Division of Respiratory Medicine, Hospital for Sick Children, 555 University Avenue, Toronto, Ontario M5G 1X8, Canada.
Curr Pharm Des. 2012;18(5):614-27. doi: 10.2174/138161212799315984.
Cystic fibrosis is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that lead to abnormalities in transepithelial ion transport in the airways of affected patients. Lung disease is the major contributor to morbidity and mortality in patients with cystic fibrosis but recommended therapeutic interventions so far have focussed on symptom control rather than treatment of the underlying causes of the disease. New therapies that are currently in pre-clinical and clinical testing include CFTR pharmacotherapy, drugs targeting other ion channels, and hydrators of the cystic fibrosis airways. The current status of these and other new developments in the treatment of cystic fibrosis are reviewed.
囊性纤维化是由囊性纤维化跨膜电导调节因子 (CFTR) 基因突变引起的，导致受影响患者气道上皮细胞间离子转运异常。肺部疾病是导致囊性纤维化患者发病率和死亡率的主要原因，但迄今为止推荐的治疗干预措施主要集中在控制症状上，而不是针对疾病的根本原因进行治疗。目前处于临床前和临床测试阶段的新疗法包括 CFTR 药物治疗、针对其他离子通道的药物以及囊性纤维化气道的保湿剂。本文综述了这些疗法以及囊性纤维化治疗中其他新进展的现状。
