Transfer RNAs (tRNAs) play a significant role in protein synthesis. Their potential to treat genetic diseases has gained much attention. tRNAs function at specific sites in the ribosome, the cellular machinery that makes and synthesizes a protein from mRNA molecules. In the sequencing process, tRNAs link the genetic code with the requisite amino acids that together form the polypeptide chains that make up proteins.

Research and development in tRNA therapeutics continue. For example, researchers are still trying to decipher how the structures of tRNA molecules influence molecular stability. Understanding tRNA dynamics is important, as it gives researchers a better understanding of these diseases.

RNA medicines often target specific gene sequences, and can be effective against rare diseases such as sickle cell, cystic fibrosis, hemophilia, Duchenne muscular dystrophy, and Ehlers-Danlos Syndrome. Notably, tRNA therapies can be deployed to treat people based on shared genetic mutations rather than their specific disease phenotypes, including many common and many rare disorders.