Engineered mutant α-ENaC subunit mRNA delivered by lipid nanoparticles reduces amiloride currents in cystic fibrosis–based cell and mice models
Anindit Mukherjee, Kelvin D. MacDonald, Jeonghwan Kim, Michael Henderson, Yulia Eygeris, Gaurav Sahay
Abstract
KO mice. These data suggest that mRNA-based ENaC inhibition is a powerful strategy for reducing mucus dehydration and has therapeutic potential for treating CF in all patients, independent of genotype.
Topics & Concepts
Epithelial sodium channelAmilorideCystic fibrosis transmembrane conductance regulatorCystic fibrosisTransfectionSodium channelSodiumMucociliary clearanceChemistryMutantCell biologyBiophysicsBiologyInternal medicineBiochemistryMedicineGeneLungOrganic chemistryCystic Fibrosis Research AdvancesNeonatal Respiratory Health ResearchEnergy Harvesting in Wireless Networks