CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-Thalassemia.

Frangoul H, Hanna R, Walters MC, Kao RL, Carroll C, McManus M, Chang KH, Jaskolka MC, Kim K, Yu Q, Badamosi N, Mei B, Afonja O, Thompson A, EdiThal Investigators