Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice.
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Nat Biomed Eng. 2023 May;7(5):616-628. doi: 10.1038/s41551-023-01026-0. Epub 2023 Apr 17.
Nat Biomed Eng. 2023.
PMID: 37069266
Free PMC article.