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An in Vivo Model of Somatic Cell Gene Therapy for Human Severe Combined Immunodeficiency

1991; American Association for the Advancement of Science; Volume: 251; Issue: 4999 Linguagem: Inglês

10.1126/science.1848369

1095-9203

Giuliana Ferrari, Silvano Rossini, Raffaella Giavazzi, Daniela Maggioni, Nadia Nobili, Monica Soldati, Grace E. Ungers, Fulvio Mavilio, Eli Gilboa, Claudio Bordignon,

Virus-based gene therapy research

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.