Final Program - American Society of Gene & Cell Therapy
Final Program - American Society of Gene & Cell Therapy
Final Program - American Society of Gene & Cell Therapy
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Abstract Directory, Saturday, May 18, 2013<br />
Saturday, May 18, 2013<br />
Poster Session III, continued<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
Neurologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II<br />
590 (Moved to Poster Session II after Abstract 391)<br />
Intracranial AAV <strong>Gene</strong> <strong>Therapy</strong> Extends the Life Span<br />
<strong>of</strong> GM2 Gangliosidosis Cats >Four-Fold with No Clinical<br />
Evidence <strong>of</strong> Vector Toxicity<br />
Allison M. Bradbury, Victoria J. McCurdy, Aime K. Johnson, Heather<br />
Gray-Edwards, Brandon L. Brunson, Ashley N. Randle, Nancy R.<br />
Cox, Miguel Sena-Esteves, Douglas R. Martin.<br />
591<br />
Prophylactic and Therapeutic Treatment <strong>of</strong> Pain<br />
in a Varicella- Zoster Virus (VZV) Rodent Model <strong>of</strong><br />
Post-Herpetic Neuralgia (PHN) Using an HSV Vector<br />
Expressing Enkephalin<br />
William F. Goins, Jean-Marc Guedon, Mingdi Zhang, Joseph C.<br />
Glorioso, Paul R. Kinchington.<br />
592<br />
AAV <strong>Gene</strong> <strong>Therapy</strong> Normalizes Disease Phenotype<br />
and Extends Life Span >Four-Fold in Feline GM1<br />
Gangliosidosis<br />
Aime K. Johnson, Victoria J. McCurdy, Allison M. Bradbury, Heather<br />
Gray-Edwards, Ashley N. Randle, Misako Hwang, Nancy R. Cox,<br />
Stanley G. Leroy, Miguel Sena-Esteves, Douglas R. Martin.<br />
593<br />
MECP2e1 Is<strong>of</strong>orm-Speciic Lentivirus Rescues Rett<br />
Syndrome Neuronal Soma Size Defects in a <strong>Cell</strong><br />
Autonomous Manner<br />
Ugljesa Djuric, Aaron Cheung, Alina Piekna, Jason Hendry, Joel<br />
Ross, Peter Pasceri, Dae-Sung Kim, James Ellis.<br />
594<br />
Immune Response Activation and Neurotoxicity after<br />
Intrathecal Infusion <strong>of</strong> AAV9 Vector Encoding a Non-Self<br />
Reporter Protein in the Non-Human Primate<br />
Lluis Samaranch, Waldy San Sebastian, Adrian P. Kells, John<br />
Bringas, Phillip Pivirotto, John Forsayeth, Kryst<strong>of</strong> S. Bankiewicz.<br />
595<br />
Intracranial Injection <strong>of</strong> AAVrh8-mbgal in a GM1<br />
Gangliosidosis Mouse Model Delays Disease Onset and<br />
Signiicantly Extends Life Span<br />
Cara M. Weismann, Jennifer Ferreira, Stacy Maitland, Miguel<br />
Sena-Esteves.<br />
596<br />
Enhanced CNS Transduction and Transgene Expression<br />
by rAAV Capsid Tyrosine Mutants<br />
Nicholas M. Kanaan, Caryl E. Sortwell, Sara E. Gombash, Nathan<br />
M. Kuhn, William W. Hauswirth, Sanford E. Boye, Vince A. Chiodo,<br />
Tessa Grabinski, Fredric P. Manfredsson.<br />
597<br />
Peripheral Blood White <strong>Cell</strong>s Present Molecular<br />
Signatures <strong>of</strong> Neurodegeneration and Respond to a<br />
Single Intravenous rAAV9-hNAGLU <strong>Gene</strong> Delivery in<br />
MPS IIIB Mice<br />
Bartholomew J. Naughton, F. Jason Duncan, Tierra Ware, Darren<br />
Murrey, Aaron Meadows, Douglas M. McCarty, Haiyan Fu.<br />
598<br />
The Therapeutic Potential <strong>of</strong> MicroRNA Regulation in a<br />
Rat Intracerebral Hemorrhage Model<br />
Jangsup Moon, Jeong-Min Kim, Jung Ick Byun Byun, Kon Chu,<br />
Keun-Hwa Jung, Soon-Tae Lee, Jin-Hee Kim, Dong-Kyu Park, So-<br />
Yoon Kim, Jae-Kyu Roh.<br />
599<br />
Adeno-Associated Virus Type 6 Is Retrogradely<br />
Transported in the Non-Human Primate Brain,<br />
Implications for <strong>Gene</strong> Delivery in Huntington’s Disease<br />
Waldy San Sebastian, Lluis Samaranch, Adrian P. Kells, John<br />
Bringas, Phillip Pivirotto, John Forsayeth, Kryst<strong>of</strong> S. Bankiewicz.<br />
600<br />
Herpes Simplex Virus 2 <strong>Gene</strong>, ICP10PK, Protects<br />
Against Neuronal <strong>Cell</strong> Death Associated with Valproic<br />
Acid-Induced Autism Spectrum Disorder<br />
Dominique Bollino, Laure Aurelian.<br />
601<br />
Targeted <strong>Gene</strong> Transfer into the Ependymal <strong>Cell</strong>s<br />
and Long-Term Enzyme Replacement in the CSF by<br />
Intraventricular Injection <strong>of</strong> AAV Vector Serotype 1<br />
Yoshiyuki Yamazaki, Kohei Hironaka, Yukihiko Hirai, Koichi<br />
Miyake, Takashi Shimada.<br />
602<br />
Neuroprotective Effect <strong>of</strong> a <strong>Cell</strong>-Free Extract Derived<br />
from Human Adipose Stem <strong>Cell</strong>s in Experimental Stroke<br />
Models<br />
Jung Ick Byun, Daejong Jeon, Kon Chu, Soon-Tae Lee, Keun-Hwa<br />
Jung, Jae-Jun Ban, Hye-Jin Yoon, Seungmoon Jung, Hyunwoo Yang,<br />
Byung Sun Kim, Ji Ye Choi, Jeong-Min Kim, Chong-Hyun Won,<br />
Manho Kim, Sang Kun Lee, Jae-Kyu Roh.<br />
603<br />
Artiicial MicroRNAs Against Spliced Variants <strong>of</strong><br />
the <strong>Gene</strong> C9ORF72, the Major Cause for Familial<br />
Amyotrophic Lateral Sclerosis<br />
Gabriela Toro, Florie Borel, Peter Sapp, Lina Song, Robert Brown,<br />
Christian Mueller.<br />
604<br />
Reduction <strong>of</strong> Hyperammonemia In Vitro and In Vivo by<br />
a Baculovirus-Delivered Glutamine Synthetase as a New<br />
Approach for the Treatment <strong>of</strong> Hepatic Encephalopathy<br />
Miguel A. Torres-Vega, Lizbeth M. Vázquez-Carcaño, Roxana<br />
Y. Vargas-Jerónimo, Ana G. Montiel-Martínez, Rosa M. Muñoz-<br />
Fuentes, Misael Uribe, Absalom Zamorano-Carrillo, Ana R. Pastor-<br />
Flores, Laura A. Palomares.<br />
132<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
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