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.
T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years / R.M. Blaese, K.W. Culver, A.D. Miller, C.S. Carter, T. Fleisher, M. Clerici, G.M. Shearer, L. Chang, Y. Chiang, P. Tolstoshev, J.J. Greenblatt, S.A. Rosenberg, H. Klein, M. Berger, C.A. Mullen, W.J. Ramsey, L. Muul, R.A. Morgan, W.F. Anderson. - In: SCIENCE. - ISSN 0036-8075. - 270:5235(1995), pp. 475-480.
T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years
M. Clerici;
1995
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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