The Role of MicroRNAs in Neurodegeneration: Insights from Huntington's Disease.

IF 4.6 2区医学 Q1 NEUROSCIENCES

Molecular Neurobiology Pub Date : 2025-07-01 Epub Date: 2025-02-26 DOI:10.1007/s12035-025-04750-7

Reda M Mansour, Abanoub A S Shaker, Ahmed I Abulsoud, Sherif S Abdel Mageed, Alaa Ashraf, Elsayed G E Elsakka, Mohammed I Dahab, Mohamed M Sadek, Farah A Awad, Radwa H Lutfy, Hanan Elimam, Ahmed H I Faraag, Yara A Nassar, Mohamed A Ali, Osama A Mohammed, Mustafa Ahmed Abdel-Reheim, Ahmed S Doghish

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引用次数: 0

Abstract

MicroRNA (miRNAs) is a single non-coding strand with a small sequence of approximately 21-25 nucleotides, which could be a biomarker or act as a therapeutic agent for disease. This review explores the dynamic role of miRNAs in Huntington's disease (HD), encompassing their regulatory function, potential as diagnostic biomarker tools, and emerging therapeutic applications. We delved into the dysregulation of specific miRNAs in HD, for instance, downregulated levels of miR-9 and miR-124 and increased levels of miR-155 and miR-196a. These alterations highlight the promise of miRNAs as non-invasive tools for early HD detection and disease progression monitoring. Moving beyond diagnosis, the exciting potential of miRNA-based therapies. By mimicking downregulated miRNAs or inhibiting dysregulated ones, we can potentially restore the balance of mutant target gene expression and modify disease progression. Recent research using engineered miRNAs delivered via an adeno-associated virus (AAV) vector in a transgenic HD minipig model demonstrates encouraging results in reducing mutant HD and improving motor function.

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微rna在神经退行性变中的作用：来自亨廷顿病的见解。

MicroRNA （mirna）是一种具有大约21-25个核苷酸的小序列的非编码单链，可以作为生物标志物或作为疾病治疗剂。这篇综述探讨了mirna在亨廷顿病（HD）中的动态作用，包括它们的调节功能，作为诊断生物标志物工具的潜力，以及新兴的治疗应用。我们深入研究了HD中特定mirna的失调，例如，miR-9和miR-124水平下调，miR-155和miR-196a水平升高。这些变化凸显了mirna作为早期HD检测和疾病进展监测的非侵入性工具的前景。除了诊断之外，基于mirna的疗法的潜力令人兴奋。通过模仿下调的mirna或抑制失调的mirna，我们可以潜在地恢复突变靶基因表达的平衡，并改变疾病的进展。最近的研究表明，通过腺相关病毒（AAV）载体在转基因HD迷你猪模型中传递工程化mirna，在减少突变HD和改善运动功能方面取得了令人鼓舞的结果。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Molecular Neurobiology 医学-神经科学

CiteScore

9.00

自引率

2.00%

发文量

480

审稿时长

1 months

期刊介绍： Molecular Neurobiology is an exciting journal for neuroscientists needing to stay in close touch with progress at the forefront of molecular brain research today. It is an especially important periodical for graduate students and "postdocs," specifically designed to synthesize and critically assess research trends for all neuroscientists hoping to stay active at the cutting edge of this dramatically developing area. This journal has proven to be crucial in departmental libraries, serving as essential reading for every committed neuroscientist who is striving to keep abreast of all rapid developments in a forefront field. Most recent significant advances in experimental and clinical neuroscience have been occurring at the molecular level. Until now, there has been no journal devoted to looking closely at this fragmented literature in a critical, coherent fashion. Each submission is thoroughly analyzed by scientists and clinicians internationally renowned for their special competence in the areas treated.