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RNA 疗法的历史与未来展望。

RNA therapeutics history and future perspectives.

机构信息

Hanoi University of Public Health, Hanoi, Vietnam.

Yenphong Medical Center, Bacninh, Vietnam.

出版信息

Prog Mol Biol Transl Sci. 2024;203:99-114. doi: 10.1016/bs.pmbts.2024.01.004. Epub 2024 Jan 24.

Abstract

Ribonucleic acid (RNA) therapeutics have significantly used RNA-based drugs to the prevention and treatment of diseases as effective messenger RNA-based vaccines in response to the COVID-19 pandemic. The RNA therapeutics with five classes including antisense oligonucleotide, small interfering RNA, microRNA, APTAMER and messenger RNAs are being quickly developed to treat various human diseases as neurological disease, cardiovascular disease, genetic and rare disease, cancer disease, coronavirus disease… which cannot be treated by other conventional drugs as small molecule-based drugs and antibodies. Therefore, the discovery of these RNA therapeutics created a new avenue for treatment of various human diseases. This chapter demonstrates the history of important discoveries in RNA biology and their impact on key developments in RNA therapeutics as well as the advantages of RNA therapeutics; RNA therapeutics describes the action mechanisms and examples of RNA-based drugs approved for treatment of various disease; and RNA therapeutics discusses delivery methods for RNA therapeutics to target organs and cells. In conclusion, this chapter is designed to offer an updated important development and advance of RNA therapeutics for the prevention and treatment of various human diseases.

摘要

核糖核酸(RNA)疗法已将基于 RNA 的药物大量用于疾病的预防和治疗,例如针对 COVID-19 大流行的有效信使 RNA 疫苗。正在快速开发 RNA 疗法的五个类别,包括反义寡核苷酸、小干扰 RNA、microRNA、适配体和信使 RNA,以治疗各种人类疾病,如神经疾病、心血管疾病、遗传和罕见疾病、癌症疾病、冠状病毒疾病……这些疾病无法通过其他传统药物(如基于小分子的药物和抗体)治疗。因此,这些 RNA 疗法的发现为治疗各种人类疾病开辟了新途径。本章展示了 RNA 生物学的重要发现历史及其对 RNA 疗法关键发展的影响,以及 RNA 疗法的优势;RNA 疗法描述了已批准用于治疗各种疾病的基于 RNA 的药物的作用机制和实例;并讨论了 RNA 疗法的递药方法,以靶向器官和细胞。总之,本章旨在提供有关 RNA 疗法在预防和治疗各种人类疾病方面的最新重要发展和进展。

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