Cystic fibrosis (CF) is a serious, potentially fatal inherited disease resulting from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which encodes an ion channel protein that helps regulate the flow of chloride ions and water in and out of epithelial cells lining the lungs, pancreas and other organs.

These CFTR mutations prevent the protein from functioning normally and ultimately lead to a buildup of mucus in the lungs and airways, impaired pancreatic secretion and other aspects of CF pathology that compromise patient health.