In vivo base editing reduces liver cysts in autosomal dominant polycystic kidney disease.
Ibel A, Bhardwaj R, Yilmaz DE, Kong S, Wendlinger S, Cordero C, Papaioannou D, Papazian M, Schönauer R, Meng Q, Eckardt KU, Hassan F, Volpe I, Klämbt V, Halbritter J, Fedeles S, Krappitz M, Kaminski MM. In vivo base editing reduces liver cysts in autosomal dominant polycystic kidney disease. Mol Ther. 2025 Nov 05; 33(11):5373-5382.