, deliberation, and communication of scientific, medical, ethical, legal, and social issues.

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NIH-RAID

6001 Executive Blvd, Room 2141 Bethesda, MD 20892 Telephone: (301) 594-4660 Fax: (301) 402-1501 E-mail: [email protected] Website: http://nihroadmap.nih.gov/raid/ Booth Number: 305

NIH-RAID makes available, at no cost, certain resources needed for the development of new therapeutic agents. Available services include: production, bulk supply, GMP manufacturing, formulation, development of an assay suitable for pharmacokinetic testing, and animal toxicology. NIH-RAID is not a grant program; successful projects gain access to government contract resources. Omnia Biologics, Inc.

12111 Parklawn Drive Rockville, MD 20852 Telephone: (301) 984-5928 Fax: (301) 984-2515 E-mail: [email protected] Website: www.omniabiologics.com Booth Number: 212

Omnia Biologics provides cost effective outsourcing of product development services to the biotechnology, pharmaceutical, and academic biologic drug developers. These services include process development, cGMP cell and virus banking, comprehensive cGMP manufacturing services, BSL-3 containment, assay development, formulation and fill/finish. Omnia has experience with a variety of vaccine, gene therapy and protein therapeutic platforms. Penn Vector Core

125 South 31st Street, Suite 2000 Philadelphia, PA 19104-3403 Telephone: (215) 573-0633 Fax: (215) 573-0300 E-mail: [email protected] Website: http://www.med.upenn.edu/gtp/vector_core.shtml Booth Number: 309

Penn Vector Core is a state-of-the-art vector production facility for investigators both within and external to the University of Pennsylvania. The main objective of the Core is to make various types of high quality viral vectors for gene transfer/ Vaccine studies at the preclinical and basic research level.

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Exhibitor Descriptions Philips Research

345 Scarborough Road Briarcliff Manor, NY 10510 Telephone: (914) 945-6188 Fax: (914) 945-6580 E-mail: [email protected] Website: www.research.philips.com/TIPS Booth Number: 501

ReGenX Biosciences

750 17th St. NW Suite 1100 Washington, DC 20006 Telephone: (202) 785-7438 Fax: (202) 785-7439 E-mail: [email protected] Website: www.regenxbio.com

Philips Research has developed the Therapy Imaging Probe System (TIPS) device. TIPS is a turnkey solution for all pre-clinical research needs using focused ultrasound for non-invasive gene delivery/transfection and site-targeted drug delivery using ultrasound and microbubble agents.

Booth Number: 407

Polyplus-transfection

Sartorius Stedim North America Inc.

Bioparc Bd S. Brant Illkirch, Alsace 67401 France Telephone: +33390406180 Fax: +33390406181 E-mail: [email protected] Website: www.polyplus-transfection.com Booth Number: 507

Polyplus-transfection is a Biotech company dedicated to the R & D and marketing of transfection reagents. Polyplus provides state-of-the-art quality reagents for standard in vitro DNA transfection to human gene therapy, as well as for protein production in synthetic medium, siRNA delivery and protein delivery in living cells. Puresyn, Inc.

87 Great Valley Parkway Malvern, PA 19355 Telephone: (610) 640-0800 Fax: (610) 640-0808 E-mail: [email protected] Website: www.puresyn.com Booth Number: 308

Puresyn, Inc., is a Separations and Purification Technology Company that develops, manufactures and markets state-of-the-art products and contract manufacturing services for nucleic acids and other biological molecules utilizing our proprietary polymer technology, PolyFlo® for production and purification of gene delivery products including plasmids and recombinant viruses at scales and customized specifications.

ReGenX is a research and development stage biopharmaceutical company developing novel therapeutics and research tools based on adeno-associated viral vectors, or AAV vectors. 5 Orville Drive Bohemia, NY 11716 Telephone: (800) 368-7178 Fax: (631) 254-2453 E-mail: [email protected] Website: www.sartorius-stedim.com Booth Number: 306

Sartorius Stedim Biotech is a leading provider of equipment and services for the development, quality assurance and production processes of the biopharmaceutical industry as well as the public sector. With integrated solutions covering fermentation, filtration, purification, fluid management, and lab technologies, Sartorius Stedim Biotech focuses on single-use technologies and value-added services to meet requirements of the industries it serves. Seattle’s Convention and Visitors Bureau

701 Pike Street, Suite 800 Seattle, WA 98101 Telephone: (206) 461-5800 Fax: (206) 461-5855 E-mail: [email protected] Website: www.visitseattle.org Booth Number: 500

Seattle is two cities in one. It’s a world-class metropolis set within, beautiful natural surroundings, offering the best of urban lifestyle while embracing the outdoors.  Many of the city’s top visitor attractions are within mere blocks of the Washington State Convention Center, including Pike Place Market. Seattle is metronatural. 

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Exhibitor Descriptions St. Jude Children’s Research Hospital

TriLink BioTechnologies, Inc.

Booth Number: 207

Booth Number: 314

St. Jude Children’s Research Hospital is a non-profit biomedical research institution located in Memphis, TN, in the United States where 190 basic science and clinical researchers investigate the molecular basis of both normal cellular and diseased processes.  Visit our booth to discuss and apply for Postdoctoral Fellowship positions for PhD, MD, PharmD, and DVM students.

TriLink BioTechnologies manufactures custom oligonucleotides, modified nucleoside triphosphates and CleanAmp™ PCR products. In addition, custom chemistry, contract research services and ISO/QSR compliant cGMP production facilities are offered. TriLink’s strong proficiency in nucleoside and oligonucleotide chemistry means high purity, modified compounds and expert technical support. For more information visit www.trilinkbiotech.com.

262 Danny Thomas Place Memphis, TN 38105 Telephone: (901) 595-2750 Fax: (901) 595-5376 E-mail: [email protected] Website: www.stjude.org

Techulon, Inc.

9955 Mesa Rim Road San Diego, CA 92121 Telephone: (858) 546-0004 Fax: (858) 546-0020 E-mail: [email protected] Website: www.trilinkbiotech.com

2200 Kraft Drive Suite 2475 Blacksburg, VA 24060 Telephone: (415) 602-0481 Fax: (206) 202-0418 E-mail: [email protected] Website: www.techulon.com

Vandalia Research

Booth Number: 613

Booth Number: 513

Techulon develops and markets biodegradable transfection reagents that exhibit high efficacy and very low cytotoxicity, especially in sensitive primary cells. Techulon welcomes inquiries from researchers and from companies interested in developing therapeutic applications. Thermo Scientific Genomics

601 Genome Way Suite 2100 Huntsville, AL 35806 Telephone: (256) 704-4848 Fax: (256) 704-4849 E-mail: [email protected] Website: www.thermo.com/gen Booth Number: 213

The Thermo Scientific genomics portfolio, which includes Dharmacon and Open Biosystems RNAi and gene expression technologies and ABgene PCR products, provides novel tools to help our customers unlock the underlying molecular mechanisms of disease from cancer, to diabetes to viral infection and develop new therapies to fight it.

EXHIBITOR PROSPECTUS final program

PO Box 2705 Huntington, WV 25726-2705 Telephone: (304) 529-0803 Fax: (888) 550-8220 E-mail: [email protected] Website: www.vandaliaresearch.com VGXI, Inc.

2700 Research Forest Drive Suite 180 The Woodlands, TX 77381 Telephone: (281) 296-7300 E-mail: [email protected] Website: www.vgxii.com Booth Number: 607

VGXI, Inc. has over 13 years experience manufacturing injectable-grade DNA plasmids for gene therapy research. A leading contract manufacturing organization (CMO), our fermentation services are designed to meet the unique needs of gene therapy researchers at every stage of product development – from research to commercial supply. This comprehensive offering lets researchers avoid the risks associated with switching suppliers just prior to clinical trials. Our mission is to support biomedical researchers in their effort to rid the world of deadly and debilitating diseases. We do this by leveraging our experience and patented technologies to maximize our clients’ chances of clinical success.

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Exhibitor Descriptions Waisman Clinical Biomanufacturing Facility

1500 Highland Avenue Madison, WI 53705 Telephone: (608) 263-8940 E-mail: [email protected] Website: www.GMPbio.org Booth Number: 214

The Waisman Clinical Biomanufacturing Facility (WCBF) is a dedicated manufacturing facility that provides cGMP manufacturing services for producing biotherapeutics for early-stage human clinical trials. Services that are provided by the WCBF range from process development to clinical production and product fill and finish, all with full quality system support. Wiley-Blackwell

350 Main St. Malden, MA 02148 Telephone: (800) 532-5954 Fax: (781) 338-8552 E-mail: [email protected] Website: www.wiley.com Booth Number: 413

Wiley-Blackwell publishes quality journals in gene and cell therapy including Stem Cells, The Journal of Gene Medicine, and Journal of Tissue Engineering and Regenerative Medicine, and must-read books including Immunology of Gene Therapy, Delivery Technologies for Biopharmaceuticals and Viral Therapy of Cancer. See our full collection at: www.wileyblackwell.com. 

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Exhibitor Information General Information

The Exhibit Hall is located in Exhibit Hall C of the Marriott Wardman Park Hotel. All participants are urged to allow adequate time in your daily schedule to visit the exhibits. The companies, organizations and institutions exhibiting at the ASGCT 13th Annual Meeting will be providing you with the latest information on products and services directly related to your professional needs, as well as recruiting for open positions.

Exhibitor Badge Policy

Exhibits will be open Wednesday, May 19 through Friday, May 21, 2010.

Admission to the Exhibit Hall will be by name badge only. Security guards will monitor exhibit hall entrances for proper I.D. Exhibit booth personnel must show an official ASGCT exhibitor name badge in order to gain access to the exhibit hall during installation, show, or dismantlement hours. Independent contractor personnel, hired by an exhibitor to install and dismantle their display, do not need to show exhibitor badges, but should possess identification that they are employed by the independent contractor. If independent contractors do not possess proper identification, they should obtain a name badge at the on-site exhibitor registration desk.

Exhibitor Registration

Endorsement Disclaimer

Exhibit Dates

The Exhibitor Registration Desk is located in the atrium, right outside Exhibit Hall C. Exhibitor Registration Desk Hours

Wednesday, May 19 Thursday, May 20 Friday, May 21

Exhibit Installation

Wednesday, May 19

Exhibit Hall Hours

Wednesday, May 19 Thursday, May 20 Thursday, May 20 Friday, May 21 Dismantlement

Friday, May 21

9:00 am – 7:00 pm 7:00 am – 5:00 pm 7:00 am – 5:00 pm 9:00 am – 5:00 pm 7:00 pm – 9:00 pm 9:15 am – 10:30 am 6:15 pm – 8:15 pm 6:30 pm – 8:30 pm 8:30 pm – 10:30 pm

Note: All exhibit booths must be staffed during hall hours. Packing of equipment/literature and the dismantling of exhibits will not be permitted until after 8:30 pm on Friday, May 21, 2010.

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Products and services displayed in the Exhibit Hall or advertised in this program occur by contractual business arrangements between ASGCT and participating companies and organizations. These arrangements do not constitute or imply an endorsement by ASGCT of these products and services.

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Washington, DC Fun Facts In Washington, DC, you’ll enjoy access to fascinating, FREE attractions and historic sights. Touch a moon rock, marvel at the Hope Diamond, view Dorothy’s Ruby Red slippers or explore Native American culture at the Smithsonian Institution’s fifteen Washington, DC area facilities. Discover treasures like the Gutenberg Bible at the Library of Congress, the only da Vinci painting in North America at the National Gallery of Art and historic documents like the Declaration of Independence at the National Archives. The National Zoo is also located within walking distance of the Marriott Wardman Park Hotel.

Hotel Information

Away from these celebrated federal sites, Washington, D.C. unwinds into a fascinating network of neighborhoods where visitors discover trendy boutiques, hip bars and restaurants, plus art galleries, historic homes and lush parks and gardens. Shoppers love the store-lined streets of Georgetown, while jazz music fans won’t want to miss a trip to U Street, where Duke Ellington played his first notes. The city’s international character shines through in its Adams Morgan and Dupont Circle neighborhoods, two prime destinations for eclectic dining and nightlife and the historic center of the city’s embassy community.

This Washington DC restaurant features an Old English menu. Enjoy sandwiches, soups, pizzas and microbrews. Open for lunch and dinner.

The arrival of several new eateries has made the nation’s capital a prime destination for dining out, with many of the city’s top tables located in the downtown Penn Quarter neighborhood. DC is also earning new recognition as a thriving performing arts town, with 65 professional theatre companies based in the metropolitan area presenting edgy world premieres and celebrated Broadway musicals throughout the year. It’s easy to get around town thanks to DC’s pedestrian-friendly streets and its safe, efficient public transportation system—including Metrorail and the new Circulator bus—it’s easy to get from your hotel to Washington, DC’s attractions. For more information, visit www.washington. org.

Marriott Wardman Park Hotel

2660 Woodly Road NW Washington, DC Phone: (202) 328-2000 The hotel has several places you can grab a bit to eat, which include: Harry’s Pub:

Illy’s

Grab a bite to go at this coffeehouse featuring specialty coffees, teas and fresh baked goods. Stone’s Throw Restaurant & Bar:

A steakhouse offering steaks, chops and seafood dishes that is open for breakfast, lunch and dinner Woodley’s Market

This gourmet deli serves breakfast items, snacks and gourmet takeout sandwiches and meals. Or walk through the neighborhood and enjoy over 20 eatery options.

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Abstract Directory Thursday, May 20, 2010 Oral Abstract Session 230 AAV Vectors: Vector Trafficking 2:00 PM - 4:00 PM Room: Marriott Ballroom: Salons 2 & 3

Thursday, May 20th

1 2:00 PM Engineering AAV for Improved In Vitro Infectivity, In Vivo Tropism and Onset of Expression Ru Xiao, Sadik H. Kassim, Lauren E. Mays, Deirdre McMenamin, Regina Munden, James M. Wilson, Luk H. Vandenberghe. 2 2:15 PM Targeting Directed Evolution of AAV Capsids for Specific Therapeutic Applications Steven J. Gray, Bonita L. Blake, Matthew Hirsch, Hugh E. Criswell, Sarah Nicolson, Wuping Li, Thomas J. McCown, R. Jude Samulski. 3 2:30 PM Enhanced Hepatic Transgene Expression Following Intravenous Delivery of Novel Stem Cell-Derived Recombinant AAV Vectors Laura J. Smith, Ariel Kauss, Kamehameha K. Wong, Saswati Chatterjee. 4 2:45 PM AAV Capsid Sequence Significantly Influences Intracellular Viral Trafficking in Polarized Human Airway Epithelial Cells Nicholas W. Keiser, Ziying Yan, Diana Lei-Butters, John F. Engelhardt. 5 3:00 PM Cell Entry Pathway Defines Efficiency and Specificity of rAAV Targeting Vectors Silke Uhrig, Jessica Sallach, David Gomez Almarza, Luca Perabo, Sabine Eming, Fernando Laguzzi Larcher, Michael Hallek, Hildegard Büning.

Oral Abstract Session 231 DNA Vectorology & Gene Targeting 2:00 PM - 4:00 PM Room: Maryland Suite 9 2:00 PM A Simplified Approach for Robust Minicircle DNA Vector Production Using a New Genetically-Modified E. Coli Producer Strain ZYCY10P3S2T Zhi-Ying Chen, Cheng-Yi He, Mark A. Kay. 10 2:15 PM Targeted Transgene Integration in Epidermal Stem Cells by Zinc Finger Nucleases and Integrase-Defective Lentiviral Vectors Giulietta Maruggi, Angelo Lombardo, Francesca Miselli, Pietro Genovese, Michael C. Holmes, Philip D. Gregory, Alessandra Recchia, Fulvio Mavilio, Luigi Naldini. 11 2:30 PM Zinc Finger Nucleases Drive Efficient Gene Disruption and SiteSpecific Gene Addition in Human HSC Nathalia G. Holt, Kevin G. Haworth, Kathleen C. Burke, Jianbin Wang, Kenneth Kim, Geoffrey Friedman, Philip D. Gregory, Michael C. Holmes, Paula M. Cannon. 12 2:45 PM Manipulating piggyBacTransposon Chromosomal Integration SiteSelection in Human Cells Claudia Kettlun, Daniel Galvan, Aparna Kaja, Edward Rebar, Matthew H. Wilson. 13 3:00 PM Optimized Recovery of Integration Sites from Gene-Modified Cells Using Mu Transposition In Vitro Troy L. Brady, Nirav Malani, Gary P. Wang, Charles C. Berry, Philippe Leboulch, Salima Hacein-Bey-Abina, Marina Cavazzana-Calvo, Harri Savilahti, Frederic D. Bushman.

6 3:15 PM Novel Cardiac-Tropic AAV Vectors by DNA Shuffling and In Vivo Screening Zhenhua Yuan, Lin Yang, Chunping Qiao, Juan Li, Xiao Xiao.

14 3:15 PM An In Vitro-Identified High Affinity Nucleosome Positioning Signal Is Capable of Transiently Positioning a Nucleosome In Vivo Lia E. Gracey, Zhi-Ying Chen, Jay M. Maniar, Anton Valouev, Arend Sidow, Andrew Z. Fire, Mark A. Kay.

7 3:30 PM Intravenously Administered rAAV9 Vector Efficiently Transduces Tissues In Vivo Via Caveolin-1-Independent Transport across the Capillary Endothelial Cell Barrier Kei Adachi, Nicole M. Kotchey, Maliha Zahid, Hiroyuki Nakai.

15 3:30 PM Hyperactive piggyBac-Mediated Gene Transfer in Somatic Cells In Vivo Joseph E. Doherty, Liqin Zhou, Kosuke Yusa, Allan Bradley, Nancy L. Craig, Matthew H. Wilson.

8 3:45 PM Directed Evolution of Adeno-Associated Virus Variants That Evade Human Neutralizing Antibodies Daniel Stone, James T. Koerber, Federico Mingozzi, Gregory M. Podsakoff, Katherine High, David V. Schaffer.

16 3:45 PM Homing Endonucleases for Gene Therapy Adi Barzel, Hilla Giladi, Einat Sachar, Ludmila Rivkin, Eyal Privman, Mor Paldor, Tal Pupko, Toni Cathomen, Eithan Galun, Martin Kupiec.

EXHIBITOR PROSPECTUS final program

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

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Abstract Directory Thursday, May 20, 2010  Oral Abstract Session 232 Neurologic & Ophthalmic Gene & Cell Therapy 2:00 PM - 4:00 PM Room: Wilson ABC 17 2:00 PM AAV-Mediated Gene Therapy Restores Visual Function and Behavior to a Mouse Model of Leber Congential Amaurosis-1 (LCA1) Shannon E. Boye, Sanford L. Boye, Jijing Pang, Renee C. Ryals, Andy W. Neeley, Drew Everhart, Yumiko Umino, Joseph C. Besharse, Robert Barlow, William W. Hauswirth.

24 3:45 PM Patterns of Motor Function Recovery in a Murine Model of Severe Menkes Disease Rescued by Brain-Directed AAV5 Gene Therapy Plus Copper Anthony Donsante, Stephen G. Kaler.

Oral Abstract Session 233 Cancer – Immunotherapy T Cell Studies 2:00 PM - 4:00 PM Room: Thurgood Marshall W

19 2:30 PM Brain Gene Therapy for Metachromatic Leucodystrophy: Towards Clinical Trial Caroline Sevin, Dolan Sondhi, Françoise Piguet, Marie-Anne Colle, Sylvie Raoul, Thomas Roujeau, Monique Piraud, Ornella Ahouansou, Celine Bouquet, Marie-Thérèse Vanier, Philippe Moullier, Yan Cherel, Neil R. Hackett, Michel Zerah, Patrick Aubourg, Ronald G. Crystal, Nathalie Cartier.

26 2:15 PM Adenovirus Mediated Expression of Flt3L and HSV1-TK within the Brain Tumor Microenvironment Elicits Higher Therapeutic Efficacy Than Dendritic Cells’ Vaccination Yohei Mineharu, Gwendalyn D. King, Marianela Candolfi, Weidong Xiong, Hikmat Assi, Kader Yagiz, Kurt M. Kroeger, Patricia Lin, B. Serguei, Pedro R. Lowenstein, Maria G. Castro.

20 2:45 PM Neonatal Intracerebroventricular Infusion of AAV2/8 Vector Leads to High Levels of Human a-L-Iduronidase Expression in the Brain and Complete Neurophenotypic Correction in a Murine Model of Mucopolysaccharidosis Type I Daniel A. Wolf, Andrew W. Lenander, Zhenhong Nan, Lalitha R. Belur, Debra Swanson, Chester B. Whitley, Walter C. Low, R. Scott McIvor.

27 2:30 PM Pharmacokinetics of Anti-PSMA Gene-Modified T Cells Infused after Non-Myeloablative Conditioning in Prostate Cancer Anthony J. Bais, Qiangzhong Ma, Hannah Boss, Esther L. Beaudoin, Richard P. Junghans.

21 3:00 PM Circuit-Specific Expression of Channelrhodopsin Restores Visual Function in Blind rd1, rd16, and rho -/- Mice Alan Horsager, Jian Wen Liu, Edward S. Boyden, A. Cyrus Arman, Kimberly A. Silka, Mehdi Doroudchi, Shannon E. Boye, Benjamin C. Matteo, Alapakkam P. Sampath, William W. Hauswirth. 22 3:15 PM Combination Therapy for the Treatment of Infantile Neuronal Ceroid Lipofuscinosis (INCL) Shannon L. Macauley-Rambach, Marie S. Roberts, Mark S. Sands. 23 3:30 PM rAAV Delivery of a-Synuclein-Specific Single Chain Antibodies Attenuates Dopamine Loss in a Mouse Model of Parkinson’s Disease Kathleen Maguire-Zeuss, William J. Bowers, Wade Narrow, Eric Yehling, Howard J. Federoff.

28 2:45 PM Binary Control of Tumor-Specific Cytotoxic T Lymphocytes by Transgenic IL7 and IL7r Expression Juan F. Vera, Ceidy Sanchez, Sujatha Muralidharan, Helen E. Heslop, Cliona M. Rooney, Gianpietro Dotti, Malcolm K. Brenner. 29 3:00 PM Multi-Tumor-Antigen-Specific Cytotoxic T Lymphocytes for Cancer Immunotherapy Ulrike Gerdemann, Juan F. Vera, Anne S. Christin, Russell C. Cruz, Helen E. Heslop, Malcolm K. Brenner, Catherine M. Bollard, Cliona M. Rooney, Ann M. Leen. 30 3:15 PM Simultaneous Comparison of T Cells Expressing 1st or 2nd Generation CARs in Human Subjects with B-Cell Malignancies: Contribution of Costimulatory Endodomains B. Savoldo, C. Ramos, C. Bollard, E. Liu, M. Mims, M. Keating, A. Gee, Z. Mei, C. Rooney, H. Heslop, M. Brenner, G. Dotti.

Thursday, May 20th

18 2:15 PM Adeno-Associated Virus Vector Expressing an Amyloid beta 1-42 Intrabody Rescues White Matter Pathology during Early Alzheimer’s Disease Maya K. Desai, Michael A. Mastrangelo, Deborah A. Ryan, Kelly L. Sudol, Wade C. Narrow, William J. Bowers.

25 2:00 PM Adoptive Transfer of Peripheral Blood Lymphocytes Genetically Modified To Express a T Cell Receptor Recognizing Carcinoembryonic Antigen into Patients with Metastatic Colorectal Cancer Induced Inflammatory Colitis without Mediating Anti-Tumor Effects Maria R. Parkhurst, James C. Yang, Russell C. Langan, Steven A. Feldman, Mark E. Dudley, Paul F. Robbins, Steven A. Rosenberg.

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Abstract Directory Thursday, May 20, 2010 Oral Abstract Session 233 — continued Cancer – Immunotherapy T Cell Studies 2:00 PM - 4:00 PM Room: Thurgood Marshall W

Thursday, May 20th

31 3:30 PM Prolonged Regression of Lymphoma and Elimination of CD19+ Cells in a Patient That Received Anti-CD19 Chimeric Antigen ReceptorTransduced T Cells J. N. Kochenderfer, M. E. Dudley, R. A. Morgan, I. Maric, M. Raffeld, M. StetlerStevenson, J. E. Janik, W. H. Wilson, C. M. Yuan, S. A. Rosenberg. 32 3:45 PM Marked Regression of Adenopathy Following Infusion of Autologous T Cells Genetically Targeted to the CD19 Antigen in a Patient with Bulky CLL Renier J. Brentjens, Isabelle Riviere, Mark Frattini, Xiuyan Wang, Clare Taylor, Malgorzata Olszewska, Oriana Borquez-Ojeda, Shirley Bartido, Jolanta Stefanski, Raymond Yeh, Michel Sadelain.

Oral Abstract Session 234 Cancer-Oncolytic Viruses I

37 3:00 PM Insect Cell Carriers for Systemic Delivery of Oncolytic Viruses Dominic G. Roy, Anthony T. Power, Fabrice Le Boeuf, John C. Bell. 38 3:15 PM Clinical Translational Development of rVSV(MD51)-M3: Biodistribution and Pharm/Tox Studies in Rats and Monkeys Marcia Meseck, Nelson Humala, Boxun Xie, Swan Thung, Susan Morgello, Gregg Goldschlager, Savio Woo. 39 3:30 PM A High-Throughput Pharmaco-Viral Approach Identifies Oncolytic Novel Virus Sensitizers Jean-Simon Diallo, Fabrice Le Boeuf, Frances Lai, Julie Cox, Markus Vaha-Koskela, Hesham Abdelbary, Heather MacTavish, Katherine Waite, Theresa Falls, Jenny Wang, Ryan Brown, Jan E. Blanchard, Eric D. Brown, David H. Kirn, John Hiscott, Harry Atkins, Brian D. Lichty, John C. Bell. 40 3:45 PM Identification of Adenovirus Modulator Genes in Human Cancer Cells by Whole Genome RNAi Library Screening Christie Vermeulen, Nikki Tol, Ida van der Meulen-Muileman, Winald R. Gerritsen, Victor W. van Beusechem.

Oral Abstract Session 235 Immunologic & Host Responses in Gene & Cell Therapy

2:00 PM - 4:00 PM Room: Marriott Ballroom Salon 1

2:00 PM - 4:00 PM Room: Virginia Suite

33 2:00 PM Demonstration of Delivery and Antitumoral Activity of JX-594, a Targeted Multi-Mechanistic Oncolytic Poxvirus, Following a Single Intravenous Infusion in Patients with Refractory Metastatic Cancers James Burke, Joe Stephenson, Laura Chow, Derek Jonker, Andrew Haas, Jorge Nieva, Kara Sabourin, Adina Pelusio, Caroline Breitbach, Taeho Hwang, Anne Moon, Kelley Parato, Manijeh Daneshmand, John C. Bell, David H. Kirn.

41 2:00 PM Repeated Autologous Infusions of Cells Modified with VSV-GPseudotyped Lentiviral Vector VRX496 Can Induce Anti-VSV-G Humoral Response in Humans without Clinical Consequence or Effect on Modified Cells Persistence: Findings from Lexgenleucel-T Phase 2 Clinical Trial Franck Lemiale, Lindsay Shovlin, Kris Andre, Gerard McGarrity, Tessio Rebello, Laurent Humeau.

34 2:15 PM Highly Attenuated Vaccinia Virus with microRNA-Regulated Oncolysis for Cancer Virotherapy Mina Hikichi, Minoru Kidokoro, Hisatoshi Shida, Hideaki Tahara, Takafumi Nakamura. 35 2:30 PM A Phase 1 Trial of Intravesical (IVE) CG0070 in Patients with Superficial Bladder Cancer after BCG Failure James M. Burke, Don Lamm, James Mckiernan, John Nemunaitis, Joe Stephenson, Max Meng, James Arsenau, Junko Aimi, Daniel Maslyar. 36 2:45 PM A Phase 1 Trial of the Novel Infectivity-Enhanced Bicistronic Adenovirus Ad5.SSTR/TK.RGD in Recurrent Gynecologic Cancer Kenneth H. Kim, Kellie Schneider, Minghui Wang, Janis O’Malley, Wen Wan, Meredith A. Preuss, Raymond D. Harris, Rosemarie Aurigemma, Gene P. Siegal, Kurt Zinn, David T. Curiel, Ronald D. Alvarez.

EXHIBITOR PROSPECTUS final program

42 2:15 PM Intensive Pharmacological Immunosuppression Allows for Repetitive Liver Gene Transfer with Recombinant Adenovirus in Nonhuman Primates Antonio Fontanellas, Sandra Hervás-Stubbs, Itsaso Mauleón, Juan Dubrot, Uxua Mancheño, María Collantes, Ana Sampedro, Carmen Unzu, Carlos Alfaro, Asis Palazón, Rafael Enríquez de Salamanca, Cristian Smerdou, Alberto Benito, Jesús Prieto, Iván Peñuelas, Ignacio Melero. 43 2:30 PM Induction of Regulatory T Cells as an Approach To Modulate CTL Responses Against AAV Capsid Epitopes Daniel J. Hui, Gary Pien, Etiena Basner-Tschakarjan, Federico Mingozzi, Jonathan D. Finn, Shangzhen Zhou, William Martin, Anne S. De Groot, Katherine A. High.

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Abstract Directory Thursday, May 20, 2010  Oral Abstract Session 235 — continued Immunologic & Host Responses in Gene & Cell Therapy 2:00 PM - 4:00 PM Room: Virginia Suite 44 2:45 PM Long-Term Phenotypic Correction of Hemophilia A after Neonatal Administration of AAV Chuhong Hu, Fides D. Lay, Joseph Springer, Ronald W. Busuttil, Gerald S. Lipshutz.

46 3:15 PM Autologous Dendritic Cell Therapy for Tolerance Induction Gautam Sule, Masataka Suzuki, Racel Cela, John Rodgers, Brendan Lee. 47 3:30 PM Differential Transgene Expression in Dendritic Cells with Unique Regulatory Cassettes Impacts Efficacy in Pompe Disease Baodong Sun, Ping Zhang, Xiao Yi Yang, Timothy C. Clay, Dwight D. Koeberl. 48 3:45 PM Impact of the Host Environment on the Control of Gene TransferInduced Immune Responses with miRNA142.3p-Regulated Vectors Florence Boisgérault, David-Alexandre Gross, Maxime Ferrand, Sylvie Darocha, Jérôme Poupiot, Marc Bartoli, Isabelle Richard, Anne Galy.

Oral Abstract Session 236 Oligonucleotide & RNAi Therapeutics I 2:00 PM - 4:00 PM Room: Delaware Suite 49 2:00 PM Systemic Delivery of Antisense Morpholino Corrects RNA MisSplicing and Reduces Myotonia in a Transgenic Mouse Model of Myotonic Dystrophy Type 1 Thurman M. Wheeler, Yong-Fu Li, Krzysztof Sobczak, Paul A. Morcos, Charles A. Thornton. 50 2:15 PM Triplex-Induced Correction of IDUA Gene Mutations as a Novel Hurler Syndrome Therapy Jake del Campo, Karla D’Agostino, Ranjit S. Bindra, Peter M. Glazer.

52 2:45 PM Constitutive Long-Term Expression of shRNA in Liver Induces Cellular Stress Miwon Ahn, Scott R. Witting, Rafaela Ruiz, Romil Saxena, Nuria Morral. 53 3:00 PM In Vivo AAV Delivered Allele Specific shRNA for the Knockdown of Alpha-1 Antitrypsin Christian Mueller, Bryan O’Sullivan-Murphy, Qiushi Tang, Terence R. Flotte. 54 3:15 PM Expression Determinants of Mammalian Argonaute Proteins in Mediating Gene Silencing Paul N. Valdmanis, Shuo Gu, Praveen Sethupathy, Mark A. Kay. 55 3:30 PM Antisense Oligonucleotide-Mediated Therapy Increases SMN Levels in the CNS and Prolongs Survival in a Severe Mouse Model of Spinal Muscular Atrophy Marco A. Passini, Jie Bu, Amy M. Richards, Lisa M. Stanek, S. P. Sardi, Yimin Hua, Gene Hung, Lamya S. Shihabuddin, C. F. Bennett, Adrian R. Krainer, Seng H. Cheng. 56 3:45 PM Multiple Exon-Skipping Using Cell-Penetrating Morpholinos for Dystrophic Dogs Toshifumi Yokota, Takashi Saito, Nobuyuki Urasawa, Tetsuya Nagata, Akinori Nakamura, Ryszard Kole, Peter Sazani, Terence Partridge, Shin’ichi Takeda, Eric Hoffman.

Poster Session I 6:15 PM - 8:15 PM Room: Exhibit Hall C & B-South

AAV Vectors I 57 Enveloped AAV Vectors for Gene Transfer Applications Casey A. Maguire, Lucia Mincheva-Nilsson, Vladimir Baranov, Bakhos A. Tannous, Miguel Sena-Esteves, Xandra O. Breakefield, Johan Skog. 58 Chimpanzee-Derived Novel Natural Variants of AAV9: Vector Development and Interrogation of Correlations between Capsid Structure and Vector Biology Li Zhong, Jun Xie, Qing Xie, Kim Van Vliet, Qiang Wang, Mengxin Li, Qin Su, Ran He, Jason Goetzmann, Mavis Mckenna, Terry Flotte, Guangping Gao. 59 Gene Delivery to the Pig Cone-Enriched Retina Highlights SpeciesSpecific Differences in AAV-Mediated Photoreceptor Transduction Claudio Mussolino, Francesco Viola, Michele Della Corte, Settimio Rossi, Umberto Di Vicino, Simona Neglia, Monica Doria, Francesco Testa, Roberto Giovannoni, Manuela Crasta, Massimo Giunti, Edoardo Villani, Marialuisa Lavitrano, Maria Laura Bacci, Roberto Ratiglia, Francesca Simonelli, Alberto Auricchio, Enrico M. Surace.

Thursday, May 20th

45 3:00 PM Impact of Pre-Existing Neutralizing Antibodies on AAV8-Mediated, Liver-Directed Gene Transfer and Vector Biodistribution in Nonhuman Primates Lili Wang, Roberto Calcedo, Peter L. Bell, Martin W. Lock, Rebecca L. Grant, James M. Wilson.

51 2:30 PM Using rAAV Delivered miRNAs To Knockdown Misfolded Human Alpha 1 Antitrypsin in a Transgenic Mouse Model Christian Mueller, Qiushi Tang, Bryan O’Sullivan Murphy, Terence R. Flotte.

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Abstract Directory Thursday, May 20, 2010 Poster Session I — continued 6:15 PM - 8:15 PM Room: Exhibit Hall C & B-South

AAV Vectors I

Thursday, May 20th

60 Enhancing AAV2 Transduction by Anti-Microtubule Drugs through RhoA GTPase Activation Ping-Jie Xiao, Richard J. Samulski. 61 Tyrosine Mutations in the Capsid of AAV Vectors Alters Transduction Properties in the Retina Hilda Petrs-Silva, Astra Dinculescu, Seok H. Min, Li Zhong, Sergei Zolotukhin, Arun Srivastava, Alfred Lewin, William W. Hauswirth. 62 Riding Along with AAV: Mechanistic Studies on Vesicular Trafficking Pathways of AAV Serotypes Shen Shen, Xin Ming, Aravind Asokan, Rudy L. Juliano. 63 Effects of DNA Methylation on AAV Integration and Transgene Expression Diptiman Chanda, Jonathan A. Hensel, Jerome T. Higgs, Niroop Kaza, Rajat Grover, Selvarangan Ponnazhagan. 64 Efficient Transduction of Mouse Liver and Other Organs Following Intravenous Administration of a Recombinant Porcine AAV Alexander J. Bello, Allan R. Chand, Monica Doria, Kaylie Tran, Mariacarmela Allocca, Alberto Auricchio, Gary Kobinger. 65 Rational Design Meets Directed Evolution for Tailoring the AAV Capsid Luk H. Vandenberghe, Ru Xiao, Christie L. Bell, Kim Van Vliet, Jing Lu, Mavis AgbandjeMcKenna, James M. Wilson. 66 AAV5 Transduction and Transgene Expression and Bioactivity in Fibroblast-Like Synoviocytes from Rheumatoid Arthritis Patients Caroline J. Aalbers, Scott A. Loiler, Federico Mingozzi, Paul-Peter Tak, Margriet J. Vervoordeldonk. 67 Pre-Injection of Polymers Can Increase the In Vivo Transduction Efficiency of Adeno-Associated Virus Type 2 Gilles Moulay, Sylvie Boutin, Carole Masurier, Daniel Scherman, Antoine Kichler.

68 Therapeutic Expression of FIX at Reduced Doses Using AAV-Protein Phosphotase-5 Helper Virus Brandon K. Sack, Jayandharan R. Giridhara, Zhong Li, Herzog W. Roland, Srivastava Arun. 69 A Scalable Process for Purification of Recombinant Adeno-Associated Virus Serotype 9 Jingmin Zhou, Xiao Yang, Linda Couto, Katherine A. High, Guang Qu. 70 Adeno- Associated Virus Type 2 Rep Proteins Mediate Preferential Integration of a Lentiviral Vector Victor J. McAlister, Tshaka Cunningham, Anthony T. Craig, Roland A. Owens. 71 High Throughput Assay for Quantifying In Vitro AAV Vector Transduction Efficiency in ARPE19 Cells Renee C. Ryals, Sanford L. Boye, Shannon Boye, Andrew Neely, William W. Hauswirth. 72 Optimization of Parameters for AAV Production in Insect Cells and Conditions for Baculovirus Storage Haifeng Chen.

Adenovirus and Other DNA Virus Vectors I 73 Radiation-Induced Liver Fibrosis Is Mitigated by Gene Therapy Against Transforming Growth Factor-b Signaling in the Rat Du Shi-Suo, Zeng Zhao-Chong, Zhou Jian, Tan Yun-shan, Zhang Zheng-Yu, Zeng Hai-Ying, Liu Zhong-shan. 74 Protection of Salivary Glands from Irradiation Damage by hMnSOD Gene Transfer Ana P. Cotrim, Anne M. Rowzee, A. Sowers, James B. Mitchell, Bruce J. Baum. 75 Intrathecal Injection of HDAd Vectors Results in Long-Term Transgene Expression in Neuroepithelial Cells and Neurons Pasquale Piccolo, Scott Dindot, Nathan Grove, Donna Palmer, Nicola Brunetti-Pierri. 76 Virion-Associated Co-Factor HMGB1 Facilitates Transposition from the HSV/Sleeping Beauty Amplicon Vector Platform Suresh de Silva, Louis T. Lotta, Clark A. Burris, William J. Bowers. 77 hKGF Gene Transfer for the Prevention of Radiation-Induced Salivary Hypofunction Changyu Zheng, Ana P. Cotrim, Anne M. Rowzee, William D. Swaim, Anastasia Sowers, James B. Mitchell, Bruce J. Baum. 78 Generation of Replication-Defective Adenovirus Vector Expressing HCV Structural Gene CE1E2 and Comparative Immunogenicity in Mice with Different Delivery Routes Jie Guan, Yao Deng, Xiao Yin, Wenjie Tan, Xiaobing Wu.

EXHIBITOR PROSPECTUS final program

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

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Abstract Directory Thursday, May 20, 2010  Poster Session I — continued 6:15 PM - 8:15 PM Room: Exhibit Hall C & B-South

Adenovirus and Other DNA Virus Vectors I 79 Interaction between Adenoviral Vector-Transduced Dendritic CellBased Cancer Vaccine and T Regulatory or T Helper 17 Cells Adele Y. Wang, Sarah Q. Crome, Kristy M. Jenkins, Jeffrey A. Medin, Jonathan L. Bramson, Megan K. Levings.

81 FX Domain Fused to Single-Chain Antibodies Improve Oncolytic Effect of Ad5 In Vitro and In Vivo Christopher Y. Chen, Michael A. Barry. 82 Sustained Inhibition of Hepatitis B Virus Replication In Vivo Using Helper-Dependent Adenovirus Vectors Carol Crowther, Betty Mowa, Felix Salazar, Patricia Marion, Abdullah Ely, Patrick Arbuthnot. 83 Adenovirus Serotype 5/35 Is Excellent for Genetic Engineering of Tumor-Directed T Cells Hannah Karlsson, Camilla Lindqvist, Bengt Simonsson, Ulla Olsson-Strömberg, Angelica S. I. Loskog. 84 Bipartite Adenoviral Vector Encoding hHGF and hXIAP for Improved Human Islet Transplantation Hao Wu, A-Rum Yoon, Chae-Ok Yun, Ram I. Mahato. 85 Vector Prime-Protein Boost Vaccine for Patients with Therapy Induced Lymphopenia Tae Hee Han, Yucheng Tang, Yeon Hee Park, Pingchuan Li, Hakan Akbulut, Line Petersen, Jonathan Maynard, Albert Deisseroth. 86 The Effect of Surface Modification of Adenovirus with an ArginineGrafted Bioreducible Polymer on Transduction Efficiency and Immunogenicity in Cancer Gene Therapy Pyung-Hwan Kim, Tae-il Kim, James W. Yockman, Sung Wan Kim, Chae-Ok Yun.

87 Safe and Efficient Delivery of Meganucleases for Lentivirus-Mediated Gene Targeting Araksya Izmiryan, Alix Bourdel, Arnaud Jollet, Frédéric Pâques, Olivier Danos. 88 Site-Specific Genome Modification of Human Primary T Lymphocytes To Improve the Safety and Efficacy of Adoptive Immunotherapy Pietro Genovese, Elena Provasi, Angelo Lombardo, Zulma Magnani, Andreas Reik, Pei-Qi Liu, Oscar Muniz Pello, Jurgen Kuball, Philip D. Gregory, Michael C. Holmes, Philip D. Greenberg, Chiara Bonini, Luigi Naldini. 89 Single-Strand Nicks Induce Homologous Recombination with Less Toxicity Than Double-Strand Breaks Using an AAV Template Michael J. Metzger, Audrey McConnell-Smith, Barry L. Stoddard, A. Dusty Miller. 90 Targeted Gene Insertion in Human Chromosomes Using a Homing Endonuclease Byoung Y. Ryu, Michael T. Certo, Mikhail Garibov, Andrew M. Scharenberg, David J. Rawlings. 91 Cell Synchronization by Serum Shock Facilitates rAAV-Mediated Gene Targeting Yonglun Luo, Emil Kofod-Olsen, Jenny Blechingberg, Rikke Christensen, Nicklas Heine Staunstrup, Lars Bolund, Charlotte Brandt Sørensen. 92 Critical Amino Acid Residues within the PhiC31 Integrase DNA Binding Domain Affect Recombination Activities in Mammalian Cells Raphael Liesner, Wenli Zhang, Nadja Noske, Anja Ehrhardt. 93 Chromatin Structure of Two Genomic Sites for Targeted Transgene Integration in Induced Pluripotent Stem Cells Ruan van Rensburg, Oleg Denisenko, Karol Bomsztyk, Andre Lieber. 94 Targeted Plasmid Integration into the PiggyBac Transposon by an Engineered Zinc Finger Recombinase Charles A. Gersbach, Russell M. Gordley, Thom Gaj, Andrew C. Mercer, Carlos F. Barbas, III. 95 Development of Autonomously Working Zinc-Finger Nuclease Pairs for Targeted Genome Engineering Cem Söllü, Kaweh Montazami-Astaneh, Morgan L. Maeder, Stacey Thibodeau-Beganny, J. Keith Joung, Toni Cathomen. 96 Adeno-Associated Virus-2 (AVV2) Integrase Mediates Specific Integration to Locus AAVS1 on Chromosome 19q13.3 in a Hematopoietic Cell Line Ashley E. Dunfee, Joshua R. Clevenger, Cynthia E. Dunbar, Andre Larochelle.

Thursday, May 20th

80 Replicating Adenoviruses That Harbors the ADP Protein and the Sodium Iodide Symporter Protein (NIS) under the Control of the Major Late Adenovirus Promoter Miguel A. Trujillo, Julia Davydova, Michael J. Oneal, Samantha McDonough, Masato Yamamoto, John C. Morris.

Targeting Gene Modification and Integration

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Abstract Directory Thursday, May 20, 2010 Poster Session I — continued 6:15 PM - 8:15 PM Room: Exhibit Hall C & B-South

Targeting Gene Modification and Integration

Thursday, May 20th

97 Gene Correction in Mouse Liver Hiroyuki Kamiya, Masayuki Uchiyama, Jingshu Piao, Yoshimichi Nakatsu, Teruhisa Tsuzuki, Hideyoshi Harashima. 98 A Transient Cold Shock Enhances Zinc-Finger Nuclease-Mediated Gene Disruption Yannick Doyon, Vivian M. Choi, Danny Xia, Thuy D. Vo, Michael C. Holmes, Philip D. Gregory. 99 Single-Polarity rAAV May Enhance Hepatic Gene Targeting In Vivo Nicole K. Paulk, Markus Grompe.

Physical Methods of Delivery 100 Over-Expression of Adiponectin and Adiponectin Receptor 2 Gene by the Method of Hydrodynamic Gene Delivery Prevents Weight Gain and Development of Insulin Resistance in AKR/J Mice Yongjie Ma, Dexi Liu. 101 Duration of Expression after Gene Electrotransfer to Skin Depends on the Gene of Interest Anita Gothelf, Jens Eriksen, Pernille Hojman, Julie Gehl. 102 Intravenous Delivery of pDNA and siRNA into Muscle by UltrasoundMediated Bubble Liposomes Yoichi Negishi, Yoko E. Takahashi, Yuko Ishii, Ryo Suzuki, Kazuo Maruyama, Yukihiko Aramaki. 103 Prototype Development and Pre-Clinical Immunogenicity Analysis of a Novel Minimally Invasive Electroporation Device Kate E. Broderick, Jay McCoy, Xuefei Shen, Feng Lin, Amir Khan, Matthew Morrow, Stephen Kemmerrer, David B. Weiner, Niranjan Y. Sardesai. 104 Ultrasound-Assisted Non-Viral Gene Transfer to the Salivary Glands Michael J. Passineau, Lee Zourelias, Laurie Machen, Paul C. Edwards, Raymond L. Benza. 105 Development of Electroporation Formulation for Cell Therapy Mike Flanagan, Gang Yu, Jeffry Gimble, Shulin Li.

EXHIBITOR PROSPECTUS final program

106 Ca2+ Electrotransfer; a Quick Stop Procedure Against Transgenic Expression Pernille Hojman, Iben Spanggaard, Julie Gehl, Hanne Gissel. 107 In Vivo Gene Delivery to the Skin by Electroporation Using a Multiple Electrode Array Siqi Guo, Amy Donate, Gaurav Basu, Kathryn Lundberg, Richard Heller. 108 Hydrodynamic Gene Delivery Efficiency Is Reduced in Fibrotic Liver in Rats Tian Zhou, Dexi Liu. 109 Efficiency of Electroporation Delivery of Peptide Nucleic Acid (PNA) Depends on PNA Charge and Electrotransfer Protocol Mette Joergensen, Birgit Agerholm-Larsen, Peter E. Nielsen, Julie Gehl. 110 Biodistribution of Plasmid AMEP, a Potent Antitumor Gene Product for the Treatment of Metastatic Melanomas Celine Bouquet, Elisabeth Dam, Veronique Trochon-Joseph, Valerie Polard, Vincent Sainte-Catherine, Emilienne Soma, Sophie Lebel-Binay. 111 In Vivo Intracellular Injection Needle (IvIn) Technology: A New Needle for Improved Cellular Uptake of DNA in a Live Animal Lars Frelin, Gustaf Ahlén, Matti Sällberg. 112 A Study on Techniques for Safety Assessment of Gene Therapy Products Using Electroporation Min Joung Choi, Il Ung Oh, Sung Yeol Han, Oh Seok Kwon, Seung Hwa Hong. 113 Toxicology and Safety Studies of Plasmid AMEP, a New Efficient Antitumor Gene Product for the Treatment of Metastatic Melanomas Celine Bouquet, Veronique Trochon-Joseph, Valerie Polard, Vincent Sainte-Catherine, Emilienne Soma, Sophie Lebel-Binay. 114 Enhancement of the Immune Response of a Noval DNA Vaccine Encoding Conserved NS3 and Core Fusion Gene of HCV Injected by Intradermal Electrotransfer in Mice Xiao Yin, Jian Lu, Wenjie Tan, Jie Guan, Li Ruan. 115 Electroporation-Enhanced DNA Prime/Protein Boost Strategy To Improve Cross-Clade Immunogenicity of HIV-1 Envelope Antigen Kate E. Broderick, K. Muthumani, Xuefei Shen, Jian Yan, Feng Lin, Janess Mendoza, Amir Khan, Niranjan Y. Sardesai, David B. Weiner. 116 Effectiveness of Impedance Spectroscopy To Monitor Impact of an Electroporation Protocol on Tissue J. Anthony Llewellyn, Loree Heller, Richard A. Gilbert.

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Chemical and Molecular Conjugates I 117 Modular Ternary Polyacridine DNA Polyplexes for Targeted Gene Delivery Nicholas J. Baumhover, Christian A. Fernandez, Sanjib Khargharia, Kevin G. Rice.

119 Interplay of Clathrin and Caveolin Trafficking Pathways in PolymerBased Gene Delivery Mark E. Hwang, Rahul K. Keswani, Daniel W. Pack. 120 Folate Linked Targeting Systems Increase Transfection Efficiency and Specificity in Human Epithelial Cells Nathalie Carmoy, Emmanuel Mornet, Céline Lainé, Loic Lemiegre, Tony Le Gall, Danielle Gillet, Mattias Lindberg, Rémi Marianowski, Pierre Lehn, Thierry Benvegnu, Tristan Montier. 121 In Vivo Gene Delivery from Modified PLG Scaffolds Misael O. Aviles, Lonnie D. Shea. 122 The Influence of Charge Density and Substituent Composition on Imidazolium-Containing Polymers for Mediated DNA Delivery Michael H. Allen, Matthew D. Green, Timothy E. Long. 123 Concentrated PEI Formulations Retain Biological Efficacy Following Long-Term Storage at 4°C Lee A. Davies, Graciela A. Nunez-Alonso, Stephen C. Hyde, Deborah R. Gill. 124 Charge Density and Molecular Weight of Polymeric Gene Carrier Are Key Parameters Influencing Its DNA Compaction Ability and Transfection Efficiency Yong Ren, Xuan Jiang, Hai-Quan Mao. 125 Designing Imidazole-Containing Polymeric Vectors with Controlled Charge Densities for Gene Delivery Matthew D. Green, John M. Layman, Alec S. Good, Sean M. Ramirez, Lulit A. Affin, Michael H. Allen, Timothy E. Long.

127 Role of Glycosaminoglycans in Peptide-Mediated DNA Delivery Rangeetha J. Naik, Pallavi Chandra, Anindo Chatterjee, Munia Ganguli. 128 Galactosylated-DPC Nanoparticles for Targeted Hepatic Gene Delivery In Vivo Keun-Sik Kim, Yeon Kyung Lee, Jung Seok Kim, Jin Ee Back, Sang Il Park, Hwa Yeon Jeong, Young Eun Shin, Yong Serk Park. 129 Effect of Molecular Weight on Plasmid DNA Delivery Efficiency of Palmitic Acid Substituted Polyethylenimine K. C. B. Remant, Breanne Landry, Hasan Uludag. 130 Electrospun Fibers with the Core-Loading of DNA and Poly(ethyleneimine) Complex as Potential Tissue Engineering Scaffold Ye Yang, Long Cheng, Shuhui He, Jie Zou, Fang Chen, Xiaohong Li. 131 A Novel Cationic Lipophosphoramide with Phytanyl Chains: Gain in Transfection Efficiency and Clinical Approach Concerning the Response to Lipofection Mattias Lindberg, Nathalie Carmoy, Christophe Lorilleux, Aurore Fraix, Tony Le Gall, Pierre Lehn, Paul-Alain Jaffrès, Tristan Montier. 132 Cationic Peptide-Based DNA Delivery In Vitro: The Role of DNA Condensation and Release Anita Mann, Garima Thakur, Vasundhara Shukla, Rangeetha Naik, Anand Kamal Singh, Richa Khanduri, Munia Ganguli.

Genetic and Metabolic Diseases Gene & Cell Therapy I 133 Modeling and Correcting Fibrous Dysplasia with Lentiviral Vectors Isabella Saggio, Cristina Remoli, Stefania Cersosimo, Stefania Piersanti, Paolo Bianco, Mara Riminucci. 134 Bone Marrow Transplantation Cures Hemophilia A Antonia Follenzi, Sanj Raut, Simone Merlin, Rita Sarkar, Sanjeev Gupta. 135 Cell Autonomy in Methylmalonic Acidemia (MMA): Implications for Gene and Cell Therapy Irini Manoli, Justin R. Sysol, Randy J. Chandler, Jennifer Sloan, Patricia Zerfas, Victoria Hoffmann, Mones Abu-Asab, Maria Tsokos, Charles P. Venditti.

Thursday, May 20th

118 Biodegradable Polyamino Acid-Based Polycations for Safe and Effective Gene Transfer Minimizing Cumulative Toxicity Keiji Itaka, Takehiko Ishii, Kazunori Kataoka.

126 Bioreducible Poly(amido ethyleneimine) and Its Polyethylene Glycol Graft Copolymer Analog Formulations Offer Ease of Use in Optimizing Improved Nucleic Acid Delivery James W. Yockman, Jonathan H. Brumbach, Young Won Lee, Chao Lin, Johan F. J. Enbersen, Jan Feijen, Sung Wan Kim.

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Genetic and Metabolic Diseases Gene & Cell Therapy I

Thursday, May 20th

136 Efficacious Gene Therapy in Pompe Disease Requires Mannose-6Phosphate Receptor Expression Baodong Sun, Deeksha Bali, Y.-T. Chen, Dwight D. Koeberl. 137 Development of a Safe Hematopoietic Stem Cell (HSC) Mobilization Strategy for Gene Therapy of Thalassemia Evangelia Yannaki, Thalia Papayannopoulou, Erica Jonlin, Ioannis Batsis, Pamela Becker, Fani Zervou, Angeliki Xagorari, Garyfalia Karponi, Varnavas Constantinou, Achilles Anagnostopoulos, Athanasios Fassas, George Stamatoyannopoulos. 138 Long Term Expression in Liver from scAAV Seems Derived from Integrated Vector Genomes Paula S. M. Miranda, Maude Flageul, Christine Kaeppel, Lysbeth ten Bloemendaal, Dominique Aubert, Nowrouzi Ali, Manfred Schmidt, Nicolas Ferry, Piter J. Bosma. 139 Human Amnion Epithelial (AE) Stem Cell Transplant Improves Disease Phenotype and Survival in Mouse Models of Intermediate Maple Syrup Urine Disease (MSUD) K. J. Skvorak, K. Dorko, M. Hansel, F. Marongiu, V. Tahan, K. M. Gibson, Q. Sun, T. Bottiglieri, E. Arning, J. Davila, S. Strom. 140 Gene Therapy Approaches Combining the Immunomodulatory Properties of Liver and IGF-I To Prevent Type I Diabetes Sabrina Tafuro, Xavier M. Anguela, Judith Agudo, Alba Casellas, David Callejas, Mercè Obach, Carles Roca, Albert Ruzo, Fàtima Bosch. 141 Hepatocyte Engineering and Genetic Modification Approaches of Hemophilia Hepatocytes within a Living Mouse Kazuo Ohashi, Kohei Tatsumi, Chise Tateno, Hiroyuki Nakai, Anja Ehrhardt, Rie Utoh, Katsutoshi Yoshizato, Teruo Okano. 142 Serotype Optimization of an AAV Vector for Gene Therapy in Canine and Murine Glycogen Storage Disease Type Ia (GSD-Ia) Xiaoyan Luo, Amanda K. Demaster, Songtao Li, Kyha Williams, Talmage T. Brown, Daniel M. Kozink, Andrew Bird, Dwight D. Koeberl.

