Therapeutic adenine base editing corrects nonsense mutation and improves visual function in a mouse model of Leber congenital amaurosis
Dong Hyun Jo, Hyeon‐Ki Jang, Chang Sik Cho, Jun Hee Han, Gahee Ryu, Youngri Jung, Sangsu Bae, Jeong Hun Kim
This study uses adenine base editing to correct a nonsense mutation in a mouse model of LCA, improving visual function.
bioRxiv · Jan 7, 2021 · PreprintGenetics