This new approach targets disorders including cystic fibrosis, muscular dystrophy, and some cancers. In lab-grown tissue from a cystic fibrosis patient who did not respond to existing drugs, researchers restored production of a full-length protein and gave Trikafta something it could act on. The patient’s cells responded when the drug was combined with engineered transfer [...]
Researchers at the University of Toronto have developed a new therapeutic approach that aims to treat various genetic diseases, including cystic fibrosis and muscular dystrophy. The technique uses modified transfer RNA (tRNA) to bypass mutations in...