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

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

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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.

Original languageEnglish (US)
Pages (from-to)1363-1366
Number of pages4
JournalScience
Volume251
Issue number4999
DOIs
StatePublished - Jan 1 1991
Externally publishedYes

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Cite this

Ferrari, G., Rossini, S., Giavazzi, R., Maggioni, D., Nobili, N., Soldati, M., Ungers, G., Mavilio, F., Gilboa, E., & Bordignon, C. (1991). An in vivo model of somatic cell gene therapy for human severe combined immunodeficiency. Science, 251(4999), 1363-1366. https://doi.org/10.1126/science.1848369