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Study of an RNA-Focused DNA-Encoded Library Informs Design of a Degrader of a r(CUG) Repeat Expansion

18 Nov 2022

Abstract excerpt

A solid-phase DNA-encoded library (DEL) was studied for binding the RNA repeat expansion r( CUG ) exp, the causative agent of the most common form of adult-onset muscular dystrophy, myotonic dystrophy type 1 (DM1). A variety of uncharged and novel RNA binders were identified to selectively bind r( CUG ) exp by using a two-color flow cytometry screen. The cellular activity of one binder was augmented by attaching...

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PMID: 36399603
DOI: 10.1021/jacs.2c08883

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