Engineered mutant α-ENaC subunit mRNA delivered by lipid nanoparticles reduces amiloride currents in cystic fibrosis–based cell and mice models

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The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

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Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

Harry Heijerman, Edward F. McKone, D.G. Downey et al. · The Lancet · 2019 · 1.4K citations · Full text

Life Sciences, Genetics, Pharmacology +14

Evidence for Periciliary Liquid Layer Depletion, Not Abnormal Ion Composition, in the Pathogenesis of Cystic Fibrosis Airways Disease

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Veterinary Medicine, Pathobiology, Physiology +13

CFTR as a cAMP-Dependent Regulator of Sodium Channels

M. Jackson Stutts, Cecilia M. Canessa, John C. Olsen et al. · Science · 1995 · 1.1K citations

Life Sciences, Cell Biology, Molecular Biology +17