The Biogenesis of miRNAs and Their Role in the Development of Amyotrophic Lateral Sclerosis.

The Biogenesis of miRNAs and Their Role in the Development of Amyotrophic Lateral Sclerosis.
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miRNA 的生物发生及其在肌萎缩侧索硬化症发展中的作用

DOI:
10.3390/cells11030572
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发表时间:
2022-02-07
期刊:
影响因子:
6
通讯作者:
Wang X
Wang X
中科院分区:
生物学2区
文献类型:
--
作者:
Liu J;Zhou F;Guan Y;Meng F;Zhao Z;Su Q;Bao W;Wang X;Zhao J;Huo Z;Zhang L;Zhou S;Chen Y;Wang X

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肌萎缩侧索硬化症(ALS)是一种影响上下运动神经元的神经退行性疾病。由于ALS尚无有效的治疗方法,因此筛选关键的基因治疗靶点尤为重要。microRNA(miRNAs)的发现彻底改变了传统的基因调控观点。miRNAs是一类非编码的单链RNA分子,参与转录后基因表达的调控。最近的进展还表明,miRNA是许多疾病的生物标志物,包括神经退行性疾病。本文就miRNAs在ALS发病机制中的作用及其在ALS基因治疗中的应用作一综述。miRNAs靶向不同途径的潜力为ALS治疗开辟了新的途径。
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that affects upper and lower motor neurons. As there is no effective treatment for ALS, it is particularly important to screen key gene therapy targets. The identifications of microRNAs (miRNAs) have completely changed the traditional view of gene regulation. miRNAs are small noncoding single-stranded RNA molecules involved in the regulation of post-transcriptional gene expression. Recent advances also indicate that miRNAs are biomarkers in many diseases, including neurodegenerative diseases. In this review, we summarize recent advances regarding the mechanisms underlying the role of miRNAs in ALS pathogenesis and its application to gene therapy for ALS. The potential of miRNAs to target diverse pathways opens a new avenue for ALS therapy.
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