13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010113Abstract DirectoryFriday, May 21, 2010Poster Session II— continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthCardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>432Identification <strong>of</strong> AAV6 as Most Efficient Vector for Transvascular<strong>Gene</strong> Transfer into Porcine Myocardium Based on an In Vitro Modelfor Prediction <strong>of</strong> the Cardiac <strong>Gene</strong> Transfer PerformanceJan Ksienzyk, Philip W. Raake, Philipp Schlegel, Changguang Shan, Christophe Weber,Jürgen A. Kleinschmidt, Hugo A. Katus, Oliver J. Müller.433Percutaneous Transendocardial Adminstration <strong>of</strong> AAV6 and AAV8Vectors to Rhesus Macaques Results in Highly Efficient and GlobalCardiac <strong>Gene</strong> TransferGuangping Gao, Meg M. Sleeper, Lawrence Bish, Lan Sun, You Lu, Jiachuan Duan,Chunyan Hu, Li Wang, H. Lee Sweeney.434<strong>Gene</strong> Transfer <strong>of</strong> VEGF165, HGF, Urokinase and Their CombinationsUsing Novel PC4W Plasmid for Induction <strong>of</strong> Vasculogenesis in MiceHind Limb Ischemia ModelPavel Makarevich, Zoya Tsokolaeva, Alexander Shevelev, Igor Rybalkin, EugeniyShevchenko, Irina Beloglazova, Tatyana Vlasik, Elena Parfyonova, Vsevolod A. Tkachuk.435PEGylation <strong>of</strong> Helper-Dependent Adenoviral Vector-MediatedLong-Term Expression <strong>of</strong> Human Apolipoprotein A-I ReducesAtherosclerosis in LDLR Deficient MiceEleonora Leggiero, Dalila Astone, Piyanuch Wonganan, Cristina Mazzaccara, GiuseppeLabruna, Luciana Sacchetti, Francesco Salvatore, Maria Croyle, Lucio Pastore.436AAV/IL13-<strong>Gene</strong> Delivery Lowers Atherogenesis in LDLR KO MiceJunaid A. Khan, Maohua Cao, Yong Liu, Paul L. Hermonat.437AAV/hSTAT3-<strong>Gene</strong> Delivery Lowers Atherosclerosis in LDLR KO Miceon High Cholesterol DietJunaid A. Khan, Maohua Cao, Bum-Yong Kang, Jawahar L. Mehta, Paul L. Hermonat.438Intramuscular Delivery <strong>of</strong> ACRX-100 Enhances Angiogenesis inRabbits with Hind Limb IschemiaTimothy J. Miller, Joseph M. Pastore, Rahul A. Aras, Marc S. Penn.439A Comparison <strong>of</strong> Three Liposomal Formulations for <strong>Gene</strong> Deliveryfrom Coronary Artery StentsSandra Ganly, Sean O. Hynes, Faisal Sharif, Karl McCullagh, Valerie Barron, Jim Crowley,Peter McHugh, Timothy O’Brien, Udo Greiser.440Efficient and Localized <strong>Gene</strong> Transference in Rat Heart byElectroporationLeonardo P. Carvalho, Eduardo G. Yasumura, Vivian Somoto, Priscila M. A. Denapoli,Flavia H. Silva, Valderez B. Valero, Hans F. Dohmann, Christina M. Takiya, Sang W. Han.Ophthalmic Disorders and Technological Advances441<strong>Gene</strong> Delivery <strong>of</strong> Erythropoietin Protects Retinal Ganglion <strong>Cell</strong>s inDBA/2J Glaucomatous MiceTimothy A. Sullivan, Eldon E. Geisert, Tonia S. Rex.442Pan-Retinal Secretion <strong>of</strong> GDNF Following Intravitreal Transduction <strong>of</strong>Müller Glia Slows Photoreceptor Loss in Retinal DegenerationDeniz Dalkara, Kathleen D. Kolstad, Meike Visel, Ryan R. Klimczak, Karen Guerin, DavidV. Schaffer, John G. Flannery.443AAV-Mediated <strong>Gene</strong> Supply <strong>of</strong> Non-Erythropoietic ErythropoietinDerivatives Improves Photoreceptor Morphology and Function inModels <strong>of</strong> Retinal Degenerative DiseasesPasqualina Colella, Carolina Iodice, Umberto Di Vicino, Alberto Auricchio.444Early Indicators <strong>of</strong> Biological Efficacy in a Phase 1/2 <strong>Gene</strong> <strong>Therapy</strong>Trial for Leber Congenital Amaurosis (LCA)Shalesh Kaushal, Margaret Humphries, Elena Filippova, George Asdourian, RadouilTzekov, Michael Stalvey, Terrence Flotte.445Positive Responses in a Mouse Model <strong>of</strong> Leber Congenital Amaurosisafter Treatment with Compacted DNA NanoparticlesKiichiro Okano, Tadao Maeda, Linas Padegimas, Tomasz H. Kowalczyk, Sharon M.Oette, Christopher R. Gedeon, Ozge Sesenoglu-Laird, Susannah L. Hyatt, Karla A. Dines,Elena Tyr, Nikia L. Beal, Robert C. Moen, Krzyszt<strong>of</strong> Palczewski, Mark J. Cooper.446Adeno-Associated Virus Mediated <strong>Gene</strong> <strong>Therapy</strong> with a RhodopsinRibozyme for Treatment <strong>of</strong> Autosomal Dominant Retinitis Pigmentosa(Adrp)Haoyu Mao, Marina S. Gorbatyuk, William W. Hauswirth, Alfred S. Lewin.447POD DNA Nanoparticles Rescue an Adult Mouse Model <strong>of</strong> RetinalDegenerationSarah P. Read, Siobhan M. Cashman, Rajendra Kumar-Singh.448Protection Against Laser-Induced Choroidal NeovascularizationFollowing Delivery <strong>of</strong> an Adenovirus Expressing a Soluble Form <strong>of</strong>Human CD59 to Murine RetinaSiobhan M. Cashman, Kasmir Ramo, Rajendra Kumar-Singh.449Dose Finding Study in Galactosialidosis Mice Using scAAV2/8-LP1-ppca VirusHuimin Hu, Elida Gomero, Eric Bonten, John Gray, Christopher Calabrese, Arthur Nienhuis,Alessandra d’Azzo.Friday, May 21 st
114<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryFriday, May 21 stFriday, May 21, 2010Poster Session II— continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthOphthalmic Disorders and Technological Advances450Characterization <strong>of</strong> 12 AAV Vectors for Intravascular Delivery ToTarget CNS and Detarget Non-CNS Tissues by miRNA Regulation:Implications in Treatment <strong>of</strong> Canavan DiseaseHongwei Zhang, Bin Yang, Jun Xie, Qing Xie, Qin Su, Ran He, Miguel Sena-Esteves,Zuoshang Xu, Guangping Gao.451Efficient Expression <strong>of</strong> Smn from Integration-Deficient LentiviralVectorsSherif O. Ahmed, Hugo Peluffo, Edmund Foster, Natalia Lago, Lawrence Moon, ThomasHutson, Ping Yip, Klaus Wanisch, Stephen B. McMahon, Rafael J. Yáñez-Muñoz.452Efficacy <strong>of</strong> an Adenovirus Vector/Cocaine Conjugate-Based Anti-Cocaine Vaccine in Shielding the Brain from Systemically AdministeredCocaineMartin J. Hicks, Bishnu P. De, Jonathan B. Rosenberg, Jesse T. Davidson, Amira Y.Moreno, Kim D. Janda, Neil R. Hackett, Stephen M. Kaminsky, Stefan Worgall, RonaldG. Crystal.453<strong>Gene</strong>tically Engineered Suppressor <strong>Cell</strong>s Target CNS and InhibitActivated Effector T-<strong>Cell</strong>sMoa Fransson, Elena Piras, Hao Wang, Ida Rasmusson, Katarina LeBlanc, Eva Brittebo,Angelica S. I. Loskog.Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II454First <strong>Gene</strong>ration AAVmicroutrophin Vector Infused into the IsolatedPelvic Limb <strong>of</strong> a Canine Model for Duchenne Muscular DystrophyMihail Petrov, Marilyn Mitchell, Alock Malik, Andy Mead, Frederick Balzer, Leonard Su,Jacqueline Farag, Benjamin Kozyak, Kapil Gopal, Charles Bridges, Janet Bogan, MartinChilders, Joe Kornegay, Hansell Stedman.455Characterization <strong>of</strong> A20 as an Inhibitor <strong>of</strong> NF-kB Activation inDystrophic MiceRakshita Charan, Paula R. Clemens.456Plasticity <strong>of</strong> Skeletal Muscle <strong>Cell</strong>s during Muscle Injury and Repair –A Dedifferentiation StudyXiaodong Mu, Hairong Peng, Johnny Huard, Yong Li.457Increased Muscle Regeneration in Hindlimb and Diaphragm <strong>of</strong> mdxMice Treated with AAV9 Mini-Dystrophin and Octalysine-NEMOBinding Domain PeptideDaniel P. Reay, Geno Raggi, Bing Wang, Xiao Xiao, Paul D. Robbins, Paula R. Clemens.458External, Non-Invasive Monitoring <strong>of</strong> Progressive CardiorespiratoryDysfunction in a Canine Model <strong>of</strong> DMDAndrew Mead, Alock Malik, Mihail Petrov, Martin Childers, Janet Bogan, JosephKornegay, Hansell Stedman.459The Polyproline Site in Hinge 2 Influences the Functional Capacity <strong>of</strong>Truncated DystrophinsGlen B. Banks, Luke M. Judge, James M. Allen, Jeffrey S. Chamberlain.460Combining <strong>Gene</strong> and Stem <strong>Cell</strong> <strong>Therapy</strong> in the Treatment <strong>of</strong>DysferlinophatiesDaniele Parolini, Claire Navarro, Andrea Farini, Mirella Meregalli, Marzia Belicchi, PaolaRazini, M. Krahn, Louis Garcia, Nicolas Lévis, Yvan Torrente.461Deletion <strong>of</strong> Functional Domains <strong>of</strong> Dp116 Leads to AlteredSarcolemmal Expression and Subcellular Localization When Expressedin Skeletal MuscleAndrea L. Arnett, Guy L. Odom, Kristy Boyle, Eric Finn, Jeffrey S. Chamberlain.462A New Therapeutic Approach for Duchenne Muscular Dystrophy:Restoration <strong>of</strong> the Dystrophin Reading Frame with a MeganucleaseJacques P. Tremblay, Pierre Chapdelaine, Christophe Pichavant, Joel Rousseau, FrédéricPâques.463Regulatory Cassettes Derived from the Slow Troponin I <strong>Gene</strong>Confer High-Level Expression in Skeletal Muscle after <strong>Gene</strong> TransferMediated by Helper-Dependent Adenovirus and Recombinant AAVRénald Gilbert, Mehdi Bendjelloul, Yué Zeng, Claude Guérin, Nancy Larochelle, GeorgeKarpati, Bernard Massie, Josephine Nalbantoglu.464<strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Muscular Dystrophy with Engineered Cd133+ <strong>Cell</strong>sAndrea Farini, Mirella Meregalli, Daniele Parolini, Marzia Belicchi, Simona Maciotta,Paola Razini, Joao da Silva Bizario, Luis Garcia, Nereo Bresolin, Yvan Torrente.465Improved Regenerative Capacity <strong>of</strong> Adult Stem <strong>Cell</strong>s Isolated fromInjured Skeletal Muscle <strong>of</strong> MouseXiaodong Mu, Ian Bellayr, Haiying Pan, Yong Li.466Sarcolemmal nNOS Anchoring Reveals a Functional Differencebetween Dystrophin and UtrophinYongping Yue, Dejia Li, Luke Judge, Akshay Bareja, Yi Lai, Kay E. Davies, Jeffrey S.Chamberlain, Dongsheng Duan.EXHIBITOR PROSPECTUSfinal program
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