Missense suppressor tRNA therapeutics correct disease-causing alleles by misreading the genetic code

In all living cells, transfer RNAs (tRNAs) act as adaptor molecules, linking the nucleic acid sequences of gene products to the accurate production of proteins by the ribosome. Translation fidelity relies on accurate aminoacylation of tRNAs and faithful decoding of each codon through interactions with the anticodon of a cognate aminoacyl-tRNA. Although translation is a high-fidelity process, cells are tolerant to significantly elevated levels of mistakes in protein synthesis.1