Gene therapy using hematopoietic stem cells: Sisyphus approaches the crest.
Gene transfer targeting cells of the blood and immune system was one of the first areas of investigation in the field of gene therapy. Despite the encouraging results achieved in early studies using murine bone marrow, the task of gene transfer into human hematopoietic stem cells proved to be far more difficult. As a result, progress has been disappointingly slow and initial clinical trials generally failed to achieve significant levels of gene marking. The continued application of new advances in vectorology and hematopoietic stem cell biology has now led to improvements in preclinical models that are being translated into clinical trials. The progress and remaining problems are discussed in this review article.
Halene
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Department of Pediatrics, Childrens Hospital Los Angeles, University of Southern California School of Medicine, CA 90027, USA.
Kohn
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D000256:Adenoviridae / Q000235:genetics; D000818:Animals; D018952:Antigens, CD34 / Q000032:analysis; D002986:Clinical Trials as Topic; D015870:Gene Expression; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000235:genetics; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000378:metabolism; D006801:Humans; D007108:Immune Tolerance; D051379:Mice; D012190:Retroviridae / Q000235:genetics; D014161:Transduction, Genetic
D018952:Antigens, CD34