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