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

20 Oct 1995

Abstract excerpt

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...

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Identifiers

PMID: 7570001
DOI: 10.1126/science.270.5235.475

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Source receipt: pubmed, retrieved 7 Sept 2026.

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T Lymphocyte-Directed Gene Therapy for ADA <sup>−</sup> SCID: Initial Trial Results After 4 YearsPMID 7570001Read ↗

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