Researchers at the University of Toronto developed an engineered form of transfer RNA, or tRNA, that helps cells read past premature stop signals in genetic instructions.
In tests involving cystic fibrosis, the approach helped cells make a full-length CFTR protein instead of stopping early. The researchers saw the restored protein in human airway cells, mouse models and patient-derived organoids.
The team also tested the RNA alongside an existing cystic fibrosis medicine. In the patient-derived organoids, the combination produced a response when either treatment alone had little effect.
The study was published in Science on August 27. The work is still early, and researchers say they must develop reliable delivery systems for different organs before the approach could become a treatment.
