Peladeau, Christine; Adam, Nadine; Bronicki, Lucas M.; Coriati, Adele; Thabet, Mohamed; Al-Rewashdy, Hasanen; Vanstone, Jason; Mears, Alan; Renaud, Jean-Marc; Holcik, Martin; Jasmin, Bernard J. published the artcile< Identification of therapeutics that target eEF1A2 and upregulate utrophin A translation in dystrophic muscles>, HPLC of Formula: 6054-98-4, the main research area is muscular dystrophy relaxant utrophin A eEF1A2 therapeutic target.
Abstract: Up-regulation of utrophin in muscles represents a promising therapeutic strategy for the treatment of Duchenne Muscular Dystrophy. We previously demonstrated that eEF1A2 associates with the 5’UTR of utrophin A to promote IRES-dependent translation. Here, we examine whether eEF1A2 directly regulates utrophin A expression and identify via an ELISA-based high-throughput screen, FDA-approved drugs that upregulate both eEF1A2 and utrophin A. Our results show that transient overexpression of eEF1A2 in mouse muscles causes an increase in IRES-mediated translation of utrophin A. Through the assessment of our screen, we reveal 7 classes of FDA-approved drugs that increase eEF1A2 and utrophin A protein levels. Treatment of mdx mice with the 2 top leads results in multiple improvements of the dystrophic phenotype. Here, we report that IRES-mediated translation of utrophin A via eEF1A2 is a critical mechanism of regulating utrophin A expression and reveal the potential of repurposed drugs for treating DMD via this pathway.
Nature Communications published new progress about Anterior tibial muscle. 6054-98-4 belongs to class alcohols-buliding-blocks, and the molecular formula is C14H8N2Na2O6, HPLC of Formula: 6054-98-4.
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