Liu Ning, Bassel-Duby Rhonda
Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, TX, 75390, USA,
Results Probl Cell Differ. 2015;56:165-90. doi: 10.1007/978-3-662-44608-9_8.
The identification of microRNAs (miRNA) in vertebrates has uncovered new mechanisms regulating skeletal muscle development and disease. miRNAs are inhibitors and act by silencing specific mRNAs or by repressing protein translation. In many cases, miRNAs are involved in physiological or pathological stress, suggesting they function to exacerbate or protect the organism during stress or disease. Although many skeletal muscle diseases differ in clinical and pathological manifestations, they all have a common feature of dysregulation of miRNA expression. In particular, analysis of miRNA expression patterns in skeletal muscle diseases reveals miRNA signatures, showing many miRNAs are dysregulated during disease. Emerging identification of miRNA targets and involvement in genetic regulatory networks serve to reveal new regulatory pathways in skeletal muscle biology. This chapter features the findings pertaining to skeletal muscle miRNAs in skeletal muscle development and disease and highlights therapeutic applications of miRNA-based technology in diagnosis and treatment of skeletal muscle myopathies.
脊椎动物中微小RNA（miRNA）的鉴定揭示了调节骨骼肌发育和疾病的新机制。miRNA起抑制作用，通过使特定mRNA沉默或抑制蛋白质翻译来发挥作用。在许多情况下，miRNA参与生理或病理应激，这表明它们在应激或疾病期间起到加剧或保护机体的作用。尽管许多骨骼肌疾病在临床和病理表现上有所不同，但它们都有一个共同特征，即miRNA表达失调。特别是，对骨骼肌疾病中miRNA表达模式的分析揭示了miRNA特征，表明许多miRNA在疾病期间表达失调。miRNA靶标的新发现以及它们参与遗传调控网络，有助于揭示骨骼肌生物学中的新调控途径。本章介绍了与骨骼肌发育和疾病中骨骼肌miRNA相关的研究结果，并强调了基于miRNA的技术在骨骼肌肌病诊断和治疗中的治疗应用。
