A small molecule that binds an RNA repeat expansion stimulates its decay via the exosome complex

Many diseases are caused by toxic RNA repeats. Herein, we designed a lead small molecule that binds the structure of the r(CUG) repeat expansion [r(CUG)(exp)] that causes myotonic dystrophy type 1 (DM1) and Fuch’s endothelial corneal dystrophy (FECD) and rescues disease biology in patient-derived ce...

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Détails bibliographiques
Publié dans:Cell Chem Biol
Auteurs principaux: Angelbello, Alicia J., Benhamou, Raphael I., Rzuczek, Suzanne G., Choudhary, Shruti, Tang, Zhenzhi, Chen, Jonathan L., Roy, Madhuparna, Wang, Kye Won, Yildirim, Ilyas, Jun, Albert S., Thornton, Charles A., Disney, Matthew D.
Format: Artigo
Langue:Inglês
Publié: 2020
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Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC7855261/
https://ncbi.nlm.nih.gov/pubmed/33157036
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.chembiol.2020.10.007
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