Article

Exon-Skipping Antisense Oligonucleotides for Cystic Fibrosis Therapy

2021-08-11

Abstract excerpt

Mutations in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene cause cystic fibrosis (CF), and the CFTR-W1282X nonsense mutation causes a severe form of CF. Although Trikafta and other CFTR-modulation therapies benefit most CF patients, targeted therapy for patients with the W1282X mutation is lacking. The CFTR-W1282X protein has residual activity, but is expressed at a very low level due to...

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Literature Corpus work: ed77e626-2bb0-530c-bb68-863a7f841850
DOI: 10.1101/2021.08.11.455936

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