Targeting circular RNAs as a therapeutic approach: current strategies and challenges.
Targeting circular RNAs as a therapeutic approach: current strategies and challenges.
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靶向环状 RNA 作为治疗方法：当前策略和挑战。
DOI：
10.1038/s41392-021-00569-5
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发表时间：
2021-05-21
影响因子：
39.3
通讯作者：
Yang BB
中科院分区：
文献类型：
--
作者：
He AT;Liu J;Li F;Yang BB
近年来，环状RNA（circRNA）的研究取得了重大进展。越来越多的证据表明，circRNA在许多细胞过程中发挥重要作用，其失调与多种疾病的发病机制有关。CircRNA是高度稳定的，通常以组织或细胞类型特异性的方式表达。因此，目前正在探索它们作为潜在的治疗靶点。功能获得和功能丧失的方法通常分别使用circRNA表达质粒和基于RNA干扰的策略进行。这些策略具有可以使用纳米颗粒和外泌体递送系统减轻的限制。此外，最近的研究表明，cre-lox系统可用于以细胞特异性方式敲除circRNAs。虽然仍处于开发的早期阶段，但CRISPR/Cas 13系统已显示出以高特异性和效率敲除circRNAs的前景。在这篇综述中，我们描述了circRNA的性质和功能，并强调其在疾病中的意义。我们总结了可用于过表达或敲低circRNA作为治疗方法的策略。最后，我们讨论了主要挑战，并提出了基于circRNA的治疗方法的未来发展方向。
Significant progress has been made in circular RNA (circRNA) research in recent years. Increasing evidence suggests that circRNAs play important roles in many cellular processes, and their dysregulation is implicated in the pathogenesis of various diseases. CircRNAs are highly stable and usually expressed in a tissue- or cell type-specific manner. Therefore, they are currently being explored as potential therapeutic targets. Gain-of-function and loss-of-function approaches are typically performed using circRNA expression plasmids and RNA interference-based strategies, respectively. These strategies have limitations that can be mitigated using nanoparticle and exosome delivery systems. Furthermore, recent developments show that the cre-lox system can be used to knockdown circRNAs in a cell-specific manner. While still in the early stages of development, the CRISPR/Cas13 system has shown promise in knocking down circRNAs with high specificity and efficiency. In this review, we describe circRNA properties and functions and highlight their significance in disease. We summarize strategies that can be used to overexpress or knockdown circRNAs as a therapeutic approach. Lastly, we discuss major challenges and propose future directions for the development of circRNA-based therapeutics.
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