miRNAs are small, noncoding RNAs that not only regulate gene expression within cells, but might also constitute promising extracellular biomarkers for a variety of pathologies, including the progressive muscle-wasting disorder Duchenne Muscular Dystrophy (DMD). A set of muscle-enriched miRNAs, the myomiRs (miR-1, miR-133, and miR-206) are highly elevated in the serum of patients with DMD and in dystrophin-deficient animal models. Furthermore, circulating myomiRs might be used as pharmacodynamic biomarkers, given that their levels can be restored towards wild-type levels following exon skipping therapy in dystrophic mice. The relationship between muscle pathology and extracellular myomiR release is complex, and incompletely understood. Here, we discuss current progress leading towards the clinical utility of extracellular miRNAs as putative DMD biomarkers, and their possible contribution to muscle physiology.

miRNA是小的非编码RNA，不仅可以调节细胞内的基因表达，而且还可以构成各种疾病的有希望的细胞外生物标志物，包括进行性肌肉萎缩症杜兴氏肌营养不良症（DMD）。一组富含肌肉的miRNA，即myomiRs（miR-1，miR-133和miR-206）在DMD患者的血清中和肌营养不良蛋白缺陷的动物模型中高度升高。此外，循环的myomiRs可以用作药效生物标志物，因为在营养不良的小鼠中，在外显子跳过治疗后，其水平可以恢复为野生型水平。肌肉病理学与细胞外myomiR释放之间的关系是复杂的，尚未完全理解。这里，