Cystic fibrosis airway inflammation enables elexacaftor/ tezacaftor/ivacaftor-mediated rescue of N1303K CFTR mutation

ERJ OPEN RESEARCH(2024)

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摘要
Rescue of N1303K CFTR by highly effective modulator therapy (HEMT) is enabled by CF airway inflammation. These findings suggest that evaluation of HEMT for rare CFTR mutations must be performed under inflammatory conditions relevant to CF airways. https://bit.ly/3tTcoJE.
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关键词
cystic fibrosis airway inflammation,cystic fibrosis,mutation,ivacaftor-mediated
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