Potentiator ivacaftor abrogates pharmacological correction of DF508 CFTR in cystic fibrosis

, 246ra96 (2014); 6 Sci Transl Med et al. Deborah M. Cholon CFTR in cystic fibrosis F508 ∆ Potentiator ivacaftor abrogates pharmacological correction of Editor's Summary potentiators make CFTR less stable, accelerating the removal of this channel from the cell membrane. and Veit and coauthors now show that combining the two types of drugs does not work effectively, because surface. Other drugs, called ''correctors,'' help bring mutant CFTR to the cell surface, but two manuscripts by Cholon cell ''potentiator,'' which means that it improves the activity of mutant CFTR but cannot work if there is no CFTR on the CF, but it is not effective enough to treat the severest and most common form of this disease. Ivacaftor is a (CFTR) ion channel, resulting in pulmonary and other complications. Ivacaftor is the only targeted drug approved for Cystic fibrosis (CF) is a genetic disease caused by mutations of the CF transmembrane conductance regulator Potentiating Trouble