Progress towards hematopoietic stem cell gene therapy.
The introduction of recombinant genetic material into human cells for therapeutic purposes offers tremendous potential. However, almost from the beginning, the application of gene therapy has been characterized by the striking discrepancy between its promise and realization. Over the past 15 years, much has been learned about the various gene transfer systems and the requirements for efficient hematopoietic stem cell gene transfer. In the current review, we will summarize recent improvements in hematopoietic stem cell gene transfer, describe some of the promising results from recent clinical applications and the impediments that remain.
Fred Hutchinson Cancer Research Center, University of Washington, Seattle, WA 98109-1024, USA.
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D000818:Animals; D001709:Biotechnology; D016207:Cytokines / Q000008:administration & dosage; D005199:Fanconi Anemia / Q000628:therapy; D005776:Gaucher Disease / Q000628:therapy; D015870:Gene Expression; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006105:Granulomatous Disease, Chronic / Q000628:therapy; D015658:HIV Infections / Q000628:therapy; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006453:Hemoglobinopathies / Q000628:therapy; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D009369:Neoplasms / Q000628:therapy; D012190:Retroviridae / Q000235:genetics; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy; D016092:Spumavirus / Q000235:genetics
D016207:Cytokines