Novel gene therapy approach creates new route to tackle rare, inherited diseases

A new study reveals a novel approach and robust technology platform for suppressing nonsense mutations using engineered transfer RNA (tRNA) molecules. The research shows that modified tRNAs can efficiently and accurately repair nonsense mutations with any amino acid. The findings by researchers at the University of Iowa, The Wistar Institute, the Cystic Fibrosis Foundation Therapeutics Lab, and Integrated DNA Technologies Inc., were published Feb. 18, 2019 in Nature Communications.