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A small molecule induces readthrough of cystic fibrosis CFTR nonsense mutations

An experimental drug 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.

from Latest Science News -- ScienceDaily https://www.sciencedaily.com/releases/2021/07/210719110521.htm
A small molecule induces readthrough of cystic fibrosis CFTR nonsense mutations A small molecule induces readthrough of cystic fibrosis CFTR nonsense mutations Reviewed by cmakigo on July 19, 2021 Rating: 5

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