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Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells
Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, encoding an anion channel that conducts chloride and bicarbonate across epithelial membranes. Mutations that disrupt pre-mRNA splicing occur in >15% of CF cases....
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| Published in: | Nucleic Acids Res |
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| Main Authors: | , , |
| Format: | Artigo |
| Language: | Inglês |
| Published: |
Oxford University Press
2020
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| Subjects: | |
| Online Access: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7367209/ https://ncbi.nlm.nih.gov/pubmed/32520327 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkaa490 |
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