In vivo base editing reduces liver cysts in autosomal dominant polycystic kidney disease
Antonia Ibel, Rishi Bhardwaj, Duygu Elif Yılmaz, Shuhan Kong, Sarah Wendlinger, Carlos Cordero, Dimitra Papaioannou, Maria Papazian, Ria Anne-Rose Schönauer, Qiyao Meng, Kai‐Uwe Eckardt, Fatima Hassan, Isabel Volpe, Verena Klämbt, Jan Halbritter, , Michael M. Kaminski
This study shows that CRISPR base editing can correct pathogenic PKD1 variants in mice, reducing liver cysts and increasing polycystin-1 expression.
Molecular Therapy · Aug 21, 2025Peer-reviewedGenetics