Gene Transfer Vectors for Clinical Application

Gene Transfer Vectors for Clinical Application

1st Edition - March 6, 2012

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  • Editor: Theodore Friedmann
  • eBook ISBN: 9780123865106
  • Hardcover ISBN: 9780123865090

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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.

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


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

Table of Contents

  • Preface: The successful clinical use of viral vectors for human gene therapy.

    Theodore Friedmann

    1. General principles of retrovirus vector design
    2. Tammy Chang And Jiing-Kuan Yee

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

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

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

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

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

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

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

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

    19. Lentiviral Hematopoietic Cell Gene Therapy for X-linked Adrenoleukodystrophy
    20. Nathalie Cartier, Salima Hacein-Bey-Abina , Cynthia C. Bartholomae, Pierre Bougnères , Manfred Schmidt, Christof Von Kalle, Alain Fischer, Marina Cavazzana-Calvo and Patrick Aubourg

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

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

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

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

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

    31. 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
    32. Marina O’Reilly, Allan Shipp, Eugene Rosenthal, Robert Jambou, Tom Shih, Maureen Montgomery, Linda Gargiulo, Amy Patterson and Jacqueline Corrigan-Curay

    33. Regulatory structures for gene therapy medicinal products in the European Union

    Bettina Klug, Patrick Celis, Melanie Carr and Jens Reinhardt

Product details

  • No. of pages: 440
  • Language: English
  • Copyright: © Academic Press 2012
  • Published: March 6, 2012
  • Imprint: Academic Press
  • eBook ISBN: 9780123865106
  • Hardcover ISBN: 9780123865090

About the Serial Volume Editor

Theodore Friedmann

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