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Oral administration of erythromycin decreases RNA toxicity in myotonic dystrophy

10 Dec 2015

Abstract excerpt

OBJECTIVE: Myotonic dystrophy type 1 (DM1) is caused by the expansion of a CTG repeat in the 3' untranslated region of DMPK. The transcripts containing an expanded CUG repeat (CUG (exp)) result in a toxic gain-of-function by forming ribonuclear foci that sequester the alternative splicing factor muscleblind-like 1 (MBNL1). Although several small molecules reportedly ameliorate RNA toxicity, none are ready for...

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PMID: 26783549
DOI: 10.1002/acn3.271

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