13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 93Abstract DirectoryThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthChemical and Molecular Conjugates I117Modular Ternary Polyacridine DNA Polyplexes for Targeted <strong>Gene</strong>DeliveryNicholas J. Baumhover, Christian A. Fernandez, Sanjib Khargharia, Kevin G. Rice.118Biodegradable Polyamino Acid-Based Polycations for Safe andEffective <strong>Gene</strong> Transfer Minimizing Cumulative ToxicityKeiji Itaka, Takehiko Ishii, Kazunori Kataoka.119Interplay <strong>of</strong> Clathrin and Caveolin Trafficking Pathways in Polymer-Based <strong>Gene</strong> DeliveryMark E. Hwang, Rahul K. Keswani, Daniel W. Pack.120Folate Linked Targeting Systems Increase Transfection Efficiency andSpecificity in Human Epithelial <strong>Cell</strong>sNathalie Carmoy, Emmanuel Mornet, Céline Lainé, Loic Lemiegre, Tony Le Gall, DanielleGillet, Mattias Lindberg, Rémi Marianowski, Pierre Lehn, Thierry Benvegnu, TristanMontier.121In Vivo <strong>Gene</strong> Delivery from Modified PLG ScaffoldsMisael O. Aviles, Lonnie D. Shea.122The Influence <strong>of</strong> Charge Density and Substituent Composition onImidazolium-Containing Polymers for Mediated DNA DeliveryMichael H. Allen, Matthew D. Green, Timothy E. Long.123Concentrated PEI Formulations Retain Biological Efficacy FollowingLong-Term Storage at 4°CLee A. Davies, Graciela A. Nunez-Alonso, Stephen C. Hyde, Deborah R. Gill.124Charge Density and Molecular Weight <strong>of</strong> Polymeric <strong>Gene</strong> CarrierAre Key Parameters Influencing Its DNA Compaction Ability andTransfection EfficiencyYong Ren, Xuan Jiang, Hai-Quan Mao.125Designing Imidazole-Containing Polymeric Vectors with ControlledCharge Densities for <strong>Gene</strong> DeliveryMatthew D. Green, John M. Layman, Alec S. Good, Sean M. Ramirez, Lulit A. Affin,Michael H. Allen, Timothy E. Long.126Bioreducible Poly(amido ethyleneimine) and Its PolyethyleneGlycol Graft Copolymer Analog Formulations Offer Ease <strong>of</strong> Use inOptimizing Improved Nucleic Acid DeliveryJames W. Yockman, Jonathan H. Brumbach, Young Won Lee, Chao Lin, Johan F. J.Enbersen, Jan Feijen, Sung Wan Kim.127Role <strong>of</strong> Glycosaminoglycans in Peptide-Mediated DNA DeliveryRangeetha J. Naik, Pallavi Chandra, Anindo Chatterjee, Munia Ganguli.128Galactosylated-DPC Nanoparticles for Targeted Hepatic <strong>Gene</strong>Delivery In VivoKeun-Sik Kim, Yeon Kyung Lee, Jung Seok Kim, Jin Ee Back, Sang Il Park, Hwa YeonJeong, Young Eun Shin, Yong Serk Park.129Effect <strong>of</strong> Molecular Weight on Plasmid DNA Delivery Efficiency <strong>of</strong>Palmitic Acid Substituted PolyethylenimineK. C. B. Remant, Breanne Landry, Hasan Uludag.130Electrospun Fibers with the Core-Loading <strong>of</strong> DNA andPoly(ethyleneimine) Complex as Potential Tissue Engineering ScaffoldYe Yang, Long Cheng, Shuhui He, Jie Zou, Fang Chen, Xiaohong Li.131A Novel Cationic Lipophosphoramide with Phytanyl Chains: Gain inTransfection Efficiency and Clinical Approach Concerning the Responseto Lip<strong>of</strong>ectionMattias Lindberg, Nathalie Carmoy, Christophe Lorilleux, Aurore Fraix, Tony Le Gall,Pierre Lehn, Paul-Alain Jaffrès, Tristan Montier.132Cationic Peptide-Based DNA Delivery In Vitro: The Role <strong>of</strong> DNACondensation and ReleaseAnita Mann, Garima Thakur, Vasundhara Shukla, Rangeetha Naik, Anand Kamal Singh,Richa Khanduri, Munia Ganguli.