Identification of drug modifiers for RYR1-related myopathy using a multi-species discovery pipeline

Ryanodine receptor type I-related myopathies (RYR1-RMs) are a common group of childhood muscle diseases associated with severe disabilities and early mortality for which there are no available treatments. The goal of this study is to identify new therapeutic targets for RYR1-RMs. To accomplish this,...

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Udgivet i:eLife
Main Authors: Volpatti, Jonathan R, Endo, Yukari, Knox, Jessica, Groom, Linda, Brennan, Stephanie, Noche, Ramil, Zuercher, William J, Roy, Peter, Dirksen, Robert T, Dowling, James J
Format: Artigo
Sprog:Inglês
Udgivet: eLife Sciences Publications, Ltd 2020
Fag:
Human Biology and Medicine
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7202896/
https://ncbi.nlm.nih.gov/pubmed/32223895
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.7554/eLife.52946
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