Exon-Skipping Antisense Oligonucleotides for Cystic Fibrosis Therapy
Young Jin Kim, Nicole Sivetz, Jessica Layne, Dillon M. Voss, Lucia Yang, Qian Zhang, Adrian R. Krainer
This paper develops an exon-skipping antisense oligonucleotide (ASO) strategy to increase CFTR protein levels in patients with the W1282X mutation by bypassing nonsense-mediated mRNA decay.
Proceedings of the National Academy of Sciences · Jan 10, 2022Peer-reviewedMolecular Biology