CFTR: New insights into structure and function and implications for modulation by small molecules
CFTR: New insights into structure and function and implications for modulation by small molecules
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DOI：
10.1016/j.jcf.2019.10.021
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发表时间：
2020-03-01
影响因子：
5.2
通讯作者：
Sheppard, David N.
中科院分区：
文献类型：
--
作者：
Kleizen, Bertrand;Hunt, John F.;Sheppard, David N.
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) 基础知识的有力组合。在这里，我们讨论最新的发现，包括临床批准的药物如何恢复突变型 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 靶向疗法有望获得监管部门批准，但仍需要付出艰苦的努力来充分理解 CFTR 的结构和功能，以开发更好的药物，使 CF 患者能够过上充实而积极的生活。 (C) 2019 年由 Elsevier B.V. 代表欧洲囊性纤维化协会发布。
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. (C) 2019 Published by Elsevier B.V. on behalf of European Cystic Fibrosis Society.