Cellular Protein Chemistry, Department of Chemistry, Utrecht University, Utrecht, the Netherlands.
Department of Biological Sciences, Columbia University, New York, NY, USA.
J Cyst Fibros. 2020 Mar;19 Suppl 1:S19-S24. doi: 10.1016/j.jcf.2019.10.021. Epub 2019 Nov 21.
Structural biology and functional studies are a powerful combination to elucidate fundamental knowledge about the cystic fibrosis transmembrane conductance regulator (CFTR). Here, we discuss the latest findings, including how clinically-approved drugs restore function to mutant CFTR, leading to better clinical outcomes for people with cystic fibrosis (CF). Despite the prospect of regulatory approval of a CFTR-targeting therapy for most CF mutations, strenuous efforts are still needed to fully comprehend CFTR structure-and-function for the development of better drugs to enable people with CF to live full and active lives.
结构生物学和功能研究是阐明囊性纤维化跨膜电导调节器（CFTR）基本知识的有力组合。在这里，我们讨论了最新的发现，包括临床批准的药物如何恢复突变 CFTR 的功能，从而为囊性纤维化（CF）患者带来更好的临床结果。尽管针对大多数 CF 突变的 CFTR 靶向治疗有获得监管批准的前景，但仍需要付出巨大努力，以充分理解 CFTR 的结构和功能，从而开发出更好的药物，使 CF 患者能够过上充实和积极的生活。
