Study Describes New Compound for Nonsense Mutations

Study Describes New Compound for Nonsense Mutations

300044

Study Describes New Compound for Nonsense Mutations

A novel compound that may hold promise for treating the roughly 11% of cases of cystic fibrosis (CF) that are caused by nonsense mutations has been described in a new study. The study, “A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1 depletion,” was published in the journal Nature Communications. CF is caused by mutations in a gene called CFTR. Mutations in this gene lead to dysfunction of the protein that the…

You must be logged in to read/download the full post.