Introduction to section IV: biophysical methods to approach CFTR structure.
Introduction to section IV: biophysical methods to approach CFTR structure.
复制标题
第四节介绍：研究 CFTR 结构的生物物理方法。
DOI：
10.1007/978-1-61779-117-8_21
复制
发表时间：
2011
期刊：
影响因子：
--
通讯作者：
Thomas,PhilipJ
中科院分区：
文献类型：
--
作者：
Mendoza,JuanL;Schmidt,André;Thomas,PhilipJ
CFTR 低效折叠成功能性三维结构是导致大多数囊性纤维化病例的基本病理生理机制。了解 CFTR 的结构以及将这些突变置于结构背景中将为开发囊性纤维化的靶向治疗方法提供关键信息。作为一种含有无序区域的大型多面体膜蛋白，完整的 CFTR 一直难以使用 X 射线晶体学解析高分辨率结构。以下章节总结了当前通过利用 NMR、电子显微镜和计算方法以及开发 CFTR 相关领域的实验模型来规避这些障碍的努力。
Inefficient folding of CFTR into a functional three-dimensional structure is the basic pathophysiologic mechanism leading to most cases of cystic fibrosis. Knowledge of the structure of CFTR and placement of these mutations into a structural context would provide information key for developing targeted therapeutic approaches for cystic fibrosis. As a large polytopic membrane protein containing disordered regions, intact CFTR has been refractory to efforts to solve a high-resolution structure using X-ray crystallography. The following chapters summarize current efforts to circumvent these obstacles by utilizing NMR, electron microscopy, and computational methodologies and by development of experimental models of the relevant domains of CFTR.