Base editing of the HBG promoter induces potent fetal hemoglobin expression with no detectable off-target mutations in human HSCs.
Han W, Qiu HY, Sun S, Fu ZC, Wang GQ, Qian X, Wang L, Zhai X, Wei J, Wang Y, Guo YL, Cao GH, Ji RJ, Zhang YZ, Ma H, Wang H, Zhao M, Wu J, Bi L, Chen QB, Li Z, Yu L, Mou X, Yin H, Yang L, Chen J, Yang B, Zhang Y.
Han W, et al. Among authors: zhang yz, zhang y.
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
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