Pharmacology & ToxicologybioRxivPreprint
Structural engineering of dual-targeting antisense oligonucleotides enhances exon 44 skipping for treating Duchenne muscular dystrophy
Grant M. Shoffner, Florian Barthélémy, Justin Pi…·Feng Guo
This study develops 3D-bridge antisense oligonucleotides that enhance exon 44 skipping in dystrophin mRNA, showing improved binding affinity and efficacy compared to conventional ASOs.
DOI 10.64898/2026.10.01.754576