Final Program - American Society of Gene & Cell Therapy

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Abstract Directory, Friday, May 17, 2013 Poster Session II, continued 5:30 PM - 7:30 PM Room: Exhibit Hall C/D AAV Vectors II 333 High-Eficiency Transduction of Primary Human Monocyte-Derived Dendritic Cells by Recombinant AAV6 Vectors Containing Mutations in Surface-Exposed Serine and Threonine Residues George Aslanidi, Angela Rivers, Luis Ortiz, Jhell Pandya, Chen Ling, Kim Van Vliet, Mavis Agbandje-McKenna, Arun Srivastava. 334 Optimization of the Capsid of Recombinant Adeno- Associated Virus 2 Vectors: The Final Threshold George Aslanidi, Angela Rivers, Luis Ortiz, Chen Ling, Kim Van Vliet, Lakshmanan Govindasamy, Mavis Agbandje-McKenna, Arun Srivastava. 335 Directed Evolution of AAV Capsid for Targeted Cancer Therapy Damien Marsic, Yuan Lu, Chunxia Cao, Dane Phelan, Lakshmanan Govindasamy, Frederic J. Kaye, Mavis Agbandje-McKenna, Steve Ghivizzani, Sergei Zolotukhin. 336 Structures of the Retargeted AAV-DJ Vector in the Presence and Not of Receptor-Analog Sucrose Octasulfate Qing Xie, Nancy Meyer, Thomas F. Lerch, Dustin McCraw, Michael Spillman, Scott Stagg, Michael S. Chapman. 337 Improved AAV1 and AAV9 Vectors for Gene Delivery Balaji Balakrishnan, Akshaya Krishnagopal, Dwaipayan Sen, Alok Srivastava, Giridhara r Jayandharan. 338 Plasmid Integration within AAVS1 in AAV Producer Cell Lines Amy Frederick, Yuxia Luo, John Martin, Jeffery Ardinger, Donna Armentano, Sam Wadsworth, Karen Vincent. 339 Short Hairpin RNA Delivered by Adeno-Associated Virus Vector Eficiently Reduced Target Gene Expression in Mouse Primary Cortical Neurons Yun Lin, Holly Carlisle, Tom Wolfe, Danny Ortuno, Trish Amarante, Huiren Zhao, Steve Wood, Cherylene Plewa, Mike Wyszynski, Paul Kassner, Ki Jeong Lee. 340 AAV Vectors Puriied from Culture Medium with a Simple and Rapid Protocol Transduce Eficiently Murine Liver, Muscle and Retina Monica Doria, Antonella Ferrara, Alberto Auricchio. DNA Vectorology & Gene Targeting I 341 Minimally Invasive Electroporation of Synthetic DNA Vaccines for the Induction CTL and Functional Humoral Immune Responses Against HIV Megan Wise, Karuppiah Muthumani, Kate E. Broderick, Amir S. Khan, Janess Mendoza, Maria Yang, Jian Yan, Natalie Hutnick, Niranjan Y. Sardesai, David B. Weiner. 342 An E3-14.7K Peptide That Promotes Microtubules- Mediated Transport of Plasmid DNA Increases Polyplexes Transfection Eficiency Lucie Pigeon, Cristine Gonçalves, David Gosset, Chantal Pichon, Patrick Midoux. 343 Benchtop DNA Synthesizer: Oligo-Templated Polymerization (OTP) Adi Barzel, Mark A. Kay. 344 Nanoparticle-Mediated Gene Delivery for Rhodopsin- Associated Retinitis Pigmentosa Zongchao Han, Rasha Makkia, Junjing Guo, Miles J. Merwin, Mark J. Cooper, Muna I. Naash. 345 Hybrid Flp-TALE and Cre-TALE Recombinases with Designed Target Speciicity as Genome Engineering Tools Feng Li, Riddhi Shah, Eugenia Voziyanova, Yuri Voziyanov. 346 Permanent Genetic Modiication of Dividing Cells Using Episomally Maintained S/MAR DNA Vectors and the Correction of a Cancer Phenotype in Renal Tumour Cells Suet Ping Wong, Richard P. Harbottle. 347 Collagen VII Gene Delivery Via Sleeping Beauty Transposon in COL7A1-Deicient Keratinocytes from Epidermolysis Bullosa Patients Maria Carmela Latella, Fabienne Cocchiarella, Giandomenico Turchiano, Zsuzsanna Izsvak, Zoltan Ivics, Manuel Gonçalves, Fulvio Mavilio, Fernando Larcher, Alessandra Recchia. 348 DNAJB4, a Heat Shock Rotein hsp40 Homolog, Activates Dendritic Cells and Functions as a Molecular Adjuvant When Incorporated into Cancer DNA Vaccine Hsing-Yu Chen, Jeremy. J.W. Chen, Chi-Chen Lin. 349 Surface-Stabilized Calcium Phosphate Nano-Crystals for Eficient Gene Delivery Min Sang Lee, Kyuri Lee, Nak Won Kim, Ji Hoon Jeong. Friday, May 17, 2013 Final Program SALT LAKE CITY, UTAH May 15–18, 2013 113
