13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010133Abstract DirectorySaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CChemical and Molecular Conjugates III754Optimization <strong>of</strong> Sendai F/HN Viroplexes for Efficient Transfection <strong>of</strong>Plasmid DNA and siRNA to Suspension <strong>Cell</strong>sJung Seok Kim, Yeon Kyung Lee, Jin Ee Baek, Sang Il Park, Hwa Yeon Jeong, Hong SungKim, Keun Sik Kim, Yong Serk Park.755Comparative Evaluation <strong>of</strong> Disulfide-Linked and Hydrophobically-Modified PEI for Plasmid Delivery In VitroK. C. B. Remant, Breanne Landry, Hasan Uludag.756Linear Polyethylenimine Prepared by Partial Acid Hydrolysis <strong>of</strong> Poly(2-ethyl-2-oxazoline) for DNA and siRNA Delivery In VitroJulio C. Fernandes, Xingping Qiu, Francoise M. Winnik, Mohamed Benderdour, XiaolingZhang, Kerong Dai, Qin Shi.757Impact <strong>of</strong> Counterion and Hydrophobic Anchor on PhysicochemicalCharacteristics and Transfection Efficiency for a Series <strong>of</strong> PyridiniumCationic Lipid-Based LipoplexesSushma Savarala, Stephanie L. Wunder, Marc A. Ilies.<strong>Gene</strong>tic and Metabolic Diseases <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> III758AAV-Mediated Liver <strong>Gene</strong> Transfer Results in Long-Term Expression<strong>of</strong> Therapeutic Levels <strong>of</strong> ARSB in MPS VI CatsGabriella Cotugno, Patrizia Annunziata, Anita Capalbo, Thomas O’Malley, PatriciaO’Donnell, Fabio Russo, Elvira De Leonibus, Mark Haskins, Alberto Auricchio.759Beta-Glucuronidase Expressed from AAV Is Poorly Modified withMannose-6-Phosphate Residues and Fails to Completely CorrectMucopolysaccharidosis VII in DogsGuangping Gao, Peter L. Bell, Mark E. Haskins, Lisa A. Kisling, Richard Steet, Luk H.Vandenberghe, Hongwei Yu, Julio C. Sanmiguel, James M. Wilson.760<strong>Gene</strong> Transfer To Induce <strong>Cell</strong>ular Reprogramming for the Treatment <strong>of</strong>DiabetesNeil Phillips, Markus Grompe, Mark A. Kay.761Salivary Gland hPTH <strong>Gene</strong> Transfer: Interesting PossibilitiesJanik Adriaansen, Paola Perez, Changyu Zheng, Michael T. Collins, Bruce J. Baum.762Efficient <strong>Gene</strong> Transfer to the Pancreas Using ssAAV for Prevention<strong>of</strong> Type 1 DiabetesEduard Ayuso, Veronica Jimenez, Judith Agudo, Ariana Salavert, Sergio Muñoz, AlbaCasellas, Merce Obach, Maria Molas, Marta Moya, Xavier Leon, Jesus Ruberte, FatimaBosch.763Tolerance Induction to Coagulation Factor IX Using <strong>Gene</strong> ModifiedPrimary B <strong>Cell</strong>sBabak Moghimi, Irene Zolotukhin, Ou Cao.764 (Moved to Poster Session II after Abstract 422)Microporous Polymer Scaffolds for Islet TransplantationRomie F. Gibly, Lonnie D. Shea.765AAV2- and AAV9 -Mediated <strong>Gene</strong> Transfer <strong>of</strong> LDL Receptor toHypercholesterolemic WHHL RabbitsElisa Vähäkangas, Anniina Laurema, Atsushi Miyanohara, Seppo Ylä-Herttuala.766Hairpin Ribozymes Encoded in Adenoviral Vectors Reduce Drinking inAlcohol Dependent RatsAmalia Sapag, Thergiory Irrazábal, María Elena Quintanilla, Lutske Tampier.767Sustained Therapeutic Levels <strong>of</strong> FVIII Activity from Lentivirally-Engineered Blood Outgrowth Endothelial <strong>Cell</strong>s Requires <strong>Cell</strong> Stabilityand Augmented AngiogenesisHideto Matsui, Margareth Ozelo, Andrea Labelle, Carol Hegadorn, Patrick Thompson,Erin Burnnet, Christine Hough, David Lillicrap.768Effects <strong>of</strong> Neonatal Intracranial AAV and Systemic Lentiviral <strong>Gene</strong><strong>Therapy</strong> in Sanfilippo Syndrome Type B MiceCoy D. Heldermon, Elizabeth Qin, Kevin K. Ohlemiller, Erik Herzog, Carole Vogler, DavidF. Wozniak, Mark S. Sands.769Laminin-111: A Highly Potent Therapeutic Agent To Treat DuchenneMuscular DystrophySebastien Goudenege, Yann Lamarre, Nicolas Dumont, Joel Rousseau, Skuk Daniel,Jacques P. Tremblay.770Layered Scaffolds for Islet Transplantation and <strong>Gene</strong> DeliveryJohn G. Graham, Lonnie D. Shea.771Lentiviral Vector Correction <strong>of</strong> Fabry Disease Encompassing a <strong>Cell</strong>Fate Control Safety ElementMatthew D. Scaife, Sean P. Devine, Anton Neschadim, Natalia A. Pacienza, Jeffrey A.Medin.772Wolman Disease (LIPA p.G87V) Genotype Frequency in Patients <strong>of</strong>Iranian-Jewish DescentYadira Valles-Ayoub, Daniel No, Saghi Esfandiarifard, Zeshan Khokher, Chai Saechao,Marvin Pietruszka, Daniel Darvish.Saturday, May 22 nd
