Molecular BiologybioRxivPreprint
One-Size-Fits-Many: Antisense oligonucleotides for rescuing splicing mutations in hotspot exons
Chaorui Duan, Stephen Rong, Luke Buerer…·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.
2 citations