Bui Nhat-Le, Chu Dinh-Toi
Center for Biomedicine and Community Health, International School, Vietnam National University, Hanoi, Vietnam; Faculty of Applied Sciences, International School, Vietnam National University, Hanoi, Vietnam.
Center for Biomedicine and Community Health, International School, Vietnam National University, Hanoi, Vietnam; Faculty of Applied Sciences, International School, Vietnam National University, Hanoi, Vietnam.
Prog Mol Biol Transl Sci. 2024;203:1-12. doi: 10.1016/bs.pmbts.2023.12.020. Epub 2024 Jan 25.
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.
RNA疗法是一个生物学术语,指将基于RNA的分子用于医学目的。得益于针对新冠病毒的mRNA疫苗生产的成功,RNA疗法已越来越受到全球科学家的关注和研究。它被视为传统药物有前景的替代方案之一。在第一章中,我们概述了RNA疗法发展的历史和前景。本章还解释了不同基于RNA的分子的潜在机制,包括反义寡核苷酸、干扰RNA(iRNA)、适体和mRNA,从降解mRNA到使靶向蛋白失活。尽管RNA疗法有许多优点,但在设计RNA化学结构和递送载体方面的挑战仍需讨论。我们描述了与药物疗效呈正相关的药物递送系统开发中的先进技术。我们的目的是在介绍“RNA疗法”这本书后续章节中包括某些疾病的临床应用等大量更详细内容之前,为读者提供RNA疗法的一般背景知识。