<strong>Gene</strong>tic and Metabolic Diseases <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I133Modeling and Correcting Fibrous Dysplasia with Lentiviral VectorsIsabella Saggio, Cristina Remoli, Stefania Cersosimo, Stefania Piersanti, Paolo Bianco,Mara Riminucci.134Bone Marrow Transplantation Cures Hemophilia AAntonia Follenzi, Sanj Raut, Simone Merlin, Rita Sarkar, Sanjeev Gupta.135<strong>Cell</strong> Autonomy in Methylmalonic Acidemia (MMA): Implications for<strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong>Irini Manoli, Justin R. Sysol, Randy J. Chandler, Jennifer Sloan, Patricia Zerfas, VictoriaH<strong>of</strong>fmann, Mones Abu-Asab, Maria Tsokos, Charles P. Venditti.Thursday, May 20 th
94<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryThursday, May 20 thThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-South<strong>Gene</strong>tic and Metabolic Diseases <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I136Efficacious <strong>Gene</strong> <strong>Therapy</strong> in Pompe Disease Requires Mannose-6-Phosphate Receptor ExpressionBaodong Sun, Deeksha Bali, Y.-T. Chen, Dwight D. Koeberl.137Development <strong>of</strong> a Safe Hematopoietic Stem <strong>Cell</strong> (HSC) MobilizationStrategy for <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> ThalassemiaEvangelia Yannaki, Thalia Papayannopoulou, Erica Jonlin, Ioannis Batsis, Pamela Becker,Fani Zervou, Angeliki Xagorari, Garyfalia Karponi, Varnavas Constantinou, AchillesAnagnostopoulos, Athanasios Fassas, George Stamatoyannopoulos.138Long Term Expression in Liver from scAAV Seems Derived fromIntegrated Vector GenomesPaula S. M. Miranda, Maude Flageul, Christine Kaeppel, Lysbeth ten Bloemendaal,Dominique Aubert, Nowrouzi Ali, Manfred Schmidt, Nicolas Ferry, Piter J. Bosma.139Human Amnion Epithelial (AE) Stem <strong>Cell</strong> Transplant Improves DiseasePhenotype and Survival in Mouse Models <strong>of</strong> Intermediate MapleSyrup Urine Disease (MSUD)K. J. Skvorak, K. Dorko, M. Hansel, F. Marongiu, V. Tahan, K. M. Gibson, Q. Sun, T.Bottiglieri, E. Arning, J. Davila, S. Strom.140<strong>Gene</strong> <strong>Therapy</strong> Approaches Combining the ImmunomodulatoryProperties <strong>of</strong> Liver and IGF-I To Prevent Type I DiabetesSabrina Tafuro, Xavier M. Anguela, Judith Agudo, Alba Casellas, David Callejas, MercèObach, Carles Roca, Albert Ruzo, Fàtima Bosch.141Hepatocyte Engineering and <strong>Gene</strong>tic Modification Approaches <strong>of</strong>Hemophilia Hepatocytes within a Living MouseKazuo Ohashi, Kohei Tatsumi, Chise Tateno, Hiroyuki Nakai, Anja Ehrhardt, Rie Utoh,Katsutoshi Yoshizato, Teruo Okano.142Serotype Optimization <strong>of</strong> an AAV Vector for <strong>Gene</strong> <strong>Therapy</strong> in Canineand Murine Glycogen Storage Disease Type Ia (GSD-Ia)Xiaoyan Luo, Amanda K. Demaster, Songtao Li, Kyha Williams, Talmage T. Brown, DanielM. Kozink, Andrew Bird, Dwight D. Koeberl.143Systemic Delivery <strong>of</strong> Bioactive Glucagon-Like Peptide 1 FollowingAdenoviral Vector-Mediated <strong>Gene</strong> Transfer to Murine SubmandibularGlandsAntonis Voutetakis, Anne M. Rowzee, Ana P. Cotrim, Changyu Zheng, Trushar Rathod,Tulin Yanik, Y. P. Loh, Bruce