Article
Base editing of the HBG promoter induces potent fetal hemoglobin expression with no detectable off-target mutations in human HSCs.
Cell stem cell - 7 Dec 2023
Han Wenyan, Qiu Hou-Yuan, Sun Shangwu, Fu Zhi-Can, Wang Guo-Quan, Qian Xiaowen, Wang Lijie, Zhai Xiaowen, Wei Jia, Wang Yichuan, Guo Yi-Lin, Cao Guo-Hua, Ji Rui-Jin, Zhang Yi-Zhou, Ma Hongxia, Wang Hongsheng, Zhao Mingli, Wu Jing, Bi Lili, Chen Qiu-Bing, Li Zifeng, Yu Ling, Mou Xiaodun, Yin Hao, Yang Li, Chen Jia, Yang Bei, Zhang Ying
Abstract excerpt
Reactivating silenced γ-globin expression through the disruption of repressive regulatory domains offers a therapeutic strategy for treating β-hemoglobinopathies. Here, we used transformer base editor (tBE), a recently developed cytosine base editor with no detectable off-target mutations, to disrupt transcription-factor-binding motifs in hematopoietic stem cells. By performing functional screening of six motifs...
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