摘要
小干涉RNA(small interfering RNA,siRNA)是目前研究的比较活跃的一种小RNA。利用siRNA使基因沉寂从而调节目的蛋白的功能,在疾病的基因治疗中有巨大的应用潜力。该文从siRNA在哺乳动物整体水平研究的角度,对近年来siRNA的载体策略以及siRNA导入体内的方法和途径等方面的研究进展作一综述,并讨论了siRNA在体内应用的优势和可能存在的问题。
Small interfering RNA (siRNA) is emerging as a powerful tool for in vivo research and for use as potential therapeutic agents. This review demonstrates the examples of the applications of siRNA as a new modality in gene therapy which can elicit down-regulation of gene expression and has prodigious potential in the treatment of diseases in mammals. Before siRNA tech-nique is used in vivo, efficient strategies of delivery must be designed, such as vectors and routes of delivery. The problems that should be considered before applying this technology are outlined.
出处
《中国药理学通报》
CAS
CSCD
北大核心
2007年第2期155-157,共3页
Chinese Pharmacological Bulletin