An in Vivo Model of Somatic Cell Gene Therapy for Human Severe Combined Immunodeficiency
- 15 March 1991
- journal article
- Published by American Association for the Advancement of Science (AAAS) in Science
- Vol. 251 (4999), 1363-1366
- https://doi.org/10.1126/science.1848369
Abstract
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency (SCID), a candidate genetic disorder for somatic cell gene therapy. Peripheral blood lymphocytes from patients affected by ADA- SCID were transduced with a retroviral vector for human ADA and injected into immunodeficient mice. Long-term survival of vector-transduced human cells was demonstrated in recipient animals. Expression of vector-derived ADA restored immune functions, as indicated by the presence in reconstituted animals of human immunoglobulin and antigen-specific T cells. Retroviral vector gene transfer, therefore, is necessary and sufficient for development of specific immune functions in vivo and has therapeutic potential to correct this lethal immunodeficiency.Keywords
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