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Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects

5 May 2014

Abstract excerpt

Myotonic dystrophy type 1 (DM1) is a dominantly inherited neuromuscular disorder resulting from expression of RNA containing an expanded CUG repeat (CUG(exp)). The pathogenic RNA is retained in nuclear foci. Poly-(CUG) binding proteins in the Muscleblind-like (MBNL) family are sequestered in foci, causing misregulated alternative splicing of specific pre-mRNAs. Inhibitors of MBNL1-CUG(exp) binding have been shown...

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PMID: 24799433
DOI: 10.1093/nar/gku275

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