Base editing of the HBG promoter induces potent fetal hemoglobin expression with no detectable off-target mutations in human HSCs.
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Cell Stem Cell. 2023 Dec 7;30(12):1624-1639.e8. doi: 10.1016/j.stem.2023.10.007. Epub 2023 Nov 20.
Cell Stem Cell. 2023.
PMID: 37989316