Tasarlanmış tRNA hatalı dur sinyallerinin aşılmasına yardım ediyor

About one in ten cystic fibrosis cases involves a nonsense mutation that inserts a premature stop signal into the instructions for making CFTR protein. Current medicines do not adequately help this group. An NIH-funded team engineered suppressor transfer RNA, or sup-tRNA, to recognise a faulty signal and insert the missing amino acid so protein production could continue. A chemical modification made the molecule more active and stable while reducing immune activation. Researchers then screened roughly 1,000 lipids to identify a nanoparticle carrier capable of delivering the cargo without a virus. In mice, an inhaled formulation reached lung epithelial cells and increased full-length CFTR production. In intestinal organoids derived from a patient, sup-tRNA combined with the approved drug Trikafta significantly improved CFTR function, although neither treatment worked alone in that model. The approach does not alter DNA, but these preclinical results still require human safety and effectiveness studies.