Polycystic kidney disease: genes, proteins, animal models, disease mechanisms and therapeutic opportunities

J Intern Med. 2007 Jan;261(1):17-31. doi: 10.1111/j.1365-2796.2006.01743.x.


An increased understanding of the genetic, molecular and cellular mechanisms responsible for the development of polycystic kidney disease has laid out the foundation for the development of rational therapies. Many animal models where these therapies can be tested are currently available. This review summarizes the rationale for these treatments, the results of preclinical trials and the prospects for clinical trials, some already in early phases of implementation.

Publication types

  • Review

MeSH terms

  • Animals
  • Calcium / metabolism
  • Cell Membrane / metabolism
  • Controlled Clinical Trials as Topic
  • Homeostasis
  • Humans
  • Kidney / metabolism
  • Membrane Proteins / genetics
  • Membrane Proteins / metabolism
  • Mice
  • Models, Animal
  • Polycystic Kidney Diseases* / genetics
  • Polycystic Kidney Diseases* / metabolism
  • Polycystic Kidney Diseases* / therapy
  • Rats


  • Membrane Proteins
  • Calcium