57th ASH Annual Meeting and Exposition (December 5-8, 2015): 801. Gene Therapy and Transfer: Poster I

Gene Therapy and Transfer Oral and Poster Abstracts
Poster

Hall A, Level 2 (Orange County Convention Center)

2035

Long-Term Increase in Fetal Hemoglobin Expression in Nonhuman Primates Following Transplantation of Autologous Bcl11a Nuclease-Edited HSCs

Olivier Humbert, PhD^* and Hans-Peter Kiem, MD

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA

2036

High Titer Lentivector from a Stable Lenti-Producer Efficiently Corrects CD34+ Hematopoietic Stem Cells from Patients with p47phox-Deficient Chronic Granulomatous Disease

Uimook Choi, Ph.D.¹^*, Narda Theobald¹^*, Throm E Robert, Ph.D.²^*, John Gray, Ph.D.³^*, David J. Rawlings, MD⁴^*, Brian P. Sorrentino, MD⁵, Harry Malech¹ and Suk See De Ravin, MD¹^*

¹LHD, NIAID, NIH, Bethesda, MD
²Division of Experimental Hematology, St Jude Children’s Research Hospital, Memphis, TN
³Research & Developement, Audentes Therapeutics, San Francisco, CA
⁴Seattle Children's Research Institute, Seattle, WA
⁵Division of Experimental Hematology, St. Jude Children's Research Hospital, Memphis, TN

2037

Single-Cell-State Culture of Human Pluripotent Stem Cells Increases Transfection Efficiency

Takenobu Nii, PhD¹^*, Hiroshi Kohara, PhD²^*, Tomotoshi Marumoto, MD, PhD¹^*, Tetsushi Sakuma, PhD³^*, Takashi Yamamoto, PhD³^* and Kenzaburo Tani, MD, PhD²

¹Division of Molecular and Clinical Genetics, Medical Institute of Bioregulation, Kyushu University, Fukuoka, Japan
²Project Division of ALA Advanced Medical Research, The Institute of Medical Science, The University of Tokyo, Tokyo, Japan
³Department of Mathematical and Life Sciences, Graduate School of Science, Hiroshima University, Hiroshima, Japan

2038

The Light Chain of Rat Factor VIII Confers Its Superior Cofactor Activity

Qizhao Wang, PhD¹^*, Jenni Firrman, PhD²^*, Katie A Pokiniewski²^*, Wenjing Cao, M.D., Ph.D.³^*, Hongying Wei, PhD¹^*, Roland Herzog, PhD⁴ and Weidong Xiao, PhD^1,2,5^*

¹Sol Sherry Thrombosis Research Center, Temple university, Philadelphia, PA
²Department of Microbiology and Immunology, Temple university, Philadelphia, PA
³Division of Laboratory Medicine, Department of Pathology, The University of Alabama at Birmingham, Birmingham, AL
⁴Pediatrics/CMT, University of Florida, Gainesville, FL
⁵Cardiovascular Research Center, Temple university, Philadelphia, PA

2039

Ultrasound Combined with Microbubbles Mediated Factor VIII Plasmid Gene Therapy in Hemophilia a Mice

Shuxian Song, PhD¹^*, James Harrang¹^*, Bryn Smith¹^* and Carol H. Miao, PhD^1,2

¹Seattle Children's Research Institute, Seattle, WA
²Department of Pediatrics, University of Washington, Seattle, WA

2040

Adeno-Associated Virus 8 (AAV8) Vector Genome Biodistribution into Body Fluids Following Single Intravenous Administration of BAX 335 Gene Therapy for Hemophilia B

Boyan Litchev, MD¹, Paul E. Monahan, MD² and Anne Prener¹^*

¹Baxalta US, Inc., Cambridge, MA
²Pediatrics, Harold R. Roberts Comprehensive Hemophilia Treatment Center, Gene Therapy Center, University of North Carolina, Chapel Hill, NC

2041

AAV Gene Therapy for MPS1 Corneal Clouding and Joint Stiffness

Melisa A. Vance, PhD¹, Matthew Hirsch, PhD²^*, Richard Jude Samulski, PhD³^*, Joanne Kurtzberg, MD⁴, Laurie Goodrich, DVM, PhD, ACVS Diplomate⁵^* and Llanga Telmo, Bsc⁶^*

¹UNC gene therapy center/ Hematology department, University of North Carolina at Chapel Hill, Chapel Hill, NC
²UNC gene therapy center/ Ophthalmology department, University of North Carolina at Chapel Hill, Chapel Hill
³UNC gene therapy, University of North Carolina at Chapel Hill, Chapel Hill, NC
⁴Division of Pediatric Blood and Marrow Transplantation, Department of Pediatrics, Duke University Medical Center, Durham, NC
⁵College of Veterinary Medicine, Colorado State University, Fort Collins, CO
⁶UNC gene Therapy center, University of North Carolina at Chapel Hill, Chapel Hill, MO

2042

Pharmacodynamic Profile and Clinical Response in Patients with B-Cell Malignancies of Anti-CD19 CAR T-Cell Therapy

