Noncoding RNAs (ncRNAs), such as miRNAs and long noncoding RNAs, are key regulators of gene expression at the post-transcriptional level and represent promising therapeutic targets and biomarkers for several human diseases, including Duchenne and Becker muscular dystrophies (DMD/BMD). A role for ncRNAs in the pathogenesis of muscular dystrophies has been suggested, even if it is still incompletely understood. Here, we discuss current progress leading towards the clinical utility of ncRNAs for DMD/BMD. Long and short noncoding RNAs are differentially expressed in DMD/BMD and have a mechanism of action via targeting mRNAs. A subset of muscle-enriched miRNAs, the so-called myomiRs (miR-1, miR-133, and miR-206), are increased in the serum of patients with DMD and in dystrophin-defective animal models. Interestingly, myomiRs might be used as biomarkers, given that their levels can be corrected after dystrophin restoration in dystrophic mice. Remarkably, further evidence demonstrates that ncRNAs also play a role in dystrophin expression; thus, their modulations might represent a potential therapeutic strategy with the aim of upregulating the dystrophin protein in combination with other oligonucleotides/gene therapy approaches.

中文翻译：

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Duchenne 和 Becker 肌营养不良症中的非编码 RNA：在发病机制以及未来预后和治疗前景中的作用。


非编码 RNA (ncRNA)，例如 miRNA 和长链非编码 RNA，是转录后水平基因表达的关键调节因子，代表了多种人类疾病的有前景的治疗靶点和生物标志物，包括杜氏肌营养不良症和贝克尔肌营养不良症 (DMD/BMD)。人们已经提出 ncRNA 在肌营养不良症发病机制中的作用，尽管这一作用仍不完全清楚。在这里，我们讨论 ncRNA 在 DMD/BMD 临床应用方面的最新进展。长非编码RNA和短非编码RNA在DMD/BMD中表达差异，并且具有通过靶向mRNA的作用机制。富含肌肉的 miRNA 的一个子集，即所谓的 myomiR（miR-1、miR-133 和 miR-206），在 DMD 患者和肌营养不良蛋白缺陷动物模型的血清中含量增加。有趣的是，myomiRs 可能被用作生物标志物，因为它们的水平可以在营养不良小鼠的肌营养不良蛋白恢复后得到纠正。值得注意的是，进一步的证据表明 ncRNA 也在肌营养不良蛋白表达中发挥作用。因此，它们的调节可能代表一种潜在的治疗策略，其目的是与其他寡核苷酸/基因治疗方法相结合，上调肌营养不良蛋白。