siRNA Versus miRNA as Therapeutics for Gene Silencing.
siRNA Versus miRNA as Therapeutics for Gene Silencing.
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DOI：
10.1038/mtna.2015.23
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发表时间：
2015-09-15
期刊：
影响因子：
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通讯作者：
Leung SW
中科院分区：
文献类型：
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作者：
Lam JK;Chow MY;Zhang Y;Leung SW
小干扰RNAs(SiRNAs)和微RNAs(MiRNAs)是二十多年前发现的在基因调控中具有重要作用的非编码RNAs。它们最近被研究为治疗包括癌症和感染在内的一系列疾病的新型治疗剂。基于siRNA和miRNA的药物的临床试验已经开始。SiRNAs和miRNAs有许多相似之处，都是短的双链RNA分子，通过靶向信使RNA在转录后水平发挥基因沉默作用，但它们的作用机制和临床应用是不同的。SiRNAs和miRNAs的主要区别在于前者具有高度的特异性，只有一个mRNA靶点，而后者有多个靶点。因此，siRNAs和miRNAs的治疗方法非常不同。因此，本文就治疗性siRNAs和miRNAs在作用机制、理化性质、递送和临床应用等方面进行了比较。此外，还讨论了开发这两类RNA作为治疗药物所面临的挑战。
Discovered a little over two decades ago, small interfering RNAs (siRNAs) and microRNAs (miRNAs) are noncoding RNAs with important roles in gene regulation. They have recently been investigated as novel classes of therapeutic agents for the treatment of a wide range of disorders including cancers and infections. Clinical trials of siRNA- and miRNA-based drugs have already been initiated. siRNAs and miRNAs share many similarities, both are short duplex RNA molecules that exert gene silencing effects at the post-transcriptional level by targeting messenger RNA (mRNA), yet their mechanisms of action and clinical applications are distinct. The major difference between siRNAs and miRNAs is that the former are highly specific with only one mRNA target, whereas the latter have multiple targets. The therapeutic approaches of siRNAs and miRNAs are therefore very different. Hence, this review provides a comparison between therapeutic siRNAs and miRNAs in terms of their mechanisms of action, physicochemical properties, delivery, and clinical applications. Moreover, the challenges in developing both classes of RNA as therapeutics are also discussed.