Identification of therapeutics that target eEF1A2 and upregulate utrophin A translation in dystrophic muscles

One potential approach for the treatment of Duchenne muscular dysrophy is to increase expression of the dystrophin homolog utrophin. Here, the authors show that eEF1A2 regulates utrophin expression, and show that 2 FDA-approved drugs upregulate eEIF1A2 and utrophin level in mice, leading to improvem...

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Autores principales: Christine Péladeau, Nadine Adam, Lucas M. Bronicki, Adèle Coriati, Mohamed Thabet, Hasanen Al-Rewashdy, Jason Vanstone, Alan Mears, Jean-Marc Renaud, Martin Holcik, Bernard J. Jasmin
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2020
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Acceso en línea:https://doaj.org/article/6bc7a05ce66e4135bb35be45df6c5df1
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Sumario:One potential approach for the treatment of Duchenne muscular dysrophy is to increase expression of the dystrophin homolog utrophin. Here, the authors show that eEF1A2 regulates utrophin expression, and show that 2 FDA-approved drugs upregulate eEIF1A2 and utrophin level in mice, leading to improvement of the dystrophic phenotype.