Key Features
- Chapters provide an invaluable resource for academics, researchers and students alike
- International board of authors
- This volume covers such topics as general principles of retrovirus vector design, chronic granulomatous disease (CGD), gene therapy for blindness, and retrovirus genetic strategy and vector design
Description
This volume of Methods in Enzymology looks at Gene Transfer Vectors for Clinical Application. The chapters provide an invaluable resource for academics, researchers and students alike. With an international board of authors, this volume covers such topics as General principles of retrovirus vector design, Chronic granulomatous disease (CGD), Gene therapy for blindness, and Retrovirus genetic strategy and vector design.
Gene Transfer Vectors for Clinical Application, 1st Edition
Preface: The successful clinical use of viral vectors for human gene therapy.
Theodore Friedmann
- General principles of retrovirus vector design
Tammy Chang And Jiing-Kuan Yee
- Strategies for retrovirus-based correction of severe, combined immunodeficiency (SCID)
Alain Fischer, Salima Hacein-Bey and Marina Cavazzana-Calvo
- Retrovirus and lentivirus vector design and methods of cell conditioning
Samantha Cooray, Stephen J Howe and Adrian J Thrasher
- Analysis of the clonal repertoire of gene corrected cells in gene therapy
Anna Paruzynski, Hanno Glimm, Manfred Schmidt and Christof von Kalle
- Developing novel lentiviral vectors into clinical products
Anna Leath and Kenneth Cornetta
- Lentivirus vectors in beta-thalassemia
Emmanuel Payen, Charlotte Colomb, Olivier Negre, Yves Beuzard, Kathleen Hehir and Philippe Leboulch
- Gene Therapy for Chronic Granulomatous Disease
Elizabeth M. Kang, and Harry L. Malech
- Alternative splicing caused by lentiviral integration in the human genome
Arianna Moiani and Fulvio Mavilio
- Genotoxicity assay for gene therapy vectors in tumor prone Cdkn2a-/- mice
Eugenio Montini and Daniela Cesana
- Lentiviral Hematopoietic Cell Gene Therapy for X-linked Adrenoleukodystrophy
Nathalie Cartier, Salima Hacein-Bey-Abina , Cynthia C. Bartholomae, Pierre Bougnères , Manfred Schmidt, Christof Von Kalle, Alain Fischer, Marina Cavazzana-Calvo and Patrick Aubourg
- Retroviral replicating vectors in cancer
Christopher R. Logg , Joan M. Robbins , Douglas J. Jolly , Harry E. Gruber , and Noriyuki Kasahara
- Adeno-associated virus vectorology, manufacturing and clinical applications
Joshua C. Grieger and R. Jude Samulski
- Gene Delivery To The Retina: From Mouse To Man
Jean Bennett, Daniel C. Chung, and Albert Maguire
- Generation of hairpin-based RNAi vectors for biological and therapeutic application
Ryan L. Boudreau and Beverly L. Davidson
- Recombinant adeno-associated viral vector reference standards
Philippe Moullier and Richard O. Snyder
- NIH oversight of human gene transfer research involving retroviral, lentiviral and adeno-associated virus vectors and the role of the NIH recombinant DNA advisory committee draft
Marina O’Reilly, Allan Shipp, Eugene Rosenthal, Robert Jambou, Tom Shih, Maureen Montgomery, Linda Gargiulo, Amy Patterson and Jacqueline Corrigan-Curay
- Regulatory structures for gene therapy medicinal products in the European Union
Bettina Klug, Patrick Celis, Melanie Carr and Jens Reinhardt
