IMPMC, Sorbonne Universités - UPMC Université Paris 06, UMR CNRS 7590, Muséum National d'Histoire Naturelle, IRD UMR 206, 4 Place Jussieu, Paris, France.
Molecular Medicine, The Hospital for Sick Children, Toronto M5G 0A4, Canada.
J Cyst Fibros. 2018 Mar;17(2S):S5-S8. doi: 10.1016/j.jcf.2017.08.008. Epub 2017 Aug 31.
Structural studies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein are critical to understand molecular mechanisms involved in gating of the apical anion channel as well as the way in which the gating is regulated, especially by the regulatory region (R region). They are also instrumental for understanding the root cause of cystic fibrosis (CF) and supporting the development of therapeutic strategies. In this short review, we summarize recent progress in the knowledge of the CFTR 3D structure and briefly discuss implications for CF drug development.
囊性纤维化跨膜电导调节因子（CFTR）蛋白的结构研究对于理解顶端阴离子通道门控的分子机制以及门控调节方式（特别是通过调节区域[R 区域]）至关重要。这些研究对于理解囊性纤维化（CF）的根本原因以及支持治疗策略的发展也具有重要意义。在这篇简短的综述中，我们总结了 CFTR 三维结构知识的最新进展，并简要讨论了其对 CF 药物开发的影响。
