Article
Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis.
Science translational medicine - 22 Jun 2016
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
Abstract excerpt
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 two drugs-the prototypical CFTR potentiator VX-770 (ivacaftor/KALYDECO) and the CFTR corrector VX-809 (lumacaftor)-in...
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