Molecular pathways and therapies in autosomal-dominant polycystic kidney disease

Physiology (Bethesda). 2015 May;30(3):195-207. doi: 10.1152/physiol.00032.2014.

Abstract

Autosomal-dominant polycystic kidney disease (ADPKD) is the most prevalent inherited renal disease, characterized by multiple cysts that can eventually lead to kidney failure. Studies investigating the role of primary cilia and polycystins have significantly advanced our understanding of the pathogenesis of PKD. This review will present clinical and basic aspects of ADPKD, review current concepts of PKD pathogenesis, evaluate potential therapeutic targets, and highlight challenges for future clinical studies.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Review

MeSH terms

  • Animals
  • Cilia
  • Genetic Predisposition to Disease
  • Genetic Testing
  • Humans
  • Kidney* / drug effects
  • Kidney* / metabolism
  • Kidney* / pathology
  • Molecular Targeted Therapy
  • Mutation*
  • Phenotype
  • Polycystic Kidney, Autosomal Dominant / diagnosis
  • Polycystic Kidney, Autosomal Dominant / epidemiology
  • Polycystic Kidney, Autosomal Dominant / genetics*
  • Polycystic Kidney, Autosomal Dominant / metabolism
  • Polycystic Kidney, Autosomal Dominant / therapy*
  • Predictive Value of Tests
  • Risk Factors
  • Signal Transduction
  • TRPP Cation Channels / genetics*
  • Treatment Outcome

Substances

  • TRPP Cation Channels
  • polycystic kidney disease 1 protein
  • polycystic kidney disease 2 protein