Efficient and in situ correction of hemoglobin Constant Spring mutation by prime editing in human hematopoietic cells

Autor: Shao, Congwen, Liu, Qing, Xu, Jinchao, Zhang, Jianxiang, Zhang, Chengpeng, Xin, Ye, Ye, Yuhua, Lin, Bin, Zhang, Xinhua, Cheng, Li, Xu, Xiangmin, Xu, Peng
Zdroj: In Molecular Therapy - Nucleic Acids 10 December 2024 35(4)
Databáze: ScienceDirect