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Showing 1 to 1 of 1 for “"MBNL1-CUGexp interaction"”.
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Advances in myotonic dystrophy type 1 drug discovery through design of novel ligands and mechanism establishment
… 1 (DM1) is caused by an expanded CUG repeat (CUGexp) that sequesters muscleblind-like 1 protein (MBNL1), a protein that regulates alternative splicing. CUGexp RNA is a validated drug target for this currently untreatable disease. Herein, we describe the development of a bioactive small …