Engineered botulinum neurotoxin B with improved efficacy for targeting human receptors

Nat Commun. 2017 Jul 3;8(1):53. doi: 10.1038/s41467-017-00064-y.

Abstract

Botulinum neurotoxin B is a Food and Drug Administration-approved therapeutic toxin. However, it has lower binding affinity toward the human version of its major receptor, synaptotagmin II (h-Syt II), compared to mouse Syt II, because of a residue difference. Increasing the binding affinity to h-Syt II may improve botulinum neurotoxin B's therapeutic efficacy and reduce adverse effects. Here we utilized the bacterial adenylate cyclase two-hybrid method and carried out a saturation mutagenesis screen in the Syt II-binding pocket of botulinum neurotoxin B. The screen identifies E1191 as a key residue: replacing it with M/C/V/Q enhances botulinum neurotoxin B binding to human synaptotagmin II. Adding S1199Y/W or W1178Q as a secondary mutation further increases binding affinity. Mutant botulinum neurotoxin B containing E1191M/S1199Y exhibits ~11-fold higher efficacy in blocking neurotransmission than wild-type botulinum neurotoxin B in neurons expressing human synaptotagmin II, demonstrating that enhancing receptor binding increases the overall efficacy at functional levels. The engineered botulinum neurotoxin B provides a platform to develop therapeutic toxins with improved efficacy.Humans are less sensitive to the therapeutic effects of botulinum neurotoxin B (BoNT/B) than the animal models it is tested on due to differences between the human and the mouse receptors. Here, the authors engineer BoNT/B to improve its affinity to human receptors and enhance its therapeutic efficacy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acetylcholine Release Inhibitors / pharmacology
  • Animals
  • Botulinum Toxins, Type A / genetics*
  • Botulinum Toxins, Type A / metabolism
  • Botulinum Toxins, Type A / pharmacology
  • Humans
  • Mutagenesis, Site-Directed
  • Neurons / drug effects
  • Neurons / metabolism
  • Patch-Clamp Techniques
  • Protein Binding / genetics
  • Rats
  • Recombinant Proteins
  • Synaptotagmin II / metabolism*
  • Two-Hybrid System Techniques

Substances

  • Acetylcholine Release Inhibitors
  • Recombinant Proteins
  • SYT2 protein, human
  • Synaptotagmin II
  • rimabotulinumtoxinB
  • Botulinum Toxins, Type A