An introduction to RNA therapeutics and their potentials

核糖核酸 适体 计算生物学 小干扰RNA 寡核苷酸 信使核糖核酸 生物 核酸结构 基因 遗传学
作者
Nhat Le Bui,Dinh‐Toi Chu
出处
期刊:Progress in Molecular Biology and Translational Science [Academic Press]
标识
DOI:10.1016/bs.pmbts.2023.12.020
摘要

RNA therapeutics is a biological term regarding the usage of RNA-based molecules for medical purposes. Thanks to the success of mRNA-vaccine production against COVID-19, RNA therapeutics has gained more and more attention and investigation from worldwide scientists. It is considered as one of the promising alternatives for conventional drugs. In this first chapter, we presented an overview of the history and perspectives of RNA therapeutics' development. This chapter also explained the underlying mechanisms of different RNA-based molecules, including antisense oligonucleotide, interfering RNA (iRNA), aptamer, and mRNA, from degrading mRNA to inactivating targeted protein. Although there are many advantages of RNA therapeutics, its challenges in designing RNA chemical structure and the delivery vehicle need to be discussed. We described advanced technologies in the development of drug delivery systems that are positively correlated to the efficacy of the drug. Our aim is to provide a general background of RNA therapeutics to the audience before introducing plenty of more detailed parts, including clinical applications in certain diseases in the following chapters of the "RNA therapeutics" book.
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