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 71<br />
ABSTRACTS<br />
FRIDAY, JUNE 3<br />
418 2:30 PM<br />
rAAV-Mediated Expression <strong>of</strong> Adiponectin Receptor<br />
(AdipoR2) cDNA in Muscle Prevents the Development<br />
<strong>of</strong> Obesity in DIO Rats<br />
George Aslanidi, Stanislav Shklayev, Erik Kohlbrenner, Vadim<br />
Kroutov, Glenn Walter, Martha Campbell-Thompson, Sergei<br />
Zolotukhin<br />
419 2:45 PM<br />
<strong>Gene</strong>ration <strong>of</strong> Transgene Product-Specific Regulatory<br />
CD4+CD25+ T <strong>Cell</strong>s by Hepatic AAV <strong>Gene</strong> Transfer<br />
O. Cao, E. Dobrzynski, B. L. Mingle, L. Wang, R. W. Herzog<br />
420 3:00 PM<br />
Tropism-Modified Adeno-Associated Virus Vector<br />
Mediates Targeting <strong>of</strong> Brain Vascular Endothelium In Vivo<br />
Yong Hong Chen, Beverly L. Davidson<br />
421 3:15 PM<br />
Liver Contains Sufficient Sulfatase Modifying Factor for<br />
4-Sulfatase and Correction <strong>of</strong> MPS VI with Neonatal<br />
Retroviral <strong>Gene</strong> <strong>Therapy</strong> in Cats<br />
Mark Haskins, Thomas O’Malley, Bin Wang, Jason Metgcalf,<br />
Ping Wang, John Hopwood, Katherine P. Ponder<br />
422 3:30 PM<br />
Dominant-Negative Interference in the Pah enu2 Mouse<br />
Model <strong>of</strong> PKU: Effectiveness <strong>of</strong> Vectors Expressing<br />
Either Modified Forms <strong>of</strong> Phenylalanine Hydroxylase<br />
(PAH) or Ribozymes Plus a Hardened PAH mRNA<br />
Catherine E. Charron, O. P. Perera, Stacy L. Porvasnik, Mandy<br />
Blackburn, Alfred S. Lewin, Philip J. Laipis<br />
423 3:45 PM<br />
Increased Incidence <strong>of</strong> Hepatocellular Change and<br />
Neoplastic Disease in AAV-WPRE Treated Pah enu2 Mice<br />
Jennifer E. Embury, Catherine E. Charron, Philip J. Laipis<br />
424 4:00 PM<br />
Intracranial AAV5 Synergizes with Non-Myeloablative<br />
Bone Marrow Transplantation in the Murine Model <strong>of</strong><br />
Globoid-<strong>Cell</strong> Leukodystrophy<br />
Darshong Lin, Anthony Donsante, Shannon Macauley, Beth<br />
Levy, Carole Vogler, Mark Sands<br />
425 4:15 PM<br />
AAV2-Mediated Expression <strong>of</strong> Iduronate Sulfatase in<br />
Peripheral Tissues and the CNS in<br />
Mucopolysaccharidosis II Mouse Model by Combined<br />
Intravenous and Intracisternal Vector Delivery<br />
Lu Kang, Haiyan Fu, Jerillyn S. Jennings, Joseph Muenzer<br />
Oral Abstract Session 334<br />
Neurologic Disorders<br />
Room: 265/266<br />
426 2:00 PM<br />
Cure <strong>of</strong> Established Disease and Direct Evidence <strong>of</strong><br />
<strong>Cell</strong>ular Cross-Correction in the Nervous System <strong>of</strong><br />
Metachromatic Leukodystrophy Mice after HSC-Based<br />
<strong>Gene</strong> <strong>Therapy</strong><br />
Alessandra Biffi, Alessia Capotondo, Angelo Quattrini, Ubaldo<br />
Del Carro, Riccardo Brambilla, Stefania Fasano, Sergio<br />
Marchesini, Claudio Bordignon, Luigi Naldini<br />
427 2:15 PM<br />
AAV2- and AAV5-Mediated CNS Delivery <strong>of</strong> Human<br />
CLN2 Reduces Lysosomal Storage in a Mouse Model <strong>of</strong><br />
Late Infantile Neuronal Ceroid Lip<strong>of</strong>uscinosis<br />
Marco A. Passini, Dolan Sondhi, James C. Dodge, Jie Bu,<br />
Wendy Yang, Neil R. Hackett, Stephen M. Kaminsky,<br />
Mukarram El-Banna, Qinwen Mao, Beverly L. Davidson<br />
David E. Sleat, Peter Lobel, Ronald G. Crystal, Gregory R.<br />
Stewart<br />
428 2:30 PM<br />
A Novel Approach for the Treatment <strong>of</strong> the Neurological<br />
Symptoms <strong>of</strong> MPS VII (Sly Disease)<br />
Brian J. Spencer, Inder M. Verma<br />
429 2:45 PM<br />
AAV5-Mediated Delivery <strong>of</strong> Human Aryl Sulfatase A<br />
(hARSA) Prevents Sufatide Storage and Neuropathological<br />
Phenotype in Metachromatic Leukodystrophy (MLD)<br />
Mice<br />
Nathalie Cartier, Caroline Sevin, Abdellatif Benraiss, Peter<br />
DeDeyn, Delphine Bonnin, Marie-T Vanier, Moullier Philippe,<br />
Volkmar Gieselmann, Patrick Aubourg<br />
430 3:00 PM<br />
Immune System Regulation <strong>of</strong> Transgene Expression<br />
from High-Capacity Gutless Adenoviral Vectors in the<br />
Mouse Brain: Very Long Term Expression Following Pre-<br />
Immunization<br />
Carlos Barcia, Jeffrey M. Zirger, Weidong Xiong, Philip Ng,<br />
Donna Palmer, Maria G. Castro, Pedro R. Lowenstein<br />
431 3:15 PM<br />
In Utero CNS Delivery <strong>of</strong> an Integrating HSV-1<br />
Amplicon Vector Leads to Prolonged, Neuron-Specific<br />
<strong>Gene</strong> Expression<br />
William J. Bowers, Michael A. Mastrangelo, Ann E. Casey,<br />
Hilary A. Southerland, Kathleen A. Maguire-Zeiss, Howard J.<br />
Feder<strong>of</strong>f<br />
432 3:30 PM<br />
Molecular Chimerism Prevents Experimental Autoimmune<br />
Encephalomyelitis (EAE)<br />
Herena Eixarch, Carmen Espejo, Francisco Vidal, Silvia Garces,<br />
Mireia Castillo, Alex Bote, Elisabeth Kadar, Marta Rosal,<br />
Xavier Montalban, Jordi Barquinero
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