50th ASH Annual Meeting and Exposition (December 6 - 9, 2008): Gene Therapy and Transfer Poster I

Sunday, December 7, 2008: 6:00 PM-8:00 PM

Hall A (Moscone Center)

First in Human Engraftment of Anti-HIV Lentiviral Vector Gene Modified CD34+ Peripheral Blood Progenitor Cells in the Treatment of AIDS Related Lymphoma (ARL)

Amrita Krishnan, MD, FACP¹, John A. Zaia, MD², John Rossi PhD¹^*, David DiGiusto PhD¹^*, Michael Kalos PhD¹^*, Larry Couture PhD¹^*, Joseph Alvarnas MD¹^*, Mingjie Li PhD¹^*, Haitang Li PhD¹^*, Jiing-Kuan Yee, PhD¹^*, Priscilla Yam FIBMS¹^*, Shirley Li MD¹^*, Shu Mi MD¹^*, Lijing Li MA¹^*, David Hsu PhD¹^*, Michelle Wardlow RN¹^*, Lupe Duarte CCRC¹^*, Anitha Rao MS¹^* and Stephen J. Forman, MD¹^*

¹City of Hope National Medical Center, Duarte, CA
²Division of Virology, City of Hope, Duarte, CA

2349

Retroviral Gene Therapy for X-Linked Chronic Granulomatous Disease: Results from Phase I/II Trial

Joong Gon Kim, MD, PhD¹^*, Hyo Seop Ahn, MD, PhD¹^*, Hyoung Jin Kang, MD, PhD¹, Sujeong Kim, PhD², Youngtae Hong, PhD²^*, Chang-Wan Joo, MS²^*, Nam-Kyung Yoon, MS²^* and Sunyoung Kim, DPhil²^*

¹Department of Pediatrics, Seoul National University College of Medicine, Seoul, South Korea
²ViroMed, Seoul, South Korea

2350

Long-Term Vector Integration Site Analysis Following Retroviral Mediated Gene Transfer to Hematopoietic Stem Cells for the Treatment of HIV Infection

Jun Hayakawa, MD, PhD¹, Matthew Hsieh, MD¹, Naoya Uchida, MD¹, Kareem Washington, PhD¹^*, Oswald Phang¹^*, Elizabeth Kang, MD²^* and John F. Tisdale, MD¹

¹MCHB, NHLBI, NIH, Bethesda, MD
²NIAID,NIH, Bethesda, MD

2351

Comprehensive and Unbiased Integration Site Analysis in Clinical Gene Therapy

Richard Gabriel¹^*, Anna Paruzynski¹^*, Ralph Eckenberg²^*, Cynthia C. Bartholomae¹^*, Ali Nowrouzi¹^*, Wei Wang, MSc¹^*, Kerstin Schwarzwaelder¹^*, Anne Arens³^*, Romy Kirsten¹^*, Annette Deichmann¹^*, Claudia Ball¹^*, Steven J. Howe⁴^*, Alessandra Recchia⁵^*, Claudia Cattoglio⁶^*, Kamaljit S. Balaggan⁷^*, Rafael J. Yáñez-Muñoz⁸^*, Robin R. Ali⁷^*, Fulvio Mavilio⁵, Bobby Gaspar⁴^*, Adrian Thrasher, MD, PhD⁴^*, Hanno Glimm, MD¹, Christof von Kalle¹^*, William Saurin²^* and Manfred Schmidt¹^*

¹Department of Translational Oncology, National Center for Tumor Diseases (NCT) and German Cancer Research Center (DKFZ), Heidelberg, Germany
²Genomining, Montrouge, France
³Genomics and Proteomics Core Facilities, German Cancer Research Center (DKFZ), Heidelberg, Germany
⁴Molecular Immunology Unit, Institute of Child Health, University College London, London, United Kingdom
⁵Department of Biomedical Sciences, University of Modena and Reggio Emilia, Milan, Italy
⁶Istituto Scientifico H. San Raffaele, Milan, Italy
⁷Institute of Ophthalmology, University College London
⁸School of Biomedical Sciences, Royal Holloway-University of London

2352

Evaluating the Safety and Efficacy of Anti-HIV Transgenic Cells in a Humanized Mouse Model (NOD/SCIDγ_c^–/–) for HIV Stem Cell Gene Therapy

Joseph S. Anderson, Ph.D.^*, Jon Walker^*, Louisa Wirthlin^*, John Javien^*, Jan A. Nolta, Ph.D. and Gerhard Bauer^*

Stem Cell Program, Department of Internal Medicine and Office of the Dean, UC Davis Health System, University of California, Davis, Sacramento, CA

2353

Development of An HIV1-Based Lentiviral Vector Able to Transduce Both Human and Rhesus Blood Cells

