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Article

SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease

6 Dec 2012

Abstract excerpt

X-linked lymphoproliferative disease (XLP1) arises from mutations in the gene encoding SLAM-associated protein (SAP) and leads to abnormalities of NKT-cell development, NK-cell cytotoxicity, and T-dependent humoral function. Curative treatment is limited to allogeneic hematopoietic stem cell (HSC) transplantation. We tested whether HSC gene therapy could correct the multilineage defects seen in SAP(-/-) mice....

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Identifiers

PMID: 23223356
DOI: 10.1182/blood-2012-07-445858

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