摘要
神经退行性疾病如阿尔茨海默病,帕金森病,肌萎缩性侧索硬化症,亨廷顿病和朊病毒不能及时有效地用常规疗法治疗。这强调了对替代治疗方法的需求。在这方面,基于基因的疗法已经被采用作为潜在可行的替代疗法,其中microRNA(miRNA)方法近年来经历了巨大的爆炸。由于miRNA已经被证明涉及包括神经退行性疾病在内的几种疾病的发病机制,因此它们作为诊断和预后生物标志物的候选者,作为药物反应的预测物和作为治疗剂被强烈研究。在这篇综述中,我们评估了直接和间接miRNA模拟物和抑制剂在体内和体外模型中对神经变性相关基因的调控的可行性,突出了与基于miRNA的治疗相关的优点和缺点,并总结了相关技术并且试图将miRNA递送到中枢神经系统用于治疗目的,特别是关于外泌体。此外,我们描述了一种新的方法,对包括神经退行性疾病在内的广泛疾病的治疗具有很大的希望。该方法基于解决将miRNA掺入外泌体以增加包装并递送至中枢神经系统和其他作用位点的miRNA的数量和质量。
关键词: MicroRNA,神经退行性疾病,基于miRNA的治疗,外泌体,miRNA递送,miRNA模拟物,miRNA抑制剂,治疗。
Current Gene Therapy
Title:Neurodegenerative Disorders Treatment: The MicroRNA Role
Volume: 17 Issue: 5
关键词: MicroRNA,神经退行性疾病,基于miRNA的治疗,外泌体,miRNA递送,miRNA模拟物,miRNA抑制剂,治疗。
摘要: Neurodegenerative diseases such as Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis, Huntington’s disease and prion disease are not timely and effectively treated using conventional therapies. This emphasizes the need for alternative therapeutic approaches. In this respect, gene-based therapies have been adopted as potentially feasible alternative therapies, where the microRNA (miRNA) approach has experienced a great explosion in recent years. Because miRNAs have been shown to be implicated in the pathogenesis of several diseases including neurodegenerative diseases, they are intensely studied as candidates for diagnostic and prognostic biomarkers, as predictors of drug response and as therapeutic agents. In this review, we evaluate the feasibility of both direct and indirect miRNA mimics and inhibitors toward the regulation of neurodegenerative-related genes both in vivo and in vitro models, highlight the advantages and drawbacks associated with miRNA-based therapy, and summarize the relevant techniques and approaches attempted to deliver miRNAs to the central nervous system for therapeutic purposes, with particular regard to the exosomes. Additionally, we describe a new approach that holds great promise for the treatment of a wide range of diseases including neurodegenerative disorders. This approach is based on addressing the incorporation of miRNAs into exosomes to increase the quantity and quality of miRNA packed and delivered to the central nervous system and other sites of action.
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Neurodegenerative Disorders Treatment: The MicroRNA Role, Current Gene Therapy 2017; 17 (5) . https://dx.doi.org/10.2174/1566523218666180119120726
DOI https://dx.doi.org/10.2174/1566523218666180119120726 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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