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健康与疾病状态下心脏和骨骼肌中差异调节的微小RNA:潜在的药物靶点?

MicroRNAs differentially regulated in cardiac and skeletal muscle in health and disease: potential drug targets?

作者信息

Winbanks Catherine E, Ooi Jenny Yy, Nguyen Sally S, McMullen Julie R, Bernardo Bianca C

机构信息

Baker IDI Heart and Diabetes Institute, Monash University, Melbourne, Vic., Australia.

出版信息

Clin Exp Pharmacol Physiol. 2014 Sep;41(9):727-37. doi: 10.1111/1440-1681.12281.

DOI:10.1111/1440-1681.12281
PMID:25115402
Abstract

The identification of non-coding RNA species, previously thought of as 'junk' DNA, adds a new dimension of complexity to the regulation of DNA, RNA and protein. MicroRNAs are short non-coding RNA species that control gene expression, are dysregulated in settings of cardiac and skeletal muscle disease and have emerged as promising therapeutic targets. MicroRNAs specifically enriched in cardiac and skeletal muscle are called myomiRs and play an important role in cardiac pathology and skeletal muscle biology. Moreover, microRNA profiles are altered in response to exercise and disease; thus, their potential as therapeutic drug targets is being widely explored. In the cardiovascular field, therapeutic inhibition of microRNAs has been shown to be effective in improving cardiac outcome in preclinical cardiac disease models. MicroRNAs that promote skeletal muscle regeneration are attractive therapeutic targets in muscle wasting conditions where regenerative capacity is compromised.

摘要

非编码RNA种类的鉴定,以前被认为是“垃圾”DNA,为DNA、RNA和蛋白质的调控增加了一个新的复杂层面。微小RNA是控制基因表达的短非编码RNA种类,在心脏和骨骼肌疾病中失调,并已成为有前景的治疗靶点。在心脏和骨骼肌中特异性富集的微小RNA被称为肌微小RNA,在心脏病理学和骨骼肌生物学中起重要作用。此外,微小RNA谱会因运动和疾病而改变;因此,它们作为治疗药物靶点的潜力正在被广泛探索。在心血管领域,微小RNA的治疗性抑制已被证明在临床前心脏疾病模型中能有效改善心脏结局。促进骨骼肌再生的微小RNA在再生能力受损的肌肉萎缩病症中是有吸引力的治疗靶点。

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