EXHIBITOR PROSPECTUS final program

143 Systemic Delivery of Bioactive Glucagon-Like Peptide 1 Following Adenoviral Vector-Mediated Gene Transfer to Murine Submandibular Glands Antonis Voutetakis, Anne M. Rowzee, Ana P. Cotrim, Changyu Zheng, Trushar Rathod, Tulin Yanik, Y. P. Loh, Bruce J. Baum, Niamh X. Cawley. 144 A Non-Viral, GNE-Lipoplex Treatment to Correct Sialylation Defects in Gne-Mutant (M712T) Mice Tal Yardeni, Carla Ciccone, Shelley Hoogstraten-Miller, Daniel Darvish, Yair Anikster, Phil Maples, Chris Jay, William A. Gahl, John Nemunaitis, Marjan Huizing. 145 Lentivirally Induced Over-Expression in the Mouse Hypothalamus; a Study of the Effects of Melanin Stimulating Hormones (MSH) in Obesity and Diabetes Kim Eerola, Siru Virtanen, Eriika Savontaus, Mikko Savontaus. 146 Alternative Methods for Ex Vivo Gene Transfer to Primary Murine B Cell Babak Moghimi, Ou Cao. 147 A Full Characterization of the CAG 140 Huntington’s Disease Mouse Model Aaron C. Rising, Eileen Denovan-Wright, Ron Mandel.

Musculo-skeletal Gene & Cell Therapy I 148 Assessment of Pre-Clinical Safety of GMP Grade GNE-Lipoplexes after Repeat Intravenous Injections in Balb/c Mice Anagha P. Phadke, Chris M. Jay, Phillip B. Maples, Neil Senzer, John Nemunaitis, Alex W. Tong. 149 Pre-Existing Antibodies to AAV8 Attenuates Micro-Dystrophin Expression Following Targeted Vascular Delivery Louise R. Rodino-Klapac, Chrystal Montgomery, William Bremer, Nancy Davis, Kimberly Shontz, Vinod Malik, Katherine Campbell, Thomas J. Preston, Zarife Sahenk, K. Reed Clark, Brian D. Coley, Christopher M. Walker, Jerry R. Mendell, Louis G. Chicoine. 150 AAV Microgene Transfer Reveals the Cellular Motif for DystrophinMediated Sarcolemmal Neuronal Nitric Oxide Synthase (nNOS) Localization Yi Lai, Yongping Yue, Dongsheng Duan. 151 Cell Therapeutic Approach to Duchenne Muscular Dystrophy Using Myogenic Differentiation of Multipotent Mesenchymal Stromal Cells Yuko N. Kasahara, Hiromi H. Kinoh, Hironori Okada, Jin-Hong Shin, Akiyo Nishiyama, Sachiko O. Hosoyama, Michiko W. Maeda, Akinori Nakamura, Takashi Okada, Shin’ichi Takeda.

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Musculo-skeletal Gene & Cell Therapy I 152 Splice Site Strength and Nonsense-Associated Exon Skipping in the DMD Gene Kevin M. Flanigan, Diane M. Dunn, Jerry R. Mendell, Alan Pestronk, Julaine M. Florence, United Dystrophinopathy Project Consortium, Robert B. Weiss.

154 Exon Exchange Approach To Repair Duchenne Dystrophin Transcripts Stéphanie Lorain, Cécile Peccate, Maëva Le Hir, Graziella Griffith, Thomas Voit, Luis Garcia. 155 Gentamicin Treatment of Duchenne Muscular Dystrophy Reinforces the Potential for Mutation Suppression Therapy Vinod Malik, Louise R. Rodino-Klapac, Laurence Viollet, Cheryl Wall, Wendy King, Roula Al-Dahhak, Sarah Lewis, Christopher J. Shilling, Janaiah Kota, John Hayes, John D. Mahan, Katherine J. Campbell, Brenda Banwell, Majed Dasouki, Victoria Watts, Kumaraswamy Sivakumar, Ricardo Bien-Willner, Kevin M. Flanigan, Zarife Sahenk, Richard J. Barohn, Christopher M. Walker, Jerry R. Mendell. 156 Reverse Perfusion Affords Efficient Whole-Body Gene Transfer to Muscle in the Large Animal Frederick Balzer, Jacqueline Farag, Alock Malik, Mihail Petrov, Andrew Mead, Benjamin Kozyak, Kapil Gopal, Marilyn Mitchell, Connie Choi, Jessie Chen, David Low, Charles Yarnall, Charles Bridges, Hansell Stedman. 157 Manganese Superoxide Dismutase Plasmid/Liposome Complex (MnSOD-PL) and Mitochondrial Targeted Nitroxide JP4-039 Ameliorate Irradiation-Induced Delay in Bone Wound Healing Jean-Claude Rwigema, Abhay Gokhale, Michael Epperly, Julie Glowacki, Tracy Dixon, Ken Patrene, G. David Roodman, Peter Wipf, Joel Greenberger. 158 Genetic Correction of Netherton Syndrome Keratinocyte Stem Cells Waseem Qasim, Katerina Semenova, Gill Talbot, John Harper, Adrian Thrasher, Fernando Larcher, Wei Di.

160 Validation of Intravascular Administration of Adeno-Associated Vvirus for Muscular Dystrophies in a Non-Human Primate Model Aravind Asokan, Julia Hegge, Joshua C. Grieger, Chengwen Li, Swati Yadav, Jade J. Samulski , Xiao Xiao, Juan Li, Jon Wolff, R. Jude Samulski, Scott W. J. McPhee.

Infectious Diseases and Vaccines: Vaccine Related Gene Transfer Research 161 Suppression of Cocaine-Induced Hyperactivity in Mice Pre-Treated with an Adenovirus-Based Anti-Cocaine Vaccine Jonathan B. Rosenberg, Martin L. Hicks, Bishnu P. De, Stephen M. Kaminsky, Amira Y. Moreno, Kim D. Janda, Stefan Worgall, Ronald G. Crystal. 162 Translation of Electroporation-Mediated DNA Vaccine Delivery into a Phase I Clinical Trial Evaluating an HIV DNA Vaccine in Healthy Volunteers Claire F. Evans, Sandhya Vasan, Karen E. Dolter, Arlene Hurley, Sarah J. Schlesinger, Drew Hannaman, David F. Gardiner, Daniel P. Dugin, Yaoxing Huang, Josephine Cox, Tony Tarragona, Len Dally, Carol Smith, Claudia Schmidt, Harriet Park, Jill Gilmour, Patricia Fast, Robert Bernard, David D. Ho. 163 Longterm Protective Immunity Against Lethal Pulmonary Challenge with the Pseudomonas aeruginosa Following Mucosal Administration of AdC7-OprF Expressing the P. aeruginosa Protein OprF Anja Krause, Wen Zhu, Yaqin Xu, Ju Joh, Ronald G. Crystal, Stefen Worgall. 164 Characterization of Adenoviruses Shed from the Macaque Gut and Evaluation of Immune Responses to Adenoviruses in the Macaque in the Context of Future Adenoviral Vaccine Studies Soumitra Roy, Roberto Calcedo, C. Angelica Medina-Jaszek, Qiuyue Qin, Jennifer Miliaresis, Surina Boyd, Diane G. Carnathan, Michael R. Betts, James M. Wilson. 165 A Lentiviral HIV Vaccine Candidate Elicits Long-Term Humoral and Cellular HIV-Specific Immunity and Minimal Neutralizing Activity Benyam Asefa, Delia Ye, Nikolay Korokhov, Laurent M. Humeau, Franck Lemiale. 166 Coexpressed RIG-I Agonist Enhances Humoral Immune Response to Influenza DNA Vaccine Jeremy M. Luke, Clague P. Hodgson, James A. Williams.

Thursday, May 20th

153 Long Term Antisense Treatment in Dystrophic Mouse Models for Duchenne Muscular Dystrophy Annemieke Aartsma-Rus, Christa L. de Winter, Hans A. Heemskerk, Judith C. van Deutekom, Gert-Jan B. van Ommen.

159 Adenoviral Human Vascular Endothelial Growth Factor-D Gene Transfer Causes Angiogenesis and Growth and Enlargement of Lymphatic Vessels in Ischemic Mouse Hind Limbs Galina Dragneva, Ivana Kholová, Svetlana Laidinen, Petra Korpisalo, Pasi Tuunanen, Nina Kaskenpää, Suvi Heinonen, Felicitas Yongabi, Anna Stefanska, Varpu Marjomäki, Olli Gröhn, Seppo Ylä-Herttuala.

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Thursday, May 20th

Infectious Diseases and Vaccines: Vaccine Related Gene Transfer Research 167 Genetically Engineered Bifidobacterium Displaying Salmonella-Antigen Protects Mice from Lethal Challenge of Salmonella Typhimurium in a Murine Typhoid Fever Model Toshiro Shirakawa, Sakura Yamamoto, Jun Wada, Takane Katayama, Takumi Jikimoto, Masakuni Nakamura, Shohiro Kinoshita, Kyung-Mi Lee, Masato Kawabata. 168 Signaling Lymphocytic Activation Molecule (SLAM, CD150)-Blind Measles Virus Is Attenuated and Induces Strong Adaptive Immune Responses in Rhesus Monkeys Vincent Leonard, Gregory Hodge, Jorge Reyes-del Valle, Michael McChesney, Roberto Cattaneo. 169 Evaluation of Mismatched Immunity To Expand Cross-Protection Against Influenza Virus Ami Patel, Isabelle Meunier, Kaylie N. Tran, Anders Leung, Stephane Pillet, Darwyn Kobasa, Veronika von Messling, Gary P. Kobinger. 170 Immune Response Against Measles Virus Encoding Helicobacter pylori Neutrophil-Activating Protein Antigen Ianko D. Iankov, Iana Haralambieva, Evanthia Galanis. 171 Generation of Chimeric GB Virus B Clones Encoding the Hepatitis C Virus E1E2p7 and coreE1E2p7 toward the Infected Marmoset Models Tingting Li, Lifang Shuai, Zixuan Chen, Anqi Wang, Wenjing Wang, Chengyao Li. 172 Characterization of Immune Response in Mice Induced by Recombinant Vaccinia Virus (Tiantan) Based Multivalent H5N1 Avian Influenza Vaccines Wen Wang, Yao Deng, Wenjie Tan, Hong Chen, Li Ruan, Yuelong Shu. 173 The Effects of the Calreticulin on the Immune Response Induced by DNA Vaccine Containing the Classical Swine Fever Virus E2 Gene in Mouse Model Ming Kun Hsieh, Chia Chin You, Chienjin Huang. 174 Evaluation of the Multi-Electrode Array for Use in DNA Vaccination Against Bacillus anthracis Amy L. Donate, Richard Heller.

EXHIBITOR PROSPECTUS final program

Cancer – Immunotherapy I 175 Enhancing the Priming of Tumor Antigen Specific T Cell Responses Using Oncolytic Viruses Phonphimon Wongthida, Rosa Diaz, Feorillo Galivo, Karen Kaluza, Jill Thompson, Timothy Kottke, Richard Vile. 176 AdCD40L Immunogene Therapy for Bladder Carcinoma – A Phase I/ IIa Trial Per-Uno Malmstrom, Angelica S. I. Loskog, Camilla A. Lindqvist, Sara M. Mangsbo, Moa Fransson, Alkwin Wanders, Truls Gardmark, Thomas H. Totterman. 177 Treatment of Refractory Cancer Patients with a Serotype 5/3 Chimeric Oncolytic Adenovirus Expressing Granulocyte Macrophage Colony-Stimulating Factor (GM-CSF): Virological, Immunological and Clinical Endpoints Anniina Koski, Sari Pesonen, Vincenzo Cerullo, Sophie Escutenaire, Elina Haavisto, Lotta Kangasniemi, Pekka Simula, Timo Joensuu, Akseli Hemminki. 178 IL-15 Reduces Suppressive Effects of Regulatory T Cells (Tregs) on Antigen-Specific Cytotoxic T Lymphocytes (CTLs) S. Perna, B. De Angelis, D. Pagliara, L. Zhang, C. Rooney, H. Heslop, M. Brenner, G. Dotti, B. Savoldo. 179 PiggyBac Transposon Modified EBV Specific Human T Lymphocytes for HER2-Positive Cellular Immunotherapy In Vivo Yozo Nakazawa, Nabil Ahmed, Vita Salsman, Ann Leen, Stephen Gottschalk, Matthew H. Wilson, Cliona M. Rooney. 180 Adenovirus-Mediated Intratumoral Expression of Immunostimulatory Molecules Together with Systemic Depletion of Regulatory T-Cells Induces Tumor-Destructive Immune Responses in Mouse Models Ying Liu, Jonas Persson, Sebastian Tuve, Roma Yumul, ZongYi Li, Steve Roffler, Andre Lieber. 181 Key Role of B Cell as Antigen Presenting Cells in the Anti-Tumor Immunity Induced by a Combined Immune-Stimulatory/Conditional Cytotoxic Therapy for Glioblastoma Marianela Candolfi, Kader Yagiz, James F. Curtin, Hikmat Assi, Mia Wibowo, Gabrielle Alzadeh, David Foulad, Kurt Kroeger, Anderson Alden, Chunyan Liu, Robert Dunn, Pedro R. Lowenstein, Maria G. Castro. 182 Genetically Engineered Malignant Glioma-Specific T Cells Resistant to Glucocorticoids Seogkyoung Kong, Richard P. Junghans, Prakash Sampath. 183 Adoptive Transfer of T Cells Genetically Targeted to the MUC16 Antigen Eradicates Established Peritoneal Ovarian Tumors in SCIDBeige Mice Alena A. Chekmasova, Thapi D. Rao, David R. Spriggs, Renier J. Brentjens.

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Cancer – Immunotherapy I

185 A New Human-Derived Reporter Gene Suitable for Clinical PET Imaging of T-Cell Trafficking Yury Likar, Juan Zurita, Larissa Shenker, Maxim Moroz, Shangde Cai, Anton Neschadim, Jeffrey Medin, Hedvig Hricak, Michel Sadelain, Vladimir Ponomarev. 186 Inhibition of Mice Colon Tumor Growth by Il-15 Immunogene Therapy Xianghui He, Weidong Li, Yifeng Wang, Linan Hou, Liwei Zhu. 187 Development of High-Performance HVJ-E for Cancer Immune Therapy Kotaro Saga, Katsuto Tamai, Takehiko Yamazaki, Yasufumi Kaneda. 188 Combined Gene Therapy Using Adenovirus Expressing Interleukin-12, Granulocyte-Macrophage Colony-Stimulating Factor, and Thymidine Kinase with Prodrug Ganciclovir Ji-seong Kim, Kyoung-ju Choi, Chae-Ok Yun. 189 Safety Data of MGN1601, a Tumor Vaccine, Made of Allogeneic, Transfected and Irradiated Tumor Cells in Combination with an Immunomodulator for the Treatment of Metastatic Renal Cell Carcinoma Manuel Schmidt, Baerbel Volz, Matthias Schroff, Kerstin Kapp, Christiane Kleuss, Marina Tschaika, Burghardt Wittig. 190 Development of an HLA-A*0201 Restricted T Cell Receptor That Recognizes Peptide Epitopes from MAGE-A3 and MAGE-A12 for Targeted Adoptive T Cell Immunotherapy of Multiple Cancer Types Nachimuthu Chinnasamy, Jennifer A. Wargo, Zhiya Yu, Timothy L. Frankel, John P. Riley, Maria R. Parkhurst, Steven A. Feldman, Nicholas P. Restifo, Paul F. Robbins, Steven A. Rosenberg, Richard A. Morgan.

191 (Moved to Oral Abstract Session 413) A Comparative Study of Stem Cell-Based Cell Carrier Systems for the Systemic Delivery of an Oncolytic Adenovirus for Antiglioma Therapy Matthew A. Tayler, Atique U. Ahmed, Ilya V. Ulasov, Irina Y. Balyasnikova, Maciej S. Lesniak. 192 A Novel Coxsackievirus B Shows Remarkable Oncolytic Capacity Against Lung Cancer Shohei Miyamoto, Hiroyuki Inoue, Meiko Yamada, Xin Meng, Takafumi Nakamura, Yasuo Urata, Hiroyuki Shimizu, Kenzaburo Tani. 193 Preclinical and Clinical Data with a Fully Serotype 3 Oncolytic Adenovirus Ad3-hTERT-E1A in the Treatment of Advanced Solid Tumors Otto Hemminki, Gerd Bauerschmitz, Silvio Hemmi, Sergio Lavilla-Alonso, Iulia Diaconu, Anna Kanerva, Vincenzo Cerullo, Minna Oksanen, Elina Haavisto, Timo Joensuu, Sari Pesonen, Akseli Hemminki. 194 Mechanism of Tumour Penetration by Oncolytic Viruses – Investigation in a 3 Dimensional In Vitro Model C. A. Campion, C. Harty, G. C. O’Sullivan, J. K. Collins. 195 Mechanisms of Cyclophophamide Enhancing Anti-Tumor Effects of Oncolytic Vesicular Stomatitis Virus for Breast Cancer Treatment Hongxun Wang, Stephen Russell, Jian Qiao. 196 Clinical Characterization of the Adenovirus Death Protein-Potentiated Oncolytic Adenovirus VRX007 Alex W. Tong, Anagha P. Phadke, Staci Horvath, William Wold, John Nemunaitis, Neil Senzer. 197 Safety and Immunological Effects of Corticosteroids in Patients Treated with Oncolytic Adenoviruses Maria Rajecki, Mari Raki, Andreas Helminen, Sophie Escutenaire, Sari Pesonen, Vincenzo Cerullo, Timo Joensuu, Elina Haavisto, Laura Ahtiainen, Akseli Hemminki. 198 Effect of Decorin on Overcoming the Extracellular Matrix Barrier for Oncolytic Virotherapy Il-Kyu Choi, Young-Sook Lee, Ji Young Yoo, A-Rum Yoon, Hye Jin Choi, Daniela G. Seidler, Chae-Ok Yun. 199 Chondroitinase ABC1 Mediated Enhancement of OV Spread and Efficacy Nina Dmitrieva, Mariano Viapiano, Balveen Kaur. 200 Use of PET Imaging for In-Vivo Monitoring of Replication Competent Retrovirus (RCR) Transduction of Malignant Gliomas Weijun Wang, Hui Wang, Ryan Park, Hancheng Cai, Peter S. Conti, Nori Kasahara, Thomas C. Chen.

Thursday, May 20th

184 Adoptive Transfer of Autologous T Cells Targeted to Prostate Specific Membrane Antigen (PSMA) for the Treatment of Castrate Metastatic Prostate Cancer Oriana Borquez-Ojeda, Jolanta Stefanski, Xiuyan Wang, Jason Plotkin, Jinrong Qu, Luis Rodriguez, Elliot Servais, Kei Suzuki, Larrisa Shenker, Vladimir Ponomarev, Susan Slovin, Michel Sadelain, Isabelle Rivière.

Cancer-Oncolytic Viruses I

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Cancer-Oncolytic Viruses I

Thursday, May 20th

201 Systemic Cancer Screening Using an Oncolytic HSV To Induce TumorSelective Expression of a Secretable Serum Biomarker Andrew W. Browne, Jennifer L. Leddon, Mark A. Currier, Jon P. Williams, Nancy Ratner, Timothy P. Cripe. 202 Therapeutic and Tumor-Specific Immunity Induced by Combination of Dendritic Cells and Oncolytic Adenovirus Expressing IL-12 and 4-1BBL Song-Nan Zhang, Jing-Hua Huang, Joo-Hang Kim, Kyung-Ju Choi, Il-Kyu Choi, Mingul Lee, Chae-Ok Yun. 203 Oncolytic Herpes Simplex Virus Encoding Interferon b Gene for Squamous Cell Carcinoma of the Head and Neck Hongtao Li, Stephen J. Russell. 204 Targeted Suicide Gene Therapy with Oncolytic Adenovirus Coding for FCU1, a Fusion Protein of Cytosine Deaminase and Uracilphosphoribosyltransferase João D. Dias, Ilkka Liikanen, Kilian Guse, Johann Foloppe, Marta Sloniecka, Iulia Diaconu, Ville Rantanen, Minna Eriksson, Tanja Hakkarainen, Monika Lusky, Philippe Erbs, Sophie Escutenaire, Anna Kanerva, Sari Pesonen, Vincenzo Cerullo, Akseli Hemminki.

Cancer-Targeted Gene & Cell Therapy I

209 Therapeutic Efficacy of a Lentiviral Mediated Thymidine Kinase/ Ganciclovir Treatment in an Immunocompetent Rat Malignant Glioma Model Jere T. Pikkarainen, Hanna P. Lesch, Haritha Samaranayake, Minna U. Kaikkonen, AnnMarie Määttä, Seppo Ylä-Herttuala. 210 Insertion of Transcription Factor Nuclear Factor Kappa B Binding Sites into Plasmid DNA Expressing a Suicide Gene Facilitates Increased Small Cell Lung Cancer Cell Death In Vitro Due to Increased Nuclear Uptake Frederik Cramer, Camilla L. Christensen, Hans S. Poulsen. 211 Preirradition Gene Therapy Against Transforming Growth Factor-b Signal Is Radioprotective for the Liver Zhao-Chong Zeng, Shi-Suo Du, Ming Qiang, Zheng-Yu Zhang. 212 Rabbit VX-2 Brain Tumor Model for Development of Improved Vector Administration Techniques Farizan Ahmad, Agnieszka Pacholska, Anna Hyvärinen, Venla Tuppurainen, Thomas Wirth, Seppo Ylä-Herttuala. 213 Nano-Flow 2D-HPLC MALDI-TOF MS/MS for Differentially Expressed Protein Analysis in Breast Cancer Patient Samples Courtney Haddock, R. B. Holtz, Neil Senzer, Phillip B. Maples, John Nemunaitis. 214 In Vitro Characterization for In Vivo Translation of a Novel Non-Viral Gene Delivery Vector Luke J. Higgins, Gloria L. Hwang, Regina H. Katzenberg, Nishita Kothary, Daniel Y. Sze, Lawrence V. Hofmann.

205 Controlled Nuclear Localization of p53 in Breast Cancer Cells Mohanad Mossalam, Carol S. Lim.

215 Altering the Tropism of Herpesvirus saimiri to Selectively Infect Hepatocellular Carcinoma (HCC) Cells Susan Turrell, Adrian Whitehouse.

206 Effect of Transcriptionally Targeted Suicide Gene Therapy for Small Cell Lung Cancer In Vitro and In Vivo by DOTAP/Cholesterol- and Adenoviral-Mediated Delivery Camilla L. Christensen, Torben Gjetting, Frederik Cramer, Skovgaard Hans.

216 Improved Gene Transfer into Renal Carcinoma Cells Using an Adenovirus Vector Shuji Terao, Bishnu Acharya, Toru Suzuki, Katsuyuki Hamada, Hiroyuki Mizuguchi, Akinobu Gotoh.

207 NF-kB Activity Regulates MSC Accumulation at Tumor Sites Ryosuke Uchibori, Hiroyuki Mizuguchi, Tomonori Tsukahara, Masashi Urabe, Hiroaki Mizukami, Akihiro Kume, Keiya Ozawa.

217 Prostate Cancer Gene Therapy Using Adenovirus Expressing a Novel Apoptosis-Inducing Gene and Its Conjugation with Chemotherapy Xiongwen Zhang, Ben Beheshti, Yi Lu.

208 Dynamics of Therapy of Malignant Melanoma with Vesicular Stomatitis Virus Jyoti Dev, Chetan P. Offord, Diana M. Rommelfanger, Richard G. Vile, Zeljko Bajzer, David Dingli.

218 Development and Preclinical Testing of Large Batches of SNS01-A – An eIF5A-Based Gene Therapy Nanoparticle Designed for the Treatment of Multiple Myeloma Catherine A. Taylor, Zhongda Liu, Terence Tang, Bin Ye, Bruce Galton, Kathleen A. Donovan, John A. Lust, John E. Thompson, Richard Dondero.

EXHIBITOR PROSPECTUS final program

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Abstract Directory Thursday, May 20, 2010  Poster Session I — continued 6:15 PM - 8:15 PM Room: Exhibit Hall C & B-South 219 Angiotensin II Type 2 Receptor Gene Mediated Apoptosis of Human Lung Adenocarcinoma A549 Cells Yanling Zhang, Ling Zhang, Yongxin Gao, Baihong Chen, Hongwei Li.

Hematologic and Immunologic Gene & Cell Therapy I

221 Use of the Natural Human Artemis Promoter To Regulate Human Artemis Expression Prevents Toxicity and Corrects Artemis Deficient SCID after Ex Vivo Lentiviral Transduction in a Murine Model of the Disease Megan M. Multhaup, Kelly Podetz-Pedersen, Sweta Gurram, Andrea Karlen, Debra Swanson, Nikunj V. Somia, Bruce R. Blazar, Morton J. Cowan, R. Scott McIvor, McIvor Lab. 222 Lack of Enhancer-Blocking Activity by 400bp cHS4 Insulator in a Human T- Lymphoid Cell line Disha A. Mody, Taihe Lu, Sheng Zhou, John Gray, Brian P. Sorrentino. 223 Liver-Directed Intravascular Fetal Delivery of scAAV Facilitates Sustained Transgene Expression with Evidence of Transplacental Trafficking and Possible Vector Integration Events C. N. Mattar, A. C. Nathwani, N. Dighe, S. Waddington, J. McIntosh, A. Biswas, N. Fisk, A. M. Davidoff, A. David, D. Peebles, A. Nowrouzi, C. Kappel, M. Schmidt, M. Choolani, J. Chan. 224 Helper-Dependent Adenoviral Vector Gene Therapy Mediates LongTerm Low-Level Correction of Hemophilia A in the Murine Model after Neonatal Gene Transfer Chuhong Hu, Fides D. Lay, Racel G. Cela, Masataka Suzuki, Joseph Springer, Ronald W. Busuttil, Brendan Lee, Gerald S. Lipshutz. 225 Lentiviral Vector Mediated Gene Therapy for the Treatment of Wiskott-Aldrich Syndrome: In Vitro and In Vivo Preclinical Studies with Human CD34+ Cells Samantha Scaramuzza, Marita Bosticardo, Anna Ripamonti, Maria C. Castiello, Luca Biasco, Giuliana Vallanti, Marina Radrizzani, Anne Galy, Robbert Bredius, Barbara Cappelli, Maria G. Roncarolo, Luigi Naldini, Anna Villa, Alessandro Aiuti.

227 Development of a Gene Transfer System for Wiskott-Aldrich Syndrome Using Foamy Virus Vectors Toru Uchiyama, Masaki Shimizu, Fabio Candotti. 228 Integration Site Distribution in Mice Following Therapeutic Gene Transfer To Treat Beta-Thalassemia Keshet Ronen, Olivier Negre, Nirav Malani, Maria Denaro, Beatrix Gillet-Legrand, Philippe Leboulch, Julian D. Down, Emmanuel Payen, Frederic D. Bushman. 229 Validation of the Tranduction of Patients Hematopoietic Progenitor Cells with a Clinical-Grade Lentiviral Vector Developed for WiskottAldrich Syndrome Gene Therapy Salima Hacein-Bey Abina, Elena De Falco, Johanna Blondeau, Sabine Charrier, Laure Caccavelli, Mike Blundell, Alain Fischer, Alessandro Aiuti, Adrian J. Thrasher, Marina Cavazzana-Calvo, Anne Galy. 230 Generation of an Optimized Lentiviral Vector Encoding a HighExpression fVIII Transgene for Gene Therapy of Hemophilia A Jennifer M. Johnston, Robby Moot, Bagirath Gangadharan, Christopher B. Doering, H. Trent B. Spencer. 231 Biodistribution of a High Dose of Intraarticularly Administered AAV-5 Vectors to Lewis Rats Scott A. Loiler, Karin de Cortie, Paul P. Tak, Margriet J. Vervoordeldonk. 232 Ex Vivo Lentiviral Transduction of the Human Purine Nucleoside Phosphorylase (PNP) Gene in Bone Marrow Derived Hematopoietic Stem Cells: An In Vivo Model of Gene Therapy for PNP Deficient T-Cell Immunodeficiency Kelly M. Podetz-Pedersen, Megan M. Multhaup, Andrea D. Karlen, Debra L. Swanson, K. Scott Baker, Nikunj V. Somia, R. Scott McIvor. 233 Signed Outside: A Surface Marker System for Transgenic Cytoplasmic Proteins Vital Wohlgensinger, Reinhard A. Seger, Martin D. Ryan, Janine Reichenbach, Ulrich Siler. 869 (Moved from Poster Session III) Myelospecific Self-Inactivating Gammaretroviral Vectors for p47phox CGD Gene Therapy Vital Wohlgensinger, Christian Brendel, Axel Schambach, Manuel Grez, Janine Reichenbach, Reinhard A. Seger, Ulrich Siler.

Thursday, May 20th

220 Modeling X-Linked Chronic Granulomatous Disease Using Neutrophils Differentiated from Patient-Derived Induced Pluripotent Stem Cells Colin L. Sweeney, Jizhong Zou, Uimook Choi, Bin-Kuan Chou, Linzhao Cheng, Harry L. Malech.

226 Development of B-Lineage Specific Lentiviral Vectors for Use in Gene Therapy for B-Cell Disorders Blythe D. Sather, Brigid V. Sterling, Byoung Y. Ryu, Stéphanie Baron, David J. Rawlings.

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Abstract Directory Thursday, May 20, 2010 Poster Session I — continued 6:15 PM - 8:15 PM Room: Exhibit Hall C & B-South

Gene Regulation

Thursday, May 20th

234 Low Levels of Expression of CD18 Using the pgk Promoter in a Foamy Viral Vector To Treat Animals with Canine Leukocyte Adhesion Deficiency Thomas R. Bauer, Jr., Erik M. Olson, Laura M. Tuschong, James M. Allen, Yi Li, Tanya H. Burkholder, Dennis D. Hickstein, David W. Russell. 235 A Dual Self-Inactivating Lentiviral Vector System for MifepristoneInducible Bicistronic Transgene Expression in Primary Neural Progenitor Cells Michael A. Mooney, Kenneth H. Fischbeck, Tyler Mark Pierson. 236 A Novel Human Artificial Chromosome Vector Derived from Chromosome 14 (14HAC): Higher Transgene Expression System Using Sub-Telomere Sequences Minoru Kakeda, Keiko Nagata, Akiko Sano, Kanako Osawa, Akiyo Okazaki, Shingo Shitara, Yasuhiro Kazuki, Mitsuo Oshimura, Kazuma Tomizuka. 237 Effective Gene Delivery to Wounds by Transplantation of Minced Skin Particles Transfected with Adenoviral-VEGF Taro Koyama, Florian Hackl, Pejman Aflaki, Juri Bergmann, Baraa Zuhaili, Emily Waisbren, Feng Yao, Elof Eriksson. 238 MicroRNA-122 Regulation Prevents Liver Toxicity of Suicide Gene Therapy Gang Wang, Wenhong Tian, Yunfan Liu, Wenjie Tan, Xiaobing Wu. 239 Regulation of Endogenous GAD67 Expression Using Artificial Transcription Factors Erika Elgstrand Wettergren, Fredrik Gussing, Maike Sest, Torbjörn Gräslund, Cecilia Lundberg. 240 Enhanced In Vivo Transgene Expression by Two-Step Transcription Amplification Genki Kanda, Hiroshi Ochiai, Hideyoshi Harashima, Hiroyuki Kamiya. 241 Targeted Knockdown of Serum Response Factor in Hearts of Adult SRF Conditional Knock-Out Mice by AAV9-Mediated Transfer of CreRecombinase Stanislas M. Werfel, Andreas Jungmann, Rolf Sprengel, Jürgen A. Kleinschmidt, Alfred Nordheim, Hugo A. Katus, Oliver J. Müller.

EXHIBITOR PROSPECTUS final program

242 Pro-Proliferative Functions of Drosophila Small Mitochondrial Heat Shock Protein 22 in Human Cells Jihoon Ryu, Renu Wadhwa, Ran Gao, Il-Kyu Choi, Genevieve Morrow, Kamal Rajput, Inwook Kim, Sunil C. Kaul, Robert M. Tanguay, Chae-Ok Yun. 243 Interleukin-(IL)-24 Regulates Epithelial to Mesenchymal Transition (EMT) Transcription Factors Corbin Goerlich, Manish Shanker, Jiankang Jin, Sendurai Mani, Rajagopal Ramesh. 244 Transient Epigenetic Gene Therapy as a Rising Arm of a Universal Stem Cell Gene Therapy Platform both for Ex Vivo and In Vivo Applications Roger Bertolotti.

Oligonucleotide & RNAi Therapeutics I 245 The Application of “Bi-Functional” Short Hairpin RNA in A FurinshRNA and GM-CSF (FANG) Autologous Vaccine for Cancer Therapy Zhaohui Wang, Connor Phalon, Padmasini Kumar, Chris M. Jay, Donald D. Rao, Alex W. Tong, Phillip B. Maples, Neil Senzer, John Nemunaitis. 246 A pH-Sensitive Fusogenic Peptide Modified siRNA-Encapsulating Nanoparticles Exerts the Gene Silencing Synergistically with TumorSpecific Cleavable PEG-Lipid Yu Sakurai, Hiroto Hatakeyama, Hidetaka Akita, Motoi Oishi, Yukio Nagasaki, Shiroh Futaki, Hideyoshi Harashima. 247 Systemic Administration of Bifunctional shRNA Lipoplex Targeting PDX-1 for Murine Model of Insulinoma and Pancreatic Cancer Shihe Liu, Zhaohui Wang, Chris M. Jay, Donald D. Rao, Nancy S. Templeton, Phillip B. Maples, Neil Senzer, F. C. Brunicardi, John Nemunaitis. 248 Augmentation of Docetaxel Anti-tumor Activity by a StathminSpecific Bi-Functional shRNA Alex W. Tong, Yang Yu, Donald D. Rao, Phillip B. Maples, Neil Senzer, John Nemunaitis. 249 RNAi- and U1i-Mediated Gene Therapy Annemart Koornneef, Pavlina Konstantinova, Bas Blits, Richard van Logtenstein, Tita Ritsema, Puri Fortes, Harald Petry. 250 Lentiviral Delivery of EzH2 shRNA Inhibits Tumor Growth of Epithelial Ovarian Cancer Ramesh B. Batchu, Aamer M. Qazi, Assaad Semaan, Shelly M. Seward, Sreedhar Chamala, Christopher S. Bryant, Robert Morris, Sanjeev Kumar, Christopher Steffes, Masood A. Shammas, Donald W. Weaver. 251 Prostate Tumor Radiosensitization through PSMA-Aptamer-Targeted siRNA Knock-Down of DNA Repair Pathways Xiahoua Ni, Yonggong Zhang, Theodore L. DeWeese, Shawn E. Lupold.

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Abstract Directory Thursday, May 20, 2010  Poster Session I — continued 6:15 PM - 8:15 PM Room: Exhibit Hall C & B-South

Oligonucleotide & RNAi Therapeutics I 252 Preclinical Safety and Toxicity Assessment of Stathmin 1 Bifunctional shRNA Formulation Following a Single Intravenous Injection in Rats Anagha P. Phadke, Chris M. Jay, Donald D. Rao, Neil Senzer, Phillip B. Maples, John Nemunaitis, Alex W. Tong.

254 The Novel Comb Shaped Amphiphilic Polyallylamine for Potential Oral Delivery of siRNA to Gastrointestinal Cancers: Physicochemical Characterizations and In Vitro Evaluations Jianfeng Guo, Colin J. Thompson, Woei Ping Cheng, Caitriona O’Driscoll. 255 Optimalization of Polymerase II sh/miRNA Expression for Therapeutic Applications Piotr Maczuga, Tita Ritsema, Annemart Koorneef, Harald Petry, Sander van Deventer, Pavlina Konstantinova. 256 The Effect of Helper Lipids on Cationic Lipid-Based siRNA Delivery System Guoqin Niu, Hartmut Derendorf, Jeffrey A. Hughes. 257 c-Cbl shRNA-Expressing Adenovirus Functions as a Sensitizer of TRAIL-Induced Apoptosis Seeun Oh, So Y. Kim, Bo K. Sun, Dongho Kim, Suhung Lee, Yong J. Lee, Joo-Hang Kim, Jae J. Song. 258 ncRNAs Originating from the Dystrophin Gene as Biomarker for Assessing Antisense Therapy Matteo Bovolenta, Annarita Armaroli, Marcella Neri, Simona Brioschi, Marina Fabris, Chiara Scotton, Sofia Falzarano, Paola Rimessi, Emanuele Valli, Giovanni Perini, Luciano Merlini, Francesca Gualandi, Alessandra Ferlini. 259 Efficient In Vivo Delivery of Cholesterol-Conjugated siRNA to the Liver Using Endogenous Chylomicron Remnant Kazutaka Nishina, Kie Yoshida, Yoshitaka Uno, Hiroya Kuwahara, Wenying Piao, Hidehiro Mizusawa, Takanori Yokota.

260 Controlled Nonviral Gene Delivery and Expression Using Stable Neural Stem Cell Line Transfected with a Hypoxia-Inducible Gene Expression System Yoon Ha, MengLu Liu, KeungNyun Kim, DoHeum Yoon. 261 Non-Viral Carriers Deliver mRNA Encoding Marker Proteins in HeLa cells and mRNA Encoding CXCR4 in Mesenchymal Stem Cells Joanna Rejman, Geertrui Tavernier, Joseph Demeester, Stefaan S. D. Desmedt. 262 Genetic Modification of Neural Stem Cells Ravi K. Chandra. 263 APOA-1 Is a Novel Marker of Erythroid Cell Maturation from Hematopoietic Stem Cells in Mice and Humans Tomoko Inoue, Daisuke Sugiyama, Ryo Kurita, Tatsuo Oikawa, Kasem Kulkeaw, Hirotaka Kawano, Yoshie Miura, Michiyo Okada, Youko Suehiro, Atsushi Takahashi, Tomotoshi Marumoto, Hiroyuki Inoue, Norio Komatsu, Kenzaburo Tani. 264 A Stem Cell Gene Therapy Approach for Enhancing the Efficacy of Breast Cancer Therapy by Trastuzumab (Herceptin) Ines Beyer, Jonas Persson, ZongYi Li, Ying Liu, Roma Yumul, Andre Lieber. 265 Ovarian Cancer Stem Cells Are in an Epithelial/Mesenchymal Hybrid State and Have Multipotential Capacity for Differentiation Robert Strauss, ZongYi Li, Ying Liu, Nicole Urban, Charles Drescher, Jiri Bartek, Andre Lieber. 266 SB-T-1214 Dramatically Downregulates Stem Cell-Related Gene Expression Profiles in Metastatic Colon Cancer Cells Yan Leyfman, Yuan Wang, Iwao Ojima, Galina Botchkina. 267 Identifying Genes and Signaling Pathways Regulating the Quiescent State in Muscle Stem (Satellite) Cells Kristy Boyle, Jeffrey S. Chamberlain. 268 Pathway Analysis of Multipotent Mesenchymal Stromal Cells Using BacMam Tools Lucas Chase, Thomas Machleidt, Mahendra S. Rao, Uma Lakshmipathy. 269 The Placental Microvasculature Is a Rich Source of Highly Proliferative Endothelial Colony Forming Cells Capable of Developing Functioning Vessels In Vivo Brian M. Rapp, Reza Saadatzadeh, Janak Bhasvar, Richard Ofstein, Michael P. Murphy. 270 microRNA Function during Human Mesenchymal Stem Cell Adipogenic Differentiation Annette Enright, Vladimir Benes, Georgina Shaw, Frank Barry, Linda Howard.

Thursday, May 20th

253 Application of siRNA-Containing Nanoparticles for In Vitro and In Vivo Gene Silencing in Tumor by Controlling Intracellular Trafficking Hiroto Hatakeyama, Erika Ito, Hidetaka Akita, Motoi Oishi, Yukio Nagasaki, Shiroh Futaki, Hideyoshi Harashima.

Stem Cell Biology

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Abstract Directory Thursday, May 20, 2010 Poster Session I — continued 6:15 PM - 8:15 PM Room: Exhibit Hall C & B-South

Stem Cell Biology

Thursday, May 20th

271 A Novel Fluorescence/Fluorine MRI Dual-Mode Reagent for Cell Tracking and Determination of Cell Fate Anthony G. Balducci, Brooke Helfer, Amy K. Wesa, Jelena Janjic, Eric Ahrens. 272 The Stromal Vascular Fraction of Human Term Placenta Is a Rich Source of Pluripotent Mesenchymal Stem Cells Mohammad R. Saadatzadeh, Janak Bhavsar, Brian M. Rapp, Peter J. Moreno, Michael P. Murphy, Michael P. Murphy. 273 The HSV-2 Protein, ICP10PK, Potentiates the Neuroprotective Activity of Stem Cells Kyle A. Wilson, Jennifer M. Laing, Aurelian Laure.

Cell Processing and Vector Production I 274 Process Improvements for cGMP Manufacture of Lexgenleucel-T for HIV Treatment Reuben Cohen, Yelena Skripchenko, Tony Encinas, Gerard J. McGarrity, Shuyuan Zhang. 275 TAG Vaccine Clinical Trial Manufacturing Phillip B. Maples, Padmasini Kumar, Yang Yu, Beena O. Pappen, Joseph Kuhn, John Nemunaitis. 276 Development and GMP Validation of Large Scale Lentiviral Transduction of CD34+ Cells for Metacromatic Leukodystrophy and Wiskott-Aldrich Syndrome Gene Therapy Clinical Trials Giuliana Vallanti, Chiara Pozza, Francesca Rossetti, Letizia Martelli, Giuseppina Marano, Alessandra Gatti, Lucia Turchetto, Paola Massariello, Monica Salomoni, Claudia Benati, Sergio Fracchia, Alessandro Lorini, Marco Dieci, Raul Ziliotto, Alessandra Biffi, Alessandro Aiuti, Claudio Bordignon, Luigi Naldini, Marina Radrizzani. 277 Large Scale rVSV Viral Vector Production in a cGMP Facility Lara J. Ausubel, Marcia Meseck, Ivy Derecho, Patricia Lopez, Christine Knoblauch, Ross McMahon, Jonathan Anderson, Nina Dunphy, Valerie Quezada, Mathew Luo, David Hsu, Savio Woo, Larry Couture. 278 Challenges in the Manufacturing of Retroviral Gene Therapy Vectors Ana F. Rodrigues, Ana I. Amaral, Manuel J. T. Carrondo, Paula M. Alves, Ana S. Coroadinha.

EXHIBITOR PROSPECTUS final program

279 Production of cGMP Grade Lentiviral Vector Lara J. Ausubel, Christine Knoblauch, Patricia Lopez, Ross McMahon, Jonathan Anderson, Valerie Quezada, Nina Dunphy, Ivy Derecho, Mathew Luo, David Hsu, Larry Couture. 280 Design and Operation of a New State-of-the-Art Multi-Product cGMP Cleanroom Facility for Vector Production and Cell Manipulation Johannes C. M. van der Loo, William P. Swaney, Kathryn Mangopoulos, Michele Beuerlein, Diana L. Nordling, Lilith R. Reeves, Punam Malik. 281 Highly Efficient Manufacturing of Clinical Grade Lentiviral Vector in a New, State of the Art Academic Good Manufacturing Practice (GMP) Facility Gerhard Bauer, Joseph Anderson, Matt Lindsey, Jan Nolta. 282 The Role of JNK in Lentivirus Gene Transfer Meng Horng Lee, Roshan Padmashali, Stelios T. Andreadis.

Late Breaking Science I 922 HSV-mediated Transfer of the Sox11 Gene and Peripheral Nerve Regeneration Xiaotang Jing, Shaohua Huang, Joseph C. Glorioso, Kathryn M. Albers 923 Real-Time In Vivo Imaging of Neural Stem Cells for Targeted Therapy of Medulloblastoma Margarita Gutova, Vazgen Kandalyyan, Ira Harutyunyan, Joseph Najbauer, Rex A. Moats, Gregory M. Shackleford, Michael E. Barish and Karen S. Aboody 924 Clinical Trial Entry of a Novel Prodrug Activator Gene Transfer Technology for the Treatment of Cancer H. Gruber, C. Ibanez, A. daSilva, W. Gunzburg, D. Pertschuk, D. Gessner, N. Kasahara, C.R. Logg, O. Perez, D. Ostertag, J. Robbins, D.J. Jolly 925 Integration-Defective Lentiviral Vectors for Hemophilia: A Safer Platform for Immune Tolerance Induction and Reduced Genotoxicity Janka Mátrai, Alessio Cantore, Cynthia C. Bartholomae, A Annoni, Abel AcostaSanchez, Ermira Samara-Kuko, Liesbeth De Waele, Ling Ma, Tim C. Nichols, Christof von Kalle, Roncarolo, Marie-Gracia, Marinee K.L. Chuah, Manfred Schmidt, Thierry VandenDriessche, Luigi Naldini 926 Abstract Withdrawn 927 Targeting CD70 Positive Malignancies with T cells Expressing a CD27-Zeta Chimeric Antigen Receptor Reveals Important Costimulatory Role of CD27 D. Shaffer, B Savoldo, G. Dotti, D Spencer, S. Kenney, C.M. Rooney, S. Gottschalk

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Abstract Directory Thursday, May 20, 2010  Poster Session I — continued 6:15 PM - 8:15 PM Room: Exhibit Hall C & B-South

Late Breaking Science I 928 Engineering Aptamers Against HIV-1 RT for Improved Antiviral Bioactivity Margaret J. Lange, Angela S. Whately, Donald H. Burke

930 Genotoxic Potential of scAAV Vectors in Mouse Liver Lucia E. Rosas, Kimberly Zaraspe, Douglas M. McCarty 931 In vivo Persistence of ex vivo Expanded CCR7+CD45RA+ T Cells Using a RetroNectin Stimulation Method Tatsuji Enoki, Nobuko Muraki, Akiko Kato, Mitsuko Ideno, Junichi Mineno 932 Self-inactivating ASLV Based Vectors for the Gene Therapy of X-CGD Kerstin B. Kaufmann, Julia Suerth, Christian Brendel, Axel Schambach, Christopher Baum, Manuel Grez

Thursday, May 20th

929 Directed Integration of New Insulated Lentiviral Vectors to the Heterochromatin towards Safer Gene Transfer to Stem Cells Alexandre Artus, Caroline Duros, Yaïr Botbol, Simone Schultz, Manfred Schmidt, Christof von Kalle, Marc Lavigne, Odile Y Cohen-Haguenauer

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Abstract Directory Friday, May 21, 2010 Plenary Abstract Session 300 Plenary Abstract Session 8:00 AM - 10:00 AM Room: Marriott Ballroom Salons 1, 2 & 3 283 8:05 AM Therapeutic Transgene Expression in Patient-Specific iPS Cells after Selection of “Safe Harbors” for Lentiviral Vector Integration Eirini P. Papapetrou, Gabsang Lee, Nirav Malani, Isabelle Riviere, Frederic D. Bushman, Lorenz Studer, Michel Sadelain. 284 8:25 AM A Novel Lentiviral Expression System Reveals Patchwork Colony Formation during Reprogramming to Induced Pluripotent Stem Cells Eva Warlich, Johannes Kuehle, Tobias Cantz, Martijn Brugman, Tobias Maetzig, Melanie Galla, Christine Völkel, Adam Filipczyk, Stephan Halle, Hannes Klump, Hans Schöler, Timm Schroeder, Christopher Baum, Axel Schambach.

Friday, May 21st

285 8:45 AM Targeted Genomic Modification and Inactivation of the CCR5 Gene Using PNA-Induced Homologous Recombination Erica B. Schleifman, Ranjit S. Bindra, Jean Leif, Nicole Ali McNeer, Jacob Del Campo, Faye Rogers, Pradeep Uchil, Leonard D. Shultz, W. Mark Saltzman, Priti Kumar, Dale Griener, Peter M. Glazer. 286 9:05 AM Determining the Specificity of Zinc Finger Nucleases by Analyzing Insertion Site Distribution of Integrase Defective Lentiviral Vectors (IDLV) Trapped into DNA Double Strand Breaks Richard Gabriel, Angelo Lombardo, Anne Arens, Pietro Genovese, Michael C. Holmes, Philip D. Gregory, Manfred Schmidt, Luigi Naldini, Christof von Kalle. 287 9:25 AM Systemic Administration of Dendrimer- and Aptamer-siRNA Conjugates Efficiently Suppresses HIV-1 Infection Jiehua Zhou, Preston C. Neff, Xiaoxuan Liu, Jane Zhang, Haitang Li, Piotr Swiderski, Ling Peng, Ramesh Akkina, John J. Rossi. 288 9:45 AM CASPALLO: Phase I Clinical Trial of Allodepleted T Cells Transduced with Inducible Caspase 9 Suicide Gene after Haploidentical Stem Cell Transplantation Antonio Di Stasi, Siok K. Tey, Yuriko Fujita, Russell Cruz, Caridad Martinez, Alana Kennedy-Nasser, George Carrum, Carlos A. Ramos, Stephen M. Gottschalk, Karin Straathof, Lin Yu-Feng, Enli Liu, April Durett, Bambi Grilley, Hao Liu, Barbara Savoldo, Eric Yvon, Teresita Lopez, Oumar Diouf, Deborah Lyon, Adrian Gee, Ellen Vitetta, John Schindler, Catherine M. Bollard, Robert A. Krance, David M. Spencer, Gianpietro Dotti, Cliona M. Rooney, Helen E. Heslop, Malcolm K. Brenner.

EXHIBITOR PROSPECTUS final program

Oral Abstract Session 320 Clinical Gene & Cell Therapy Oral Abstract Session 2:00 PM - 4:00 PM Room: Marriott Ballroom Salons 2 & 3 289 2:00 PM Therapeutic DNA Vaccination Followed by Standard-of-Care Therapy in Patients with Chronic Hepatitis C: A Rapid Clearance of Viremia Matti Sallberg, Lars Frelin, Helmut M. Diepolder, Maria C. Jung, Iacob Mathiesen, Michael Fons, Gustaf Ahlen, Margaret Chen, Ola Weiland. 290 2:15 PM Sustained Alpha-Sarcoglycan Gene Expression in LGMD2D Following Gene Transfer Jerry R. Mendell, Louise R. Rodino-Klapac, Xiomara Rosales-Quintero, Brian D. Coley, Gloria Galloway, Sarah Lewis, Vinod Malik, Christopher J. Shilling, Barry J. Byrne, Thomas Conlon, Katherine J. Campbell, William G. Bremer, Christopher M. Walker, Zarife Sahenk, K. Reed Clark. 291 2:30 PM Advanced Generation RGD-Modified Oncolytic Adenovirus ICOVIR-7 in the Treatment of Cancer Refractory to Other Modalities Petri Nokisalmi, Sari Pesonen, Sophie Escutenaire, Mari Raki, Vincenzo Cerullo, Ramon Alemany, Juanjo Rojas, Maria Rajecki, Lotta Kangasniemi, Anna Kanerva, Timo Joensuu, Laura Ahtiainen, Akseli Hemminki. 292 2:45 PM Final Results of Phase I Trial of GMCSF Gene-TGFb2 Antisense Gene Autologous Tumor Cell (TAG) Vaccine in Advanced Cancer John Nemunaitis, Padmasini Kumar, Neil Senzer, Joseph Kuhn, Minal Barve, Jairo Olivares, Gerald Edelman, Cynthia Bedell, Yang Yu, Mitchell Magee, Beena O. Pappen, Gladice Wallraven, Alex W. Tong, Phillip B. Maples. 293 3:00 PM Administration of Tumor-Specific Cytotoxic T Lymphocytes Engineered to Resist TGF-ß to Subjects with EBV-Associated Lymphomas Catherine M. Bollard, Gianpietro Dotti, Stephen Gottschalk, Enli Liu, Andrea M. Sheehan, George Carrum, Hao Liu, Chung-Che Chang, Adrian P. Gee, Malcolm K. Brenner, Helen E. Heslop, Cliona M. Rooney. 294 3:15 PM Efficient Engraftment of MGMTP140K Gene-Modified CD34+ Cells Following Nonmyeloablative BCNU Conditioning in Patients with Glioblastoma Jennifer E. Adair, Brian C. Beard, Alexander Spence, Jason Rockhill, Daniel L. Silbergeld, Robert Rostomily, Pamela Becker, Marc Chamberlain, Maciej Mrugala, Hans-Peter Kiem. 295 3:30 PM Thymic Renewal and Anti-Leukemic Effect in Adults after Haplodentical Transplantation and Suicide Gene Therapy Luca Vago, Giacomo Oliveira, Maddalena Noviello, Corrado Soldati, Domenico Ghio, Roberto Nicoletti, Immacolata Brigida, Alessandro Aiuti, Maria Teresa Lupo Stanghellini, Jacopo Peccatori, Attilio Bondanza, Katharina Fleischhauer, Claudio Bordignon, Fabio Ciceri, Chiara Bonini.

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Abstract Directory Friday, May 21, 2010 Oral Abstract Session 320 I  — continued Clinical Gene & Cell Therapy Oral Abstract Session 2:00 PM - 4:00 PM Room: Marriott Ballroom Salons 2 & 3 296 3:45 PM Update on a Clinical Gene Therapy Trial for Adenosine Deaminase Deficient Severe Combined Immune Deficiency (ADA-SCID) Kit L. Shaw, Yeong “Christopher” Choi, Linda Muul, Robert Sokolic, Denise A. Carbonaro, Alan Ikeda, Monika Smogorzewska, Jayashree Jagadeesh, Elizabeth Garabedian, Ami Shah, Neena Kapoor, Michael S. Hershfield, Fabio Candotti, Donald B. Kohn.

303 3:30 PM Erasable Non-Integrating RNA Replicon Vectors for Generation of Immaculate iPS Cells Free from Transgenes and Sequence-Interference Hiroshi Ban, Noemi Fusaki, Toshiaki Tabata, Koichi Saeki, Akihiro Iida, Makoto Inoue, Mamoru Hasegawa. 304 3:45 PM Self-Inactivating Alpharetroviral Vectors with a Split-Packaging Design Julia D. Sürth, Tobias Mätzig, Melanie Galla, Ute Modlich, Christopher Baum, Axel Schambach.

Oral Abstract Session 322 AAV Vectors: Vector Biology

Oral Abstract Session 321 RNA Virus Vectors

2:00 PM - 4:00 PM Room: Marriott Ballroom Salon 1

2:00 PM - 4:00 PM Room: Virginia Suite

305 2:00 PM Dissecting rAAV Integration Frequency in Non-Human Primates by Deep Sequencing Ali Nowrouzi, Magalie Penaud-Budloo, Christine Kaeppel, Anne Arens, Caroline Le Guiner, Adrien Leger, Karim Zaoui, Christian Weber, Richard O. Snyder, Christof von Kalle, Philippe Moullier, Manfred Schmidt.

297 2:00 PM Sensitive In Vivo Models To Assess the Risk of Insertional Mutagenesis in the Liver upon Vector Systemic Delivery Marco Ranzani, Daniela Cesana, Manfred Schmidt, Francesca Sanvito, Fabrizio Benedicenti, Cynthia Bartholomä, Maurilio Ponzoni, Alessio Cantore, Lucia Sergi Sergi, Claudio Doglioni, Christof VonKalle, Luigi Naldini, Eugenio Montini.

299 2:30 PM Specific Insertions of Zinc Finger Domains into Gag-Pol Yields Engineered Retroviral Vectors with Selective Integration Properties Kwang-il Lim, Ryan Klimczak, Julie Yu, David Schaffer. 300 2:45 PM The D4Z4 Insulator Protects Retrovirus Vectors from Silencing in Stem Cells Sylvie Rival-Gervier, Susan Anderson, Mandy Lo, Peter Pasceri, James Ellis. 301 3:00 PM Expression of the LEDGF Integrase Binding Domain (IBD) Severely Restricts HIV-1 Replication and Drives Evolution of Phenotypically Distinctive Integrase Mutants Dyana T. Saenz, Anne Meehan, Mary E. Peretz, Eric M. Poeschla. 302 3:15 PM Transient and Highly Efficient Delivery of Sleeping Beauty Transposase and Flp Recombinase mRNA in Target Cells Via RTDeficient Retroviral Particles Melanie Galla, Axel Schambach, Tobias Maetzig, Niels Heinz, Zsuzsanna Izsvak, Zoltan Ivics, Christopher Baum.

