《生命科学》 2007, 19(2):

RNA干扰在疾病治疗方面的应用研究

褚　亮¹，刘新垣^{1,2 *}

1中国科学院上海生命科学研究院生物化学与细胞生物学研究所，上海200031；2浙江理工大学生命科学学院，杭州310018

摘　要：
摘　要：RNA干扰是由双链RNA引起的序列特异的基因沉默现象。由于RNA干扰能在细胞组织及动物模型中沉默疾病相关基因，因此，RNA干扰也是各种疾病治疗的有效手段。在哺乳动物细胞内诱导RNA干扰可以通过导入小干扰RNA(siRNA)，或是以质粒、病毒为载体表达短的发夹RNA(shRNA)而实现。本文介绍了RNA干扰在疾病治疗方面的应用，并就其面临的挑战进行讨论。
关键词：RNA干扰；小干扰RNA；短的发夹RNA；基因沉默

Progress in RNA interference therapeutics

CHU Liang¹, LIU Xinyuan^{1,2 *}

1 Institute of Biochemistry and Cell Biology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai 200031, China; 2 Xinyuan Institute of Medicine and Biotechnology, Zhejiang Sci-Tech University, Hangzhou 310018, China

Abstract: Abstract: RNA interference (RNAi) is a conserved biologic response to double-stranded RNA that resulted in the sequence-specific silencing of target gene expression. RNAi is a valuable tool in the treatment of diseases for its potency to silence disease-associated genes in tissue culture and animal models. RNAi can be induced in mammalian cells by the introduction of chemically synthesized small interfering RNAs (siRNAs) or by plasmid and viral vectors that express short-hairpin RNAs (shRNAs) that can be processed to siRNAs by the cellular machinery. In this review, we focus on the potential therapeutic use of RNAi. Also reviewed, are the challenges involved in developing RNAi for clinical use.
Key words: RNA interference; small interfering RNA; short-hairpin RNA; gene silence