Research ArticleBOTULISM

Neuronal delivery of antibodies has therapeutic effects in animal models of botulism

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Science Translational Medicine  06 Jan 2021:
Vol. 13, Issue 575, eabd7789
DOI: 10.1126/scitranslmed.abd7789

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Nontoxic botulinum for drug delivery

Botulism is a severe and potentially fatal disease characterized by muscle paralysis. The causing agent, botulinum neurotoxins (BoNTs), has the ability to enter motor neurons and to block neurotransmission. In two independent studies, Miyashita et al. and McNutt et al. used nontoxic derivative of BoNT to deliver therapeutic antibodies against BoNTs in neurons. Miyashita et al. targeted BoNT/A and BoNT/B and reported therapeutic effects in mice. Using a similar approach targeting BoNT/A, McNutt et al. increased survival after lethal challenge in mice, guinea pigs, and monkeys. This approach provided a safe and effective treatment against BoNT intoxication and could be exploited for targeting other intracellular proteins in neurons.

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