CFTR and cystic fibrosis: In fact, ASO treatment in non-CF and CFTR-F508del homozygous donor cells in comparison to donor cells with either one or two copies of CFTR-W1282X showed an increase in ASO-induced exon 23 skipping that correlated with the number of CFTR-W1282X alleles (SI Appendix, Fig. S3D).