Cystic fibrosis airway inflammation enables elexacaftor/tezacaftor/ivacaftor-mediated rescue of N1303K CFTR mutation.
Martina GentzschBrooke BakerDeborah M CholonCharissa W KamCameron J McKinzieKatherine A DespotesSusan E BoylesNancy L QuinneyCharles R EstherCarla M P RibeiroPublished in: ERJ open research (2024)
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.