Lieberman Judy, Song Erwei, Lee Sang-Kyung, Shankar Premlata
Center for Blood Research and Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA.
Trends Mol Med. 2003 Sep;9(9):397-403. doi: 10.1016/s1471-4914(03)00143-6.
RNA interference (RNAi) is an evolutionarily conserved mechanism for silencing gene expression by targeted degradation of mRNA. Short double-stranded RNAs, known as small interfering RNAs (siRNA), are incorporated into an RNA-induced silencing complex that directs degradation of RNA containing a homologous sequence. RNAi has been shown to work in mammalian cells, and can inhibit viral infection and control tumor cell growth in vitro. Recently, it has been shown that intravenous injection of siRNA or of plasmids expressing sequences processed to siRNA can protect mice from autoimmune and viral hepatitis. RNAi could provide an exciting new therapeutic modality for treating infection, cancer, neurodegenerative disease and other illnesses.
RNA干扰（RNAi）是一种通过靶向降解mRNA使基因表达沉默的进化保守机制。短双链RNA，即小干扰RNA（siRNA），被整合到RNA诱导沉默复合体中，该复合体指导含有同源序列的RNA的降解。RNAi已被证明在哺乳动物细胞中起作用，并且在体外可抑制病毒感染和控制肿瘤细胞生长。最近，研究表明静脉注射siRNA或表达加工成siRNA序列的质粒可保护小鼠免受自身免疫性肝炎和病毒性肝炎的侵害。RNAi可能为治疗感染、癌症、神经退行性疾病和其他疾病提供一种令人兴奋的新治疗方式。
