Skeletal Muscle MicroRNAs: Their Diagnostic and Therapeutic Potential in Human Muscle Diseases.
Skeletal Muscle MicroRNAs: Their Diagnostic and Therapeutic Potential in Human Muscle Diseases.
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DOI：
10.3233/jnd-140058
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发表时间：
2015
影响因子：
3.3
通讯作者：
Kunkel LM
中科院分区：
文献类型：
--
作者：
Alexander MS;Kunkel LM
MicroRNAs （miRNAs）是一种21-24核苷酸的小rna，能够调节多种组织的多种信号通路。在早期发育、组织再生、癌症和各种其他疾病状态期间，MicroRNAs的表达水平受到动态调控和变化。最近，从全血清和肌肉活检中分离出微小rna，以确定特定神经肌肉疾病状态的独特诊断特征。在神经肌肉疾病细胞系和动物模型中对microrna的功能研究已经阐明了它们在神经肌肉疾病进展和病理中的重要性。microRNAs改变整个信号通路表达的能力开启了其独特的能力，可作为治疗疾病的潜在治疗切入点。在这里，我们将回顾各种神经肌肉疾病中关键microrna及其失调的最新发现。此外，我们将强调当前用于调节关键microrna表达的策略，因为它们在一些神经肌肉疾病的临床治疗中已经成为重要的参与者。
MicroRNAs (miRNAs) are small 21–24 nucleotide RNAs that are capable of regulating multiple signaling pathways across multiple tissues. MicroRNAs are dynamically regulated and change in expression levels during periods of early development, tissue regeneration, cancer, and various other disease states. Recently, microRNAs have been isolated from whole serum and muscle biopsies to identify unique diagnostic signatures for specific neuromuscular disease states. Functional studies of microRNAs in cell lines and animal models of neuromuscular diseases have elucidated their importance in contributing to neuromuscular disease progression and pathologies. The ability of microRNAs to alter the expression of an entire signaling pathway opens up their unique ability to be used as potential therapeutic entry points for the treatment of disease. Here, we will review the recent findings of key microRNAs and their dysregulation in various neuromuscular diseases. Additionally, we will highlight the current strategies being used to regulate the expression of key microRNAs as they have become important players in the clinical treatment of some of the neuromuscular diseases.