细胞外微小 RNA 作为杜氏肌营养不良症生物标志物的潜力。
Biomarker Potential of Extracellular miRNAs in Duchenne Muscular Dystrophy.
机构信息
Department of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford, OX1 3QX, UK; Institute of Neurology, Sobell Department of Motor Neuroscience and Movement Disorders, University College London, London, Queen Square, London, WC1N 3BG, UK.
Department of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford, OX1 3QX, UK.
出版信息
Trends Mol Med. 2017 Nov;23(11):989-1001. doi: 10.1016/j.molmed.2017.09.002. Epub 2017 Oct 5.
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.
miRNAs 是小的非编码 RNA，不仅可以在细胞内调节基因表达，而且可能构成各种病理状态的有前途的细胞外生物标志物，包括进行性肌肉萎缩症杜氏肌营养不良症 (DMD)。一组肌肉丰富的 miRNAs，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 可用作药效学生物标志物，因为它们的水平可以在肌营养不良小鼠的外显子跳跃治疗后恢复到野生型水平。肌肉病理学和细胞外 myomiR 释放之间的关系很复杂，尚未完全理解。在这里，我们讨论了使细胞外 miRNA 作为潜在的 DMD 生物标志物的临床应用的最新进展，以及它们对肌肉生理学的可能贡献。
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