O.32Genome editing of expanded CTG repeats within the human DMPK gene reduces nuclear RNA foci in muscle of DM1 mice

Myotonic dystrophy type 1 (DM1) is the most common inherited myopathy in adults and belongs to the class of the nucleotide repeats disorders. In particular, DM1 is caused by the expansion of the trinucleotide CTG within the 3′ untranslated region (3′UTR) of the myotonic dystrophy protein kinase gene (DMPK). The number of CTG repeats varies from 5 to 37 in unaffected individuals, and from 51 to several thousands in DM1 patients, and severity of...