307 2:30 PM Interaction with Homologous Recombination Pathways Leads To Loss of Functional Recombinant AAV (rAAV) Genomes Marcela P. Cataldi, Douglas M. McCarty. 308 2:45 PM High-Throughput Sequencing of AAV Vector Integrants To Assess Potential for Genotoxicity Hojun Li, Nirav Malani, Shari R. Hamilton, Alexander Schlachterman, Giulio Bussadori, Shyrie E. Edmonson, Rachel Shah, Federico M. Mingozzi, J. F. Wright, Valder R. Arruda, Frederic D. Bushman, Katherine A. High. 309 3:00 PM A Self-Complimentary rAAV Vector Genome but Not Variations in the Capsid Sequence Magnifies the Innate Immune Response Following Liver Directed Delivery Ashley T. Martino, Masataka Suzuki, Brendan Lee, Roland W. Herzog. 310 3:15 PM A Proteomic Analysis of the Protein Composition in Recombinant AAV Vector Preparations Biao Dong, Xunbao Duan, Fayaz Khazi, Kirk Chu, Jihong Dai, Jenni A. Snyder, Hui Lu, Weidong Xiao. 311 3:30 PM Downregulation of DNA-Damage Response Proteins in Postmitotic Tissues Correlates with Increased AAV Transduction Jasmina Lovric, Miguel Mano, Lorena Zentilin, Mauro Giacca.

Friday, May 21st

298 2:15 PM Systemic Injection in a Tumor Prone Mouse Unravels the Residual Genotoxicity of Self-Inactivating Lentiviral Vectors Daniela Cesana, Marco Ranzani, Cynthia Bartholomä, Manfred Schmidt, Fabrizio Benedicenti, Monica Volpin, Christof VonKalle, Luigi Naldini, Eugenio Montini.

306 2:15 PM Non-Canonical Integrating AAV Vectors as a Safe Tool for the Future of Gene Therapy Leszek Lisowski, Zhongya Wang, Ashley Lau, Feijie Zhang, Markus Grompe, Mark A. Kay.

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Abstract Directory Friday, May 21, 2010 Oral Abstract Session 322  — continued AAV Vectors: Vector Biology 2:00 PM - 4:00 PM Room: Marriott Ballroom Salon 1 312 3:45 PM Innate Immune Response towards Adeno-Associated Viral (AAV) Vectors in Human Liver Non-Parenchymal Cells Marianna Hoesel, Mathias Broxtermann, Hanna Janicki, Knud Esser, Silke Arzberger, Miriam John von Freyend, Dirk Stabenow, Percy Knolle, Michael Hallek, Ulrike Protzer, Hildegard Buening.

Oral Abstract Session 323 Gene Therapy for Lysosomal Storage Diseases and Other Genetic Disorders 2:00 PM - 4:00 PM Room: Delaware Suite

Friday, May 21st

313 2:00 PM Towards a Safe and Effective Gene Therapy Approach for Globoid Cell Leukodystrophy Ilaria Visigalli, Silvia Ungari, Bernhard Gentner, Sabata Martino, Daniela Ungaro, Letterio S. Politi, Lucia Sergi Sergi, Ubaldo Del Carro, Angelo Quattrini, Luigi Naldini, Alessandra Biffi. 314 2:15 PM Mesenchymal Stem Cell Therapy for the Twitcher Mouse Model of Krabbe’s Disease Cynthia Ripoll, Mette Flaat, Jessica Eiermann, Jeanne Perkins, Brittni Scruggs, Marjorie McCants, Michelle Eagle, William Wimley, Cynthia Trygg, Jeffrey Gimble, Bruce Bunnell. 315 2:30 PM Therapeutic Efficacy of Hematopoietic Stem Cell Gene Therapy for Hurler Type 1 Mucopolysaccharidosis Stefania Delai, Ilaria Visigalli, Letterio S. Politi, Carmela Di Domenico, Emanuela Mrak, Riccardo Brambilla, Federica Cerri, Daniela Ungaro, Merel Stok, Francesca Sanvito, Ubaldo Del Carro, Alessandro Rubinacci, Katherine P. Ponder, Angelo Quattrini, Paola Di Natale, Luigi Naldini, Alessandra Biffi. 316 2:45 PM Therapeutic Potential of Genetically Modified Neural Stem Cells (NSCs) in a Mouse Model of Globoid Cells Leukodystrophy (GLD) Margherita Neri, Alessandra Ricca, Silvia Ungari, Ilaria Visigalli, Alessandra Biffi, Ilaria Di Girolamo, Sabata Martino, Lucia Sergi Sergi, Luigi Naldini, Angela Gritti. 317 3:00 PM Adeno-Associated Virus and Tetanus Toxin Fragment C Based Vectors for Gene Therapy in the CNS Carlos Gay-Antaki, John H. Wolfe.

EXHIBITOR PROSPECTUS final program

318 3:15 PM Lineage Specific Lentiviral Mpl Expression Rescues Hematopoietic Stem Cell Defects in Mpl-/- Mice Dirk Heckl, Daniel C. Wicke, Johann Meyer, Axel Schambach, Johanna Krause, Matthias Ballmaier, Christopher H. Baum, Ute Modlich. 319 3:30 PM Safety of Gene Therapy for Thalassemia Riccardo Mezzadra, Emanuela Roselli, Claudia Rossi, Francesca Tiboni, Giacomo Mandelli, Sarah Marktel, Fulvio Mavilio, Giulietta Maruggi, Marco Andreani, Guido Lucarelli, Giuliana Ferrari. 320 3:45 PM Long-Term Phenotypic Correction of Skin by Fetal Gene Transfer in a Lethal Murine Model of Epidermolysis Bullosa Masayuki Endo, Philip W. Zoltick, Antenata Radu, Jiang Qiujie, Chihiro Matsui, Peter M. Marinkovich, Jouni Uitto, Alan W. Flake.

Oral Abstract Session 324 Physical Methods of Delivery 2:00 PM - 4:00 PM Room: Wilson ABC 321 2:00 PM Bile Duct Infusion of Plasmid DNA Mediated High Level of Transgene Expression in Rat Liver Xuan Jiang, Derek Leong, Deng Pan, Hai-Quan Mao. 322 2:15 PM Image-Guided, Computer-Controlled Hydrodynamic Gene Delivery to Liver Results in Therapeutic Level of Human Alpha-1 Antitrypsin in Pigs and Dogs Kenya Kamimura, Takeshi Suda, Guisheng Zhang, Dexi Liu. 323 2:30 PM Targeting Magnetic Nanoparticles Impregnated with Adenovirus to Stented Arteries Using Uniform-Field Induced Magnetization in the Rat Carotid Model of Restenosis Michael Chorny, Ilia Fishbein, Richard Adamo, Robert J. Levy. 324 2:45 PM Ultrasound-Activated Microbubbles for Gene Transfer: In Vivo Efficiency and Confocal Microscopy Real Time Intracellular Investigations Anthony Delalande, Patrick Midoux, Michiel Postema, Chantal Pichon. 325 3:00 PM Long-Term Gene Expression after Hydrodynamic Delivery of Sleeping Beauty Transposons to Canine Liver Using Balloon Catheters Perry B. Hackett, Myra Urness, Jason B. Bell, Erik R. Olson, Elena L. Aronovich, R. Scott McIvor. 326 3:15 PM Efficient Microbubble- and Ultrasound-Mediated Plasmid DNA Transfer into a Specific Rat Liver Lobe Via a Targeted Injection and Acoustic Exposure Using a Novel Ultrasound System Shuxian Song, Misty L. Noble, Liping Chen, Samuel S. Sun, Andrew A. Brayman, Carol H. Miao.

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Abstract Directory Friday, May 21, 2010 Oral Abstract Session 324  — continued Physical Methods of Delivery 2:00 PM - 4:00 PM Room: Wilson ABC

334 3:15 PM Tumor-Selectivity of Replication-Competent Retrovirus (RCR) Vectors Regulated by Prostate-Specific Promoters and Cytotoxic Effect of Suicide Gene Therapy Mediated by RCR Vectors In Vitro Shuichi Kamijima, Takahiro Kimura, Kazunori Haga, Kei Hiraoka, Akihito Inagaki, Christopher R. Logg, Chinghai Kao, Bernard H. Bochner, Noriyuki Kasahara.

327 3:30 PM A Novel Prototype Device for Electroporation-Enhanced DNA Vaccine Delivery to Both Skin and Muscle Feng Lin, Jay McCoy, Xuefei Shen, Janess Mendoza, Kate E. Broderick, Jian Yan, Steve Kemmerrer, David B. Weiner, Niranjan Y. Sardesai.

335 3:30 PM Translational Phase I Study in Advanced Cancer Patients of DualFunction Autologous (FANG) Vaccine Incorporating Bifunctional shRNAfurin and GMCSF Transgene Expression Neil Senzer, Padmasini Kumar, Joseph Kuhn, Minal Barve, Cynthia Bedell, Yang Yu, Alex W. Tong, Phillip B. Maples, John Nemunaitis.

328 3:45 PM Safety and Feasibility of Transvenous Limb Perfusion with Saline in Human Muscular Dystrophy William Powers, Zheng Fan, Keith Kocis, Robert Valley, Manisha Chopra, James Howard, Jr, Joseph Muenzer.

336 3:45 PM Efficient Gene Transfer in Quiescent Healthy and Cancer B Cells by a Measles Virus gp Pseudotyped LV Cecilia Frecha, Caroline Costa, Camille Levy, Didier Negre, Andrea Maisner, Stephen J. Russell, Nicholas Rachinel, Gilles Salles, Francois-Loic Cosset, Els Verhoeyen.

Oral Abstract Session 325 Cancer-Targeted Gene & Cell Therapy

Oral Abstract Session 326 Hematologic/Immunologic: Basic

2:00 PM - 4:00 PM Room: Thurgood Marshall W

330 2:15 PM Comparative Analysis of Enzyme and Pathway Engineering Strategies for Suicide Gene Therapy Applications Adam J. Johnson, Andressa Ardiani, Kinta Serve, Marilyn Sanchez-Bonilla, Margaret E. Black. 331 2:30 PM Conditional and Effective Gene Knock-Down in Proangiogenic Macrophages To Disrupt Tumor Angiogenesis Ferdinando Pucci, Roberta Mazzieri, Bernhard Gentner, Luigi Naldini, Michele De Palma. 332 2:45 PM Cell Fusion between Mesenchymal Stem Cells and Cancer Cells: A Mechanism of Cancer Progression Yan Xiao, Dirk H. Hondmann, Carla J. Aldrich, Chenzhong Kuang. 333 3:00 PM Next-Generation Lentiviral Cell Fate Control Gene Therapy Based on Engineered Variants of Human Deoxycytidine Kinase Anton Neschadim, Arnon Lavie, Takeya Sato, Daniel H. Fowler, Vladimir Ponomarev, Jeffrey A. Medin.

337 2:00 PM Safely Exploiting Hematopoeitic Stem and Progenitor Cell Specific microRNAs To Target Transgene Expression to the Differentiated Progeny Bernhard Gentner, Alice Giustacchini, Francesco Boccalatte, Massimo Saini, Giulia Schira, Luigi Naldini. 338 2:15 PM Therapeutic Fetal Hemoglobin Production in Erythroid Progeny of Normal and b-Thalassemic Human CD34+ Cells Using Lentiviral Vectors Encoding g-Globin or shRNA To Down Modulate the Transcriptional Repressor BCL11A Andrew Wilber, Vijay G. Sankaran, Phillip W. Hargrove, Yoon-Sang Kim, Eleni Papanikolaou, Nicholas P. Anagnou, Stuart H. Orkin, Arthur W. Nienhuis, Derek A. Persons. 339 2:30 PM Potentially Therapeutic Levels of Fetal Hemoglobin in Erythroid Progeny of Normal and b-Thalassemic CD34+ Cells Following Lentivirus-Mediated Delivery of a Zinc-Finger Transcriptional Activator Targeting the g-Globin Gene Promoters Andrew Wilber, Ulrich Tschulena, Phillip W. Hargrove, Yoon-Sang Kim, Carlos F. Barbas, III, Derek A. Persons, Arthur W. Nienhuis. 340 2:45 PM Development of an S/MAR Based Episomal Vector of a Specific ZincFinger Activator That Mediates Gamma-Globin Gene Activation, for the Gene Therapy of Hemoglobinopathies Eleana Stavrou, Eleni Lagadinou, Nikolas Zoumbos, Alexandros Spyridonidis, Carlos Barbas, III, Kenneth Peterson, Aglaia Athanassiadou.

Friday, May 21st

329 2:00 PM A Genetically Engineered Multifunctional Vector for HER2 Targeted Cancer Suicide Gene Therapy Yuhua Wang, Arash Hatefi.

2:00 PM - 4:00 PM Room: Maryland Suite

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Abstract Directory Friday, May 21, 2010 Oral Abstract Session 326— continued Hematologic/Immunologic: Basic 2:00 PM - 4:00 PM Room: Maryland Suite 341 3:00 PM Engineered Human Regulatory T Cells Expressing Lentiviral PDL1 under Cell Fate Control Prevent Lethal Xenogeneic GVHD Shoba Amarnath, James Wang, Courtney Mangus, James Riley, Bruce Levine, Carl June, Jeffrey Medin, Daniel Fowler. 342 3:15 PM Correction of Murine Hemophilia A Following Nonmyeloablative Transplantation of Hematopoietic Stem Cells Engineered To Express a Bioimproved Human FVIII Using a Safety-Augmented, B-Lymphoid Lineage-Restricted Lentiviral Vector Ali Ramezani, Sara Karandish, Lynnsey A. Zweier-Renn, Teresa S. Hawley, Robert G. Hawley.

Friday, May 21st

343 3:30 PM Lentiviral Vectors Containing an Endogenous Btk Promoter Restore B and Myeloid Lineage Expression and Function in a Murine Model of X-Linked Agammaglobulinemia Hannah M. Kerns, Byoung Y. Ryu, Brigid V. Stirling, Blythe D. Sather, Alexander Astrakhan, Stephanie Humblet-Baron, Denny Liggitt, David J. Rawlings. 344 3:45 PM Early Gestational Gene Transfer of IL-10 by Lentiviral Vector Can Prevent Arthritis in a Murine Model Jessica L. Roybal, Masayuki Endo, Antoneta Radu, Carlyn Todorow, Philip W. Zoltick, Alan W. Flake.

Poster Session II 6:30 PM - 8:30 PM Room: Exhibit Hall C & B-South

RNA Virus Vectors I 345 Expression Profile and Epigenetic Landscape Influence in a CellSpecific Fashion Integration Site Selection and In Vivo Distribution of MLV-Vector in Patients Luca Biasco, Alessandro Ambrosi, Danilo Pellin, Cynthia Bartholomae, Immacolata Brigida, Maria Grazia Roncarolo, Clelia Di Serio, Christof Von Kalle, Manfred Schmidt, Alessandro Aiuti. 346 High-Definition Mapping of Retroviral Integration Sites Defines the Fate of Allogeneic T Cells after Donor Lymphocyte Infusion Alessandra Recchia, Claudia Cattoglio, Danilo Pellin, Giulietta Maruggi, Serena Kimi Perna, Alessandro Ambrosi, Clelia Di Serio, Nirav Malani, Frederic D. Bushman, Cynthia Bartholomae, Manfred Schmidt, Chiara Bonini, Fulvio Mavilio.

EXHIBITOR PROSPECTUS final program

347 Genome Wide Characterization of Insertion Profiles of Integration Deficient Lentiviral Vectors Using Two-Sided Non-Restrictive and Standard LAM-PCR Wei Wang, Cynthia C. Bartholomae, Anne Arens, Richard Gabriel, Hanno Glimm, Christof von Kalle, Manfred Schmidt. 348 Multiple Pathways Facilitate Transduction of Quiescent Human CD34+ Cells by HIV-1 Vectors Cathy X. Wang, Charles DeRossi, Bruce E. Torbett. 349 Targeted Lentiviral Gene Delivery to Cells Expressing c-fms or IL-2 Receptor Anil Chandrashekran, Giedre Milinkeviciute, Rosana Cardoso, Fouzia Abdulrasul, Shao-An Xue, Nick Dibb, Carol Readhead, Colin Casimir. 350 Regulation of Transgene Expression In Vivo by Proteasomal Degradation Khalid Tai, Fredrik Gussing, Christina Isaksson, Luis Quintino, Mari Iwamoto, Tom J. Wandless, Cecilia Lundberg. 351 Clonal Analysis Allows Characterization and Risk Assessment of Viral-Cellular Fusion Transcripts Generated by Transcriptional Termination Read-Through in Keratinocytes Transduced with SelfInactivating Retroviral Vectors David Almarza, Fernando Larcher, Rodolfo Murillas, Fulvio Mavilio. 352 Evaluation of Cytokine Effects on Lentiviral Transduction of Human CD34+ Cells Naoya Uchida, Kareem N. Washington, Matthew M. Hsieh, John F. Tisdale. 353 Towards Quantitative High Throughput Clonality Screening: Redefining the Frontier of Retroviral Integration Analysis Troy B. Hawkins, Aparna Jasti, Jessica Dantzer, Nate Van Dusen, Brandon Downing, Sean Mooney, Mary C. Dinauer, Keithanne Mockaitis, Kenneth G. Cornetta. 354 Development of a pCL20c-Backbone Based HIV Lentivirus Vector That Achieves Efficient Functional Correction of Neutrophils Arising from Transduced CD34+ Hematopoietic Progenitor Cells of Patients with X-Linked Chronic Granulomatous Disease Uimook Choi, Robert E. Throm, Elizabeth M. Kang, Suk See DeRavin, Narda Theobald, Gilda M. Linton, Brian P. Sorrentino, Mary C. Dinauer, John T. Gray, Harry L. Malech. 355 Packaging Heterologous Retroviral Vectors into HIV-1 Viral Particles Adam S. Cockrell, Henriette van Praag, Nicholas Santistevan, Thomas J. McCown, Bonita Blake, Hong Ma, John Olsen, Tal Kafri. 356 High Throughput Vector Integration Analysis with SeqMap 2.0 Troy B. Hawkins, Jessica Dantzer, Brandon Peters, Mary Dinauer, Keithanne Mockaitis, Sean Mooney, Kenneth G. Cornetta.

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Abstract Directory Friday, May 21, 2010 Poster Session II— continued 6:30 PM - 8:30 PM Room: Exhibit Hall C & B-South

RNA Virus Vectors I 357 ‘Self’ Marker CD47 on Viral Envelopes Nisha Sosale, Richard K. Tsai, Irena Ivanovska, Philip W. Zoltick, Dennis E. Discher. 358 Lentiviral Microarrays for High-Throughput and Real-Time Monitoring of Gene Expression Dynamics Jun Tian, Stella Allimperti, Pedro Lei, Stelios T. Andreadis. 359 Fibrin-Conjugated Pseudotyped Lentivirus for Cell-Controlled and Spatially Localized Gene Delivery on Microarrayed Surfaces Roshan Padmashali, Pedro Lei, Stelios Andreadis.

366 The Hybrid AAV2.5 Vector Transduces Neurons Bordering the Third and Fourth Ventricles after Lateral Ventricular Infusion in Rats Dawn E. Bowles, Chengwen Li, Joseph E. Rabinowitz, R. Jude Samulski, Thomas J. McCown. 367 Differential Lymphatic Transport of Chimeric and Naturally Occurring AAV Strains Jana L. Phillips, Roland Cheung, Chengwen Li, Aravind Asokan. 368 Optimizing Liver Transduction of the Low Seroprevalent AAV rh32.33 Luk H. Vandenberghe, Ru Xiao, James M. Wilson. 369 Role of a Cellular Chaperone Protein, FKBP52, in Uncoating of AAV2 Vectors Mengxin Li, Giridhara R. Jayandharan, Baozheng Li, Chen Ling, Wenqin Ma, Arun Srivastava, Li Zhong.

361 Purification of Lentivirus Vector by Iodixanol Self-Forming Density Gradient Julia Transfiguracion, Sven Ansorge, Rosa Tran, Amine Kamen.

371 Optimizing Capsid Composition for the Production of Recombinant Adeno-Associated Virus Using the Baculovirus Expression System Kirsten Erger, David Markusic, Ramaz Geguchadze, Barry J. Byrne, Sergei Zolotukhin.

AAV Vectors II 362 rAAV-Mediated Delivery of Micro RNA Scavengers Leads to Efficient and Stable Knock-Down of Cognate Micro RNAs, Upregulation of Their Natural Target Genes and Phenotypic Changes in Mice Jun Xie, Jennifer Broderick, Qing Xie, Hongwei Zhang, Ran He, Qin Su, Phillip D. Zamore, Guangping Gao. 363 Activation of the Non-Canonical NF-kB Pathway by AAV2 Vectors: Implications in Immune Response and Gene Therapy Giridhara R. Jayandharan, George Aslanidi, George Q. Perrin, Li Zhong, Angela E. Rivers, Ashley T. Martino, Roland W. Herzog, Arun Srivastava. 364 Multifunctional Peptide Ligands for AAV Capsid Recognition Nagesh Pulicherla, Jana L. Phillips, Aravind Asokan. 365 The Rous Sarcoma Virus Promoter Carried by a rAAV Vector Is Resistant to DNA Methylation in Primate Muscle and Liver Adrien Léger, Magalie Penaud-Budloo, Michael L. Nickerson, Caroline Le Guiner, Philippe Moullier, Richard O. Snyder.

372 Genetically Re-Engineered AAV Rep78 Identifies Sequence-Restricted Inhibitory Regions Affecting Adenoviral Replication Varsha Sitaraman, Patrick Hearing, Steffen Mueller, Charles Ward, Dmitri Gnatenko, Steven Skiena, Eckard Wimmer, Wadie F. Bahou. 373 Improved and Persistent Expression with Novel AAV Serotypes for Neonatal Gene Therapy Chuhong Hu, Fides D. Lay, Joseph Springer, Ronald W. Busuttil, Gerald S. Lipshutz. 374 Optimization of AAV Vectors for Improved Therapeutic Protein Expression in the Canine Models for Duchenne Muscular Dystrophy Alock Malik, Marilyn Mitchell, Andrew Mead, Mike Petrov, Hansell Stedman. 375 rAAV1 Infect Mouse Bone Marrow-Derived Dendritic Cells through Receptors with a2,3 and a2,6 Sialic Acids Yuanqing Lu, Sihong Song. 376 Using AAV Vector as Carrier for Biosensor: Detecting microRNA’s Activities by Reverse Infection of AAV Vectors Wenhong Tian, Xiaoyan Dong, Gang Wang, Shuping Tan, Xiaobing Wu.

Friday, May 21st

360 Improved Transduction Efficiency of Human Amniotic Mesenchymal Stem Cells Using Optimized Lentiviral Vectors F. Pisano, P. Danieli, E. Cervio, C. Ciuffreda, M. Roccio, G. Viarengo, M. Gnecchi.

370 Strategies To Achieve Enhanced Transduction of Primary Human Dendritic Cells by AAV2 Vectors and Reduced Cytotoxic T Cell Response to Viral Capsids and Transgene Products George Aslanidi, George Q. Perrin, Giridhara R. Jayandharan, Angela E. Rivers, Li Zhong, Arun Srivastava.

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Abstract Directory Friday, May 21, 2010 Poster Session II— continued 6:30 PM - 8:30 PM Room: Exhibit Hall C & B-South 377 Clearance of Herpes Simplex Virus by Orthogonal Manufacturing Process of Recombinant Adeno-Associated Virus (rAAV) Vectors Guo-jie Ye, David R. Knop, Darby L. Thomas, Lijun Wang, Jeffrey D. Chulay.

Adenovirus and Other DNA Virus Vectors II 378 Production of a Novel Class of Human Polyomavirus JC Vectors Using Baculovirus Expression System Kirsten E. Erger, Oleg Gorbatyuk, George Aslanidi, Ramaz Geguchadze, Barry J. Byrne, Sergei Zolotukhin. 379 Induction of Cell Surface Integrins in Lymphomas and Potential Applications to Gene Therapy Ann Marie O’Neill, Farruk M. L. Kabir, David T. Curiel, Laura Timares, Bruce F. Smith.

Friday, May 21st

380 FX-Binding Ablated Ad5 Do Not Transduce Hepatocytes but Exhibit Splenic Uptake at High Doses Raul Alba, Angela C. Bradshaw, Robert A. McDonald, Lynda Coughlan, Alan L. Parker, Ashley M. Miller, Nico van Rooijen, Stuart A. Nicklin, Andrew H. Baker. 381 Targeting Adenovirus Cell Entry Via Tumor-Associated EphA2 by Insertion of a Ligand Peptide into Different Loops of the Short Fiber of Ad41 Michael Behr, Dirk M. Nettelbeck. 382 Induction of a Prostate Specific Promoter and Replication Kinetics by p21/Waf-1 shRNA Armed CRAd Naser Uddin Hoti, Sadaf Mustafa, Wasim Chowdhury, Ronald Rodriguez. 383 Capacitance-Based Real Time Monitoring of Virus Transduction Joung-Woo Choi. 384 Inhibition of PD-1 Greatly Augments Therapeutic Effect of Suicide Gene Therapy for Gastrointestinal Cancers Hae-hyun Seo, Mi-Hyang Lee, In-Hoo Kim, Seong-Phil Shin, Kyung-Hee Chun, Sang-Jin Lee. 385 Method for Fast and Easy Adoption of First-Generation Adenoviral Vectors to High-Capacity Adenoviral Vectors Martin A. Hausl, Christina Rauschhuber, Richard Voigtlander, Zsolt Ruzsics, Anja Ehrhardt.

EXHIBITOR PROSPECTUS final program

386 PAUF Targeting RNA-Based Gene Therapy Sue Kyung Kim, Eun Ok Kim, Hyun Mi Dae, Ju Young Moon, Kyun Heo, In-Hoo Kim, Yun-Hee Kim. 387 Combining Oncolytic Adenoviruses and Antibody Therapy Dominik E. Dorer, Roland Kontermann, Dirk M. Nettelbeck. 388 Direct Injection of HDAd into the Liver Is as Effective but Less Toxic Than Intravenous Injection for Crigler-Najjar Syndrome Gene Therapy Franesco Vetrini, Nathan Grove, Donna Palmer, Philip Ng, Nicola Brunetti-Pierri. 389 Dual-Expression Adenoviral Vectors as Vaccine Carriers Xiangyang Zhou, Heng Chen, Dongming Zhou, Ang Bian, Hildegund C. Ertl. 390 Optimized Adenovirus Targeting through Virus-Displayed Peptide Library Screening in a Prostate Cancer Model Ping Wu, Tarana A. Kudrolli, Wasim H. Chowdhury, Minzhi M. Liu, Ronald Rodriguez, Shawn E. Lupold. 391 Human Adenovirus Type 40 Vector Engineering for Intestinal Mucosa Targeting Satoshi Yamasaki, Eric Brown, Julia Davydova, Selwyn M. Vickers, Masato Yamamoto.

Chemical and Molecular Conjugates II 392 Macropinocytosis Is the Major Pathway Leading to Transfection for a Charge-Reversal Amphiphile Xiao-Xiang Zhang, Phil Allen, Mark W. Grinstaff. 393 Ornithine-Based Cationic Lipids: Highly Charged Dendritic Headgroups, New Lipoplex Phases and Degradable Lipids Kai K. Ewert, Alexandra Zidovska, Rahau S. Shirazi, Joel Quispe, Bridget Carragher, Clinton S. Potter, Cyrus R. Safinya. 394 Nanoparticle-Mediated Delivery of Genes and Imaging Agents to the Brain Stephen L. Hart, Alison Bienemann, Steven Gill, Panos Kyrtatos, Andrew S. Lowe, Mark Lythgoe, Edward A. White, Michele J. Writer. 395 In Vitro and In Vivo Transfection Efficiency of DNA Nanoparticles Made with Histidinylated lPEI and Plasmid DNA Containing NFkB DNA Binding Sequence Patrick Midoux, Gilles Breuzard, Ludivine Billiet, Emilie Bertrand, Tony Le Gall, Tristan Montier, Pierre Lehn, Hervé Chéradame, Chantal Pichon, Philippe Guégan.

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Abstract Directory Friday, May 21, 2010 Poster Session II— continued 6:30 PM - 8:30 PM Room: Exhibit Hall C & B-South

Chemical and Molecular Conjugates II 396 Intracellular Trafficking and Decondensation Kinetics of ChitosanpDNA Polyplexes Marc Thibault, Surendra Nimesh, Lavertu Marc, Buschmann Michael. 397 Dual Function Effect on PEI-Mediated Gene Delivery by Diphtheria Toxin-Derived Ligand and Fusogenic Domains Takeshi Nagasaki, Shinji Kakimoto, Hideki Azuma.

405 Novel L-Lysine Reducible Copolymers for Efficient Nonviral Gene Delivery Mohamed M. Ismail Nounou, Samuel Chung, Kalyopy Emmanouil, Thu Pham, Zhuoyuan Lu, Malavosklish Bikram. 406 JL1-Directed Small Interfering RNA Delivery Using Anti-JL1 ImmunoSendai Viroplexes to T-Leukemic Cells Jung Seok Kim, Yeon Jyung Lee, Jin Ee Baek, Sang Il Park, Hwa Yeon Jeong, Hong Sung Kim, Keun Sik Kim, Sang Soon Yoon, Kyeong Cheon Jung, Hyung Geun Song, Yong Serk Park. 407 Antibody-Directed Drug Delivery Using Protein A-Based Carriers Nahyun Kim, Hong-Seok Ban, Priti Kumar, Sang-Kyung Lee.

Genetic and Metabolic Diseases Gene & Cell Therapy II 408 Lentiviral Vector-Transduced Hematopoietic Stem Cell Gene Therapy for Cystinosis Brian A. Yeagy, Frank Harrison, Matthew V. Tadlock, Daniel R. Salomon, Stephanie Cherqui.

399 Efficient Gene Transfer to Umbilical Cord Blood-Derived Mesenchymal Stem Cells Mediated by MR Imageable Inorganic-Organic Hybrid Conjugate Yong Sook Kim, In-Kyu Park, Jin Sook Kwon, Myung Ho Jeong, Youngkeun Ahn.

409 Creation and Rescue of a Severe Hyperammonaemic Phenotype in Ornithine Transcarbamylase Deficient spfash Mice Using rAAV and shRNA Sharon C. Cunningham, Cindy Y. Kok, Allison P. Dane, Kevin H. Carpenter, Ian E. Alexander.

400 Histidine-Rich Amphipathic Peptides Promote Efficient Delivery of Nucleic Acids into Mammalian Cells Antoine Kichler, A. James Mason, Christian Leborgne, Daniel Scherman, Burkhard Bechinger. 401 MHC Class-I Mediated Antigen Presentation by a Non-Viral T-MEND Vector Expressing a High Luciferase Activity in Murine Derived Dendritic Cells (JAWS-II) Sharif M. Shaheen, Hidetaka Akita, Takashi Nakamura, Shiroh Futaki, Atsushi Yamashita, Ryo Katoono, Nobuhiko Yui, Hideyoshi Harashima. 402 Evaluation of Polymer-DNA Nanoparticles for Gene Transfer to Cardiac Muscles Xiaohong Qiu, Lepeng Zeng, Jon Urban, Alena Nikolskaya, Sarah Audet. 403 Chemical Manipulation of AAV Tissue Tropism Eric D. Horowitz, Jana L. Phillips, Aravind Asokan. 404 Versatile Self-Assembling Nanoparticles for Enhanced siRNA Delivery into Mammalian Cells Gulam H. Dar, Madhusudhana R. Nalam, Vijaya Gopal.

410 Long Term Correction of Biochemical and Neurological Abnormalities of Metachromatic Leukodystrophy Model Mice by Systemic Neonatal Injection of Serotype 9 AAV Vector Noriko Miyake, Koichi Miyake, Nagisa Asakawa, Motoko Yamamoto, Takashi Shimada. 411 Neural Stem Cells as a Therapeutic for Krabbe’s Disease Mette Flaat, Cynthia Ripoll, Jimin Yu, Jessica Eiermann, Jeanne Fisher-Perkins, Brittni Scruggs, Helen Paige Leonard, Amy Lin, Marjorie McCants, Michelle Eagle, William Wimley, Bruce A. Bunnell. 412 AAV8 Mediated Expression of LDLR Ameliorates Hypercholesterolemia and Regresses Pre-Existing Atherosclerosis in a Humanized Mouse Model of Familial Hypercholesterolemia Sadik H. Kassim, Luk H. Vandenberghe, Christian Hinderer, Dawn Marchadier, Peter L. Bell, Hui Li, Debra Cromley, Aisha Wilson, Hongwei Yu, James M. Wilson, Daniel J. Rader. 413 Widespread Enzymatic Correction of CNS Tissues by a Single Intracerebral Injection of Therapeutic Lentiviral Vector in Leukodystrophy Mouse Models Annalisa Lattanzi, Claudio Maderna, Margherita Neri, Ilaria Di Girolamo, Sabata Martino, Aldo Orlacchio, Mario Amendola, Luigi Naldini, Angela Gritti.

Friday, May 21st

398 Tumour Therapy with Listeria Monocytogenes Vector-Mediated DNA Delivery Jan P. Van Pijkeren, David Morrissey, Michelle Cronin, Simon Rajendran, Gerald C. O’Sullivan, Cormac G. M. Gahan, Mark Tangney.

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Genetic and Metabolic Diseases Gene & Cell Therapy II 414 Gene Therapy Ex Vivo in the ADA-Deficient Mouse: Increased Survival and Efficacy with Short-Term Enzyme Replacement Therapy Post Gene Therapy Denise A. Carbonaro, Xiangyang Jin, Donald B. Kohn. 415 Towards Targeted Transgene Integration in Human Hematopoietic and Induced Pluripotent Stem Cells Hongjie Wang, Ying Liu, Michael C. Holmes, Philip D. Gregory, Andre Lieber. 416 Optimization of the Expression Cassette Using an AAV2/8 Based Vector for the Treatment of Ornithine Transcarbamylase Deficiency Lili Wang, Huan Wang, Hiroki Morizono, Peter L. Bell, David Jones, Mark L. Batshaw, James M. Wilson.

Friday, May 21st

417 Generation of Early AMD Mouse Model by Induction of Oxidative Stress Soojung Gibson, Jian W. Liu, Yun Le, Wiliam W. Hauswirth, Alfred S. Lewin. 418 Increased Expression of Human Glucocerebrosidase Activity in Bone Marrow of Gaucher Mice Following Macrophage Mediated Gene Therapy Arlene T. Lim, Edward I Ginns. 419 Cell Sheet Engineering toward Bioengineering of Functional Neo-Islets Takahiro Saito, Kazuo Ohashi, Hirofumi Shimizu, Rie Utoh, Kazuya Ise, Masayuki Yamato, Teruo Okano, Mitsukazu Gotoh. 420 Liver-Specific Over-Expression of PGC-1a Does Not Induce a Diabetic Phenotype Luke Roode, Chunping Qiao, Ruhang Tang, Jiangjang Jiang, Juan Li, Xiao Xiao. 421 Promoter Selection for AAV Vector-Mediated Factor IX Expression in Skeletal Muscle Akihiro Kume, Hiroya Yagi, Hiroaki Mizukami, Masashi Urabe, Tomonori Tsukahara, Akira Ishiwata, Jun Mimuro, Seiji Madoiwa, Tsukasa Ohmori, Yoichi Sakata, Keiya Ozawa. 422 Development of HIV-1 Based Lentiviral Vectors Expressing PreTrans-Splicing Molecules (PTMs) for Liver-Directed Therapies Madaiah Puttaraju, S. Gary Mansfield, Jun Wang, Nikolay Korokhov, Jenice D’Costa, Ziping C. Chen, Katherine Radd, Gerard J. McGarrity, Laurent M. Humeau.

EXHIBITOR PROSPECTUS final program

764 (Moved from Poster Session III) Microporous Polymer Scaffolds for Islet Transplantation Romie F. Gibly, Lonnie D. Shea.

Cardiovascular Gene & Cell Therapy 423 AAV-Mediated Intramyocardial VEGF-B167 Expression Exerts a Potent Cardioprotective Effect in Small and Large Animal Models of Myocardial Infarction and Heart Failure Lorena Zentilin, Vincenzo Lionetti, Uday Puligadda, Silvia Moimas, Serena Zacchigna, Martino Pepe, Mohammed Mamdani, Khaled Qanud, Xiabin Xu, Thomas H. Hintze, Fabio A. Recchia, Mauro Giacca. 424 Osteopontin Enhances the Angiogenic Potential of Endothelial Progenitor Cells Erin E. Vaughan, Angela Duffy, Timothy O’Brien. 425 Arginine and Tetrahydrobiopterin Synergistically Potentiate the Antirestenotic Effect of Vascular Gene Therapy with Inducible Nitric Oxide Synthase Ilia Fishbein, Peter Sobolewki, Richard F. Adamo, Ivan S. Alferiev, Ariel Fishbein, Marina Bakay, Michael Chorny, Robert J. Levy. 426 Intra-Myocardial Administration of ACRX-100 Promotes Cardiac Benefit and Improves Angiogenesis in a Porcine Model of Heart Failure Joseph M. Pastore, Timothy J. Miller, Rahul A. Aras, Marc S. Penn. 427 Strategies for Promoting Survival of Transplanted Mesenchymal Stem Cells in a Myocardial Infarction Model Lisa McGinley, Daniel O’Toole, Aoife Duffy, Xizhe Chen, Alessia Stocca, Jill McMahon, Timothy O’Brien. 428 Systemic Delivery of Interleukin-10 by an AAV Vector Attenuates Obesity-Related Glomerulopathy in Zucker Fatty Rats Manabu Ogura, Masashi Urabe, Akira Onishi, Takayuki Ito, Tomonori Tsukahara, Hiroaki Mizukami, Akihiro Kume, Eiji Kusano, Keiya Ozawa. 429 AAV/B7H1-Gene Delivery Lowers Th1 Cytotoxic T Lymphocyte Response In Vitro and Atherogenesis In Vivo Maohua Cao, Junaid A. Khan, Yong Liu, Paul L. Hermonat. 430 Dose-Dependent Angiogenesis by Human and Murine VEGF Is Species-Specific Edin Mujagic, Roberto Gianni-Barrera, Marianna Trani, Lorenz Gürke, Michael Heberer, Thomas Wolff, Andrea Banfi. 431 AAV-Sema3A-Mediated Recruitment of Neuropilin-1-Expressing Mononuclear Cells (NEMs) Contributes to Vessel Stabilization and Normalization Alessandro Carrer, Serena Zacchigna, Silvia Moimas, Lucia Pattarini, Lorena Zentilin, Mauro Giacca.

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Cardiovascular Gene & Cell Therapy 432 Identification of AAV6 as Most Efficient Vector for Transvascular Gene Transfer into Porcine Myocardium Based on an In Vitro Model for Prediction of the Cardiac Gene Transfer Performance Jan Ksienzyk, Philip W. Raake, Philipp Schlegel, Changguang Shan, Christophe Weber, Jürgen A. Kleinschmidt, Hugo A. Katus, Oliver J. Müller. 433 Percutaneous Transendocardial Adminstration of AAV6 and AAV8 Vectors to Rhesus Macaques Results in Highly Efficient and Global Cardiac Gene Transfer Guangping Gao, Meg M. Sleeper, Lawrence Bish, Lan Sun, You Lu, Jiachuan Duan, Chunyan Hu, Li Wang, H. Lee Sweeney. 434 Gene Transfer of VEGF165, HGF, Urokinase and Their Combinations Using Novel PC4W Plasmid for Induction of Vasculogenesis in Mice Hind Limb Ischemia Model Pavel Makarevich, Zoya Tsokolaeva, Alexander Shevelev, Igor Rybalkin, Eugeniy Shevchenko, Irina Beloglazova, Tatyana Vlasik, Elena Parfyonova, Vsevolod A. Tkachuk.

436 AAV/IL13-Gene Delivery Lowers Atherogenesis in LDLR KO Mice Junaid A. Khan, Maohua Cao, Yong Liu, Paul L. Hermonat. 437 AAV/hSTAT3-Gene Delivery Lowers Atherosclerosis in LDLR KO Mice on High Cholesterol Diet Junaid A. Khan, Maohua Cao, Bum-Yong Kang, Jawahar L. Mehta, Paul L. Hermonat. 438 Intramuscular Delivery of ACRX-100 Enhances Angiogenesis in Rabbits with Hind Limb Ischemia Timothy J. Miller, Joseph M. Pastore, Rahul A. Aras, Marc S. Penn. 439 A Comparison of Three Liposomal Formulations for Gene Delivery from Coronary Artery Stents Sandra Ganly, Sean O. Hynes, Faisal Sharif, Karl McCullagh, Valerie Barron, Jim Crowley, Peter McHugh, Timothy O’Brien, Udo Greiser.

Ophthalmic Disorders and Technological Advances 441 Gene Delivery of Erythropoietin Protects Retinal Ganglion Cells in DBA/2J Glaucomatous Mice Timothy A. Sullivan, Eldon E. Geisert, Tonia S. Rex. 442 Pan-Retinal Secretion of GDNF Following Intravitreal Transduction of Müller Glia Slows Photoreceptor Loss in Retinal Degeneration Deniz Dalkara, Kathleen D. Kolstad, Meike Visel, Ryan R. Klimczak, Karen Guerin, David V. Schaffer, John G. Flannery. 443 AAV-Mediated Gene Supply of Non-Erythropoietic Erythropoietin Derivatives Improves Photoreceptor Morphology and Function in Models of Retinal Degenerative Diseases Pasqualina Colella, Carolina Iodice, Umberto Di Vicino, Alberto Auricchio. 444 Early Indicators of Biological Efficacy in a Phase 1/2 Gene Therapy Trial for Leber Congenital Amaurosis (LCA) Shalesh Kaushal, Margaret Humphries, Elena Filippova, George Asdourian, Radouil Tzekov, Michael Stalvey, Terrence Flotte. 445 Positive Responses in a Mouse Model of Leber Congenital Amaurosis after Treatment with Compacted DNA Nanoparticles Kiichiro Okano, Tadao Maeda, Linas Padegimas, Tomasz H. Kowalczyk, Sharon M. Oette, Christopher R. Gedeon, Ozge Sesenoglu-Laird, Susannah L. Hyatt, Karla A. Dines, Elena Tyr, Nikia L. Beal, Robert C. Moen, Krzysztof Palczewski, Mark J. Cooper. 446 Adeno-Associated Virus Mediated Gene Therapy with a Rhodopsin Ribozyme for Treatment of Autosomal Dominant Retinitis Pigmentosa (Adrp) Haoyu Mao, Marina S. Gorbatyuk, William W. Hauswirth, Alfred S. Lewin. 447 POD DNA Nanoparticles Rescue an Adult Mouse Model of Retinal Degeneration Sarah P. Read, Siobhan M. Cashman, Rajendra Kumar-Singh. 448 Protection Against Laser-Induced Choroidal Neovascularization Following Delivery of an Adenovirus Expressing a Soluble Form of Human CD59 to Murine Retina Siobhan M. Cashman, Kasmir Ramo, Rajendra Kumar-Singh. 449 Dose Finding Study in Galactosialidosis Mice Using scAAV2/8-LP1ppca Virus Huimin Hu, Elida Gomero, Eric Bonten, John Gray, Christopher Calabrese, Arthur Nienhuis, Alessandra d’Azzo.

Friday, May 21st

435 PEGylation of Helper-Dependent Adenoviral Vector-Mediated Long-Term Expression of Human Apolipoprotein A-I Reduces Atherosclerosis in LDLR Deficient Mice Eleonora Leggiero, Dalila Astone, Piyanuch Wonganan, Cristina Mazzaccara, Giuseppe Labruna, Luciana Sacchetti, Francesco Salvatore, Maria Croyle, Lucio Pastore.

440 Efficient and Localized Gene Transference in Rat Heart by Electroporation Leonardo P. Carvalho, Eduardo G. Yasumura, Vivian Somoto, Priscila M. A. Denapoli, Flavia H. Silva, Valderez B. Valero, Hans F. Dohmann, Christina M. Takiya, Sang W. Han.

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Ophthalmic Disorders and Technological Advances

Friday, May 21st

450 Characterization of 12 AAV Vectors for Intravascular Delivery To Target CNS and Detarget Non-CNS Tissues by miRNA Regulation: Implications in Treatment of Canavan Disease Hongwei Zhang, Bin Yang, Jun Xie, Qing Xie, Qin Su, Ran He, Miguel Sena-Esteves, Zuoshang Xu, Guangping Gao.

457 Increased Muscle Regeneration in Hindlimb and Diaphragm of mdx Mice Treated with AAV9 Mini-Dystrophin and Octalysine-NEMO Binding Domain Peptide Daniel P. Reay, Geno Raggi, Bing Wang, Xiao Xiao, Paul D. Robbins, Paula R. Clemens. 458 External, Non-Invasive Monitoring of Progressive Cardiorespiratory Dysfunction in a Canine Model of DMD Andrew Mead, Alock Malik, Mihail Petrov, Martin Childers, Janet Bogan, Joseph Kornegay, Hansell Stedman. 459 The Polyproline Site in Hinge 2 Influences the Functional Capacity of Truncated Dystrophins Glen B. Banks, Luke M. Judge, James M. Allen, Jeffrey S. Chamberlain.

451 Efficient Expression of Smn from Integration-Deficient Lentiviral Vectors Sherif O. Ahmed, Hugo Peluffo, Edmund Foster, Natalia Lago, Lawrence Moon, Thomas Hutson, Ping Yip, Klaus Wanisch, Stephen B. McMahon, Rafael J. Yáñez-Muñoz.

460 Combining Gene and Stem Cell Therapy in the Treatment of Dysferlinophaties Daniele Parolini, Claire Navarro, Andrea Farini, Mirella Meregalli, Marzia Belicchi, Paola Razini, M. Krahn, Louis Garcia, Nicolas Lévis, Yvan Torrente.

452 Efficacy of an Adenovirus Vector/Cocaine Conjugate-Based AntiCocaine Vaccine in Shielding the Brain from Systemically Administered Cocaine Martin J. Hicks, Bishnu P. De, Jonathan B. Rosenberg, Jesse T. Davidson, Amira Y. Moreno, Kim D. Janda, Neil R. Hackett, Stephen M. Kaminsky, Stefan Worgall, Ronald G. Crystal.

461 Deletion of Functional Domains of Dp116 Leads to Altered Sarcolemmal Expression and Subcellular Localization When Expressed in Skeletal Muscle Andrea L. Arnett, Guy L. Odom, Kristy Boyle, Eric Finn, Jeffrey S. Chamberlain.

453 Genetically Engineered Suppressor Cells Target CNS and Inhibit Activated Effector T-Cells Moa Fransson, Elena Piras, Hao Wang, Ida Rasmusson, Katarina LeBlanc, Eva Brittebo, Angelica S. I. Loskog.

Musculo-skeletal Gene & Cell Therapy II 454 First Generation AAVmicroutrophin Vector Infused into the Isolated Pelvic Limb of a Canine Model for Duchenne Muscular Dystrophy Mihail Petrov, Marilyn Mitchell, Alock Malik, Andy Mead, Frederick Balzer, Leonard Su, Jacqueline Farag, Benjamin Kozyak, Kapil Gopal, Charles Bridges, Janet Bogan, Martin Childers, Joe Kornegay, Hansell Stedman.

462 A New Therapeutic Approach for Duchenne Muscular Dystrophy: Restoration of the Dystrophin Reading Frame with a Meganuclease Jacques P. Tremblay, Pierre Chapdelaine, Christophe Pichavant, Joel Rousseau, Frédéric Pâques. 463 Regulatory Cassettes Derived from the Slow Troponin I Gene Confer High-Level Expression in Skeletal Muscle after Gene Transfer Mediated by Helper-Dependent Adenovirus and Recombinant AAV Rénald Gilbert, Mehdi Bendjelloul, Yué Zeng, Claude Guérin, Nancy Larochelle, George Karpati, Bernard Massie, Josephine Nalbantoglu. 464 Cell Therapy of Muscular Dystrophy with Engineered Cd133+ Cells Andrea Farini, Mirella Meregalli, Daniele Parolini, Marzia Belicchi, Simona Maciotta, Paola Razini, Joao da Silva Bizario, Luis Garcia, Nereo Bresolin, Yvan Torrente.

455 Characterization of A20 as an Inhibitor of NF-kB Activation in Dystrophic Mice Rakshita Charan, Paula R. Clemens.

465 Improved Regenerative Capacity of Adult Stem Cells Isolated from Injured Skeletal Muscle of Mouse Xiaodong Mu, Ian Bellayr, Haiying Pan, Yong Li.

456 Plasticity of Skeletal Muscle Cells during Muscle Injury and Repair – A Dedifferentiation Study Xiaodong Mu, Hairong Peng, Johnny Huard, Yong Li.

466 Sarcolemmal nNOS Anchoring Reveals a Functional Difference between Dystrophin and Utrophin Yongping Yue, Dejia Li, Luke Judge, Akshay Bareja, Yi Lai, Kay E. Davies, Jeffrey S. Chamberlain, Dongsheng Duan.

EXHIBITOR PROSPECTUS final program

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Musculo-skeletal Gene & Cell Therapy II 467 Detailed Physical Mapping of Deletion Breakpoints in the GSHP Dog Model for DMD Alock Malik, Acong Xu, Jesse Chen, Andrew Mead, Martin Childers, Janet Bogan, Scott Schatzberg, Joe Kornegay, Hansell Stedman. 468 Lentiviral Vector Mediated Delivery of Full-Length Dystrophin for Gene Therapy of Muscular Dystrophy En Kimura, Katsuhisa Uchio, Tomohiro Suga, tatsuya Koide, Yuji Uchida, Yasushi Maeda, Satoshi Yamoshita, Jeffrey S. Chamberlain, Makoto Uchino.

Cancer – Immunotherapy II

476 TCR Engineered and In Vitro Expanded T Lymphocytes Recapitulate the Properties of Central Memory T Cells Shicheng Yang, Zhili Zheng, Ling Zhang, Steven A. Rosenberg, Richard A. Morgan. 477 Phase I Safety and Immunogenicity of HPV 16 and 18 DNA Vaccines Delivered Via Electroporation Jian Yan, Panyupa Pankhong, Xuefei Shen, Mary Giffear, Jessica Lee, Drea Harris, Deanna Diaz, Amir Khan, Mark Bagarazzi, Jean Boyer, David B. Weiner, Niranjan Y. Sardesai. 478 Improving Adoptive T Cell Therapy by Transduction of TIL with a Vector That Mediates the Inducible Expression of IL-12 in the Tumor Microenvironment Ling Zhang, Sid P. Kerkard, Shicheng Yang, Zhili Zheng, Zhiya Yu, Nichaolas P. Restifo, Steven A. Rosenberg, Richard A. Morgan. 479 Robust and Sustained Ex Vivo Expansion of NK Cells with Artificial APCs Bearing Membrane-Bound IL-21 Is Associated with a Distinct Phenotype and Transcriptional Profile Cecele J. Denman, Vladimir Senyukov, Lisa M. Kopp, Maria Lima de Silva, Harjeet Singh, Lenka Hurton, Simon Olivares, Sourindra Maiti, Mary H. Huls, Richard E. Champlin, Laurence J. N. Cooper, Dean A. Lee. 480 STAT3-Inactivated Dendritic Cell Vaccination Could Induce Stronger Anti-Tumor Immune Responses Hidetoshi Sumimoto, Tomoko Iwata-Kajihara, Hiroyuki Mizuguchi, Kiyoshi Takeda, Yutaka Kawakami. 481 Combination Therapy for Melanoma Using Virotherapy and Alternate TLR Stimulating Agonists Rosa Diaz, Marta Compte-Grau, Elizabeth Ilett, Phonphimon Wongthida, Feorillo Galivo, Timothy Kottke, Jill Thompson, Alan Melcher, Richard Vile. 482 Bid Is a Key Factor for Eliciting the Antiproliferative Activity of IFN-a Mediated by TRAIL Takaya Tsuno, Josef Mejido, Tongmao Zhao, Terry Phillips, Kathryn Zoon. 483 Oral Adeno-Associated Virus-neu (AAV-neu) Immunotherapy Inhibits the Growth of neu+ Murine Breast Cancer Jason C. Steel, Giovanni Di Pasquale, Charmaine A. Ramlogan, Caroline Morris, John A. Chiorini, John C. Morris.

Friday, May 21st

469 Elimination of Cancer Stem Cells with Genetically Engineered T Cells Expressing a Stage Specific Embryonic Antigen-1 (SSEA1) Chimeric Antigen Receptor Antonina V. Kurtova, Juan Vera, An Lu, Adham Bear, Aaron E. Foster. 470 Targeting Cancer Stem Cells in Osteosarcoma Alexia Ghazi, Nino Rainusso, Vita Salsman, Helen Heslop, Steven Gottschalk, Nabil Ahmed. 471 CMV-Specific HER2-Redirected T Cells for the Adoptive Immunotherapy of Glioblastoma Alexia Ghazi, Vita Salsman, Yvonne Kew, Donald Shaffer, Suzanne Powell, Robert Grossman, Michael Scheurer, Ann Leen, Catherine Bollard, Helen Heslop, Stephen Gottschalk, Nabil Ahmed. 472 Combining mTor Inhibitors with Rapamycin-Resistant T Cells: A TwoPronged Approach to Tumor Elimination Leslie E. Huye, Barbara Savoldo, Gianpietro Dotti, Matthew H. Wilson, Cliona M. Rooney. 473 Evaluating the Combination of 5’Azacytidine and MAGE-A4-Specific T Cell Therapy as a Potential Immunotherapeutic Strategy for Refractory Hodgkin’s Lymphoma Conrad R. Cruz, Ulrike Gerdemann, Jessica A. Shafer, Ann M. Leen, Tamara Tripic, Terzah M. Horton, Anas Younes, Cliona M. Rooney, Helen E. Heslop, Catherine M. Bollard. 474 The iCD40.MyD88 Dendritic Cell (DC) Gene Switch Leads to Potent Anti-Tumor Therapy Natalia Lapteva, Priyadharshini Narayanan, Mamatha Seethammagari, Kevin M. Slawin, David M. Spencer.

475 Emozolomide Inhibits the Migration of Immune Cells into the Brain Tumor Microenvironment and Their Systemic Expansion, but Exerts a Synergistic Anti-Tumor Effect When Combined with Combined Conditional Cytotoxic/Immune-Gene Therapy Maria G. Castro, Marianela Candolfi, Kader Yagiz, Kurt M. Kroeger, David Foulad, Garbielle Ahlzadeh, Matthew Tesarfreund, Pedro R. Lowenstein.

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Cancer – Immunotherapy II 484 Tumor-Specific Antitumor Activity of mIL-10 Revealed by Hydrodynamic Tail Vein Injection of pCMV-mIL Plasmids in TumorBearing Mice Jianfeng Li, Li Hai, Shulin Li, Dexi Liu. 485 Liver Specific, Inducible, AAV-Mediated IL12 Delivered Resulted in a Strong Anti Tumoral Inmune Response in a Murine Model of Liver Metastasis Lucia Vanrell, Laura Blanco, Pedro Berraondo, Marianna Di Scala, Astrid Paneda, Victor Baldim, Jesus Prieto, Gloria González-Aseguinolaza.

Friday, May 21st

486 AdCD40L Gene Therapy Enhances the Efficacy of Chemotherapy in Experimental Bladder Cancer Lina S. E. Liljenfeldt, Lisa H. Christiansson, Katerina Gkirtzimanaki, Angelica S. I. Loskog, Aristides G. Eliopoulos. 487 Xenograft Expanded FANG Autologous Tumor Cell Vaccine Development and Manufacturing for Clinical Use Phillip B. Maples, Padmasini Kumar, Yang Yu, Beena O. Pappen, David Monsma, Stephanie Scott, Dawna Dylewski, Craig Webb, Neil Senzer, John Nemunaitis.

