CFTR Modulators Small Molecules and Peptides

The cystic fibrosis transmembrane conductance regulator (CFTR) is a 12-transmembrane ATP-binding cassette (ABC) transporter that functions as a cAMP-dependent chloride channel. It is localized to the apical membrane of epithelial cells within the lungs, liver, pancreas, digestive tract, reproductive tract and skin, where it is involved in fluid transport.

CFTR is composed of five domains: two membrane-spanning domains (MSD1, MSD2) that function as a chloride channel; two nucleotide-binding ...