MicroRNA therapeutics: a new niche for antisense nucleic acids

MicroRNA therapeutics: a new niche for antisense nucleic acids
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DOI:
10.1016/j.molmed.2006.01.004
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发表时间:
2006-03-01
影响因子:
13.6
通讯作者:
Hammond, SM
Hammond, SM
中科院分区:
医学1区
文献类型:
--
作者:
Hammond, SM

文献摘要

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MicroRNA分子(MiRNAs)是RNA干扰途径的自然触发因素。20年前,在秀丽隐杆线虫中发现了第一个被鉴定的miRNA,LIN-4。这种模式生物最初是一种好奇的东西,现在已经扩展到生物学的几乎所有领域;现在人类中已证实的miRNA基因有326个,预计总数将达到1000个。每个miRNA都有可能调控数百个mRNAs;因此,很可能没有几条生物途径不受miRNA调控的影响。最近的证据表明,miRNAs可能是包括癌症在内的一系列疾病的可行治疗靶点。Stoffel和他的同事最近的一篇文章证明了在体内对miRNAs的显著有效抑制,从而为进入前景光明的miRNA疗法的新领域提供了一个切入点。
MicroRNA molecules (miRNAs) are naturally occurring triggers of the RNA-interference pathway. The first identified miRNA, lin-4, was discovered in Caenorhabditis elegans > 20 years ago. What began as a curiosity in this model organism has expanded into almost every area of biology; there are now 326 confirmed miRNA genes in humans and the total is predicted to reach 1000. Each miRNA has the potential to regulate hundreds of mRNAs; therefore, there are likely to be few biological pathways not impacted by miRNA regulation. Recent evidence has suggested that miRNAs might be viable therapeutic targets for a wide range of diseases, including cancer. A recent article by Stoffel and colleagues has demonstrated remarkably effective inhibition of miRNAs in vivo, thus providing an entry point into the promising new arena of miRNA therapeutics.