FINAL PROGRAM - American Society of Gene & Cell Therapy

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13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010121Abstract DirectoryFriday, May 21, 2010Poster Session II — continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthCell Processing and Vector Production II575Escherichia coli Plasmid DNA Fermentation Strain and Process-SpecificEffects on Vector Yield, Quality and Transgene ExpressionAaron E. Carnes, Jeremy M. Luke, Justin M. Vincent, Clague P. Hodgson, James A.Williams.576An Assay To Monitor the Residual Contamination in the PlasmidDNA Manufacturing Area Based on the Correlation of Plasmid DNAConcentration to TOCRameswari Rameswaran, Lee Allen, Shane Johnson, Henry Hebel.577Counting and Sizing of Viral Vector Particles and Aggregates byNanoparticle Tracking Analysis (NTA)Andrew Malloy, Duncan Griffiths, Patrick Hole, Bob Carr.578Manufacturing and Stability Testing of a GNE-Lipoplex for a ClinicalTrial for the Treatment of Hereditary Inclusion Body Myopathy Type(HIBM)Chris M. Jay, Courtney Haddock, Phillip B. Maples, John Nemunaitis.579Dissolved Oxygen and pH Profile Evolution in Shake Flasks during293 Cell Growth and Infection with AdenovirusAna Carina Silva, Daniel Simão, Marcos F. Q. Sousa, Pedro E. Cruz, Manuel J. T.Carrondo, Paula M. Alves.Late Breaking Science II933Targeted Lentiviral Vectors for the Gene Therapy of X-Linked ChronicGranulomatous DiseaseChristian Brendel, Stefan Schultze-Strasser, Uta Müller-Kuller,Serap Tokmak, HanaKunkel, Linping Chen-Wichmann, Stefan Stein, Eva Rudolf, Michael Antoniou, ManuelGrez934Meganucleases: A Novel Anti-Viral ApproachJulianne Smith, Roman Galetto, Stephanie Grosse, Cécile Schiffer Mannioui, SylvainArnould, Marine Gailledrat, Philippe Duchateau, Frédéric Pâques & Carole Desseaux935Monomeric Designer T Cells are More Potent against IL13Ra2Expressing GBMSeogkyoung Kong, Richard P Junghans, , Prakash Sampath936pSuperShuttle – An Easy Method for Modifications at any Site in theAdenoviral Genome by Conventional Bacterial RecombinationDaniel Oberg, Nick R Lemoine, Gunnel Hallden937Generation and Characterization of Lipid-Encapsulated AAV vectors:A Strategy to Evade Neutralization by AAV AntibodiesJ. Fraser Wright, Sissel Lund-KatzFriday, May 21 st
122American Society of Gene & Cell TherapyAbstract DirectorySaturday, May 22 ndSaturday, May 22, 2010Oral Abstract Session 410Musculo-skeletal Gene & Cell Therapy11:00 AM - 1:00 PMRoom: Delaware Suite580 11:00 AMIrradiation of Adult mdx Mice Prior to Full-Length Murine DystrophincDNA Transfer Results in a Delayed and Diminished Anti-DystrophinHost Immune ResponseSaman Eghtesad, Heng Zheng, Hiroyuki Nakai, Michael W. Epperly, Paula R. Clemens.581 11:15 AMTransient Expression of a Therapeutic Dystrophin Transgene inDuchenne Muscular Dystrophy Revealed by T Cell Mediated ImmunityJerry R. Mendell, Katherine Campbell, Louise R. Rodino-Klapac, Zarife Sahenk, ChristopherShilling, Sarah Lewis, Dawn Bowles, Steven Gray, Chengwin Li, Gloria Galloway, VinodMalik, Brian Coley, K. Reed Clark, Juan Li, Xiao Xiao, Jade Samulski, Scott McPhee, R.Jude Samulski, Christopher M. Walker.582 11:30 AMInduction of