T Lymphocyte-Directed Gene Therapy for ADAminus SCID: Initial Trial Results After 4 Years.
Blaese, R. Michael *; Culver, Kenneth W.; Miller, A. Dusty; Carter, Charles S.; Fleisher, Thomas; Clerici, Mario **; Shearer, Gene; Chang, Lauren; Chiang, Yawen; Tolstoshev, Paul; Greenblatt, Jay J.; Rosenberg, Steven A.; Klein, Harvey; Berger, Melvin; Mullen, Craig A. ***; Ramsey, W. Jay; Muul, Linda; Morgan, Richard A.; Anderson, W. French sign ****
[Report] Science. 270(5235):475-480, October 20, 1995.

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In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children with severe combined immunodeficiency (ADAminus SCID). The number of blood T cells normalized as did many cellular and humoral immune responses. Gene treatment ended after 2 years, but integrated vector and ADA gene expression in T cells persisted. Although many components remain to be perfected, it is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease.

Copyright (C) 1995 by the American Association for the Advancement of Science