Constitutive activation of G protein-coupled receptors and diseases: insights into mechanisms of activation and therapeutics

Pharmacol Ther. 2008 Nov;120(2):129-48. doi: 10.1016/j.pharmthera.2008.07.005. Epub 2008 Aug 9.

Abstract

The existence of constitutive activity for G protein-coupled receptors (GPCRs) was first described in 1980s. In 1991, the first naturally occurring constitutively active mutations in GPCRs that cause diseases were reported in rhodopsin. Since then, numerous constitutively active mutations that cause human diseases were reported in several additional receptors. More recently, loss of constitutive activity was postulated to also cause diseases. Animal models expressing some of these mutants confirmed the roles of these mutations in the pathogenesis of the diseases. Detailed functional studies of these naturally occurring mutations, combined with homology modeling using rhodopsin crystal structure as the template, lead to important insights into the mechanism of activation in the absence of crystal structure of GPCRs in active state. Search for inverse agonists on these receptors will be critical for correcting the diseases cause by activating mutations in GPCRs. Theoretically, these inverse agonists are better therapeutics than neutral antagonists in treating genetic diseases caused by constitutively activating mutations in GPCRs.

Publication types

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

MeSH terms

  • Animals
  • Disease Models, Animal
  • Drug Inverse Agonism*
  • Humans
  • Mutation
  • Receptors, G-Protein-Coupled / agonists
  • Receptors, G-Protein-Coupled / genetics
  • Receptors, G-Protein-Coupled / metabolism*
  • Rhodopsin / genetics
  • Rhodopsin / metabolism
  • Sequence Homology, Amino Acid
  • Structural Homology, Protein

Substances

  • Receptors, G-Protein-Coupled
  • Rhodopsin