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

RM Blaese, KW Culver, AD Miller, CS Carter, T Fleisher… - Science, 1995 - science.org
RM Blaese, KW Culver, AD Miller, CS Carter, T Fleisher, M Clerici, G Shearer, L Chang…
Science, 1995science.org
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 …
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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