A small molecule induces readthrough of cystic fibrosis CFTR nonsense mutations

(University of Alabama at Birmingham) 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 are also caused by nonsense mutations.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news