Cystic fibrosis (CF) is a genetic disease that is caused by mutations of the gene encoding for Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein. Mutations in the CFTR gene lead to impaired or loss of protein function over time resulting in severe damage to lungs, digestive system, and other organs in the body. A major problem in treating CF is the diversity of the genetic defect. Over 2000 different mutations have been identified in CF patients: just 12 of these are represented in 50% of the CF population, with more than 1900 mutations distributed amongst the other 50%.


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