Cancer-Oncolytic Viruses II 488 Optimization of Measles Virus Infected Mesenchymal Stem Cell Carriers for Clinical Testing in Patients with Recurrent Ovarian Cancer Kah-Whye Peng, Emily K. Mader, Greg Butler, Marla Sommerfield, Sean C. Dowdy, Allan B. Dietz. 489 Expression of Interferon Alpha from an Oncolytic Adenovirus Vector Decreases Toxicity but Retains the Anti-Tumor Efficacy of the Vector Jacqueline F. Spencer, Debanjan Dhar, Elena V. Shashkova, Karoly Toth, William S. M. Wold. 490 Measles Virus Encoding H. pylori Neutrophil-Activating Protein for Treatment of Metastatic Breast Cancer Ianko D. Iankov, Cory Allen, Stephen J. Russell, Mark J. Federspiel, Evanthia Galanis. 491 Carrier Cells Delivery of Inducible Oncolytic Adenovurs for Brain Tumor Hiroshi Nakashima, E. A. Chiocca.

EXHIBITOR PROSPECTUS final program

492 Optimizing the Genomic Stability, Transgene Expression, and Therapeutic Efficacy of Replication-Competent Retrovirus Vectors for Prodrug Activator Gene Transfer Kei Hiraoka, Logg R. Christopher, Akihito Inagaki, Hiroshi Matsumoto, Oscar Diago, Omar Perez, Joan Robbins, Harry Gruber, Douglas Jolly, Noriyuki Kasahara. 493 Gene Therapy for Head and Neck Squamous Carcinoma Cells by IAI.3B Promoter-Driven Oncolytic Adenovirus-Infected Carrier Cells Katsuyuki Hamada, Ting Zhang, Koh-ichi Nakashiro, Hiroyuki Hamakawa. 494 Evaluation of Monocytes and Cancer Cells as Carriers for Oncolytic Adenoviruses in Immune-Competent Models of Pancreatic Cancer in Syrian Hamsters Eva Garcia-Aragoncillo, Sergia Bortolanza, Maria Buñuales, Jesus Prieto, Ruben Hernandez-Alcoceba. 495 Combination Therapy for Breast Cancer Using Oncolytic Viruses and Histone Deacetylase Inhibitors Hongxun Wang, Stephen J. Russell, Jian Qiao. 496 Combination Oncolytic Virotherapy Timothy Kottke, Jill Thompson, Konstantin Doronin, Sonia Guedan Carrio, William Wold, Ramon Alemany, Richard Vile. 497 Conditionally Replicative Adenovirus with an Adenoviral Major Late Promoter-Driven Imaging Cassette Predicts In Vivo Anti-Tumor Effect Julia Davydova, Tatyana Gavrikova, Eric J. Brown, Xianghua Luo, David T. Curiel, Selwyn M. Vickers, Masato Yamamoto. 498 Susceptibility of Neuroblastoma Primary Tumor-Initiating Cells to Oncolytic Herpes Simplex Virus Is Determined by Nectin-1 Expression Pin-Yi Wang, Mark A. Currier, Margaret H. Collins, Betsy A. DiPasquale, Loen Hansford, David Kaplan, Sean Lawler, E. Antonio Chiocca, Timothy P. Cripe. 499 Activity of Oncolytic Measles Virus Strains Against Brain Tumor Stem Cells Cory Allen, Mark A. Schroeder, Jann N. Sarkaria, Mark J. Federspiel, Stephen J. Russell, Evanthia Galanis. 500 Replication-Competent Retrovirus Vector-Mediated Suicide Gene Therapy Achieves Significant Therapeutic Efficacy Against Human Malignant Mesothelioma Xenografts Yoshiko Kawasaki, Norie Yamaoka, Nobuyuki Terada, Haruki Okamura, Noriyuki Kasahara, Shuji Kubo. 501 Oncolytic Adenovirus with Somatostatin Motif for Selective Infection of Neuroendocrine Tumors Di Yu, Justyna Leja, Berith Nilsson, Magnus Essand.

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Abstract Directory Friday, May 21, 2010 Poster Session II — continued 6:30 PM - 8:30 PM Room: Exhibit Hall C & B-South

Cancer-Oncolytic Viruses II 502 Silencing a p53 Inhibitor in Cancer Cells Increases Killing Potency of Oncolytic Adenovirus Christie Vermeulen, Nikki Tol, Winald R. Gerritsen, Victor W. van Beusechem. 503 Safety of Intravenous Ad5-Mda-7, a Potential Anti-Metastatic Agent in Gynecologic Cancer Kenneth H. Kim, Meredith A. Preuss, Minghui Wang, Anand C. Annan, Justin A. Barnes, Devanand Sarkar, Steven Grant, Paul Dent, Paul B. Fisher, Ronald D. Alvarez, David T. Curiel. 504 Characterization of a Novel Dual-Lock Oncolytic Cadvex-Vector That Combines Efficient Prostate-Specific Replication with Cytokine Expression Brian D. Hoel, Min Luo, David A. Einfeld, Danher Wang, John Y. Dong.

506 Adenovirus Vectors Transductionally Retargeted to Her2/neu for Prostate Tumour Treatment Maria K. Magnusson, Robert Kraaij, Corrina M. A. De Ridder, Leif Lindholm.

Cancer-Targeted Gene & Cell Therapy II 507 The Potential of Conditionally Replicating Adenovirus Expressing TIMP2 for Ovarian Cancer Therapy Sherry W. Yang, James J. Cody, Joanne T. Douglas, Ronald D. Alvarez, Selvarangan Ponnazhagan. 508 Mechanisms of the Tmpk/AZT-System Bystander Effect; Cell Fate Control Gene Therapy for Anti-Cancer Treatment Takeya Sato, Anton Neschadim, Jun Sukegawa, Teruyuki Yanagisawa, Jeffrey A. Medin. 509 Truncated CD19 Fused with Mutant Human Tmpk: A Versatile Cell Fate Control Safety Cassette Matthew D. Scaife, Sean P. Devine, Anton Neschadim, Natalia A. Pacienza, Daniel H. Fowler, Jeffrey A. Medin.

511 A Liposome Formulation with High Accumulation in Tumor Tissue for Targeted Delivery of Nucleic Acid Torben Gjetting, Camilla L. Christensen, Frederik Cramer, Hans S. Poulsen, Thomas L. Andresen. 512 15-LO-1 Induced Apoptosis Inhibits Tumour Growth and Extends Survival in Rat Glioma Model Agnieszka Pacholska, Helena Viita, Farizan Ahmad, Anna Hyvärinen, Thomas Wirth, Seppo Ylä-Herttuala. 513 An Efficient Targeted Gene Therapy Using Brain Tumor-Specific Promoter Toshio Yawata, Eri Ishida, Yu Kawanishi, Masakazu Tamura, Keiji Shimizu. 514 Nitric Oxide Synthase One Homologous Recombinant Negative (NOS1 -/-) Mice Are Protected from Thoracic Irradiation Induced Seizures by Esophageal Administration of Manganese Superoxide Dismutase Plasmid Liposome Complex (MnSOD-PL) Malolan Rajagopalan, Jean-Claude Rwigema, Brandon Stone, Michael Epperly, Julie Goff, Tracy Dixon, Darcy Franicola, Joel Greenberger. 515 Maternal Manganese Superoxide Dismutase Plasmid/Liposome Complex (MnSOD-PL) Gene Therapy of E13 Pregnant Mice Reduces Total Body Irradiation Induced Growth Retardation in Newborn Mice Michael Epperly, Tracy Dixon, Xichen Zhang, Benjamin Greenberger, Paavani Komanduri, Hong Wang, Joel Greenberger. 516 Adenovirus-Relaxin Gene Therapy on Keloids: Attenuated Proliferative Response and Collagen Degradation Il-Kyu Choi, Il-Kyu Lee, Yong Oock Kim, Dong Kyun Rah, Chae-Ok Yun. 517 Inducing Systemic Anti-Tumor Immune Responses through Direct Tumor Targeting G. Campbell Kaynor, Liaomin Peng, Jorge Sanchez-Salazar, Yang-Xin Fu, Xinzhong Wang. 518 Angiotensin Type 2 Receptor Mediated Apoptosis of Human Prostate Cancer Cells Hongwei Li, Yanfei Qi, Chengyao Li, Leah N. Braseth, Yongxin Gao, Arseniy E. Shabashvili, Michael J. Katovich, Colin Sumners. 519

Abstract Withdrawn

520 Regulatory Submission Process for Gene and Cellular Therapies Gladice Wallraven, Phillip B. Maples, John Nemunaitis.

Friday, May 21st

505 A Probasin Promoter, Conditionally Replicating Adenovirus that Expresses the Sodium Iodide Symporter (NIS) for Radiovirotherapy of Prostate Cancer Miguel A. Trujillo, Michael J. Oneal, Samantha McDonough, Rui Qin, John C. Morris.

510 Muscle Directed Systemic Cancer Gene Therapy for Lymphoma Using Type 8 AAV Vector Expressing mda-7/IL24 Nanya Wang, Bai Fan, Koichi Miyake, Noriko Miyake, Takashi Shimada.

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Abstract Directory Friday, May 21, 2010 Poster Session II  — continued 6:30 PM - 8:30 PM Room: Exhibit Hall C & B-South

Hematologic and Immunologic Gene & Cell Therapy II 521 Lentiviruses Containing a Modified Ankyrin-1 Promoter Direct Uniform, Copy-Number-Dependent Expression of a g-Globin Gene at Therapeutic Levels Faith Harrow, Karina Laflamme, Amanda Cline, David M. Bodine. 522 Non-Myeloablative Conditioning Regimen To Increase Engraftment of Gene-Modified Hematopoietic Stem Cells Teiko Sumiyoshi, Chang J. Lee, Roger Hollis, Francesca Giannoni, Stan F. Louie, Alice F. Tarantal, Donald B. Kohn.

Friday, May 21st

523 In Vivo Selection of Hematopoietic Stem Cells by Expressing a Truncated Epo Receptor (tEpoR) Maria Rosa Lidonnici, Francesca Tiboni, Giacomo Mandelli, Claudia Cattoglio, Margherita Galletti, Alexis Grande, Fulvio Mavilio, Giuliana Ferrari. 524 Successful Treatment of Canine Leukocyte Adhesion Deficiency Using a SIN Lentiviral Vector with the PGK Promoter Expressing Canine CD18 Michael J. Hunter, Huifen Zhao, Laura M. Tuschong, Tanya H. Burkholder, Derek A. Persons, Dennis D. Hickstein. 525 Diminished End Organ Damage and Correction of Anemia in Sickle Cell Mice Treated with Non-Myeloablative Busulfan Conditioning and g-Globin Lentiviral Vector Transduced HSCs Tamara I Pestina, Phillip W. Hargrove, Huifen Zhao, Peter Vogel, Derek A. Persons. 526 Small but Powerful, Predicting the Outcome of Gene Therapy Trial Using NSG Mice Tomoyasu Higashimoto, Elke Grassman, Jose A. Cancelas, Punam Malik. 527 Development of a Bioengineered Recombinant Factor VIII Therapeutic Using a Lentiviral Production Platform Christopher B. Doering, Gabriela Denning, Richard Gautney, Boro Dropulic, Lajos Baranyi, Andre J. Roy, Bagirath Gangadharan, Ernest T. Parker, Pete Lollar, H. Trent Spencer. 528 Transgenic Expression of Cytidine Deaminase (CDD) for Myeloprotective Purposes Nico Lachmann, Nils Pfaff, Sebastian Brennig, Doreen Lüttge, Tobias Cantz, Christopher Baum, Axel Schambach, Thomas Moritz.

EXHIBITOR PROSPECTUS final program

529 Development of a Pre-Clinical Model for the Gene Therapy Treatment of Wiskott-Aldrich Syndrome (WAS) Alexander Astrakhan, Byoung Ryu, Brigid Stirling, Blythe Sather, Jit Khim, Mikhail Garibov, Stephanie Humblet-Baron, Hans Ochs, David J. Rawlings. 530 Lentiviral Gene Therapy of Murine Hematopoietic Stem Cells Using Codon Optimized IL2RG cDNA: A Comparison of Multiple Promoter Elements and Transplant Conditions Marshall W. Huston, Niek P. van Til, Trudi P. Visser, Roya Sawari, Shazia Arshad, Gerard Wagemaker. 531 Human Fetal Liver HSCs Demonstrate High Transduction Rates and Sustained Transgene Expression Following Implantation Niraja Dighe, Maroun Khoury, Mark Chong, Citra Mattar, Michael N. Antoniou, Jianzhu Chen, Mahesh Choolani, Jerry Chan. 532 FANG Autologous Tumor Cell Vaccine Development and Manufacturing Phillip B. Maples, Padmasini Kumar, Yang Yu, Beena O. Pappen, Chris M. Jay, Zhaohui Wang, Donald D. Rao, Joseph Kuhn, John Nemunaitis, Neil Senzer. 533 Atomic Force Microscopy (AFM) Shows Lower Membrane Resistance of Human T Cells Rendered Deficient in WASP by RNAi Véronique Pariétti, Ruthger Van Zwieten, Malgorzata Lekka, Nicolas Mermod, Anne Galy. 534 A Combination of Codon Optimization and Amino Acid Substitution in the Human Factor IX Gene Yields a High Level of Superactive Factor IX Thipparat Suwanmanee, William Hannah, Tong Gui, Hong Ma, Adam S. Cockrell, Paul E. Monahan, Tal Kafri.

Immunologic & Host Responses in Gene & Cell Therapy 535 In Vivo Expansion of Regulatory T Cells with IL2-mAb Complexes Prevents and Modulates Anti-Factor VIII Immune Responses in Hemophilia A Mice after Factor VIII Plasmid-Mediated Gene Therapy Chao-Lien Liu, Peiqing Ye, Benjamin Yen, Baowei Peng, Carol H. Miao. 536 Phase I Safety and Pharmacokinetic Study of Systemic p53 Gene Therapy (SGT-53) Using Immunolipoplexes Targeted by AntiTransferrin Receptor (TfR) Single Chain Antibody Fragment (scFv): Updated Results Neil Senzer, John Nemunaitis, Cynthia Bedell, Robert Nunan, Henry Allen, Leanne Sleer, Esther Chang. 537 Induction of Type I Interferon (IFN) by Adenovirus-Encoded Small RNAs Tomoko Yamaguchi, Kenji Kawabata, Emi Kouyama, Takayuki Suzuki, Fuminori Sakurai, Shinnosuke Kurachi, Kazufumi Katayama, Hiroyuki Mizuguchi.

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Abstract Directory Friday, May 21, 2010 Poster Session II — continued 6:30 PM - 8:30 PM Room: Exhibit Hall C & B-South

Immunologic & Host Responses in Gene & Cell Therapy 538 Immunological Effects of Oral and Intravenous Cyclophosphamide in Cancer Patients Treated with Oncolytic Adenoviruses Vincenzo Cerullo, Sophie Escutenaire, Sari Pesonen, Iulia Diaconu, Liisa Sirrka, Anna Kanerva, Lotta Kangasniemi, Elina Haavisto, Minna Oksanen, Tiimo Joensuu, A. KariojaKallio, Petteri Arstila, Akseli Hemminki. 539 The Role of TGF-b and IL-10 in the Development of Immune Tolerance Following Hepatic Gene Therapy Brad E. Hoffman, Ashley T. Martino, Brandon K. Sack, Roland W. Herzog. 540 Murine Hepatocytes Transduced with Y-F Mutant Capsid Are More Resistant to Killing by Capsid Specific CD8+ T Cells Ashley Martino, David M. Markusic, Arun Srivastava, Roland W. Herzog. 541

Abstract Withdrawn

548 Overcoming Anti-AAV Neutralizing Antibodies with Alternate Serotype and Pharmacological Strategies Federico Mingozzi, Margriet J. Vervoordeldonk, Yifeng Chen, Shyrie Edmonson, Rogier Thurlings, Katherine High, Paul P. Tak. 549 Switching the Fiber Knob of Oncolytic Adenoviruses To Avoid Neutralizing Antibodies in Human Cancer Patients Mari Raki, Iulia Diaconu, Merja Sarkioja, Sophie Escutenaire, Lotta Kangasniemi, Anna Kanerva, Vincenzo Cerullo, Timo Joensuu, Sari Pesonen, Akseli Hemminki. 550 Depletion of B Cells by Anti-CD20 Partially Regulates Anti-FVIII Antibody Production in the Nonviral Gene Therapy Model Peiqing Ye, Baowei Peng, Marilyn Kehry, David J. Rawlings, Carol H. Miao. 551 Sheep Harbor Naturally-Occurring Antibodies to Human AAV: A New Large Animal Model for AAV Immunology Joseph Tellez, Jonathan D. Finn, Nick Tschernia, Graça Almeida-Porada, Valder R. Arruda, Christopher D. Porada. 552 Effects of Various TLR/Inflammation Pathways on Humoral and Cellular Immune Response to rAAV1 in Mice Muriel Sudres, Virginie Vasseur, Séverine Ciré, Bernhard Ryffel, Anne Galy.

543 AAV8 Vectors Evade CTL Mediated Destruction by Failing To Activate a Potent Inflammatory Response Suryanarayan Somanathan, Ekaterina Breous, Peter L. Bell, James M. Wilson.

553 Monitoring and Suppression of Immune Responses to AAV-Mediated Gene Therapy in Canine Muscle Zejing Wang, Rainer Storb, Donghoon Lee, Martin Kushmeric, Baocheng Chu, Jeffery Chamberlain, Stainly Riddell, Stephen Tapscott.

544 Robust and Sustained Factor IX Expression by IV Administration of AAV8-Based Vectors in Macaques Hiroaki Mizukami, Jun Mimuro, Akira Ishiwata, Hiroya Yagi, Tsukasa Ohmori, Seiji Madoiwa, Tomonori Tsukahara, Masashi Urabe, Akihiro Kume, Yoichi Sakata, Keiya Ozawa.

554 Prevalence of Neutralizing Antibodies Against Adeno-Associated Virus 2, 8 and 9 in Non-Human Primate Colonies Hiroya Yagi, Hiroaki Mizukami, Tomonori Tsukahara, Masashi Urabe, Hiromi Hamada, Akihiro Kume, Hiroyuki Yoshikawa, Keiya Ozawa.

545 Induction of Transgene Immunological Tolerance by a Single Injection of miR-142-Regulated Integrase Defective Lentiviral Vector Andrea Annoni, Alessio Cantore, Lucia Sergi Sergi, Luigi Naldini, Maria Grazia Roncarolo.

Oligonucleotide & RNAi Therapeutics II

546 T Cell Hyperactivity in Cmah Null Mice: A Pre-Clinical rAAV Immunotoxicity Model? George Buchlis, Federico Mingozzi, Daniel J. Hui, Harre Downey, Virginia Haurigot, Shangzhen Zhou, Ajit Varki, Katherine A. High.

555 Dual Function BAFF Receptor Aptamers Block BAFF1 Ligand Induced Proliferation and Deliver siRNAS to NHL Cells Katrin Tiemann, Stephen J. Forman, John J. Rossi, Jiehua Zhou. 556 Efficient Delivery of siRNAs into Primary Cells In Vitro and Systemically In Vivo Using Chemically Modified PepFect Peptides Samir El Andaloussi, Edvard C. I. Smith, Ülo Langel.

Friday, May 21st

542 The Innate Immune Response to Intravenously Injected Adenoviral Vectors: Role of Endosomal Escape in MAP Kinase Activation and the Cytokine/Chemokine Response Jeffrey S. Smith, Zhili Xu, Jie Tian, Donna J. Palmer, Philip Ng, Andrew P. Byrnes.

547 Complement-Mediated Clearance of AAV by Tissue Resident Macrophages Limits Transduction of Target Cells Chelsea M. Bolyard, Jeffrey S. Bartlett.

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Abstract Directory Friday, May 21, 2010 Poster Session II — continued 6:30 PM - 8:30 PM Room: Exhibit Hall C & B-South

Oligonucleotide & RNAi Therapeutics II 557 Reduction of Mutant Huntingtin by Antisense Oligonucleotides Ameliorates Phenotypic Deficits in YAC128 Mouse Model of Huntington’s Disease Jonathan A. Fidler, Lisa M. Stanek, Wendy Yang, Amy M. Richards, Edward Wancewicz, Curt Mazur, Gene Hung, C. Frank Bennett, Seng H. Cheng, Lamya S. Shihabuddin. 558 Antibody-Mediated Targeting of pH-Responsive Nanoparticles Facilitates siRNA Delivery to Cancer Cells Maria C. Palanca-Wessels, Richelle Cutler-Strom, Anthony C. Convertine, Fan Lee, Michael F. Press, Scott E. James, Patrick S. Stayton, Oliver W. Press.

Friday, May 21st

559 Adenoviral shRNA-Mediated Knockdown of TNFR1(P55) Either Locally in the Knee Joint or Systemically Ameliorates CollagenInduced Arthritis Onno J. Arntz, Jeroen Geurts, Sharon Veenbergen, Miranda B. Bennink, Wim B. van den Berg, Fons A. J. van de Loo. 560 Specific and Efficacious RNA Aptamer Against Hepatic Metastasis of Colon Cancer Seong-Wook Lee, Young Ju Lee, Seung Ryul Han, Min jung Kim, Hanna Cho, Won Il Lee, Jin-Sook Jeong. 561 Development of Aptamer Therapeutics Against Cell Surface Receptors for Lymphoma and Auto-Immune Diseases Elizabeth D. Pratico, David Boczkowski, Smita Nair, Bruce A. Sullenger. 562 A Microscopic Study of the Intracellular Fate of Cationic Mediated siRNA Delivery Meysam Abbasi, Rakesh Bhatnagar, Hasan Uludag. 563 Deriving Effective Anti-HIV Guide Sequences from Enhanced Primary microRNA Secondary Structures Fiona van den Berg, Patrick Arbuthnot, Marc S. Weinberg. 564 “Bifunctional” shRNA Strategy for Cancer Gene and Cell Therapy Donald D. Rao, Zhaohui Wang, Padmasini Kumar, Yang Yu, Shihe Liu, Chris M. Jay, Nancy S. Templeton, Courtney Haddock, John Vorhies, Alex W. Tong, Phillip B. Maples, Neil Senzer, F. C. Brunicardi, John Nemunaitis.

EXHIBITOR PROSPECTUS final program

565 Feasibility and Effectiveness of Exon 51 skipping in Human-Like mdx Mutation Yoshitsugu Aoki, Toshifumi Yokota, Takashi Saito, Akinori Nakamura, Tetsuya Nagata, Hitoshi Okazawa, Shin’ichi Takeda. 566 The Effect of RNA Interfering a1,3GT and NF-kB in Cardiac Xenotransplantation Feng Qi, Chengyu Lv, Yachuan Gu, Xianghui He, Liwei Zhu. 567 Inhibition of Breast Cancer Cell Growth Via Silencing IKKe Bin Qin, Ravi S. Shukla, Kun Cheng. 568 Cationic Polymer Mediated siRNA Delivery for P-glycoprotein (P-gp) Down-Regulation in Tumor Cells Meysam Abbasi, Afsaneh Lavasanifar, Michael Weinfeld, Hasan Uludag. 569 Gene Editing Induces Double-Stranded Breakage in Mammalian Cells Melissa A. Bonner, Eric B. Kmiec.

Cell Processing and Vector Production II 570 Development of a MDCK Cell Line for the Manufacturing of Canine Adenovirus Type 2 (CAV-2) Vectors Virgínia Santiago, Eric Kremer, Paula M. Alves, Ana S. Coroadinha. 571 Production of a rAAV1-CB-hAAT Human Clinical Trial Lot by rHSV Co-Infection of Suspension BHK Cells David R. Knop, Brad Grimm, Lijun Wang, Darby L. Thomas, Guo-jie Ye. 572 RNA Based Plasmid Selection System for Antibiotic-Free Plasmid DNA Vector Production Aaron E. Carnes, Jeremy M. Luke, Justin M. Vincent, Clague P. Hodgson, James A. Williams. 573 Robust Recombinase-Free System Production of Helper-Dependent Adenoviral Vectors with Negligible Contamination by Delayed Packaging Process Dan Cots, Raul Alba, Patrick Hearing, Eric J. Kremer, Maria Mercedes Segura, Assumpció Bosch, Miguel Chillón. 574 Removal of Clinically Relevant Contaminants from cGMP Grade Preparations of Plasmid DNA: Implications for Non-Viral Gene Therapy and DNA-Based Vaccines Nancy S. Templeton, Pervez Firozi, Wei Zhang, Li Chen, Florante A. Quiocho, Kim C. Worley, Phillip B. Maples, John Nemunaitis.

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Abstract Directory Friday, May 21, 2010 Poster Session II — continued 6:30 PM - 8:30 PM Room: Exhibit Hall C & B-South

Cell Processing and Vector Production II 575 Escherichia coli Plasmid DNA Fermentation Strain and Process-Specific Effects on Vector Yield, Quality and Transgene Expression Aaron E. Carnes, Jeremy M. Luke, Justin M. Vincent, Clague P. Hodgson, James A. Williams. 576 An Assay To Monitor the Residual Contamination in the Plasmid DNA Manufacturing Area Based on the Correlation of Plasmid DNA Concentration to TOC Rameswari Rameswaran, Lee Allen, Shane Johnson, Henry Hebel. 577 Counting and Sizing of Viral Vector Particles and Aggregates by Nanoparticle Tracking Analysis (NTA) Andrew Malloy, Duncan Griffiths, Patrick Hole, Bob Carr.

579 Dissolved Oxygen and pH Profile Evolution in Shake Flasks during 293 Cell Growth and Infection with Adenovirus Ana Carina Silva, Daniel Simão, Marcos F. Q. Sousa, Pedro E. Cruz, Manuel J. T. Carrondo, Paula M. Alves.

933 Targeted Lentiviral Vectors for the Gene Therapy of X-Linked Chronic Granulomatous Disease Christian Brendel, Stefan Schultze-Strasser, Uta Müller-Kuller,Serap Tokmak, Hana Kunkel, Linping Chen-Wichmann, Stefan Stein, Eva Rudolf, Michael Antoniou, Manuel Grez 934 Meganucleases: A Novel Anti-Viral Approach Julianne Smith, Roman Galetto, Stephanie Grosse, Cécile Schiffer Mannioui, Sylvain Arnould, Marine Gailledrat, Philippe Duchateau, Frédéric Pâques & Carole Desseaux 935 Monomeric Designer T Cells are More Potent against IL13Ra2 Expressing GBM Seogkyoung Kong, Richard P Junghans, , Prakash Sampath 936 pSuperShuttle – An Easy Method for Modifications at any Site in the Adenoviral Genome by Conventional Bacterial Recombination Daniel Oberg, Nick R Lemoine, Gunnel Hallden 937 Generation and Characterization of Lipid-Encapsulated AAV vectors: A Strategy to Evade Neutralization by AAV Antibodies J. Fraser Wright, Sissel Lund-Katz

Friday, May 21st

578 Manufacturing and Stability Testing of a GNE-Lipoplex for a Clinical Trial for the Treatment of Hereditary Inclusion Body Myopathy Type (HIBM) Chris M. Jay, Courtney Haddock, Phillip B. Maples, John Nemunaitis.

Late Breaking Science II

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Abstract Directory Saturday, May 22, 2010 Oral Abstract Session 410 Musculo-skeletal Gene & Cell Therapy 11:00 AM - 1:00 PM Room: Delaware Suite 580 11:00 AM Irradiation of Adult mdx Mice Prior to Full-Length Murine Dystrophin cDNA Transfer Results in a Delayed and Diminished Anti-Dystrophin Host Immune Response Saman Eghtesad, Heng Zheng, Hiroyuki Nakai, Michael W. Epperly, Paula R. Clemens. 581 11:15 AM Transient Expression of a Therapeutic Dystrophin Transgene in Duchenne Muscular Dystrophy Revealed by T Cell Mediated Immunity Jerry R. Mendell, Katherine Campbell, Louise R. Rodino-Klapac, Zarife Sahenk, Christopher Shilling, Sarah Lewis, Dawn Bowles, Steven Gray, Chengwin Li, Gloria Galloway, Vinod Malik, Brian Coley, K. Reed Clark, Juan Li, Xiao Xiao, Jade Samulski, Scott McPhee, R. Jude Samulski, Christopher M. Walker. 582 11:30 AM Induction of Oral Immunotolerance to rAAV9-Microdystrophin in Canine X-Linked Muscular Dystrophy Hiromi H. Kinoh, Naoko Yugeta, Hironori Okada, Yuko N. Kasahara, Takashi Okada, Shin’ichi Takeda. 583 11:45 AM Baculovirus-Engineered Mesenchymal Stem Cells Heal the CriticalSized Femoral Segmental Bone Defects in Rabbits Chin-Yu Lin, Yu-Han Chang, Kun-Ju Lin, Tzu-Chen Yen, Wen-Yi Lo, Chia-Hsin Luo, ChingLung Tai, Yu-Chen Hu. 584 12:00 PM Genetic Evidence for a Structural Role of a–dystrobrevin-3 within the Dystrophin-Glycoprotein Complex Guy L. Odom, Glen B. Banks, Dewayne Townsend, Marvin E. Adams, Joseph M. Metzger, Stanely C. Froehner, Jeffrey S. Chamberlain.

Saturday, May 22nd

585 12:15 PM rAAV8-Mediated Protein-Anchoring Therapy for Targeting Collagen Q-Tailed Acetylcholinesterase to the Neuromuscular Junction Mikako Ito, Yumi Suzuki, Takashi Okada, Takayasu Fukudome, Toshiro Yoshimura, Akio Masuda, Shin’ichi Takeda, Eric Krejci, Kinji Ohno. 586 12:30 PM A Single Patient, Multiple Intravenous (IV) Infusion, Clinical Trial of GNE-Lipoplex for Therapy of Hereditary Inclusion Body Myopathy (HIBM) Gregory Nemunaitis, Chris M. Jay, Neil Senzer, Henry Allen, Anagha P. Phadke, Nancy S. Templeton, Phillip B. Maples, John Nemunaitis.

EXHIBITOR PROSPECTUS final program

587 12:45 PM Successful Expression of a R16/17 Micro-Dystrophin Gene in Dystrophic Dogs with a Tyrosine-Mutant AAV Vector and a Brief Course of Immune Suppression Jin-Hong Shin, Yi Lai, Yongping Yue, Arun Srivastava, Bruce F. Smith, Dongsheng Duan.

Oral Abstract Session 411 Chemical and Molecular Conjugates 11:00 AM - 1:00 PM Room: Virginia Suite 588 11:00 AM Transcription Factor Binding by Plasmid DNA Is Needed for Interaction with Microtubules and Trafficking in Cells Melissa A. Badding, Erin E. Vaughan, David A. Dean. 589 11:15 AM Quantitative Analysis of the Effect of Functional Peptides on Intracellular Trafficking of Polymer Gene Delivery Systems Nicole M. Moore, Denis Pristinski, Shelly E. Sakiyama-Elbert. 590 11:30 AM The Effects of PNA-Based prePEGylation on the In Vitro and In Vivo Properties of DNA Polyplexes Peter G. Millili, Ulhas P. Naik, Millicent O. Sullivan. 591 11:45 AM Particle Tracking for Intracellular Trafficking of Octaarginine-Modified Liposome: Comparative Study with Adenovirus Hidetaka Akita, Kaoru Enoto, Tomoya Masuda, Hiroyuki Mizuguchi, Tomomi Tani, Hideyoshi Harashima. 592 12:00 PM Advanced In Vitro Models Combined with Mathematical Representations Yield New Quantitative Understanding of Synthetic Gene Delivery System Performance Michelle C. Lowe, Todd D. Giorgio. 593 12:15 PM Gene Therapy of the Blood Brain Barrier: Systemic Delivery of shRNA-Producing Plasmid DNA for Transient Suppression of MRP4 Drug Efflux Transporter Protein Hongwei Zhang, Michelle L. Varney, Rakesh K. Singh, Serguei V. Vinogradov. 594 12:30 PM PEGylated Polyacridine-Peptide DNA Open-Polyplexes with Long Pharmacokinetic Half-Life Have Improved Hydrodynamic Stimulated Gene Expression Christian A. Fernandez, Nicholas J. Baumhover, Jason Duskey, Sanjib Khargharia, Kevin G. Rice. 595 12:45 PM Successful Gene Delivery with Receptor-Targeted PAMAM Dendrimers Occurs Via Caveolae-Mediated Pathways Mark E. Hwang, William R. Gunther, Daniel W. Pack.

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Abstract Directory Saturday, May 22, 2010

Oral Abstract Session 413 Cancer-Oncolytic Viruses II

Oral Abstract Session 412 Cardiovascular Gene & Cell Therapy 11:00 AM - 1:00 PM Room: Wilson ABC 596 11:00 AM Systemic Delivery of AAV2/9 Vectors Improve Cardiac Function in Pompe Disease Darin J. Falk, Meghan S. Soustek, Cathryn S. Mah, Denise A. Cloutier, Sean A. Germain, Barry J. Byrne. 597 11:15 AM Coordinated Co-Expression of VEGF164 and PDGF-BB Prevents Aberrant Angiogenesis and Accelerates Vascular Stabilization Roberto Gianni-Barrera, Silvia Reginato, Thomas Wolff, Lorenz Gürke, Michael Heberer, Andrea Banfi. 598 11:30 AM Mitral Valve Disease Is Due to Abnormal Collagen Structure in Canine Mucopolysaccharidosis I and VII and Can Be Reduced with Neonatal Intravenous Injection of a Gamma Retroviral Vector Katherine P. Ponder, Meg M. Sleeper, Susan Wu, Patricia O’Donnell, Mark E. Haskins. 599 11:45 AM AAV9-Mediated proBNP Delivery Improves Diastolic Performance in a Model of Spontaneous Hypertensive Rats Jason M. Tonne, Jarryd M. Campbell, Diego Bellavia, Elise A. Oehler, Fernando L. Martin, Alessandro Cataliotti, John C. Burnett, Yasuhiro Ikeda. 600 12:00 PM In Vivo Over-Expression of a b-Adrenergic Receptor Kinase Inhibitor Using scAAV6-Mediated Molecular Cardiac Surgery with Recirculating Delivery (MCARD™) Enhances Cardiac Function JaBaris D. Swain, Michael G. Katz, Anthony Fargnoli, Marina Sumaroka, Catherine Tomasulo, Mihail Petrov, James J. Pilla, Hansell H. Stedman, Walter J. Koch, Joseph Rabinowitz, Charles R. Bridges. 601 12:15 PM AAV-Mediated SERCA2a Expression Improves the Electrocardiographic Profile of Aged mdx Mice Jin-Hong Shin, Brian P. Bostick, Yongping Yue, Roger J. Hajjar, Dongsheng Duan.

603 12:45 PM SDF-1a-Engineered Cell-Based Therapy for Diabetic Wound Healing Zhao-Jun Liu, Runxia Tian, Weijun An, Ying Zhuge, Yan Li, Hongwei Shao, Bianca Habib, Omaida C. Velazquez.

604 11:00 AM High Resolution Analysis of the Intratumoral Spread of an Intravenously Administered Oncolytic Virus Shruthi Naik, Rebecca Nace, Elizabeth Hadac, Glen Barber, Stephen J. Russell. 605

Abstract Withdrawn

191 11:15 AM A Comparative Study of Stem Cell-Based Cell Carrier Systems for the Systemic Delivery of an Oncolytic Adenovirus for Antiglioma Therapy Matthew A. Tayler, Atique U. Ahmed, Ilya V. Ulasov, Irina Y. Balyasnikova, Maciej S. Lesniak. 606 11:30 AM Combination of Anti Angiogenic Cancer Therapy with Systemic Oncolytic Virotherapy To Treat Established Tumors Timothy Kottke, Geoff Hall, Jose Pulido, Rosa Diaz, Jill Thompson, Debabrata Mukhopadhyay, Heung Chong, Peter Selby, Matt Coffey, Hardev Pandha, John Chester, Alan Melcher, Kevin Harrington, Richard Vile. 607 11:45 AM The Type III Interferon IL-28 Mediates Anti Tumor Efficacy of Oncolytic VSV in a Fully Immune Competent Model Phonphimon Wongthida, Rosa Diaz, Feorillo Galivo, Timothy Kottke, Jill Thompson, Jose Pulido, Kevin Pavelko, Larry Pease, Alan Melcher, Richard Vile. 608 12:00 PM A New Oncolytic Herpes Simplex Virus That Synergizes with Small Molecule Inhibitors of PI3K/Akt To Treat Malignant Glioma Ryuichi Kanai, Matthew Gardner, Hiroaki Wakimoto, Robert L. Martuza, Samuel D. Rabkin. 609 12:15 PM Improving the Lytic Capacity of a Conditionally Replicative Adenoviruses by Incorporating Promoter Elements Responsive to Tumor Environmental Conditons Diego L. Viale, Veronica M. Lopez, Eduardo G. Cafferata, David Gould, Yuti Chenajovsky, Osvaldo L. Podhajcer. 610 12:30 PM Defining the Role of Innate Immune Mechanisms in the Efficacy of Oncolytic VSV Virotherapy in a Fully Immune Competent Model Phonphimon Wongthida, Rosa Diaz, Feorillo Galivo, Timothy Kottke, Jill Thompson, Jose Pulido, Alan Melcher, Richard Vile. 611 12:45 PM Differential Effects of VSV and Cyclophosphamide on NK Cells Leads to Antagonistic Effects on Anti-Tumor Therapy of a Murine Mesothelioma Model Candice L. Willmon, Jill Thompson, Timothy Kottke, Stephen Albelda, Richard Vile.

Saturday, May 22nd

602 12:30 PM RNAi-Mediated Knockdown of HMG CoA Reductase In Vitro and In Vivo Increases Transgene Expression from Genomic LDLR Mini-Gene Vectors Olivia C. Hibbitt, Sarina Agkatsev, Carina Owen, Len Seymour, Keith M. Channon, Richard Wade-Martins.

11:00 AM - 1:00 PM Room: Marriott Ballroom Salons 2 & 3

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Abstract Directory Saturday, May 22, 2010 Oral Abstract Session 414 Hematologic/Immunologic: Clinical, Preclinical and Safety 11:00 AM - 1:00 PM Room: Maryland Suite 612 11:00 AM Polyclonal Reconstitution of the Hematopoietic System in Two Patients of a WAS Clinical Gene Therapy Trial Anna Paruzynski, Kaan Boztug, Claudia Ball, Ali Nowrouzi, Christina Lulay, Marie Böhm, Sonja Naundorf, Klaus Kühlke, Rainer Blasczyk, Irina Kondratenko, Laszló Maródi, Hanno Glimm, Christoph Klein, Christof von Kalle, Manfred Schmidt. 613 11:15 AM Sustained Partial Phenotypic Correction of Hemophilia B in the Dog by a microRNA-Regulated Lentiviral Vector Alessio Cantore, Marco Ranzani, Lucia Sergi Sergi, Giuliana Vallanti, Marina Radrizzani, Brian Brown, Eugenio Montini, Timothy Nichols, Luigi Naldini. 614 11:30 AM Timing of Anti-T Cell Immunosuppressive Therapy Is Critical for the Safety of Liver Mediated AAV2 Transduction in Rhesus Macaques Jonathan D. Finn, Patricia Favaro, J. Fraser Wright, Federico Mingozzi, Katherine A. High, Valder R. Arruda. 615 11:45 AM Factor IX-R338L (FIX Padua) as a Novel Alternative for the Treatment of Canine Severe Hemophilia B Jonathan D. Finn, Paolo Simioni, Nicholas Iacobelli, Shangzhen Zhou, Timothy C. Nichols, Katherine A. High, Valder R. Arruda.

Saturday, May 22nd

616 12:00 PM Erythropoeisis Sustained 1 Year by the EPODURE BioPump in Patients with Chronic Kidney Disease: Further Results of Phase I/II Proof of Concept Trial Baruch S. Stern, Ehud Shoshani, Andrew L. Pearlman, Philip Ng, Allen R. Nissenson, Eithan Galun, Anatole Besarab, Avi Rimler, Haim Goltzmann, Reem Miari, Alex Okun, Haddas Bookay, Tamar Jibly, Javier A. Abrameto, Michal Elhalel.

619 12:45 PM Lentiviral Gene Transfer under Optimized Cytokine Conditions Facilitates Polyclonal Hematopoiesis of Recipient Mice Tobias Maetzig, Martijn H. Brugman, Olga S. Kustikova, Zhixiong Li, Melanie Galla, Niels Heinz, Ute Modlich, Bernhard Schiedlmeier, Axel Schambach, Christopher Baum.

Oral Abstract Session 415 Lung and Respiratory Disease Gene & Cell Therapy 11:00 AM - 12:00 PM Room: Thurgood Marshall W 620 11:00 AM Generation of a Transgenic Gut-Corrected CFTR Knockout Ferret Model with Intestinal-Specific Expression of Ferret CFTR Xiaoming Liu, Xingshen Sun, John T. Fisher, Meihui Luo, Hongshu Shui, Ziying Yan, Yaling Yi, Michael J. Goodheart, Gergely Lukacs, David Meyerholz, John F. Engelhardt. 621 11:15 AM Persistent Long Term Lentiviral Correction of the CFTR Gene Defect in Cystic Fibrosis Mice Patricia Cmielewski, Donald Anson, David W. Parsons. 622 11:30 AM Development of Optimized hCFTR Gene Sequence and Prolonged Expression Vector for Cystic Fibrosis Therapy Linas Padegimas, Karla A. Dines, Samuel J. Adams, Sharon M. Oette, Susannah L. Hyatt, Olena Tyr, Christopher R. Gedeon, Ozge Sesenoglu-Laird, Robert C. Moen, Tomasz H. Kowalczyk, Mark J. Cooper. 623 11:45 AM Expression of Human Alpha-1 Antitrypsin in Mice and Dogs Following AAV6 Vector-Mediated Gene Transfer to the Lungs Christine L. Halbert, David K. Madtes, Andrew E. Vaughan, Zejing Wang, Rainer Storb, Stephen J. Tapscott, A. Dusty Miller.

Oral Abstract Session 416 Gene Regulation 12:15 PM - 1:15 PM Room: Thurgood Marshall W

617 12:15 PM Lentiviral Gene Therapy of RAG1 Severe Combined Immunodeficiency: Risks and Implications for Treatment Niek P. van Til, Roya Sarwari, Trudi P. Visser, Helen de Boer, Yuedan Li, Julia Hauer, Chantal Lagresle-Peyrou, Olivier Danos, Arnaud Jollet, Fang Zhang, Adrian Thrasher, Barbara Cassani, Monique M. Verstegen, Anna Villa, Marina Cavazzana-Calvo, Gerard Wagemaker.

624 12:15 PM Engineered Zinc Finger Transcription Factors for the Treatment of Gain of Function Mutations Claudio Mussolino, Daniela Sanges, Ciro Bonetti, Elena Marrocco, Umberto Di Vicino, Valeria Marigo, Alberto Auricchio, Germana Meroni, Enrico M. Surace.

618 12:30 PM Disruption of Glucocorticoid Regulation in Therapeutic Human T Cells with Zinc Finger Nucleases Rebecca A. Gardner, Andreas Reik, Carolina Berger, Seitaro Terakura, Philip D. Gregory, Michael C. Jensen, Michael C. Holmes, Stanley R. Riddell.

625 12:30 PM AAV Vectors for Tissue-Specific, Regulated Expression in Heart, Liver and Lung Shu-Jen Chen, Maria P. Limberis, Lawrence Bish, Ruben Hovhannisyan, Sheena Saighal, Odella Jno-Charles, H. Lee Sweeney, Arbans Sandhu, Julie Johnston, James M. Wilson.

EXHIBITOR PROSPECTUS final program

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Abstract Directory Saturday, May 22, 2010 Oral Abstract Session 416 — continued Gene Regulation 12:15 PM - 1:15 PM Room: Thurgood Marshall W 626 12:45 PM Repairing Huntingtin RNA by Trans-Splicing Hansjorg Rindt, Tristan H. Coady, Christian L. Lorson. 627 1:00 PM Towards Personalized Arthritis Gene Therapy: Screening of Computationally-Designed Promoters in an In Vivo Mouse Model and in Human Synovial Fibroblasts Eline A. Vermeij, Jeroen Geurts, Onno J. Arntz, Miranda B. Bennink, Wim B. van den Berg, Fons A. J. van de Loo.

Oral Abstract Session 417 Stem Cell Therapies 11:00 AM - 1:00 PM Room: Marriott Ballroom Salon 1 628 11:00 AM Contribution of PDGFRa-Positive Bone Marrow Cells for Epithelial Regeneration in Genetic Blistering Skin Disease, RDEB Katsuto Tamai, Takenao Chino, Takehiko Yamazaki, Yasufumi Kaneda. 629 11:15 AM Adult Muscle-Derived Stem Cells Rescue Progeria Stem Cell Dysfunction and Promote Angiogenesis Mitra Lavasani, Andria R. Robinson, Aiping Lu, Joseph M. Feduska, Jeremy S. Tilstra, Paul D. Robbins, Laura J. Niedernhofer, Johnny Huard. 630 11:30 AM AAV-2 Delivered Factor IX Is Secreted with Full Clotting Activity from Human Adipose Stromal Cells Differentiated towards Hepatocytes Katherine T. Marcucci, Katie Bisordi, Shangzhen Zhou, Keith L. March, Elliot D. Rosen, Katherine A. High.

632 12:00 PM Stealth Delivery: Combining Site-Specific Integration and Cassette Design To Achieve Robust Expression without Impacting Endogenous Gene Expression Angelo Lombardo, Daniela Cesana, Pietro Genovese, Elena Provasi, Bruno Di Stefano, Margherita Neri, Vania Broccoli, Angela Gritti, Michael C. Holmes, Philip D. Gregory, Chiara Bonini, Luigi Naldini.

634 12:30 PM Simple and Efficient Preparation of Immaculate Human iPS Cells with Transgene-Free and No Chromosomal Damages Using an Extranuclear Manipulation System of Genetic Information, PlasmExTM Noemi Fusaki, Hiroshi Ban, Koichi Saeki, Toshiaki Tabata, Makoto Inoue, Mamoru Hasegawa. 635 12:45 PM Myogenic Potential Enhancement of Muscle Stem Cell by Reducing NF-kB Expression Aiping Lu, Jonathan Proto, Ying Tang, Mitra Lavasani, Bing Wang, Jeremy S. Tilstra, Laura J. Niedernhofer, Denis C. Guttridge, Paul D. Robbins, Johnny Huard.

Oral Abstract Session 430 Adenovirus and Other DNA Virus Vectors 3:00 PM - 5:00 PM Room: Maryland Suite 636 3:00 PM Mapping of Transposon Integration Sites in Murine and Canine Liver after Transposition from a High-Capacity Adenoviral Vector Wenli Zhang, Martin Hausl, Nadine Muether, Anja Ehrhardt. 637 3:15 PM Endogenous miRNA-Regulation Can Be Used To Enhance Transgene Product-Directed Immune Responses Induced by Adenovirus-Based Genetic Vaccines Matthias W. Kron, Tatjana Engler, Stefan Kochanek, Florian Kreppel. 638 3:30 PM Novel Class of Adenovirus Vector “Capsid-Displaying” the RetroOriented Human Complement Inhibitor DAF Has a Significantly Improved Safety Profile In Vivo Sergey S. Seregin, Yasser A. Aldhamen, Daniel M. Appledorn, Zachary C. Hartman, Nathaniel J. Schuldt, Jeannine Scott, Sarah S. Godbehere, Haixiang Jiang, Michael M. Frank, Andrea Amalfitano. 639 3:45 PM Sustained Therapeutic hFIX Levels in Rhesus Macaques without Neutralizing Anti-hFIX Antibody Development Following CatheterBased Delivery of HDAd Nicola Brunetti-Pierri, Nathan C. Grove, Priti M. Patel, Donna Palmer, Arthur Beaudet, Charles Mullins, Philip Ng. 640 4:00 PM Fully Retargeted HSV-1 Infection Directed by Re-Engineered Glycoprotein D (gD) Is Augmented by Hyperactive gB Mutations Hiroaki Uchida, Janet Chan, William F. Goins, Paola Grandi, Kenji Nakano, Izumi Kumagai, Justus B. Cohen, Joseph C. Glorioso. 641 4:15 PM Gene Expression Profiling of Adenovirus 5 Infections: Identifying Key Pathways That Affect Oncolytic Adenovirus Replication Dominik E. Dorer, Frank Holtrup, Kurt Fellenberg, Jörg Hoheisel, Dirk M. Nettelbeck.

Saturday, May 22nd

631 11:45 AM Nonhuman Primate Cord Blood Expansion and Transplantation Korashon L. Watts, R. Keith Humphries, Hans-Peter Kiem.

633 12:15 PM Generation of Insulin-Secreting Cells from Human Induced Pluripotent Stem Cells Tayaramma Thatava, Karen Squillace, Adam Armstrong, Yogish Kudva, Yasuhiro Ikeda.

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Abstract Directory Saturday, May 22, 2010 Oral Abstract Session 430 — continued Adenovirus and Other DNA Virus Vectors 3:00 PM - 5:00 PM Room: Maryland Suite 642 4:30 PM The Cytoplasmic Sensor, NOD2, Induces the Innate Immune Response and Transgene Silencing of Vector after Systemic Administration of Helper-Dependent Adenovirus Masataka Suzuki, Vincenzo Cerullo, Racel G. Cela, Terry K. Bertin, Christian Clarke, Lee H. Brendan. 643 4:45 PM CAdVax-Vectored Multivalent Filovirus Vaccine Protects NHP Against Challenge at 1000 Times the Lethal Dose of Ebola and Marburg Viruses John Y. Dong, Danher Wang, William D. Pratt.

Oral Abstract Session 431 Infectious Diseases and Vaccines

649 4:15 PM Induction of Immune Responses Against Multiple Species of Ebolavirus Using DNA and Retroviral-Based Virus-Like Particles Bearing the Envelope Glycoprotein of a Single Species Zaire Ebolavirus Wu Ou, Josie Delisle, Carolyn Wilson. 650 4:30 PM Low Levels of CXCR4 Are Sufficient for HIV-1 Entry and Infection Jinyun Yuan, Charles DeRossi, Michael C. Holmes, Philip D. Gregory, Bruce E. Torbett. 651 4:45 PM Overexpression of the SLAM Receptor Adaptor Molecule EAT-2 as a Novel Strategy for Enhancing Cellular Immunity to HIVantigens Yasser A. Aldhamen, Daniel M. Appledorn, Sergey S. Seregin, Joyce C. Liu, Nathan J. Schuldt, Sarah S. Godbehere, Andrea Amalfitano.

3:00 PM - 5:00 PM Room: Delaware Suite

Oral Abstract Session 432 Cancer – Immunotherapy II

644 3:00 PM Chim3 as a New Transgene for Anti-HIV-1/AIDS Gene Therapy: Antiviral Effect and Survival Advantage Features in a Preclinical In Vitro Model Simona Porcellini, Luca Alberici, Francesco Gubinelli, Gian-Paolo Rizzardi, Chiara Bovolenta.

3:00 PM - 5:00 PM Room: Marriott Ballroom Salons 2 & 3

645 3:15 PM In Vivo Selection of CD4+ T Cells Transduced with a GammaRetroviral Vector Expressing a Single-Chain Intrabody Directed Against HIV-1 Tat Stephen E. Braun, Ran Taube, Quan Zhu, Fay E. Wong, Akikazu Murakami, Erick Kamau, Markryan Dwyer, Angela Carville, R. Paul Johnson, Wayne A. Marasco.

Saturday, May 22nd

648 4:00 PM DNA Vaccination with a Consensus H1HA Influenza Immunogen Induces Immune Responses Capable of Protecting from a Lethal Novel A/H1N1-2009 and A/H1N1-1918 Challenge M. P. Morrow, J. Yan, A. Khan, M. Pope, A. Patel, G. Kobinger, D. B. Weiner, N. Y. Sardesai.

646 3:30 PM Safety and Efficacy of Autologous CD4+ T cells Transduced with a Lentiviral Vector Delivering Anti-HIV RNA Antisense env in HIV+ Subjects Failing One or More HAART Regimens Tessio Rebello, David Stein, Gary Blick, Richard Greenberg, Clifford Kinder, Andrew Zolopa, Gerard McGarrity. 647 3:45 PM Lentiviral Delivery of TRIM-Cyclophilin Restriction Factors Against HIV-1 Emma Chan, Torsten Schaller, Lu Zhao, Adrian Thrasher, Greg Towers, Waseem Qasim.

EXHIBITOR PROSPECTUS final program

652 3:00 PM A Powerful Retroviral Vector for TCR Gene Therapy: Silencing of Endogenous TCR Improved Expression and Anti-Tumor Reactivity of Transduced Tumor-Specific TCRs in Human Lymphocytes Sachiko Okamoto, Hiroaki Ikeda, Hiroshi Fujiwara, Masaki Yasukawa, Hiroshi Shiku, Junichi Mineno. 653 3:15 PM Organ Specific Oncolytic Virus Incorporated Antigen Libraries (OSOVIAL) for the Immunotherapy of Cancer Jose Pulido, Timothy Kottke, Jill Thompson, Peter Selby, Alan Melcher, Richard Vile. 654 3:30 PM Editing Human Lymphocyte Specificity for Safe and Effective Adoptive Immunotherapy of Leukemia Elena Provasi, Pietro Genovese, Zulma Magnani, Angelo Lombardo, Andreas Reik, Liu Pei-Qi, Oscar Muniz Pello, Jurgen Kuball, Attilio Bondanza, Giulia Casorati, Philip D. Gregory, Claudio Bordignon, Michael C. Holmes, Philip D. Greenberg, Luigi Naldini, Chiara Bonini. 655 3:45 PM Constitutive 4-1BBL Expression Dramatically Increases Proliferation, Survival and In Vivo Persistence of CD19-Targeted T Cells, Promoting Complete Tumor Elimination Maud Condomines, Jason Plotkin, John C. Markley, Gertrude Gunset, Michel Sadelain.

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Abstract Directory Saturday, May 22, 2010 Oral Abstract Session 432 — continued Cancer – Immunotherapy II 3:00 PM - 5:00 PM Room: Marriott Ballroom Salons 2 & 3

664 4:00 PM Treatment with the TLR9 Agonist MGN1703 in Patients with Metastatic Solid Tumors – EFFICACY Results of a Phase 1 Clinical Study Martin R. Weihrauch, Manuel Schmidt, Heike Richly, Bahar Bahar, Michael S. von Bergwelt-Baildon, Ulrich Hacker, Alexander Shimabukuro-Vornhagen, Marina Tschaika, Burghardt Wittig, Max E. Scheulen.

656 4:00 PM An A20-Silenced, Stroma-Targeted DC Vaccine Reverses the Immunosuppressive Tumor Microenviroment Resulting in Potent Antitumor Activity Xiao-Tong Song, Xiaoou Zhou, Lisa Rollins, Cliona M. Rooney, Stephen Gottschalk.

665 4:15 PM miR-7 Inhibits Schwannoma Tumor Growth by Directly Targeting p21-Activated Kinase 1 and Epidermal Growth Factor Receptor Okay Saydam, Ozlem Senol, Arda Mizrak, Thomas Wurdinger, Anat O. StemmerRachamimov, Anna M. Krichevsky, Xandra O. Breakefield.

657 4:15 PM A CD46-Targeting Recombinant Protein that Increases the Therapeutic Efficacy of Monoclonal Antibodies for Cancer Hongjie Wang, Ying Liu, Zong Li, Akseli Hemminki, Andre Lieber.

666 4:30 PM Nanoparticles are Effective Vehicles for Systemic Delivery of 2’OMePS Antisense Oligonucleotides in Exon Skipping-Mediated Dystrophin Restoration Alessandra Ferlini, Marina Fabris, Elena Bassi, Sofia Falzarano, Daniela Perrone, Francesca Gualandi, Patrizia Sabatelli, Luciano Merlini, Paola Rimessi.

658 4:30 PM Treatment of Cancer Patients with Integrin-Targeted Oncolytic Adenoviruses Ad5-D24-RGD and Ad5-RGD-D24-GMCSF Sari Pesonen, Vincenzo Cerullo, Sophie Escutenaire, Mari Raki, Lotta Kangasniemi, Eerika Karli, Elina Haavisto, Minna Oksanen, Timo Joensuu, Anna Kanerva, Akseli Hemminki. 659 4:45 PM Effective Dendritic Cell Immuno-Gene Therapy of Established ErbB2Expressing Tumors Using Pentostatin-Based Host Immune Depletion and Subsequent Host Th1/Tc1 Cell Transfer Miriam Mossoba, Jason Foley, Anu Gangopadhyay, Kris Ylaya, Matthew Winterton, Justin Taylor, Paul Massey, Stephen M. Hewitt, Jeffrey A. Medin, Daniel H. Fowler.

