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Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

20 Apr 2016

Abstract excerpt

X-linked severe combined immunodeficiency (SCID-X1) is a profound deficiency of T, B, and natural killer (NK) cell immunity caused by mutations inIL2RGencoding the common chain (γc) of several interleukin receptors. Gamma-retroviral (γRV) gene therapy of SCID-X1 infants without conditioning restores T cell immunity without B or NK cell correction, but similar treatment fails in older SCID-X1 children. We used a...

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PMID: 27099176
DOI: 10.1126/scitranslmed.aad8856

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