
With newer tools for gene editing, is it time to revisit genetic therapy for cystic fibrosis?
Cystic Fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene on chromosome 7. These abnormalities affect the chloride channels in the mucus producing epithelial cells. With these channels not working properly, water is unable to leave the cells to enter the mucus; thus, leading to sticky mucus which obstructs airways and ducts in the body.