The epithelium as a target for therapy in cystic fibrosis

Cystic fibrosis (CF) is characterized by a solute transport defect in epithelial tissues. In the lungs, this defect culminates in the dehydration of the airway surface and mucus accumulation, ultimately leading to chronic bacterial infection. To date, the current therapeutic approaches used to treat CF primarily focus on the secondary manifestations of the disease (e.g. bacterial infection, viscous mucus). However, new therapeutic approaches are targeting the underlying ion transport defect in cystic fibrosis, with the aim of restoring the function of the cystic fibrosis transmembrane conductance regulator, stimulating alternative chloride channels, inhibiting sodium absorption, and utilizing hyperosmotic agents to rehydrate the airway surface. Although still in the development phase, these approaches, used by themselves or in combination, show great promise in the treatment of CF.