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Gene correction for SCID-X1 in long-term hematopoietic stem cells

9 Apr 2019

Abstract excerpt

Abstract Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an effective therapy for monogenic diseases of the blood and immune system. Here we describe an approach for X-linked sSevere cCombined iImmunodeficiency (SCID-X1) using targeted integration of a cDNA into the endogenous start codon to functionally correct disease-causing mutations throughout the gene. Using a CRISPR-Cas9/AAV6...

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PMID: 30967552
DOI: 10.1038/s41467-019-09614-y

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