Molecular BiologybioRxivPreprint
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…·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.
DOI 10.64898/2026.06.29.735365