Systems pharmacology of cystic fibrosis transmembrane regulator correctors and potentiators

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Cystic fibrosis is a life limiting disease caused by defective or deficient cystic fibrosis trans-membrane conductance regulator activity. The recent approval of lumacaftor combined with ivacaftor targets 70% of CF patients with F508del-CFTR. Unfortunately, the clinical efficacy of these first-in-class drugs is limited by our limited understanding of their pharmacology. This innovative systems pharmacology project will have a significant potential in improving clinical practice worldwide.