An in vivo model of somatic cell gene therapy for human severe combined immunodeficiency. |
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Authors: | G Ferrari S Rossini R Giavazzi D Maggioni N Nobili M Soldati G Ungers F Mavilio E Gilboa C Bordignon |
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Institution: | Laboratory of Hematology, Istituto Scientifico H.S. Raffaele, Milano, Italy. |
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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. |
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