Oral Abstract Session 433 Oligonucleotide & RNAi Therapeutics II 3:00 PM - 5:00 PM Room: Thurgood Marshall W 660 3:00 PM Alleviation of Potentially Adverse shRNA Off-Targeting Via AAV Vector-Encoded Passenger Strand Decoys Stefan Mockenhaupt, Nina Schürmann, Dirk Grimm.

662 3:30 PM Delivery of Chemo-Sensitizing siRNAs to HER2-Positive Breast Cancer Cells Using RNA Aptamers Kristina W. Thiel, William H. Thiel, Xiuying Liu, Justin P. Dassie, Katie R. Stockdale, William M. Rockey, James O. McNamara, II, Paloma H. Giangrande. 663 3:45 PM Methods for Isolating Cell-Internalizing RNA Aptamers for Targeted siRNA Delivery William H. Thiel, Kristina W. Thiel, Justin P. Dassie, Liu Xiuying, Katie R. Stockdale, William M. Rockey, James O. McNamara, II, Paloma H. Giangrande.

Oral Abstract Session 434 Cell Processing and Vector Production 3:00 PM - 5:00 PM Room: Marriott Ballroom Salon 1 668 3:00 PM GMP Production of Self-Complementary Serotype 8 AAV Vector for Treatment of Hemophilia B James A. Allay, John S. Coleman, Arthur W. Nienhuis, Andrew Davidoff, Amit C. Nathwani, Susan Sleep, John T. Gray. 669 3:15 PM Production of Lentiviral Vectors in Suspension Cells Using Recombinant Baculoviruses Hanna P. Lesch, Anna E. Laitinen, Lauri M. Laitinen, Jere T. Pikkarainen, Haritha Samaranayake, Seppo Ylä-Herttuala, Kari J. Airenne. 670 3:30 PM Increased Therapeutic Effect of Cell Transplantation for Wound Healing in Diabetic Mice by Prolonging the Survival of Transplanted Cells Using Adhesamine, a Synthetic Adhesion Molecule Tetsuya Suehara, Makiya Nishikawa, Yuki Takahashi, Sayumi Yamazoe, Motonari Uesugi, Yoshinobu Takakura. 671 3:45 PM Characterization of Positioning Effect of pol III-shRNA Transcription Unit in scAAV Vector Genome on the Packaging Efficiency and Functionality of shRNA Silencing Jun Xie, Ran He, Qin Su, Guangping Gao. J. Telen, Bruce A. Sullenger.

Saturday, May 22nd

661 3:15 PM Multiplexing of snoRNAs for HIV-1 Inhibition Jane Zhang, John J. Rossi.

667 4:45 PM Therapy for Vaso-Occlusion in Sickle Cell Disease Includes RNA Aptamer Angela D. Burnette, Jun-ichi Nishimura, Milena Batchvarova, Shahid M. Nimjee, Rahima Zenadi, Marilyn J. Telen, Bruce A. Sullenger.

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Abstract Directory Saturday, May 22, 2010 Oral Abstract Session 434  — continued Cell Processing and Vector Production 3:00 PM - 5:00 PM Room: Marriott Ballroom Salon 1 672 4:00 PM Rapid Production of High Titer Virus Using a Concentrated Retrovirus Expansion Device Ceidy Sanchez, Gianpietro Dotti, John Wilson, Cliona M. Rooney, Malcolm K. Brenner, Juan F. Vera. 673 4:15 PM A Novel Approach to Developing Recombinant AAV Producing Cell Lines Jingmin Zhou, Katherine A. High, Guang Qu. 674 4:30 PM An Efficient and Complete Platform for the Production and Use of Lentiviral Vectors Thomas Quinn, Chris Mello, Lily Lee, Mei Fong, Dwayne Bisgrove, Michael Haugwitz, Hiroaki Sagawa. 675 4:45 PM Development, Scale-Up and Technology Transfer for Rapid, Automated, cGMP Compliant, Large-Volume Suspension Cell Based Process for Lentiviral Vector Production James Brady, Linhong Li, Scott R. Witting, Cornell Allen, Rama Shivakumar, Aparna Jasti, Kenneth G. Cornetta, Madhusudan V. Peshwa.

Oral Abstract Session 435 Gene Therapy for Inborn Errors of Metabolism

Saturday, May 22nd

3:00 PM - 5:00 PM Room: Wilson ABC

678 3:30 PM Update of Stem Cell Gene Therapy Clinical Trial for ADA-Deficiency in Japan Makoto Otsu, Masafumi Yamada, Yoshihiro Maeyama, Miyuki Kida, Satoru Nakajima, Nariaki Toita, Norikazu Hatano, Ryouji Kobayashi, Osamu Tatsuzawa, Junichi Mineno, Masafumi Onodera, Pawan Bali, Michael S. Hershfield, Fabio Candotti, Nobuaki Kawamura, Yukio Sakiyama, Tadashi Ariga. 679 3:45 PM Rescue and Long-Term Outcome of a Murine Model of Methylmalonic Acidemia Following Liver-Directed, AAV-Mediated Gene Therapy Randy J. Chandler, Nuria Carillo-Carrasco, Suma Chandrasekaran, Charles P. Venditti. 680 4:00 PM AAV Vector Mediated Enzyme Replacement Therapy of Hypophosphatasia (HPP) Model Mice Tae Matsumoto, Seiko Yamamoto, Hideo Orimo, Koichi Miyake, Noriko Miyake, Sonoko Narisawa, José Luis Millán, Yoshitaka Fukunaga, Takashi Shimada. 681 4:15 PM rDNA-AAV Vectors for the Treatment of Phenylketonuria Nick Morcinek, Laura M. Roy, Zhongya Wang, Markus Grompe, Cary Harding. 682 4:30 PM Safety and Efficiency of Site-Specific Integration AAV-rDNA Vectors Zhongya Wang, Mark Kay, Markus Grompe. 683 4:45 PM Functional Improvement of LGMD Hamsters by AAV-hMG53Mediated Sarcolemma Repair Bo He, Chunping Qiao, Bin Xiao, Juan Li, Zhenhua Yuan, Christina Mayer, Jianjie Ma, Xiao Xiao.

Oral Abstract Session 436 Late Breaking Science 3:00 PM - 5:00 PM Room: Virginia Suite

676 3:00 PM Early Gestational Gene Transfer with Lentiviral Vector Driven by a Liver Specific Promoter Targets the Fetal Liver and Improves Phenotype in a Murine Model of Wilson’s Disease Jessica L. Roybal, Masayuki Endo, Antoneta Radu, Carlyn Todorow, Philip W. Zoltick, Alan W. Flake.

914 3:00 PM Postexposure Protection of Nonhuman Primates against a Lethal Ebola Virus Challenge by RNA Interference Marjorie Robbins, Amy CH Lee, Joan B Geisbert, Anna N Honko, Vandana Sood, Joshua C Johnson, Susan de Jong, Iran Tavakoli, Adam Judge, Thomas W Geisbert, Lisa E Hensley, Ian MacLachlan.

677 3:15 PM A Combination of Pharmacological and Liver-Directed Gene Therapy Results in Long-Term Phenotypic Correction of a Mouse Model of Argininosuccinic Aciduria Philippe M. Campeau, Oleg A. Shchelochkov, Sandesh C. Sreenath Nagamani, Nicola Brunetti-Pierri, Donna Palmer, Muralidhar H. Premkumar, Harsha K. Garg, Anilkumar K. Reddy, Yaoping Tang, Nathan S. Bryan, Ayelet Erez, Brendan H. Lee.

915 3:15 PM A Clinical Study to Evaluate the Safety and Efficacy of pGM169/ GL67A Administered to the Nose and Lungs of Individuals with Cystic Fibrosis Stephen C Hyde, Jane C Davies, Uta Griesenbach, Tracy Higgins, Eric WFW Alton, A Christopher Boyd, J Alastair Innes, David Porteous, Deborah R Gill.

EXHIBITOR PROSPECTUS final program

916 3:30 PM Long-Term Safety and Transgene Expression in Monkeys Administered Lentiviral and AAV Vectors In Utero AF Tarantal, CCI Lee, C Mall, BJ Byrne, DB Kohn.

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Abstract Directory Saturday, May 22, 2010 Oral Abstract Session 436 — continued Late Breaking Science 3:00 PM - 5:00 PM Room: Virginia Suite 917 3:45 PM rAAV- Mediated Therapeutic Silencing of miR-122 Leads to Rapid and Significant Reduction of LDL in DLDR-/-/Apobec1A-/- Mice Jun Xie, Jennifer Broderick, Qing Xie, Hongwei Zhang, Ran He, Chengjian Li, Qin Su, Li Zhong, Terence R. Flotte, Phillip D. Zamore, Guangping Gao. 918 4:00 PM Hyperactive Transposons for Genetic Modification of Induced Pluripotent and Adult Stem Cells: A Novel Non-Viral Paradigm for Coaxed Differentiation Eyayu Belay, Janka Mátrai, Abel Acosta-Sanchez, Ling Ma, Mattia Quattrocelli, Lajos Mátés, Pau Sancho-Bru, Martine Geraerts, Bing Yan, Joris Vermeesch, Ermira SamaraKuko, Zoltán Ivics, Catherine Verfaillie, Maurillio Sampaolesi, Zsuzsanna Izsvák, Thierry VandenDriessche, Marinee K.L. Chuah. 919 4:15 PM Promoting the Migration and Integration of Schwann Cells after Transplantation into Lesioned Spinal Cord by Lentiviral Mediated Expression of Polysialic Acid Juan Luo, Xuenong Bo, Dongsheng Wu, John Yeh, Peter M Richardson, Yi Zhang. 920 4:30 PM Rescue of the Spinal Muscular Atrophy Phenotype in a Mouse Model by Early Postnatal Delivery of SMN Kevin D Foust, Xueyong Wang, Vicki L McGovern, Lyndsey Braun, Adam K Bevan, Amanda M Haidet, Thanh T Le, Pablo R Morales, Mark M Rich, Arthur H M Burghes, Brian K Kaspar. 921 4:45 PM Aptamer-Mediated Delivery of siRNAs and miRNA-Based siRNA Triggers to the Nucleus of Multiple Cancer Cell Types Jonathan Kotula, Bruce Sullenger.

Poster Session III

RNA Virus Vectors II 684 Insights on the Mechanisms of Measles Virus Entry into Cells Matthew K. Ennis, Stephen J. Russell, David Dingli.

686 In Vivo Immunization with Integration Deficient Lentiviral Vectors Biliang Hu, Pin Wang. 687 “Same Day” Ex Vivo Regional Gene Therapy: A Novel Approach To Enhance Bone Repair Mandeep S. Virk, O. Sugiyama, Sang H. Park, Jay R. Lieberman. 688 The R Peptide Sequence of XMRV Envelope Glycoprotein Determined the Pseudotyping Specificity with HIV-1 and MLV Vectors Toshie Sakuma, Suk See De Ravin, Harry L. Malech, Yasuhiro Ikeda. 689 Virus-Receptor Mediated Transduction of Dendritic Cells among Ross River Virus and Semliki Forest Virus Pseudotyped Lentivectors Steven Froelich, April Tai, Katie Stroup, Adnan Zubair, Pin Wang. 690 Effect of TLX1 Homeobox Gene Expression on Insertional Immortalization by an Insulated Self-Inactivating Retroviral Vector Lynnsey A. Zweier-Renn, Teresa S. Hawley, Ali Ramezani, Irene Riz, Robert G. Hawley. 691 Transcriptional Pulsing of Retroviral Transgenes in Mouse Embryonic Stem Cells Mandy Y. M. Lo, Akitsu Hotta, Sylvie Rival-Gervier, James Ellis. 692 The Cellular Fibronectin Matrix Is a Key Factor for the Attachment of VSV-G Pseudotyped Vector Particles and Its Cell Type Specific Complexity Provides a Mechanism for Delayed Cell Entry Josha A. Woodward, Amy M. Skinner, Peter Kurre. 693 Targeting of Lentiviral Vectors and Positioning of Transduced Cells by Magnetic Nanoparticles and Magnetic Field Katrin Zimmermann, Christina Trueck, Olga Mykhaylyk, Ulrich M. Becher, Bernhard Gleich, Daniela Wenzel, Bernd Fleischmann, Christian Plank, Alexander Pfeifer. 694 Pseudotyped Hepatitis C Virus Particle Could Be a Liver Target BioMissile Xiaojing Lin, Yonghui Zhang, Yue Wang. 695 Regulation of Cytokine Expression by Lentivirus-Encoded RNA Antagomirs Rasmus O. Bak, Line B. Petersen, Brian Moldt, Karin Stenderup, Cecilia Rosada, Lars Aagaard, Tomas N. Dam, Jacob G. Mikkelsen. 696 Effects of Replicators on Retroviral Variegation and Extinction Meihong Liu, Maribeth V. Eiden.

Saturday, May 22nd

6:00 PM - 8:00 PM Room: Exhibit Hall C

685 The Principal Function of the Lentiviral Central DNA Flap Is Defense Against APOBEC3 Protein Restriction Chunling Hu, Dyana T. Saenz, Hind Fadel, Mary E. Peretz, William Walker, Eric M. Poeschla.

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Abstract Directory Saturday, May 22, 2010 Poster Session III — continued 6:00 PM - 8:00 PM Room: Exhibit Hall C

AAV Vectors III 697 Late Gestation Intrauterine Gene Transfer of AAV9 in Non-Human Primates Preferentially Transduces the Central Nervous System, Liver, Skeletal and Cardiac Musculature C. N. Mattar, N. Johana, A. Fisk, A. Biswas, J. Lu, M. H. Tan, L. G. Tan, A. Rahim, S. Waddington, M. Choolani, J. Chan. 698 Integrin-Linked Endocytosis Directs AAV2 and AAV1 to a Highly Efficient Transduction Pathway Paul M. Kaminsky, Nicholas W. Keiser, John F. Engelhardt. 699 Computationally-Guided Rational Design of Tropism-Modified AAV Vectors Adam S. Davis, William C. Ray, Thais Federici, Nicholas M. Boulis, Jeffrey S. Bartlett. 700 Identification of Human Hepatocyte Growth Factor Receptor as a Cellular Receptor/Co-Receptor for AAV3 Chen Ling, Yuan Lu, Jasmine K. Kalsi, Giridhara R. Jayandharan, Binbin Cheng, Samantha W. Y. Gee, Katherine E. McGoogan, Li Zhong, Arun Srivastava. 701 Rapid Transgene Expression and Long-Term Phenotypic Correction of a Lethal Mouse Model of Methylmalonic Acidemia Using rAAV9Mediated Gene Delivery Julien S. Senac, Randy J. Chandler, Charles P. Venditti.

Saturday, May 22nd

702 Widespread Transduction of Brain Parenchyma after Intraventricular Injection of AAV9 Annagiusi Gargiulo, Enrico M. Surace. 703 AAV1.9-3 Exhibiting Significantly Impaired Infectivity and Substantially Delayed Blood Clearance, Is a Novel Detargeted Platform of AAV Random Peptide Display Libraries Kei Adachi, Nicole M. Kotchey, Kimimasa Tobita, Hiroyuki Nakai. 704 Development of the D[+]-Sequence-Deleted ssAAV2 Vectors: Enhanced Transgene Expression In Vitro and In Vivo Chen Ling, Giridhara R. Jayandharan, Baozheng Li, Yuan Lu, Wenqin Ma, Arun Srivastava.

705 Influence of Murine Parvovirus on Adeno-Associated Virus VectorRelated Expression in Murine Models Sara A. Collins, Gerald C. O’Sullivan, Patrick T. Harrison, Martina F. Scallan, Mark Tangney. 706 Involvement of the Glucocorticoid Receptor Signaling in AAV2 VectorMediated Transgene Expression Yuan Lu, Chen Ling, Giridhara R. Jayandharan, Angela E. Rivers, Arun Srivastava. 707 AAV9-Mediated Catalase Expression in Mitochondria Improves Exercise Performance in mdx Mice Dejia Li, Dongsheng Duan. 708 Development of a Liver Specific Tet-On Inducible System for AAV Vectors Lucia Vanrell, Laura Blanco, Lilianne Tenenbaum, Jesus Prieto, Gloria GonzálezAseguinolaza. 709 Intracortical Delivery of an Adeno-Associated Viral Vector Expressing the Glutamate Transporter (GLT1) Decreases Stroke-Induced Extracellular Glutamate Overflow and Cerebral Infarction after Transient Middle Cerebral Artery Occlusion in Rats Brandon K. Harvey, Mikko T. Airavaara, Jason Hinzman, Matthew J. Chiocco, Hui Shen, Douglas B. Howard, Greg A. Gerhardt, Barry J. Hoffer, Yun Wang. 710 The Potential Role of Chromatin Structure in Adeno-Associated Virus Type 2 Locus-Specific Integration at AAVS1 Victor J. McAlister, Roland A. Owens. 711 Augmentation of Traditional Ad/AAV Helper Plasmids with HPV E1 Increases rAAV DNA Replication and Virus Production in 293 Cells Sarmistha Bandyopadhyay, Maohua Cao, Yong Liu, Paul L. Hermonat.

Adenovirus and Other DNA Virus Vectors III 712 Hexon-Modifications of hAd5 Based Helper-Virus for High-Capacity Adenoviral Vector Production and Limitations Imposed by HVRExchanges Utilizing Serotype Switch Martin A. Hausl, Zsolt Ruzsics, Anja Ehrhardt. 713 Use of Cell-Penetrating Peptides To Induce a Bystander Effect Ying Shen, William Yu, John G. Hay, Harald Sauthoff. 714 Highly Sensitive Detection of Prostate Cancer Cells In Vivo by a Dual Adenoviral TSTA Trans-Amplification System Makoto Sato, Frédéric Pouliot, Steve Huyn, Lily Wu. 715 Coagulation Factor X-Mediated Adenoviral Transduction Is Dependent on Heparan Sulfate Proteoglycan Sulfation Angela C. Bradshaw, Alan L. Parker, Stuart A. Nicklin, John H. McVey, Andrew H. Baker.

EXHIBITOR PROSPECTUS final program

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Abstract Directory Saturday, May 22, 2010 Poster Session III — continued 6:00 PM - 8:00 PM Room: Exhibit Hall C

Adenovirus and Other DNA Virus Vectors III 716 Towards the Generation of a Baculovirus Display Library for Cell Targeting Marcel Westenberg, Helen M. Soedling, Linda J. Nicholson, Derek A. Mann, Colin T. Dolphin. 717 Broad Spectrum Activity of the HSV-2 Vector DRR Enhances Neuroprotection and Includes Crosstalk between Neurons and Glial Cells Jennifer M. Laing, Laure Aurelian. 718 Enhanced Transduction and Replication of Fiber-Modified Adenovirus in Murine Primary T Cells Sadhak Sengupta, Ilya V. Ulasov, Atique U. Ahmed, Maciej S. Lesniak. 719 Cloning of the Human Herpesvirus-6A (HHV-6A) Genome into the Bacterial Artificial Chromosomes (BACs) towards the Creation of Helper-Less Amplicon Vectors for Potential Use in Lymphocyte Gene Therapy and Vaccination Niza Frenkel, Ronen Borenstein, Haim Zeigerman, Adi Museri, Pnina Schlesinger. 720 Adenovirus-Mediated Decorin Expression Induces Cell Death through Activation of p53 and Mitochondrial Apoptosis A-Rum Yoon, Jung-Sun Lee, Chae-Ok Yun. 721 PEG-Modified Adenovirus Conjugated with Herceptin Improves Therapeutic Value by Active Targeting through Her2/neu with an Enhanced Safety Profile Joung-Woo Choi.

723 Ad5-Ad48 Hexon Hypervariable Region Substitutions Affect Viral Biodistribution and Inflammatory Responses In Vivo Lynda Coughlan, Angela Bradshaw, Laura Denby, Stuart A. Nicklin, Jerome Custers, Nico van Rooijen, Dan H. Barouch, Andrew H. Baker.

725 The Knob Domain Replacement Strategy Is Broadly Applicable to a Wide Variety of Adenovirus Fiber Proteins Galina Mikheeva, Natalya Belousova, Victor Krasnykh.

DNA Vectorology & Gene Targeting 726 Transcriptional Repression of Transgenes Delivered by Sleeping Beauty, piggyBac and Tol2 DNA Transposon Vectors Nynne Sharma, Rasmus O. Bak, Nicklas H. Staunstrup, Gernot Wolf, Jacob G. Mikkelsen. 727 Duration of Expression from CpG-Free Plasmids Following Hydrodynamic Delivery to the Mouse Ian A. Pringle, Mary M. Connolly, Anna E. Lawton, Lee A. Davies, Stephen C. Hyde, Deborah R. Gill. 728 A Liver-Specific Promoter Enhances Expression of alpha-L-Iduronidase in Sleeping Beauty Transposon-Mediated Gene Therapy of Murine MPS I Elena L. Aronovich, Jason B. Bell, Brenda L. Koniar, Perry B. Hackett. 729 New Generation, S/MAR Based, Episomal Vectors with High Potential for Gene Transfer into Hematopoietic Progenitor Cells Eleana Stavrou, Vassileios Lazaris, Eirini Papapetrou, Aristeidis Giannakopoulos, Dimitra Kokkinou, Louiza Toutouna, Alexandros Spyridonidis, Nikolas Zoumbos, Aglaia Athanassiadou. 730 High-Yield, Non-Viral Production of Engineered Cells through Sleeping Beauty Transposon-Mediated Gene Integration Coupled with Selective Amplification Nataly A. Kacherovsky, Maja Zavaljevski, Carl A. Blau, Cecilia M. Giachelli, Suzie H. Pun. 731 A Combinatorial Approach for Targeted, Systemic Delivery Using Small Molecules and Reversible Masking to Bypass Non-Specific Uptake In Vivo Nancy S. Templeton, Qiaoyun Shin, Andrew T. Nguyen, Yu Angell, Defeng Deng, ChangRim Na, Kevin Burgess, David D. Roberts, F. C. Brunicardi, Phillip B. Maples, John Nemunaitis. 732 Using Helper Virus-Free Packaged HSV-1/EBV Hybrid Amplicons To Induce Directed Cell Motility toward SDF-1a Yasser M. El-Sherbini, Mark Stevenson, Richard Wade-Martins, Len W. Seymour. 733 A Novel System for the Evaluation of Genotoxicity for Gene Therapy Vectors Jun Zhan, Matthew M. Wielgosz, John Gray, Arthur W. Nienhuis.

Saturday, May 22nd

722 Study on Testing Method for Adeno-Associated Virus (AAV) of Adventitious Virus Yejin Choi, Nam Hee Kim, Keum S. Choi, Keerang Park, Younjoo Park.

724 Evaluation of a Porcine Adenovirus 3 Vector as a Post-Exposure Vaccine Candidate Ami Patel, Suresk K. Tikoo, Gary P. Kobinger.

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DNA Vectorology & Gene Targeting 734 A Liver-Directed Targeting Tether Vector System To Increase Uptake in Liver Cells Jason B. Bell, Erik Olson, R. Scott McIvor, Perry B. Hackett. 735 Cells Labeled with Fatty Acid-Oligonucleotide Conjugates as Potentially Suitable for Tissue Engineering Galina E. Pozmogova, Marina A. Zaitseva, Andrey N. Chuvilin, Grigory G. Borisenko. 736 An Enhanced Non-Viral Expression Vector (EEV) – For Cancer Treatment Patrick F. Forde, Mira Sadadcharam, Marcel De Kruijf, Slawomir P. Salwa, Michael G. Bourke, Gerald C. O’Sullivan, Declan M. Soden. 737 Development of Post-Translational Tumor-Targeted-Cytokine Vector for Systemic Treatment of Malignant Disease Jeffry J. Cutrera, Denada Dibra, Xuexing Xia, Shulin Li. 738 Improved Antibiotic-Free Plasmid Vector Design by Incorporation of Transient Expression Enhancers Jeremy M. Luke, Justin M. Vincent, Clague P. Hodgson, James A. Williams. 739 Genomic DNA Reduction for Therapeutic DNA Manufacture Ying Cai, Debra Bailey, Reto P. Bazzani, Henry Hebel, Stephen C. Hyde, Stephen Rodriguez, Deborah R. Gill.

Saturday, May 22nd

740 Preclinical Efficacy and Safety of Electroporation-Mediated Delivery of a Plasmid Vector Manufactured in the Absence of Antibiotic Selection Karen E. Dolter, Cedric Y. Szpirer, Philippe Gabant, Drew Hannaman, Claire F. Evans. 741 Hepatic Stellate Cell-Specific TGF-b1 Gene Silencing for Treating Liver Fibrosis Ningning Yang, Ram I. Mahato.

Chemical and Molecular Conjugates III 742 Nuclear Enclosure of Nanospheres in Artificial Nuclei and in Living Cells Nathalie Symens, Katrien Remaut, Jo Demeester, Stefaan C. De Smedt.

EXHIBITOR PROSPECTUS final program

743 Analysis of Intracellular Trafficking of MPC-Coated Multifunctional Envelope-Type Nano Device (MPC-MEND) in Isolated Hepatocyte Masami Ukawa, Hidetaka Akita, Tomoya Masuda, Yasuhiro Hayashi, Tomohiro Konno, Kazuhiko Ishihara, Hideyoshi Harashima. 744 Photo-Activated Release of DNA from Liposome Complexes Caroline M. LaManna, Jiazuo H. Feng, Hrvoje Lusic, Mark W. Grinstaff. 745 A Novel Cationic Lipophosphoramide with Di-Unsaturated Lipid Chains: Synthesis, Physico-Chemical Properties, and Transfection Activities Tony Le Gall, Damien Loiseau, Erwan Picquet, Nathalie Carmoy, Jean Jacques Yaouanc, Laure Burel Deschamps, Pascal Delépine, Philippe Giamarchi, Paul Alain Jaffrès, Pierre Lehn, Tristan Montier. 746 Modifying DNA Nanoparticles with Biotin for Versatile Ligand Substitution Wenchao Sun, Pamela B. Davis. 747 An Integrative Approach To Monitor Gene Delivery to Airway Epithelium by Nanocomplex Vectors and Radioimaging Agents Maria D. I. Manunta, Aristides Tagalakis, Christopher O’Callaghan, Stephen L. Hart. 748 Development of Small, Homogenous Non-Viral Gene Vector Using Mono-Cationic Detergents Ryosuke Suzuki, Yuma Yamada, Hideyoshi Harashima. 749 Development of DNA Complex-Releasing Systems Using Biodegradable Apatite Cement and Therapeutic Effect in Tumor Model Mice Tomoko Ito, Yoshiyuki Koyama, Makoto Otsuka. 750 Investigation of Cross-Linking Peptides as Gene Delivery Vectors Anton V. Kiselev, Anna A. Egorova, Arto O. Urtti, Vladislav S. Baranov. 751 Rapid and Highly Efficient Magnetofection of IL-10 to Extremely Susceptible Vascular Endothelial Cells Using Hybrid Iron Oxide-PEI Conjugate Yong Sook Kim, In-Kyu Park, Jin Sook Kwon, Myung Ho Jeong, Youngkeun Ahn. 752 Cocaine Conjugated to Disrupted Adenovirus – A Synthetic/ Viral Protein Hybrid Strategy To Elicit High Titers Against NonImmunogenic Addictive Small Molecules Bishnu P. De, Martin J. Hicks, Jonathan B. Rosenberg, Stefan Worgall, Stephen M. Kaminsky, Amira Y. Moreno, Kim D. Janda, Ronald G. Crystal. 753 Bisphosphonate-Mediated Gene-Eluting Metal Stent as Gene Vector Delivery Using Antibody for Localized Gene Delivery Yong Wang, Cunxian Song, Robert J. Levy.

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Abstract Directory Saturday, May 22, 2010 Poster Session III — continued 6:00 PM - 8:00 PM Room: Exhibit Hall C

Chemical and Molecular Conjugates III 754 Optimization of Sendai F/HN Viroplexes for Efficient Transfection of Plasmid DNA and siRNA to Suspension Cells Jung Seok Kim, Yeon Kyung Lee, Jin Ee Baek, Sang Il Park, Hwa Yeon Jeong, Hong Sung Kim, Keun Sik Kim, Yong Serk Park. 755 Comparative Evaluation of Disulfide-Linked and HydrophobicallyModified PEI for Plasmid Delivery In Vitro K. C. B. Remant, Breanne Landry, Hasan Uludag. 756 Linear Polyethylenimine Prepared by Partial Acid Hydrolysis of Poly (2-ethyl-2-oxazoline) for DNA and siRNA Delivery In Vitro Julio C. Fernandes, Xingping Qiu, Francoise M. Winnik, Mohamed Benderdour, Xiaoling Zhang, Kerong Dai, Qin Shi. 757 Impact of Counterion and Hydrophobic Anchor on Physicochemical Characteristics and Transfection Efficiency for a Series of Pyridinium Cationic Lipid-Based Lipoplexes Sushma Savarala, Stephanie L. Wunder, Marc A. Ilies.

Genetic and Metabolic Diseases Gene & Cell Therapy III 758 AAV-Mediated Liver Gene Transfer Results in Long-Term Expression of Therapeutic Levels of ARSB in MPS VI Cats Gabriella Cotugno, Patrizia Annunziata, Anita Capalbo, Thomas O’Malley, Patricia O’Donnell, Fabio Russo, Elvira De Leonibus, Mark Haskins, Alberto Auricchio.

760 Gene Transfer To Induce Cellular Reprogramming for the Treatment of Diabetes Neil Phillips, Markus Grompe, Mark A. Kay. 761 Salivary Gland hPTH Gene Transfer: Interesting Possibilities Janik Adriaansen, Paola Perez, Changyu Zheng, Michael T. Collins, Bruce J. Baum.

763 Tolerance Induction to Coagulation Factor IX Using Gene Modified Primary B Cells Babak Moghimi, Irene Zolotukhin, Ou Cao. 764 (Moved to Poster Session II after Abstract 422) Microporous Polymer Scaffolds for Islet Transplantation Romie F. Gibly, Lonnie D. Shea. 765 AAV2- and AAV9 -Mediated Gene Transfer of LDL Receptor to Hypercholesterolemic WHHL Rabbits Elisa Vähäkangas, Anniina Laurema, Atsushi Miyanohara, Seppo Ylä-Herttuala. 766 Hairpin Ribozymes Encoded in Adenoviral Vectors Reduce Drinking in Alcohol Dependent Rats Amalia Sapag, Thergiory Irrazábal, María Elena Quintanilla, Lutske Tampier. 767 Sustained Therapeutic Levels of FVIII Activity from LentivirallyEngineered Blood Outgrowth Endothelial Cells Requires Cell Stability and Augmented Angiogenesis Hideto Matsui, Margareth Ozelo, Andrea Labelle, Carol Hegadorn, Patrick Thompson, Erin Burnnet, Christine Hough, David Lillicrap. 768 Effects of Neonatal Intracranial AAV and Systemic Lentiviral Gene Therapy in Sanfilippo Syndrome Type B Mice Coy D. Heldermon, Elizabeth Qin, Kevin K. Ohlemiller, Erik Herzog, Carole Vogler, David F. Wozniak, Mark S. Sands. 769 Laminin-111: A Highly Potent Therapeutic Agent To Treat Duchenne Muscular Dystrophy Sebastien Goudenege, Yann Lamarre, Nicolas Dumont, Joel Rousseau, Skuk Daniel, Jacques P. Tremblay. 770 Layered Scaffolds for Islet Transplantation and Gene Delivery John G. Graham, Lonnie D. Shea. 771 Lentiviral Vector Correction of Fabry Disease Encompassing a Cell Fate Control Safety Element Matthew D. Scaife, Sean P. Devine, Anton Neschadim, Natalia A. Pacienza, Jeffrey A. Medin. 772 Wolman Disease (LIPA p.G87V) Genotype Frequency in Patients of Iranian-Jewish Descent Yadira Valles-Ayoub, Daniel No, Saghi Esfandiarifard, Zeshan Khokher, Chai Saechao, Marvin Pietruszka, Daniel Darvish.

Saturday, May 22nd

759 Beta-Glucuronidase Expressed from AAV Is Poorly Modified with Mannose-6-Phosphate Residues and Fails to Completely Correct Mucopolysaccharidosis VII in Dogs Guangping Gao, Peter L. Bell, Mark E. Haskins, Lisa A. Kisling, Richard Steet, Luk H. Vandenberghe, Hongwei Yu, Julio C. Sanmiguel, James M. Wilson.

762 Efficient Gene Transfer to the Pancreas Using ssAAV for Prevention of Type 1 Diabetes Eduard Ayuso, Veronica Jimenez, Judith Agudo, Ariana Salavert, Sergio Muñoz, Alba Casellas, Merce Obach, Maria Molas, Marta Moya, Xavier Leon, Jesus Ruberte, Fatima Bosch.

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Abstract Directory Saturday, May 22, 2010 Poster Session III — continued 6:00 PM - 8:00 PM Room: Exhibit Hall C

Neurodegenerative Disorders, Spinal Cord and Trauma 773 Delayed Inhibition of Soluble Tumor Necrosis Factor Signaling Blocks the Progressive Phase of Dopaminergic Neuron Death in a Rat Model of Parkinson’s Disease Ashley S. Harms, Kelly M. Ruhn, Steve Varghese, Isaac Trevino, Malu G. Tansey. 774 Identification and Analysis of a Novel g-Secretase Inhibitory Gene Hiroki Hayashi, Hironori Nakagami, Nobutaka Koibuchi, Munehisa Shimamura, Yasufumi Kaneda.

782 A Comparison of AAV Serotypes and Routes of Administration for Delivery to the Spinal Cord Brooke R. Snyder, Thais Federici, Eric Quach, Steven Gray, Cary H. Leung, Nicholas M. Boulis. 783 Engineered Zinc Finger Protein Improves Microvascular Recovery, Neuronal Sparing and Functional Recovery in Modeled Traumatic Brain Injury in Rats Eugene Park, Ishita Siddiq, Elaine Liu, S. Kaye Spratt, Richard Surosky, Gary Lee, Dale Ando, Marty Giedlin, Andrew J. Baker.

Musculo-skeletal Gene & Cell Therapy III

775 Effects of Alpha 7 Nicotinic Receptor Gene Delivery on Processes Underlying Cell Viability and Cell Differentiation Yan Ren, Lin Zhang, Edwin M. Meyer, Jeffrey A. Hughes, Sihong Song.

784 Blocking the Myostatin Signal Improves the Success of Human Myoblast Transplantation in Dystrophic Mice Raouia Fakhfakh, Annick Michaud, Jacques P. Tremblay.

776 Delivery of hGDNF to Brain Using DNA Nanoparticles: Neuroprotection of the Nigrostriatal Pathway David M. Yurek, Anita M. Fletcher, Linas Padegimas, Tomasz H. Kowalczyk, Mark J. Cooper.

785 High Force Myosin Correlated with the Early Degeneration of Jaw Closing Muscle in the GRMD Model of DMD Andrew F. Mead, Alock Malik, Mihail Petrov, Martin Childers, Marilyn Mitchell, Janet Bogan, Joe Kornegay, Brittani Nickens, Hansell Stedman.

777 ProSavin, a Gene Therapy for Parkinson’s Disease Bechir Jarraya, Helene Lepetit, Scott Ralph, James Miskin, Jean-Marc Gurruchaga, Maria Vinti, Gilles Fenelon, Pierre Brugiere, K. Abay, I. Gabriel, Sabrina Boulet, Caroline Jan, Susan Kingsman, P. Cesaro, Philippe Hantraye, Philippe Remy, Kyriacos Mitrophanous, Stephane Palfi.

786 Comparison Studies of rAAV6 Transduction Efficiency in Adult and Aged Muscles Patryk Konieczny, James M. Allen, Eric Finn, Jeffrey S. Chamberlain.

778 Primate Hippocampal Gene Delivery Via Frameless Stereotaxis Cary H. Leung, Michele A. Kliem, Brooke R. Snyder, Thais Federici, Thomas J. McKown, Scott W. J. McPhee, Nicholas M. Boulis.

Saturday, May 22nd

781 HSV Gene Transfer of a Poreless TRPV1 Receptor Reduces PainRelated Behavior in Rodents James R. Goss, William F. Goins, Rahul Srinivasan, Joseph C. Glorioso.

779 Delivery Co-Interventions in Gene Transfer and Cell Transplantation: A Meta-Analysis of Surgical Risk for Trials Involving Inoculation to Deep Brain Structures Jonathan Kimmelman, Katherine Duckworth, Tim Ramsay, Marina Emborg, Tiffini Voss. 780 Modification of the Inhibitory Environment of Spinal Cord Injury by Lentiviral Vector Containing NT-3 and shRNA for NG2 Eleanor M. Donnelly, Nicolas N. Madigan, Gemma E. Rooney, Anthony J. Windebank, Timothy O’Brien, Siobhan S. McMahon.

EXHIBITOR PROSPECTUS final program

787 Gene Transfer of Signals Critical for Allograft Healing Using Immobilized Freeze Dried Adeno-Associated Viral Vectors Mette Koefoed, Alexandra G. Pedersen, Michael Ulrich-Vinther, Thomas G. Jensen, Kjeld Soballe. 788 Rescue from Respiratory Dysfunction by Transduction of Full-Length Dystrophin to Diaphragm Via the Peritoneal Cavity in Muscular Dystrophy Mouse Ishizaki Masatoshi, Yasushi Maeda, En Kimura, Makoto Uchino. 789 Restoration of Wild-Type DMD mRNA in an Immortalized Human Fibroblast Line from an Exon 8-9 Duplicated DMD Patient Ling Zhao, Gillian Butler-Browne, Vincent Mouly, Soraya Chaouch, Kamel Mamchaoui, Steve D. Wilton, Kevin M. Flanigan. 790 Expression of Micro-Dystrophin and Full-Length Dystrophin in Mouse and Dog Muscles Christophe Pichavant, Pierre Chapdelaine, Joe N. Kornegay, João C. S. Bizario, Xiao Xiao, Jacques P. Tremblay.

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Abstract Directory Saturday, May 22, 2010 Poster Session III — continued 6:00 PM - 8:00 PM Room: Exhibit Hall C

Musculo-skeletal Gene & Cell Therapy III 791 A Comparison of Adeno-Associated Virus Mediated Gene Transfer into Healthy and Dystrophin-Deficient Skeletal Muscles Gilles Moulay, Carole Masurier, Pascal Bigey, Daniel Scherman, Antoine Kichler. 792 In Vivo Reprogramming into the Myogenic Lineage and Improving Engraftment in the mdx Mouse Model of DMD Lindsey A. Muir, Jeffrey S. Chamberlain. 793 Gene Transfer-Driven Inhibition of Cardiac Ankyrin Repeat Protein as a Potential Therapy for the Correction of Muscular Dystrophies Nathalie F. Danièle, Laurence Suel-Petat, Isabelle Richard. 794 Background Strain and Natural Selection Improves Survival of HIBM Mouse Model Yadira Valles-Ayoub, Zeshan Khokher, Arman Haghighatgoo, Daniel No, Saghi Esfandiarifard, Chai Saechao, Jennifer Kay, Christopher Creencia, Sheila Darvish, Daniel Darvish. 795 Combining Stem Cells and Exon Skipping Approach To Treat Muscular Dystrophies Mirella Meregalli, Daniele Parolini, Andrea Farini, Marzia Belicchi, Giuseppe D’Antona, Simona Maciotta, Paola Razini, Alberto Lerario, Serena Bonfiglio, Roberto Bottinelli, Luis Garcia, Nereo Bresolin, Yvan Torrente.

798 Assessing Preclinical Safety of a CCR5-Specific Zinc Finger Nuclease in Human CD4+T Cells Using the NSG Immunodeficient Mouse Model S. Kaye Spratt, Marty Giedlin, Richard Carroll, Shuguang Jiang, Dawn Maier, Gwendolyn Binder-Scholl, Gary Lee, Lynn Truong, Bruce Levine, Michael Holmes, Philip Gregory, Carl June, Dale Ando. 799 Multivirus-Specific T Cell Immunotherapy To Prevent or Treat Infections of Allogeneic Stem Cell Transplant Recipients Ann M. Leen, Ulrike Gerdemann, Carlos A. Ramos, Tamara Tripic, Malcolm K. Brenner, Cliona M. Rooney, Juan F. Vera, Helen E. Heslop. 800 Inhibition of HIV-1 Replication in VRX496-Modified CD4 T Cells: Importance of Cytoplasmic dsRNA Components Randall Merling, Brit J. Hart, Ziping C. Chen, Laurent M. Humeau, Nikolay Korokhov. 801 Engineering Resistance to CXCR4-Tropic HIV: A Cutting Edge Approach Craig B. Wilen, Michael C. Holmes, Jianbin Wang, Philip D. Gregory, Carl H. June, Robert W. Doms. 802 Development of an Unbiased Method for Characterizing Viral Populations Using Next Generation Sequencing Technology Stephanie M. Willerth, Helder L. Pedro, Laurent M. Humeau, Adam P. Arkin, David V. Schaffer. 803 Long Term Persistence of VRX496-Modified CD4+ T Lymphocytes Following Lexgenleucel-T Infusions for the Treatment of HIV/AIDS: Results from a Phase 2 Clinical Trial Laurent M. Humeau, Kris Andre, Karen Bengtson, Shuyuan Zhang, Gerard McGarrity, Tessio Rebello. 804 Generation of Restriction Factor-Transgenic Cats Using Lentiviral Transgenesis Pimprapar Wongsrikeao, Dyana T. Saenz, Thomas A. Rinkoski, Takeshige Otoi, Eric M. Poeschla.

Infectious Diseases and Vaccines: Non-Vaccine Related Gene Transfer Research

805 Combinatorial Latency Reactivation for HIV-1 Subtypes and Variants John C. Burnett, John J. Rossi, Kwang-il Kim, Adam P. Arkin, David V. Schaffer.

797 Delineation of RNA Antisense env Effects on HIV Quasispecies Recovered from Lexgenleucel-T Treated Subjects: Influences of Sequence Similarity of the RNA Antisense with the Targeted HIV env Transcript and VRX496-Modified Cell Persistence Alexander Pasternak, Nikolay Korokhov, Vladimir Lukashov, Laurent M. Humeau.

806 Highly Efficient Expression and Purification of Hepatitis C Virus Core Protein in E.coli Yixin Bian, Guozhen Qiu, Tingting Li, Zixuan Chen, Wenjing Wang, Chengyao Li. 807 Molecular Epidemiology of HCV Infection among Recovery and Chronic Blood Donors in Guangdong, China Tingting Li, Jinfeng Zeng, Zixuan Chen, Lifang Shuai, Anqi Wang, Chengyao Li.

Saturday, May 22nd

796 Gene and Cell Therapy Approaches Tto Treating the Dysferlinopathies Bradley A. Williams, Nilah Monnier, Douglas E. Albrecht, Nupur Garg, Laura E. Rufibach, Esther Hwang, Angela Salerno, Plavi Mittal.

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Abstract Directory Saturday, May 22, 2010 6:00 PM - 8:00 PM Room: Exhibit Hall C

816 Tumor Antigen Delivery to Secondary Lymphoid Organs by Interleukin-12 Gene Modified T Cells Adham S. Bear, Xiatong Song, Meghan E. Turnis, Stephen M. Gotschalk, Cliona M. Rooney, Aaron E. Foster.

Infectious Diseases and Vaccines: Non-Vaccine Related Gene Transfer Research

817 Pluronic Based Formulations Are Promising Anticancer DNA Vaccines Caroline Roques, Zagit Gaymalov, Jedd D. Wolchok, Alexander V. Kabanov.

Poster Session III — continued

808 Occult Hepatitis B Virus Infection in Shenzhen, China Xin Zheng, Yixin Bian, Ling Zhang, Wenjing Wang, Lifang Shuai, Anqi Wang, Jean-Pierre Allain, Daniel Candotti, Chengyao Li.

Cancer – Immunotherapy III 809 Time for TEA: T Cells Expanded on Artificial Antigen Presenting Cells? Hillary N. Gibbons, Carrie Yuen, Sergio Giralt, Carl H. June, Laurence J. N. Cooper. 810 Oxaliplatin Increases the Antitumor Effect of Vector-Delivered Interleukin-12 Expression in a Murine Model of Hepatic Colorectal Cancer Metastases Manuela Gonzalez-Aparicio, Itsaso Mauleon, Pilar Alzuguren, Sandra Hervas-Stubbs, Uxua Manchengo, Julien Crettaz, Gloria González-Aseguinolaza, Jose Medina-Echeverz, Pedro Berraondo, Jesus Prieto, Ruben Hernandez-Alcoceba. 811 Optimizing Dendritic Cell Vaccination by Combination with Oncolytic Adenovirus Coexpressing Interleukin-12 and GranulocyteMacrophage Colony Stimulating Factor Song-Nan Zhang, Jing-Hua Huang, Ji Young Yoo, Kyung-Ju Choi, Chae-Ok Yun. 812 Bacterial Vector Mediated Vaccination for Prostate Cancer Garrett Casey, Sarfraz Ahmad, Michelle Cronin, Simon Rajendran, Mark Tangney, Gerald C. O’Sullivan.

Saturday, May 22nd

813 Evaluation of Long-Term Transgene Expression in piggyBac-Modified Human T Lymphocytes Yozo Nakazawa, Sunandan Saha, Daniel Galvan, Cliona M. Rooney, Matthew H. Wilson. 814 Engineered Drug Resistant Immunocompetent Cell Therapy for Neuroblastoma Anindya Dasgupta, David McCarty, H. Trent Spencer. 815 Genetically Mediated Constitutive Expression of CD40L by T Cells Will Induce Enhanced Immunogenicity of B-Cell Malignancies Kevin J. Curran, Raymond Yeh, Beatrijs Seinstra, Yan Nikhamin, Yelena Usachenko, Renier J. Brentjens.

EXHIBITOR PROSPECTUS final program

818 Efficient Induction of Humoral and Cellular Immune Responses Against Her-2/neu by Combination of Naked DNA and Adenoviral Vectors Expressing Genetically Engineered Her-2/neu Jae-Gyun Jeong, Dong-Sik Kim, Sung-Won Lim, Kyu-Woong Shim, Hwang-Jae Lee, SungMin Oh, Jin-A Chae, Yeon-Sook Cha, Seong-Hyun Ho, Youngtae Hong, Woong Hahn, Sujeong Kim, Jong-Mook Kim, Seungshin Yu, Chang-Yuil Kang, Sunyoung Kim. 819 Autocrine, Not Paracrine, Interferon-Gamma Gene Delivery Enhances Ex Vivo Antigen-Specific Cytotoxic T Lymphocyte Stimulation and Killing Dazhi Zhang, Liu Yong, Chang Xuan You, Rongcheng Lou, Paul L. Hermonat. 820 Potent Transfer of Human CD40L to Chronic Myeloid Leukemia Stem Cells Via an AdCD40L Genetically Engineered Bystander Cell Line Lisa H. Christiansson, Lina Liljenfeldt, Bengt Simonsson, Ulla Olsson-Strömberg, Angelica S. I. Loskog. 822 Preclinical Efficacy Data of MGN1601, a Tumor Vaccine Comprising 4-Fold Gene-Modified and Irradiated Allogeneic Tumor Cells in Combination with a DNA-Based Immunomodulator for the Treatment of Metastatic Renal Carcinoma Baerbel Volz, Manuel Schmidt, Kerstin Kapp, Matthias Schroff, Marina Tschaika, Burghardt Wittig. 823 Electrically Mediated Delivery of Plasmid DNA-Encoding Cytokines for the Generation of an Enhanced Immune Response Against Melanoma Bernadette Marerro, Shawna Shirley, Loree Heller, Richard Heller. 824 Cytokine-Based Log-Scale Expansion of Functional Human Dendritic Cells Yui Harada, Yasuji Ueda, Hiroaki Kinoh, Aki Furuya, Makoto Inoue, Mamoru Hasegawa, Tomohiko Ichikawa, Yoshikazu Yonemitsu. 825 Small Plasmid/PEI/Anionic Polysaccharide Ternary Complex Particles: Toxicity and Animal Clinical Study Yoshiyuki Koyama, Masahiko Tojyo, Chieko Yoshihara, Katsuyuki Hamada, Tomoko Ito. 826 Imiquimod, a TLR7 Agonist, Delays Brain Tumor Growth through Suppression of Treg in Brain Zhengming Xiong, Shoko Toma, John Ohlfest.

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Abstract Directory Saturday, May 22, 2010 Poster Session III — continued 6:00 PM - 8:00 PM Room: Exhibit Hall C

Cancer – Immunotherapy III 827 Strategy Escalation: An Emerging Paradigm for Safe Clinical Development of T Cell Gene Therapies R. P. Junghans.

Cancer-Oncolytic Viruses III 828 Combination of Promoter and microRNA Detargeting Is Greatly Advantageous in Attenuation of Adenoviral Replication in Hepatic Cells Justyna Leja, Berith Nilsson, Di Yu, Magnus Essand. 829 Combining Oncolytic Herpes Simplex Virus and Chemotherapy: G47D and Temozolomide Synergize in Killing Glioma Stem Cells Ryuichi Kanai, Hiroaki Wakimoto, Robert L. Martuza, Samuel D. Rabkin. 830 Transcriptional Targeting of an Oncolytic HSV-1 to Midkine-Positive Tumors Arturo R. Maldonado, Chuck Klanke, Anil G. Jegga, Bruce J. Aronow, Yonatan Y. Mahller, Timothy P. Cripe, Timothy M. Crombleholme. 831 Preclinical Studies Support the Efficacy of Prodrug Activator Gene Transfer in Experimental Glioma Models by Enhanced ReplicationCompetent Retrovirus Vector Akihito Inagaki, Kei Hiraoka, Christopher R. Logg, Hiroshi Matsumoto, Joan Robbins, Omar Perez, Harry Gruber, Douglas Jolly, Noriyuki Kasahara. 832 Intrapleural Administration of Third Generation Oncolytic HSV1 (G47D) Is Highly Efficacious in Orthotopic Malignant Pleural Mesothelioma Models Masamichi Takahashi, Yasushi Ino, Nobuhito Saito, Tomoki Todo.

835 Inhibition of Autophagy Has Negative Impact on Virus Production Ilya V. Ulasov, Atique U. Ahmed, Bart Thaci, Natalya V. Kaverina, Maciej S. Lesniak. 836 Armed and Transcriptionally Driven Oncolytic HSV-1 Enhances Antitumor Efficacy In Vitro and In Vivo Ji-Young Yoo, Amy Haseley, Anna Bratasz, Kimerly Powell, Balveen Kaur. 837 Determining Adenovirus Titers Following Extraction of the Virus from Tumors in the Presence of Anti-Adenovirus Antibodies Debanjan Dhar, Karoly Toth, William S. M. Wold. 838

Abstract Withdrawn

839 The Use of Third Generation Oncolytic HSV-1 Expressing Luciferase for Demonstration of Real-Time Biodistribution and Dynamics Yihan Wu, Yasushi Ino, Tomoki Todo. 840 Highly Attenuated Vaccinia Virus as a Potential Oncolytic Agent for Cancer Virotherapy Takafumi Nakamura, Minoru Kidokoro, Mina Hikichi, Hisatoshi Shida, Hideaki Tahara. 841 Enhanced Tumor Killing by a Mortalin Targeting Modified Oncolytic Adenovirus Jung-Sun Lee, Ji Young Yoo, Jihoon Ryu, Tomoko Yaguchi, Sunil C. Kaul, Renu Wadhwa, Chae-Ok Yun. 842 Increased Oncolytic Ability of a Double RGD-Modified Adenovirus in Ovarian Cancer Lena J. Gamble, Qiana L. Matthews, Anton V. Borovjagin, David T. Curiel.

Cancer-Targeted Gene & Cell Therapy III 843 Increasing the Efficacy of Monoclonal Antibody Therapy of Breast Cancer through Transient Remodeling of Intercellular Junctions Jonas Persson, Max Drescher, ZongYi Li, Roma Yumul, Ying Liu, Hongjie Wang, Akseli Hemminki, Pascal Fender, Andre Lieber. 844 Improvement of Cancer Gene Therapy by Incorporating a Chicken Hypersensitive Site-4 Insulator into TSTA-Based Tumor-Targeting Vector Chien-Fu Chen, Yu-Ping Sher, Mien-Chie Hung, Chia-Ling Hsieh.

Saturday, May 22nd

833 Engineered Measles Viruses for Oncolytic Therapy of Pancreatic Cancer Sascha Bossow, Christian Grossardt, Stefanie Sawall, Christoph Springfeld, Achim Temme, Roberto Cattaneo, Christof von Kalle, Guy Ungerechts.

834 Incorporation of a Mutated E1A and a Chimeric 5/3 Fiber Improved the Therapeutic Efficacy of a Novel CRAd (Ad-F512) on Disseminated Ovarian Cancer Veronica M. Lopez, Angel A. Rivera, Lorena Benedetti, Diego L. Viale, Kristopher J. Kimball, Minghui Wang, Alicia J. Bravo, Christine Dorvault, Joanne Douglas, Zeng B. Zhu, Ronald D. Alvarez, David T. Curiel, Osvaldo L. Podhajcer.

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Abstract Directory Saturday, May 22, 2010 Poster Session III — continued 6:00 PM - 8:00 PM Room: Exhibit Hall C

Cancer-Targeted Gene & Cell Therapy III 845 Cytokine Induced Killer (CIK) Cells for Cell Therapy of Acute Myeloid Leukemia (AML): Improvement of Their Immune Activity by Expression of CD33-Specific Chimeric Receptors (CR) Virna Marin, Irene Pizzitola, Valentina Agostoni, Helene Finney, Alastair Lawson, Martin Pule, Raphael Rousseau, Andrea Biondi, Ettore Biagi. 846 Exosome-Mediated Suicide Gene Therapy for Brain Tumors Arda Mizrak, Senol Ozlem, Baris G. Ozdener, Leonora Balaj, Johan Skog, Xandra O. Breakefield, Okay Saydam. 847 Eradication of Therapy-Resistant Human Prostate Tumors Using an Ultrasound Guided Site-Specific Cancer Terminator Virus Delivery Approach Pier Paolo Claudio, Rounak Nande, Mary A. Teter, Paul B. Fisher, Sarkar Denavand, Luigi Claudio, Adelaide Greco, Candace Howard. 848 Systemic Tumour Targeting with Orally Administered Non-Pathogenic Bacteria Michelle Cronin, David Morrissey, Simon Rajendran, Douwe van Sinderen, Gerald C. O’Sullivan, Mark Tangney. 849 Transient Intratumoral Relaxin Expression Results in Tumor Stroma Degradation and Improves Breast Cancer Therapy by Herceptin ZongYi Li, Ying Liu, Roma Yumul, Max Drescher, Jonas Persson, Ines Beyer, Andre Lieber.

Saturday, May 22nd

850 Developing a Multi-Modal Gene-Based Imaging and Therapy To Treat Disseminated Hormone Refractory Prostate Cancer Ziyue Jiang, Makoto Sato, Lily Wu. 851 Adenoviruses-Mediated Expression and Cell-Mediated Delivery of Interferon-Lambda Produced Anti-Tumor Effects to Human Esophageal Carcinoma In Vivo Masatoshi Tagawa, Quanhai Li, Kiyoko Kawamura, Shinya Okamoto, Shan Yang, Hiroshi Kobayashi, Muneo Numasaki, Yuji Tada, Kenzo Hiroshima, Hideaki Shimada. 852 Inhibition of Angiogenesis and Suppression of Colorectal Cancer Metastatic to the Liver Using the Sleeping Beauty Transposon System Lalitha R. Belur, Kelly M. Podetz-Petersen, Brent Sorenson, Josh Parker, Cathy Carlson, Daniel Saltzman, Ramakrishnan Sundaram, R. Scott McIvor.

