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

Cystic fibrosis (CF) results from mutations in the chloride-conducting CF transmembrane conductance regulator (CFTR) gene. Airway dehydration and impaired mucociliary clearance in CF is proposed to result in tonic epithelial sodium channel (ENaC) activity, which drives amiloride-sensitive electrogen...

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Dettagli Bibliografici
Pubblicato in:Sci Adv
Autori principali: Mukherjee, Anindit, MacDonald, Kelvin D., Kim, Jeonghwan, Henderson, Michael I., Eygeris, Yulia, Sahay, Gaurav
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Association for the Advancement of Science 2020
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7673816/
https://ncbi.nlm.nih.gov/pubmed/33208364
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.abc5911
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