Small-molecule activation of lysosomal TRP channels ameliorates Duchenne muscular dystrophy in mouse models

Duchenne muscular dystrophy (DMD) is a devastating disease caused by mutations in dystrophin that compromise sarcolemma integrity. Currently, there is no treatment for DMD. Mutations in transient receptor potential mucolipin 1 (ML1), a lysosomal Ca(2+) channel required for lysosomal exocytosis, prod...

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Veröffentlicht in:Sci Adv
Hauptverfasser: Yu, Lu, Zhang, Xiaoli, Yang, Yexin, Li, Dan, Tang, Kaiyuan, Zhao, Zifan, He, Wanwan, Wang, Ce, Sahoo, Nirakar, Converso-Baran, Kimber, Davis, Carol S., Brooks, Susan V., Bigot, Anne, Calvo, Raul, Martinez, Natalia J., Southall, Noel, Hu, Xin, Marugan, Juan, Ferrer, Marc, Xu, Haoxing
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Association for the Advancement of Science 2020
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Research Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7032923/
https://ncbi.nlm.nih.gov/pubmed/32128386
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.aaz2736
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