RNA Drugs

Abstract

RNA drugs are a new group of drugs that delivers RNAs or similar structures inside the body to achieve the therapeutic effect. This is a promising direction in drug development to treat serious and rare genetic diseases more specifically and effectively. In reality, the genetic systems and protein synthesis processes of living organisms are extremely complex, so the development of RNA drugs faces many difficulties. To achieve success, many different studies have been carried out to address issues such as finding suitable RNAs, synthesizing similar RNA structures, stabilizing RNA structures, and introducing drugs into targeted cells. Since the first RNA drug was officially approved by the FDA (2004), 10 RNA drugs in total have been approved to date. Among them, two vaccines, appearing at the time when much needed support to cope with the new SARS-CoV-2 variants, were developed using mRNA technology. With these achievements, scientists can have more confidence in the possibilities of evolving a new drug group that is more specific and effective, which is RNA drugs. This review briefly introduces the group of drugs that use RNAs, RNA structural analogs, and RNA biomarkers to develop novel drugs for application in the diagnosis, prevention, and treatment of disease. Keywords: RNA drugs; mRNA; the protein; vaccines; RNA diagnostics; small molecule drugs; RNA target. References [1] U. Sahin, K. Karikó, Ö. Türeci, Mrna-Based Therapeutics-Developing A New Class of Drugs, Nature Reviews Drug Discovery, Vol. 13, No. 10, 2014, pp. 759-780.[2] T. H. Nguyen, T. M. H. Pham, M. K. Tu, Pharmacogenetics: Prospects and Issues. Journal of Pharmacy, No. 54, Vol. 456, 2014, pp. 2-6.[3] A. M. Yu, Y. H. Choi, M. J. Tu, Rna Drugs and Rna Targets for Small Molecules: Principles, Progress, and Challenges, Pharmacological Reviews, Vol. 72, No. 4, 2020, pp. 862-898.[4] M. A. Hendaus, F. A. 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