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微小 RNA 在杜氏肌营养不良症发病机制中的作用。

The Role of MicroRNA in the Pathogenesis of Duchenne Muscular Dystrophy.

机构信息

Department of Physiology, Pomeranian Medical University, 70-111 Szczecin, Poland.

1st Chair and Department of Cardiology, Medical University of Warsaw, Banacha 1A, 02-097 Warsaw, Poland.

出版信息

Int J Mol Sci. 2024 Jun 1;25(11):6108. doi: 10.3390/ijms25116108.

Abstract

Duchenne muscular dystrophy (DMD) is an X-linked progressive disorder associated with muscle wasting and degeneration. The disease is caused by mutations in the gene that encodes dystrophin, a protein that links the cytoskeleton with cell membrane proteins. The current treatment methods aim to relieve the symptoms of the disease or partially rescue muscle functionality. However, they are insufficient to suppress disease progression. In recent years, studies have uncovered an important role for non-coding RNAs (ncRNAs) in regulating the progression of numerous diseases. ncRNAs, such as micro-RNAs (miRNAs), bind to their target messenger RNAs (mRNAs) to suppress translation. Understanding the mechanisms involving dysregulated miRNAs can improve diagnosis and suggest novel treatment methods for patients with DMD. This review presents the available evidence on the role of altered expression of miRNAs in the pathogenesis of DMD. We discuss the involvement of these molecules in the processes associated with muscle physiology and DMD-associated cardiomyopathy.

摘要

杜氏肌营养不良症(DMD)是一种与肌肉萎缩和退化相关的 X 连锁进行性疾病。该疾病由编码肌营养不良蛋白的基因突变引起,肌营养不良蛋白是一种将细胞骨架与细胞膜蛋白连接的蛋白质。目前的治疗方法旨在缓解疾病症状或部分恢复肌肉功能,但不足以抑制疾病进展。近年来,研究发现非编码 RNA(ncRNA)在调节多种疾病的进展中起着重要作用。ncRNA,如 micro-RNAs(miRNAs),与它们的靶信使 RNA(mRNA)结合以抑制翻译。了解涉及失调 miRNA 的机制可以改善 DMD 患者的诊断并提出新的治疗方法。本综述介绍了 miRNA 表达改变在 DMD 发病机制中的作用的现有证据。我们讨论了这些分子在与肌肉生理学和 DMD 相关的心肌病相关的过程中的参与。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6c42/11172814/b28d9dfe5243/ijms-25-06108-g001.jpg

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