Article

Systematic deletion of symmetrical <i>CFTR</i> exons reveals new therapeutic targets for exon skipping antisense oligonucleotides

2024-08-29

Abstract excerpt

There is a major need for therapeutics that treat diseases caused by pathogenic gene variants that disrupt protein open-reading frames. Splice-switching antisense oligonucleotides (ASOs) offer a potential solution by inducing the skipping of exons containing these variants, removing them from the mRNA and correcting the open-reading frame. Cystic fibrosis (CF), caused by disruption of the CF transmembrane regulato...

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Literature Corpus work: f09be3f6-40d0-5bcb-b649-3119b1258a5f
DOI: 10.1101/2024.08.28.607949

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