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 53<br />
ABSTRACTS<br />
THURSDAY, JUNE 2<br />
AAV Vectors: Disease Applications<br />
110<br />
Performance <strong>of</strong> Different AAV Serotype Vectors Following<br />
Injection into the Deep Cerebellar Nuclei <strong>of</strong><br />
ASMKO Mouse Brain<br />
James C. Dodge, Jennifer Clarke, Anthony Song, Jie Bu, Qi<br />
Zhao, Tatyana V. Taksir, Denise Griffiths, Lamya S.<br />
Shihabuddin, Catherine R. O’Riordan, Marco A. Passini Ed H.<br />
Schuchman, Gregory R. Stewart<br />
111<br />
AAV7, 8, and 9 Are More Efficient and Less Immunogenic<br />
Vectors for Muscle-Directed <strong>Gene</strong> <strong>Therapy</strong> for<br />
Hemophilia B<br />
Lili Wang, Jean-Pierre Louboutin, Yan Li, James M. Wilson<br />
112<br />
Strong Transgene-Specific Immune Responses Can Be<br />
Elicited with Novel Adeno-Associated Virus Vectors in<br />
Mice<br />
Yan Zhi, Julie Johnston, Di Wu, Joanita Figueredo, Martin<br />
Lock, Peter Bell, Guangping Gao, James M. Wilson<br />
113<br />
Manipulating AAV2 Tropism for Enhanced Delivery <strong>of</strong><br />
AAV Vectors to the Spinal Cord<br />
Adam Davis, Matthew Stachler, Wenfang Shi, James Liu,<br />
Nicholas Boulis, Jeffrey Bartlett<br />
114<br />
Efficient Arterial Formation at the Sites <strong>of</strong> Adult Neo-<br />
Angiogenesis Requires the Recruitment <strong>of</strong> Bone Marrow<br />
<strong>Cell</strong>s through the Neuropilin-1 (NP-1) Receptor<br />
Serena Zacchigna, Nikola Arsic, Lucia Pattarini, Silvia<br />
Moimas, Alessandro Carrer, Lorena Zentilin, Alessandro Salvi,<br />
Gianfranco Sinagra, Mauro Giacca<br />
115<br />
In Vivo Expression <strong>of</strong> Human Cob(I)alamin<br />
Adenosyltransferase Using rAAV Serotypes 1,2 and 8, an<br />
Approach to <strong>Gene</strong> <strong>Therapy</strong> for Methylmalonic Aciduria<br />
(MMA)<br />
Kirsten E. Erger, Thomas J. Conlon, Travis L. Cossette, Thomas<br />
A. Bobik, Terence R. Flotte<br />
116<br />
Functional Comparison <strong>of</strong> Four CFTR rAAV Vectors<br />
with Alternative Transcriptional Elements in <strong>Cell</strong> Lines<br />
and Polarized Human CF Airway Epithelia<br />
Liang Zhang, Benjamin Dutzar, Keith Munson, Barrie Carter,<br />
John F. Engelhardt<br />
117<br />
AAV-Mediated Erythropoietin <strong>Gene</strong> Transfer Protects<br />
from <strong>Gene</strong>tic and Light-Induced Retinal Degeneration<br />
Mariacarmela Allocca, Tonia S. Rex, Luciano Domenici, Jean<br />
Bennet, Victor M. Rivera, Alberto Auricchio<br />
118<br />
Self-Complementary AAV Vectors Pseudotyped with<br />
Either Serotype 5 or 8 Capsids Give Therapeutic Human<br />
Factor IX Levels at Significantly Reduced Doses in Non-<br />
Human Primates<br />
John T. Gray, Andrew M. David<strong>of</strong>f, Amit C. Nathwani<br />
119<br />
Intraperitoneal Transduction <strong>of</strong> Adeno-Associated Virus 2<br />
Expressing Angiostatin and Endostatin Synergistically<br />
Augments Paclitaxel <strong>Therapy</strong> and Tumor-Free Survival in<br />
a Mouse Model <strong>of</strong> Epithelial Ovarian Cancer<br />
Tatyana Isayeva, Changchun Ren, Selvarangan Ponnazhagan<br />
120<br />
Stimulation <strong>of</strong> the Synthesis <strong>of</strong> Type-II Collagen and<br />
Extracellular Matrix in Human Normal and Osteoarthritic<br />
Articular Cartilage Explants In Situ by<br />
Overexpression <strong>of</strong> Human sox9 by rAAV-Mediated <strong>Gene</strong><br />
Transfer<br />
Henning Madry, Tanja Thurn, Dieter Kohn, Ernest F.<br />
Terwilliger, Magali Cucchiarini<br />
121<br />
VEGF-Induced Angiogenesis Does Not Have a Favorable<br />
Effect on Muscle Perfusion: A Relevant Role <strong>of</strong> Long-<br />
Term Angiopoietin-1 Expression in the Formation <strong>of</strong><br />
Functional Blood Vessels<br />
Serena Zacchigna, Ennio Tasciotti, Nikola Arsic, Oreste Sorace,<br />
Marini Cecilia, Silvia Pardini, Debora Petroni, Claudia<br />
Kusmic, Lucia Pattarini, Silvia Moimas GianMario Sambuceti,<br />
Mauro Giacca<br />
122<br />
Enhanced Expression <strong>of</strong> Glutamate Decarboxylase 65<br />
Significantly Improves Rat Parkinsonian Symptom<br />
Boyoung Lee, Young Ran Nam, Sung Jin Kim, Jin Hwan Oh,<br />
Yoon Hee Choi, Jin Woo Chang, Heuiran Lee<br />
123<br />
rAAV-pdx-1 Augmented the Insulin-Producing <strong>Cell</strong>s<br />
Hua Li, Karen S. Lam, Sidney Tam, Weidong Xiao, Paul Tam,<br />
Ruian Xu<br />
124<br />
The “Perivascular Pump” Driven by Arterial Pulsation Is<br />
a Powerful Mechanism for the Distribution <strong>of</strong> Therapeutic<br />
Molecules within the Brain<br />
Piotr Hadaczek, Hanna Mirek, Laszlo Tamas, Martha C.<br />
Bohn, Charles Noble, John W. Park, Kryst<strong>of</strong> Bankiewicz<br />
125<br />
AAV Vector-Mediated Neonatal <strong>Gene</strong> Transfer: Efficient<br />
Transgene Expression in Muscles after Intraperitoneal<br />
Cavity Vector Injection<br />
Tsuyoshi Ogura, Hiroaki Mizukami, Jun Mimuro, Takashi<br />
Okada, Hiromi Hamada, Akihiro Kume, Hiroyuki Yoshikawa,<br />
Yoichi Sakata, Keiya Ozawa
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