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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| Autori principali: | , , , , , , , , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
Nature Portfolio
2023-10-01
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| Serie: | Nature Communications |
| Accesso online: | https://doi.org/10.1038/s41467-023-42036-5 |
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