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Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs

9 Dec 2022

Abstract excerpt

Severe combined immunodeficiency (SCID) is a group of disorders caused by mutations in genes involved in the process of lymphocyte maturation and function. CRISPR-Cas9 gene editing of the patient's own hematopoietic stem and progenitor cells (HSPCs) ex vivo could provide a therapeutic alternative to allogeneic hematopoietic stem cell transplantation, the current gold standard for treatment of SCID. To eliminate...

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PMID: 36618262
DOI: 10.1016/j.omtn.2022.12.006

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