476. In Vivo Repopulation of Syngenic Hematopoietic Cells Expressing GFP Transfected by Recombinant Sendai Virus–Toward Development of ‘Cytoplasmic Gene Therapy’ for Hematopoietic Disorders

Gene therapy targeting hematopoietic cells is a hopeful strategy to treat inherited hematopoietic disorders. Recent success in clinical gene therapy for X-linked SCID using a retoroviral vector expressing common gamma-chain encouraged physicians, patients and scientists; however serious adverse event, namely T-cell leukemia, related to the insertion of pro-viral genome into the host chromosome mediated by retroviral vector is a hazard to development the gene therapy strategies in this field. We have developed a novel RNA viral vector for efficient gene transfer, namely recombinant Sendai virus (SeV).