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环状 RNA 进出细胞:环状 RNA 的治疗用途。

Circular RNAs in and out of Cells: Therapeutic Usages of Circular RNAs.

机构信息

Graduate School of Medical Science and Engineering, Korea Advanced Institute for Science and Technology (KAIST), Daejeon 34141, Korea.

BioMedical Research Center, KAIST, Daejeon 34141, Korea.

出版信息

Mol Cells. 2023 Jan 31;46(1):33-40. doi: 10.14348/molcells.2023.2170. Epub 2023 Jan 18.

Abstract

RNAs are versatile molecules that are primarily involved in gene regulation and can thus be widely used to advance the fields of therapeutics and diagnostics. In particular, circular RNAs which are highly stable, have emerged as strong candidates for use on next-generation therapeutic platforms. Endogenous circular RNAs control gene regulatory networks by interacting with other biomolecules or through translation into polypeptides. Circular RNAs exhibit cell-type specific expression patterns, which can be altered in tissues and body fluids depending on pathophysiological conditions. Circular RNAs that are aberrantly expressed in diseases can function as biomarkers or therapeutic targets. Moreover, exogenous circular RNAs synthesized can be introduced into cells as therapeutic molecules to modulate gene expression networks . Depending on the purpose, synthetic circular RNA sequences can either be identical to endogenous circular RNA sequences or artificially designed. In this review, we introduce the life cycle and known functions of intracellular circular RNAs. The current stage of endogenous circular RNAs as biomarkers and therapeutic targets is also described. Finally, approaches and considerations that are important for applying the available knowledge on endogenous circular RNAs to design exogenous circular RNAs for therapeutic purposes are presented.

摘要

RNAs 是多功能分子,主要参与基因调控,因此可以广泛用于推进治疗学和诊断学领域。特别是,高度稳定的环状 RNA 已成为下一代治疗平台应用的有力候选物。内源性环状 RNA 通过与其他生物分子相互作用或通过翻译为多肽来控制基因调控网络。环状 RNA 表现出细胞类型特异性表达模式,这些模式可以根据病理生理条件在组织和体液中改变。在疾病中异常表达的环状 RNA 可以作为生物标志物或治疗靶点。此外,合成的环状 RNA 可以作为治疗分子被引入细胞,以调节基因表达网络。根据目的的不同,合成的环状 RNA 序列可以与内源性环状 RNA 序列相同,也可以是人工设计的。在这篇综述中,我们介绍了细胞内环状 RNA 的生命周期和已知功能。还描述了内源性环状 RNA 作为生物标志物和治疗靶点的现状。最后,提出了将内源性环状 RNA 的现有知识应用于设计用于治疗目的的外源性环状 RNA 时需要考虑的方法和注意事项。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/295b/9880607/5a3775f4fd7a/molce-46-1-33-f1.jpg

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