Systems pharmacology of cystic fibrosis transmembrane regulator correctors and potentiators

囊性纤维化是由囊性纤维化跨膜传导调节剂活性缺陷或不足引起的限制生命的疾病。最近批准的Lumacaftor联合Ivacaftor靶向70%的F508 del-CFTR CF患者。不幸的是，由于我们对其药理学的了解有限，这些一流药物的临床疗效受到限制。这一创新的系统药理学项目将在改善全球临床实践方面具有重大潜力。

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.