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MicroRNA-Based Therapeutic Perspectives in Myotonic Dystrophy

9 Nov 2019

Abstract excerpt

Myotonic dystrophy involves two types of chronically debilitating rare neuromuscular diseases: type 1 (DM1) and type 2 (DM2). Both share similarities in molecular cause, clinical signs, and symptoms with DM2 patients usually displaying milder phenotypes. It is well documented that key clinical symptoms in DM are associated with a strong mis-regulation of RNA metabolism observed in patient's cells. This...

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PMID: 31717488
DOI: 10.3390/ijms20225600

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