Sequence determinants of efficient exon 44 skipping in Duchenne muscular dystrophy define design principles for steric-blocking antisense oligonucleotides
Enyang Han, Katherine Webster, Tiberiu Loredan Stan, C. Tanganyika-de Winter, Elizabeth M. van der Pijl, Jordan Tahquechi, Brian Heglar, Chad Koehler, Irinna Papangeli, Donnie Mackenzie, Brett E. Crawford, Annemieke M Aartsma-Rus, Tom A. Hartl
The paper identifies sequence and chemical features of ASOs that efficiently induce exon 44 skipping in DMD and defines design principles for therapeutic ASOs.
bioRxiv · Jul 1, 2026 · PreprintMolecular Biology