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Article

A simple ligand that selectively targets CUG trinucleotide repeats and inhibits MBNL protein binding

8 Sept 2009

Abstract excerpt

This work describes the rational design, synthesis, and study of a ligand that selectively complexes CUG repeats in RNA (and CTG repeats in DNA) with high nanomolar affinity. This sequence is considered a causative agent of myotonic dystrophy type 1 (DM1) because of its ability to sequester muscleblind-like (MBNL) proteins. Ligand 1 was synthesized in two steps from commercially available compounds, and its...

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PMID: 19805260
DOI: 10.1073/pnas.0901824106

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