Article
Potentiator synergy in rectal organoids carrying S1251N, G551D, or F508del CFTR mutations.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Sept 2016
Dekkers Johanna F, Van Mourik Peter, Vonk Annelotte M, Kruisselbrink Evelien, Berkers Gitte, de Winter-de Groot Karin M, Janssens Hettie M, Bronsveld Inez, van der Ent Cornelis K, de Jonge Hugo R, Beekman Jeffrey M
Abstract excerpt
The potentiator VX-770 (ivacaftor/KALYDECO™) targets defective gating of CFTR and has been approved for treatment of cystic fibrosis (CF) subjects carrying G551D, S1251N or one of 8 other mutations. Still, the current potentiator treatment does not normalize CFTR-dependent biomarkers, indicating the need for development of more effective potentiator strategies. We have recently pioneered a functional CFTR assay...
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