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 93<br />
ABSTRACTS<br />
SATURDAY, JUNE 4<br />
Oral Abstract Session 413<br />
Molecular Conjugates: Therapeutic Applications<br />
Room: 120/124<br />
779 10:15 <strong>AM</strong><br />
Oral Delivery <strong>of</strong> Non-Viral DNA Nanoparticles for<br />
Hemophilia A<br />
Katherine Bowman, Rita Sarkar, Sanj Raut, Neelima Shah,<br />
Xuli Wang, Hai-Quan Mao, Kam W. Leong<br />
780 10:30 <strong>AM</strong><br />
A Novel Multi-Component Molecular Conjugate Vector<br />
for Highly Efficient p53-Mediated Tumor-Specific <strong>Gene</strong><br />
<strong>Therapy</strong><br />
Stanley Saamoah-M<strong>of</strong>fatt, Sandra Wiehle, Richard J. Cristiano<br />
781 10:45 <strong>AM</strong><br />
The Effect <strong>of</strong> Overexpression <strong>of</strong> Lysyl Oxidase on<br />
Dermal Wound Healing<br />
Ying-Ka I. Lau, Jennifer L. West<br />
782 11:00 <strong>AM</strong><br />
Poly(glycoamidoamine)s Deliver DNA with High<br />
Efficacy to Cardiomyoblast <strong>Cell</strong>s<br />
Yemin Liu, Suiwen He, W. Keith Jones, Theresa M. Reineke<br />
783 11:15 <strong>AM</strong><br />
Transient Transduction <strong>of</strong> Repopulating Human CD34+<br />
<strong>Cell</strong>s Using Nanoparticles in a Clinically Applicable Ex<br />
Vivo Protocol<br />
Jesper Bonde, Dustin J. Maxwell, David A. Hess, Ryan Lahey,<br />
David Piwnica-Worms, Jan A. Nolta<br />
784 11:30 <strong>AM</strong><br />
Promoter-Dependence <strong>of</strong> Plasmid-Pluronics Targeted<br />
alpha-Galactosidase A Expression in Skeletal Muscle <strong>of</strong><br />
Fabry Mice<br />
Matthieu D. Lavigne, Marita Pohlschmidt, Javier F. Novo,<br />
Valery Alakhov, Dariusz C. Gorecki<br />
785 11:45 <strong>AM</strong><br />
<strong>Cell</strong>-Permeable Peptides Mediated <strong>Gene</strong> Delivery in<br />
Hepatocytes In Vitro and In Vivo<br />
Zhenping Shen, Carol H. Miao<br />
786 12:00 PM<br />
Synthesis <strong>of</strong> Cationic Biodegradable Polymers for<br />
Targeted <strong>Gene</strong> Delivery to Hepatocytes<br />
Gregory T. Zugates, Daniel Anderson, Ingrid Lawhorn, Robert<br />
Langer<br />
Oral Abstract Session 414<br />
Muscle and Connective Tissue: <strong>Gene</strong> <strong>Therapy</strong> for<br />
Muscular Dystrophy<br />
Room: 230/231<br />
787 10:15 <strong>AM</strong><br />
Lentivirus Mediated Dystrophin Expression in mdx<br />
Muscles<br />
En Kimura, Sheng Li, Brent Fall, Miki Haraguchi, Leonard<br />
Meuse, Jeffery S. Chamberlain<br />
788 10:30 <strong>AM</strong><br />
Antisense <strong>Therapy</strong> for Duchenne Muscular Dystrophy: A<br />
Realistic Possibility<br />
Qi L. Lu, Adam Rabinowitz, HaiFang Yin, Julia Alter, George<br />
Bou-Gharios, Terrence Partridge<br />
789 10:45 <strong>AM</strong><br />
Stable Genome Alteration <strong>of</strong> the Dystrophin <strong>Gene</strong> for<br />
the Treatment <strong>of</strong> Duchenne Muscular Dystrophy<br />
(DMD) Due to Frame-Shift Mutations Using Oligonucleotide-Mediated<br />
<strong>Gene</strong> Editing<br />
Carmen Bertoni, Thomas A. Rando<br />
790 11:00 <strong>AM</strong><br />
Evaluation <strong>of</strong> Immune Responses to Dystrophin in<br />
Humanized mdx HLA-A*0201 Dystrophic Mice:<br />
Application in Duchenne Muscular Dystrophy <strong>Gene</strong><br />
<strong>Therapy</strong> after U7 snRNA-Mediated Exon Skipping<br />
Florent Ginhoux, Sabrina Turbant, Marylène Leboeuf, David<br />
A. Gross, François Lemonnier, Luis Garcia, Olivier Danos, Jean<br />
Davoust<br />
791 11:15 <strong>AM</strong><br />
Immunity to AAV-Mediated <strong>Gene</strong> <strong>Therapy</strong> in a Random-<br />
Bred Canine Model <strong>of</strong> Duchenne Muscular Dystrophy<br />
Zejing Wang, Michael J. Blankinship, Paul Gregorevic, Marie-<br />
Terese Little, Rainer J. Storb, James M. Allen, Stephen J.<br />
Tapscott, Jeffrey S. Chamberlain, Christian S. Kuhr<br />
792 11:30 <strong>AM</strong><br />
Canine Mini-Dystrophin <strong>Gene</strong> Transfer by AAV1 in mdx<br />
Mice Ameliorates Dystrophic Pathology and Protects<br />
Membrane Integrity<br />
Bing Wang, Juan Li, Liqiao Zhou, Chunping Qiao, Mengnan<br />
Tian, Tong Zhu, Janet Bogan, Joseph Kornegay, Xiao Xiao<br />
793 11:45 <strong>AM</strong><br />
Efficient Expression <strong>of</strong> the 6 kb Mini-Dystrophin <strong>Gene</strong><br />
by Trans-Splicing Adeno-Associated Viral (AAV) Vector<br />
Restores the Entire Dystrophin-Associated Glycoprotein<br />
Complex and Reduces Contraction-Induced Damage in<br />
the Mdx Mouse Model <strong>of</strong> Duchenne Muscular Dystrophy<br />
Yi Lai, Yongping Yue, Mingju Liu, Arka Ghosh, John F.<br />
Engelhardt, Jeffrey S. Chamberlain, Dongsheng Duan
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