Messenger RNA Therapeutics: Start of a New Era in Medicine

Abstract

Last decade has witnessed tremendous growth in the new promising treatment options based on mRNA, RNAi, antisense RNA, and RNA aptamers, the four classes of RNA-based therapeutics. Among these, mRNA-based therapy is centered on producing proteins within the cells to supplant deficient or abnormal proteins and in vaccination to a target pathogen. The potential of mRNA therapeutics is evident from the two major mRNA vaccines approved for COVID-19: developed by Moderna and by Pfizer. Nonetheless, mRNA therapeutic potential extends far beyond this, such as in treating genetic diseases, cancers, and other infectious diseases. Given the potential of mRNA therapeutics, this chapter is written to provide the reader an insight into the features of several synthetic mRNA platforms, production, purification; strategies to increase the stability and reduce the immunogenicity of therapeutic mRNA molecules; delivery methods of these mRNAs in vivo; and their applications, safety, and efficacy. Graphical abstract: A detailed diagram of the chemically modified mRNA, with the in vitro delivery modes on the left, and the myriad of applications, namely the treatment of major genetic diseases on the right. The IVT mRNA is represented with more details above the diagram.