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, Thursday, May 16, 2013<br />
Oral Abstract Session 233, continued<br />
Stem <strong>Cell</strong> Engineering and <strong>Therapy</strong><br />
2:00 PM - 4:00 PM<br />
Room: Ballroom D<br />
Oral Abstract Session 234<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I<br />
2:00 PM - 4:00 PM<br />
Room: 150 DEFG<br />
Thursday, May 16, 2013<br />
81 2:30 PM<br />
“Semi-Universal Donor Stem <strong>Cell</strong>s” That Express a<br />
Single Class I Human Leukocyte Antigen (HLA) A*0201<br />
Can Present Peptides to Allorestricted CD8+ T-<strong>Cell</strong>s<br />
German Gornalusse, Marvin Festag, Roli Hirata, Laura Riolobos,<br />
Cameron Turtle, Stan Riddell, David W. Russell.<br />
82 2:45 PM<br />
Targeted Correction <strong>of</strong> X-CGD iPSCs by TALEN-Mediated<br />
<strong>Gene</strong> Transfer to the CYBB Locus<br />
Colin L. Sweeney, Uimook Choi, Jizhong Zou, Suk See DeRavin,<br />
Harry L. Malech.<br />
83 3:00 PM<br />
<strong>Gene</strong>tically Corrected Myoblasts Derived from<br />
Dystrophic hIPSCs Fused with Existing Muscle Fibers<br />
in Immunodeicient mdx Mice and Led to Expression <strong>of</strong><br />
Micro-Dystrophin and a-Sarcoglycan<br />
Chantale Maltais, Carl Lebel, Joël Rousseau, Jacques P. Tremblay.<br />
84 3:15 PM<br />
<strong>Gene</strong>ration <strong>of</strong> Functional Monocyte/Macrophages<br />
by <strong>Gene</strong>tic Correction <strong>of</strong> Patient-Speciic iPSC in<br />
Congenital Pulmonary Alveolar Proteinosis<br />
Nico Lachmann, Christine Happle, Doreen Lüttge, Mania<br />
Ackermann, Adele Mucci, Nicolaus Schwerk, Martin Wetzke, Sylvia<br />
Merkert, Axel Schambach, Gesine Hansen, Thomas Moritz.<br />
85 3:30 PM<br />
Clonogenic Hepatocyte Progenitors from the Liver,<br />
Pancreas and Gall Bladder<br />
Craig Dorrell, Meritxell Huch, Yuhan Wang, Hans Clevers, Markus<br />
Grompe.<br />
86 3:45 PM<br />
The Activation <strong>of</strong> the Wnt3a/b-Catenin Pathway<br />
Induced Maturation <strong>of</strong> Impaired Neutrophils as Well<br />
as the Endoplasmic Reticulum Stress in Induced<br />
Pluripotent Stem <strong>Cell</strong> Derived from a Severe Congenital<br />
Neutropenia Patient with ELANE Mutation<br />
Takafumi Hiramoto, Yasuhiro Ebihara, Yoko Mizoguchi, Kazuhiro<br />
Nakamura, Kiyoshi Yamaguchi, Kazuko Ueno, Naoki Nariai, Shinji<br />
Mochizuki, Shohei Yamamoto, Masao Nagasaki, Yoichi Furukawa,<br />
Kenzaburo Tani, Hiromitsu Nakauchi, Masao Kobayashi, Kohichiro<br />
Tsuji.<br />
87 2:00 PM<br />
The Effect <strong>of</strong> Neonatal <strong>Gene</strong> <strong>Therapy</strong> on Dysostosis<br />
Multiplex in Mucopolysaccaridosis VII Dogs after a<br />
Decade<br />
Katherine P. Ponder, Elizabeth M. Xing, Van W. Knox, Patricia A.<br />
O’Donnell, Yuli Liu, Susan Wu, Mark E. Haskins.<br />
88 2:15 PM<br />
scAAV Provides Sustained Expression <strong>of</strong> a Homologous<br />
Therapeutic Transgene in Large Mammalian Joints,<br />
with Enhanced Expression in Osteoarthritis<br />
Rachael S. Watson, Ted Broome, Andrew Smith, Patrick Colahan,<br />
Steven Ghivizzani.<br />
89 2:30 PM<br />
Dual Axis <strong>Gene</strong>-<strong>Therapy</strong> Using Stem <strong>Cell</strong>s<br />
Overexpressing TGF-b3 in Combination with IL-1b and<br />
TNF-a RNA Silencing for Osteoarthritis Control in a<br />
Large Animal Osteochondral Chip Fracture Model<br />
Alan J. Nixon, Ashlee E. Watts, Laila Begum.<br />
90 2:45 PM<br />
Respiratory Function in a Canine Model <strong>of</strong> X-Linked<br />
Myotubular Myopathy Following Regional Limb<br />
Infusion with Recombinant AAV8-MTM1<br />
Melissa Goddard, Romain Joubert, Karine Poulard, Robert Grange,<br />
Michael Lawlor, Christelle Moal, Thibaud Jamet, Nathalie Danièle,<br />
Samia Martin, Christel Rivière, Caroline Hammer, Laetitia Van<br />
Wittenberghe, Erin Mitchell, Alban Vignaud, Carole Masurier,<br />
Philippe Moullier, Alan Beggs, Anna Buj-Bello, Barbara Smith,<br />
Barry Byrne, Martin Childers.<br />
91 3:00 PM<br />
Vascular Delivery <strong>of</strong> AAV.Micro-Dystrophin Using<br />
Whole Limb Re-Circulation in the Non-Human Primate<br />
as a <strong>Therapy</strong> for DMD<br />
Louis G. Chicoine, Chrystal L. Montgomery, Danielle A. Grifin,<br />
Kristin N. Heller, Kim Shontz, Thomas J. Preston, William G.<br />
Bremer, Christopher M. Walker, K. R. Clark, Jerry R. Mendell,<br />
Louise R. Rodino-Klapac.<br />
92 3:15 PM<br />
Safety and Histological Signs <strong>of</strong> Eficacy Following<br />
Systemic AAV9-CMV-Microutrophin <strong>Gene</strong> <strong>Therapy</strong> in a<br />
Canine Model for Duchenne Muscular Dystrophy<br />
Alock Malik, Andrew Mead, Mike Petrov, Marilyn Mitchell, Leonard<br />
Su, Frederick Balzer, Jacqueline Farag, Janet Bogan, Dan Bogan, Joe<br />
Kornegay, Hansell Stedman.<br />
93 3:30 PM<br />
A Xenograft Model <strong>of</strong> Skeletal Muscle for Preclinical<br />
<strong>Gene</strong> <strong>Therapy</strong> Studies<br />
Kathryn R. Wagner, Tracy Y. Zhang, Terence A. Partridge, Takako<br />
Jone, Oliver King, Fedik Rahimov, Qin Su, Guangpin Gao, Louis M.<br />
Kunkel, Robert H. Brown, Jr, Miguel Sena-Esteves.<br />
94<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
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