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