2′F-ANA-LET7 mediated utrophin upregulation for DMD therapy
Abstract:
The invention relates to compositions and methods for enhancing or upregulating utrophin protein production and methods for treating myopathies, such as Duchenne Muscular Dystrophy (DMD). Specifically, the invention relates to compositions, such as oligonucleotides, and methods for enhancing or upregulating utrophin in a subject by blocking binding of let-7c miRNA to the utrophin mRNA 3 untranslated region (UTR).
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