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RNA therapy is defined as the use of RNA-based molecules to prevent or treat disease. As recent research has shown, the field of RNA therapy has made significant advances. In particular, the ability to target diverse genetic materials in the body and the rapid development of drugs is why we can expect to see more research efforts focused on developing RNA-based therapeutics in the future. In this special issue, we have selected some of the most important topics related to RNA therapy. We cover the latest microRNA (miRNA) therapeutics in clinical trials, the latest efforts to deliver small interfering RNAs (siRNAs) to diverse organs, the disease association of RNA fragments derived from transfer RNAs (tRNAs), and the immune response induced by messenger RNA (mRNA) vaccines, which has received much attention. The combination of research in these different areas will enable us to overcome a range of diseases that were previously difficult to treat or prevent.