GABAA receptor signalling mechanisms revealed by structural pharmacology

Nature. 2019 Jan;565(7740):454-459. doi: 10.1038/s41586-018-0832-5. Epub 2019 Jan 2.

Abstract

Type-A γ-aminobutyric (GABAA) receptors are ligand-gated chloride channels with a very rich pharmacology. Some of their modulators, including benzodiazepines and general anaesthetics, are among the most successful drugs in clinical use and are common substances of abuse. Without reliable structural data, the mechanistic basis for the pharmacological modulation of GABAA receptors remains largely unknown. Here we report several high-resolution cryo-electron microscopy structures in which the full-length human α1β3γ2L GABAA receptor in lipid nanodiscs is bound to the channel-blocker picrotoxin, the competitive antagonist bicuculline, the agonist GABA (γ-aminobutyric acid), and the classical benzodiazepines alprazolam and diazepam. We describe the binding modes and mechanistic effects of these ligands, the closed and desensitized states of the GABAA receptor gating cycle, and the basis for allosteric coupling between the extracellular, agonist-binding region and the transmembrane, pore-forming region. This work provides a structural framework in which to integrate previous physiology and pharmacology research and a rational basis for the development of GABAA receptor modulators.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Allosteric Regulation / drug effects
  • Alprazolam / chemistry*
  • Alprazolam / pharmacology
  • Benzodiazepines / chemistry
  • Benzodiazepines / pharmacology
  • Bicuculline / chemistry*
  • Bicuculline / pharmacology
  • Binding, Competitive / drug effects
  • Cryoelectron Microscopy*
  • Diazepam / chemistry*
  • Diazepam / pharmacology
  • GABA Modulators / chemistry
  • GABA Modulators / pharmacology
  • Humans
  • Ligands
  • Models, Molecular
  • Nanostructures / chemistry
  • Picrotoxin / chemistry*
  • Picrotoxin / pharmacology
  • Receptors, GABA-A / chemistry*
  • Receptors, GABA-A / metabolism*
  • Signal Transduction / drug effects*

Substances

  • GABA Modulators
  • Ligands
  • Receptors, GABA-A
  • Picrotoxin
  • Benzodiazepines
  • Diazepam
  • Bicuculline
  • Alprazolam