Gene Transfer Vectors for Clinical Application

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