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


Book information

  • Published: March 2012
  • ISBN: 978-0-12-386509-0

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