Crystal structure of a human GABAA receptor

Nature. 2014 Aug 21;512(7514):270-5. doi: 10.1038/nature13293. Epub 2014 Jun 8.

Abstract

Type-A γ-aminobutyric acid receptors (GABAARs) are the principal mediators of rapid inhibitory synaptic transmission in the human brain. A decline in GABAAR signalling triggers hyperactive neurological disorders such as insomnia, anxiety and epilepsy. Here we present the first three-dimensional structure of a GABAAR, the human β3 homopentamer, at 3 Å resolution. This structure reveals architectural elements unique to eukaryotic Cys-loop receptors, explains the mechanistic consequences of multiple human disease mutations and shows an unexpected structural role for a conserved N-linked glycan. The receptor was crystallized bound to a previously unknown agonist, benzamidine, opening a new avenue for the rational design of GABAAR modulators. The channel region forms a closed gate at the base of the pore, representative of a desensitized state. These results offer new insights into the signalling mechanisms of pentameric ligand-gated ion channels and enhance current understanding of GABAergic neurotransmission.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Benzamidines / chemistry
  • Benzamidines / metabolism
  • Benzamidines / pharmacology
  • Binding Sites
  • Cell Membrane / chemistry
  • Cell Membrane / metabolism
  • Conserved Sequence
  • Crystallography, X-Ray
  • Drug Design
  • GABA-A Receptor Agonists / chemistry
  • GABA-A Receptor Agonists / metabolism
  • GABA-A Receptor Agonists / pharmacology
  • Genetic Predisposition to Disease
  • Glycosylation
  • Humans
  • Models, Molecular
  • Mutation / genetics
  • Polysaccharides / chemistry
  • Polysaccharides / metabolism
  • Protein Structure, Quaternary
  • Protein Structure, Tertiary
  • Protein Subunits
  • Receptors, GABA-A / chemistry*
  • Receptors, GABA-A / genetics
  • Synaptic Transmission

Substances

  • Benzamidines
  • GABA-A Receptor Agonists
  • Polysaccharides
  • Protein Subunits
  • Receptors, GABA-A
  • benzamidine

Associated data

  • PDB/4COF