Gene therapy using hematopoietic stem cells.
While gene therapy using hematopoietic stem cells was the first area of investigation in the field, success has proven elusive. However, significant progress has been achieved recently in methods for more effective gene transfer and expression. In addition to greatly improved results using retroviral vectors, adeno-associated vectors and lentiviral vectors appear to be promising for stable transduction of hematopoietic stem cells. These advances, documented in animal transplant models, are now being applied to clinical trials.
Kohn
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Department of Pediatrics, University of Southern California School of Medicine, Children's Hospital, Los Angeles 90027, USA. dkohn@chla.usc.edu
D000256:Adenoviridae / Q000235:genetics / Q000276:immunology; D000818:Animals; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000494:pharmacology; D001855:Bone Marrow Diseases / Q000139:chemically induced / Q000235:genetics / Q000628:therapy; D002986:Clinical Trials as Topic; D004195:Disease Models, Animal; D004285:Dogs; D019008:Drug Resistance, Neoplasm / Q000235:genetics; D015967:Gene Expression Regulation, Viral; D030342:Genetic Diseases, Inborn / Q000235:genetics / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics / Q000276:immunology; D015658:HIV Infections / Q000628:therapy; D015497:HIV-1; D018380:Hematopoietic Stem Cell Transplantation; D006453:Hemoglobinopathies / Q000235:genetics / Q000628:therapy; D006801:Humans; D007223:Infant; D016086:Lentivirus / Q000235:genetics / Q000276:immunology; D051379:Mice; D016688:Mice, Inbred NOD; D018345:Mice, Knockout; D016513:Mice, SCID; D011323:Primates; D011828:Radiation Chimera; D011993:Recombinant Fusion Proteins / Q000276:immunology / Q000502:physiology; D012190:Retroviridae / Q000235:genetics / Q000276:immunology; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy; D014183:Transplantation, Heterologous; D014184:Transplantation, Homologous
D000970:Antineoplastic Agents; D011993:Recombinant Fusion Proteins