Validation of Effective Therapeutic Targets for ADPKD Using Animal Models.

Various polycystic kidney disease (PKD) animal models including Pkd1- or Pkd2-deficient mice have been developed and efficiently utilized to identify novel therapeutic targets as well as elucidate multiple mechanisms of cyst formation in PKD. Based on several successful in vivo studies, preclinical approaches using PKD animal models would shed light on the development of potential therapeutic strategies for PKD. Here, we provide an update on the current evidence obtained by the in vivo evaluation of PKD therapeutic candidates and discuss the effect of therapeutic targets.