Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis

• Published in: Science translational medicine Vol. 8; no. 344; p. 344ra84
• Main Authors: Dekkers, Johanna F, Berkers, Gitte, Kruisselbrink, Evelien, Vonk, Annelotte, de Jonge, Hugo R, Janssens, Hettie M, Bronsveld, Inez, van de Graaf, Eduard A, Nieuwenhuis, Edward E S, Houwen, Roderick H J, Vleggaar, Frank P, Escher, Johanna C, de Rijke, Yolanda B, Majoor, Christof J, Heijerman, Harry G M, de Winter-de Groot, Karin M, Clevers, Hans, van der Ent, Cornelis K, Beekman, Jeffrey M
• Format: Journal Article
• Language: English
• Published: United States 22.06.2016
• Subjects: Aminophenols - pharmacology; Aminopyridines - pharmacology; Benzodioxoles - pharmacology; Biological Assay - methods; Chlorides - metabolism; Cystic Fibrosis - drug therapy; Cystic Fibrosis - genetics; Cystic Fibrosis - metabolism; Cystic Fibrosis Transmembrane Conductance Regulator - genetics; Cystic Fibrosis Transmembrane Conductance Regulator - metabolism; Genotype; Humans; In Vitro Techniques; Mutation - genetics; Organoids - drug effects; Organoids - metabolism; Quinolones - pharmacology

Identifying subjects with cystic fibrosis (CF) who may benefit from cystic fibrosis transmembrane conductance regulator (CFTR)-modulating drugs is time-consuming, costly, and especially challenging for individuals with rare uncharacterized CFTR mutations. We studied CFTR function and responses to tw...