Emerging Opportunities in Human Pluripotent Stem-Cells Based Assays to Explore the Diversity of Botulinum Neurotoxins As Future Therapeutics

Overview

Journal Int J Mol Sci

Publisher MDPI

Specialties Biochemistry
Chemistry
Molecular Biology

Date 2021 Jul 24

PMID 34299143

Citations 2

Authors

Juliette Duchesne de Lamotte

Anselme Perrier

Cecile Martinat

Camille Nicoleau

Affiliations

Soon will be listed here.

Abstract

Botulinum neurotoxins (BoNTs) are produced by Clostridium botulinum and are responsible for botulism, a fatal disorder of the nervous system mostly induced by food poisoning. Despite being one of the most potent families of poisonous substances, BoNTs are used for both aesthetic and therapeutic indications from cosmetic reduction of wrinkles to treatment of movement disorders. The increasing understanding of the biology of BoNTs and the availability of distinct toxin serotypes and subtypes offer the prospect of expanding the range of indications for these toxins. Engineering of BoNTs is considered to provide a new avenue for improving safety and clinical benefit from these neurotoxins. Robust, high-throughput, and cost-effective assays for BoNTs activity, yet highly relevant to the human physiology, have become indispensable for a successful translation of engineered BoNTs to the clinic. This review presents an emerging family of cell-based assays that take advantage of newly developed human pluripotent stem cells and neuronal function analyses technologies.

Citing Articles

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Understanding Clinical Effectiveness and Safety Implications of Botulinum Toxin in Children: A Narrative Review of the Literature.

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Human-Relevant Sensitivity of iPSC-Derived Human Motor Neurons to BoNT/A1 and B1.

Schenke M, Prause H, Bergforth W, Przykopanski A, Rummel A, Klawonn F Toxins (Basel). 2021; 13(8).

PMID: 34437455 PMC: 8402508. DOI: 10.3390/toxins13080585.

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