T Lymphocyte-Directed Gene Therapy for ADA <sup>−</sup> SCID: Initial Trial Results After 4 Years
R. Michael Blaese(National Institutes of Health), Kenneth W. Culver(National Institutes of Health), A. Dusty Miller(Cape Town HVTN Immunology Laboratory / Hutchinson Centre Research Institute of South Africa), Charles S. Carter(Carter Center), Thomas A. Fleisher(Carter Center), Mario Clerici(National Institutes of Health), Gene M. Shearer(National Institutes of Health), Lauren A. Chang(National Heart Lung and Blood Institute), Ya‐Wen Chiang(Research Institute for Genetic and Human Therapy), Paul Tolstoshev(Research Institute for Genetic and Human Therapy), Jay J. Greenblatt(National Institutes of Health), Steven A. Rosenberg(National Institutes of Health), Harvey G. Klein(Carter Center), Melvin Berger(Case Western Reserve University), Craig A. Mullen(National Institutes of Health), W. Jay Ramsey(National Institutes of Health), Linda Muul(National Human Genome Research Institute), Richard A. Morgan(National Human Genome Research Institute), W. French Anderson(National Heart Lung and Blood Institute)
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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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