DataSheet1_Cell-Penetrating Botulinum Neurotoxin Type A With Improved Cellular Uptake and Therapeutic Index.docx (2.09 MB)
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posted on 2022-02-11, 04:47 authored by Xuan Wei, Lu Li, Yiwen Wu, Jia LiuBotulinum neurotoxin serotype A (BoNTA) is widely used for treating neuromuscular disorders. Despite of the various marketed products, BoNTA is known to have small therapeutic index ranging from 5 to 15. In the present study, we designed and characterized engineered BoNTA proteins with fusion of cell-penetrating peptides (CPPs). We have shown that CPPs, particularly a recently reported zinc finger protein could improve the cellular uptake and intramuscular therapeutic index of BoNTA. Our study has shed the light on developing next-generation neuromuscular modulators using CPP fusion.
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