Article

Antisense oligonucleotide-mediated correction of <i>CFTR</i> splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells

2020-05-14

Abstract excerpt

<h4>ABSTRACT</h4> 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 more than 15% of CF cases. One common CFTR splicing mutation is CFTR c.3718-2477C>T (3849+10kbC>T), which creates...

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Literature Corpus work: 45cf7430-1c4a-5726-a576-9a9b492ddf7b
DOI: 10.1101/2020.05.12.089417

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