more
Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy
This study advances next-generation exon 51 skipping antisense oligonucleotides (AONs) for Duchenne muscular dystrophy (DMD) by incorporating novel chemical modifications like 5-methylcytosine and locked nucleic acids (LNAs). Screening 100+ AONs identified an optimized target site, achieving 65-fold higher exon skipping and restoring 30-40% dystrophin levels in preclinical models. These AONs demonstrated improved muscle integrity, enhanced motor function, and a favorable safety profile, marking a major step toward safer, more effective DMD therapies.
