Corydon Thomas Juhl, Mikkelsen Jacob Giehm
Aarhus Universitet, Institut for Human Genetik, Arhus C.
Ugeskr Laeger. 2006 Dec 11;168(50):4401-4.
RNA interference (RNAi) is an evolutionarily conserved cellular mechanism by which gene expression is suppressed by double-stranded RNA (dsRNA) in a sequence-specific manner. Enzymatically processed dsRNA molecules (siRNAs) target and facilitate cleavage of mRNA. By harnessing the RNAi machinery using synthetic or DNA-encoded RNA effectors, the siRNA technology offers new therapeutic strategies for genetic diseases. We outline the basic mechanisms of RNAi and present state-of-the-art examples of in vivo siRNA delivery towards development of RNAi-based individualized drugs.
RNA干扰(RNAi)是一种进化上保守的细胞机制,通过该机制,双链RNA(dsRNA)以序列特异性方式抑制基因表达。经过酶促加工的dsRNA分子(小干扰RNA,siRNAs)靶向并促进mRNA的切割。通过使用合成或DNA编码的RNA效应物来利用RNAi机制,siRNA技术为遗传疾病提供了新的治疗策略。我们概述了RNAi的基本机制,并展示了在体内递送siRNA以开发基于RNAi的个体化药物的最新实例。