CAS, a division of the American Chemical Society 2540 Olentangy River Road, Columbus, Ohio 43202, United States.
College of Veterinary Medicine, Kansas State University, Manhattan, Kansas 66506, United States.
J Med Chem. 2022 May 26;65(10):6975-7015. doi: 10.1021/acs.jmedchem.2c00024. Epub 2022 May 9.
In the past decade, there has been a shift in research, clinical development, and commercial activity to exploit the many physiological roles of RNA for use in medicine. With the rapid success in the development of lipid-RNA nanoparticles for mRNA vaccines against COVID-19 and with several approved RNA-based drugs, RNA has catapulted to the forefront of drug research. With diverse functions beyond the role of mRNA in producing antigens or therapeutic proteins, many classes of RNA serve regulatory roles in cells and tissues. These RNAs have potential as new therapeutics, with RNA itself serving as either a drug or a target. Here, based on the CAS Content Collection, we provide a landscape view of the current state and outline trends in RNA research in medicine across time, geography, therapeutic pipelines, chemical modifications, and delivery mechanisms.
在过去的十年中,研究、临床开发和商业活动发生了转变,开始利用 RNA 的许多生理作用来开发医学用途。随着用于 COVID-19 mRNA 疫苗的脂质-RNA 纳米颗粒的快速成功开发,以及几种批准的基于 RNA 的药物,RNA 已一跃成为药物研究的前沿。除了在产生抗原或治疗性蛋白质方面发挥 mRNA 的作用之外,许多类型的 RNA 在细胞和组织中发挥调节作用。这些 RNA 具有作为新型治疗药物的潜力,RNA 本身可以作为药物或靶标。在这里,我们基于 CAS 内容集,提供了一个全景视图,概述了 RNA 药物研究在时间、地理位置、治疗管道、化学修饰和输送机制方面的现状和趋势。