Gene Transfer Vectors for Clinical Application

Other | December 31, 2012

byTheodore C. Friedman, Theodore C. Friedman

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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.



  • 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

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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 ...

Format:OtherDimensions:440 pages, 1 × 1 × 1 inPublished:December 31, 2012Publisher:Academic PressLanguage:English

The following ISBNs are associated with this title:

ISBN - 10:0123865107

ISBN - 13:9780123865106

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Table of Contents

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

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