Rectal organoid-guided CFTR modulator therapy restores lung function in a cystic fibrosis patient with the rare 1677delTA/R334W genotype
Georgia Mitropoulou, Nathalie Brandenberg, Sylke Hoehnel, Camilla Ceroni, Zisis Balmpouzis, Sylvain Blanchon, Gian Dorta, Alain Sauty, Angela Koutsokera
Abstract
<b><i>Ex vivo</i> analysis of intestinal organoids has great translational potential as a precision medicine tool for the prediction of clinical efficacy of CFTR modulators and, for patients with rare variants, may hold the key to these life-changing therapies</b> http://bit.ly/3G1FkDp
Topics & Concepts
OrganoidCystic fibrosisEx vivoMedicineLungLung functionCystic fibrosis transmembrane conductance regulatorGenetic enhancementInternal medicineBioinformaticsIn vivoPathologyCancer researchBiologyNeuroscienceGeneGeneticsCystic Fibrosis Research AdvancesNeonatal Respiratory Health ResearchInhalation and Respiratory Drug Delivery