T Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years

1995/10/20 by R. Michael Blaese, Kenneth W. Culver, A. Dusty Miller +21 · 27 citations
Biochemistry, Genetics and Molecular Biology · Medicine · #Virus-based gene therapy research #CAR-T cell therapy research #RNA Interference and Gene Delivery

paper · doi: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.

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