PDF(Pubmed)
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)是与肌肉萎缩和退化相关的X连锁进行性疾病。这种疾病是由编码肌营养不良蛋白的基因突变引起的，一种连接细胞骨架和细胞膜蛋白的蛋白质。目前的治疗方法旨在缓解疾病的症状或部分挽救肌肉功能。然而,它们不足以抑制疾病进展。近年来,研究揭示了非编码RNA（ncRNA）在调节多种疾病进展中的重要作用。ncRNAs,如micro-RNAs(miRNA)，结合到它们的靶信使RNA(mRNA)以抑制翻译。了解涉及失调的miRNA的机制可以改善诊断，并为DMD患者提供新的治疗方法。这篇综述提供了关于miRNA表达改变在DMD发病机理中的作用的可用证据。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。
