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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Detalhes bibliográficos
Publicado no:Cell Chem Biol
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: 2020
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Article
Acesso em linha: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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