Arianne Perez, PhD¹^*, Lynn Navale, MS¹^*, John M. Rossi, MS¹^*, Yueh-wei Shen, MS¹^*, Yizhou Jiang, PhD¹^*, Marika Sherman, MS¹^*, Armen Mardiros, PhD¹, Sean C. Yoder, MS¹^*, William Y. Go, MD, PhD¹, Steven A. Rosenberg, MD, PhD²^*, Jeff Wiezorek, MD, MS¹^*, Margo R. Roberts, PhD¹^*, David D. Chang, MD, PhD¹^*, James N. Kochenderfer, MD³ and Adrian Bot, MD, PhD¹^*

¹Kite Pharma, Santa Monica, CA
²Surgery Branch, NCI, NIH, Bethesda, MD
³Experimental Transplantation and Immunology Branch, National Cancer Institute/National Institutes of Health, Bethesda, MD

2043

Automated Lentiviral Transduction of T Cells with Cars Using the Clinimacs Prodigy

Ulrike Mock*, PhD¹^*, Lauren Nickolay*, PhD¹^*, Gordon Weng-Kit Cheung²^*, Hong Zhan, PhD¹^*, Karl Peggs, MBBChir, MRCP, FRCPath³^*, Ian CD Johnston, PhD⁴^*, Andrew Kaiser, PhD⁴^*, Martin Pule, PhD²^*, Adrian Thrasher, MD, PhD¹^* and Waseem Qasim, MBBS PhD¹^*

¹Institute of Child Health / Molecular and Cellular Immunology Unit, University College London, London, United Kingdom
²Cancer Institute, University College London, London, United Kingdom
³CANCER INSTITUTE, UCL, London, United Kingdom
⁴Research & Development, Miltenyi Biotec GmbH, Bergisch Gladbach, Germany

2044

Development of Optimized Protocol for Generation of NK Cells Expressing Chimeric Antigen Receptors from Hematopoietic Stem Cells for Cancer Immunotherapy

Amie Patel¹^*, Laurel Christine Truscott, MD² and Satiro N. De Oliveira, MD³

¹Western University of Health Sciences, Pomona, CA
²Ucla-Mattel Children's Hospital, Los Angeles, CA
³Dept. of Pediatrics, University of California, Los Angeles, Los Angeles, CA

2045

Megatal, Crispr/Cas9, and Talen T-Cell Receptor Gene Editing

Mark J. Osborn, PhD¹^*, Beau R Webber, PhD²^*, Friederike Knipping³^*, Cara-lin Lonetree²^*, Anthony DeFeo²^*, Amber McElroy²^*, Colby Starker, PhD²^*, Richard Gabriel⁴^*, Sarah Merkel²^*, Keli L. Hippen, PhD⁵^*, Daniel Voytas, PhD²^*, Christof von Kalle, MD⁶^*, Manfred Schmidt, PhD³^*, Jeffrey S. Miller, MD², Jordan Jarjour, PhD⁷^*, Alexander Astrakhan, PhD⁷^*, Andrew M Scharenberg, MD⁸^*, Jakub Tolar, M.D., Ph.D.¹ and Bruce R. Blazar, MD⁹

¹Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN
²University of Minnesota, Minneapolis, MN
³German Cancer Research Center (DKFZ), Heidelberg, Germany
⁴Department of Translational Oncology, Heidelberg, Germany
⁵Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, MN
⁶Department of Translational Oncology, National Center for Tumor Diseases and German Cancer Research Center, Heidelberg, Germany
⁷BlueBird Biosciences, Seattle, WA
⁸Seattle Children's Research Institute, Seattle, WA
⁹Division of Pediatrics, Department of Medicine, University of Minnesota Cancer Center, Minneapolis, MN

2046

First Clinical Application of Talen Engineered Universal CAR19 T Cells in B-ALL

Waseem Qasim, MBBS PHD¹^*, Persis Jal Amrolia²^*, Sujith Samarasinghe, MD, PhD³^*, Sara Ghorashian, MD, PhD, FRCPath¹^*, Hong Zhan, PhD⁴^*, Sian Stafford, PHD¹^*, Katie Butler, PHD¹^*, Gul Ahsan⁵^*, Kimberly Gilmour⁵^*, Stuart Adams, PHD⁵^*, Danielle Pinner⁵^*, Robert Chiesa⁵^*, Steve Chatters, PHD⁵^*, Sue Swift, PHD¹^*, Nicholas Goulden, MD, PhD³, Karl Peggs, MBBChir, MRCP, FRCPath⁶^*, Adrian J Thrasher, MD, PhD¹^*, Paul Veys²^* and Martin Pule, PhD⁷^*

¹INSTITUTE OF CHILD HEALTH, UCL, London, United Kingdom
²Bone Marrow Transplantation Department, Great Ormond Street Hospital, London, United Kingdom
³Department of Haematology, Great Ormond Street Hospital For Children, London, United Kingdom
⁴Institute of Child Health / Molecular and Cellular Immunology Unit, University College London, London, United Kingdom
⁵GREAT ORMOND STREET HOSPITAL, London, United Kingdom
⁶CANCER INSTITUTE, UCL, London, United Kingdom
⁷Cancer Institute, University College London, London, United Kingdom

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^*signifies non-member of ASH

801.P1.44 801. Gene Therapy and Transfer: Poster I