Engineered tRNA Restores Protein in Cystic Fibrosis Cells

University of Toronto researchers used chemically modified tRNA delivered via lipid nanoparticles to bypass nonsense mutations and restore full-length CFTR protein in cells from a cystic fibrosis patient who did not respond to existing drugs. Combined with Trikafta, the cells responded, and the protein persisted for over 40 days in human airway cells.

• Nonsense mutations cause about 11% of inherited genetic disorders
• Restored CFTR protein persisted over 40 days in airway cells
• tRNA was delivered with lipid nanoparticles redesigned for the cargo
• The study was published in the journal Science