CFTR

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators act on an epithelial chloride channel. The CFTR gene was cloned in 1989, and its most common disease-associated variant deletes phenylalanine 508 (F508del). Potentiators raise the open probability of the channel, as reported in 2009 for ivacaftor under the code VX-770. Correctors such as lumacaftor and VX-661 improve folding and trafficking of F508del-CFTR to the plasma membrane. Inhibitors serve to probe channel function, among them CFTRinh-172, a thiazolidinone described in 2002, and the quinoxalinedione PPQ-102. Ataluren promotes readthrough of premature stop codons and is used with nonsense variants of the gene. Other chloride-conducting proteins are listed under the sibling category Chloride Channels.