Kim Young-Kook
Department of Biochemistry, Chonnam National University Medical School, Hwasun, Korea.
Chonnam Med J. 2020 May;56(2):87-93. doi: 10.4068/cmj.2020.56.2.87. Epub 2020 May 25.
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.
最近的研究在细胞中鉴定出了包括非编码RNA在内的多种RNA及其各种作用机制。这些RNA调节多种细胞途径，因此有望成为治疗人类疾病的重要靶点。随着对其广泛的功能研究，基于RNA的治疗技术近年来有了很大发展。经过多年研究以及各种反复试验，基于反义RNA和小干扰RNA的药物已被开发出来，目前正在临床中使用。此外，基于RNA适体和信使RNA的药物研发也在积极进行中。随着这些基于RNA的药物的发展，人们已经开发出多种策略来有效地将RNA药物转运到细胞中。RNA疗法相对于现有的基于小分子或单克隆抗体的疗法具有许多优势，包括其靶向细胞中所有基因的潜力。本综述将介绍RNA疗法的历史，并解释RNA疗法以及市场上或临床试验中的基于RNA的药物的基本概念。此外，还将讨论RNA疗法的未来潜力。
