基于环状RNA开发核酸药物研究进展

Research advances in nucleic acid drugs developed based on circular RNAs

摘要:

核酸药物是继化学药物和蛋白质药物之后的第三代药物，其中mRNA疫苗在抗击新型冠状病毒中发挥了重要作用。mRNA疫苗因其开发周期短，制备工艺成熟，在应对病毒变异、支持全球供应上具有显著优势。相比mRNA，环状RNA (circular RNA, circRNA)是一类具有更稳定的结构、更长的半衰期、更弱免疫原性等优点的分子，并且已被证实可以高效表达蛋白产物，由此可见环状RNA药物的开发前景更为广阔。虽然环状RNA的生物学功能有大量研究，但基于环状RNA开发核酸药物的研究还很少。本文概述了环状RNA的基本信息，总结了环状RNA的合成路径及作用机制，并讨论了该领域的发展方向，希望为环状RNA药物的研发提供一些启示和帮助。

Abstract:

The development and clinical application of nucleic acid drugs has been a trendy field. One of the notable examples is mRNA vaccines, which have been used in the fighting against SARS-CoV-2. With short development cycles and mature preparation processes, mRNA vaccines demonstrate advantages in the global supply and in response to virus mutations. Circular RNAs (circRNAs) are a group of nucleic acid molecules with more stable structure, longer half-life, and weaker immunogenicity than mRNAs. Studies have proven that circRNAs can efficiently express protein products, indicating their potential in drug development. Despite extensive studies on the biogenesis and biological functions of circRNAs, there is limited research on developing nucleic acid drugs based on circRNAs. This article provides an overview of circRNAs, including their basic information, synthesis routes, and mechanisms, and discusses the future development directions of this field, hoping to provide inspiration for the research and development of drugs based on circRNAs.