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

Proceedings of the National Academy of Sciences, Volume 123, Number 2, January 2026.
SignificanceThis work presents a class of triplet nucleic acid ligands that combine the targeting precision of antisense oligonucleotides with the compactness of small molecules to selectively engage the expanded RNA-CUG repeats involved in Myotonic…