Journal article Open Access

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

Blaese, R. Michael; Culver, Kenneth W.; Miller, A. Dusty; Shearer, G.; Carter, Charles S.; Clerici, M.; Tolstoshev, P.; Fleisher, Thomas; Chang, L.; Rosenberg, S. A.; Chiang, Y.; Mullen, C. A.; Greenblatt, J. J.; Ramsey, W. J.; Klein, H.; Muul, L.; Berger, M.; Morgan, R. A.; Anderson, W. F.

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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