Metabolic Changes in Polycystic Kidney Disease as a Potential Target for Systemic Treatment

Int J Mol Sci. 2020 Aug 24;21(17):6093. doi: 10.3390/ijms21176093.

Abstract

Autosomal recessive and autosomal dominant polycystic kidney disease (ARPKD, ADPKD) are systemic disorders with pronounced hepatorenal phenotypes. While the main underlying genetic causes of both ARPKD and ADPKD have been well-known for years, the exact molecular mechanisms resulting in the observed clinical phenotypes in the different organs, remain incompletely understood. Recent research has identified cellular metabolic changes in PKD. These findings are of major relevance as there may be an immediate translation into clinical trials and potentially clinical practice. Here, we review important results in the field regarding metabolic changes in PKD and their modulation as a potential target of systemic treatment.

Keywords: ADPKD; ARPKD; cellular metabolism; cilia; fibrocystin; polycystin.

Publication types

  • Review

MeSH terms

  • Humans
  • Metabolome / physiology
  • Molecular Targeted Therapy / methods
  • Molecular Targeted Therapy / trends*
  • Polycystic Kidney Diseases / drug therapy*
  • Polycystic Kidney Diseases / genetics
  • Polycystic Kidney Diseases / metabolism*
  • Polycystic Kidney, Autosomal Dominant / drug therapy
  • Polycystic Kidney, Autosomal Dominant / genetics
  • Polycystic Kidney, Autosomal Dominant / metabolism
  • Polycystic Kidney, Autosomal Recessive / drug therapy
  • Polycystic Kidney, Autosomal Recessive / genetics
  • Polycystic Kidney, Autosomal Recessive / metabolism