J. Baum, Niamh X. Cawley.144A Non-Viral, GNE-Lipoplex Treatment to Correct Sialylation Defectsin Gne-Mutant (M712T) MiceTal Yardeni, Carla Ciccone, Shelley Hoogstraten-Miller, Daniel Darvish, Yair Anikster, PhilMaples, Chris Jay, William A. Gahl, John Nemunaitis, Marjan Huizing.145Lentivirally Induced Over-Expression in the Mouse Hypothalamus;a Study <strong>of</strong> the Effects <strong>of</strong> Melanin Stimulating Hormones (MSH) inObesity and DiabetesKim Eerola, Siru Virtanen, Eriika Savontaus, Mikko Savontaus.146Alternative Methods for Ex Vivo <strong>Gene</strong> Transfer to Primary Murine B<strong>Cell</strong>Babak Moghimi, Ou Cao.147A Full Characterization <strong>of</strong> the CAG 140 Huntington’s Disease MouseModelAaron C. Rising, Eileen Denovan-Wright, Ron Mandel.Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I148Assessment <strong>of</strong> Pre-Clinical Safety <strong>of</strong> GMP Grade GNE-Lipoplexesafter Repeat Intravenous Injections in Balb/c MiceAnagha P. Phadke, Chris M. Jay, Phillip B. Maples, Neil Senzer, John Nemunaitis, AlexW. Tong.149Pre-Existing Antibodies to AAV8 Attenuates Micro-DystrophinExpression Following Targeted Vascular DeliveryLouise R. Rodino-Klapac, Chrystal Montgomery, William Bremer, Nancy Davis, KimberlyShontz, Vinod Malik, Katherine Campbell, Thomas J. Preston, Zarife Sahenk, K. ReedClark, Brian D. Coley, Christopher M. Walker, Jerry R. Mendell, Louis G. Chicoine.150AAV Microgene Transfer Reveals the <strong>Cell</strong>ular Motif for Dystrophin-Mediated Sarcolemmal Neuronal Nitric Oxide Synthase (nNOS)LocalizationYi Lai, Yongping Yue, Dongsheng Duan.151<strong>Cell</strong> Therapeutic Approach to Duchenne Muscular Dystrophy UsingMyogenic Differentiation <strong>of</strong> Multipotent Mesenchymal Stromal <strong>Cell</strong>sYuko N. Kasahara, Hiromi H. Kinoh, Hironori Okada, Jin-Hong Shin, Akiyo Nishiyama,Sachiko O. Hosoyama, Michiko W. Maeda, Akinori Nakamura, Takashi Okada, Shin’ichiTakeda.EXHIBITOR PROSPECTUSfinal program
- Page 1 and 2:
FINAL PROGRAMAmerican Society of Ge
- Page 4:
13 th AnnUAL MEETing | Washington,
- Page 7 and 8:
6American Society of Gene & Cell Th
- Page 12 and 13:
13 th AnnUAL MEETing | Washington,
- Page 20 and 21:
13 th AnnUAL MEETing | Washington,
- Page 22 and 23:
13 th AnnUAL MEETing | Washington,
- Page 24 and 25:
13 th AnnUAL MEETing | Washington,
- Page 26 and 27:
13 th AnnUAL MEETing | Washington,
- Page 28 and 29:
13 th AnnUAL MEETing | Washington,
- Page 30 and 31:
13 th AnnUAL MEETing | Washington,
- Page 32 and 33:
13 th AnnUAL MEETing | Washington,
- Page 34 and 35:
13 th AnnUAL MEETing | Washington,
- Page 36 and 37:
13 th AnnUAL MEETing | Washington,
- Page 38 and 39:
13 th AnnUAL MEETing | Washington,
- Page 40 and 41:
13 th AnnUAL MEETing | Washington,
- Page 42 and 43:
13 th AnnUAL MEETing | Washington,
- Page 44 and 45: 13 th AnnUAL MEETing | Washington,
- Page 46 and 47: 13 th AnnUAL MEETing | Washington,
- Page 48 and 49: 13 th AnnUAL MEETing | Washington,
- Page 50 and 51: 13 th AnnUAL MEETing | Washington,
- Page 52 and 53: 13 th AnnUAL MEETing | Washington,
- Page 54 and 55: 13 th AnnUAL MEETing | Washington,
- Page 56 and 57: 13 th AnnUAL MEETing | Washington,
- Page 58 and 59: 13 th AnnUAL MEETing | Washington,
- Page 60 and 61: 13 th AnnUAL MEETing | Washington,
- Page 62 and 63: 13 th AnnUAL MEETing | Washington,
