Longer-acting and highly potent chimaeric inhibitors of excessive exocytosis created with domains from botulinum neurotoxin A and B

Biochem J. 2012 May 15;444(1):59-67. doi: 10.1042/BJ20120100.

Abstract

Various human neurogenic hyper-excitability disorders are successfully treated with type A or B BoNT (botulinum neurotoxin). The BoNT/A complex is widely used because of its longer-lasting benefits; also, autonomic side-effects are more often reported for BoNT/B. To establish if this distinct effect of BoNT/B could be exploited therapeutically, BoNT/A was modified so that it would bind the more abundant BoNT/B acceptor in rodents while retaining its desirable persistent action. The advantageous protease and translocation domain of BoNT/A were recombinantly combined with the acceptor-binding moiety of type B [H(C)/B (C-terminal half of BoNT/B heavy chain)], creating the chimaera AB. This purified protein bound the BoNT/B acceptor, displayed enhanced capability relative to type A for intraneuronally delivering its protease, cleaved SNAP-25 (synaptosome-associated protein of 25 kDa) and induced a more prolonged neuromuscular paralysis than BoNT/A in mice. The BA chimaera, generated by substituting H(C)/A (C-terminal half of BoNT/A heavy chain) into BoNT/B, exhibited an extremely high specific activity, delivered the BoNT/B protease via the BoNT/A acceptor into neurons, or fibroblast-like synoviocytes that lack SNAP-25, cleaving the requisite isoforms of VAMP (vesicle-associated membrane protein). Both chimaeras inhibited neurotransmission in murine bladder smooth muscle. BA has the unique ability to reduce exocytosis from non-neuronal cells expressing the BoNT/A-acceptor and utilising VAMP, but not SNAP-25, in exocytosis.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Botulinum Toxins / genetics*
  • Botulinum Toxins, Type A / genetics*
  • Cells, Cultured
  • Exocytosis / drug effects*
  • Female
  • Fibroblasts / drug effects
  • Fibroblasts / metabolism
  • In Vitro Techniques
  • Mice
  • Muscle, Smooth / drug effects
  • Muscle, Smooth / physiology
  • Neuromuscular Junction / drug effects
  • Neuromuscular Junction / physiopathology
  • Neurons / drug effects
  • Neurons / metabolism
  • Paralysis / chemically induced
  • Peptide Hydrolases / genetics
  • Peptide Hydrolases / metabolism
  • Phrenic Nerve / drug effects
  • Phrenic Nerve / physiopathology
  • Protein Structure, Tertiary
  • Rats
  • Rats, Sprague-Dawley
  • Recombinant Fusion Proteins / genetics
  • Recombinant Fusion Proteins / pharmacology*
  • Synaptic Transmission / drug effects
  • Synaptosomal-Associated Protein 25 / metabolism
  • Synovial Membrane / cytology
  • Urinary Bladder / drug effects
  • Urinary Bladder / physiology
  • Vesicle-Associated Membrane Protein 2 / genetics
  • Vesicle-Associated Membrane Protein 2 / metabolism

Substances

  • Recombinant Fusion Proteins
  • Synaptosomal-Associated Protein 25
  • Vesicle-Associated Membrane Protein 2
  • rimabotulinumtoxinB
  • Peptide Hydrolases
  • Botulinum Toxins
  • Botulinum Toxins, Type A