The promise of microRNA replacement therapy.
The promise of microRNA replacement therapy.
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DOI：
10.1158/0008-5472.can-10-2010
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发表时间：
2010-09-15
期刊：
影响因子：
11.2
通讯作者：
Winkler M
中科院分区：
文献类型：
--
作者：
Bader AG;Brown D;Winkler M
微小RNA（microRNAs，miRNAs）是一类天然的RNA干扰因子，近年来被认为是治疗性干预的重要靶点。开发miRNA治疗剂的基本原理是基于这样的前提，即异常表达的miRNA在人类疾病的发展中起关键作用，并且通过拮抗或恢复miRNA功能来纠正这些miRNA缺陷可以提供治疗益处。虽然miRNA拮抗剂在概念上类似于其他抑制性疗法，但通过miRNA替代来恢复miRNA的功能是一种不太好表征的方法。在这里，我们讨论了miRNA替代的具体性质，并回顾了最近的例子，探讨了癌症动物模型中的miRNA模拟物的治疗传递。
MicroRNAs (miRNAs), a class of natural RNA-interfering agents, have recently been identified as attractive targets for therapeutic intervention. The rationale for developing miRNA therapeutics is based on the premise that aberrantly expressed miRNAs play key roles in the development of human disease and that correcting these miRNA deficiencies by either antagonizing or restoring miRNA function may provide a therapeutic benefit. While miRNA antagonists are conceptually similar to other inhibitory therapies, restoring the function of a miRNA by miRNA replacement is a less well characterized approach. Here, we discuss the specific properties of miRNA replacement and review recent examples that explored the therapeutic delivery of miRNA mimics in animal models of cancer.