Abstract Directory, Friday, May 17, 2013 Friday, May 17, 2013 Poster Session II, continued 5:30 PM - 7:30 PM Room: Exhibit Hall C/D DNA Vectorology & Gene Targeting I 350 Polymeric Micellar Nanocarriers for Gene and Oligonucleotide Delivery Kazunori Kataoka. 351 Methods of Facilitating Patient Participation in DNA Based Experimental Therapeutic Phase I Cancer Studies Wanda Strange, Cynthia Bedell, Shannon Cagnina, Jeanne Jones, Patricia Brown, Gladice Wallraven, John Nemunaitis. 352 Manufacturing Developments for Scale-Up of Autologous Phase II Ovarian Tumor Tissue FANG Vaccine Nicolas Taquet, John Nemunaitis, Phillip B. Maples. Gene & Cell Therapy of Diabetes, Metabolic and Genetic Diseases I 353 Ultrasound-Guided Hepatic Injections of HDAd Vector Are More Eficient and Safer Than Systemic Intravenous Injections for Liver-Directed Gene Therapy of Crigler- Najjar Syndrome Type I Nunzia Pastore, Edoardo Nusco, Pasquale Piccolo, Sigismondo Castaldo, Francesco Vetrini, Donna Palmer, Libor Vitek, Philip Ng, Nicola Brunetti-Pierri. 354 Gene Therapy for Liver Diseases Using Non-Viral Minicircle-DNA Vector Hiu Man Viecelli, Richard P. Harbottle, Suet Ping Wong, Marinne Chuah, Thierry VandenDriessche, Cary O. Harding, Beat Thony. 355 Hepatic Delivery of Genes Encoding Therapeutic Proteins or Mirnas Using Hyperactive Transposons: A Platform Technology for Gene Therapy and Functional Genomics In Vivo Mario Di Matteo, Pieter De Bleser, Ermira Samara-Kuko, Marinee Chuah, Thierry VandenDriessche. 356 Liver Progenitor Cells Isolated from Adult Mice Fed a Choline-Deicient, Ethionine-Supplemented Diet Can Engraft, Expand and Mature in the Injured Liver of the FRG Mouse Model Gustavo de Alencastro, Grant J. Logan, Allison P. Dane, Sophia H. Y. Liao, George C. T. Yeoh, Ian E. Alexander. 357 Long-Term Phenotypic Correction of Hemophilia A by Non-Viral PiggyBac Transposon Vector Hideto Matsui, Mitsuhiko Sugimoto, Midori Shima, Naoko Fujimoto, Akitsu Hotta. 358 Long-Term Secretion of Arylsulfatase A without Antibody Formation after AAV Mediated Neonatal Gene Transfer into MLD Model Mice Noriko Miyake, Koichi Miyake, Motoko Yamamoto, Takashi Shimada. 359 In Vitro and In Vivo Characterization of an Engineered Arylsulfatase B for Therapy of Mucopolysaccharidosis VI Rita Ferla, Pamela Claudiani, Gabriella Cotugno, Aaron Petrey, Richard Steet, Alberto Auricchio. 360 Gene Therapy for MPS VI Is Effective in Cats without Pre-Existing Immunity to AAV8 Rita Ferla, Thomas O’Malley, Roberto Calcedo, Patricia O’Donnell, Ping Wang, Gabriella Cotugno, Pamela Claudiani, James M. Wilson, Mark Haskins, Alberto Auricchio. 361 Correlation of Gene Transfer and Transduction to Chloride Current Defect from rAAV2/5 Vector Derived Transgene DCFTR in Murine CF Nasal Airway Gang Shi, Kelvin D. MacDonald, Christian Mueller, Terence R. Flotte, Anne C. Fischer. 362 Helper-Dependent Adenoviral Vectors for Liver- Directed Gene Therapy of Primary Hyperoxaluria Type 1 Raffaele Castello, Stefania D”Aria, Roberta Borzone, Pasquale Piccolo, Patrizia Annunziata, Nicola Brunetti-Pierri. 363 Fetal Gene Therapy at 0.4G with AAV-hFIX or AAV-hFX Mediates Therapeutic Transgene Expression Despite Growth and a Positive Immune Response in a Non- Human Primate Model Citra Mattar, Cecilia Rosales, Rowell Fragata, Nuryanti Johana, Simon Waddington, Arijit Biswas, Mahesh Choolani, Amit Nathwani, Jerry Chan. 364 AAV Vector-Mediated Iduronidase Gene Delivery in a Murine Model of Mucopolysaccharidosis Type I: Comparing Different Routes of Delivery to the CNS Lalitha Belur, Zhenhong Nan, Kelly M. Podetz-Pedersen, Carolyn A. Fairbanks, Leah R. Hanson, William H. Frey, Karen Kozarsky, Walter C. Low, R. Scott McIvor. 114 Final Program SALT LAKE CITY, UTAH May 15–18, 2013
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ASGCT Officers and Board of Directo
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Annual Meeting Exhibitors - as of A
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Committee Rosters Advisory Council
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Committee Rosters Program Committee
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Abstract Reviewers Cancer - Immunot
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Outstanding New Investigator Awards
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Faculty List Thomas W. Chalberg, Jr
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Faculty List Ann M. Leen, PhD Baylo
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Faculty List Evan Y. Snyder, MD PhD
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General Meeting Information Continu
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General Meeting Information Speaker
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Salt Palace Convention Center Floor
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Salt Palace Convention Center Floor
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Program Schedule, Wednesday, May 15
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Final Program - American Society of Gene & Cell Therapy

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