Autosomal dominant polycystic kidney disease (ADPKD) is a leading cause of end-stage renal disease. The vasopressin V2 receptor (VPV2R) antagonist OPC31260 has been effective in two animal models of PKD with pathologies that are probably related. Here we show, in a mouse model of ADPKD (Pkd2−/tm1Som), a similar cellular phenotype and response to OPC31260 treatment, with reduction of renal cyclic AMP (cAMP) levels, prevention of renal enlargement, marked inhibition of cystogenesis and protection of renal function.
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This work was supported by National Institutes of Health grant DK44863 (V.E.T.) and by a grant from the Polycystic Kidney Disease Foundation (V.H.G.). OPC31260 was a gift from Otsuka Pharmaceutical. Technical assistance was provided by Ming Li.
V.H.G. is listed as the inventor on the US patent, held by the University of Kansas Medical Center, entitled, “Treatment of polycystic kidney disease using vasopressin V2 receptor antagonists.”
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Torres, V., Wang, X., Qian, Q. et al. Effective treatment of an orthologous model of autosomal dominant polycystic kidney disease. Nat Med 10, 363–364 (2004). https://doi.org/10.1038/nm1004
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