Gene Transfer Vectors for Clinical Application: Volume 507

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.

Zusammenfassung
A resource for academics, researchers and students alike. It covers topics such as general principles of retrovirus vector design, chronic granulomatous disease (CGD), gene therapy for blindness, and retrovirus genetic strategy and vector design.

Inhalt

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