Molecular BiologyProceedings of the National Academy of SciencesPeer-reviewed
Exon-Skipping Antisense Oligonucleotides for Cystic Fibrosis Therapy
Young Jin Kim, Nicole Sivetz, Jessica Layne…·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.
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