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Article

Cell Membrane Integrity in Myotonic Dystrophy Type 1: Implications for Therapy

23 Mar 2015

Abstract excerpt

Myotonic Dystrophy type 1 (DM1) is a multisystemic disease caused by toxic RNA from a DMPK gene carrying an expanded (CTG•CAG)n repeat. Promising strategies for treatment of DM1 patients are currently being tested. These include antisense oligonucleotides and drugs for elimination of expanded RNA or prevention of aberrant binding to RNP proteins. A significant hurdle for preclinical development along these lines...

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PMID: 25799359
DOI: 10.1371/journal.pone.0121556

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