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Science 15 March 1991:
Vol. 251. no. 4999, pp. 1363 - 1366
DOI: 10.1126/science.1848369
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Articles

Science, Vol 251, Issue 4999, 1363-1366
Copyright © 1991 by American Association for the Advancement of Science

articles

An in vivo model of somatic cell gene therapy for human severe combined immunodeficiency

G Ferrari, S Rossini, R Giavazzi, D Maggioni, N Nobili, M Soldati, G Ungers, F Mavilio, E Gilboa, and C Bordignon

Laboratory of Hematology, Istituto Scientifico H.S. Raffaele, Milano, Italy.

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.


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