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 />
Poster Session III, continued<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
DNA Vectorology & <strong>Gene</strong> Targeting II<br />
573<br />
Eficient Delivery DNA Vaccine for Alzheimer’s Disease<br />
by Triggered Release <strong>of</strong> Polyplexes from Microneedles<br />
Kim Nak Won, Lee Kyuri, Lee Min Sang, Jeong Ji Hoon.<br />
574<br />
Improved Lentiviral Formats for Genome Editing Using<br />
Zinc Finger Nucleases<br />
Monika Gjoka, Kevin Kang, Yuping Huang, Gregory D. Davis.<br />
575<br />
CFTR Reporter Construct for Optimization <strong>of</strong> Airway<br />
Epithelium Differentiation<br />
Myra E. Gordon, Alaleh Esmaeili-Shandiz, Shingo Suzuki, Roy G.<br />
Sargent, Dieter C. Gruenert.<br />
576<br />
Non-Viral <strong>Gene</strong> Delivery to Oral Squamous <strong>Cell</strong><br />
Carcinoma and Cervical Carcinoma <strong>Cell</strong>s and Suicide<br />
<strong>Gene</strong> <strong>Therapy</strong><br />
Nejat Düzgünes, Aruna Singh, Senait Gebremedhin, Krystyna<br />
Konopka.<br />
577<br />
Orphan Product Development for DNA Therapeutics<br />
Gladice Wallraven, Phillip B. Maples, John Nemunaitis.<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Diabetes, Metabolic<br />
and <strong>Gene</strong>tic Diseases II<br />
578<br />
Development <strong>of</strong> Lentiviral <strong>Gene</strong> <strong>Therapy</strong> for Type VII<br />
Collagen Deicient Epidermolysis Bullosa<br />
Christos Georgiadis, Ekaterina Semenova, Andy P. South, Adrian<br />
Thrasher, John A. McGrath, Wei-Li Di, Waseem Qasim.<br />
579<br />
Reprogramming <strong>of</strong> Murine Gallbladder <strong>Cell</strong>s towards<br />
the Pancreatic b-<strong>Cell</strong> Fate In Vitro<br />
Yuhan Wang, Ray D. Hickey, Markus Grompe.<br />
580<br />
Fetal <strong>Gene</strong> <strong>Therapy</strong> at Early and Late Gestation:<br />
Analysis <strong>of</strong> Outcomes and Postnatal Vector Challenge in<br />
a Macaque Model<br />
Citra Mattar, Cecilia Rosales, Rowell Fragata, Nuryanti Johana,<br />
Simon Waddington, Arijit Biswas, Mahesh Choolani, Amit<br />
Nathwani, Jerry Chan.<br />
581<br />
Factors Inluencing Direct Reprogramming <strong>of</strong> Primary<br />
Human Extrahepatic Biliary <strong>Cell</strong>s into Pancreatic beta<br />
<strong>Cell</strong>s<br />
Feorillo Galivo, Eric Benedetti, Craig Dorrell, Olga Smirnova,<br />
Jonathan Schug, Klaus Kaestner, Markus Grompe.<br />
582<br />
Mesenchymal Stem <strong>Cell</strong>s Do Not Prevent Alloantibody<br />
Response Against IDUA Used To Treat MPSI<br />
Priscila K. Matsumoto, Roberta S. Stilhano, Flavia H. da Silva,<br />
Vivian Y. Samoto, Giovani B. Perez, Sang W. Han.<br />
583<br />
Comparison <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> Vectors in a New<br />
Hypomorphic Model <strong>of</strong> Propionic Acidemia<br />
Adam J. Guenzel, Sean E. Hoherr, Matthew Hillestad, Mary Barry,<br />
Eric Weaver, Jan P. Kraus, Dietrich Matern, Michael A. Barry.<br />
584<br />
Transcription Activator-Like Effector Nuclease-Based<br />
Genome Editing for Mucopolysaccharidosis Type I<br />
Mark J. Osborn, Colby G. Starker, Anthony P. DeFeo, Daniel F.<br />
Voytas, Bruce R. Blazar, Jakub Tolar.<br />
585<br />
<strong>Gene</strong> <strong>Therapy</strong> for Mouse Model <strong>of</strong> Krabbe Disease<br />
Hiroshi Kobayashi, Masamichi Ariga, Takahiro Fukuda, Takeo<br />
Iwamoto, Sayoko Izuka, Yota Shimada, Yoshikatsu Eto, Toya<br />
Ohashi.<br />
586<br />
Choroid Plexus-Directed Viral <strong>Gene</strong> <strong>Therapy</strong> as a<br />
Source <strong>of</strong> alpha-N-Acetyl-Glucosaminidase Enzyme<br />
Replacement in Cerebrospinal Fluid for Sanilippo B<br />
Syndrome<br />
Patricia I. Dickson, Shih-hsin Kan, Steven Q. Le, Maria Reina<br />
Haddad, Eun-Young Choi, Anthony Donsante, Stephen G. Kaler.<br />
587<br />
Mitigation <strong>of</strong> DNA Damage Responses and Oxidative<br />
Stress in <strong>Cell</strong>s with Impaired Telomerase Function<br />
Larisa Pereboeva, Erik Westin, Mary Gannon, Lawrence Lamb,<br />
Aloysius Klingelhutz, Frederick Goldman.<br />
588 Abstract Withdrawn<br />
Neurologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II<br />
589<br />
CNS Administration <strong>of</strong> an AAVrh.10 <strong>Gene</strong> Transfer<br />
Vector Expressing the Human ARSA cDNA to Nonhuman<br />
Primates Provides Widespread Activity across the CNS<br />
Jonathan B. Rosenberg, Dolan Sondhi, David G. Rubin, Sébastien<br />
Monette, Alvin Chen, Sara A. Cram, Benjamin Van de Graaf, Bishnu<br />
P. De, Stephen M. Kaminsky, Caroline Sevin, Patrick Aubourg,<br />
Ronald G. Crystal.<br />
Saturday, May 18, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
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