Journal
BONE MARROW TRANSPLANTATION
Volume 41, Issue 2, Pages 199-205Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/sj.bmt.1705895
Keywords
gene therapy; hematopoietic stem cell; immunodeficiency
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Gene therapy using autologous hematopoietic stem cells (HSC) that are corrected with the normal gene may have a beneficial effect on blood cell production or function, without the immunologic complications of allogeneic HSC transplantation. Childhood immunological diseases are highly favorable candidates for responses to gene therapy using HSC. Hemoglobinopathies, lysosomal and metabolic disorders and defects of hematopoietic stem and progenitor cells should also be ameliorated by gene therapy using autologous HSC. At present, gene therapy has been beneficial for patients with XSCID, ADA-deficient SCID and chronic granulomatous disease. The principle that partial marrow conditioning increases engraftment of gene-corrected HSC has been demonstrated. Clinical trials are being developed in Europe and the United States to treat several other genetic blood cell disorders. This progress is tempered by the serious complication observed in XSCID patients developing T lymphoproliferative disease. New methods for gene transfer (lentiviral and foamy viral vectors, semi-viral systems and gene correction) may retain or further increase the efficacy and decrease the risks from gene therapy using HSC. Ultimately, the relative benefits and risks of autologous gene therapy will be weighed against other available options (for example, allogeneic HSCT) to determine the treatment of choice.
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