Figure 1.

Transformative clinical potential of tRNA medicines.A, a single tRNA medicine can readthrough a shared genetic mutation, such as a premature termination codon, across different genes independent of the mutation location along the transcript. B, the clinical impact of this modality feature enables a novel grouping of patients, not based on their clinical presentation or on the identity of the gene mutated as is traditionally done, but based on the underlying mutation patients carry. GSD, glycogen storage disease; HEM A, hemophilia A; HEM B, hemophilia B; MMA, methylmalonic aciduria; OTC, ornithine transcarbamylase; PKU, phenylketonuria.