EXHIBITOR PROSPECTUS final program

853 Plasmid AMEP, a New Efficient Antitumor Gene Transfer Product for the Treatment of Metastatic Melanomas Celine Bouquet, Veronique Trochon-Joseph, Valerie Polard, Lluis M. Mir, Michel Perricaudet, Sophie Lebel-Binay. 854 Adenovirus-Delivered Short Hairpin-Structured RNA for Microphage Migration Inhibitory Factor Silencing Inhibits Human Prostate Cancer Cell Proliferation Ling Zhang, Anqi Wang, Chengyao Li, Hongwei Li, Leah N Braseth, Arseniy E Shabashvili, Colin Sumners. 855 Monitoring of Electroporation-Mediated Diphtheria Toxin A Expression in Prostate Cancer Cells Grown in SCID Mice Christine Goepfert, Felix J. Frey, Brigitte M. Frey. 856 Clinical Gene Transfer in the Community: Ten Years of Local IBC Review Jan P. Vleck.

Hematologic and Immunologic Gene & Cell Therapy III 857 High Incidence of Vector Integration near Cancer-Related Genes within Primitive Hematopoietic Stem Cells (HSC) after Fetal Gene Transfer with g-Retroviral Vectors Joe Tellez, Josh Wood, Graça Almeida-Porada, Chris D. Porada. 858 Successful Treatment of Canine Leukocyte Adhesion Deficiency Using a MSCV-Promoter Based Foamy Viral Vector and Low-Dose Busulfan Conditioning Thomas R. Bauer, Jr., Erik M. Olson, Laura M. Tuschong, James M. Allen, Tanya H. Burkholder, David W. Russell, Dennis D. Hickstein. 859 Genetically Engineered VIP-Expressing Dendritic Cells Are Therapeutic in the Autoimmune Mouse Model of Experimental Autoimmune Encephalomyelitis Miguel G. Toscano, Mario Skarica, Weiming Kong, Doina Ganea. 860 Long-Term Preclinical Evaluation of Lentiviral Vectors for SCID-X1 Gene Therapy in a SCID-X1 Mouse Model Sheng Zhou, Suk See DeRavin, Zhijun Ma, Taihe Lu, John T. Gray, Harry L. Malech, Brian P. Sorrentino. 861 Transduction of Human CD34+ Derived NOG Repopulating Cells with an Insulated SIN Lentiviral Vector for SCID-X1 Produced from a Stable Producer Cell Line Michael R. Greene, Yoon-Sang Kim, Timothy Lockey, Paolo Fagone, Robert E. Throm, John T. Gray, Derek A. Persons, Brian P. Sorrentino.

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Abstract Directory Saturday, May 22, 2010 Poster Session III — continued 6:00 PM - 8:00 PM Room: Exhibit Hall C

Hematologic and Immunologic Gene & Cell Therapy III 862 Prevention of Atopic Dermatitis by Plasmid-Based Sustained Transgene Expression of Interferon g in NC/NGA Mice Kayoko Hattori, Makiya Nishikawa, Kanitta Watcharanurak, Akihiko Ikoma, Kenji Kabashima, Hiroyasu Toyota, Yuki Takahashi, Rei Takahashi, Yoshihiko Watanabe, Yoshinobu Takakura. 863 Expression of an Antibody Against CTLA4 from Tumor Infiltrating Leukocytes Induces Efficient Anti-Tumor Immune Responses in a Mouse Model for Breast Cancer Jonas Persson, Ying Liu, Roma Yumul, ZongYi Li, Steve Roffler, Andre Lieber. 864 Foamy Virus Vectors with MGMT-P140K Mediate Efficient Selection in a Canine Pyruvate Kinase Deficiency Model for Hematopoietic Stem Cell Gene Therapy Brian C. Beard, Robert A. Wu, Christina Ironside, Punam Malik, Kiem Hans-Peter, Trobridge D. Grant. 865 Modeling of Congenital Erythropoietic Porphyria by RNAInterference: A New Tool for Preclinical Gene Therapy Evaluation Hubert de Verneuil, Elodie Robert-Richard, Magalie Lalanne, Isabelle Lamrissi-Garcia, Véronique Guyonnet-Duperat, Emmanuel Richard, Frédéric Mazurier, Cécile Ged, François Moreau-Gaudry. 866 Gene Corrected Neutrophils from Induced Pluripotent Stem Cells Derived from an X-CGD Disease Model Sayandip Mukherjee, Giorgia Santilli, Michael P. Blundell, Elena Almarza, Juan Bueren, Adrian J. Thrasher.

868 Sleeping Beauty-Mediated Therapeutic Gene Transfer into Hematopoietic Stem Cells Kendra A. Hyland, Erik R. Olson, Xianzheng Zhou, R. Scott McIvor.

870 Delivery of Soluble ICAM-1 in NOD Salivary Glands Can Result in B Cell Proliferation and Immunoglobulin Production Jelle L. Vosters, Nienke Roescher, Paul P. Tak, John A. Chiorini. 871 Modulation of CXCR4 Expression by Novel Growth Factor Cocktail on Stem Cells Somayeh Shahrokhi, Kamran Alimoghaddam, Massoumeh Ebtekar, Zohre Sharifi, Seyed Hamidolah Ghaffari, Ali Akbar Pourfathollah, Mahboobeh Mohseni, Ardeshir Ghavamzadeh.

Lung and Respiratory Disease Gene & Cell Therapy 872 Efficient Gene Delivery to Pig Airways Using Helper-Dependent Adenoviral Vectors Huibi Cao, Jonathan C. Yeung, Cathleen Duan, Allan Coates, Kitty Leung, Shaf Keshavjee, Jim Hu. 873 SIV Vector Pseudotyped with SeV-F/HN Envelope Proteins Produces Long Lasting Expression in the Murine Lung, Is Readministrable and Transfects Human Airway Models Uta Griesenbach, Makoto Inoue, Cuixiang Meng, Andrea M. Brum, Raymond Farley, Nikki Newman, Eiji Akiba, Mamoru Hasegawa, Eric W. F. W. Alton. 874 Airway Lentiviral Gene Transfer In Marmosets David Parsons, Richard Bright, Darren Miller, Don Anson. 875 Pulmonary Delivery of mRNA: In Vitro and In Vivo Evaluation Oliwia Najder, Joanna Rejman, Stefaan De Smedt, Joseph Demeester, Mario Van Poucke, Luc Peelman, Niek N. Sanders. 876 Inhibiting Bap31 by Dominant Negative p20 Does Not Promote CFTR Translocation to the Cell Membrane Yadong Zhang, Wei Zhang, Min Li, T. C. Hwang, Yihong Ye, Dongsheng Duan. 877 Targeted siRNA Delivery to Lung Epithelia Reduces Mucus Secretion in a Mouse Model for Allergic Asthma Hyo Sung Choi, Jae Wook Jung, Hong Seok Ban, Nahyun Kim, Priti Kumar, Sang kyung Lee. 878 The T1a Promoter Mediates Nuclear Import of Plasmid DNA into Alveolar Epithelial Type I Cells Lynn F. Gottfried, David A. Dean.

Saturday, May 22nd

867 The Comparison of Various Promoters for Wiskott Aldrich Syndrome Gene Therapy Using Insulated Lentiviral Vectors Rachel M. Koldej, Deepti Banka, Gael Carney, Hui Fen Zhao, Derek Persons, Arthur W. Nienhuis.

869 (Moved to Poster Session I after Abstract 233) Myelospecific Self-Inactivating Gammaretroviral Vectors for p47phox CGD Gene Therapy Vital Wohlgensinger, Christian Brendel, Axel Schambach, Manuel Grez, Janine Reichenbach, Reinhard A. Seger, Ulrich Siler.

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Abstract Directory Saturday, May 22, 2010 Poster Session III — continued 6:00 PM - 8:00 PM Room: Exhibit Hall C

Lung and Respiratory Disease Gene & Cell Therapy 879 Effects of LPC on Lung Mechanics in Non-Human Primates P. Hiatt, R. McConnell, N. Grove, D. Dang, N. Brunetti-Pierri, D. Palmer, A. Beaudet, P. Ng. 880 Repeat Aerosol Delivery of Concentrated PEI/pDNA to the Sheep Lung Gerry McLachlan, Lee A. Davies, Cath M. Gordon, Christina Vrettou, Eilidh Baker, Peter Tennant, Alison Baker, Rebecca L. Coles, Dominique McCormick, Stephanie G. SumnerJones, Stephen C. Hyde, Deborah R. Gill, D. David S. Collie. 881 Pseudotype Switching as a Strategy To Permit Efficient ReAdministration of Lentiviral Vectors to Mouse Airways John C. Olsen, Angela Giddings, Manij Patel.

Oligonucleotide & RNAi Therapeutics III 882 Antinociceptive Potentiation and Attenuation of Tolerance by Intrathecal b-Arrestin 2 siRNA in Rats Chung-Ren Lin, Kuan-Hung Chen, Chih-Hsien Wu, Yi-Sen Chen.

Saturday, May 22nd

883 siRNA Cationic Delivery Systems for Therapeutic Applications Anne-Laure D. Bolcato-Bellemin, Marie-Elise Bonnet, Gaëlle Deglane, Nathalie Lenne, Patrick Neuberg, Clément Paris, Jean-Paul Behr, Patrick Erbacher.

887 Safety Results of a Phase 1 Clinical Study for Treatment of Patients with Advanced Solid Tumors with the TLR9 Agonist MGN1703 Max E. Scheulen, Manuel Schmidt, Michael S. von Bergwelt-Baildon, Ulrich Hacker, Alexander Shimabukuro-Vornhagen, Heike Richly, Bahar Nokay, Marina Tschaika, Burghardt Wittig, Martin R. Weihrauch. 888 Parallel Assessment of Silencing Potency of Conventional SiRNAs vs. SiRNA Variants Nicholas M. Snead, Min-sun Song, Kumi Sakurai, John J. Rossi. 889 Inhibition of Viral Hepatitis with Non-Coding RNAs Jovanna Gonzalez-Rojas, Amaya Abad, Lorea Blazquez, Nerea Razquin, Xabier Abad, Puri Fortes. 890 Screening of Nature Compounds for Enhancing Antisense Oligonucleotide-Induced Exon Skipping of Dystrophin Gene In Vitro and In Vivo Bo Wu, Caryn Cloer, Peijuan Lu, Mingxing Wang, Wei Li, Kazuo Koike, Qi Long Lu. 891 A Combination of PHD2 Prolyl Hydroxylase Silencing and Biocompatible Polyplex Nanocarrier Enhances Therapeutic Angiogenesis Shourong Wu, Nobuhiro Nishiyama, Keiji Itaka, Takuya Hashimoto, Hiroyuki Koyama, Mitsunobu R Kano, Kazunori Kataoka. 892 Development of a Novel, RNAi-Based Therapeutic Targeting Pancreatic Duodenal Homebox-1 (PDX-1) for Insulinoma and Pancreatic Cancer Zhaohui Wang, Shi-he Liu, Donald D. Rao, Phillip B. Maples, Neil Senzer, John Nemunaitis, F. C. Brunicardi.

884 Therapeutic Effect of Intravenous Administration of New Type Hybrid Staple-Modified NFkB Decoy Oligonucleotides on Inflammatory Bowel Disease Model Mice Kazunari Ozaki, Hirofumi Makino, Takashi Miyake, Yasushi Takeya, Motokuni Aoki, Hiromi Rakugi, Ryuichi Morishita.

893 Systemic Delivery of Highly Active Functionalized LipopolyamineFormulated siRNA to Murine Lung with Reduction of Inflammation by Dexamethasone Pre-Treatment Jeff Sparks, Kevin J. Polach, Richard T. Congo, Gregory Slobodkin, Angela Rea-Ramsey, Casey Pence, Majed Matar, Diane McClure, Jennifer Rice, David Ulkoski, Elaine Brunhoeber, Leslie Wilkinson, Khursheed Anwer, Jason G. Fewell.

885 Artificial miRNA Clusters for Targeting Multiple Regions of the HCV Genome Xiao Yang, Virginia Haurigot, Shangzhen Zhou, Linda B. Couto.

894 Ornithine and Tryptophan as an Efficient Polycation for siRNA Delivery to Tumor Cells Yusuke Sato, Hiroto Hatakeyama, Hideyoshi Harashima.

886 Design and Pre-Clinical Evaluation of Adeno-Associated Viral (AAV) Vectors for Therapeutic Inhibition of Disease-Associated miRNAs Nina Schürmann, Ellen Hoffmann, Dirk Grimm.

895 Influence of 5’ Terminal Triphosphate in Short Hairpin RNA Targeting HCV Replicon on Antiviral Activity and Innate Immune Response Kyungbo Kim, Bokhui Lee, Soo-Ryoon Ryoo, Dal-Hee Min, Dong-Eun Kim. 896 Enhanced Delivery of GFP siRNA Using New Amino Lipid-Based Cationic Liposomes Hyun-Ki Kim, Enkhzaya Davaa, Chang-seon Myung, Jeong-Sook Park.

EXHIBITOR PROSPECTUS final program

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

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Abstract Directory Saturday, May 22, 2010 Poster Session III — continued 6:00 PM - 8:00 PM Room: Exhibit Hall C

Oligonucleotide & RNAi Therapeutics III 897 Gene Editing Activity in C. elegans Directed by Single-Stranded Oligonucleotides Kerry A. Falgowski, Eric B. Kmiec.

Stem Cell Therapies 898 Engraftment into the Liver of Transplanted Adipose Tissue-Derived Stromal Cells Detected by Noninvasive Bioluminescence Imaging Giuliana Di Rocco, Antonietta Gentile, Annalisa Antonini, Maurizio C. Capogrossi, Gabriele Toietta. 899 Establishment of iPS Cells Derived from the New World Monkey of Common Marmoset Saori Yamaguchi, Tomotoshi Marumoto, Hirotaka Kawano, Shinya Shimoda, Yoshie Miura, Tomoko Inoue, Ryo Kurita, Hiroyuki Miyoshi, Erika Sasaki, Atsushi Takahashi, Kenzaburo Tani. 900 Umbilical Cord Blood-Derived Mesenchymal Stem Cells Acquire Cardiac Phenotype with Oxytocin Treatment and Enhance Cardiac Recovery after Myocardial Infarction Yong Sook Kim, Jin Sook Kwon, In-Kyu Park, Myung Ho Jeong, Youngkeun Ahn. 901 Ex Vivo Expansion and Enrichment of Murine Adult Neural Stem Cells for Gene Therapy of Neurogenetic Diseases Sushma Chaubey, John H. Wolfe. 902 Dual Effect of Mesenchymal Stem Cells Genetically Modified with a Single-Chain Antibody Against EGFRvIII on the Targeting and Growth of EGFRvIII Expressing Experimental Glial Tumors Irina V. Balyasnikova, Sherise D. Ferguson, Yu Han, Maciej S. Lesniak.

904 The Granules Isolated from Mast Cells Have Increased the Survival of Bone Marrow-Derived Mesenchymal Stem Cells Jin Sook Kwon, Yong Sook Kim, In-Kyu Park, Myung Ho Jeong, Youngkeun Ahn.

906 Enrichment of Bone Marrow Stem Cells In Vivo by Dissociation- and Proliferation-Inducing Compounds Augments Bone Growth in a Mouse Segmental Bone Defect Model Sanjay Kumar, Selvarangan Ponnazhagan. 907 A Delayed Treatment of Mesenchymal Stem Cells Is Effective in Rescuing Mice with Established LPS-Induced Acute Lung Injury Shirley H. J. Mei, Colleen H. Parker, Conrad Liles, Duncan J. Stewart. 908 Lentiviral Vector-Mediated Transduction in Murine SMA Embryonic Stem Cells Derived from Spinal Muscular Atrophy (SMAI) Mice Arianna Malgieri, Andrea Luchetti, Massimo Sanchez, Maria Bellocchi, Paola Spitalieri, Douglas A. Kerr, Giuseppe Novelli, Federica Sangiuolo. 909 Development of a Reporter System for I-SceI-Mediated Genome Editing in Canine Hematopoietic Stem Cells Nina M. Muñoz, Grant D. Trobridge, Brian C. Beard, Byoung Y. Ryu, Andrew M. Scharenberg, Hans-Peter Kiem. 910 Liver Disease Correction with Cells Isolated from Cadaveric Liver Laura Erker, Hisaya Azuma, Laura Eaton, Eric Benedetti, Bryan Jensen, Andrew Y. Lee, Milton Finegold, Holger Willenbring, Markus Grompe. 911 Efficient Gene Transfer into CD34+ Hematopoietic Stem Cells Using Clinical Grade Lentiviral Vector Expressing the Human Beta-Globin Gene Clare Taylor, Leda Ferro, Jason Plotkin, Xiuyan Wang, Michel Sadelain, Isabelle Rivière1. 912 Embryonic Stem Cell Therapy Enhances Tendon Regeneration in a Large Animal Flexor Tendonitis Model Alan J. Nixon, Ashlee A. Watts, Oleg Kopyov. 913 Generation of Cystic Fibrosis Induced Pluripotent Stem (iPS) Cells for Gene Therapy R. G. Sargent, Y. Yang, A. Lee, S. Kim, L. Ye, J. Chang, D. C. Gruenert.

Saturday, May 22nd

903 Expression of Coagulation Factors from Murine iPS-Derived Liver Cells Kohei Tatsumi, Yoshihiko Sakurai, Kazuo Ohashi, Shogo Kasuda, Junko Kato, Teruo Okano, Midori Shima.

905 Large-Scale Expansion of Undifferentiated Human Embryonic Stem Cells in Serum-Free Suspension Culture Vincent C. Chen, Sylvana Couture, Jonathan Auerbach, Araz Toumadje, Lara Ausubel, David Hsu, Larry Couture.

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Abstract Directory Saturday, May 22, 2010 Poster Session III — continued 6:00 PM - 8:00 PM Room: Exhibit Hall C

Late Breaking Science III 938 Blockage of Viral RNA Transport of REV- and CTE-Dependent Lentiviral Vectors Encoding NUP98 Fusion Genes Yildirim Dogan, David C Dorn, Sutapa Sahay, Anna Franceschino, Malcolm AS Moore. 939 Novel Helper-Independent piggyBac Plasmids for Gene Delivery Approaches Johann Urschitz, Chad Walton, Jesse Owens, Miyuri Kawasumi, Hideaki Yamashiro, Ilko Stoytchev, Joel Marh, Craig J. Coates, Joseph M. Kaminski, Pawel Pelczar, Ralph V. Shohet, Stefan Moisyadi 940 Transgene Trafficking to Nuclear Periphery is a Barrier to Transfection in a Lipid Substituted Nonviral Cationic Polymer Charlie Y-M Hsu, Hasan Uludag. 941 Studies on the Biodistribution Evaluation of Gene Therapy Products using the Real Time-PCR Song Hyun, Choi Min Joung, Seo Su Jin, Han Sung Yeol, Lee Yeon Ho, Park Youn Joo, Ahn Kwang Soo, Kim Tae Geun. 942 Activity, Integration Preferences, and Localization of TcBuster; a Novel hAT Transposon System Lauren E. Woodard, Aparna Kaja, Robert H. Hice, Peter W. Atkinson, Nancy L. Craig, Matthew H. Wilson.

Saturday, May 22nd

943 Overcoming the Lipofection Resistance of Oral Cancer Cells: Polyplexes and Adenoviruses Senait Gebremedhin, Krystyna Konopka, Ilya Ulasov, Maciej Lesniak, Nejat Düzgünes. 944 Association of CD44 Expression with Hormone Receptor Status in Breast Cancer P Olivera, E Ortiz, R Romero, A Palacios, R Morales, G Cervantes, S Galván, E Langley, A García-Carrancá.

EXHIBITOR PROSPECTUS final program

945 Systemic Apoptotic Gene Therapy for Pancreatic Cancer: Multi-Dose Toxicity Study of the BikDD Nanoparticle K Barnhart, M Javle, S Miller, J Hsu, J McMannis, P Smith, J Abbruzzese, MC Hung. 946 Phosphatidylserine Immobilization of Lentivirus for Localized Gene Transfer Seungjin Shin, Hannah M. Tuinstra, David M. Salvay, Lonnie D. Shea. 947 Engineering Adenovirus Serotype 5 Vectors to Increase Hepatocyte Transduction for Liver Gene Therapy Reeti Khare, Michael A. Barry

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index A

Aagaard, Lars . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695 Aalbers, Caroline J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 66 Aartsma-Rus, Annemieke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 153 Abad, Amaya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 889 Abad, Xabier . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 889 Abay, K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Abbasi, Meysam . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 562, 568 Abdelbary, Hesham . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39 Abdulrasul, Fouzia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349 Abrameto, Javier A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616 Abu-Asab, Mones . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135 Acharya, Bishnu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 216 Adachi, Kei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 7, 703 Adair, Jennifer E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294 Adamo, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 323 Adamo, Richard F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 425 Adams, Marvin E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 584 Adams, Samuel J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 622 Adriaansen, Janik . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 761 Affin, Lulit A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 125 Aflaki, Pejman . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237 Agbandje-McKenna, Mavis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 65 Agerholm-Larsen, Birgit . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 109 Agkatsev, Sarina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 602 Agostoni, Valentina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845 Agudo, Judith . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140, 762 Ahlén, Gustaf . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 111 Ahlen, Gustaf . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 289 Ahlzadeh, Garbielle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 475 Ahmad, Farizan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 212, 512 Ahmad, Sarfraz . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 812 Ahmed, Atique U. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 191, 718, 835 Ahmed, Nabil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 179, 470, 471 Ahmed, Sherif O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451 Ahn, Miwon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 52 Ahn, Youngkeun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 399, 751, 900, 904 Ahouansou, Ornella . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19 Ahrens, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 271 Ahtiainen, Laura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 197, 291 Aimi, Junko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 35 Airavaara, Mikko T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709 Airenne, Kari J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 669 Aiuti, Alessandro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225, 229, 276, . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295, 345 Akbulut, Hakan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85 Akiba, Eiji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 873 Akita, Hidetaka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 246, 253, 401, 591, 743 Akkina, Ramesh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287

Alba, Raul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380, 573 Albelda, Stephen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 611 Alberici, Luca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 644 Albrecht, Douglas E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796 Al-Dahhak, Roula . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155 Alden, Anderson . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181 Aldhamen, Yasser A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638, 651 Aldrich, Carla J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 332 Alemany, Ramon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 291, 496 Alexander, Ian E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 409 Alfaro, Carlos . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Alferiev, Ivan S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 425 Ali, Nowrouzi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138 Alimoghaddam, Kamran . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871 Allain, Jean-Pierre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 808 Allay, James A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 668 Allen, Cornell . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 675 Allen, Cory . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 490, 499 Allen, Henry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 536, 586 Allen, James M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234, 459, 786, 858 Allen, Lee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 576 Allen, Michael H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 122, 125 Allen, Phil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 392 Allimperti, Stella . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 358 Allocca, Mariacarmela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 64 Almarza, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 351 Almarza, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 866 Almeida-Porada, Graça . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 551, 857 Alton, Eric W. F. W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 873 Alvarez, Ronald D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36, 503, 507, 834 Alves, Paula M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 278, 570, 579 Alzadeh, Gabrielle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181 Alzuguren, Pilar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 810 Amalfitano, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638, 651 Amaral, Ana I. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 278 Amarnath, Shoba . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 341 Ambrosi, Alessandro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 345, 346 Amendola, Mario . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 413 An, Weijun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603 Anagnostopoulos, Achilles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137 Anagnou, Nicholas P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 338 Anderson, Jonathan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279 Anderson, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 281 Anderson, Susan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 300 Ando, Dale . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783, 798 Andre, Kris . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 41, 803 Andreadis, Stelios . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 359 Andreadis, Stelios T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 358, 282 Andreani, Marco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319

143

144

American Society of Gene & Cell Therapy

Abstract Author Index Andresen, Thomas L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 511 Angell, Yu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 731 Anguela, Xavier M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140 Anikster, Yair . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144 Annan, Anand C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 503 Annoni, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 545 Annunziata, Patrizia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 758 Anson, Don . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 874 Anson, Donald . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 621 Ansorge, Sven . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 361 Antonini, Annalisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 898 Antoniou, Michael N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531 Anwer, Khursheed . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893 Aoki, Motokuni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 884 Aoki, Yoshitsugu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 565 Appledorn, Daniel M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638, 651 Aramaki, Yukihiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 102 Aras, Rahul A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 426, 438 Arbuthnot, Patrick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 82, 563 Ardiani, Andressa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 330 Arens, Anne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 286, 305, 347 Ariga, Tadashi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678 Arkin, Adam P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 802, 805 Arman, A. Cyrus . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21 Armaroli, Annarita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258 Armstrong, Adam . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 633 Arnett, Andrea L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 461 Arning, E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139 Arntz, Onno J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 559, 627 Aronovich, Elena L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 325, 728 Aronow, Bruce J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 830 Arruda, Valder R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308, 551, 614, 615 Arsenau, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 35 Arshad, Shazia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 530 Arstila, Petteri . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 538 Arun, Srivastava . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 68 Arzberger, Silke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312 Asakawa, Nagisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 410 Asdourian, George . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 444 Asefa, Benyam . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 165 Aslanidi, George . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 363, 370, 378 Asokan, Aravind . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 62, 160, 364, 367, 403 Assi, Hikmat . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26, 181 Astone, Dalila . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435 Astrakhan, Alexander . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 343, 529 Athanassiadou, Aglaia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 340, 729 Atkins, Harry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39 Aubert, Dominique . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138 Aubourg, Patrick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19

EXHIBITOR PROSPECTUS final program

Audet, Sarah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 402 Auerbach, Jonathan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 905 Aurelian, Laure . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 717 Auricchio, Alberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59, 64, 443, 624, 758 Aurigemma, Rosemarie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36 Ausubel, Lara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 905 Ausubel, Lara J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279 Aviles, Misael O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 121 Ayuso, Eduard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762 Azuma, Hideki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 397 Azuma, Hisaya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910

b

Bacci, Maria Laura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59 Back, Jin Ee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128 Badding, Melissa A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 588 Baek, Jin Ee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 406, 754 Bagarazzi, Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 477 Bahar, Bahar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 664 Bahou, Wadie F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372 Bailey, Debra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 739 Bais, Anthony J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 27 Bajzer, Zeljko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 208 Bak, Rasmus O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695, 726 Bakay, Marina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 425 Baker, Alison . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880 Baker, Andrew H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380, 715, 723 Baker, Andrew J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783 Baker, Eilidh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880 Baker, K. Scott . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 232 Balaj, Leonora . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 846 Baldim, Victor . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 485 Balducci, Anthony G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 271 Bali, Deeksha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 136 Bali, Pawan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678 Ball, Claudia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612 Ballmaier, Matthias . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 318 Balyasnikova, Irina V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 902 Balyasnikova, Irina Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 191 Balzer, Frederick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 454 Ban, Hiroshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303, 634 Ban, Hong Seok . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 877 Ban, Hong-Seok . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 407 Bandyopadhyay, Sarmistha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 711 Banfi, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 430, 597 Banka, Deepti . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 867 Banks, Glen B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 459, 584 Banwell, Brenda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155 Baranov, Vladimir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Baranov, Vladislav S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 750 Baranyi, Lajos . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527 Barbas, III, Carlos . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 340 Barbas, III, Carlos F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 94, 339 Barber, Glen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 604 Bareja, Akshay . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 466 Barlow, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17 Barnes, Justin A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 503 Barohn, Richard J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155 Baron, Stéphanie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 226 Barouch, Dan H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 723 Barron, Valerie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439 Barry, Frank . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 270 Barry, Michael A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 81 Bartek, Jiri . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 265 Bartholomae, Cynthia C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 298, 345, 346, 347 Bartido, Shirley . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32 Bartlett, Jeffrey S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 547, 699 Bartoli, Marc . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48 Barve, Minal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 292, 335 Barzel, Adi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16 Basner-Tschakarjan, Etiena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43 Bassi, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 666 Basu, Gaurav . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 107 Batchu, Ramesh B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250 Batchvarova, Milena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 667 Batshaw, Mark L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 416 Batsis, Ioannis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137 Bauer, Gerhard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 281 Bauer, Jr., Thomas R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234, 858 Bauerschmitz, Gerd . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 193 Baum, Bruce J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 74, 77, 143, 761 Baum, Christopher . . . . . . . . . . . . . . . . . . . . . . . 284, 302, 304, 318, 528, 619 Baumhover, Nicholas J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 117, 594 Bazzani, Reto P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 739 Beal, Nikia L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445 Bear, Adham . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 469 Bear, Adham S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 816 Beard, Brian C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294, 864, 909 Beaudet, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879 Beaudet, Arthur . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 639 Beaudoin, Esther L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 27 Becher, Ulrich M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693 Bechinger, Burkhard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 400 Becker, Pamela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137, 294 Bedell, Cynthia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 292, 335, 536 Beheshti, Ben . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 217 Behr, Jean-Paul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883

Behr, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 381 Belicchi, Marzia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460, 464, 795 Bell, Christie L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 65 Bell, Jason B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 325, 728, 734 Bell, John C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33, 37, 39 Bell, Peter L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 45, 412, 416, 543, 759 Bellavia, Diego . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599 Bellayr, Ian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 465 Bello, Alexander J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 64 Bellocchi, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908 Beloglazova, Irina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434 Belousova, Natalya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 725 Belur, Lalitha R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20, 852 Benati, Claudia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276 Benderdour, Mohamed . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 756 Bendjelloul, Mehdi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463 Benedetti, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910 Benedetti, Lorena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 834 Benedicenti, Fabrizio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 298 Benes, Vladimir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 270 Bengtson, Karen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 803 Benito, Alberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Bennett, C. F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55 Bennett, C. Frank . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 557 Bennink, Miranda B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 559, 627 Benvegnu, Thierry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120 Benza, Raymond L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 104 Berger, Carolina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 618 Bergmann, Juri . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237 Bernard, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Berraondo, Pedro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 485, 810 Berry, Charles C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13 Bertin, Terry K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 642 Bertolotti, Roger . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 244 Bertrand, Emilie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 395 Besarab, Anatole . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616 Besharse, Joseph C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17 Betts, Michael R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 164 Beuerlein, Michele . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 280 Beyer, Ines . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 264, 849 Bhasvar, Janak . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 269 Bhatnagar, Rakesh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 562 Bhavsar, Janak . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 272 Biagi, Ettore . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845 Bian, Ang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 389 Bian, Yixin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 806, 808 Bianco, Paolo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 133 Biasco, Luca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225, 345 Bienemann, Alison . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394

145

146

American Society of Gene & Cell Therapy

Abstract Author Index Bien-Willner, Ricardo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155 Biffi, Alessandra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276, 313, 315, 316 Bigey, Pascal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 791 Bikram, Malavosklish . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 405 Billiet, Ludivine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 395 Binder-Scholl, Gwendolyn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 798 Bindra, Ranjit S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 50, 285 Biondi, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845 Bird, Andrew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 142 Bisgrove, Dwayne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 674 Bish, Lawrence . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433, 625 Bisordi, Katie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 630 Biswas, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223, 697 Bizario, João C. S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 790 Black, Margaret E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 330 Blake, Bonita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 355 Blake, Bonita L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2 Blanchard, Jan E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39 Blanco, Laura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 485, 708 Blasczyk, Rainer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612 Blau, Carl A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 730 Blazar, Bruce R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221 Blazquez, Lorea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 889 Blechingberg, Jenny . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91 Blick, Gary . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 646 Blits, Bas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 249 Blondeau, Johanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 229 Blundell, Michael P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 866 Blundell, Mike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 229 Boccalatte, Francesco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 337 Bochner, Bernard H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334 Boczkowski, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 561 Bodine, David M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 521 Bogan, Janet . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 454, 458, 467, 785 Böhm, Marie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612 Boisgérault, Florence . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48 Bolcato-Bellemin, Anne-Laure D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883 Bollard, C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30 Bollard, Catherine . . . . . . . . . . . . . . . . . . . . . . . . . . . . 29, 288, 293, 471, 473 Bolund, Lars . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91 Bolyard, Chelsea M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 547 Bomsztyk, Karol . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 93 Bondanza, Attilio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295, 654 Bonetti, Ciro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 624 Bonfiglio, Serena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 795 Bonini, Chiara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88, 295, 346, 632, 654 Bonner, Melissa A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 569 Bonnet, Marie-Elise . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883

EXHIBITOR PROSPECTUS final program

Bonten, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 449 Bookay, Haddas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616 Bordignon, Claudio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276, 295, 654 Borenstein, Ronen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 719 Borisenko, Grigory G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 735 Borovjagin, Anton V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 842 Borquez-Ojeda, Oriana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32, 184 Bortolanza, Sergia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 494 Bosch, Assumpció . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 573 Bosch, Fàtima . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140 Bosch, Fatima . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762 Bosma, Piter J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138 Boss, Hannah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 27 Bossow, Sascha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 833 Bosticardo, Marita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225 Bostick, Brian P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 601 Botchkina, Galina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 266 Bottiglieri, T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139 Bottinelli, Roberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 795 Boulet, Sabrina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Boulis, Nicholas M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 699, 778, 782 Bouquet, Celine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19, 110, 113, 853 Bourdel, Alix . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 87 Bourke, Michael G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 736 Boutin, Sylvie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 67 Bovolenta, Chiara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 644 Bovolenta, Matteo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258 Bowers, William J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 18, 23, 76 Bowles, Dawn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 581 Bowles, Dawn E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 366 Boyd, Surina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 164 Boyden, Edward S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21 Boye, Sanford L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17, 71 Boye, Shannon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 71 Boye, Shannon E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17, 21 Boyer, Jean . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 477 Boyle, Kristy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 267, 461 Boztug, Kaan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612 Bradley, Allan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 15 Bradshaw, Angela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 723 Bradshaw, Angela C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380, 715 Brady, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 675 Brady, Troy L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13 Brambilla, Riccardo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315 Bramson, Jonathan L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 79 Braseth, Leah N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 518 Braseth, Leah N . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 854 Bratasz, Anna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 836 Braun, Stephen E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Bravo, Alicia J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 834 Brayman, Andrew A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 326 Breakefield, Xandra O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57, 665, 846 Bredius, Robbert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225 Breitbach, Caroline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33 Bremer, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149 Bremer, William G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 290 Brendan, Lee H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 642 Brendel, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 869 Brenner, Malcolm K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 28, 29, 30, . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 178, 288, 293, 672, 799 Brennig, Sebastian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 528 Brentjens, Renier J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32, 183, 815 Breous, Ekaterina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 543 Bresolin, Nereo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 464, 795 Breuzard, Gilles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 395 Bridges, Charles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 454 Bridges, Charles R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600 Bright, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 874 Brigida, Immacolata . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295, 345 Brioschi, Simona . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258 Brittebo, Eva . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 453 Broccoli, Vania . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 632 Broderick, Jennifer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 362 Broderick, Kate E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103, 115, 327 Brown, Brian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 613 Brown, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 391 Brown, Eric D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39 Brown, Eric J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 497 Brown, Ryan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39 Brown, Talmage T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 142 Browne, Andrew W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 201 Broxtermann, Mathias . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312 Brugiere, Pierre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Brugman, Martijn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284 Brugman, Martijn H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 619 Brum, Andrea M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 873 Brumbach, Jonathan H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 126 Brunetti-Pierri, N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879 Brunetti-Pierri, Nicola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 75, 388, 639, 677 Brunhoeber, Elaine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893 Brunicardi, F. C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 247, 564, 731, 892 Bryan, Nathan S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677 Bryant, Christopher S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250 Bu, Jie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55 Buchlis, George . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 546 Buening, Hildegard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312 Bueren, Juan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 866 Büning, Hildegard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5

Bunnell, Bruce . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314 Bunnell, Bruce A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 411 Buñuales, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 494 Burel Deschamps, Laure . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 745 Burgess, Kevin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 731 Burke, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33 Burke, James M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 35 Burke, Kathleen C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11 Burkholder, Tanya H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234, 524, 858 Burnett, John C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599, 805 Burnette, Angela D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 667 Burnnet, Erin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767 Burris, Clark A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 76 Bushman, Frederic D. . . . . . . . . . . . . . . . . . . . . . . . . . . 13, 228, 283, 308, 346 Bussadori, Giulio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308 Busuttil, Ronald W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 44, 224, 373 Butler, Greg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 488 Butler-Browne, Gillian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 789 Byrne, Barry J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 290, 371, 378, 596 Byrnes, Andrew P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 542

C

Caccavelli, Laure . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 229 Cafferata, Eduardo G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 609 Cai, Hancheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 200 Cai, Shangde . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185 Cai, Ying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 739 Calabrese, Christopher . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 449 Calcedo, Roberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 45, 164 Callejas, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140 Campbell, Jarryd M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599 Campbell, Katherine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 581 Campbell, Katherine J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155, 290 Campeau, Philippe M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677 Campion, C. A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 194 Cancelas, Jose A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 526 Candolfi, Marianela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26, 181, 475 Candotti, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 808 Candotti, Fabio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 227, 296, 678 Cannon, Paula M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11 Cantore, Alessio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 545, 613 Cantz, Tobias . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284, 528 Cao, Huibi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 872 Cao, Maohua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 429, 436, 437, 711 Cao, Ou . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 146, 763 Capalbo, Anita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 758 Capogrossi, Maurizio C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 898 Cappelli, Barbara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225 Carbonaro, Denise A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296, 414

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148

American Society of Gene & Cell Therapy

Abstract Author Index Cardoso, Rosana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349 Carillo-Carrasco, Nuria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 679 Carlson, Cathy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 852 Carmoy, Nathalie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120, 131, 745 Carnathan, Diane G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 164 Carnes, Aaron E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 572, 575 Carney, Gael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 867 Carpenter, Kevin H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 409 Carr, Bob . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 577 Carragher, Bridget . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 393 Carrer, Alessandro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 431 Carroll, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 798 Carrondo, Manuel J. T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 278, 579 Carrum, George . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288, 293 Cartier, Nathalie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19 Carvalho, Leonardo P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440 Carville, Angela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645 Casellas, Alba . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140, 762 Casey, Garrett . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 812 Cashman, Siobhan M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 447, 448 Casimir, Colin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349 Casorati, Giulia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 654 Cassani, Barbara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617 Castiello, Maria C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225 Castro, Maria G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26, 181, 475 Cataldi, Marcela P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 307 Cataliotti, Alessandro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599 Cathomen, Toni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16, 95 Cattaneo, Roberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 168, 833 Cattoglio, Claudia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 346, 523 Cavazzana-Calvo, Marina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13, 229, 617 Cawley, Niamh X. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 143 Cela, Racel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 46 Cela, Racel G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 224, 642 Cerri, Federica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315 Cersosimo, Stefania . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 133 Certo, Michael T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 90 Cerullo, Vincenzo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 177, 193, 197, . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204, 291, 538, 549, 642, 658 Cervio, E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 360 Cesana, Daniela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 298, 632 Cesaro, P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Chéradame, Hervé . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 395 Cha, Yeon-Sook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Chae, Jin-A . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Chamala, Sreedhar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250 Chamberlain, Jeffery . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553 Chamberlain, Jeffrey S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 267, 459, . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 461, 466, 468, 584, 786, 792

EXHIBITOR PROSPECTUS final program

Chamberlain, Marc . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294 Champlin, Richard E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479 Chan, Emma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 647 Chan, J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223, 697 Chan, Janet . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640 Chan, Jerry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531 Chand, Allan R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 64 Chanda, Diptiman . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 63 Chandler, Randy J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135, 679, 701 Chandra, Pallavi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 127 Chandra, Ravi K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 262 Chandrasekaran, Suma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 679 Chandrashekran, Anil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349 Chang, Chung-Che . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 293 Chang, Esther . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 536 Chang, J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 913 Chang, Yu-Han . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583 Channon, Keith M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 602 Chaouch, Soraya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 789 Chapdelaine, Pierre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 462, 790 Charan, Rakshita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 455 Charrier, Sabine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 229 Chase, Lucas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 268 Chatterjee, Anindo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 127 Chatterjee, Saswati . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 3 Chaubey, Sushma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 901 Chekmasova, Alena A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 183 Chen, Baihong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 219 Chen, Chien-Fu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 844 Chen, Christopher Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 81 Chen, Dong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605 Chen, Fang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 130 Chen, Haifeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 72 Chen, Heng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 389 Chen, Hong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 172 Chen, Jesse . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 467 Chen, Jessie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156 Chen, Jianzhu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531 Chen, Kuan-Hung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 882 Chen, Li . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 574 Chen, Liping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 326 Chen, Margaret . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 289 Chen, Shu-Jen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 625 Chen, Thomas C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 200 Chen, Vincent C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 905 Chen, Xizhe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 427 Chen, Y.-T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 136 Chen, Yifeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 548 Chen, Yi-Sen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 882

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Chen, Zhi-Ying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 9, 14 Chen, Ziping C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 422, 800 Chen, Zixuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 171, 806, 807 Chenajovsky, Yuti . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 609 Cheng, Binbin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 700 Cheng, Kun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 567 Cheng, Linzhao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 220 Cheng, Long . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 130 Cheng, Seng H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55, 557 Cheng, Woei Ping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 254 Cherel, Yan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19 Cherqui, Stephanie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 408 Chester, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606 Cheung, Roland . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 367 Chicoine, Louis G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149 Childers, Martin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 454, 458, 467, 785 Chillón, Miguel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 573 Chinnasamy, Nachimuthu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 190 Chino, Takenao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 628 Chiocca, E. A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 491 Chiocca, E. Antonio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 498 Chiocco, Matthew J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709 Chiorini, John A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 483, 870 Cho, Hanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 560 Choi, Connie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156 Choi, Hye Jin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 198 Choi, Hyo Sung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 877 Choi, Il-Kyu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 198, 202, 242, 516 Choi, Joung-Woo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 383, 721 Choi, Keum S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 722 Choi, Kyoung-ju . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 188 Choi, Kyung-Ju . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 202, 811 Choi, Min Joung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 112 Choi, Uimook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 220, 354 Choi, Vivian M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 98 Choi, Yejin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 722 Choi, Yeong “Christopher” . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296 Chong, Heung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606 Chong, Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531 Choolani, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223, 697 Choolani, Mahesh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531 Chopra, Manisha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 328 Chorny, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 323, 425 Chou, Bin-Kuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 220 Chow, Laura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33 Chowdhury, Wasim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 382 Chowdhury, Wasim H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 390 Christensen, Camilla L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 206, 210, 511 Christensen, Rikke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91

Christiansson, Lisa H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 486, 820 Christin, Anne S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 29 Christopher, Logg R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 492 Chu, Baocheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553 Chu, Kirk . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310 Chulay, Jeffrey D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 377 Chun, Kyung-Hee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 384 Chung, Samuel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 405 Chuvilin, Andrey N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 735 Ciccone, Carla . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144 Ciceri, Fabio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295 Ciré, Séverine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 552 Ciuffreda, C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 360 Clark, K. Reed . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 290, 581 Clarke, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 642 Claudio, Luigi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 847 Claudio, Pier Paolo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 847 Clay, Timothy C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 47 Clemens, Paula R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 455, 457, 580 Clevenger, Joshua R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 96 Cline, Amanda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 521 Cloer, Caryn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 890 Cloutier, Denise A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 596 Cmielewski, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 621 Coady, Tristan H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 626 Coates, Allan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 872 Cockrell, Adam S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 355, 534 Cody, James J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 507 Coffey, Matt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606 Cohen, Justus B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640 Cohen, Reuben . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 274 Colella, Pasqualina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 443 Coleman, John S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 668 Coles, Rebecca L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880 Coley, Brian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 581 Coley, Brian D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 290 Collantes, María . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Colle, Marie-Anne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19 Collie, D. David S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880 Collins, J. K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 194 Collins, Margaret H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 498 Collins, Michael T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 761 Collins, Sara A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 705 Compte-Grau, Marta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 481 Condomines, Maud . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 655 Congo, Richard T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893 Conlon, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 290 Connolly, Mary M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 727 Constantinou, Varnavas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137

149

150

American Society of Gene & Cell Therapy

Abstract Author Index Conti, Peter S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 200 Convertine, Anthony C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558 Cooper, Laurence J. N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479, 809 Cooper, Mark J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622, 776 Cornetta, Kenneth G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353, 356, 675 Coroadinha, Ana S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 278, 570 Cosset, Francois-Loic . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336 Costa, Caroline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336 Cotrim, Ana P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 74, 77, 143 Cots, Dan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 573 Cotugno, Gabriella . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 758 Coughlan, Lynda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380, 723 Couto, Linda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 69 Couto, Linda B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 885 Couture, Larry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279, 905 Couture, Sylvana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 905 Cowan, Morton J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221 Cox, Josephine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Cox, Julie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39 Craig, Anthony T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 70 Craig, Nancy L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 15 Cramer, Frederik . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 206, 210, 511 Crasta, Manuela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59 Creencia, Christopher . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 794 Crettaz, Julien . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 810 Cripe, Timothy P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 201, 498, 830 Criswell, Hugh E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2 Crombleholme, Timothy M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 830 Crome, Sarah Q. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 79 Cromley, Debra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 412 Cronin, Michelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 398, 812, 848 Crowley, Jim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439 Crowther, Carol . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 82 Croyle, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435 Cruz, Conrad R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 473 Cruz, Pedro E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 579 Cruz, Russell . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Cruz, Russell C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 29 Crystal, Ronald G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19, 161, 163, 452, 752 Cunningham, Sharon C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 409 Cunningham, Tshaka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 70 Curiel, David T. . . . . . . . . . . . . . . . . . . . . . . . . . . 36, 379, 497, 503, 834, 842 Curran, Kevin J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 815 Currier, Mark A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 201, 498 Curtin, James F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181 Custers, Jerome . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 723 Cutler-Strom, Richelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558 Cutrera, Jeffry J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 737

EXHIBITOR PROSPECTUS final program

D

da Silva Bizario, Joao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 464 Dae, Hyun Mi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 386 D’Agostino, Karla . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 50 Dai, Jihong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310 Dai, Kerong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 756 Dalkara, Deniz . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 442 Dally, Len . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Dam, Elisabeth . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 110 Dam, Tomas N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695 Dane, Allison P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 409 Daneshmand, Manijeh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33 Dang, D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879 Danièle, Nathalie F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 793 Daniel, Skuk . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 769 Danieli, P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 360 Danos, Olivier . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 87, 617 D’Antona, Giuseppe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 795 Dantzer, Jessica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353, 356 Dar, Gulam H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 404 Darocha, Sylvie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48 Darvish, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144, 772, 794 Darvish, Sheila . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 794 Dasgupta, Anindya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 814 Dasouki, Majed . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155 Dassie, Justin P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662, 663 Davaa, Enkhzaya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 896 David, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223 Davidoff, A. M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223 Davidoff, Andrew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 668 Davidson, Jesse T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 452 Davies, Kay E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 466 Davies, Lee A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 123, 727, 880 Davila, J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139 Davis, Adam S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 699 Davis, Nancy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149 Davis, Pamela B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 746 Davydova, Julia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 80, 391, 497 d’Azzo, Alessandra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 449 D’Costa, Jenice . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 422 De, Bishnu P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 161, 452, 752 De Angelis, B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 178 de Boer, Helen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617 de Cortie, Karin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 231 De Falco, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 229 De Groot, Anne S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43 De Kruijf, Marcel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 736 De Leonibus, Elvira . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 758 De Palma, Michele . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 331

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index De Ridder, Corrina M. A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 506 de Silva, Suresh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 76 De Smedt, Stefaan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 875 De Smedt, Stefaan C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 742 de Verneuil, Hubert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865 de Winter, Christa L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 153 Dean, David A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 588, 878 Deglane, Gaëlle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883 Deisseroth, Albert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85 Del Campo, Jacob . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285 del Campo, Jake . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 50 Del Carro, Ubaldo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 315 Delépine, Pascal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 745 Delai, Stefania . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315 Delalande, Anthony . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 324 Delisle, Josie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 649 Della Corte, Michele . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59 Demaster, Amanda K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 142 Demeester, Jo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 742 Demeester, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 261, 875 Denapoli, Priscila M. A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440 Denaro, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 228 Denavand, Sarkar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 847 Denby, Laura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 723 Deng, Defeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 731 Deng, Yao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 78, 172 Denisenko, Oleg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 93 Denman, Cecele J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479 Denning, Gabriela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527 Denovan-Wright, Eileen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 147 Dent, Paul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 503 DeRavin, Suk See . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 354, 860 Derecho, Ivy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279 Derendorf, Hartmut . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 256 DeRossi, Charles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 348, 650 Desai, Maya K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 18 Desmedt, Stefaan S. D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 261 Dev, Jyoti . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 208 Devine, Sean P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 509, 771 DeWeese, Theodore L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 251 Dhar, Debanjan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 489, 837 Di, Wei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 158 Di Domenico, Carmela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315 Di Girolamo, Ilaria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 316, 413 Di Natale, Paola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315 Di Paolo, Nelson C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 541 Di Pasquale, Giovanni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 483 Di Rocco, Giuliana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 898 Di Scala, Marianna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 485

Di Serio, Clelia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 345, 346 Di Stasi, Antonio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Di Stefano, Bruno . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 632 Di Vicino, Umberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59, 443, 624 Diaconu, Iulia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 193, 204, 538, 549 Diago, Oscar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 492 Diallo, Jean-Simon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39 Dias, João D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204 Diaz, Deanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 477 Diaz, Rosa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 175, 481, 606, 607, 610 Dibb, Nick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349 Dibra, Denada . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 737 Dieci, Marco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276 Diepolder, Helmut M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 289 Dietz, Allan B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 488 Dighe, N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223 Dighe, Niraja . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531 Dinauer, Mary . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 356 Dinauer, Mary C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353, 354 Dinculescu, Astra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 61 Dindot, Scott . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 75 Dines, Karla A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622 Dingli, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 208, 684 Diouf, Oumar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 DiPasquale, Betsy A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 498 Discher, Dennis E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 357 Dixon, Tracy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157, 514, 515 Dmitrieva, Nina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 199 Doering, Christopher B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 230, 527 Doglioni, Claudio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297 Doherty, Joseph E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 15 Dohmann, Hans F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440 Dolphin, Colin T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 716 Dolter, Karen E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162, 740 Doms, Robert W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 801 Donate, Amy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 107 Donate, Amy L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 174 Dondero, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218 Dong, Biao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310 Dong, John Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 504, 643 Dong, Xiaoyan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 376 Donnelly, Eleanor M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 780 Donovan, Kathleen A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218 Donsante, Anthony . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 24 Dorer, Dominik E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 387, 641 Doria, Monica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59, 64 Dorko, K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139 Doronin, Konstantin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 496 Doroudchi, Mehdi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21 Dorvault, Christine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 834 Dotti, G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30, 178

151

152

American Society of Gene & Cell Therapy

Abstract Author Index Dotti, Gianpietro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 28, 288, 293, 472, 672 Douglas, Joanne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 834 Douglas, Joanne T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 507 Dowdy, Sean C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 488 Down, Julian D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 228 Downey, Harre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 546 Downing, Brandon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353 Doyon, Yannick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 98 Dragneva, Galina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159 Drescher, Charles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 265 Drescher, Max . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 843, 849 Dropulic, Boro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527 Du, Shi-Suo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 211 Duan, Cathleen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 872 Duan, Dongsheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 150, 466, . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 587, 601, 707, 876 Duan, Jiachuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433 Duan, Xunbao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310 Dubrot, Juan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Duckworth, Katherine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 779 Dudley, M. E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31 Dudley, Mark E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25 Duffy, Angela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 424 Duffy, Aoife . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 427 Dugin, Daniel P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Dumont, Nicolas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 769 Dunbar, Cynthia E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 96 Dunfee, Ashley E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 96 Dunn, Diane M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 152 Dunn, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181 Dunphy, Nina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279 Durett, April . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Duskey, Jason . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 594 Dwyer, Markryan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645 Dylewski, Dawna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 487

E

Eagle, Michelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314, 411 Eaton, Laura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910 Ebtekar, Massoumeh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871 Edelman, Gerald . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 292 Edmonson, Shyrie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 548 Edmonson, Shyrie E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308 Edwards, Paul C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 104 Eerola, Kim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 145 Eghtesad, Saman . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 580 Egorova, Anna A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 750 Ehrhardt, Anja . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 92, 141, 385, 636, 712 Eiden, Maribeth V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 696

EXHIBITOR PROSPECTUS final program

Eiermann, Jessica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314, 411 Einfeld, David A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 504 El Andaloussi, Samir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 556 Elgstrand Wettergren, Erika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 239 Elhalel, Michal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616 Eliopoulos, Aristides G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 486 Ellis, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 300, 691 El-Sherbini, Yasser M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 732 Ely, Abdullah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 82 Emborg, Marina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 779 Eming, Sabine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5 Emmanouil, Kalyopy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 405 Enbersen, Johan F. J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 126 Encinas, Tony . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 274 Endo, Masayuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320, 344, 676 Engelhardt, John F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4, 620, 698 Engler, Tatjana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 637 Ennis, Matthew K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 684 Enoto, Kaoru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 591 Enríquez de Salamanca, . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Rafael 42 Enright, Annette . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 270 Epperly, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157, 514, 515 Epperly, Michael W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 580 Erbacher, Patrick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883 Erbs, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204 Erez, Ayelet . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677 Erger, Kirsten . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 371 Erger, Kirsten E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 378 Eriksen, Jens . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 101 Eriksson, Elof . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237 Eriksson, Minna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204 Erker, Laura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910 Ertl, Hildegund C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 389 Escutenaire, Sophie . . . . . . . . . . . . . . . . . . 177, 197, 204, 291, 538, 549, 658 Esfandiarifard, Saghi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 772, 794 Essand, Magnus . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 501, 828 Esser, Knud . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312 Evans, Claire F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162, 740 Everhart, Drew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17 Ewert, Kai K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 393

F

Fabris, Marina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258, 666 Fadel, Hind . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 685 Fagone, Paolo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 861 Fakhfakh, Raouia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 784 Falgowski, Kerry A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 897

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Falk, Darin J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 596 Falls, Theresa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39 Falzarano, Sofia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258, 666 Fan, Bai . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 510 Fan, Zheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 328 Farag, Jacqueline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 454 Fargnoli, Anthony . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600 Farini, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460, 464, 795 Farley, Raymond . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 873 Fassas, Athanasios . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137 Fast, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Favaro, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 614 Federici, Thais . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 699, 778, 782 Federoff, Howard J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 23 Federspiel, Mark J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 490, 499 Feduska, Joseph M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 629 Feijen, Jan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 126 Feldman, Steven A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25, 190 Fellenberg, Kurt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 641 Fender, Pascal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 843 Fenelon, Gilles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Feng, Jiazuo H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 744 Ferguson, Sherise D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 902 Ferlini, Alessandra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258, 666 Fernandes, Julio C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 756 Fernandez, Christian A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 117, 594 Ferrand, Maxime . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48 Ferrari, Giuliana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319, 523 Ferro, Leda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 911 Ferry, Nicolas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138 Fewell, Jason G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893 Fidler, Jonathan A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 557 Filipczyk, Adam . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284 Filippova, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 444 Finegold, Milton . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910 Finn, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 461, 786 Finn, Jonathan D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43, 551, 614, 615 Finney, Helene . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845 Fire, Andrew Z. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14 Firozi, Pervez . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 574 Fischbeck, Kenneth H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 235 Fischer, Alain . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 229 Fishbein, Ariel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 425 Fishbein, Ilia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 323, 425 Fisher, John T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620 Fisher, Paul B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 503, 847 Fisher-Perkins, Jeanne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 411 Fisk, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 697 Fisk, N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223

