Global ETD Search

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Showing 1 to 1 of 1 for “"RNA binder"”.

  1. Approaches to the assembly of potent therapeutic agents for the treatment of myotonic dystrophy

    … caused by an expansion of the CTG repeat, whose RNA transcript sequesters the MBNL1 protein into nuclear foci, leading to the misregulation of various pre-mRNAs. Based on a published x-ray structure of a r(CUG)6 hairpin, a benzamidinium ligand was rationally designed and found to selectively bind …

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