肌特异性microRNA作为杜氏肌营养不良生物标志物的研究进展

A review on muscle-specific microRNAs as the biomarker for Duchenne muscular dystrophy

摘要:

microRNA（miRNA）是一种长度约22nt的非编码单链RNA，主要在转录后调控基因的表达。目前miRNA已成为多种疾病潜在的生物标志物，如肿瘤、白血病、神经系统疾病等。肌特异性miRNA在杜氏肌营养不良（DMD）患者的骨骼肌中富集，且在DMD发病机制中起重要作用。鉴于肌酸激酶诊断DMD的特异性有限，如其水平与病情轻重无明显关联，故探索肌特异性miRNA是否能成为理想的DMD生物标志物具有重要临床意义，该文将对这一领域研究进展进行综述。

Abstract:

MicroRNA (miRNA) is a non-coding single-stranded RNA with a length of approximately 22 nucleotides and is mainly responsible for the regulation of gene expression at the post-transcriptional level. At present, miRNA have become potential biomarkers for various diseases such as tumor, leukemia, and nervous system disease. Muscle-specific microRNAs are enriched in the skeletal muscle of patients with Duchenne muscular dystrophy (DMD) and also play an important role in the pathogenesis of DMD. Creatine kinase has limited specificity in the diagnosis of DMD since its level is not significantly associated with disease severity, and therefore, it is of great clinical significance to explore whether muscle-specific microRNAs can be used as ideal biomarkers for DMD. This article reviews the research advances in this field.