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, Friday, May 17, 2013<br />
Friday, May 17, 2013<br />
Poster Session II, continued<br />
5:30 PM - 7:30 PM<br />
Room: Exhibit Hall C/D<br />
Immunologic & Host Responses in <strong>Gene</strong> & <strong>Cell</strong><br />
<strong>Therapy</strong><br />
441<br />
Liver-Directed <strong>Gene</strong> <strong>Therapy</strong> with Lentiviral Vector<br />
Reverts Pre-Existing Immunity to Coagulation Factor IX<br />
and Rescues Hemophilia B Mice from the Disease<br />
Andrea Annoni, Alessio Cantore, Kevin Goudy, Mahzad Akbarpour,<br />
Fabio Russo, Sara Bartolaccini, Luigi Naldini, Maria Grazia<br />
Roncarolo.<br />
442<br />
Combination Use <strong>of</strong> TLR7 Ligand with GM-CSF <strong>Gene</strong>-<br />
Transduced Tumor Vaccines Provides Substantial<br />
Antitumor Immunity Against Poorly Immunogenic<br />
Mouse Lung Cancer <strong>Cell</strong>s<br />
Megumi Narusawa, Hiroyuki Inoue, Chika Sakamoto, Tomoko<br />
Inoue, Shohei Miyamoto, Makoto Inoue, Koichi Takayama, Mamoru<br />
Hasegawa, Yoichi Nakanishi, Kenzaburo Tani.<br />
443<br />
Elimination <strong>of</strong> Contaminating Capsid-Expressing <strong>Gene</strong>s<br />
in AAV Vectors Reduces Immune Responses to Vector<br />
Administration in a Canine <strong>Gene</strong> <strong>Therapy</strong> Model<br />
Zejing Wang, Christine L. Halbert, Donghoon Lee, Tiffany Butts,<br />
Rainer Storb, Stephen H. Tapscott, Dusty Miller.<br />
444<br />
Immune Activation and Transgene Stability Following<br />
AAV9 <strong>Gene</strong> Transfer to an Acute Myocardial Infarct<br />
Mouse Model<br />
Susan Faust, Ehre Gao, Rebecca Tenney, Jason Ohlstein, Peter Bell,<br />
Walter Koch, James Wilson, Joseph Rabinowitz.<br />
445<br />
Blood Proteins Affecting rAAV Eficiency<br />
Jerome Denard, Hans Jörg Fehling, Philippe Moullier, Tomas Voit,<br />
Fedor Svinartchouk.<br />
446<br />
B & T <strong>Cell</strong> Immunomodulation Attenuates Anaphylaxis<br />
in a Novel Mouse Model <strong>of</strong> Pompe Disease<br />
Phillip A. Doerler, Sushrusha Nayak, Roland W. Herzog, Barry J.<br />
Byrne.<br />
447<br />
Quantiication <strong>of</strong> Alpha-L-Iduronidase Expression<br />
Outcomes in Mice Following Sleeping Beauty<br />
Transposon-Mediated <strong>Gene</strong> <strong>Therapy</strong><br />
Elena L. Aronovich, Bryan C. Hall, Jason B. Bell, R. Scott McIvor,<br />
Perry B. Hackett.<br />
449<br />
Over-Expression <strong>of</strong> Interleukin-6 Alone Induces<br />
Dexamethasone-Relieved Multiple-Organ Lesion in<br />
Mice<br />
Tao Bian, Zhenhua Yuan, Chunxia Zhao, Ting Zhang, Juan Li, Xiao<br />
Xiao.<br />
Oligonucleotide & RNAi Therapeutics<br />
450<br />
AAV6-Mediated Systemic Administration <strong>of</strong> miR30-<br />
Based RNAi Hairpins Impacts Muscle Disease in the<br />
HSALR Myotonic Dystrophy Mouse Model<br />
Darren R. Bisset, Maja Zavaljevski, Carrie Stoltzman, Joel R.<br />
Chamberlain.<br />
451<br />
Peptide-Conjugated Morpholino Rescues Utrophin-<br />
Dystrophin-Deicient Mice by Restoring Dystrophin<br />
Expression and Improvement in Muscle Pathology and<br />
Function<br />
Bo Wu, Caryn Cloer, Stephanie Milazi, Mona Shaban, Sapana N.<br />
Shah, Peijuan Lu, Hong M. Moulton, Qi Long Lu.<br />
452<br />
Co-Delivery <strong>of</strong> VEGF siRNA and IL-4 into Chitosan<br />
Nanoparticles in Breast Tumor Model <strong>of</strong> Rat<br />
Emine Salva, Suna Özbas-Turan, Levent Kabasakal, Fatih Eren,<br />
Saadet Alan, Naziye Özkan, Jülide Akbuga.<br />
453<br />
Systemic Delivery <strong>of</strong> a Peptide-Linked Morpholino<br />
Oligonucleotide Neutralizes Mutant RNA Toxicity in a<br />
Mouse Model <strong>of</strong> Myotonic Dystrophy<br />
Andrew J. Leger, Leocadia M. Mosquea, Nicholas P. Clayton,<br />
I.-Huan Wu, Timothy Weeden, Carol A. Nelson, Lucy Phillips,<br />
Errin Roberts, Peter A. Piepenhagen, Seng H. Cheng, Bruce M.<br />
Wentworth.<br />
454<br />
<strong>Gene</strong> Expression from Pseudouridine and<br />
5-Methylcytidine Modiied Messenger RNAs<br />
Jiehua Zhou, Maggie L. Bobbin, Julie Escamilla-Powers, Eric<br />
Arnold, Anthony Lauer, Laura Juckem, Karl-Dimiter Bissig, Anton<br />
P. McCaffrey, John J. Rossi.<br />
455<br />
Integrin-Targeted Nanoparticle Delivery <strong>of</strong> miRNA for<br />
Anti-Tumor <strong>Therapy</strong><br />
Puthupparampil Scaria, Yijia Liu, Betty Zhou, Martin Woodle.<br />
456<br />
Multi-Subtype Targeting, Escape-Reducing (MuSTER)<br />
Anti-HIV shRNA <strong>Gene</strong> <strong>Therapy</strong><br />
Ulrike Jung, John J. Rossi.<br />
448<br />
Hepatocyte Heparan Sulfate Is Dispensable for<br />
Adenovirus Liver Transduction In Vivo<br />
Anne K. Zaiss, Erin Foley, Ding Xu, Roger Lawrence, Jeffrey D. Esko,<br />
Harvey R. Herschman.<br />
120<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
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