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author = "Mulders, Susan A. M." > : "Biological Sciences" (x)
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Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy

Description : Myotonic dystrophy type 1 (DM1) is caused by toxicity of an expanded, noncoding (CUG)n tract in DM protein kinase (DMPK) transcripts. According to current evidence the long (CUG)n segment is involved in entrapment of muscleblind (Mbnl) proteins in ribonuclear aggregates and stabilized expression of ...
Repository : Europe PubMed Central
Language(s) : English
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