Flaat, Mette . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314, 411 Flageul, Maude . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138 Flake, Alan W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320, 344, 676 Flanagan, Mike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 105 Flanigan, Kevin M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 152, 155, 789 Flannery, John G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 442 Fleischhauer, Katharina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295 Fleischmann, Bernd . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693 Fletcher, Anita M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 776 Florence, Julaine M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 152 Flotte, Terence R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 51, 53 Flotte, Terrence . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 444 Flotte, Terry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58 Foley, Jason . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659 Follenzi, Antonia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 134 Foloppe, Johann . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204 Fong, Mei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 674 Fons, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 289 Fontanellas, Antonio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Forde, Patrick F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 736 Forman, Stephen J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 555 Fortes, Puri . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 249, 889 Foster, Aaron E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 469, 816 Foster, Edmund . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451 Foulad, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181, 475 Fowler, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 341 Fowler, Daniel H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 333, 509, 659 Fracchia, Sergio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276 Fraix, Aurore . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 131 Franicola, Darcy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 514 Frank, Michael M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638 Frankel, Timothy L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 190 Fransson, Moa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 176, 453 Frattini, Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32 Frecha, Cecilia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336 Frelin, Lars . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 111, 289 Frenkel, Niza . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 719 Frey, Brigitte M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 855 Frey, Felix J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 855 Friedman, Geoffrey . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11 Froehner, Stanely C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 584 Froelich, Steven . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 689 Fu, Yang-Xin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 517 Fujita, Yuriko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Fujiwara, Hiroshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 652 Fukudome, Takayasu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 585 Fukunaga, Yoshitaka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 680 Furuya, Aki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 824 Fusaki, Noemi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303, 634 Futaki, Shiroh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 246, 253, 401

153

154

American Society of Gene & Cell Therapy

Abstract Author Index G

Gabant, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 740 Gabriel, I. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Gabriel, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 286, 347 Gahan, Cormac G. M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 398 Gahl, William A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144 Gaj, Thom . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 94 Galanis, Evanthia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 170, 490, 499 Galivo, Feorillo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 175, 481, 607, 610 Galla, Melanie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284, 302, 304, 619 Galletti, Margherita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 523 Galloway, Gloria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 290, 581 Galton, Bruce . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218 Galun, Eithan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16, 616 Galvan, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12, 813 Galy, Anne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48, 225, 229, 533, 552 Gamble, Lena J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 842 Ganea, Doina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 859 Gangadharan, Bagirath . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 230, 527 Gangeswaran, Rathi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605 Gangopadhyay, Anu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659 Ganguli, Munia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 127, 132 Ganly, Sandra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439 Gao, Dongling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605 Gao, Guangping . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 362, 433, 450, 671, 759 Gao, Ran . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242 Gao, Yongxin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 219, 518 Garabedian, Elizabeth . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296 Garcia, Louis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460 Garcia, Luis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 154, 464, 795 Garcia-Aragoncillo, Eva . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 494 Gardiner, David F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Gardmark, Truls . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 176 Gardner, Matthew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 608 Gardner, Rebecca A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 618 Garg, Harsha K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677 Garg, Nupur . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796 Gargiulo, Annagiusi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 702 Garibov, Mikhail . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 90, 529 Gatti, Alessandra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276 Gautney, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527 Gavrikova, Tatyana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 497 Gay-Antaki, Carlos . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 317 Gaymalov, Zagit . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 817 Ged, Cécile . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865 Gedeon, Christopher R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622 Gee, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30 Gee, Adrian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Gee, Adrian P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 293

EXHIBITOR PROSPECTUS final program

Gee, Samantha W. Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 700 Geguchadze, Ramaz . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 371, 378 Gehl, Julie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 101, 106, 109 Geisert, Eldon E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 441 Genovese, Pietro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10, 88, 286, 632, 654 Gentile, Antonietta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 898 Gentner, Bernhard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 331, 337 Gerdemann, Ulrike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 29, 473, 799 Gerhardt, Greg A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709 Germain, Sean A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 596 Gerritsen, Winald R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 40, 502 Gersbach, Charles A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 94 Geurts, Jeroen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 559, 627 Ghaffari, Seyed Hamidolah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871 Ghavamzadeh, Ardeshir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871 Ghazi, Alexia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 470, 471 Ghio, Domenico . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295 Giacca, Mauro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 311, 423, 431 Giachelli, Cecilia M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 730 Giamarchi, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 745 Giangrande, Paloma H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662, 663 Giannakopoulos, Aristeidis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 729 Gianni-Barrera, Roberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 430, 597 Giannoni, Francesca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 522 Gibbons, Hillary N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 809 Gibly, Romie F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 764 Gibson, K. M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139 Gibson, Soojung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 417 Giddings, Angela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 881 Giedlin, Marty . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783, 798 Giffear, Mary . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 477 Giladi, Hilla . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16 Gilbert, Rénald . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463 Gilbert, Richard A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 116 Gill, Deborah R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 123, 727, 739, 880 Gill, Steven . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394 Gillet, Danielle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120 Gillet-Legrand, Beatrix . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 228 Gilmour, Jill . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Gimble, Jeffrey . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314 Gimble, Jeffry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 105 Ginns, Edward I . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 418 Giorgio, Todd D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 592 Giovannoni, Roberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59 Giralt, Sergio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 809 Giridhara, Jayandharan R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 68 Gissel, Hanne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 106 Giunti, Massimo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59 Giustacchini, Alice . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 337

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Gjetting, Torben . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 206, 511 Gkirtzimanaki, Katerina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 486 Glazer, Peter M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 50, 285 Gleich, Bernhard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693 Glimm, Hanno . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 347, 612 Glorioso, Joseph C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640, 781 Glowacki, Julie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157 Gnatenko, Dmitri . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372 Gnecchi, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 360 Godbehere, Sarah S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638, 651 Goepfert, Christine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 855 Goerlich, Corbin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 243 Goetzmann, Jason . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58 Goff, Julie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 514 Goins, William F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640, 781 Gokhale, Abhay . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157 Goldschlager, Gregg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38 Goltzmann, Haim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616 Gomero, Elida . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 449 Gomez Almarza, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5 González-Aseguinolaza, Gloria . . . . . . . . . . . . . . . . . . . . . . . . . . . 485, 708, 810 Gonzalez-Aparicio, Manuela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 810 Gonzalez-Rojas, Jovanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 889 Good, Alec S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 125 Goodheart, Michael J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620 Gopal, Kapil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 454 Gopal, Vijaya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 404 Gorbatyuk, Marina S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 446 Gorbatyuk, Oleg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 378 Gordley, Russell M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 94 Gordon, Cath M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880 Goss, James R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 781 Gothelf, Anita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 101 Gotoh, Akinobu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 216 Gotoh, Mitsukazu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 419 Gotschalk, Stephen M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 816 Gottfried, Lynn F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 878 Gottschalk, Stephen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 179, 293, 471, 656 Gottschalk, Stephen M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Gottschalk, Steven . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 470 Goudenege, Sebastien . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 769 Gould, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 609 Gräslund, Torbjörn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 239 Gröhn, Olli . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159 Gracey, Lia E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14 Graham, John G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 770 Grande, Alexis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 523 Grandi, Paola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640 Grant, Rebecca L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 45

Grant, Steven . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 503 Grant, Trobridge D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 864 Grassman, Elke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 526 Gray, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 222, 449, 733 Gray, John T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 354, 668, 860, 861 Gray, Steven . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 581, 782 Gray, Steven J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2 Greco, Adelaide . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 847 Green, Matthew D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 122, 125 Greenberg, Philip D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88, 654 Greenberg, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 646 Greenberger, Benjamin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 515 Greenberger, Joel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157, 514, 515 Greene, Michael R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 861 Gregory, Philip . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 798 Gregory, Philip D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10, 11, 88, 98, . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 286, 415, 618, 632, 650, 654, 801 Greiser, Udo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439 Grez, Manuel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 869 Grieger, Joshua C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 160 Griener, Dale . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285 Griesenbach, Uta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 873 Griffith, Graziella . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 154 Griffiths, Duncan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 577 Grilley, Bambi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Grimm, Brad . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 571 Grimm, Dirk . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 660, 886 Grinstaff, Mark W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 392, 744 Gritti, Angela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 316, 413, 632 Grompe, Markus . . . . . . . . . . . . . . . . . . . . . . . . . 99, 306, 681, 682, 760, 910 Gross, David-Alexandre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48 Grossardt, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 833 Grossman, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 471 Grove, N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879 Grove, Nathan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 75, 388 Grove, Nathan C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 639 Grover, Rajat . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 63 Gruber, Harry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 492, 831 Gruenert, D. C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 913 Gu, Shuo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 54 Gu, Yachuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 566 Guégan, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 395 Guérin, Claude . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463 Gualandi, Francesca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258, 666 Guan, Jie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 78, 114 Gubinelli, Francesco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 644 Guedan Carrio, Sonia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 496 Guerin, Karen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 442 Gui, Tong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 534

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156

American Society of Gene & Cell Therapy

Abstract Author Index Gunset, Gertrude . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 655 Gunther, William R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 595 Guo, Jianfeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 254 Guo, Siqi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 107 Gupta, Sanjeev . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 134 Gürke, Lorenz . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 430, 597 Gurram, Sweta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221 Gurruchaga, Jean-Marc . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Guse, Kilian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204 Gussing, Fredrik . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 239, 350 Guttridge, Denis C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 635 Guyonnet-Duperat, Véronique . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865

H

Ha, Yoon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 260 Haas, Andrew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33 Haavisto, Elina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 177, 193, 197, 538, 658 Habib, Bianca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603 Hacein-Bey Abina, Salima . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 229 Hacein-Bey-Abina, Salima . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13 Hacker, Ulrich . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 664, 887 Hackett, Neil R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19, 452 Hackett, Perry B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 325, 728, 734 Hackl, Florian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237 Hadac, Elizabeth . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 604 Haddock, Courtney . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 213, 564, 578 Haga, Kazunori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334 Haghighatgoo, Arman . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 794 Hahn, Woong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Hai, Li . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 484 Hai-Ying, Zeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73 Hajjar, Roger J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 601 Hakkarainen, Tanja . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204 Halbert, Christine L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 623 Hall, Geoff . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606 Halle, Stephan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284 Hallek, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5, 312 Hamada, Hiromi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 554 Hamada, Katsuyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 216, 493, 825 Hamakawa, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 493 Hamilton, Shari R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308 Han, Sang W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440 Han, Seung Ryul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 560 Han, Sung Yeol . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 112 Han, Tae Hee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85 Han, Yu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 902 Hannah, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 534 Hannaman, Drew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162, 740 Hans, Skovgaard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 206

EXHIBITOR PROSPECTUS final program

Hansel, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139 Hansford, Loen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 498 Hans-Peter, Kiem . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 864 Hantraye, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Harada, Yui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 824 Haralambieva, Iana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 170 Harashima, Hideyoshi . . . . . . . . . . 97, 240, 246, 253, 401, 591, 743, 748, 894 Harding, Cary . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 681 Hargrove, Phillip W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 338, 339, 525 Harms, Ashley S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 773 Harper, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 158 Harrington, Kevin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606 Harris, Drea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 477 Harris, Raymond D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36 Harrison, Frank . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 408 Harrison, Patrick T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 705 Harrow, Faith . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 521 Hart, Brit J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 800 Hart, Stephen L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394, 747 Hartman, Zachary C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638 Harty, C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 194 Harvey, Brandon K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709 Hasegawa, Mamoru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303, 634, 824, 873 Haseley, Amy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 836 Hashimoto, Takuya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 891 Haskins, Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 758 Haskins, Mark E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 598, 759 Hatakeyama, Hiroto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 246, 253, 894 Hatano, Norikazu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678 Hatefi, Arash . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 329 Hattori, Kayoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 862 Hauer, Julia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617 Haugwitz, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 674 Haurigot, Virginia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 546, 885 Hausl, Martin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 636 Hausl, Martin A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 385, 712 Hauswirth, Wiliam W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 417 Hauswirth, William W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17, 21, 61, 71, 446 Hawkins, Troy B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353, 356 Hawley, Robert G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 342, 690 Hawley, Teresa S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 342, 690 Haworth, Kevin G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11 Hay, John G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 713 Hayashi, Hiroki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 774 Hayashi, Yasuhiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 743 Hayes, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155 He, Bo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 683 He, Cheng-Yi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 9 He, Ran . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 362, 450, 671

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index He, Shuhui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 130 He, Xianghui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 186, 566 Hearing, Patrick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372, 573 Hebel, Henry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 576, 739 Heberer, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 430, 597 Heckl, Dirk . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 318 Heemskerk, Hans A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 153 Hegadorn, Carol . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767 Hegge, Julia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 160 Heinonen, Suvi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159 Heinz, Niels . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 302, 619 Heldermon, Coy D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 768 Helfer, Brooke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 271 Heller, Loree . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 116, 823 Heller, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 107, 174, 823 Helminen, Andreas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 197 Hemmi, Silvio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 193 Hemminki, Akseli . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 177, 193, 197, . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204, 291, 538, 549, 657, 658, 843 Hemminki, Otto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 193 Hensel, Jonathan A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 63 Heo, Kyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 386 Hermonat, Paul L. . . . . . . . . . . . . . . . . . . . . . . . . . . . 429, 436, 437, 711, 819 Hernandez-Alcoceba, . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .Ruben 494, 810 Hershfield, Michael S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296, 678 Hervás-Stubbs, Sandra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Hervas-Stubbs, Sandra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 810 Herzog, Erik . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 768 Herzog, Roland W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 309, 363, 539, 540 Heslop, Helen E. . . . . . . . . . . . 28, 29, 30, 178, 288, 293, 470, 471, 473, 799 Hewitt, Stephen M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659 Hiatt, P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879 Hibbitt, Olivia C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 602 Hicks, Martin J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 452, 752 Hicks, Martin L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 161 Hickstein, Dennis D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234, 524, 858 Higashimoto, Tomoyasu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 526 Higgins, Luke J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 214 Higgs, Jerome T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 63 High, Katherine A. . . . . . . . . . . . 8, 43, 69, 308, 546, 548, 614, 615, 630, 673 Hikichi, Mina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 34, 840 Hinderer, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 412 Hintze, Thomas H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423 Hinzman, Jason . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709 Hiraoka, Kei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334, 492, 831 Hiroshima, Kenzo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851 Hirsch, Matthew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2 Hiscott, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39

Ho, David D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Ho, Seong-Hyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Hodge, Gregory . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 168 Hodgson, Clague P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 166, 572, 575, 738 Hoel, Brian D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 504 Hoesel, Marianna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312 Hoffer, Barry J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709 Hoffman, Brad E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 539 Hoffman, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56 Hoffmann, Ellen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 886 Hoffmann, Victoria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135 Hofmann, Lawrence V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 214 Hoheisel, Jörg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 641 Hojman, Pernille . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 101, 106 Hole, Patrick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 577 Hollis, Roger . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 522 Holmes, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 798 Holmes, Michael C. . . . . . . 10, 11, 88, 98, 286, 415, 618, 632, 650, 654, 801 Holt, Nathalia G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11 Holtrup, Frank . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 641 Holtz, R. B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 213 Hondmann, Dirk H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 332 Hong, Seung Hwa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 112 Hong, Youngtae . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Hoogstraten-Miller, Shelley . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144 Horowitz, Eric D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 403 Horsager, Alan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21 Horton, Terzah M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 473 Horvath, Staci . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 196 Hosoyama, Sachiko O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151 Hoti, Naser Uddin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 382 Hotta, Akitsu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 691 Hou, Linan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 186 Hough, Christine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767 Hovhannisyan, Ruben . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 625 Howard, Candace . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 847 Howard, Douglas B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709 Howard, Linda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 270 Howard, Jr, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 328 Hricak, Hedvig . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185 Hsieh, Chia-Ling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 844 Hsieh, Matthew M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 352 Hsieh, Ming Kun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 173 Hsu, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279, 905 Hu, Biliang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 686 Hu, Chuhong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 44, 224, 373 Hu, Chunling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 685 Hu, Chunyan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433 Hu, Huimin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 449

157

158

American Society of Gene & Cell Therapy

Abstract Author Index Hu, Jim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 872 Hu, Yu-Chen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583 Hua, Yimin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55 Huang, Chienjin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 173 Huang, Jing-Hua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 202, 811 Huang, Yaoxing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Huard, Johnny . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 456, 629, 635 Hughes, Jeffrey A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 256, 775 Hui, Daniel J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43, 546 Huizing, Marjan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144 Huls, Mary H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479 Humala, Nelson . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38 Humblet-Baron, Stephanie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 343, 529 Humeau, Laurent . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 41 Humeau, Laurent M. . . . . . . . . . . . . . . . . . . . . . 165, 422, 797, 800, 802, 803 Humphries, Margaret . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 444 Humphries, R. Keith . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 631 Hung, Gene . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55, 557 Hung, Mien-Chie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 844 Hunter, Michael J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 524 Hurley, Arlene . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Hurton, Lenka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479 Huston, Marshall W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 530 Hutson, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451 Huye, Leslie E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 472 Huyn, Steve . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 714 Hwang, Esther . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796 Hwang, Gloria L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 214 Hwang, Mark E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 119, 595 Hwang, T. C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 876 Hwang, Taeho . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33 Hyatt, Susannah L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622 Hyde, Stephen C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 123, 727, 739, 880 Hyland, Kendra A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 868 Hynes, Sean O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439 Hyvärinen, Anna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 212, 512

I

Iacobelli, Nicholas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 615 Iankov, Ianko D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 170, 490 Ichikawa, Tomohiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 824 Iida, Akihiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303 Ikeda, Alan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296 Ikeda, Hiroaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 652 Ikeda, Yasuhiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599, 633, 688 Ikoma, Akihiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 862 Ilett, Elizabeth . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 481 Ilies, Marc A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 757 Inagaki, Akihito . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334, 492, 831

EXHIBITOR PROSPECTUS final program

Ino, Yasushi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 832, 839 Inoue, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 192, 263 Inoue, Makoto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303, 634, 824, 873 Inoue, Tomoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263, 899 Iodice, Carolina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 443 Ironside, Christina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 864 Irrazábal, Thergiory . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 766 Isaksson, Christina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 350 Ise, Kazuya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 419 Ishida, Eri . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 513 Ishihara, Kazuhiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 743 Ishii, Takehiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 118 Ishii, Yuko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 102 Ishiwata, Akira . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 421, 544 Ismail Nounou, Mohamed M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 405 Itaka, Keiji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 118, 891 Ito, Erika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 253 Ito, Mikako . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 585 Ito, Takayuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 428 Ito, Tomoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 749, 825 Ivanovska, Irena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 357 Ivics, Zoltan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 302 Iwamoto, Mari . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 350 Iwata-Kajihara, Tomoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 480 Izmiryan, Araksya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 87 Izsvak, Zsuzsanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 302

J

Jaffrès, Paul-Alain . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 131, 745 Jagadeesh, Jayashree . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296 James, Scott E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558 Jan, Caroline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Janda, Kim D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 161, 452, 752 Janicki, Hanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312 Janik, J. E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31 Janjic, Jelena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 271 Jarraya, Bechir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Jasti, Aparna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353, 675 Jay, Chris . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144 Jay, Chris M. . . . . . . . . . . . . . . . . . . . 148, 245, 247, 252, 532, 564, 578, 586 Jayandharan, Giridhara R. . . . . . . . . . . . . . . . . . . 363, 369, 370, 700, 704, 706 Jegga, Anil G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 830 Jenkins, Kristy M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 79 Jensen, Bryan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910 Jensen, Michael C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 618 Jensen, Thomas G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 787 Jeong, Hwa Yeon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128, 406, 754 Jeong, Jae-Gyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Jeong, Jin-Sook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 560

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Jeong, Myung Ho . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 399, 751, 900, 904 Jian, Zhou . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73 Jiang, Guozhong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605 Jiang, Haixiang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638 Jiang, Jiangjang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 420 Jiang, Shuguang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 798 Jiang, Xuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 124, 321 Jiang, Ziyue . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 850 Jibly, Tamar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616 Jikimoto, Takumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167 Jimenez, Veronica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762 Jin, Jiankang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 243 Jin, Xiangyang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 414 Jno-Charles, Odella . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 625 Joensuu, Tiimo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 538 Joensuu, Timo . . . . . . . . . . . . . . . . . . . . . . . . . . 177, 193, 197, 291, 549, 658 Joergensen, Mette . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 109 Joh, Ju . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 163 Johana, N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 697 John von Freyend, Miriam . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312 Johnson, Adam J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 330 Johnson, R. Paul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645 Johnson, Shane . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 576 Johnston, Jennifer M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 230 Johnston, Julie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 625 Jollet, Arnaud . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 87, 617 Jolly, Douglas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 492, 831 Jones, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 416 Jonker, Derek . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33 Jonlin, Erica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137 Joung, J. Keith . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 95 Judge, Luke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 466 Judge, Luke M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 459 Juliano, Rudy L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 62 June, Carl H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 341, 798, 801, 809 Jung, Jae Wook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 877 Jung, Kyeong Cheon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 406 Jung, Maria C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 289 Junghans, R. P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 827 Junghans, Richard P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 27, 182 Jungmann, Andreas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 241

K

Kabanov, Alexander V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 817 Kabashima, Kenji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 862 Kabir, Farruk M. L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 379 Kacherovsky, Nataly A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 730 Kaeppel, Christine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138, 305 Kafri, Tal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 355, 534

Kaikkonen, Minna U. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 209 Kaja, Aparna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12 Kakeda, Minoru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236 Kakimoto, Shinji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 397 Kaler, Stephen G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 24 Kalsi, Jasmine K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 700 Kaluza, Karen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 175 Kamau, Erick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645 Kamen, Amine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 361 Kamijima, Shuichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334 Kamimura, Kenya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 322 Kaminsky, Paul M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 698 Kaminsky, Stephen M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 161, 452, 752 Kamiya, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 97, 240 Kanai, Ryuichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 608, 829 Kanda, Genki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 240 Kaneda, Yasufumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 187, 628, 774 Kanerva, Anna . . . . . . . . . . . . . . . . . . . . . . . . . . 193, 204, 291, 538, 549, 658 Kang, Bum-Yong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 437 Kang, Chang-Yuil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Kang, Elizabeth M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 354 Kangasniemi, Lotta . . . . . . . . . . . . . . . . . . . . . . . . . . 177, 291, 538, 549, 658 Kano, Mitsunobu R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 891 Kao, Chinghai . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334 Kaplan, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 498 Kapoor, Neena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296 Kapp, Kerstin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 189, 822 Kappel, C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223 Karandish, Sara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 342 Karioja-Kallio, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 538 Karlen, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221 Karlen, Andrea D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 232 Karli, Eerika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 658 Karlsson, Hannah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 83 Karpati, George . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463 Karponi, Garyfalia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137 Kasahara, Nori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 200 Kasahara, Noriyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334, 492, 500, 831 Kasahara, Yuko N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151, 582 Kaskenpää, Nina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159 Kassim, Sadik H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1, 412 Kasuda, Shogo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 903 Kataoka, Kazunori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 118, 891 Katayama, Kazufumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 537 Katayama, Takane . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167 Kato, Junko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 903 Katoono, Ryo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 401 Katovich, Michael J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 518 Katus, Hugo A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 241, 432

159

160

American Society of Gene & Cell Therapy

Abstract Author Index Katz, Michael G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600 Katzenberg, Regina H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 214 Kaul, Sunil C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242, 841 Kaur, Balveen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 199, 836 Kaushal, Shalesh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 444 Kauss, Ariel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 3 Kaverina, Natalya V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 835 Kawabata, Kenji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 537 Kawabata, Masato . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167 Kawakami, Yutaka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 480 Kawamura, Kiyoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851 Kawamura, Nobuaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678 Kawanishi, Yu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 513 Kawano, Hirotaka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263, 899 Kawasaki, Yoshiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 500 Kay, Jennifer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 794 Kay, Mark A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 9, 14, 54, 306, 682, 760 Kaynor, G. Campbell . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 517 Kaza, Niroop . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 63 Kazuki, Yasuhiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236 Keating, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30 Kehry, Marilyn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 550 Keiser, Nicholas W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4, 698 Kemmerrer, Stephen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103 Kemmerrer, Steve . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 327 Kennedy-Nasser, Alana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Kerkard, Sid P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 478 Kerns, Hannah M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 343 Kerr, Douglas A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908 Keshavjee, Shaf . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 872 Keswani, Rahul K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 119 Kettlun, Claudia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12 Kew, Yvonne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 471 Khan, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648 Khan, Amir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103, 115, 477 Khan, Junaid A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 429, 436, 437 Khanduri, Richa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 132 Khargharia, Sanjib . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 117, 594 Khazi, Fayaz . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310 Khim, Jit . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 529 Khokher, Zeshan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 772, 794 Kholová, Ivana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159 Khoury, Maroun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531 Kichler, Antoine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 67, 400, 791 Kida, Miyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678 Kidokoro, Minoru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 34, 840 Kiem, Hans-Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294, 631, 909 Kim, Dong-Eun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 895 Kim, Dongho . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 257

EXHIBITOR PROSPECTUS final program

Kim, Dong-Sik . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Kim, Eun Ok . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 386 Kim, Hong Sung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 406, 754 Kim, Hyun-Ki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 896 Kim, In-Hoo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 384, 386 Kim, Inwook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242 Kim, Ji-seong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 188 Kim, Jong-Mook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Kim, Joo-Hang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 202, 257 Kim, Jung Seok . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128, 406, 754 Kim, Kenneth . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11 Kim, Kenneth H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36, 503 Kim, Keun Sik . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 406, 754 Kim, KeungNyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 260 Kim, Keun-Sik . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128 Kim, Kwang-il . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 805 Kim, Kyungbo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 895 Kim, Min jung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 560 Kim, Nahyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 407, 877 Kim, Nam Hee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 722 Kim, Pyung-Hwan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 86 Kim, S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 913 Kim, So Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 257 Kim, Sue Kyung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 386 Kim, Sujeong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Kim, Sung Wan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 86, 126 Kim, Sunyoung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Kim, Tae-il . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 86 Kim, Yong Oock . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 516 Kim, Yong Sook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 399, 751, 900, 904 Kim, Yoon-Sang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 338, 339, 861 Kim, Yun-Hee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 386 Kimball, Kristopher J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 834 Kimmelman, Jonathan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 779 Kimura, En . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468, 788 Kimura, Takahiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334 Kinder, Clifford . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 646 King, Gwendalyn D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26 King, Wendy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155 Kingsman, Susan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Kinoh, Hiroaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 824 Kinoh, Hiromi H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151, 582 Kinoshita, Shohiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167 Kirn, David H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33, 39 Kiselev, Anton V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 750 Kisling, Lisa A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 759 Klanke, Chuck . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 830 Klein, Christoph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612 Kleinschmidt, Jürgen A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 241, 432

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Kleuss, Christiane . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 189 Kliem, Michele A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 778 Klimczak, Ryan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 299 Klimczak, Ryan R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 442 Klump, Hannes . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284 Kmiec, Eric B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 569, 897 Knoblauch, Christine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279 Knolle, Percy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312 Knop, David R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 377, 571 Kobasa, Darwyn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169 Kobayashi, Hiroshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851 Kobayashi, Ryouji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678 Kobinger, G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648 Kobinger, Gary . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 64 Kobinger, Gary P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169, 724 Koch, Walter J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600 Kochanek, Stefan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 637 Kochenderfer, J. N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31 Kocis, Keith . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 328 Koeberl, Dwight D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 47, 136, 142 Koefoed, Mette . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 787 Koerber, James T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 8 Kofod-Olsen, Emil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91 Kohn, Donald B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296, 414, 522 Koibuchi, Nobutaka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 774 Koide, tatsuya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468 Koike, Kazuo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 890 Kok, Cindy Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 409 Kokkinou, Dimitra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 729 Koldej, Rachel M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 867 Kole, Ryszard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56 Kolstad, Kathleen D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 442 Komanduri, Paavani . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 515 Komatsu, Norio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263 Kondratenko, Irina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612 Kong, Seogkyoung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 182 Kong, Weiming . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 859 Koniar, Brenda L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 728 Konieczny, Patryk . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 786 Konno, Tomohiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 743 Konstantinova, Pavlina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 249, 255 Kontermann, Roland . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 387 Koorneef, Annemart . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 255 Koornneef, Annemart . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 249 Kopp, Lisa M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479 Kopyov, Oleg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 912 Kornegay, Joe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 454, 467, 785 Kornegay, Joe N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 790 Kornegay, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 458

Korokhov, Nikolay . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 165, 422, 797, 800 Korpisalo, Petra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159 Koski, Anniina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 177 Kota, Janaiah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155 Kotchey, Nicole M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 7, 703 Kothary, Nishita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 214 Kottke, Timothy . . . . . . . . . . . . . . . . . 175, 481, 496, 606, 607, 610, 611, 653 Kouyama, Emi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 537 Kowalczyk, Tomasz H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622, 776 Koyama, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 891 Koyama, Taro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237 Koyama, Yoshiyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 749, 825 Kozink, Daniel M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 142 Kozyak, Benjamin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 454 Kraaij, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 506 Krahn, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460 Krainer, Adrian R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55 Krance, Robert A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Krasnykh, Victor . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 725 Krause, Anja . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 163 Krause, Johanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 318 Krejci, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 585 Kremer, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 570 Kremer, Eric J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 573 Kreppel, Florian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 637 Krichevsky, Anna M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 665 Kroeger, Kurt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181 Kroeger, Kurt M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26, 475 Kron, Matthias W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 637 Ksienzyk, Jan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 432 Kuang, Chenzhong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 332 Kuball, Jurgen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88, 654 Kubo, Shuji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 500 Kudrolli, Tarana A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 390 Kudva, Yogish . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 633 Kuehle, Johannes . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284 Kühlke, Klaus . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612 Kuhn, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 275, 292, 335, 532 Kulkeaw, Kasem . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263 Kumagai, Izumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640 Kumar, Padmasini . . . . . . . . . . . . . . . . . . . 245, 275, 292, 335, 487, 532, 564 Kumar, Priti . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285, 407, 877 Kumar, Sanjay . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 906 Kumar, Sanjeev . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250 Kumar-Singh, Rajendra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 447, 448 Kume, Akihiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 207, 421, 428, 544, 554 Kupiec, Martin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16 Kurachi, Shinnosuke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 537 Kurita, Ryo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263, 899

161

162

American Society of Gene & Cell Therapy

Abstract Author Index Kurre, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 692 Kurtova, Antonina V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 469 Kusano, Eiji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 428 Kushmeric, Martin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553 Kustikova, Olga S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 619 Kuwahara, Hiroya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 259 Kwon, Jin Sook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 399, 751, 900, 904 Kwon, Oh Seok . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 112 Kyrtatos, Panos . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394

L

Lab, McIvor . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221 Labelle, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767 Labruna, Giuseppe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435 Lachmann, Nico . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 528 Laflamme, Karina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 521 Lagadinou, Eleni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 340 Lago, Natalia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451 Lagresle-Peyrou, Chantal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617 Laguzzi Larcher, Fernando . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5 Lai, Frances . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39 Lai, Yi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 150, 466, 587 Laidinen, Svetlana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159 Lainé, Céline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120 Laing, Jennifer M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 273, 717 Laitinen, Anna E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 669 Laitinen, Lauri M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 669 Lakshmipathy, Uma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 268 Lalanne, Magalie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865 LaManna, Caroline M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 744 Lamarre, Yann . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 769 Lamm, Don . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 35 Lamrissi-Garcia, Isabelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865 Landry, Breanne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 129, 755 Langan, Russell C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25 Langel, Ülo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 556 Lapteva, Natalia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 474 Larcher, Fernando . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 158, 351 Larochelle, Andre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 96 Larochelle, Nancy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463 Lattanzi, Annalisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 413 Lau, Ashley . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 306 Laure, Aurelian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 273 Laurema, Anniina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 765 Lavasani, Mitra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 629, 635 Lavasanifar, Afsaneh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 568 Lavie, Arnon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 333 Lavilla-Alonso, Sergio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 193 Lavitrano, Marialuisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59

EXHIBITOR PROSPECTUS final program

Lawler, Sean . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 498 Lawson, Alastair . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845 Lawton, Anna E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 727 Lay, Fides D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 44, 224, 373 Layman, John M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 125 Lazaris, Vassileios . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 729 Le, Yun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 417 Le Boeuf, Fabrice . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 37, 39 Le Gall, Tony . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120, 131, 395, 745 Le Guiner, Caroline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 305, 365 Le Hir, Maëva . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 154 Lebel-Binay, Sophie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 110, 113, 853 LeBlanc, Katarina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 453 Leborgne, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 400 Leboulch, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13, 228 Leddon, Jennifer L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 201 Lee, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 913 Lee, Andrew Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910 Lee, Bokhui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 895 Lee, Brendan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 46, 224, 309 Lee, Brendan H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677 Lee, Chang J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 522 Lee, Dean A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479 Lee, Donghoon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553 Lee, Fan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558 Lee, Gabsang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 283 Lee, Gary . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783, 798 Lee, Hwang-Jae . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Lee, Il-Kyu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 516 Lee, Jessica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 477 Lee, Jung-Sun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 720, 841 Lee, Kyung-Mi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167 Lee, Lily . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 674 Lee, Meng Horng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 282 Lee, Mi-Hyang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 384 Lee, Mingul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 202 Lee, Sang kyung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 877 Lee, Sang-Jin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 384 Lee, Sang-Kyung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 407 Lee, Seong-Wook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 560 Lee, Suhung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 257 Lee, Won Il . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 560 Lee, Yeon Jyung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 406 Lee, Yeon Kyung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128, 754 Lee, Yong J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 257 Lee, Young Ju . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 560 Lee, Young Won . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 126 Lee, Young-Sook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 198 Leen, Ann . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 179, 471

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Leen, Ann M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 29, 473, 799 Leger, Adrien . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 305, 365 Leggiero, Eleonora . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435 Lehn, Pierre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120, 131, 395, 745 Lei, Pedro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 358, 359 Lei-Butters, Diana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4 Leif, Jean . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285 Leja, Justyna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 501, 828 Lekka, Malgorzata . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 533 Lemiale, Franck . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 41, 165 Lemiegre, Loic . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120 Lemoine, Nick R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605 Lenander, Andrew W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20 Lenne, Nathalie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883 Leon, Xavier . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762 Leonard, Helen Paige . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 411 Leonard, Vincent . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 168 Leong, Derek . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 321 Lepetit, Helene . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Lerario, Alberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 795 Lesch, Hanna P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 209, 669 Lesniak, Maciej S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 191, 718, 835, 902 Leung, Anders . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169 Leung, Cary H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 778, 782 Leung, Kitty . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 872 Levine, Bruce . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 341, 798 Levings, Megan K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 79 Lévis, Nicolas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460 Levy, Camille . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336 Levy, Robert J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 323, 425, 753 Lewin, Alfred . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 61 Lewin, Alfred S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 417, 446 Lewis, Sarah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155, 290, 581 Leyfman, Yan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 266 Li, Baozheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 369, 704 Li, Chengwen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 160, 366, 367 Li, Chengwin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 581 Li, Chengyao . . . . . . . . . . . . . . . . . . . . . . . . . . . 171, 518, 806, 807, 808, 854 Li, Dejia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 466, 707 Li, Haitang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287 Li, Hojun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308 Li, Hongtao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 203 Li, Hongwei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 219, 518, 854 Li, Hui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 412 Li, Jianfeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 484 Li, Juan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 6, 160, 420, 581, 683 Li, Linhong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 675 Li, Mengxin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 369 Li, Min . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 876

Li, Pingchuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85 Li, Quanhai . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851 Li, Shulin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 105, 484, 737 Li, Songtao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 142 Li, Tingting . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 171, 806, 807 Li, Wei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 890 Li, Weidong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 186 Li, Wuping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2 Li, Xiaohong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 130 Li, Xiaozhu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605 Li, Yan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603 Li, Yi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234 Li, Yong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 456, 465 Li, Yong-Fu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 49 Li, Yuedan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617 Li, Zhixiong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 619 Li, Zhong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 68 Li, Zong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 657 Li, ZongYi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 180, 264, 265, 843, 849, 863 Lichty, Brian D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39 Lidonnici, Maria Rosa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 523 Lieber, Andre . . . . . . . . . . . . . . . . 93, 180, 264, 265, 415, 657, 843, 849, 863 Lieberman, Jay R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 687 Liesner, Raphael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 92 Liggitt, Denny . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 343 Liikanen, Ilkka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204 Likar, Yury . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185 Liles, Conrad . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 907 Liljenfeldt, Lina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 820 Liljenfeldt, Lina S. E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 486 Lillicrap, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767 Lim, Arlene T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 418 Lim, Carol S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 205 Lim, Kwang-il . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 299 Lim, Sung-Won . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Lima de Silva, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479 Limberis, Maria P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 625 Lin, Amy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 411 Lin, Chao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 126 Lin, Chin-Yu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583 Lin, Chung-Ren . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 882 Lin, Feng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103, 115, 327 Lin, Kun-Ju . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583 Lin, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26 Lin, Xiaojing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 694 Lindberg, Mattias . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120, 131 Lindholm, Leif . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 506 Lindqvist, Camilla . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 83 Lindqvist, Camilla A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 176

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Abstract Author Index Lindsey, Matt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 281 Ling, Chen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 369, 700, 704, 706 Linton, Gilda M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 354 Lionetti, Vincenzo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423 Lipshutz, Gerald S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 44, 224, 373 Lisowski, Leszek . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 306 Liu, Chao-Lien . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 535 Liu, Chunyan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181 Liu, Dexi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 100, 108, 322, 484 Liu, E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30 Liu, Elaine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783 Liu, Enli . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288, 293 Liu, Hao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288, 293 Liu, Jian W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 417 Liu, Jian Wen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21 Liu, Joyce C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 651 Liu, Meihong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 696 Liu, MengLu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 260 Liu, Minzhi M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 390 Liu, Pei-Qi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88 Liu, Shihe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 247, 564 Liu, Shi-he . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 892 Liu, Xiaoming . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620 Liu, Xiaoxuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287 Liu, Xiuying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662 Liu, Ying . . . . . . . . . . . . . . . . . . . . . . 180, 264, 265, 415, 657, 843, 849, 863 Liu, Yong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 429, 436, 711 Liu, Yunfan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 238 Liu, Zhao-Jun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603 Liu, Zhongda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218 Llewellyn, J. Anthony . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 116 Lo, Mandy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 300 Lo, Mandy Y. M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 691 Lo, Wen-Yi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583 Lock, Martin W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 45 Lockey, Timothy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 861 Logg, Christopher R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334, 831 Loh, Y. P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 143 Loiler, Scott A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 66, 231 Loiseau, Damien . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 745 Lollar, Pete . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527 Lombardo, Angelo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10, 88, 286, 632, 654 Long, Timothy E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 122, 125 Lopez, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279 Lopez, Teresita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Lopez, Veronica M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 609, 834 Lorain, Stéphanie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 154 Lorilleux, Christophe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 131 Lorini, Alessandro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276

EXHIBITOR PROSPECTUS final program

Lorson, Christian L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 626 Loskog, Angelica S. I. . . . . . . . . . . . . . . . . . . . . . . . . . . 83, 176, 453, 486, 820 Lotta, Louis T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 76 Lou, Rongcheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 819 Louie, Stan F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 522 Lovric, Jasmina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 311 Low, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156 Low, Walter C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20 Lowe, Andrew S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394 Lowe, Michelle C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 592 Lowenstein, Pedro R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26, 181, 475 Lu, Aiping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 629, 635 Lu, An . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 469 Lu, Hui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310 Lu, J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 697 Lu, Jian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 114 Lu, Jing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 65 Lu, Peijuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 890 Lu, Qi Long . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 890 Lu, Taihe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 222, 860 Lu, Yi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 217 Lu, You . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433 Lu, Yuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 700, 704, 706 Lu, Yuanqing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 375 Lu, Zhuoyuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 405 Lucarelli, Guido . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319 Luchetti, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908 Lukacs, Gergely . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620 Lukashov, Vladimir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 797 Luke, Jeremy M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 166, 572, 575, 738 Lulay, Christina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612 Lundberg, Cecilia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 239, 350 Lundberg, Kathryn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 107 Luo, Chia-Hsin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583 Luo, Mathew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279 Luo, Meihui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620 Luo, Min . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 504 Luo, Xianghua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 497 Luo, Xiaoyan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 142 Luo, Yonglun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91 Lupo Stanghellini, Maria Teresa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295 Lupold, Shawn E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 251, 390 Lusic, Hrvoje . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 744 Lusky, Monika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204 Lust, John A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218 Lüttge, Doreen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 528 Lv, Chengyu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 566 Lyon, Deborah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Lythgoe, Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index M

Ma, Hong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 355, 534 Ma, Jianjie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 683 Ma, Qiangzhong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 27 Ma, Wenqin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 369, 704 Ma, Yongjie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 100 Ma, Zhijun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 860 Määttä, Ann-Marie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 209 Macauley-Rambach, Shannon L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 22 Machen, Laurie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 104 Machleidt, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 268 Maciotta, Simona . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 464, 795 MacTavish, Heather . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39 Maczuga, Piotr . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 255 Mader, Emily K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 488 Maderna, Claudio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 413 Madigan, Nicolas N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 780 Madoiwa, Seiji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 421, 544 Madtes, David K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 623 Maeda, Michiko W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151 Maeda, Tadao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445 Maeda, Yasushi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468, 788 Maeder, Morgan L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 95 Maetzig, Tobias . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284, 302, 619 Maeyama, Yoshihiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678 Magee, Mitchell . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 292 Magnani, Zulma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88, 654 Magnusson, Maria K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 506 Maguire, Casey A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57 Maguire-Zeuss, Kathleen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 23 Mah, Cathryn S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 596 Mahan, John D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155 Mahato, Ram I. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 84, 741 Mahller, Yonatan Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 830 Maier, Dawn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 798 Maisner, Andrea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336 Maiti, Sourindra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479 Makarevich, Pavel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434 Makino, Hirofumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 884 Malani, Nirav . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13, 228, 283, 308, 346 Maldonado, Arturo R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 830 Malech, Harry L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 220, 354, 688, 860 Malgieri, Arianna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908 Malik, Alock . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 374, 454, 458, 467, 785 Malik, Punam . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 280, 526, 864 Malik, Vinod . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 155, 290, 581 Malloy, Andrew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 577 Malmstrom, Per-Uno . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 176 Mamchaoui, Kamel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 789

Mamdani, Mohammed . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423 Mancheño, Uxua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Manchengo, Uxua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 810 Mandel, Ron . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 147 Mandelli, Giacomo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319, 523 Mangopoulos, Kathryn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 280 Mangsbo, Sara M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 176 Mangus, Courtney . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 341 Mani, Sendurai . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 243 Maniar, Jay M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14 Mann, Anita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 132 Mann, Derek A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 716 Mano, Miguel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 311 Manoli, Irini . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135 Mansfield, S. Gary . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 422 Manunta, Maria D. I. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 747 Mao, Hai-Quan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 124, 321 Mao, Haoyu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 446 Maples, Phil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144 Maples, Phillip B. . . . . . . . . . . . . . . . . . . . 148, 213, 245, 247, 248, 252, 275, . . . . . . . . . . . . . . . 292, 335, 487, 520, 532, 564, 574, 578, 586, 731, 892 Maródi, Laszló . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612 Marano, Giuseppina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276 Marasco, Wayne A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645 Marc, Lavertu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 396 March, Keith L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 630 Marchadier, Dawn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 412 Marcucci, Katherine T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 630 Marerro, Bernadette . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 823 Marianowski, Rémi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120 Maric, I. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31 Marigo, Valeria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 624 Marin, Virna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845 Marinkovich, Peter M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320 Marion, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 82 Marjomäki, Varpu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159 Markley, John C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 655 Marktel, Sarah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319 Markusic, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 371 Markusic, David M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 540 Marongiu, F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139 Marrocco, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 624 Martelli, Letizia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276 Martin, Fernando L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599 Martin, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43 Martinez, Caridad . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Martino, Ashley . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 540 Martino, Ashley T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 309, 363, 539 Martino, Sabata . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 316, 413

165

166

American Society of Gene & Cell Therapy

Abstract Author Index Martuza, Robert L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 608, 829 Maruggi, Giulietta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10, 319, 346 Marumoto, Tomotoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263, 899 Maruyama, Kazuo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 102 Masatoshi, Ishizaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 788 Maslyar, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 35 Mason, A. James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 400 Massariello, Paola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276 Massey, Paul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659 Massie, Bernard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463 Mastrangelo, Michael A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 18 Masuda, Akio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 585 Masuda, Tomoya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 591, 743 Masurier, Carole . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 67, 791 Matar, Majed . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893 Mathiesen, Iacob . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 289 Matsui, Chihiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320 Matsui, Hideto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767 Matsumoto, Hiroshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 492, 831 Matsumoto, Tae . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 680 Mattar, C. N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223, 697 Mattar, Citra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 531 Matteo, Benjamin C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21 Matthews, Qiana L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 842 Mätzig, Tobias . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 304 Mauleón, Itsaso . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Mauleon, Itsaso . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 810 Mavilio, Fulvio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10, 319, 346, 351, 523 Mayer, Christina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 683 Maynard, Jonathan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85 Mays, Lauren E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1 Mazur, Curt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 557 Mazurier, Frédéric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865 Mazzaccara, Cristina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435 Mazzieri, Roberta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 331 McAlister, Victor J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 70, 710 McCants, Marjorie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314, 411 McCarty, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 814 McCarty, Douglas M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 307 McChesney, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 168 McClure, Diane . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893 McConnell, R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879 McConnell-Smith, Audrey . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 89 McCormick, Dominique . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880 McCown, Thomas J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2, 355, 366 McCoy, Jay . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103, 327 McCullagh, Karl . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439 McDonald, Robert A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380 McDonough, Samantha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 80, 505

EXHIBITOR PROSPECTUS final program

McGarrity, Gerard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 41, 646, 803 McGarrity, Gerard J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 274, 422 McGinley, Lisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 427 McGoogan, Katherine E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 700 McHugh, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439 McIntosh, J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223 McIvor, R. Scott . . . . . . . . . . . . . . . . . . . . . . 868, 20, 221, 232, 325, 734, 852 Mckenna, Mavis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58 Mckiernan, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 35 McKown, Thomas J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 778 McLachlan, Gerry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880 McMahon, Jill . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 427 McMahon, Ross . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279 McMahon, Siobhan S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 780 McMahon, Stephen B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451 McMenamin, Deirdre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1 McNamara, II, James O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662, 663 McNeer, Nicole Ali . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285 McPhee, Scott . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 581 McPhee, Scott W. J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 160, 778 McVey, John H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 715 Mead, Andrew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 374, 458, 467 Mead, Andrew F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 785 Mead, Andy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 454 Medin, Jeffrey . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185, 341 Medin, Jeffrey A. . . . . . . . . . . . . . . . . . . . . . . . . . 79, 333, 508, 509, 659, 771 Medina-Echeverz, Jose . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 810 Medina-Jaszek, C. Angelica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 164 Meehan, Anne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 301 Mehta, Jawahar L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 437 Mei, Shirley H. J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 907 Mei, Z. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30 Mejido, Josef . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 482 Melcher, Alan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 481, 606, 607, 610, 653 Melero, Ignacio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Mello, Chris . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 674 Mendell, Jerry R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 152, 155, 290, 581 Mendoza, Janess . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 115, 327 Meng, Cuixiang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 873 Meng, Max . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 35 Meng, Xin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 192 Mercer, Andrew C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 94 Meregalli, Mirella . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460, 464, 795 Merlin, Simone . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 134 Merling, Randall . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 800 Merlini, Luciano . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258, 666 Mermod, Nicolas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 533 Meroni, Germana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 624 Meseck, Marcia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38, 277

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Metzger, Joseph M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 584 Metzger, Michael J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 89 Meunier, Isabelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169 Meyer, Edwin M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 775 Meyer, Johann . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 318 Meyerholz, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620 Mezzadra, Riccardo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319 Miao, Carol H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 326, 535, 550 Miari, Reem . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616 Michael, Buschmann . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 396 Michaud, Annick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 784 Midoux, Patrick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 324, 395 Mikheeva, Galina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 725 Mikkelsen, Jacob G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695, 726 Miliaresis, Jennifer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 164 Milinkeviciute, Giedre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349 Millán, José Luis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 680 Miller, A. Dusty . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 89, 623 Miller, Ashley M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380 Miller, Darren . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 874 Miller, Timothy J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 426, 438 Millili, Peter G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 590 Mims, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30 Mimuro, Jun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 421, 544 Min, Dal-Hee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 895 Min, Seok H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 61 Mincheva-Nilsson, Lucia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57 Mineharu, Yohei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26 Mineno, Junichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 652, 678 Ming, Xin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 62 Mingozzi, Federico . . . . . . . . . . . . . . . . . . . . . . . . . . . 8, 43, 66, 546, 548, 614 Mingozzi, Federico M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308 Mir, Lluis M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 853 Miranda, Paula S. M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138 Miselli, Francesca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10 Miskin, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Mitchell, James B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 74, 77 Mitchell, Marilyn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 374, 454, 785 Mitrophanous, Kyriacos . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Mittal, Plavi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796 Miura, Yoshie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263, 899 Miyake, Koichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 410, 510, 680 Miyake, Noriko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 410, 510, 680 Miyake, Takashi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 884 Miyamoto, Shohei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 192 Miyanohara, Atsushi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 765 Miyoshi, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 899 Mizrak, Arda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 665, 846 Mizuguchi, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . 207, 216, 480, 537, 591

Mizukami, Hiroaki . . . . . . . . . . . . . . . . . . . . . . . . . . . 207, 421, 428, 544, 554 Mizusawa, Hidehiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 259 Mockaitis, Keithanne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353, 356 Mockenhaupt, Stefan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 660 Modlich, Ute . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 304, 318, 619 Mody, Disha A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 222 Moen, Robert C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622 Moghimi, Babak . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 146, 763 Mohseni, Mahboobeh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871 Moimas, Silvia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423, 431 Molas, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762 Moldt, Brian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695 Monahan, Paul E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 534 Monnier, Nilah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796 Monsma, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 487 Montazami-Astaneh, Kaweh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 95 Montgomery, Chrystal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149 Montier, Tristan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120, 131, 395, 745 Montini, Eugenio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 298, 613 Moon, Anne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33 Moon, Ju Young . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 386 Moon, Lawrence . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451 Mooney, Michael A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 235 Mooney, Sean . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353, 356 Moore, Nicole M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 589 Moot, Robby . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 230 Morcinek, Nick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 681 Morcos, Paul A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 49 Moreau-Gaudry, François . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865 Moreno, Amira Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 161, 452, 752 Moreno, Peter J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 272 Morgan, R. A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31 Morgan, Richard A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 190, 476, 478 Morgello, Susan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38 Morishita, Ryuichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 884 Moritz, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 528 Morizono, Hiroki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 416 Mornet, Emmanuel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 120 Moroz, Maxim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185 Morral, Nuria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 52 Morris, Caroline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 483 Morris, John C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 80, 483, 505 Morris, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250 Morrissey, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 398, 848 Morrow, Genevieve . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242 Morrow, M. P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648 Morrow, Matthew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103 Mossalam, Mohanad . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 205 Mossoba, Miriam . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659

167

168

American Society of Gene & Cell Therapy

Abstract Author Index Moulay, Gilles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 67, 791 Moullier, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19, 305, 365 Mouly, Vincent . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 789 Mowa, Betty . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 82 Moya, Marta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762 Mrak, Emanuela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315 Mrugala, Maciej . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294 Mu, Xiaodong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 456, 465 Muñoz, Nina M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 909 Muñoz, Sergio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762 Mueller, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 51, 53 Mueller, Steffen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372 Muenzer, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 328 Muether, Nadine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 636 Muir, Lindsey A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 792 Mujagic, Edin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 430 Mukherjee, Sayandip . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 866 Mukhopadhyay, Debabrata . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606 Müller, Oliver J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 241, 432 Mullins, Charles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 639 Multhaup, Megan M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221, 232 Munden, Regina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1 Muniz Pello, Oscar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88, 654 Murakami, Akikazu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645 Muralidharan, Sujatha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 28 Murillas, Rodolfo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 351 Murphy, Michael P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 269, 272, 272 Museri, Adi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 719 Mussolino, Claudio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59, 624 Mustafa, Sadaf . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 382 Muthumani, K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 115 Muul, Linda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296 Mykhaylyk, Olga . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693 Myung, Chang-seon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 896

N

Na, Chang-Rim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 731 Nace, Rebecca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 604 Nagasaki, Takeshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 397 Nagasaki, Yukio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 246, 253 Nagata, Keiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236 Nagata, Tetsuya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56, 565 Naik, Rangeetha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 132 Naik, Rangeetha J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 127 Naik, Shruthi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 604 Naik, Ulhas P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 590 Nair, Smita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 561 Najder, Oliwia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 875 Nakagami, Hironori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 774

EXHIBITOR PROSPECTUS final program

Nakai, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 7, 141, 580, 703 Nakajima, Satoru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678 Nakamura, Akinori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56, 151, 565 Nakamura, Masakuni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167 Nakamura, Takafumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 34, 192, 840 Nakamura, Takashi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 401 Nakano, Kenji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640 Nakashima, Hiroshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 491 Nakashiro, Koh-ichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 493 Nakatsu, Yoshimichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 97 Nakazawa, Yozo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 179, 813 Nalam, Madhusudhana R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 404 Nalbantoglu, Josephine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463 Naldini, Luigi . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10, 88, 225, 276, 286, 297, . . . . . . . . . . . . . . . 298, 313, 315, 316, 331, 337, 413, 545, 613, 632, 654 Nan, Zhenhong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20 Nande, Rounak . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 847 Narayanan, Priyadharshini . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 474 Narisawa, Sonoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 680 Narrow, Wade . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 23 Narrow, Wade C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 18 Nathwani, A. C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223 Nathwani, Amit C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 668 Naundorf, Sonja . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612 Navarro, Claire . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460 Neeley, Andy W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17 Neely, Andrew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 71 Neff, Preston C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287 Negishi, Yoichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 102 Neglia, Simona . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59 Negre, Didier . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336 Negre, Olivier . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 228 Nemunaitis, Gregory . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 586 Nemunaitis, John . . . . . . . . . . . . . . . . 35, 144, 148, 196, 213, 245, 247, 248, . . . 252, 275, 292, 335, 487, 520, 532, 536, 564, 574, 578, 586, 731, 892 Neri, Marcella . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258 Neri, Margherita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 316, 413, 632 Neschadim, Anton . . . . . . . . . . . . . . . . . . . . . . . . . . . 185, 333, 508, 509, 771 Nettelbeck, Dirk M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 381, 387, 641 Neuberg, Patrick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883 Newman, Nikki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 873 Ng, P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879 Ng, Philip . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 388, 542, 616, 639 Nguyen, Andrew T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 731 Ni, Xiahoua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 251 Nichols, Timothy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 613 Nichols, Timothy C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 615 Nicholson, Linda J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 716 Nickens, Brittani . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 785

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Nickerson, Michael L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 365 Nicklin, Stuart A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380, 715, 723 Nicoletti, Roberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295 Nicolson, Sarah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2 Niedernhofer, Laura J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 629, 635 Nielsen, Peter E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 109 Nienhuis, Arthur W. . . . . . . . . . . . . . . . . . . . . . . 338, 339, 449, 668, 733, 867 Nieva, Jorge . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33 Nikhamin, Yan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 815 Nikolskaya, Alena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 402 Nilsson, Berith . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 501, 828 Nimesh, Surendra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 396 Nimjee, Shahid M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 667 Nishikawa, Makiya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 670, 862 Nishimura, Jun-ichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 667 Nishina, Kazutaka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 259 Nishiyama, Akiyo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151 Nishiyama, Nobuhiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 891 Nissenson, Allen R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616 Niu, Guoqin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 256 Nixon, Alan J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 912 No, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 772, 794 Noble, Misty L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 326 Nokay, Bahar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 887 Nokisalmi, Petri . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 291 Nolta, Jan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 281 Nordheim, Alfred . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 241 Nordling, Diana L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 280 Noske, Nadja . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 92 Novelli, Giuseppe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908 Noviello, Maddalena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295 Nowrouzi, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223 Nowrouzi, Ali . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 305, 612 Numasaki, Muneo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851 Nunan, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 536 Nunez-Alonso, Graciela A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 123

O

Obach, Mercè . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140 Obach, Merce . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762 O’Brien, Timothy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 424, 427, 439, 780 O’Callaghan, Christopher . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 747 Ochiai, Hiroshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 240 Ochs, Hans . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 529 Odom, Guy L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 461, 584 O’Donnell, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 598, 758 O’Driscoll, Caitriona . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 254 Oehler, Elise A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599 Oette, Sharon M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622

