Single-dose base editing corrects PKD1 mutation and extends survival in ADPKD preclinical models
by Kelley Luckstein, Mayo Clinic edited by Gaby Clark, reviewed by Andrew Zinin Editors’ notes The GIST Add as preferred source A single injection of Cbh-ABE9-dual-AAV9 decreases heart hypertrophy in Pkd1RC/RC mice. Credit: Nature Communications (2025). DOI: 10.1038/s41467-025-65997-1 Mayo Clinic researchers have developed a promising gene-editing therapy that directly corrects a genetic mutation responsible for autosomal dominant polycystic kidney disease (ADPKD), the […]
