Molecular basis of ClC-6 function and its impairment in human disease

Sci Adv. 2023 Oct 13;9(41):eadg4479. doi: 10.1126/sciadv.adg4479. Epub 2023 Oct 13.

Abstract

ClC-6 is a late endosomal voltage-gated chloride-proton exchanger that is predominantly expressed in the nervous system. Mutated forms of ClC-6 are associated with severe neurological disease. However, the mechanistic role of ClC-6 in normal and pathological states remains largely unknown. Here, we present cryo-EM structures of ClC-6 that guided subsequent functional studies. Previously unrecognized ATP binding to cytosolic ClC-6 domains enhanced ion transport activity. Guided by a disease-causing mutation (p.Y553C), we identified an interaction network formed by Y553/F317/T520 as potential hotspot for disease-causing mutations. This was validated by the identification of a patient with a de novo pathogenic variant p.T520A. Extending these findings, we found contacts between intramembrane helices and connecting loops that modulate the voltage dependence of ClC-6 gating and constitute additional candidate regions for disease-associated gain-of-function mutations. Besides providing insights into the structure, function, and regulation of ClC-6, our work correctly predicts hotspots for CLCN6 mutations in neurodegenerative disorders.

MeSH terms

  • Chloride Channels* / chemistry
  • Chloride Channels* / genetics
  • Humans
  • Ion Transport
  • Mutation
  • Neurodegenerative Diseases* / genetics
  • Structure-Activity Relationship

Substances

  • Chloride Channels
  • CLCN6 protein, human