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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Gepubliceerd in:Cell Chem Biol
Hoofdauteurs: 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.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2020
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Article
Online toegang: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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