Therapeutic base editing of human hematopoietic stem cells

Zeng, J; Wu, YX; Ren, CY; Bonanno, J; Shen, AH; Shea, D; Gehrke, JM; Clement, K; Luk, K; Yao, QM; Kim, R; Wolfe, SA; Manis, JP; Pinello, L; Joung, JK; Bauer, DE

Bauer, DE (corresponding author), Harvard Med Sch, Div Hematol Oncol, Boston Childrens Hosp,Broad Inst,Dept Pediat, Dept Pediat Oncol,Dana Farber Canc Inst,Harvard S, Boston, MA 02115 USA.

NATURE MEDICINE, 2020; 26 (4): 535

Abstract

Base editing by nucleotide deaminases linked to programmable DNA-binding proteins represents a promising approach to permanently remedy blood disorder......

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