Oral Immunotolerance to rAAV9-Microdystrophin inCanine X-Linked Muscular DystrophyHiromi H. Kinoh, Naoko Yugeta, Hironori Okada, Yuko N. Kasahara, Takashi Okada,Shin’ichi Takeda.583 11:45 AMBaculovirus-Engineered Mesenchymal Stem Cells Heal the Critical-Sized Femoral Segmental Bone Defects in RabbitsChin-Yu Lin, Yu-Han Chang, Kun-Ju Lin, Tzu-Chen Yen, Wen-Yi Lo, Chia-Hsin Luo, Ching-Lung Tai, Yu-Chen Hu.584 12:00 PMGenetic Evidence for a Structural Role of a–dystrobrevin-3 within theDystrophin-Glycoprotein ComplexGuy L. Odom, Glen B. Banks, Dewayne Townsend, Marvin E. Adams, Joseph M. Metzger,Stanely C. Froehner, Jeffrey S. Chamberlain.585 12:15 PMrAAV8-Mediated Protein-Anchoring Therapy for Targeting CollagenQ-Tailed Acetylcholinesterase to the Neuromuscular JunctionMikako Ito, Yumi Suzuki, Takashi Okada, Takayasu Fukudome, Toshiro Yoshimura, AkioMasuda, Shin’ichi Takeda, Eric Krejci, Kinji Ohno.586 12:30 PMA Single Patient, Multiple Intravenous (IV) Infusion, Clinical Trial ofGNE-Lipoplex for Therapy of Hereditary Inclusion Body Myopathy(HIBM)Gregory Nemunaitis, Chris M. Jay, Neil Senzer, Henry Allen, Anagha P. Phadke, Nancy S.Templeton, Phillip B. Maples, John Nemunaitis.587 12:45 PMSuccessful Expression of a R16/17 Micro-Dystrophin Gene inDystrophic Dogs with a Tyrosine-Mutant AAV Vector and a BriefCourse of Immune SuppressionJin-Hong Shin, Yi Lai, Yongping Yue, Arun Srivastava, Bruce F. Smith, Dongsheng Duan.Oral Abstract Session 411Chemical and Molecular Conjugates11:00 AM - 1:00 PMRoom: Virginia Suite588 11:00 AMTranscription Factor Binding by Plasmid DNA Is Needed forInteraction with Microtubules and Trafficking in CellsMelissa A. Badding, Erin E. Vaughan, David A. Dean.589 11:15 AMQuantitative Analysis of the Effect of Functional Peptides onIntracellular Trafficking of Polymer Gene Delivery SystemsNicole M. Moore, Denis Pristinski, Shelly E. Sakiyama-Elbert.590 11:30 AMThe Effects of PNA-Based prePEGylation on the In Vitro and In VivoProperties of DNA PolyplexesPeter G. Millili, Ulhas P. Naik, Millicent O. Sullivan.591 11:45 AMParticle Tracking for Intracellular Trafficking of Octaarginine-ModifiedLiposome: Comparative Study with AdenovirusHidetaka Akita, Kaoru Enoto, Tomoya Masuda, Hiroyuki Mizuguchi, Tomomi Tani,Hideyoshi Harashima.592 12:00 PMAdvanced In Vitro Models Combined with MathematicalRepresentations Yield New Quantitative Understanding of SyntheticGene Delivery System PerformanceMichelle C. Lowe, Todd D. Giorgio.593 12:15 PMGene Therapy of the Blood Brain Barrier: Systemic Delivery ofshRNA-Producing Plasmid DNA for Transient Suppression of MRP4Drug Efflux Transporter ProteinHongwei Zhang, Michelle L. Varney, Rakesh K. Singh, Serguei V. Vinogradov.594 12:30 PMPEGylated Polyacridine-Peptide DNA Open-Polyplexes with LongPharmacokinetic Half-Life Have Improved Hydrodynamic StimulatedGene ExpressionChristian A. Fernandez, Nicholas J. Baumhover, Jason Duskey, Sanjib Khargharia, KevinG. Rice.595 12:45 PMSuccessful Gene Delivery with Receptor-Targeted PAMAMDendrimers Occurs Via Caveolae-Mediated PathwaysMark E. Hwang, William R. Gunther, Daniel W. Pack.EXHIBITOR PROSPECTUSfinal program
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