A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease
镰状细胞病的主要编辑策略是重写γ-珠蛋白启动子并重新激活胎儿血红蛋白。
期刊:Blood
影响因子:23.1
doi:10.1182/blood.2024028166
Chalumeau, Anne; Bou Dames, Maria; Fontana, Letizia; Amistadi, Simone; Antoniou, Panagiotis; Loganathan, Priyanka; Mombled, Margaux; Corre, Guillaume; Peterka, Martin; Amendola, Mario; Giovannangeli, Carine; Maresca, Marcello; Miccio, Annarita; Brusson, Mégane