Editing aberrant splice sites efficiently restores ?-globin expression in ?-thalassemia.
Xu S, Luk K, Yao Q, Shen AH, Zeng J, Wu Y, Luo HY, Brendel C, Pinello L, Chui DHK, Wolfe SA, Bauer DE. Editing aberrant splice sites efficiently restores ?-globin expression in ?-thalassemia. Blood. 2019 05 23; 133(21):2255-2262.