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The regulatory roles of microRNAs toward pathogenesis and treatments in Huntington's disease
Journal of Biomedical Science ( IF 11.0 ) Pub Date : 2021-08-19 , DOI: 10.1186/s12929-021-00755-1
Tung, Chih-Wei, Huang, Pin-Yu, Chan, Siew Chin, Cheng, Pei-Hsun, Yang, Shang-Hsun

Huntington’s disease (HD) is one of neurodegenerative diseases, and is defined as a monogenetic disease due to the mutation of Huntingtin gene. This disease affects several cellular functions in neurons, and further influences motor and cognitive ability, leading to the suffering of devastating symptoms in HD patients. MicroRNA (miRNA) is a non-coding RNA, and is responsible for gene regulation at post-transcriptional levels in cells. Since one miRNA targets to several downstream genes, it may regulate different pathways simultaneously. As a result, it raises a potential therapy for different diseases using miRNAs, especially for inherited diseases. In this review, we will not only introduce the update information of HD and miRNA, but also discuss the development of potential miRNA-based therapy in HD. With the understanding toward the progression of miRNA studies in HD, we anticipate it may provide an insight to treat this devastating disease, even applying to other genetic diseases.

中文翻译:

microRNAs对亨廷顿病发病机制和治疗的调控作用

亨廷顿病(HD)是一种神经退行性疾病,被定义为由于亨廷顿基因突变引起的单基因疾病。这种疾病会影响神经元的多种细胞功能,并进一步影响运动和认知能力,导致 HD 患者出现毁灭性症状。MicroRNA (miRNA) 是一种非编码 RNA,负责细胞中转录后水平的基因调控。由于一个 miRNA 靶向多个下游基因,它可能同时调节不同的途径。因此,它提出了一种使用 miRNA 治疗不同疾病的潜在疗法,尤其是针对遗传性疾病。在这篇综述中,我们不仅会介绍 HD 和 miRNA 的更新信息,还会讨论 HD 中潜在的基于 miRNA 的治疗的发展。
更新日期:2021-08-19
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