134<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectorySaturday, May 22 ndSaturday, May 22, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CNeurodegenerative Disorders, Spinal Cord and Trauma773Delayed Inhibition <strong>of</strong> Soluble Tumor Necrosis Factor Signaling Blocksthe Progressive Phase <strong>of</strong> Dopaminergic Neuron Death in a Rat Model<strong>of</strong> Parkinson’s DiseaseAshley S. Harms, Kelly M. Ruhn, Steve Varghese, Isaac Trevino, Malu G. Tansey.774Identification and Analysis <strong>of</strong> a Novel g-Secretase Inhibitory <strong>Gene</strong>Hiroki Hayashi, Hironori Nakagami, Nobutaka Koibuchi, Munehisa Shimamura, YasufumiKaneda.775Effects <strong>of</strong> Alpha 7 Nicotinic Receptor <strong>Gene</strong> Delivery on ProcessesUnderlying <strong>Cell</strong> Viability and <strong>Cell</strong> DifferentiationYan Ren, Lin Zhang, Edwin M. Meyer, Jeffrey A. Hughes, Sihong Song.776Delivery <strong>of</strong> hGDNF to Brain Using DNA Nanoparticles:Neuroprotection <strong>of</strong> the Nigrostriatal PathwayDavid M. Yurek, Anita M. Fletcher, Linas Padegimas, Tomasz H. Kowalczyk, Mark J.Cooper.777ProSavin, a <strong>Gene</strong> <strong>Therapy</strong> for Parkinson’s DiseaseBechir Jarraya, Helene Lepetit, Scott Ralph, James Miskin, Jean-Marc Gurruchaga, MariaVinti, Gilles Fenelon, Pierre Brugiere, K. Abay, I. Gabriel, Sabrina Boulet, Caroline Jan,Susan Kingsman, P. Cesaro, Philippe Hantraye, Philippe Remy, Kyriacos Mitrophanous,Stephane Palfi.778Primate Hippocampal <strong>Gene</strong> Delivery Via Frameless StereotaxisCary H. Leung, Michele A. Kliem, Brooke R. Snyder, Thais Federici, Thomas J. McKown,Scott W. J. McPhee, Nicholas M. Boulis.779Delivery Co-Interventions in <strong>Gene</strong> Transfer and <strong>Cell</strong> Transplantation:A Meta-Analysis <strong>of</strong> Surgical Risk for Trials Involving Inoculation toDeep Brain StructuresJonathan Kimmelman, Katherine Duckworth, Tim Ramsay, Marina Emborg, Tiffini Voss.780Modification <strong>of</strong> the Inhibitory Environment <strong>of</strong> Spinal Cord Injury byLentiviral Vector Containing NT-3 and shRNA for NG2Eleanor M. Donnelly, Nicolas N. Madigan, Gemma E. Rooney, Anthony J. Windebank,Timothy O’Brien, Siobhan S. McMahon.781HSV <strong>Gene</strong> Transfer <strong>of</strong> a Poreless TRPV1 Receptor Reduces Pain-Related Behavior in RodentsJames R. Goss, William F. Goins, Rahul Srinivasan, Joseph C. Glorioso.782A Comparison <strong>of</strong> AAV Serotypes and Routes <strong>of</strong> Administration forDelivery to the Spinal CordBrooke R. Snyder, Thais Federici, Eric Quach, Steven Gray, Cary H. Leung, Nicholas M.Boulis.783Engineered Zinc Finger Protein Improves Microvascular Recovery,Neuronal Sparing and Functional Recovery in Modeled TraumaticBrain Injury in RatsEugene Park, Ishita Siddiq, Elaine Liu, S. Kaye Spratt, Richard Surosky, Gary Lee, DaleAndo, Marty Giedlin, Andrew J. Baker.Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> III784Blocking the Myostatin Signal Improves the Success <strong>of</strong> HumanMyoblast Transplantation in Dystrophic MiceRaouia Fakhfakh, Annick Michaud, Jacques P. Tremblay.785High Force Myosin Correlated with the Early Degeneration <strong>of</strong> JawClosing Muscle in the GRMD Model <strong>of</strong> DMDAndrew F. Mead, Alock Malik, Mihail Petrov, Martin Childers, Marilyn Mitchell, JanetBogan, Joe Kornegay, Brittani Nickens, Hansell Stedman.786Comparison Studies <strong>of</strong> rAAV6 Transduction Efficiency in Adult andAged MusclesPatryk Konieczny, James M. Allen, Eric Finn, Jeffrey S. Chamberlain.787<strong>Gene</strong> Transfer <strong>of</strong> Signals Critical for Allograft Healing UsingImmobilized Freeze Dried Adeno-Associated Viral VectorsMette Koefoed, Alexandra G. Pedersen, Michael Ulrich-Vinther, Thomas G. Jensen, KjeldSoballe.788Rescue from Respiratory Dysfunction by Transduction <strong>of</strong> Full-LengthDystrophin to Diaphragm Via the Peritoneal Cavity in MuscularDystrophy MouseIshizaki Masatoshi, Yasushi Maeda, En Kimura, Makoto Uchino.789Restoration <strong>of</strong> Wild-Type DMD mRNA in an Immortalized HumanFibroblast Line from an Exon 8-9 Duplicated DMD PatientLing Zhao, Gillian Butler-Browne, Vincent Mouly, Soraya Chaouch, Kamel Mamchaoui,Steve D. Wilton, Kevin M. Flanigan.790Expression <strong>of</strong> Micro-Dystrophin and Full-Length Dystrophin in Mouseand Dog MusclesChristophe Pichavant, Pierre Chapdelaine, Joe N. Kornegay, João C. S. Bizario, XiaoXiao, Jacques P. Tremblay.EXHIBITOR PROSPECTUSfinal program
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