人工合成环状RNA的治疗前景。

IF 4.8 2区 生物学 Q1 CELL BIOLOGY
Hui Chen, Yi Du, Yesheng Wang, Xue Zhang, Maolei Zhang, Xingguo Liang, Qiyi Zhao, Ming Liu
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引用次数: 0

摘要

内源性环状rna在生物体中发挥着至关重要的生理作用,并具有作为疾病诊断、预后和治疗靶向的生物标志物的巨大潜力。环状rna的闭环结构在稳定性方面具有独特的优势,使其成为可编程传递载体和平台的理想候选者,用于药物传递和治疗应用。各种人工合成技术已经被开发出来,以确保工程环状rna的可靠供应,从而使新的药物和疗法的创造成为可能。然而,关于人工合成环状rna的景观和新趋势,仍然缺乏系统的总结。本文就人工合成环状rna的制备纯化方法、序列设计与优化策略、给药技术、质量控制、应用场景、临床前和临床研究进展等方面进行综述。在此基础上,我们对该领域的临床翻译障碍和未来趋势进行了深入分析。我们相信,在科研领域和产业界的专家学者加强合作后,环状rna作为治疗手段的临床应用前景广阔。本文分为:调控RNA /RNAi/核糖开关>调控RNA在疾病和发展> RNA在疾病翻译>机制。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Therapeutic Prospects of Artificially Synthesized Circular RNA.

Endogenous circRNAs play crucial physiological roles within organisms and hold significant potential as biomarkers for disease diagnosis, prognosis, and therapeutic targeting. The closed-loop structure of circRNAs confers unique advantages in stability, making them ideal candidates for programmable delivery vectors and platforms for drug delivery and therapeutic applications. Various artificial synthesis techniques have been developed to ensure a reliable supply of engineered circular RNAs, thereby enabling the creation of novel drugs and therapies. However, there remains a notable absence of a systematic summary regarding the landscape and emerging trends of artificially synthesized circular RNAs. In this review, we provide a comprehensive overview of the preparation and purification methods, sequence design and optimization strategies, delivery technologies, quality control, application scenarios, as well as the progress in pre-clinical and clinical research on drugs and therapies related to artificially synthesized circular RNAs. Building upon this foundation, we conducted an in-depth analysis of the clinical translation hurdles and forthcoming trends in this field. We believe that clinical applications of circular RNAs as therapeutic approaches are promising after experts and scholars in both scientific research area and industry strengthen their collaboration. This article is categorized under: Regulatory RNAs/RNAi/Riboswitches > Regulatory RNAs RNA in Disease and Development > RNA in Disease Translation > Mechanisms.

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来源期刊
CiteScore
14.80
自引率
4.10%
发文量
67
审稿时长
6-12 weeks
期刊介绍: WIREs RNA aims to provide comprehensive, up-to-date, and coherent coverage of this interesting and growing field, providing a framework for both RNA experts and interdisciplinary researchers to not only gain perspective in areas of RNA biology, but to generate new insights and applications as well. Major topics to be covered are: RNA Structure and Dynamics; RNA Evolution and Genomics; RNA-Based Catalysis; RNA Interactions with Proteins and Other Molecules; Translation; RNA Processing; RNA Export/Localization; RNA Turnover and Surveillance; Regulatory RNAs/RNAi/Riboswitches; RNA in Disease and Development; and RNA Methods.
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