Clinical Trial
doi: 10.1126/science.270.5235.475.
T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years
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- PMID: 7570001
- DOI: 10.1126/science.270.5235.475
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Clinical Trial
T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years
Science.
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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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