Exon-skipping antisense oligonucleotides for cystic fibrosis therapy

Abstract excerpt

Significance Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene, which can lead to respiratory failure. To date, there is no treatment for CF caused by the CFTR- W1282X mutation located on CFTR exon 23. Nonsense-mediated messenger RNA (mRNA) decay (NMD) degrades the CFTR- W1282X mRNA, leading to low levels of functional CFTR protein. We developed a...

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