Gene transfer vectors for clinical application [electronic resource] / edited by Theodore Friedman.

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. Chapters provide an invaluable resource for academics, researchers and students alike. Iinternational 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.

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-Abina, and Marina Cavazzana-Calvo -- Retrovirus and lentivirus vector design and methods of cell conditioning / Samantha Cooray, Steven J. Howe, and Adrian J. Thrasher -- Analysis of the clonal repertoire of gene-corrected cells in gene therapy / Anna Paruzynski ... [et al.] -- Developing novel lentiviral vectors into clinical products / Anna Leath and Kenneth Cornetta -- Lentivirus vectors in [beta]-thalassemia / Emmanuel Payen ... [et al.] -- 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 Cdkn2amice / Eugenio Montini and Daniela Cesana -- Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy / Nathalie Cartier ... [et al.] -- Retroviral replicating vectors in cancer / Christopher R. Logg ... [et al.] -- 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 / Marina O'Reilly ... [et al.] -- Regulatory structures for gene therapy medicinal products in the European Union / Bettina Klug ... [et al.].

Preface: The successful clinical use of viral vectors for human gene therapy. General principles of retrovirus vector design Strategies for retrovirus-based correction of severe, combined immunodeficiency (SCID) Retrovirus and lentivirus vector design and methods of cell conditioning Analysis of the clonal repertoire of gene corrected cells in gene therapy Developing novel lentiviral vectors into clinical products Lentivirus vectors in beta-thalassemia Gene Therapy for Chronic Granulomatous Disease Alternative splicing caused by lentiviral integration in the human genome Genotoxicity assay for gene therapy vectors in tumor prone Cdkn2a-/- mice Lentiviral Hematopoietic Cell Gene Therapy for X-linked Adrenoleukodystrophy Retroviral replicating vectors in cancer Adeno-associated virus vectorology, manufacturing and clinical applications Gene Delivery To The Retina: From Mouse To Man Generation of hairpin-based RNAi vectors for biological and therapeutic application Recombinant adeno-associated viral vector reference standards 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 Regulatory structures for gene therapy medicinal products in the European Union.

Includes bibliographical references and indexes.

Description based on print version record.