Importance Cystic fibrosis, a genetic disorder defined by variants in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene, affects more than 30 000 individuals in
the US and approximately 89 000 worldwide. Absent or decreased function of the CFTR
protein is associated with multiorgan dysfunction and shortened life expectancy.
Observations CFTR is an anion channel in the apical membrane of epithelial cells. Loss of
function leads to obstructed exocrine glands. Of people with cystic fibrosis in the US …