A pothole-filling strategy for selective targeting of rCUG-repeats associated with myotonic dystrophy type 1.

ABSTRACT: We present an alternative approach to conventional small-molecule and antisense strategies for selectively targeting expanded CUG-RNA repeats associated with Myotonic Dystrophy type 1. Our alternatively designed nucleic acid ligands uniquely integrate advantageous features from both existing methods: They are compact (only three units in length), structurally resembling small molecules, yet recognize RNA targets through directional hydrogen-bonding similar to antisense oligonucleotides. Notably, these ligands exhibit greater specificity and selectivity than either approach alone. This enhanced specificity results from their bifacial recognition mechanism, wherein mismatches on one binding interface are reciprocally mirrored on the complementary face. Additionally, their short length signif

SUBMITTER: Perera JDR 

PROVIDER: S-EPMC12799113 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature