Article

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

2020-05-12

Abstract excerpt

We describe the design of a small molecule that binds the structure of a r(CUG) repeat expansion [r(CUG) exp ] and reverses molecular defects in two diseases mediated by the RNA - myotonic dystrophy type 1 (DM1) and Fuchs endothelial corneal dystrophy (FECD). Thus, a single structure-specific ligand has potential therapeutic benefit for multiple diseases, in contrast to oligonucleotide-based modalities that are c...

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Literature Corpus work: 896f466f-2420-5353-b350-d12d5310cc59
DOI: 10.1101/2020.05.11.088427

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