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CRISPR-Cas9 engineering of the RAG2 locus via complete coding sequence replacement for therapeutic applications

27 Oct 2023

Abstract excerpt

Abstract RAG2 -SCID is a primary immunodeficiency caused by mutations in Recombination-activating gene 2 (RAG2) , a gene intimately involved in the process of lymphocyte maturation and function. ex-vivo manipulation of a patient’s own hematopoietic stem and progenitor cells (HSPCs) using CRISPR-Cas9/rAAV6 gene editing could provide a therapeutic alternative to the only current treatment, allogeneic hematopoietic...

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PMID: 37891182
DOI: 10.1038/s41467-023-42036-5

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