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

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/r...

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Autors principals:	Daniel Allen, Orli Knop, Bryan Itkowitz, Nechama Kalter, Michael Rosenberg, Ortal Iancu, Katia Beider, Yu Nee Lee, Arnon Nagler, Raz Somech, Ayal Hendel
Format:	Artigo
Idioma:	Inglês
Publicat:	Nature Portfolio 2023-10-01
Col·lecció:	Nature Communications
Accés en línia:	https://doi.org/10.1038/s41467-023-42036-5
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