Rescue of multiple class II CFTR mutations by elexacaftor+tezacaftor+ivacaftor mediated in part by the dual activities of elexacaftor as both corrector and potentiator

• Published in: The European respiratory journal Vol. 57; no. 6; p. 2002774
• Main Authors: Laselva, Onofrio, Bartlett, Claire, Gunawardena, Tarini N.A., Ouyang, Hong, Eckford, Paul D.W., Moraes, Theo J., Bear, Christine E., Gonska, Tanja
• Format: Journal Article
• Language: English
• Published: England European Respiratory Society 01.06.2021
• Subjects: Aminophenols; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator - genetics; Drug Combinations; HEK293 Cells; Humans; Indoles; Mutation; Original; Pyrazoles; Pyridines; Pyrrolidines; Quinolines; Quinolones

Positive results in pre-clinical studies of the triple combination of elexacaftor, tezacaftor and ivacaftor, performed in airway epithelial cell cultures obtained from patients harbouring the class II cystic fibrosis transmembrane conductance regulator (CFTR) mutation F508del-CFTR, translated to imp...