Offord, Chetan P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 208 Ofstein, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 269 Ogura, Manabu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 428 Oh, Il Ung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 112 Oh, Seeun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 257 Oh, Sung-Min . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Ohashi, Kazuo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 141, 419, 903 Ohlemiller, Kevin K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 768 Ohlfest, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 826 Ohmori, Tsukasa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 421, 544 Ohno, Kinji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 585 Oikawa, Tatsuo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263 Oishi, Motoi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 246, 253 Ojima, Iwao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 266 Okada, Hironori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151, 582 Okada, Michiyo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263 Okada, Takashi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151, 582, 585 Okamoto, Sachiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 652 Okamoto, Shinya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851 Okamura, Haruki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 500 Okano, Kiichiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445 Okano, Teruo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 141, 419, 903 Okazaki, Akiyo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236 Okazawa, Hitoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 565 Oksanen, Minna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 193, 538, 658 Okun, Alex . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616 Olivares, Jairo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 292 Olivares, Simon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479 Oliveira, Giacomo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295 Olsen, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 355 Olsen, John C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 881 Olson, Erik . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 734 Olson, Erik M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234, 858 Olson, Erik R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 325, 868 Olsson-Strömberg, Ulla . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 83, 820 Olszewska, Malgorzata . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32 O’Malley, Janis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36 O’Malley, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 758 Oneal, Michael J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 80, 505 O’Neill, Ann Marie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 379 Onishi, Akira . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 428 Onodera, Masafumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678 Orimo, Hideo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 680 Orkin, Stuart H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 338 Orlacchio, Aldo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 413 Osawa, Kanako . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236 Oshimura, Mitsuo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236 O’Sullivan, G. C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 194 O’Sullivan, Gerald C. . . . . . . . . . . . . . . . . . . . . . . . . . 398, 705, 736, 812, 848

169

170

American Society of Gene & Cell Therapy

Abstract Author Index O’Sullivan Murphy, Bryan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 51, 53 Otoi, Takeshige . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 804 O’Toole, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 427 Otsu, Makoto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678 Otsuka, Makoto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 749 Ou, Wu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 649 Owen, Carina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 602 Owens, Roland A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 70, 710 Ozaki, Kazunari . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 884 Ozawa, Keiya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 207, 421, 428, 544, 554 Ozdener, Baris G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 846 Ozelo, Margareth . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767 Ozlem, Senol . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 846

P

Pacholska, Agnieszka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 212, 512 Pacienza, Natalia A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 509, 771 Pack, Daniel W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 119, 595 Padegimas, Linas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622, 776 Padmashali, Roshan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 282, 359 Pagliara, D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 178 Palanca-Wessels, Maria C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558 Palazón, Asis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Palczewski, Krzysztof . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445 Paldor, Mor . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16 Palfi, Stephane . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Palmer, D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 879 Palmer, Donna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 75, 388, 639, 677 Palmer, Donna J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 542 Pan, Deng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 321 Pan, Haiying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 465 Pandha, Hardev . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606 Paneda, Astrid . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 485 Pang, Jijing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17 Pankhong, Panyupa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 477 Papanikolaou, Eleni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 338 Papapetrou, Eirini . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 729 Papapetrou, Eirini P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 283 Papayannopoulou, Thalia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137 Pappen, Beena O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 275, 292, 487, 532 Pâques, Frédéric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 87, 462 Parato, Kelley . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33 Parfyonova, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434 Pariétti, Véronique . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 533 Paris, Clément . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 883 Park, Eugene . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783 Park, Harriet . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Park, In-Kyu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 399, 751, 900, 904 Park, Jeong-Sook . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 896

EXHIBITOR PROSPECTUS final program

Park, Keerang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 722 Park, Ryan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 200 Park, Sang H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 687 Park, Sang Il . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128, 406, 754 Park, Yeon Hee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85 Park, Yong Serk . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128, 406, 754 Park, Younjoo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 722 Parker, Alan L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380, 715 Parker, Colleen H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 907 Parker, Ernest T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527 Parker, Josh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 852 Parkhurst, Maria R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25, 190 Parolini, Daniele . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460, 464, 795 Parsons, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 874 Parsons, David W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 621 Partridge, Terence . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56 Paruzynski, Anna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 612 Pasceri, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 300 Passineau, Michael J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 104 Passini, Marco A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55 Pasternak, Alexander . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 797 Pastore, Joseph M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 426, 438 Pastore, Lucio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435 Patel, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648 Patel, Ami . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169, 724 Patel, Manij . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 881 Patel, Priti M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 639 Patrene, Ken . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157 Pattarini, Lucia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 431 Paulk, Nicole K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 99 Pavelko, Kevin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 607 Payen, Emmanuel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 228 Peñuelas, Iván . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Pearlman, Andrew L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616 Pease, Larry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 607 Peccate, Cécile . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 154 Peccatori, Jacopo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295 Pedersen, Alexandra G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 787 Pedro, Helder L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 802 Peebles, D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223 Peelman, Luc . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 875 Pei-Qi, Liu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 654 Pellin, Danilo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 345, 346 Peluffo, Hugo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451 Pelusio, Adina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33 Penaud-Budloo, Magalie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 305, 365 Pence, Casey . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893 Peng, Baowei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 535, 550 Peng, Hairong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 456

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Peng, Kah-Whye . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 488 Peng, Liaomin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 517 Peng, Ling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287 Penn, Marc S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 426, 438 Pepe, Martino . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423 Perabo, Luca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5 Peretz, Mary E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 301, 685 Perez, Omar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 492, 831 Perez, Paola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 761 Perini, Giovanni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258 Perkins, Jeanne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314 Perna, S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 178 Perna, Serena Kimi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 346 Perricaudet, Michel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 853 Perrin, George Q. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 363, 370 Perrone, Daniela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 666 Persons, Derek . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 867 Persons, Derek A. . . . . . . . . . . . . . . . . . . . . . . . . . . . 338, 339, 524, 525, 861 Persson, Jonas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 180, 264, 843, 849, 863 Peshwa, Madhusudan V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 675 Pesonen, Sari . . . . . . . . . . . . . . . . . . 177, 193, 197, 204, 291, 538, 549, 658 Pestina, Tamara I . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 525 Pestronk, Alan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 152 Peters, Brandon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 356 Petersen, Line . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85 Petersen, Line B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695 Peterson, Kenneth . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 340 Petrov, Mihail . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156, 454, 458, 600, 785 Petrov, Mike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 374 Petrs-Silva, Hilda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 61 Petry, Harald . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 249, 255 Pfaff, Nils . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 528 Pfeifer, Alexander . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693 Phadke, Anagha P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 148, 196, 252, 586 Phalon, Connor . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 245 Pham, Thu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 405 Phillips, Jana L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 364, 367, 403 Phillips, Neil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 760 Phillips, Terry . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 482 Piao, Jingshu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 97 Piao, Wenying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 259 Piccolo, Pasquale . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 75 Pichavant, Christophe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 462, 790 Pichon, Chantal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 324, 395 Picquet, Erwan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 745 Pien, Gary . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43 Piersanti, Stefania . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 133 Pierson, Tyler Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 235 Pietruszka, Marvin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 772

Piguet, Françoise . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19 Pikkarainen, Jere T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 209, 669 Pilla, James J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600 Pillet, Stephane . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169 Piras, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 453 Piraud, Monique . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19 Pisano, F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 360 Pizzitola, Irene . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845 Plank, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693 Plotkin, Jason . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184, 655, 911 Podetz-Pedersen, Kelly . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221 Podetz-Pedersen, Kelly M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 232 Podetz-Petersen, Kelly M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 852 Podhajcer, Osvaldo L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 609, 834 Podsakoff, Gregory M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 8 Poeschla, Eric M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 301, 685, 804 Polach, Kevin J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893 Polard, Valerie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 110, 113, 853 Politi, Letterio S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 315 Ponder, Katherine P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315, 598 Ponnazhagan, Selvarangan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 63, 507, 906 Ponomarev, Vladimir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184, 185, 333 Ponzoni, Maurilio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297 Pope, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648 Porada, Chris D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 857 Porada, Christopher D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 551 Porcellini, Simona . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 644 Postema, Michiel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 324 Potter, Clinton S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 393 Pouliot, Frédéric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 714 Poulsen, Hans S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 210, 511 Poupiot, Jérôme . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48 Pourfathollah, Ali Akbar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871 Powell, Kimerly . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 836 Powell, Suzanne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 471 Power, Anthony T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 37 Powers, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 328 Pozmogova, Galina E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 735 Pozza, Chiara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276 Pratico, Elizabeth D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 561 Pratt, William D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 643 Premkumar, Muralidhar H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677 Press, Michael F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558 Press, Oliver W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558 Preston, Thomas J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149 Preuss, Meredith A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36, 503 Prieto, Jesús . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Prieto, Jesus . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 485, 494, 708, 810 Pringle, Ian A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 727

171

172

American Society of Gene & Cell Therapy

Abstract Author Index Pristinski, Denis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 589 Privman, Eyal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16 Proto, Jonathan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 635 Protzer, Ulrike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312 Provasi, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88, 632, 654 Pucci, Ferdinando . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 331 Pule, Martin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845 Pulicherla, Nagesh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 364 Pulido, Jose . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606, 607, 610, 653 Puligadda, Uday . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423 Pun, Suzie H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 730 Pupko, Tal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16 Puttaraju, Madaiah . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 422

Q

Qanud, Khaled . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423 Qasim, Waseem . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 158, 647 Qazi, Aamer M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250 Qi, Feng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 566 Qi, Yanfei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 518 Qiang, Ming . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 211 Qiao, Chunping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 6, 420, 683 Qiao, Jian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 195, 495 Qin, Bin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 567 Qin, Elizabeth . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 768 Qin, Qiuyue . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 164 Qin, Rui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 505 Qiu, Guozhen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 806 Qiu, Xiaohong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 402 Qiu, Xingping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 756 Qiujie, Jiang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320 Qu, Guang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 69, 673 Qu, Jinrong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184 Quach, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 782 Quattrini, Angelo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 315 Quezada, Valerie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 277, 279 Quinn, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 674 Quintanilla, María Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 766 Quintino, Luis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 350 Quiocho, Florante A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 574 Quispe, Joel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 393

R

Raake, Philip W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 432 Rabinowitz, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600 Rabinowitz, Joseph E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 366 Rabkin, Samuel D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 608, 829 Rachinel, Nicholas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336 Radd, Katherine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 422 Rader, Daniel J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 412

EXHIBITOR PROSPECTUS final program

Radrizzani, Marina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225, 276, 613 Radu, Antenata . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320 Radu, Antoneta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 344, 676 Raffeld, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31 Raggi, Geno . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 457 Rah, Dong Kyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 516 Rahim, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 697 Rainusso, Nino . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 470 Rajagopalan, Malolan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 514 Rajecki, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 197, 291 Rajendran, Simon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 398, 812, 848 Rajput, Kamal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242 Raki, Mari . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 197, 291, 549, 658 Rakugi, Hiromi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 884 Ralph, Scott . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Ramesh, Rajagopal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 243 Rameswaran, Rameswari . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 576 Ramezani, Ali . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 342, 690 Ramirez, Sean M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 125 Ramlogan, Charmaine A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 483 Ramo, Kasmir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 448 Ramos, C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30 Ramos, Carlos A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288, 799 Ramsay, Tim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 779 Rantanen, Ville . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204 Ranzani, Marco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 298, 613 Rao, Donald D. . . . . . . . . . . . . . . . . . . . . . 245, 247, 248, 252, 532, 564, 892 Rao, Mahendra S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 268 Rao, Thapi D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 183 Raoul, Sylvie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19 Rapp, Brian M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 269, 272 Rasmusson, Ida . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 453 Rathod, Trushar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 143 Ratiglia, Roberto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59 Ratner, Nancy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 201 Rauschhuber, Christina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 385 Raut, Sanj . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 134 Ravin, Suk See De . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 688 Rawlings, David J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . 90, 226, 343, 529, 550 Ray, William C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 699 Razini, Paola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460, 464, 795 Razquin, Nerea . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 889 Read, Sarah P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 447 Readhead, Carol . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349 Rea-Ramsey, Angela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893 Reay, Daniel P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 457 Rebar, Edward . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12 Rebello, Tessio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 41, 646, 803 Recchia, Alessandra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10, 346

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Recchia, Fabio A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423 Reddy, Anilkumar K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677 Reeves, Lilith R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 280 Reginato, Silvia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 597 Reichenbach, Janine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 233, 869 Reik, Andreas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 88, 618, 654 Rejman, Joanna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 261, 875 Remant, K. C. B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 129, 755 Remaut, Katrien . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 742 Remoli, Cristina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 133 Remy, Philippe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Ren, Yan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 775 Ren, Yong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 124 Restifo, Nichaolas P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 478 Restifo, Nicholas P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 190 Rex, Tonia S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 441 Reyes-del Valle, Jorge . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 168 Ricca, Alessandra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 316 Rice, Jennifer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893 Rice, Kevin G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 117, 594 Richard, Emmanuel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865 Richard, Isabelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48, 793 Richards, Amy M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55, 557 Richly, Heike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 664, 887 Riddell, Stainly . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553 Riddell, Stanley R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 618 Riley, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 341 Riley, John P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 190 Rimessi, Paola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258, 666 Riminucci, Mara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 133 Rimler, Avi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616 Rindt, Hansjorg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 626 Rinkoski, Thomas A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 804 Ripamonti, Anna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225 Ripoll, Cynthia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314, 411 Rising, Aaron C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 147 Ritsema, Tita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 249, 255 Rival-Gervier, Sylvie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 300, 691 Rivera, Angel A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 834 Rivers, Angela E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 363, 370, 706 Rivière, Isabelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184 Rivière1, Isabelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 911 Riviere, Isabelle . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32, 283 Rivkin, Ludmila . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16 Riz, Irene . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 690 Rizzardi, Gian-Paolo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 644 Robbins, Joan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 492, 831 Robbins, Paul D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 457, 629, 635 Robbins, Paul F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25, 190

Robert-Richard, Elodie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 865 Roberts, David D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 731 Roberts, Marie S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 22 Robinson, Andria R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 629 Roca, Carles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140 Roccio, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 360 Rockey, William M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662, 663 Rockhill, Jason . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294 Rodgers, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 46 Rodino-Klapac, Louise R. . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 155, 290, 581 Rodrigues, Ana F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 278 Rodriguez, Luis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184 Rodriguez, Ronald . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 382, 390 Rodriguez, Stephen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 739 Roescher, Nienke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 870 Roffler, Steve . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 180, 863 Rogers, Faye . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285 Rojas, Juanjo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 291 Roland, Herzog W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 68 Rollins, Lisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 656 Rommelfanger, Diana M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 208 Roncarolo, Maria G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225 Roncarolo, Maria Grazia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 345, 545 Ronen, Keshet . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 228 Roode, Luke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 420 Roodman, G. David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157 Rooney, C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30, 178 Rooney, Cliona M. . . 28, 29, 179, 288, 293, 472, 473, 656, 672, 799, 813, 816 Rooney, Gemma E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 780 Roques, Caroline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 817 Rosada, Cecilia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695 Rosales-Quintero, Xiomara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 290 Roselli, Emanuela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319 Rosen, Elliot D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 630 Rosenberg, Jonathan B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 161, 452, 752 Rosenberg, S. A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31 Rosenberg, Steven A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25, 190, 476, 478 Rossetti, Francesca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276 Rossi, Claudia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319 Rossi, John J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287, 555, 661, 805, 888 Rossi, Settimio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59 Rostomily, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294 Roujeau, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19 Rousseau, Joel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 462, 769 Rousseau, Raphael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 845 Rowzee, Anne M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 74, 77, 143 Roy, Andre J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527 Roy, Dominic G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 37 Roy, Laura M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 681

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174

American Society of Gene & Cell Therapy

Abstract Author Index Roy, Soumitra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 164 Roybal, Jessica L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 344, 676 Ruan, Li . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 114, 172 Ruberte, Jesus . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762 Rubinacci, Alessandro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315 Rufibach, Laura E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796 Ruhn, Kelly M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 773 Ruiz, Rafaela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 52 Russell, David W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234, 858 Russell, Stephen J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 195, 203, . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336,490, 495, 499, 604, 684 Russo, Fabio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 758 Ruzo, Albert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140 Ruzsics, Zsolt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 385, 712 Rwigema, Jean-Claude . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157, 514 Ryals, Renee C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17, 71 Ryan, Deborah A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 18 Ryan, Martin D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 233 Rybalkin, Igor . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434 Ryffel, Bernhard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 552 Ryoo, Soo-Ryoon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 895 Ryu, Byoung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 529 Ryu, Byoung Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 90, 226, 343, 909 Ryu, Jihoon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242, 841

S

Saadatzadeh, Mohammad R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 272 Saadatzadeh, Reza . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 269 Sabatelli, Patrizia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 666 Sabourin, Kara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33 Sacchetti, Luciana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435 Sachar, Einat . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 16 Sack, Brandon K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 68, 539 Sadadcharam, Mira . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 736 Sadelain, Michel . . . . . . . . . . . . . . . . . . . . . . . . . 32, 184, 185, 283, 655, 911 Saechao, Chai . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 772, 794 Saeki, Koichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303, 634 Saenz, Dyana T. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 301, 685, 804 Safinya, Cyrus R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 393 Saga, Kotaro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 187 Sagawa, Hiroaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 674 Saggio, Isabella . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 133 Saha, Sunandan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 813 Sahenk, Zarife . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 155, 290, 581 Saighal, Sheena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 625 Saini, Massimo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 337 Sainte-Catherine, Vincent . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 110, 113 Saito, Nobuhito . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 832 Saito, Takahiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 419

EXHIBITOR PROSPECTUS final program

Saito, Takashi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56, 565 Sakata, Yoichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 421, 544 Sakiyama, Yukio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678 Sakiyama-Elbert, Shelly E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 589 Sakuma, Toshie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 688 Sakurai, Fuminori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 537 Sakurai, Kumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 888 Sakurai, Yoshihiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 903 Sakurai, Yu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 246 Salavert, Ariana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 762 Salazar, Felix . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 82 Salerno, Angela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796 Sallach, Jessica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5 Sallberg, Matti . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 111, 289 Salles, Gilles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336 Salomon, Daniel R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 408 Salomoni, Monica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276 Salsman, Vita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 179, 470, 471 Saltzman, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 852 Saltzman, W. Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285 Salvatore, Francesco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435 Salwa, Slawomir P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 736 Samaranayake, Haritha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 209, 669 Sampath, Alapakkam P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21 Sampath, Prakash . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 182 Sampedro, Ana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Samulski, Jade . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 581 Samulski, Jade J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 160 Samulski, R. Jude . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 2, 60, 160, 366, 581 Sanchez, Ceidy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 28, 672 Sanchez, Massimo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908 Sanchez-Bonilla, Marilyn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 330 Sanchez-Salazar, Jorge . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 517 Sanders, Niek N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 875 Sandhu, Arbans . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 625 Sands, Mark S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 22, 768 Sanges, Daniela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 624 Sangiuolo, Federica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908 Sankaran, Vijay G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 338 Sanmiguel, Julio C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 759 Sano, Akiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236 Santiago, Virgínia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 570 Santilli, Giorgia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 866 Santistevan, Nicholas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 355 Sanvito, Francesca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 315 Sapag, Amalia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 766 Sardesai, N. Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648 Sardesai, Niranjan Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103, 115, 327, 477 Sardi, S. P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Sargent, R. G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 913 Sarkar, Devanand . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 503 Sarkar, Rita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 134 Sarkaria, Jann N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 499 Sarkioja, Merja . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 549 Sarwari, Roya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617 Sasaki, Erika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 899 Sather, Blythe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 529 Sather, Blythe D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 226, 343 Sato, Makoto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 714, 850 Sato, Takeya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 333, 508 Sato, Yusuke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 894 Sauthoff, Harald . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 713 Savarala, Sushma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 757 Savilahti, Harri . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13 Savoldo, B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 30, 178 Savoldo, Barbara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288, 472 Savontaus, Eriika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 145 Savontaus, Mikko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 145 Sawall, Stefanie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 833 Sawari, Roya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 530 Saxena, Romil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 52 Saydam, Okay . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 665, 846 Sazani, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56 Scaife, Matthew D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 509, 771 Scallan, Martina F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 705 Scaramuzza, Samantha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225 Schöler, Hans . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284 Schürmann, Nina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 660, 886 Schaffer, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 299 Schaffer, David V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 8, 442, 802, 805 Schaller, Torsten . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 647 Schambach, Axel . . . . . . . . . . . . . . . . . . . . 284, 302, 304, 318, 528, 619, 869 Scharenberg, Andrew M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 90, 909 Schatzberg, Scott . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 467 Scherman, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 67, 400, 791 Scheulen, Max E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 664, 887 Scheurer, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 471 Schiedlmeier, Bernhard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 619 Schindler, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Schira, Giulia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 337 Schlachterman, Alexander . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308 Schlegel, Philipp . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 432 Schleifman, Erica B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285 Schlesinger, Pnina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 719 Schlesinger, Sarah J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Schmidt, Claudia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Schmidt, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223 Schmidt, Manfred . . . . . . . . . . . 138, 286, 297, 298, 305, 345, 346, 347, 612

Schmidt, Manuel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 189, 664, 822, 887 Schneider, Kellie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36 Schroeder, Mark A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 499 Schroeder, Timm . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284 Schroff, Matthias . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 189, 822 Schuldt, Nathan J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 651 Schuldt, Nathaniel J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638 Scott, Jeannine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638 Scott, Stephanie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 487 Scotton, Chiara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258 Scruggs, Brittni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314, 411 Seethammagari, Mamatha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 474 Seger, Reinhard A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 233, 869 Segura, Maria Mercedes . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 573 Seidler, Daniela G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 198 Seinstra, Beatrijs . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 815 Selby, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606, 653 Semaan, Assaad . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250 Semenova, Katerina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 158 Senac, Julien S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 701 Sena-Esteves, Miguel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57, 450 Sengupta, Sadhak . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 718 Senol, Ozlem . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 665 Senyukov, Vladimir . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479 Senzer, Neil . . . . . . . . . . . . . . . . . . 148, 196, 213, 245, 247, 248, 252, 292, . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 335, 487, 532, 536, 564, 586, 892 Seo, Hae-Hyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 384 Seregin, Sergey S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 638, 651 Sergi Sergi, Lucia . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 313, 316, 545, 613 Serguei, B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26 Servais, Elliot . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184 Serve, Kinta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 330 Sesenoglu-Laird, Ozge . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 445, 622 Sest, Maike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 239 Sethupathy, Praveen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 54 Sevin, Caroline . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19 Seward, Shelly M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250 Seymour, Len . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 602 Seymour, Len W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 732 Shabashvili, Arseniy E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 518, 854 Shafer, Jessica A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 473 Shaffer, Donald . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 471 Shah, Ami . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296 Shah, Rachel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308 Shaheen, Sharif M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 401 Shahrokhi, Somayeh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871 Shammas, Masood A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250 Shan, Changguang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 432 Shanker, Manish . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 243

175

176

American Society of Gene & Cell Therapy

Abstract Author Index Shao, Hongwei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603 Sharif, Faisal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 439 Sharifi, Zohre . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 871 Sharma, Nynne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 726 Shashkova, Elena V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 489 Shaw, Georgina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 270 Shaw, Kit L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296 Shayakhmetov, Dmitry M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 541 Shchelochkov, Oleg A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677 Shea, Lonnie D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 121, 764, 770 Sheehan, Andrea M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 293 Shen, Hui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709 Shen, Shen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 62 Shen, Xuefei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103, 115, 327, 477 Shen, Ying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 713 Shenker, Larissa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185 Shenker, Larrisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184 Sher, Yu-Ping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 844 Shevchenko, Eugeniy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434 Shevelev, Alexander . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434 Shi, Qin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 756 Shida, Hisatoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 34, 840 Shihabuddin, Lamya S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55, 557 Shiku, Hiroshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 652 Shilling, Christopher . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 581 Shilling, Christopher J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155, 290 Shim, Kyu-Woong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Shima, Midori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 903 Shimabukuro-Vornhagen, Alexander . . . . . . . . . . . . . . . . . . . . . . . . . . . 664, 887 Shimada, Hideaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851 Shimada, Takashi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 410, 510, 680 Shimamura, Munehisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 774 Shimizu, Hirofumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 419 Shimizu, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 192 Shimizu, Keiji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 513 Shimizu, Masaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 227 Shimoda, Shinya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 899 Shin, Jin-Hong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 151, 587, 601 Shin, Qiaoyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 731 Shin, Seong-Phil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 384 Shin, Young Eun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 128 Shirakawa, Toshiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167 Shirazi, Rahau S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 393 Shirley, Shawna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 823 Shi-Suo, Du . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73 Shitara, Shingo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236 Shivakumar, Rama . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 675 Shontz, Kimberly . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149 Shoshani, Ehud . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616

EXHIBITOR PROSPECTUS final program

Shovlin, Lindsay . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 41 Shu, Yuelong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 172 Shuai, Lifang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 171, 807, 808 Shui, Hongshu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620 Shukla, Ravi S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 567 Shukla, Vasundhara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 132 Shultz, Leonard D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285 Siddiq, Ishita . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783 Sidow, Arend . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14 Siegal, Gene P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36 Silbergeld, Daniel L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294 Siler, Ulrich . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 233, 869 Silka, Kimberly A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21 Silva, Ana Carina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 579 Silva, Flavia H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440 Simão, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 579 Simioni, Paolo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 615 Simonelli, Francesca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59 Simonsson, Bengt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 83, 820 Simula, Pekka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 177 Singh, Anand Kamal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 132 Singh, Harjeet . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 479 Singh, Rakesh K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 593 Sirrka, Liisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 538 Sitaraman, Varsha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372 Sivakumar, Kumaraswamy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155 Skarica, Mario . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 859 Skiena, Steven . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372 Skinner, Amy M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 692 Skog, Johan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57, 846 Skripchenko, Yelena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 274 Skvorak, K. J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139 Slawin, Kevin M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 474 Sleep, Susan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 668 Sleeper, Meg M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433, 598 Sleer, Leanne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 536 Sloan, Jennifer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135 Slobodkin, Gregory . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893 Sloniecka, Marta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 204 Slovin, Susan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184 Smerdou, Cristian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Smith, Bruce F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 379, 587 Smith, Carol . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Smith, Edvard C. I. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 556 Smith, Jeffrey S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 542 Smith, Laura J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 3 Smogorzewska, Monika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296 Snead, Nicholas M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 888 Snyder, Brooke R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 778, 782

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Snyder, Jenni A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310 Snyder, Richard O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 305, 365 Soballe, Kjeld . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 787 Sobczak, Krzysztof . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 49 Sobolewki, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 425 Soden, Declan M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 736 Soedling, Helen M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 716 Sokolic, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 296 Soldati, Corrado . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295 Söllü, Cem . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 95 Soma, Emilienne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 110, 113 Somanathan, Suryanarayan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 543 Somia, Nikunj V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 221, 232 Sommerfield, Marla . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 488 Somoto, Vivian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440 Sondhi, Dolan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19 Song, Cunxian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 753 Song, Hyung Geun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 406 Song, Jae J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 257 Song, Min-sun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 888 Song, Shuxian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 326 Song, Sihong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 375, 775 Song, Xiao-Tong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 656 Song, Xiatong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 816 Sørensen, Charlotte Brandt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91 Sorenson, Brent . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 852 Sorrentino, Brian P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 222, 354, 860, 861 Sosale, Nisha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 357 Sousa, Marcos F. Q. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 579 Soustek, Meghan S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 596 Sowers, A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 74 Sowers, Anastasia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 77 Spanggaard, Iben . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 106 Sparks, Jeff . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893 Spence, Alexander . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 294 Spencer, David M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288, 474 Spencer, H. Trent . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 527, 814 Spencer, H. Trent B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 230 Spencer, Jacqueline F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 489 Spitalieri, Paola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 908 Spratt, S. Kaye . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783, 798 Sprengel, Rolf . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 241 Spriggs, David R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 183 Springer, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 44, 224, 373 Springfeld, Christoph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 833 Spyridonidis, Alexandros . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 340, 729 Squillace, Karen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 633 Sreenath Nagamani, Sandesh C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677 Srinivasan, Rahul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 781

Srivastava, Arun . . . . . . . . . . . . . 61, 363, 369, 370, 540, 587, 700, 704, 706 Stabenow, Dirk . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312 Stalvey, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 444 Stamatoyannopoulos, George . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137 Stanek, Lisa M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 55, 557 Staunstrup, Nicklas H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 726 Staunstrup, Nicklas Heine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91 Stavrou, Eleana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 340, 729 Stayton, Patrick S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 558 Stedman, Hansell . . . . . . . . . . . . . . . . . . . . . . . 156, 374, 454, 458, 467, 785 Stedman, Hansell H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600 Steel, Jason C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 483 Steet, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 759 Stefanska, Anna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159 Stefanski, Jolanta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32, 184 Steffes, Christopher . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250 Stein, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 646 Stemmer-Rachamimov, Anat O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 665 Stenderup, Karin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 695 Stephenson, Joe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33, 35 Sterling, Brigid V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 226 Stern, Baruch S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 616 Stetler-Stevenson, M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31 Stevenson, Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 732 Stewart, Duncan J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 907 Stirling, Brigid . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 529 Stirling, Brigid V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 343 Stocca, Alessia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 427 Stockdale, Katie R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662, 663 Stoddard, Barry L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 89 Stok, Merel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 315 Stone, Brandon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 514 Stone, Daniel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 8 Storb, Rainer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553, 623 Straathof, Karin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Strauss, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 265 Strom, S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139 Stroup, Katie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 689 Studer, Lorenz . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 283 Su, Leonard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 454 Su, Qin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 362, 450, 671 Suda, Takeshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 322 Sudol, Kelly L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 18 Sudres, Muriel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 552 Suehara, Tetsuya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 670 Suehiro, Youko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263 Suel-Petat, Laurence . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 793 Suga, Tomohiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468 Sugiyama, Daisuke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263

177

178

American Society of Gene & Cell Therapy

Abstract Author Index Sugiyama, O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 687 Sukegawa, Jun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 508 Sule, Gautam . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 46 Sullenger, Bruce A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 561, 667 Sullivan, Millicent O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 590 Sullivan, Timothy A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 441 Sumaroka, Marina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600 Sumimoto, Hidetoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 480 Sumiyoshi, Teiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 522 Sumner-Jones, Stephanie G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880 Sumners, Colin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 518, 854 Sun, Baodong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 47, 136 Sun, Bo K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 257 Sun, Lan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433 Sun, Q. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139 Sun, Samuel S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 326 Sun, Wenchao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 746 Sun, Xingshen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620 Sundaram, Ramakrishnan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 852 Surace, Enrico M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59, 624, 702 Surosky, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 783 Sürth, Julia D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 304 Suwanmanee, Thipparat . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 534 Suzuki, Kei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 184 Suzuki, Masataka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 46, 224, 309, 642 Suzuki, Ryo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 102 Suzuki, Ryosuke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 748 Suzuki, Takayuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 537 Suzuki, Toru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 216 Suzuki, Yumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 585 Swaim, William D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 77 Swain, JaBaris D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600 Swaney, William P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 280 Swanson, Debra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20, 221 Swanson, Debra L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 232 Sweeney, Colin L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 220 Sweeney, H. Lee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433, 625 Swiderski, Piotr . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287 Symens, Nathalie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 742 Sysol, Justin R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135 Sze, Daniel Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 214 Szpirer, Cedric Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 740

T

Tabata, Toshiaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303, 634 Tada, Yuji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851 Tadlock, Matthew V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 408 Tafuro, Sabrina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 140 Tagalakis, Aristides . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 747

EXHIBITOR PROSPECTUS final program

Tagawa, Masatoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851 Tahan, V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 139 Tahara, Hideaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 34, 840 Tai, April . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 689 Tai, Ching-Lung . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583 Tai, Khalid . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 350 Tak, Paul P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 231, 548, 870 Tak, Paul-Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 66 Takahashi, Atsushi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 263, 899 Takahashi, Masamichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 832 Takahashi, Rei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 862 Takahashi, Yoko E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 102 Takahashi, Yuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 670, 862 Takakura, Yoshinobu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 670, 862 Takeda, Kiyoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 480 Takeda, Shin’ichi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56, 151, 565, 582, 585 Takeya, Yasushi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 884 Takiya, Christina M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440 Talbot, Gill . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 158 Tamai, Katsuto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 187, 628 Tampier, Lutske . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 766 Tamura, Masakazu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 513 Tan, L. G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 697 Tan, M. H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 697 Tan, Shuping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 376 Tan, Wenjie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 78, 114, 172, 238 Tang, Qiushi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 51, 53 Tang, Ruhang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 420 Tang, Terence . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218 Tang, Yaoping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 677 Tang, Ying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 635 Tang, Yucheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 85 Tangney, Mark . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 398, 705, 812, 848 Tanguay, Robert M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242 Tani, Kenzaburo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 192, 263, 899 Tani, Tomomi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 591 Tannous, Bakhos A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57 Tansey, Malu G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 773 Tapscott, Stephen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553 Tapscott, Stephen J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 623 Tarantal, Alice F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 522 Tarragona, Tony . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Tateno, Chise . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 141 Tatsumi, Kohei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 141, 903 Tatsuzawa, Osamu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678 Taube, Ran . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645 Tavernier, Geertrui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 261 Tayler, Matthew A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 191 Taylor, Catherine A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Taylor, Clare . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32, 911 Taylor, Justin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659 Telen, Marilyn J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 667 Tellez, Joe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 857 Tellez, Joseph . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 551 Temme, Achim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 833 Templeton, Nancy S. . . . . . . . . . . . . . . . . . . . . . . . . . 247, 564, 574, 586, 731 ten Bloemendaal, Lysbeth . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 138 Tenenbaum, Lilianne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 708 Tennant, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880 Terada, Nobuyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 500 Terakura, Seitaro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 618 Terao, Shuji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 216 Tesarfreund, Matthew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 475 Testa, Francesco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59 Teter, Mary A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 847 Tey, Siok K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Thaci, Bart . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 835 Thakur, Garima . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 132 Thatava, Tayaramma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 633 Theobald, Narda . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 354 Thibault, Marc . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 396 Thibodeau-Beganny, Stacey . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 95 Thiel, Kristina W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662, 663 Thiel, William H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 662, 663 Thomas, Darby L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 377, 571 Thompson, Colin J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 254 Thompson, Jill . . . . . . . . . . . . . . . . . . 175, 481, 496, 606, 607, 610, 611, 653 Thompson, John E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218 Thompson, Patrick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 767 Thornton, Charles A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 49 Thrasher, Adrian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 158, 617, 647 Thrasher, Adrian J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 866, 229 Throm, Robert E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 354, 861 Thung, Swan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38 Thurlings, Rogier . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 548 Tian, Jie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 542 Tian, Jun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 358 Tian, Runxia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603 Tian, Wenhong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 238, 376 Tiboni, Francesca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 319, 523 Tiemann, Katrin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 555 Tikoo, Suresk K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 724 Tilstra, Jeremy S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 629, 635 Timares, Laura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 379 Tisdale, John F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 352 Tkachuk, Vsevolod A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434 Tobita, Kimimasa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 703 Todo, Tomoki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 832, 839

Todorow, Carlyn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 344, 676 Toietta, Gabriele . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 898 Toita, Nariaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678 Tojyo, Masahiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 825 Tol, Nikki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 40, 502 Toma, Shoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 826 Tomasulo, Catherine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 600 Tomizuka, Kazuma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 236 Tong, Alex W. . . . . . . . . . . . . . . . . . . 148, 196, 245, 248, 252, 292, 335, 564 Tonne, Jason M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 599 Torbett, Bruce E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 348, 650 Torrente, Yvan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 460, 464, 795 Toscano, Miguel G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 859 Toth, Karoly . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 489, 837 Totterman, Thomas H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 176 Toumadje, Araz . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 905 Toutouna, Louiza . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 729 Towers, Greg . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 647 Townsend, Dewayne . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 584 Toyota, Hiroyasu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 862 Tran, Kaylie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 64 Tran, Kaylie N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169 Tran, Rosa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 361 Trani, Marianna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 430 Transfiguracion, Julia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 361 Tremblay, Jacques P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 462, 769, 784, 790 Trevino, Isaac . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 773 Tripic, Tamara . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 473, 799 Trobridge, Grant D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 909 Trochon-Joseph, Veronique . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 110, 113, 853 Trueck, Christina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693 Trujillo, Miguel A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 80, 505 Truong, Lynn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 798 Trygg, Cynthia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314 Tsai, Richard K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 357 Tschaika, Marina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 189, 664, 822, 887 Tschernia, Nick . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 551 Tschulena, Ulrich . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 339 Tsokolaeva, Zoya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434 Tsokos, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135 Tsukahara, Tomonori . . . . . . . . . . . . . . . . . . . . . . . . . 207, 421, 428, 544, 554 Tsuno, Takaya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 482 Tsuzuki, Teruhisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 97 Tuppurainen, Venla . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 212 Turchetto, Lucia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276 Turnis, Meghan E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 816 Turrell, Susan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 215 Tuschong, Laura M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234, 524, 858 Tuunanen, Pasi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159

179

180

American Society of Gene & Cell Therapy

Abstract Author Index Tuve, Sebastian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Tyr, Elena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Tyr, Olena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Tysome, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Tzekov, Radouil . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .

180 445 622 605 444

U

Uchibori, Ryosuke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 207 Uchida, Hiroaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 640 Uchida, Naoya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 352 Uchida, Yuji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468 Uchil, Pradeep . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 285 Uchino, Makoto . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468, 788 Uchio, Katsuhisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468 Uchiyama, Masayuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 97 Uchiyama, Toru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 227 Ueda, Yasuji . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 824 Uesugi, Motonari . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 670 Uhrig, Silke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5 Uitto, Jouni . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320 Ukawa, Masami . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 743 Ulasov, Ilya V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 191, 718, 835 Ulkoski, David . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893 Ulrich-Vinther, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 787 Uludag, Hasan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 129, 562, 568, 755 Umino, Yumiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17 Ungari, Silvia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 316 Ungaro, Daniela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 315 Ungerechts, Guy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 833 United Dystrophinopathy Project Consortium . . . . . . . . . . . . . . . . . . . . . . . . . 152 Uno, Yoshitaka . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 259 Unzu, Carmen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 42 Urabe, Masashi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 207, 421, 428, 544, 554 Urasawa, Nobuyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56 Urata, Yasuo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 192 Urban, Jon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 402 Urban, Nicole . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 265 Urness, Myra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 325 Urtti, Arto O. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 750 Usachenko, Yelena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 815 Utoh, Rie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 141, 419

V

Vähäkangas, Elisa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 765 Völkel, Christine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284 Vago, Luca . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 295 Vaha-Koskela, Markus . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39 Valdmanis, Paul N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 54 Valero, Valderez B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440

EXHIBITOR PROSPECTUS final program

Vallanti, Giuliana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225, 276, 613 Valles-Ayoub, Yadira . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 772, 794 Valley, Robert . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 328 Valli, Emanuele . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 258 Valouev, Anton . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14 van Beusechem, Victor W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 40, 502 van de Loo, Fons A. J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 559, 627 van den Berg, Fiona . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 563 van den Berg, Wim B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 559, 627 van der Loo, Johannes C. M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 280 van der Meulen-Muileman, Ida . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 40 van Deutekom, Judith C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 153 van Deventer, Sander . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 255 Van Dusen, Nate . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 353 van Logtenstein, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 249 van Ommen, Gert-Jan B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 153 Van Pijkeren, Jan P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 398 Van Poucke, Mario . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 875 van Praag, Henriette . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 355 van Rensburg, Ruan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 93 van Rooijen, Nico . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 380, 723 van Sinderen, Douwe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 848 van Til, Niek P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 530, 617 Van Vliet, Kim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 65 Van Zwieten, Ruthger . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 533 Vandenberghe, Luk H. . . . . . . . . . . . . . . . . . . . . . . . . . . . 1, 65, 368, 412, 759 Vanier, Marie-Thérèse . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19 Vanrell, Lucia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 485, 708 Varghese, Steve . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 773 Varki, Ajit . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 546 Varney, Michelle L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 593 Vasan, Sandhya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 162 Vasseur, Virginie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 552 Vaughan, Andrew E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 623 Vaughan, Erin E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 424, 588 Veenbergen, Sharon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 559 Velazquez, Omaida C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603 Venditti, Charles P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135, 679, 701 Vera, Juan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 469 Vera, Juan F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 28, 29, 672, 799 Verhoeyen, Els . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 336 Vermeij, Eline A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 627 Vermeulen, Christie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 40, 502 Verstegen, Monique M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617 Vervoordeldonk, Margriet J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 66, 231, 548 Vetrini, Franesco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 388 Viale, Diego L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 609, 834 Viapiano, Mariano . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 199 Viarengo, G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 360

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Vickers, Selwyn M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 391, 497 Viita, Helena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 512 Vile, Richard . . . . . . . . . . . . . . . . . . . 175, 481, 496, 606, 607, 610, 611, 653 Vile, Richard G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 208 Villa, Anna . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 225, 617 Villani, Edoardo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59 Vincent, Justin M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 572, 575, 738 Vinogradov, Serguei V. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 593 Vinti, Maria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 777 Viola, Francesco . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 59 Viollet, Laurence . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155 Virk, Mandeep S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 687 Virtanen, Siru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 145 Visel, Meike . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 442 Visigalli, Ilaria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 313, 315, 316 Visser, Trudi P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 530, 617 Vitetta, Ellen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Vlasik, Tatyana . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 434 Vleck, Jan P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 856 Vo, Thuy D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 98 Vogel, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 525 Vogler, Carole . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 768 Voigtlander, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 385 Voit, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 154 Volpin, Monica . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 298 Volz, Baerbel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 189, 822 von Bergwelt-Baildon, Michael S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 664, 887 von Kalle, Christof . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 286, 305 Von Kalle, Christof . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 345 von Kalle, Christof . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 347, 612, 833 von Messling, Veronika . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 169 VonKalle, Christof . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297, 298 Vorhies, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 564 Voss, Tiffini . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 779 Vosters, Jelle L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 870 Voutetakis, Antonis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 143 Vrettou, Christina . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 880

W

Wada, Jun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167 Waddington, S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 223, 697 Wade-Martins, Richard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 602, 732 Wadhwa, Renu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 242, 841 Wagemaker, Gerard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 530, 617 Waisbren, Emily . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237 Waite, Katherine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39 Wakimoto, Hiroaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 608, 829 Walker, Christopher M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149, 155, . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 290, 581

Walker, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 685 Wall, Cheryl . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155 Wallraven, Gladice . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 292, 520 Wan, Wen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36 Wancewicz, Edward . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 557 Wanders, Alkwin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 176 Wandless, Tom J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 350 Wang, Adele Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 79 Wang, Anqi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 171, 807, 808, 854 Wang, Bing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 457, 635 Wang, Cathy X. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 348 Wang, Danher . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 504, 643 Wang, Gang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 238, 376 Wang, Gary P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13 Wang, Hao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 453 Wang, Hong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 515 Wang, Hongjie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 415, 657, 843 Wang, Hongxun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 195, 495 Wang, Huan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 416 Wang, Hui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 200 Wang, James . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 341 Wang, Jenny . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39 Wang, Jianbin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11, 801 Wang, Jun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 422 Wang, Li . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 433 Wang, Lijun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 377, 571 Wang, Lili . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 45, 416 Wang, Minghui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36, 503, 834 Wang, Mingxing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 890 Wang, Nanya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 510 Wang, Pengju . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605 Wang, Pin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 686, 689 Wang, Pin-Yi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 498 Wang, Qiang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58 Wang, Shengdian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605 Wang, Wei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 347 Wang, Weijun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 200 Wang, Wen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 172 Wang, Wenjing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 171, 806, 808 Wang, Xinzhong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 517 Wang, Xiuyan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32, 184, 911 Wang, Yaohe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 605 Wang, Yifeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 186 Wang, Yong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 753 Wang, Yuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 266 Wang, Yue . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 694 Wang, Yuhua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 329 Wang, Yun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 709 Wang, Zejing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 553, 623

181

182

American Society of Gene & Cell Therapy

Abstract Author Index Wang, Zhaohui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 245, 247, 532, 564, 892 Wang, Zhongya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 306, 681, 682 Wanisch, Klaus . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451 Ward, Charles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372 Wargo, Jennifer A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 190 Warlich, Eva . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 284 Washington, Kareem N. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 352 Watanabe, Yoshihiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 862 Watcharanurak, Kanitta . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 862 Watts, Ashlee A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 912 Watts, Korashon L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 631 Watts, Victoria . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 155 Weaver, Donald W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 250 Webb, Craig . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 487 Weber, Christian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 305 Weber, Christophe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 432 Weihrauch, Martin R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 664, 887 Weiland, Ola . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 289 Weinberg, Marc S. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 563 Weiner, D. B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648 Weiner, David B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 103, 115, 327, 477 Weinfeld, Michael . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 568 Weiss, Robert B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 152 Wenzel, Daniela . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693 Werfel, Stanislas M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 241 Wesa, Amy K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 271 Westenberg, Marcel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 716 Wheeler, Thurman M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 49 White, Edward A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394 Whitehouse, Adrian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 215 Whitley, Chester B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20 Wibowo, Mia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181 Wicke, Daniel C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 318 Wielgosz, Matthew M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 733 Wilber, Andrew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 338, 339 Wilen, Craig B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 801 Wilkinson, Leslie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 893 Willenbring, Holger . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 910 Willerth, Stephanie M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 802 Williams, Bradley A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 796 Williams, James A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 166, 572, 575, 738 Williams, Jon P. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 201 Williams, Kyha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 142 Willmon, Candice L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 611 Wilson, Aisha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 412 Wilson, Carolyn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 649 Wilson, James M. . . . . . . . . . . . 1, 45, 65, 164, 368, 412, 416, 543, 625, 759 Wilson, John . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 672 Wilson, Kyle A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 273

EXHIBITOR PROSPECTUS final program

Wilson, Matthew H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12, 15, 179, 472, 813 Wilson, W. H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31 Wilton, Steve D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 789 Wimley, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 314, 411 Wimmer, Eckard . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 372 Windebank, Anthony J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 780 Winnik, Francoise M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 756 Winterton, Matthew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659 Wipf, Peter . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 157 Wirth, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 212, 512 Wittig, Burghardt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 189, 664, 822, 887 Witting, Scott R. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 52, 675 Wohlgensinger, Vital . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 233, 869 Wolchok, Jedd D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 817 Wold, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 196, 496 Wold, William S. M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 489, 837 Wolf, Daniel A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20 Wolf, Gernot . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 726 Wolfe, John H. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 317, 901 Wolff, Jon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 160 Wolff, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 430, 597 Wong, Fay E. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645 Wong, Kamehameha K. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 3 Wonganan, Piyanuch . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 435 Wongsrikeao, Pimprapar . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 804 Wongthida, Phonphimon . . . . . . . . . . . . . . . . . . . . . . . . . . . 175, 481, 607, 610 Woo, Savio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38, 277 Wood, Josh . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 857 Woodward, Josha A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 692 Worgall, Stefan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 161, 452, 752 Worgall, Stefen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 163 Worley, Kim C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 574 Wozniak, David F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 768 Wright, J. F. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 308 Wright, J. Fraser . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 614 Writer, Michele J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 394 Wu, Bo . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 890 Wu, Chih-Hsien . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 882 Wu, Hao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 84 Wu, Lily . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 714, 850 Wu, Ping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 390 Wu, Robert A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 864 Wu, Shourong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 891 Wu, Susan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 598 Wu, Xiaobing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 78, 238, 376 Wu, Yihan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 839 Wunder, Stephanie L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 757 Wurdinger, Thomas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 665

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index X

Xagorari, Angeliki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137 Xia, Danny . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 98 Xia, Xuexing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 737 Xiao, Bin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 683 Xiao, Ping-Jie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 60 Xiao, Ru . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1, 65, 368 Xiao, Weidong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 310 Xiao, Xiao . . . . . . . . . . . . . . . . . . . . . . . . . . 6, 160, 420, 457, 581, 683, 790 Xiao, Yan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 332 Xie, Boxun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38 Xie, Jun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 362, 450, 671 Xie, Qing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 362, 450 Xiong, Weidong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26 Xiong, Zhengming . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 826 Xiuying, Liu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 663 Xu, Acong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 467 Xu, Xiabin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423 Xu, Yaqin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 163 Xu, Zhili . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 542 Xu, Zuoshang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 450 Xue, Shao-An . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 349

Y

Yáñez-Muñoz, Rafael J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451 Yadav, Swati . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 160 Yagi, Hiroya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 421, 544, 554 Yagiz, Kader . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 26, 181, 475 Yaguchi, Tomoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 841 Yamada, Masafumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 678 Yamada, Meiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 192 Yamada, Yuma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 748 Yamaguchi, Saori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 899 Yamaguchi, Tomoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 537 Yamamoto, Masato . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 80, 391, 497 Yamamoto, Motoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 410 Yamamoto, Sakura . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 167 Yamamoto, Seiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 680 Yamaoka, Norie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 500 Yamasaki, Satoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 391 Yamashita, Atsushi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 401 Yamato, Masayuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 419 Yamazaki, Takehiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 187, 628 Yamazoe, Sayumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 670 Yamoshita, Satoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 468 Yan, J. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 648 Yan, Jian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 115, 327, 477 Yan, Ziying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4, 620 Yanagisawa, Teruyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 508

Yang, Bin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 450 Yang, James C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25 Yang, Lin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 6 Yang, Ningning . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 741 Yang, Shan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 851 Yang, Sherry W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 507 Yang, Shicheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 476, 478 Yang, Wendy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 557 Yang, Xiao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 69, 885 Yang, Xiao Yi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 47 Yang, Y. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 913 Yang, Ye . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 130 Yanik, Tulin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 143 Yannaki, Evangelia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137 Yao, Feng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237 Yaouanc, Jean Jacques . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 745 Yardeni, Tal . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 144 Yarnall, Charles . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 156 Yasukawa, Masaki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 652 Yasumura, Eduardo G. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 440 Yawata, Toshio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 513 Ye, Bin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 218 Ye, Delia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 165 Ye, Guo-jie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 377, 571 Ye, L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 913 Ye, Peiqing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 535, 550 Ye, Yihong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 876 Yeagy, Brian A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 408 Yeh, Raymond . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 32, 815 Yehling, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 23 Yen, Benjamin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 535 Yen, Tzu-Chen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 583 Yeung, Jonathan C. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 872 Yi, Yaling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 620 Yin, Xiao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 78, 114 Yip, Ping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 451 Ylä-Herttuala, Seppo . . . . . . . . . . . . . . . . . . . . . 159, 209, 212, 512, 669, 765 Ylaya, Kris . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 659 Yockman, James W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 86, 126 Yokota, Takanori . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 259 Yokota, Toshifumi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 56, 565 Yonemitsu, Yoshikazu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 824 Yong, Liu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 819 Yongabi, Felicitas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 159 Yoo, Ji Young . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 198, 811, 841 Yoo, Ji-Young . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 836 Yoon, A-Rum . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 84, 198, 720 Yoon, DoHeum . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 260 Yoon, Sang Soon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 406

183

184

American Society of Gene & Cell Therapy

Abstract Author Index Yoshida, Kie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 259 Yoshihara, Chieko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 825 Yoshikawa, Hiroyuki . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 554 Yoshimura, Toshiro . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 585 Yoshizato, Katsutoshi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 141 You, Chang Xuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 819 You, Chia Chin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 173 Younes, Anas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 473 Yu, Di . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 501, 828 Yu, Gang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 105 Yu, Hongwei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 412, 759 Yu, Jimin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 411 Yu, Julie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 299 Yu, Seungshin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 818 Yu, William . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 713 Yu, Yang . . . . . . . . . . . . . . . . . . . . . . . . . 248, 275, 292, 335, 487, 532, 564 Yu, Zhiya . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 190, 478 Yuan, C. M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 31 Yuan, Jinyun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 650 Yuan, Zhenhua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 6, 683 Yue, Yongping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 150, 466, 587, 601 Yuen, Carrie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 809 Yu-Feng, Lin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288 Yugeta, Naoko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 582 Yui, Nobuhiko . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 401 Yumul, Roma . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 180, 264, 843, 849, 863 Yun, Chae-Ok . . . . . . . . . . . . 84, 86, 188, 198, 202, 242, 516, 720, 811, 841 Yun-shan, Tan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73 Yurek, David M. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 776 Yusa, Kosuke . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 15 Yvon, Eric . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 288

Z

Zacchigna, Serena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 423, 431 Zahid, Maliha . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 7 Zaitseva, Marina A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 735 Zamore, Phillip D. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 362 Zaoui, Karim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 305 Zavaljevski, Maja . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 730 Zeigerman, Haim . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 719 Zenadi, Rahima . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 667 Zeng, Jinfeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 807 Zeng, Lepeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 402 Zeng, Yué . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 463 Zeng, Zhao-Chong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 211 Zentilin, Lorena . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 311, 423, 431 Zerah, Michel . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19 Zerfas, Patricia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135 Zervou, Fani . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 137

EXHIBITOR PROSPECTUS final program

Zhan, Jun . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 733 Zhang, Dazhi . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 819 Zhang, Fang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617 Zhang, Feijie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 306 Zhang, Guisheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 322 Zhang, Hongwei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 362, 450, 593 Zhang, Jane . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287, 661 Zhang, L. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 178 Zhang, Lin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 775 Zhang, Ling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 219, 476, 478, 808, 854 Zhang, Ping . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 47 Zhang, Shuyuan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 274, 803 Zhang, Song-Nan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 202, 811 Zhang, Ting . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 493 Zhang, Wei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 574, 876 Zhang, Wenli . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 92, 636 Zhang, Xiaoling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 756 Zhang, Xiao-Xiang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 392 Zhang, Xichen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 515 Zhang, Xiongwen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 217 Zhang, Yadong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 876 Zhang, Yanling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 219 Zhang, Yonggong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 251 Zhang, Yonghui . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 694 Zhang, Zheng-Yu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 211 Zhao, Hui Fen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 867 Zhao, Huifen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 524, 525 Zhao, Ling . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 789 Zhao, Lu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 647 Zhao, Tongmao . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 482 Zhao-Chong, Zeng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73 Zheng, Changyu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 77, 143, 761 Zheng, Heng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 580 Zheng, Xin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 808 Zheng, Zhili . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 476, 478 Zheng-Yu, Zhang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73 Zhong, Li . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 58, 61, 363, 369, 370, 700 Zhong-shan, Liu . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 73 Zhou, Dongming . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 389 Zhou, Jiehua . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287, 555 Zhou, Jingmin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 69, 673 Zhou, Liqin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 15 Zhou, Shangzhen . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43, 546, 615, 630, 885 Zhou, Sheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 222, 860 Zhou, Tian . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 108 Zhou, Xiangyang . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 389 Zhou, Xianzheng . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 868 Zhou, Xiaoou . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 656 Zhu, Liwei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 186, 566

13th AnnUAL MEETing | Washington, DC USA May 19-22, 2010

Abstract Author Index Zhu, Quan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 645 Zhu, Wen . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 163 Zhu, Zeng B. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 834 Zhuge, Ying . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 603 Zidovska, Alexandra . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 393 Ziliotto, Raul . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 276 Zimmermann, Katrin . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 693 Zinn, Kurt . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36 Zolopa, Andrew . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 646 Zolotukhin, Irene . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 763 Zolotukhin, Sergei . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 61, 371, 378 Zoltick, Philip W. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 320, 344, 357, 676 Zoon, Kathryn . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 482 Zou, Jie . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 130 Zou, Jizhong . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 220 Zoumbos, Nikolas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 340, 729 Zourelias, Lee . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 104 Zubair, Adnan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 689 Zuhaili, Baraa . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 237 Zurita, Juan . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185 Zweier-Renn, Lynnsey A. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 342, 690

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American Society of Gene & Cell Therapy

Notes

EXHIBITOR PROSPECTUS final program

Mark Your Calendars!

American Socie t y of Gene & Cell Therapy

14th Annual Meeting S e at t l e , WA

|

May 1 8 - 2 2 , 2 0 1 1

Exciting scientific session topics will include: - Stem Cell Therapeutics - Oligonucleotide Therapies - Novel Vector Development - Mechanisms of Vector Transduction - Host-Vector Interactions

ASGCT-1209-226

- Clinical Trial Advancements