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基于微小RNA的多发性硬化症治疗潜力

MiRNA-based therapeutic potential in multiple sclerosis.

作者信息

Zabalza Ana, Pappolla Agustin, Comabella Manuel, Montalban Xavier, Malhotra Sunny

机构信息

Vall Hebron University Hospital & Research Institute (VHIR), Multiple Sclerosis Centre of Catalonia (Cemcat) & Neurology Department, Universitat Autonoma de Barcelona, Barcelona, Spain.

Faculty of Medicine, University of Vic - Central University of Catalonia (UVicUCC), Vic, Spain.

出版信息

Front Immunol. 2024 Aug 29;15:1441733. doi: 10.3389/fimmu.2024.1441733. eCollection 2024.

Abstract

This review will briefly introduce microRNAs (miRNAs) and dissect their contribution to multiple sclerosis (MS) and its clinical outcomes. For this purpose, we provide a concise overview of the present knowledge of MS pathophysiology, biomarkers and treatment options, delving into the role of selectively expressed miRNAs in clinical forms of this disease, as measured in several biofluids such as serum, plasma or cerebrospinal fluid (CSF). Additionally, up-to-date information on current strategies applied to miRNA-based therapeutics will be provided, including miRNA restoration therapy (lentivirus expressing a specific type of miRNA and miRNA mimic) and miRNA inhibition therapy such as antisense oligonucleotides, small molecules inhibitors, locked nucleic acids (LNAs), anti-miRNAs, and antagomirs. Finally, it will highlight future directions and potential limitations associated with their application in MS therapy, emphasizing the need for improved delivery methods and validation of therapeutic efficacy.

摘要

本综述将简要介绍微小RNA(miRNA),并剖析它们对多发性硬化症(MS)及其临床结局的作用。为此,我们简要概述了目前关于MS病理生理学、生物标志物和治疗选择的知识,深入探讨了在血清、血浆或脑脊液(CSF)等多种生物流体中检测到的选择性表达的miRNA在该疾病临床类型中的作用。此外,还将提供有关基于miRNA的治疗方法的最新信息,包括miRNA恢复疗法(表达特定类型miRNA的慢病毒和miRNA模拟物)和miRNA抑制疗法,如反义寡核苷酸、小分子抑制剂、锁核酸(LNA)、抗miRNA和抗反义寡核苷酸。最后,将强调其在MS治疗中应用的未来方向和潜在局限性,强调改进递送方法和验证治疗效果的必要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9c1a/11390414/8c7fc4843471/fimmu-15-1441733-g001.jpg

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