囊性纤维化跨膜传导调节因子(CFTR)是一个重要的离子通道，其功能丧失导致囊性纤维化，而其过度激活导致分泌性腹泻。改善CFTR折叠(校正剂)或功能(增强剂)的小分子在临床上可用。然而，唯一的增强剂ivacaftor具有次优的药代动力学，抑制剂尚未临床开发。

Apical localisation of CFTR protein and its chloride channel activity strictly depend on interactions of its PDZ (for postsynaptic density 95/disclarge/zona occludens)-interacting domain in the C ...

New research findings add further clarity to a question that has polarized the cystic fibrosis (CF) research community. It is well established that people with cystic fibrosis have two faulty copies ...

Cystic fibrosis is a common life-shortening inherited disease that affects over 70,000 people worldwide, the majority of whom are children and young adults. Individuals living with cystic fibrosis ...

January 12, 2016 CHAPEL HILL, NC - It is well established that people with cystic fibrosis (CF) have two faulty copies of the CFTR gene, but debate continues on the question of whether certain ...

Researchers studying the root cause of cystic fibrosis have made a major advance in our understanding of silent gene changes with implications for the complexity of cystic fibrosis. Cystic fibrosis is ...

Researchers studying the root cause of cystic fibrosis have made a major advance in our understanding of silent gene changes with implications for the complexity of cystic fibrosis. The findings are ...

Mutation of one gene is all it takes to get cystic fibrosis (CF), but disease severity depends on many other genes and proteins. For the first time, researchers at the UNC School of Medicine have ...