Types of RNA therapeutics.

3区 生物学 Q2 Biochemistry, Genetics and Molecular Biology
Pouya Goleij, Mehregan Babamohamadi, Aryan Rezaee, Pantea Majma Sanaye, Mohammad Amin Khazeei Tabari, Sarvin Sadreddini, Reza Arefnezhad, Hossein Motedayyen
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引用次数: 0

Abstract

RNA therapy is one of the new treatments using small RNA molecules to target and regulate gene expression. It involves the application of synthetic or modified RNA molecules to inhibit the expression of disease-causing genes specifically. In other words, it silences genes and suppresses the transcription process. The main theory behind RNA therapy is that RNA molecules can prevent the translation into proteins by binding to specific messenger RNA (mRNA) molecules. By targeting disease-related mRNA molecules, RNA therapy can effectively silence or reduce the development of harmful proteins. There are different types of RNA molecules used in therapy, including small interfering RNAs (siRNAs), microRNAs (miRNAs), aptamer, ribozyme, and antisense oligonucleotides (ASOs). These molecules are designed to complement specific mRNA sequences, allowing them to bind and degrade the targeted mRNA or prevent its translation into protein. Nanotechnology is also highlighted to increase the efficacy of RNA-based drugs. In this chapter, while examining various methods of RNA therapy, we discuss the advantages and challenges of each.

RNA 疗法的类型。
RNA 疗法是利用小 RNA 分子靶向调节基因表达的新疗法之一。它涉及应用合成或修饰的 RNA 分子来抑制特定致病基因的表达。换句话说,它能沉默基因并抑制转录过程。RNA 疗法背后的主要理论是,RNA 分子可以通过与特定的信使 RNA(mRNA)分子结合,阻止其翻译成蛋白质。通过靶向与疾病相关的 mRNA 分子,RNA疗法可以有效抑制或减少有害蛋白质的产生。用于治疗的 RNA 分子有多种类型,包括小干扰 RNA(siRNA)、microRNA(miRNA)、aptamer、ribozyme 和反义寡核苷酸(ASO)。这些分子旨在与特定的 mRNA 序列互补,使其能够结合并降解目标 mRNA 或阻止其翻译成蛋白质。纳米技术也是提高基于 RNA 的药物疗效的亮点。本章在探讨各种 RNA 治疗方法的同时,还讨论了每种方法的优势和挑战。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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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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