Combinations of CF correctors from McGill could rescue more mutations

Researchers from McGill University discovered three novel correctors of cystic fibrosis transmembrane conductance regulator (CFTR) and showed that a combination of the small molecules could correct the most common CF mutation, F508del, as well as additional folding mutations not targeted by available CF therapies.

In a Nature Medicine paper, the researchers used biochemical screens to identify three small molecules that each correct a different component of the structural defects of the F508del CFTR mutation. In human epithelial nasal cells, the triple combination led to complete biochemical restoration of the F508del mutation and about 48% functional restoration compared with wild-type levels...