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 101<br />
ABSTRACTS<br />
SATURDAY, JUNE 4<br />
907<br />
<strong>Gene</strong> <strong>Therapy</strong> for <strong>Gene</strong>tic Lipoprotein Lipase (LPL)<br />
Deficiency; an Update<br />
Jaap Rip, Melchior C. Nierman, Jeroen A. Sierts, Colin J. Ross,<br />
Michael R. Hayden, Albertine de Jong, Paul Dijkhuizen Wim<br />
Hermens, Jaap Twisk, John J. Kastelein, Jan Albert<br />
Kuivenhoven, Janneke M. Meulenberg<br />
908<br />
Evaluation <strong>of</strong> the Sleeping Beauty Transposon System in<br />
OTC Deficient Spf-Ash Mice: A Non-Viral Approach to<br />
a <strong>Cell</strong> Autonomous Metabolic Disease<br />
Andy Wilber, Joel Frandsen, Hiroki Morizono, Mendel<br />
Tuchman, R. Scott McIvor<br />
909<br />
Sustained Correction <strong>of</strong> Glycogen Storage Disease Type<br />
II by rAAV1 Vector-Mediated <strong>Gene</strong> <strong>Therapy</strong><br />
Kerry O. Cresawn, Cathryn S. Mah, Thomas J. Fraites, Jr.,<br />
Melissa A. Lewis, Irene Zolotukhin, Barry J. Byrne<br />
910<br />
AAV8-Mediated Expression <strong>of</strong> Glucocerebrosidase<br />
Ameliorates the Storage Pathology in the D409V/null<br />
Gaucher Mouse<br />
Kerry McEachern, John Marshall, Jennifer B. Nietupski, Karen<br />
M. Brennan, Christine M. Barbon, Wei-Lien Chuang, William<br />
Kennedy, Gregory A. Grabowski, Seng H. Cheng<br />
911<br />
Comparison <strong>of</strong> rAAV Serotype, Promoter, and Treatment<br />
Age for the Correction <strong>of</strong> Glycogen Storage Disease Type<br />
II<br />
Kerry O. Cresawn, Cathryn S. Mah, Irene Zolotukhin, Melissa<br />
A. Lewis, Barry J. Byrne<br />
912<br />
Long-Term Efficacy <strong>of</strong> AAV Vector-Mediated <strong>Gene</strong><br />
<strong>Therapy</strong> in the Mouse Model for Glycogen Storage<br />
Disease Type la (GSD-Ia)<br />
Dwight D. Koeberl, Baodong Sun, Tirupapuliyur Damodaran,<br />
Talmage Brown, Andrew Bird, Ayn Schneider, David S.<br />
Millington, Y.-T. Chen<br />
913<br />
Development and Characterization <strong>of</strong> CF Airway<br />
Epithelial <strong>Cell</strong> Lines Complemented with Wild-Type and<br />
∆F508 CFTR cDNA<br />
Rosalie Maurisse, Beate Illek, Judy Cheung, Tusyoshi Shuto,<br />
Hir<strong>of</strong>umi Kai, Horst Fischer, Dieter C. Gruenert<br />
914<br />
A Dichotomous Role for Interleukin-10 in the Pathogenesis<br />
<strong>of</strong> Type 1 Diabetes<br />
Greg G. Simon, Fletcher Schwartz, Clive Wasserfall, Mark A.<br />
Atkinson<br />
915<br />
Adenovirus Transduction May Be Involved in the<br />
Transdifferentiation <strong>of</strong> the Liver to the Pancreas<br />
Alfred Y. Wang, Anja Ehrhardt, Hui Xu, Mark A. Kay<br />
Cardiovascular: Myocardial <strong>Gene</strong> <strong>Therapy</strong><br />
916<br />
Sustained Whole-Body Functional Rescue by Systemic<br />
Delivery <strong>of</strong> AAV8 Vectors in Heart Failure and Muscular<br />
Dystrophy Hamsters<br />
Tong Zhu, Liqiao Zhou, Satsuki Mori, Zhong Wang, Charles<br />
Mctiernan, Chunping Qiao, Chunlian Chen, Daowen Wang,<br />
Juan Li, Xiao Xiao<br />
917<br />
Engineered <strong>Cell</strong> <strong>Therapy</strong> for Sustained Local Myocardial<br />
Delivery <strong>of</strong> Non-Secreted Proteins<br />
Jing Bian, Niladri Mal, Matt Kiedrowski, Farhad Forudi, Marc<br />
S. Penn<br />
918<br />
Nanoparticle Mediated <strong>Gene</strong> Delivery to Magnetized<br />
Implants<br />
Benjamin B. Yellen, Michael Chorny, Ilia Fishbein, Darryl N.<br />
Williams, Candice M. Klingerman, Ivan S. Alferiev, Origene<br />
Nyanguile, Gary Friedman, Robert J. Levy<br />
919<br />
Magnetic Nanoparticle Mediated <strong>Gene</strong> and <strong>Cell</strong> Delivery<br />
Darryl N. Williams, Michael Chorny, Benjamin B. Yellen, Ilia<br />
Fishbein, Stanley J. Stachelek, Origene Nyanguile, Gary<br />
Friedman<br />
920<br />
Muscle Stem <strong>Cell</strong>s Provide Superior Infarct Repair When<br />
Compared with Myoblasts<br />
Hideki Oshima, Thomas R. Payne, Kenneth L. Urish, Tetsuro<br />
Sakai, Yiqun Ling, Burhan Gharaibeh, Kimimasa Tobita,<br />
Bradley B. Keller, James H. Cummins, Johnny Huard<br />
(Note: Abstract 921 has been moved from Poster<br />
Session III to Poster Session II and will follow Abstract<br />
639.)<br />
921<br />
Steps toward Cardiomyopathy <strong>Gene</strong> <strong>Therapy</strong>: In Vivo<br />
Expression <strong>of</strong> Human VLCAD<br />
J. Lawrence Merritt II, Jerry Vockley, Dietrich Matern, Miao He,<br />
David B. Schowalter<br />
922<br />
Paracrine Alterations <strong>of</strong> Endothelial <strong>Cell</strong>s Adhesion<br />
Complexes; Implications for Stem <strong>Cell</strong> Homing<br />
Ion S. Jovin, Jinette D. Abbott, Thomas Manes, Zhenrong Hao,<br />
Lei Li, Reed P. Hickey, Yan Huang, Dingang Liu, Martin S.<br />
Kluger, Frank J. Giordano
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