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Science 20 October 1995:
Vol. 270. no. 5235, p. 357
DOI:

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A form of severe combined immune deficiency (SCID) results from defects in the gene for adenosine deaminase (ADA); deoxyadenosine then accumulates and is toxic to T cells. Bone marrow transplants can cure this disease but donors are usually not available; some symptomatic relief can be afforded by the use of enzyme replacements, such as ADA conjugated to polyethylene glycol (PEG-ADA). Two reports discuss the results of separate clinical trials in which the ADA gene has been transferred with retroviral vectors to two children with SCID during 2-year treatment periods. Blaese et al. (p. 475) targeted T cells and showed that vector integration and ADA expression persisted 2 years after gene treatment ended. Bordignon et al. (p. 470) used two different vectors to target peripheral blood lymphocytes and bone marrow cells. T cells derived from the marrow gradually dominated the population after treatment stopped. In each trial PEG-ADA was also given, but both reports conclude that gene therapy has contributed to therapeutic improvements in SCID.

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