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

  • Blaese R
  • Culver K
  • Miller A
 et al. 
  • 295

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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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Authors

  • R. M. Blaese

  • K. W. Culver

  • A. D. Miller

  • C. S. Carter

  • T. Fleisher

  • M. Clerici

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