Recent advances in basic science have greatly expanded our understanding of the cystic
fibrosis (CF) transmembrane conductance regulator (CFTR), the chloride and bicarbonate
channel that is encoded by the gene, which is mutated in patients with CF. We review the
structure, function, biosynthetic processing, and intracellular trafficking of CFTR and discuss
the five classes of mutations and their impact on the CF phenotype. The therapeutic
discussion is focused on the significant progress toward CFTR mutation-specific therapies …