Article

Influence of Disease-Causing Mutations and Ivacaftor on the Dynamics of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Protein

2025-07-07

Abstract excerpt

Cystic fibrosis (CF), one of the most common life-shortening autosomal recessive diseases, arises from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and leads to severe respiratory and digestive dysfunction. CFTR-targeting therapies such as ivacaftor have transformed CF care by directly modulating CFTR function. However, the precise mechanism by which ivacaftor alters CFTR’s conf...

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Literature Corpus work: 487ad0cf-f256-5fd2-bba1-8b286117c672
DOI: 10.1101/2025.07.07.663434

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