Article

<i>In vivo</i> base editing reduces liver cysts in autosomal dominant polycystic kidney disease

2025-02-08

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 chara...

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Literature Corpus work: eaefeff4-53ee-596c-924b-07615fe41ef9
DOI: 10.1101/2025.02.07.636600

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