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Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy
(Elsevier, 2021-12-03)
Myotonic dystrophy type 1 (DM1) is a rare neuromuscular disease caused by expansion of unstable CTG repeats in a non-coding region of the DMPK gene. CUG expansions in mutant DMPK transcripts sequester MBNL1 proteins in ...