Article

CRISPR-Cas9 RAG2 Correction via Coding Sequence Replacement to Preserve Endogenous Gene Regulation and Locus Structure

2023-02-17

Abstract excerpt

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

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Literature Corpus work: 02a6e70a-8538-5767-96aa-3b75d87d437d
DOI: 10.21203/rs.3.rs-2565742/v1

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