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New therapeutic targets for DM1: miRNAs analysis in DM1 disease models (DM1-MIRNA)
Start date: Jul 1, 2011, End date: Jun 30, 2015,
Expansion of a non-coding CTG tract in the DMPK gene leads to myotonic dystrophy type 1 (DM1), a dominant autosomal condition clinically characterized by myotonia, muscle waste and heart conduction defects among other symptoms. Transcripts containing long CUG stretches are toxic to the cell by their ability to interfere with gene expression regulation at several levels. Chief molecular alterations ...
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