Base editing of the HBG promoter induces potent fetal hemoglobin expression with no detectable off-target mutations in human HSCs
Wenyan Han, Hou-Yuan Qiu, Shangwu Sun, Zhi-Can Fu, Guoquan Wang, Xiaowen Qian, Lijie Wang, Xiaowen Zhai, Wei Jia, Yichuan Wang, Yilin Guo, Guohua Cao, Rui-Jin Ji, Yizhou Zhang, Yizhou Zhang, Hongxia Ma, Hongsheng Wang, Mingli Zhao, Jing Wu, Lili Bi, Qiubing Chen, Zifeng Li, Ling Yu, Xiaodun Mou, Hao Yin, Li Yang, Jia Chen, Bei Yang, Ying Zhang, Ying Zhang
Topics & Concepts
BiologyPromoterFetal hemoglobinHaematopoiesisGlobinStem cellTranscription factorNucleaseTranscription (linguistics)Progenitor cellGeneticsMolecular biologyDNACell biologyGene expressionGeneFetusPregnancyPhilosophyLinguisticsHemoglobinopathies and Related DisordersCRISPR and Genetic EngineeringParvovirus B19 Infection Studies