International Journal of Molecular Sciences, Год журнала: 2025, Номер 26(3), С. 1303 - 1303
Опубликована: Фев. 3, 2025
Thirty years ago, in 1995, I proposed a fundamental treatment for Duchenne Muscular Dystrophy (DMD) using antisense oligonucleotides (ASOs) to induce exon skipping and restore dystrophin expression. DMD is progressive fatal muscular dystrophy, the establishment of an effective therapy has been pressing demand among patients worldwide. Exon-skipping utilizing ASOs garnered significant attention as one most promising treatments DMD, stimulating global research development efforts ASO technology. Two decades later, 2016, was conditionally approved by U.S. FDA first treatment. This review summarizes current status challenges ASO-based exon-skipping therapies explores prospects pseudoexon ASOs, which holds potential achieving complete cure DMD.
Язык: Английский