A Small Molecule Induces Readthrough Of Cystic Fibrosis CFTR Nonsense Mutations
An experimental drug reported in Nature Communications suggests that a “path is clearly achievable” to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations. This includes about 11 percent of cystic fibrosis patients, as well as patients with other genetic diseases, including Duchenne muscular dystrophy, β-thalassemia and numerous types of cancers, that …