Open reading frame correction using antisense oligonucleotides for the treatment of cystic fibrosis caused by CFTR-W1282X
W. E. Michaels, C. Pena-Rasgado, R. Kotaria, R. J. Bridges, M. L. Hastings
This paper shows that ASO-induced exon 23 skipping restores CFTR function in CFTR-W1282X patients by rescuing mRNA expression and chloride current.
bioRxiv · Aug 11, 2021 · PreprintCell Biology