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Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1
Keywords antisense oligonucleotides MBNL myotonic dystrophy oleic acid conjugate muscle uptake Introduction Myotonic dystrophy type 1 (DM1; MIM: 160900) is a rare genetic disorder with an overall prevalence of one in 3,000–8,000 individuals worldwide.1 DM1 is characterized by expansion of an unstable CTG microsatellite repeat in the 3′ untranslated region (UTR) of DM1 protein kinase (DMPK; MIM: 605377).
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