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非编码 RNA 靶向治疗:最新综述。

Non-Coding RNA-Targeted Therapy: A State-of-the-Art Review.

机构信息

Department of Cardiac Surgery, Centre Cardiologique du Nord, 93200 Saint-Denis, France.

出版信息

Int J Mol Sci. 2024 Mar 24;25(7):3630. doi: 10.3390/ijms25073630.

Abstract

The use of non-coding RNAs (ncRNAs) as drug targets is being researched due to their discovery and their role in disease. Targeting ncRNAs, including microRNAs (miRNAs) and long non-coding RNAs (lncRNAs), is an attractive approach for treating various diseases, such as cardiovascular disease and cancer. This seminar discusses the current status of ncRNAs as therapeutic targets in different pathological conditions. Regarding miRNA-based drugs, this approach has made significant progress in preclinical and clinical testing for cardiovascular diseases, where the limitations of conventional pharmacotherapy are evident. The challenges of miRNA-based drugs, including specificity, delivery, and tolerability, will be discussed. New approaches to improve their success will be explored. Furthermore, it extensively discusses the potential development of targeted therapies for cardiovascular disease. Finally, this document reports on the recent advances in identifying and characterizing microRNAs, manipulating them, and translating them into clinical applications. It also addresses the challenges and perspectives towards clinical application.

摘要

由于非编码 RNA(ncRNA)的发现及其在疾病中的作用,将其作为药物靶点的研究正在进行中。针对 ncRNA,包括 microRNAs(miRNAs)和长非编码 RNA(lncRNAs),是治疗各种疾病(如心血管疾病和癌症)的一种有吸引力的方法。本研讨会讨论了 ncRNA 在不同病理条件下作为治疗靶点的现状。关于 miRNA 为基础的药物,这种方法在心血管疾病的临床前和临床试验中取得了重大进展,因为传统药物治疗的局限性是显而易见的。miRNA 药物的挑战,包括特异性、传递和耐受性,将被讨论。将探讨改善其成功的新方法。此外,它还广泛讨论了针对心血管疾病的靶向治疗的潜在发展。最后,本文报告了在鉴定和表征 microRNAs、操纵它们以及将它们转化为临床应用方面的最新进展。它还解决了临床应用的挑战和前景。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a949/11011542/9e1058c07820/ijms-25-03630-g006.jpg

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