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VX‐809 mitigates disease in a mouse model of autosomal dominant polycystic kidney disease bearing the R3277C human mutation

18 Oct 2021

Abstract excerpt

Abstract Autosomal dominant polycystic kidney disease (ADPKD) is associated with the formation of renal cysts. We have devised a therapeutic approach, based on reversing the cyst phenotype from secretion to absorption by using VX‐809, a modulator of the cystic fibrosis transmembrane regulator trafficking and processing. Our goal is to test VX‐809 in RC/RC mice bearing the R3277C human mutation to demonstrate its...

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PMID: 34662459
DOI: 10.1096/fj.202101315r

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