CFTR: New insights into structure and function and implications for modulation by small molecules

Bertrand Kleizen*, John F. Hunt, Isabelle Callebaut, Tzyh Chang Hwang, Isabelle Sermet-Gaudelus, Sylvia Hafkemeyer, David N. Sheppard

*此作品的通信作者

研究成果: Article同行評審

8 引文 斯高帕斯(Scopus)

摘要

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.

原文English
頁(從 - 到)S19-S24
期刊Journal of Cystic Fibrosis
19
DOIs
出版狀態Published - 3月 2020

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