Early research shows a one-time gene therapy has potential for addressing both the liver and lung disease that patients with ...

By creating a special compound that binds FBXW7-R465C, the Northwestern team was able to direct this mutant protein to accelerate degradation of other tumor-promoting proteins. Only the mutated ...

Every person has two copies of the hemoglobin gene. Usually, both genes make a normal hemoglobin protein. When someone inherits two mutant copies of the hemoglobin gene, the abnormal form of the ...

The author is looking forward to seeing how Beam Therapeutics would do in their clinical trials of single-base DNA editing.