Thelin William R, Boucher Richard C
Cystic Fibrosis/Pulmonary Research and Treatment Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Curr Opin Pharmacol. 2007 Jun;7(3):290-5. doi: 10.1016/j.coph.2007.01.004. Epub 2007 May 1.
Cystic fibrosis (CF) is characterized by a solute transport defect in epithelial tissues. In the lungs, this defect culminates in the dehydration of the airway surface and mucus accumulation, ultimately leading to chronic bacterial infection. To date, the current therapeutic approaches used to treat CF primarily focus on the secondary manifestations of the disease (e.g. bacterial infection, viscous mucus). However, new therapeutic approaches are targeting the underlying ion transport defect in cystic fibrosis, with the aim of restoring the function of the cystic fibrosis transmembrane conductance regulator, stimulating alternative chloride channels, inhibiting sodium absorption, and utilizing hyperosmotic agents to rehydrate the airway surface. Although still in the development phase, these approaches, used by themselves or in combination, show great promise in the treatment of CF.
囊性纤维化（CF）的特征是上皮组织中的溶质转运缺陷。在肺部，这种缺陷最终导致气道表面脱水和黏液积聚，最终引发慢性细菌感染。迄今为止，用于治疗CF的现有治疗方法主要集中在该疾病的继发表现（如细菌感染、黏稠黏液）上。然而，新的治疗方法正针对囊性纤维化潜在的离子转运缺陷，目的是恢复囊性纤维化跨膜传导调节因子的功能、刺激其他氯离子通道、抑制钠吸收，并利用高渗剂为气道表面补充水分。尽管仍处于研发阶段，但这些方法单独使用或联合使用，在CF治疗中都显示出了巨大的前景。
