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FINAL PROGR AM - American Society of Gene & Cell Therapy

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<strong>AM</strong>ERICAN SOCIETY OF GENE THERAPY • Final Program 111<br />

ABSTRACTS<br />

SATURDAY, JUNE 4<br />

1067<br />

Regulated Expression <strong>of</strong> Biotherapeutic Proteins and<br />

shRNA Using an Ecdysone Receptor-Based <strong>Gene</strong> Switch<br />

Prasanna Kumar, Anand K. Katakam, Chris L. Herold<br />

1068<br />

Luciferase as an In Vivo Reporter for Selective Liver<br />

Repopulation in Adult FAH-/- Mice<br />

Kirk J. Wangensteen, Andy Wilber, Guisheng Zeng, Joel L.<br />

Frandsen, R. Scott McIvor, Stephen C. Ekker, Xin Wang<br />

1069<br />

Increased Antioxidant Levels Following MnSOD-PL<br />

Transfection Results in Increased Radioprotection <strong>of</strong><br />

Normal Tissue but Not Tumor <strong>Cell</strong>s<br />

Michael W. Epperly, Suhua Nie, Xichen Zhang, Joel S.<br />

Greenberger<br />

1070<br />

Easy Search on Clinical <strong>Gene</strong> Transfer: The Euregenethy<br />

(EC-DG Research FP5-TN) Data Base on Pre-Clinical &<br />

Clinical <strong>Gene</strong> Transfer & <strong>Therapy</strong><br />

Odile Y. D. Cohen-Haguenauer, Fatima Bosch, Manuel<br />

Carrondo, Klaus Cichutek, Nicole Deglon, Barbara Demeneix,<br />

George Dickson, Gosta Garhton, Bernd Gansbacher, Cecilia<br />

Melani Luigi Naldini, Felicia Rosenthal, Sandro Rusconi,<br />

Seppo Yla-Herttuala, Heinz Zwierzina<br />

1071<br />

Potential Utility <strong>of</strong> Adipose-Derived Stromal <strong>Cell</strong>s<br />

(ASCs) as a Carrier for Ex Vivo <strong>Gene</strong> <strong>Therapy</strong> – <strong>Gene</strong><br />

Transfer Efficiency <strong>of</strong> ASCs<br />

Rei Ogawa, Hideki Hanawa, Yukihiko Hirai, Toshiyuki<br />

Kurai, Hiroshi Mizuno, Hiko Hyakusoku, Takashi Shimada<br />

1072<br />

Superactivation <strong>of</strong> the SRað and CMV Promoters Using<br />

Designed Zinc Finger Proteins<br />

Victor V. Bartsevich, Andreas Reik, Yuanyue Zhou, Michelle<br />

Kong, Xiao-Yong Li, Lyndon Warfe, Lei Zhang, Casey C.<br />

Case, Trevor Collingwood, Philip D. Gregory Edward J. Rebar<br />

1073<br />

Down-Regulation <strong>of</strong> Epithelial Glycoprotein-2 and<br />

VEGF-D by Engineered Zinc Finger Protein Transcription<br />

Factors<br />

Willemijn M. Gommans, Robbert H. Cool, Pamela M. J.<br />

McLaughlin, Olli H. Laitinen, Seppo Yla-Herttuala, Bert J.<br />

van der Zaal, Hidde J. Haisma, Marianne G. Rots<br />

SUNDAY, JUNE 5<br />

Oral Abstract Session 510<br />

Clinical <strong>Gene</strong> <strong>Therapy</strong><br />

Room: 240 Complex<br />

1074 10:15 <strong>AM</strong><br />

The Use <strong>of</strong> <strong>Gene</strong>tically Modified Antigen Presenting<br />

<strong>Cell</strong>s to <strong>Gene</strong>rate Donor-Derived Virus-Specific Cytotoxic<br />

T Lymphocytes Reactive Against CMV and Adenoviral<br />

Antigens for Clinical Use<br />

Gary D. Myers, Ann Leen, Helen Huls, Elizabeth Buza, Jeff<br />

Chang, Robert A. Krance, George Carrum, Malcolm K.<br />

Brenner, Cliona M. Rooney, Helen E. Heslop Catherine M.<br />

Bollard<br />

1075 10:30 <strong>AM</strong><br />

Phase I Clinical Trial Demonstrates Safety and Feasibility<br />

<strong>of</strong> Autologous <strong>Cell</strong>ular <strong>Therapy</strong> with Lentiviral Modified<br />

CD4 T <strong>Cell</strong>s Expressing Anti-HIV Antisense in Patients<br />

with HAART Resistant HIV-1 Infection<br />

Boro Dropulic, Rob-Roy MacGregor, Tessio Rebello, Peter<br />

Manilla, Laurent Humeau, Xiaobin Lu, Vladimir Slepushkin,<br />

Gwendolyn K. Binder, Cathy Ybarra, Bruce L. Levine Frederic<br />

Bushman, Carl H. June<br />

1076 10:45 <strong>AM</strong><br />

Stable Polyclonal Hematopoietic Repopulation after<br />

Successful Clinical <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Chronic Granulomatous<br />

Disease (CGD)<br />

Manfred Schmidt, Kerstin Schwarzwaelder, Marion G. Ott,<br />

Stefan Stein, Hanno Glimm, Annette Deichmann, Ulrich<br />

Sieler, Dieter Hoelzer, Reinhard Seger, Manuel Grez Christ<strong>of</strong><br />

von Kalle<br />

1077 11:00 <strong>AM</strong><br />

Phase I Clinical Trial <strong>of</strong> Recombinant Adeno-Associated<br />

Virus (rAAV)-Alpha-1 Antitrypsin Vectors<br />

Mark L. Brantly, Margaret Humphries, Sihong Song, Thomas<br />

Conlon, Amy Poirier, Barry J. Byrne, Richard Snyder, Terence<br />

R. Flotte<br />

1078 11:15 <strong>AM</strong><br />

Therapeutic Misconception in Early Phase <strong>Gene</strong> Transfer<br />

Trials<br />

Gail E. Henderson, Michele E. Easter, Catherine Zimmer,<br />

Nancy M. P. King, Arlene M. Davis, Barbra Bluestone<br />

Rothschild, Larry R. Churchill, Benjamin S. Wilfond, Daniel<br />

K. Nelson<br />

1079 11:30 <strong>AM</strong><br />

Successful Myoblast Transplantation to Duchenne<br />

Muscular Dystrophy Patients<br />

Jacques P. Tremblay, Daniel Skuk, Sylvain Michel, Raynald<br />

Roy, Jean-Guy Lachance, Hélène Senay, Deschênes Louise,<br />

Goulet Marlyne, Brigitte Roy, Jean-Pierre Bouchard

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