T Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years
Open Access
- 20 October 1995
- journal article
- clinical trial
- Published by American Association for the Advancement of Science (AAAS) in Science
- Vol. 270 (5235) , 475-480
- https://doi.org/10.1126/science.270.5235.475
Abstract
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 (ADA − 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.Keywords
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