Progress towards next-generation therapeutics for cystic fibrosis

Cystic fibrosis (CF) is the most common inherited genetic condition amongst Caucasian ethnicities, affecting 1 in 2500 live births. There remains a significant unmet medical need for more and better therapies for this chronic, degenerative condition, in particular those that address the respiratory dysfunction and respiratory infections that characterise CF. CF is caused by mutations in the cystic transmembrane conductance regulator gene (CFTR). The key pathology driver of CF is dysregulated ion transport across the epithelial cell barriers that line the respiratory tract, gastrointestinal tract and other organ systems. This review focuses on the state-of-the-art advances and future directions in therapeutic strategies to combat and manage the symptoms of CF and/or restore functionality of the defective CFTR.