FINAL PROGR AM - American Society of Gene & Cell Therapy
FINAL PROGR AM - American Society of Gene & Cell Therapy
FINAL PROGR AM - American Society of Gene & Cell Therapy
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<strong>AM</strong>ERICAN SOCIETY OF GENE THERAPY • Final Program 103<br />
ABSTRACTS<br />
SATURDAY, JUNE 4<br />
Neurodegenerative Diseases<br />
939<br />
Viral Vector-Mediated Chaperone <strong>Gene</strong> <strong>Therapy</strong> in a<br />
Mouse Model <strong>of</strong> Sporadic Parkinson Disease<br />
Zhizhong Dong, David P. Wolfer, Hans-Peter Lipp, Hansruedi<br />
Bueler<br />
940<br />
Neuroprotective Effects <strong>of</strong> XIAP <strong>Gene</strong> <strong>Therapy</strong> in<br />
Models <strong>of</strong> Neurodegenerative Diseases<br />
Sergei Musatov, Joshua Goldfein, Justin Fraser, John Pena,<br />
Michael G. Kaplitt<br />
941<br />
Delayed GDNF Exposure to Mature Dopaminergic<br />
Grafts Impairs Their Ability To Improve Motor Function<br />
in Parkinsonian Rats<br />
Biljana Georgievska, Christian Winkler, Thomas Carlsson,<br />
Benjamin Lacar, Deniz Kirik<br />
942<br />
Evaluation <strong>of</strong> Short (8 Weeks) and Long-Term (3 and 5<br />
Years) <strong>Gene</strong> Transfer Following Brain Delivery <strong>of</strong> AAV-<br />
AADC Vector in Parkinsonian Monkeys<br />
Marcel M. Daadi, Philip Pivirotto, John Bringas, Janet<br />
Cunningham, John Forsayeth, Jamie L. Eberling, Kryst<strong>of</strong> S.<br />
Bankiewicz<br />
943<br />
Anti-alpha-Synuclein scFv Study<br />
Chun Zhou, Anne Messer<br />
944<br />
Silencing <strong>of</strong> Endogenous Human að-Synuclein by a<br />
Lentiviral Vector Expressing Short Hairpin RNA<br />
Mohan K. Sapru, Jonathan W. Yates, Martha C. Bohn<br />
945<br />
Prevention <strong>of</strong> Onset <strong>of</strong> Parkinson’s Disease by In Vivo<br />
<strong>Gene</strong> Transfer <strong>of</strong> Human Hepatocyte Growth Factor in<br />
Rodent and Primate Model<br />
Hiromi Koike, Akihiko Ishida, Munehisa Shimamura,<br />
Naoyuki Sato, Takuya Hayashi, Ryuichi Morishita<br />
946<br />
CD8 + T <strong>Cell</strong>s Are Partially Mediate the Immune-<br />
Mediated Silencing <strong>of</strong> Adenoviral Vector-Encoded<br />
Transgene Expression in the Rat Brain<br />
Carlos Barcia, Clare Thomas, James F. Curtin, Jeffrey M.<br />
Zirger, Weidong Xiong, Olivier Boyer, David Klatzmann,<br />
Maria G. Castro, Pedro R. Lowenstein<br />
947<br />
Effects <strong>of</strong> Natural Pre-Existing Humoral Immunity to<br />
AAV on Administration <strong>of</strong> AAV2-NTN to the Brain <strong>of</strong><br />
Non-Human Primates<br />
Marie A. Printz, Christopher D. Herzog, Alistair Wilson,<br />
Dawn Gammon, Richard Lin, Roberto Calcedo, Biplob Dass,<br />
Medhi Gasmi, Jeffrey H. Kordower, Raymond T. Bartus<br />
948<br />
Improvement <strong>of</strong> Symptoms <strong>of</strong> Parkinson’s Disease by In<br />
Vivo <strong>Gene</strong> Transfer <strong>of</strong> Human Hepatocyte Growth<br />
Factor - A Model <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> for Parkinson’s<br />
Disease<br />
Hiromi Koike, Akihiko Ishida, Munehisa Shimamura,<br />
Naoyuki Sato, Ryuichi Morishida<br />
949<br />
AAV-Parkin <strong>Gene</strong> <strong>Therapy</strong> for Alpha-Synucleinopathy as<br />
Parkinson’s Diseased Model<br />
Hideki Mochizuki, Masanori Yamada, Yoshikuni Mizuno<br />
950<br />
Biodistribution and Safety Pr<strong>of</strong>ile <strong>of</strong> CERE-120 (AAV2-<br />
NTN) Delivered to the Rodent Brain<br />
M. Landers, K. Kwok, E. Ketchum, C. Wilson, R. Lin, D.<br />
Gammon, M. Austin, L. Manlapaz, A. Wilson, J.<br />
Cunningham D. Vacante, M. Gasmi, K. Bishop, M. Printz, R.<br />
Bartus<br />
951<br />
Evaluation <strong>of</strong> HSV-Mediated Alzheimer <strong>Gene</strong> <strong>Therapy</strong><br />
Vectors Using Novel Alzheimer Mouse Model: Applications<br />
<strong>of</strong> siRNA and Neprilysin Vectors to Adjust the<br />
Metabolic Imbalance <strong>of</strong> Abð in Alzheimer’s Disease<br />
Chang-Sook Hong, James R. Goss, William F. Goins, Joseph<br />
C. Glorioso<br />
952<br />
Effects <strong>of</strong> rAAV-BDNF Expression in the Striatum <strong>of</strong> the<br />
R6/1 Mouse<br />
Edgardo Rodriguez, Eileen Denovan-Wright, Ron J. Mandel<br />
953<br />
Progressive and Selective Striatal Degeneration in<br />
Primary Neuronal Cultures Using Lentiviral Vector<br />
Coding Mutant Huntingtin Fragment<br />
Nicole Deglon, Diana Zala, Alexandra Benchoua, Patrick<br />
Aebischer, Anne D. Zurn, Emmanuel Brouillet<br />
954<br />
Demonstration <strong>of</strong> In Vivo Off-Targeting Using rAAV-<br />
Delivered Ribozymes and shRNA Molecules in a Mouse<br />
Model <strong>of</strong> Huntington’s Disease<br />
Edgardo Rodriguez, Eileen Denovan-Wright, Alfred S. Lewin,<br />
Ron J. Mandel
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