RNA 疗法及其潜力简介。

3区 生物学 Q2 Biochemistry, Genetics and Molecular Biology
Nhat-Le Bui, Dinh-Toi Chu
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引用次数: 0

摘要

RNA 疗法是将 RNA 分子用于医疗目的的生物学术语。由于针对 COVID-19 的 mRNA 疫苗的成功生产,RNA 疗法得到了全世界科学家越来越多的关注和研究。它被认为是有希望替代传统药物的药物之一。在第一章中,我们概述了 RNA 疗法的发展历史和前景。本章还解释了不同 RNA 分子(包括反义寡核苷酸、干扰 RNA (iRNA)、适配体和 mRNA)从降解 mRNA 到灭活靶蛋白的基本机制。虽然 RNA 疗法有很多优点,但其在设计 RNA 化学结构和递送载体方面的挑战也需要讨论。我们介绍了与药物疗效正相关的药物输送系统开发方面的先进技术。我们的目的是向读者介绍 RNA 疗法的一般背景,然后在《RNA 疗法》一书的后续章节中介绍更详细的内容,包括某些疾病的临床应用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
An introduction to RNA therapeutics and their potentials.

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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来源期刊
CiteScore
5.00
自引率
0.00%
发文量
110
审稿时长
4-8 weeks
期刊介绍: Progress in Molecular Biology and Translational Science (PMBTS) provides in-depth reviews on topics of exceptional scientific importance. If today you read an Article or Letter in Nature or a Research Article or Report in Science reporting findings of exceptional importance, you likely will find comprehensive coverage of that research area in a future PMBTS volume.
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