Cystic fibrosis (CF) is an inherited disorder that damages your lungs and digestive system. CF affects the cells of the body that produce mucus. These fluids are meant to lubricate the body and are typically thin and slick. CF makes these bodily flui... Read more

Cystic fibrosis (CF) is an inherited disorder that damages your lungs and digestive system. CF affects the cells of the body that produce mucus. These fluids are meant to lubricate the body and are typically thin and slick. CF makes these bodily fluids dense and sticky, which causes them to build up in the lungs, airways, and digestive tract. While advancements in research have vastly improved the quality of life and life expectancy of people with CF, most will need to treat the condition for their entire lives. Currently, there's no cure for CF, but researchers are working toward one. Cystic fibrosis (CF) is also an autosomal recessive, monogenetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The gene defect was first described 25 years ago and much progress has been made since then in our understanding of how CFTR mutations cause disease and how this can be addressed therapeutically. CFTR is a transmembrane protein that transports ions across the surface of epithelial cells. CFTR dysfunction affects many organs; however, lung disease is responsible for the vast majority of morbidity and mortality in patients with cystic fibrosis. Prenatal diagnostics, new-born screening and new treatment algorithms are changing the incidence and the prevalence of the disease. Until recently, the standard of care in cystic fibrosis treatment focused on preventing and treating complications of the disease; now, novel treatment strategies directly targeting the ion channel abnormality are becoming available and it will be important to evaluate how these treatments affect disease progression and the quality of life of patients. Less