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In vivo base editing reduces liver cysts in autosomal dominant polycystic kidney disease

21 Aug 2025

Abstract excerpt

Autosomal dominant polycystic kidney disease (ADPKD) is the most prevalent genetic kidney disorder, affecting over 10 million individuals worldwide. Cystic expansion typically progresses to kidney failure and also involves the liver with limited treatment options. Pathogenic variants in PKD1 or PKD2 account for 85%-90% of cases. Genetic re-expression of Pkd1 or Pkd2 has been shown to partially reverse key...

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PMID: 40842155
DOI: 10.1016/j.ymthe.2025.08.026

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