Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1

We address the lack of optimized antimiRs to upregulate MBNL1 in myotonic dystrophy. Through in vitro and in vivo screening and oleic acid conjugation, we identify antimiR leads that boost MBNL1, correct splicing defects, and improve muscle function, highlighting a promising RNA-targeted therapeutic strategy.