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Cells of Matter—In Vitro Models for Myotonic Dystrophy

23 May 2018

Abstract excerpt

Myotonic Dystrophy type 1 (DM1 also known as Steinert disease) is a multisystemic disorder mainly characterized by myotonia, progressive muscle weakness and wasting, cognitive impairments and cardiac defects. This autosomal dominant disease is caused by the expression of nuclear retained RNAs containing pathologic expanded CUG repeats that alters the function of RNA binding proteins in a tissue-specific manner,...

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PMID: 29875732
DOI: 10.3389/fneur.2018.00361

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