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X-linked severe combined immunodeficiency (SCID-X1) is a recessive hereditary disorder in which early T and Natural Killer (NK) lymphocyte development is blocked. The genetic disorder results from mutations in the common gamma c chain that participates in several cytokine receptors including the interleukin-2 (Il-2), Il-4, Il-7, Il-9, Il-15 receptors. SCID-X1 offers a reliable model for gene therapy as it is a lethal condition that is, in many cases, curable by allogeneic bone marrow transplantation. We have shown that retrovirus-mediated transfer of the gamma c cDNA induced gamma c chain expression and restored the function of the high-affinity IL-2 receptor on SCI-X1 EBV-transformed B-cell lines. We have the designed culture conditions to study NK-cell and T-cell development of CD34+ hematopoietic progenitor cells. In the culture systems, gamma c transduced CD34+ marrow cells from two SCID-X1 patients were able to mature into CD56+ and/or CD16+ NK cells and into CD4+ TCR alpha beta+ T cells. These preclinical results set the basis for a clinical study of ex-vivo gamma c gene transfer into CD34+ cells from SCID-X1 patients.
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F Gross, and
P Nusbaum, and
E Yvon, and
A Fischer, and
M Cavazzana-Calvo
May 2005,
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S Hacein-Bey, and
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M Cavazzana-Calvo
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S Hacein-Bey, and
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E Yvon, and
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M Cavazzana-Calvo
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S Hacein-Bey, and
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June 2015,
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S Hacein-Bey, and
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P Nusbaum, and
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A Fischer, and
M Cavazzana-Calvo
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S Hacein-Bey, and
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P Nusbaum, and
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S Hacein-Bey, and
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P Nusbaum, and
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Human gene therapy,
