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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Bibliografski detalji
Izdano u:Cell Chem Biol
Glavni autori: 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
Jezik:Inglês
Izdano: 2020
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Article
Online pristup: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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