Naoya Uchida, MD¹, Kareem Washington, PhD¹^*, Jun Hayakawa, MD, PhD¹^*, Matthew Hsieh, MD¹, Aylin C. Bonifacino²^*, Allen E. Krouse²^*, Mark E. Metzger²^*, Robert E. Donahue, VMD² and John F. Tisdale, MD¹

¹MCHB, NHLBI, NIH, Bethesda, MD
²HB, NHLBI, NIH, Rockville, MD

2354

Correction of Sickle Cell Anemia with γ-Globin Gene Delivered by Lentivirus Vector in the Setting of Myeloablative or Reduced Intensity Conditioning, and Establishing Critical Determinants for Successful Gene Therapy for Sickle Cell Disease

Ajay Perumbeti, M.D.¹^*, Tomoyasu Higashimoto, Phd.²^*, Fabrizia Urbinati, Phd.²^*, Kristy Lauderback, B.S.²^*, Anastacia Loberg, B.S.²^*, Gabriel Estevez-Pagani²^*, Herbert Meiselman, M.D.³^*, Robert Franco, Phd.⁴^*, David Witte, M.D.⁵^*, Clinton H. Joiner, MD, PhD⁶ and Punam Malik⁷

¹Div. of Hematology Oncology and Div. of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
²Div. of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
³Department of Physiology and Biophysics, University of Southern California, Keck School of Medicine, Los Angeles, CA
⁴Department of Internal Medicine, University of Cincinnati College of Medicine, Cincinnati, CA
⁵Div. of Pathology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁶Div. of Experimental Hematology and Cancer Biology, Dept of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁷Div. of Experimental Hematology and Cancer Biology, Dept of Pediatrics, Cincinnati Childrens Hospital Medical Center, Cincinnati, OH

2355

High Level Production of a Lysosomal Enzyme from HSC-Derived Erythroid Cells for Therapeutic Correction of Hurler Syndrome in Mice

Daren Wang, PhD¹^*, Wei Zhang, MS¹^*, Theodosia A. Kalfa, MD, PhD², Punam Malik, MD¹^* and Dao Pan, PhD¹^*

¹Experimental Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
²Hematology/Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH

2356

Correction of Murine Adamts13 Deficiency by Hematopoietic Progenitor Cell-Mediated Gene Therapy

Pablo Laje^*, Dezhi Shang^*, Masayuki Endo^*, Wenjing Cao^*, Philip W. Zoltick^*, Alan W. Flake^* and X. Long Zheng

Departments of General Surgery, Pathology and Laboratory Medicine, Children's Hospital of Philadelphia and University of Pennsylvania, Philadelphia, PA

2357

Correction of Fanconi Anemia Group Α in Primary Human and Murine Hematopoietic Progenitors with a Clinical Lentiviral Vector

Jason Taylor, MD, PhD¹^*, Grant D Trobridge, PhD²^*, Martin Wohlfahrt²^*, Sylvia Chien³^*, Xin Zhao, MD³^*, Brian C Beard, PhD², Hans-Peter Kiem, MD⁴ and Pamela S. Becker, MD, PhD³

¹Hematology & Medical Oncology, Oregon Health & Science University, Portland, OR
²Clinical Research Div., Fred Hutchinson Cancer Research Ctr., Seattle, WA
³Division of Hematology, University of Washington, Seattle, WA
⁴Fred Hutchinson Cancer Rsch. Ctr., Seattle, WA

2358

Non-Random Lentiviral Vector Insertions in Bone Marrow Progenitors from Fanconi Anemia Patients

Ali Nowrouzi¹^*, Africa Gonzales-Murillo²^*, Anna Paruzynski¹^*, Ariana Jacome²^*, Paula Rio²^*, Susana Navarro²^*, José Carlos Segovia²^*, Nadine Krenzer¹^*, Christof von Kalle¹^*, Guillermo Guenechea²^*, Juan A. Bueren²^* and Manfred Schmidt¹^*

¹Department of Translational Oncology, National Center for Tumor Diseases (NCT) and German Cancer Research Center (DKFZ), Heidelberg, Germany
²Division of Hematopoiesis and Gene Therapy, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and CIBER-ER, Madrid, Spain

2359

Mechanism of Reduction in Titers from Lentivirus Vectors Carrying Chromatin Insulator Elements in the 3’ LTR

Fabrizia Urbinati^*, Paritha Arumugam^*, Tomoyasu Higashimoto^*, Anil Perumbeti^*, Ping Xia^* and Punam Malik

Div. of Experimental Hematology and Cancer Biology, Dept of Pediatrics, Cincinnati Childrens Hospital Medical Center, Cincinnati, OH

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^*signifies non-member of ASH

801.P1.34 Gene Therapy and Transfer Poster I