小的基于RNA的核酸类药物代表一种有前景的新一类治疗性制剂，此类制剂沉默异常或过度活跃的致性基因，研究人员已发现了新机制，通过这种机制RNA药物能控制基因活性。Nucleic Acid Therapeutics上的一篇综合性综述文章详细描述了这些进展，这种期刊是由Mary Ann Liebert公司出版的同行评审期刊。
短链核酸，也称为小RNA，可被用于靶基因的沉默，使它们成为吸引人的药物代表。这些小RNA通过多种RNA干扰通路阻断基因表达，这些通路包括2个新发现的通路，在这2个通路中小RNA结合到存在于细胞核内的Argonaute蛋白或RNA的其他形式，例如长非编码RNA和前mRNA。
德克萨斯大学西南医学中心的Keith T. Gagnon,博士和 David R. Corey博士详细地综述了由控制基因表达的RNAi通路所共享的一般特性，重点综述了Argonaute-RNA复合物在基因调控中的潜力和其他靶向非编码RNA和前mRNA的出现形式的令人兴奋的新选项，综述的标题是"Argonaute and the Nuclear RNAs: New Pathways for RNA Mediated Control of Gene Expression"。
"RNA介导的控制基因表达的领域正在迅速展开，Gagnon 和 Corey写的文章提供了一篇生物医学研究令人兴奋并经常令人吃惊领域的高度信息化与时新的综述。我们很欣喜地发表这篇此领域的重要综述"， 北卡罗莱纳达勒姆杜克大学医学中心杜克翻译研究院的主要主编Bruce A. Sullenger博士这样说。（生物谷bioon.com）
Argonaute and the Nuclear RNAs: New Pathways for RNA-Mediated Control of Gene Expression
Keith T. Gagnon and David R. Corey
Abstract Small RNAs are a commonly used tool for gene silencing and a promising platform for nucleic acid drug development. They are almost exclusively used to silence gene expression post-transcriptionally through degradation of mRNA. Small RNAs, however, can have a broader range of function by binding to Argonaute proteins and associating with complementary RNA targets in the nucleus, including long noncoding RNAs (lncRNAs) and pre-mRNA. Argonaute-RNA complexes can regulate nuclear events like transcription, genome maintenance, and splicing. Thousands of lncRNAs and alternatively spliced pre-mRNA isoforms exist in humans, and these RNAs may serve as natural targets for regulation and therapeutic intervention. This review describes nuclear mechanisms for Argonaute proteins and small RNAs, new pathways for sequence-specific targeting, and the potential for therapeutic development of small RNAs with nuclear targets.
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