Validation of Effective Therapeutic Targets for ADPKD Using Animal Models

Adv Exp Med Biol. 2016:933:71-84. doi: 10.1007/978-981-10-2041-4_7.

Abstract

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.

Keywords: Animal model; PKD; Therapeutic target.

Publication types

  • Review

MeSH terms

  • Animals
  • Disease Models, Animal
  • Humans
  • Molecular Targeted Therapy / methods*
  • Polycystic Kidney, Autosomal Dominant / drug therapy*
  • Polycystic Kidney, Autosomal Dominant / genetics