A Comeback for Gene Therapy

Science  06 Nov 2009:
Vol. 326, Issue 5954, pp. 805-806
DOI: 10.1126/science.1181937

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Gene therapy has recently had some important successes in treating severe inherited diseases (13) after years of skepticism from the scientific community and neglect by the pharmaceutical industry. On page 818 in this issue, Cartier et al. (4) report another major advance—the successful first clinical testing of an HIV-derived vector in hematopoietic stem cell (HSC)–based gene therapy. The procedure was used to treat a severe neurodegenerative disease, X-linked adrenoleukodystrophy (ALD), and the results indicate stable expression of a therapeutic gene in a substantial fraction of patients' hematopoietic cells, as well as clinical benefits.