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Single-dose base editing corrects PKD1 mutation and extends survival in ADPKD preclinical models

[ 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 most common inherited kidney disorder. A single treatment …

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