Gene Transfer Vectors for Clinical Application book cover

Gene Transfer Vectors for Clinical Application

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.

Biochemists, biophysicists, molecular biologists, analytical chemists, and physiologists

Included in series
Methods in Enzymology

Hardbound, 440 Pages

Published: March 2012

Imprint: Academic Press

ISBN: 978-0-12-386509-0


  • Preface: The successful clinical use of viral vectors for human gene therapy.
    Theodore Friedmann
    1. General principles of retrovirus vector design
      Tammy Chang And Jiing-Kuan Yee

    2. Strategies for retrovirus-based correction of severe, combined immunodeficiency (SCID)
      Alain Fischer, Salima Hacein-Bey and Marina Cavazzana-Calvo

    3. Retrovirus and lentivirus vector design and methods of cell conditioning
      Samantha Cooray, Stephen J Howe and Adrian J Thrasher

    4. Analysis of the clonal repertoire of gene corrected cells in gene therapy
      Anna Paruzynski, Hanno Glimm, Manfred Schmidt and Christof von Kalle

    5. Developing novel lentiviral vectors into clinical products
      Anna Leath and Kenneth Cornetta

    6. Lentivirus vectors in beta-thalassemia
      Emmanuel Payen, Charlotte Colomb, Olivier Negre, Yves Beuzard, Kathleen Hehir and Philippe Leboulch

    7. Gene Therapy for Chronic Granulomatous Disease
      Elizabeth M. Kang, and Harry L. Malech

    8. Alternative splicing caused by lentiviral integration in the human genome
      Arianna Moiani and Fulvio Mavilio

    9. Genotoxicity assay for gene therapy vectors in tumor prone Cdkn2a-/- mice
      Eugenio Montini and Daniela Cesana

    10. 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

    11. Retroviral replicating vectors in cancer
      Christopher R. Logg , Joan M. Robbins , Douglas J. Jolly , Harry E. Gruber , and Noriyuki Kasahara

    12. Adeno-associated virus vectorology, manufacturing and clinical applications
      Joshua C. Grieger and R. Jude Samulski

    13. Gene Delivery To The Retina: From Mouse To Man
      Jean Bennett, Daniel C. Chung, and Albert Maguire

    14. Generation of hairpin-based RNAi vectors for biological and therapeutic application
      Ryan L. Boudreau and Beverly L. Davidson

    15. Recombinant adeno-associated viral vector reference standards
      Philippe Moullier and Richard O. Snyder

    16. 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

    17. Regulatory structures for gene therapy medicinal products in the European Union
      Bettina Klug, Patrick Celis, Melanie Carr and Jens Reinhardt


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