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Clinical Trial
. 1995 Oct 20;270(5235):475-80.
doi: 10.1126/science.270.5235.475.

T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years

R M Blaese  1 , K W Culver, A D Miller, C S Carter, T Fleisher, M Clerici, G Shearer, L Chang, Y Chiang, P Tolstoshev, J J Greenblatt, S A Rosenberg, H Klein, M Berger, C A Mullen, W J Ramsey, L Muul, R A Morgan, W F Anderson
Affiliations
Clinical Trial

T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years

R M Blaese et al. Science. .

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.

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