Engineered tRNAs suppress nonsense mutations in cells and in vivo. Engineered tRNAs suppress nonsense mutations in cells and in vivo

we introduce a strategy to repurpose sense-codon decoding tRNA into efficient suppressors of the three nonsense mutation-induced PTCs (UGA, UAG and UAA). The suppressor tRNAs restore function of a model and disease-related protein Overall design: Suppressor tRNAs are administered both in mice (intraveneous in liver or intratracheal in lung) or in immortalized cell culture and tissues/cells are collected and subjected to ribosome profiling (Ribo-Seq)