- Page 64 and 65: 13 th AnnUAL MEETing | Washington,
- Page 66 and 67: 13 th AnnUAL MEETing | Washington,
- Page 68 and 69: 13 th AnnUAL MEETing | Washington,
- Page 70 and 71: 13 th AnnUAL MEETing | Washington,
- Page 72 and 73: 13 th AnnUAL MEETing | Washington,
- Page 74 and 75: 13 th AnnUAL MEETing | Washington,
- Page 76 and 77: 13 th AnnUAL MEETing | Washington,
- Page 78 and 79: 13 th AnnUAL MEETing | Washington,
- Page 80 and 81: 13 th AnnUAL MEETing | Washington,
- Page 82 and 83: 13 th AnnUAL MEETing | Washington,
- Page 84 and 85: 13 th AnnUAL MEETing | Washington,
- Page 86 and 87: 13 th AnnUAL MEETing | Washington,
- Page 88 and 89: 13 th AnnUAL MEETing | Washington,
- Page 90 and 91: 13 th AnnUAL MEETing | Washington,
- Page 92 and 93: 13 th AnnUAL MEETing | Washington,
- Page 96 and 97: 13 th AnnUAL MEETing | Washington,
- Page 98 and 99: 13 th AnnUAL MEETing | Washington,
- Page 100 and 101: 13 th AnnUAL MEETing | Washington,
- Page 102 and 103: 13 th AnnUAL MEETing | Washington,
- Page 104 and 105: 13 th AnnUAL MEETing | Washington,
- Page 106 and 107: 13 th AnnUAL MEETing | Washington,
- Page 108 and 109: 13 th AnnUAL MEETing | Washington,
- Page 110 and 111: 13 th AnnUAL MEETing | Washington,
- Page 112 and 113: 13 th AnnUAL MEETing | Washington,
- Page 114 and 115: 13 th AnnUAL MEETing | Washington,
- Page 116 and 117: 13 th AnnUAL MEETing | Washington,
- Page 118 and 119: 13 th AnnUAL MEETing | Washington,
- Page 120 and 121: 13 th AnnUAL MEETing | Washington,
- Page 122 and 123: 13 th AnnUAL MEETing | Washington,
- Page 124 and 125: 13 th AnnUAL MEETing | Washington,
- Page 127 and 128: 126American Society of Gene & Cell
- Page 129 and 130: 128American Society of Gene & Cell
- Page 131 and 132: 130American Society of Gene & Cell
- Page 133 and 134: 132American Society of Gene & Cell
- Page 135 and 136: 134American Society of Gene & Cell
- Page 137 and 138: 136American Society of Gene & Cell
- Page 139 and 140: 138American Society of Gene & Cell
- Page 141 and 142: 140American Society of Gene & Cell
- Page 143 and 144: 142American Society of Gene & Cell
- Page 145 and 146:
144American Society of Gene & Cell
- Page 147 and 148:
146American Society of Gene & Cell
- Page 149 and 150:
148American Society of Gene & Cell
- Page 151 and 152:
150American Society of Gene & Cell
- Page 153 and 154:
152American Society of Gene & Cell
- Page 155 and 156:
154American Society of Gene & Cell
- Page 157 and 158:
156American Society of Gene & Cell
- Page 159 and 160:
158American Society of Gene & Cell
- Page 161 and 162:
160American Society of Gene & Cell
- Page 163 and 164:
162American Society of Gene & Cell
- Page 165 and 166:
164American Society of Gene & Cell
- Page 167 and 168:
166American Society of Gene & Cell
- Page 169 and 170:
168American Society of Gene & Cell
- Page 171 and 172:
170American Society of Gene & Cell
- Page 173 and 174:
172American Society of Gene & Cell
- Page 175 and 176:
174American Society of Gene & Cell
- Page 177 and 178:
176American Society of Gene & Cell
- Page 179 and 180:
178American Society of Gene & Cell
- Page 181 and 182:
180American Society of Gene & Cell
- Page 183 and 184:
182American Society of Gene & Cell
- Page 185 and 186:
184American Society of Gene & Cell
- Page 187 and 188:
186American Society of Gene & Cell
Change language