Cystic fibrosis: a complex disease and a paradigm for biomedical research]

Cystic fibrosis (CF) is a genetic disease, particularly frequent among northern Europeans and northern Americans which affects, often lethally, the function of exocrine organs (including the respiratory, digestive, genital and cutaneous mucosae). Despite its pioneering role in areas as diverse as genetics, physiology or pharmacology, CF research still faces essential unsolved questions. In particular, the factors involved in the appearance and severity of CF airway disease remain mostly unraveled so far. This review aims at presenting current views on the pathophysiological processes involved in CF, whether these views are firmly demonstrated or still hypothetical. The various experimental models, central to CF research, are also examined. Finally, emerging therapeutic options for CF (e.g. gene and protein therapy), which raise many hopes among patients, are described.