CYSTIC FIBROSIS

 

Cystic Fibrosis (CF), a genetic disease, is caused by dysfunction of the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein, which acts as a cyclic adenosine monophosphate-regulated chloride channel at the apical membrane of epithelial cells.

 

Around 50,000 European citizens suffer from this disease. Life expectancy progressed significantly last decades but remains less than 30 years.

 

Absence of normally functioning CFTR in the apical membrane of the cell, results in viscous secretions in different exocrine tissues, mainly the respiratory tract, pancreas, gastrointestinal tract, sweat glands. Increased viscosity of these secretions makes them difficult to clear and patients develop progressively exocrine gland dysfunction of multiple organ systems in childhood, resulting in chronic respiratory disease as well as other pathologies including pancreatic insufficiency,…