Final Program - American Society of Gene & Cell Therapy

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