miRNA药物的发展前景

Perspective of MiRNA Drugs

microRNA(miRNA)是一类非编码RNA(ncRNA),在转录后水平调节内源性基因表达。miRNA通过直接或间接上调或下调靶标mRNA的表达,几乎参与体内所有生理和病理过程。miRNA具有易于化学合成和修饰的优点,使其成为具有巨大治疗潜力的主要非编码RNA。本研究梳理miRNA类似物和抑制剂在癌症、心血管疾病、代谢性疾病等领域的的治疗靶点及作用机制,总结miRNA类似物和抑制剂的临床试验进展及优势,讨论miRNA药物治疗的机制、进展和存在的RNA稳定性和递送瓶颈问题及解决方案。尽管目前miRNA药物的发展尚存在一些挑战,但miRNA仍然是制药业中前景光明的靶标。

MicroRNA(miRNA)are a class of non-coding RNAs that regulate endogenous gene expression at the post-transcriptional level.It actively participates in almost all physiological and pathogenetic processes in vivo,through upregulating or downregulating the expression of target messenger RNA.The merits of easy chemical synthesis and modification make miRNA the major class of noncoding RNA with great therapeutical potential.In this artical,the miRNA drug researches in the fields of cancer,cardiovascular diseases,metabolic diseases,as well as the treatment principles and strategies were reviewed.The progresses of ongoing clinical trials of miRNA analogues and inhibitors were summarized.The mechanism,progress and challenges of the miRNA therapy were discussed.Despite the existing of some technical bottlenecks,miRNAs still remain as one of the promising therapeutical targets in the pharmaceutical industry.