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Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome

23 Feb 2016

Abstract excerpt

Loss of CD40 ligand (CD40L) expression or function results in X-linked hyper-immunoglobulin (Ig)M syndrome (X-HIGM), characterized by recurrent infections due to impaired immunoglobulin class-switching and somatic hypermutation. Previous attempts using retroviral gene transfer to correct murine CD40L expression restored immune function; however, treated mice developed lymphoproliferative disease, likely due to...

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PMID: 26903548
DOI: 10.1182/blood-2015-11-683235

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