Small interfering RNA (siRNA)-based therapeutic applications against viruses: principles, potential, and challenges.

IF 9 2区 医学 Q1 CELL BIOLOGY Journal of Biomedical Science Pub Date : 2023-10-16 DOI:10.1186/s12929-023-00981-9
Hara Kang, Yun Ji Ga, Soo Hyun Kim, Young Hoon Cho, Jung Won Kim, Chaeyeon Kim, Jung-Yong Yeh
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Abstract

RNA has emerged as a revolutionary and important tool in the battle against emerging infectious diseases, with roles extending beyond its applications in vaccines, in which it is used in the response to the COVID-19 pandemic. Since their development in the 1990s, RNA interference (RNAi) therapeutics have demonstrated potential in reducing the expression of disease-associated genes. Nucleic acid-based therapeutics, including RNAi therapies, that degrade viral genomes and rapidly adapt to viral mutations, have emerged as alternative treatments. RNAi is a robust technique frequently employed to selectively suppress gene expression in a sequence-specific manner. The swift adaptability of nucleic acid-based therapeutics such as RNAi therapies endows them with a significant advantage over other antiviral medications. For example, small interfering RNAs (siRNAs) are produced on the basis of sequence complementarity to target and degrade viral RNA, a novel approach to combat viral infections. The precision of siRNAs in targeting and degrading viral RNA has led to the development of siRNA-based treatments for diverse diseases. However, despite the promising therapeutic benefits of siRNAs, several problems, including impaired long-term protein expression, siRNA instability, off-target effects, immunological responses, and drug resistance, have been considerable obstacles to the use of siRNA-based antiviral therapies. This review provides an encompassing summary of the siRNA-based therapeutic approaches against viruses while also addressing the obstacles that need to be overcome for their effective application. Furthermore, we present potential solutions to mitigate major challenges.

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基于小干扰RNA(siRNA)的病毒治疗应用:原理、潜力和挑战。
RNA已成为抗击新发传染病的革命性和重要工具,其作用已超出其在疫苗中的应用,用于应对新冠肺炎大流行。自20世纪90年代发展以来,RNA干扰(RNAi)疗法已显示出降低疾病相关基因表达的潜力。基于核酸的疗法,包括RNAi疗法,可以降解病毒基因组并快速适应病毒突变,已成为替代疗法。RNAi是一种常用于以序列特异性方式选择性抑制基因表达的强大技术。基于核酸的治疗方法(如RNAi治疗)的快速适应性使其比其他抗病毒药物具有显著优势。例如,小干扰RNA(siRNA)是在序列互补的基础上产生的,以靶向和降解病毒RNA,这是一种对抗病毒感染的新方法。siRNA在靶向和降解病毒RNA方面的精确性导致了基于siRNA的多种疾病治疗方法的发展。然而,尽管siRNA具有良好的治疗效果,但一些问题,包括长期蛋白质表达受损、siRNA不稳定性、脱靶效应、免疫反应和耐药性,一直是使用基于siRNA的抗病毒疗法的相当大的障碍。这篇综述对基于siRNA的病毒治疗方法进行了全面总结,同时也解决了有效应用所需克服的障碍。此外,我们提出了缓解重大挑战的潜在解决方案。
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来源期刊
Journal of Biomedical Science
Journal of Biomedical Science 医学-医学:研究与实验
CiteScore
18.50
自引率
0.90%
发文量
95
审稿时长
1 months
期刊介绍: The Journal of Biomedical Science is an open access, peer-reviewed journal that focuses on fundamental and molecular aspects of basic medical sciences. It emphasizes molecular studies of biomedical problems and mechanisms. The National Science and Technology Council (NSTC), Taiwan supports the journal and covers the publication costs for accepted articles. The journal aims to provide an international platform for interdisciplinary discussions and contribute to the advancement of medicine. It benefits both readers and authors by accelerating the dissemination of research information and providing maximum access to scholarly communication. All articles published in the Journal of Biomedical Science are included in various databases such as Biological Abstracts, BIOSIS, CABI, CAS, Citebase, Current contents, DOAJ, Embase, EmBiology, and Global Health, among others.
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