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环状RNA的治疗潜力。

The therapeutic potential of circular RNAs.

作者信息

O'Leary Eoghan, Jiang Yanyi, Kristensen Lasse S, Hansen Thomas B, Kjems Jørgen

机构信息

Circio AB, Stockholm, Sweden.

Interdisciplinary Nanoscience Center, Aarhus University, Aarhus, Denmark.

出版信息

Nat Rev Genet. 2025 Apr;26(4):230-244. doi: 10.1038/s41576-024-00806-x. Epub 2025 Jan 9.

Abstract

Over the past decade, research into circular RNA (circRNA) has increased rapidly, and over the past few years, circRNA has emerged as a promising therapeutic platform. The regulatory functions of circRNAs, including their roles in templating protein translation and regulating protein and RNA functions, as well as their unique characteristics, such as increased stability and a favourable immunological profile compared with mRNAs, make them attractive candidates for RNA-based therapies. Here, we describe the properties of circRNAs, their therapeutic potential and technologies for their synthesis. We also discuss the prospects and challenges to be overcome to unlock the full potential of circRNAs as drugs.

摘要

在过去十年中,对环状RNA(circRNA)的研究迅速增加,在过去几年里,circRNA已成为一个有前景的治疗平台。circRNA的调控功能,包括其在模板蛋白翻译以及调节蛋白质和RNA功能方面的作用,以及它们的独特特性,如与信使核糖核酸(mRNA)相比具有更高的稳定性和良好的免疫学特征,使其成为基于RNA的治疗方法的有吸引力的候选者。在此,我们描述了circRNA的特性、它们的治疗潜力以及其合成技术。我们还讨论了要充分发挥circRNA作为药物的全部潜力所面临的前景和挑战。

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