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

Tuesday, December 9, 2008: 7:00 AM-9:00 AM

304-306-308 - South (Moscone Center)

Moderators:

John Tisdale and Chiara Bonini

7:00 AM

The 3’ End of the Chicken Hypersensitive Site-4 Insulator Has Properties Similar to the 5’ Insulator Core and Is Necessary in Conjunction with the Core for Full Insulator Activity

Paritha Arumugam¹^*, Fabrizia Urbinati, Phd.²^*, Chinavenmeni S. Velu, PhD³, H. Leighton Grimes, PhD⁴ and Punam Malik, MD⁵

¹Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
²Experimental Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati
³Immunobiology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁴Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁵Div. of Experimental Hematology and Cancer Biology, Dept of Pediatrics, Cincinnati Childrens Hospital Medical Center, Cincinnati, OH

7:15 AM

818

Evaluation of a γ-Globin Lentiviral Vector in Sickle Cell Mice and Pigtail Macaques

Phillip W Hargrove, M.S.¹^*, Tamara I Pestina, Ph.D.¹^*, Yoon Sang Kim, M.D., Ph.D.¹^*, John Gray, Ph.D.¹^*, Kelli Boyd, D.V.M., Ph.D.²^* and Derek Persons, MD, PhD¹

¹Hematology, St. Jude Children's Research Hospital, Memphis, TN
²Veterinary Pathology Core, St. Jude Children's Research Hospital, Memphis, TN

7:30 AM

819

Cell-Intrinsic Heterogeneity Plays An Important Role in the Development of Clonal Dominance after Retroviral Gene Transfer into Hematopoietic Stem Cells

Olga S. Kustikova^*, Bernhard Schiedlmeier^*, Martijn H. Brugman^*, Maike Stahlhut^*, Zhixiong Li and Christopher Baum

Experimental Hematology, Hannover Medical School, Hannover, Germany

7:45 AM

820

Transcription Factor Binding Sites Are Genetic Determinant of Retroviral Integration in the Human Genome

Claudia Cattoglio¹^*, Barbara Felice²^*, Davide Cittaro²^*, Annarita Miccio³^*, Giuliana Ferrari¹^*, Alessandra Recchia⁴^*, Lucilla Luzi²^* and Fulvio Mavilio⁴

¹Istituto Scientifico H. San Raffaele, Milan, Italy
²FIRC Institute of Molecular Oncology, Milan, Italy
³Abramson Research Center, Children's Hosp. of Philadelphia, Philadelphia, PA
⁴Department of Biomedical Sciences, University of Modena and Reggio Emilia, Modena, Italy

8:00 AM

821

Hematopoietic Stem Cell Gene Therapy Trial with Lentiviral Vector in X-Linked Adrenoleukodystrophy

Marina Cavazzana-Calvo, MD, PhD¹, Nathalie Cartier²^*, Salima Hacein-Bey Abina¹^*, Gabor Veres³^*, Manfred Schmidt⁴^*, Cynthia Bartholomae⁴^*, Michel Vidaud⁵^*, Liliane Dal-Cortivo¹^*, Laure Caccavelli¹^*, Nizar Malhaoui⁶^*, Pierre Frange⁶^*, Véronique Kiermer⁷^*, Denice Mittelstaedt⁷^*, Andrew Simmons⁷^*, Céline Bellesme²^*, François Lefrère¹^*, Chantale Lagresle¹^*, Stephane Blanche⁶^*, Pierre Charneau⁸^*, Muriel Audit⁹^*, Bruno L'Homme⁵^*, Jing Chao Zhao-Emonet⁵^*, Serge Fichelson¹⁰^*, Françoise Pflumio¹⁰^*, Anne Dubart-Kupperschmitt¹⁰^*, Rachel Salzman¹¹^*, Amber Salzman¹¹^*, Pierre Bougnères²^*, Christof Von Kalle⁴^*, Alain Fischer, MD, PhD¹²^* and Patrick Aubourg²^*

¹Biothérapie, Hopital Necker - Enfants Malades, Paris, France
²Endocrinologie et Neurologie Pediatrique, AP-HP Hôpital Saint Vincent de Paul, Paris, France
³Applied Genetic Technologies, Corporation, Alachua, FL
⁴Translational Oncology, National Center for Tumor Diseases (NCT) / German Cancer Research Center (DKFZ), Heidelberg, Germany
⁵Faculté des Sciences Pharmaceutiques et Biologiques, UMR745 Inserm, Université Paris Descartes,, Paris, France
⁶Unité d'Immunologie et Hématologie Pédiatrique, AP-HP Hôpital Necker - Enfants Malades, Paris, France
⁷Cell Genesys, Inc., San Francisco, CA
⁸CNRS-URA3015, Institut Pasteur, Paris, France
⁹Généthon, Paris, France
¹⁰departement d'Hématologie - INSERM U567, Institut Cochin, Paris, France
¹¹The StopALD Foundation, Houston, TX
¹²Service d'Immunologie et Hematologie Pediatrique, INSERM, Université Paris Descartes, IFR94, AP-HP Hopital Necker Enfants-Malades, Paris, France

8:15 AM

822

Immunosuppression Modulates Immune Responses to AAV Capsid in Human Subjects Undergoing Intramuscular Gene Transfer for Lipoprotein Lipase Deficiency

Daniel J Hui, Ph.D.¹^*, Federico Mingozzi, Ph.D.¹^*, Annemarie Kleefstra²^*, Janneke M Meulenberg, Ph.D.²^*, Shyrie Edmonson³^*, Denise Morin⁴^*, Daniel Gaudet, M.D., Ph.D.⁵^*, Sander van Deventer, M.D., Ph.D.²^* and Katherine A. High, M.D.³

¹Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA
²Amsterdam Molecular Therapeutics, Amsterdam, Netherlands
³Howard Hughes Medical Institute, The Children's Hospital of Philadelphia, Philadelphia, PA
⁴University of Montreal, Montreal, QC, Canada
⁵Chicoutimi University Hospital, Chicoutimi, QC, Canada

8:30 AM

823

Recombination of Endogenous TCR Chains with Retrovirally Introduced TCR Chains Can Result in Mixed T Cell Receptor Dimers Harbouring Harmful Alloreactivity

Marleen M van Loenen, MSc^*, Renate de Boer, BSc^*, Gerdien L Volbeda, BSc^*, Avital L Amir, MD^*, Renate S. Hagedoorn, MSc^*, Roelof Willemze, MD, PhD, J.H. Frederik Falkenburg, MD, PhD and Mirjam H.M. Heemskerk, PhD

Dept. of Hematology, Leiden University Medical Center, Leiden, Netherlands

8:45 AM

824

Engineering CD19-Redirected T Lymphocytes to Enhance Their Safety and Efficacy

Valentina Hoyos, MD¹^*, Barbara Savoldo, MD¹^*, Juan F Vera, MD¹^*, Concetta Quintarelli, PhD¹^*, Cliona M Rooney, PhD²^*, Helen E. Heslop, MD³, Malcolm K Brenner, MD, PhD³^* and Gianpietro Dotti, MD⁴^*

¹Pediatrics, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX
²Pediatrics, Molecular Virology and Microbiology, Immunology, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX
³Pediatrics, Medicine, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX
⁴Medicine, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX

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^*signifies non-member of ASH

801.O1.8 Gene Therapy and Transfer