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Article

Targeted genome editing restores T cell differentiation in a humanized X-SCID pluripotent stem cell disease model.

Scientific reports - 29 Sept 2017

Alzubi Jamal, Pallant Celeste, Mussolino Claudio, Howe Steven J, Thrasher Adrian J, Cathomen Toni

Abstract excerpt

The generation of T cells from pluripotent stem cells (PSCs) is attractive for investigating T cell development and validating genome editing strategies in vitro. X-linked severe combined immunodeficiency (X-SCID) is an immune disorder caused by mutations in the IL2RG gene and characterised by the absence of T and NK cells in patients. IL2RG encodes the common gamma chain, which is part of several interleukin...

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PMID: 28963568
DOI: 10.1038/s41598-017-12750-4
PMCID: PMC5622068

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