Structural characterisation of the catalytic domain of botulinum neurotoxin X - high activity and unique substrate specificity

Sci Rep. 2018 Mar 14;8(1):4518. doi: 10.1038/s41598-018-22842-4.

Abstract

Botulinum neurotoxins (BoNTs) are among the most potent toxins known and are also used to treat an increasing number of medical disorders. There are seven well-established serotypes (BoNT/A-G), which all act as zinc-dependent endopeptidases targeting specific members of the SNARE proteins required for synaptic vesicle exocytosis in neurons. A new toxin serotype, BoNT/X, was recently identified. It cleaves not only the canonical targets, vesicle associated membrane proteins (VAMP) 1/2/3 at a unique site, but also has the unique ability to cleave VAMP4/5 and Ykt6. Here we report the 1.35 Å X-ray crystal structure of the light chain of BoNT/X (LC/X). LC/X shares the core fold common to all other BoNTs, demonstrating that LC/X is a bona fide member of BoNT-LCs. We found that access to the catalytic pocket of LC/X is more restricted, and the regions lining the catalytic pocket are not conserved compared to other BoNTs. Kinetic studies revealed that LC/X cleaves VAMP1 with a ten times higher efficiency than BoNT/B and the tetanus neurotoxin. The structural information provides a molecular basis to understand the convergence/divergence between BoNT/X and other BoNTs, to develop effective LC inhibitors, and to engineer new scientific tools and therapeutic toxins targeting distinct SNARE proteins in cells.

Publication types

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

MeSH terms

  • Binding Sites
  • Botulinum Toxins, Type A / chemistry*
  • Botulinum Toxins, Type A / metabolism*
  • Catalytic Domain*
  • Clostridium botulinum / enzymology*
  • Enzyme Activation
  • Models, Molecular
  • Protein Binding
  • Protein Conformation
  • Structure-Activity Relationship
  • Substrate Specificity

Substances

  • Botulinum Toxins, Type A