Gene Transfer Vectors for Clinical Application

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