Comparison of Organoid Swelling and <i>In Vivo</i> Biomarkers of CFTR Function to Determine Effects of Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for the F508del Mutation
Simon Y. Graeber, Peter van Mourik, Annelotte M. Vonk, Evelien Kruisselbrink, Stephanie Hirtz, Cornelis K. van der Ent, Marcus Mall, Jeffrey M. Beekman
Abstract
"Comparison of Organoid Swelling and In Vivo Biomarkers of CFTR Function to Determine Effects of Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for the F508del Mutation." American Journal of Respiratory and Critical Care Medicine, 202(11), pp. 1589–1592
Topics & Concepts
IvacaftorCystic fibrosisMedicineOrganoidIn vivoMutationCystic fibrosis transmembrane conductance regulatorPathologyInternal medicineGeneticsBiologyGeneCystic Fibrosis Research AdvancesNeonatal Respiratory Health ResearchTracheal and airway disorders