Cystic fibrosis two-step

A Canadian and a U.S. team have independently shown that restoring normal function to mutant CFTR requires correcting two distinct folding steps.^1,2 The findings could provide a road map to guide the rational development of more effective therapies to treat cystic fibrosis.

CF is caused by genetic mutations in the cystic fibrosis transmembrane conductance regulator (CFTR), an ion channel that helps keep the lung epithelium hydrated and prevents mucus buildup that leads to airway obstruction and infection...