RNA Therapy: Current Status and Future Potential.
RNA Therapy: Current Status and Future Potential.
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DOI：
10.4068/cmj.2020.56.2.87
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发表时间：
2020-05-01
期刊：
影响因子：
--
通讯作者：
Kim, Young-Kook
中科院分区：
文献类型：
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作者：
Kim, Young-Kook
近年来的研究发现了包括非编码rna在内的多种rna及其在细胞中的多种作用机制。这些rna调节多种细胞通路，因此有望成为治疗人类疾病的重要靶点。随着其广泛的功能研究，基于rna的治疗技术近年来有了长足的发展。经过多年的研究和各种试验和错误，反义rna和基于小干扰rna的药物已经开发出来，现在正在临床应用。此外，基于RNA适体和信使RNA的药物开发正在积极进行研究。随着这些基于RNA的药物的发展，各种各样的策略被开发出来，以有效地将RNA药物转运到细胞中。RNA疗法比现有的基于小分子或单克隆抗体的疗法有许多优点，包括其靶向细胞中所有基因的潜力。本文将介绍RNA治疗的发展历史，阐述RNA治疗的基本概念以及目前市场上或临床试验的RNA药物。此外，还将讨论RNA治疗的未来潜力。
Recent studies identified diverse RNAs including noncoding RNAs and their various action mechanisms in the cells. These RNAs regulate a variety of cellular pathways and are therefore expected to be important targets for the treatment of human diseases. Along with their extensive functional studies, RNA-based therapeutic techniques have developed considerably in recent years. After years of research and various trial and error, antisense RNAs and small interfering RNAs-based drugs have been developed and are now being used in the clinic. In addition, active research is ongoing to develop drugs based on RNA aptamer and messenger RNA. Along with the development of these RNA-based drugs, diverse strategies have been developed to transport RNA drugs into the cells efficiently. RNA therapy has many advantages over existing small molecule or monoclonal antibody-based therapies, including its potential to target all genes in the cells. This review will introduce the history of RNA therapy, and explain the basic concepts of RNA therapy and RNA-based drugs on the market or clinical trials. In addition, the future potential of RNA therapy will be discussed.