One-Size-Fits-Many: Antisense oligonucleotides for rescuing splicing mutations in hotspot exons
Chaorui Duan, Stephen Rong, Luke Buerer, Christopher R. Neil, Juliann M. Savatt, Natasha T. Strande, William Guy Fairbrother
The paper identifies splice-disrupting mutations in hotspot exons and shows that antisense oligonucleotides (ASOs) can revert splicing defects in these exons.
bioRxiv · Dec 7, 2024 · PreprintMolecular Biology