Cheng Jerry C, Sakamoto Kathleen M
Division of Hematology-Oncology, Department of Pediatrics, Mattel Children's Hospital at UCLA, David Geffen School of Medicine at UCLA, Los Angeles, California 90095, USA.
Cell Cycle. 2004 Nov;3(11):1398-401. doi: 10.4161/cc.3.11.1240. Epub 2004 Nov 15.
RNA interference (RNAi) has emerged as the tool of choice for studying gene function. Dubbed the "breakthrough of the year" in 2002 by the journal Science, RNAi is a naturally occurring host defense mechanism that mediates the sequence-specific degradation of target mRNA transcripts and their protein products. The specificity of RNAi makes it an ideal tool for targeted therapeutics against unique fusion oncogene sequences. RNAi may also be effective against viral-mediated oncogenesis and has the potential to enhance tumor sensitivities to existing chemotherapy. The current interest in the success of RNAi-based therapies will depend on the delivery systems that protect the silencing apparatus from endogenous nucleases, sustain tissue-specific expression of the small-interfering RNAs, and prevent the activation of a destructive nonspecific host immune response.
RNA干扰（RNAi）已成为研究基因功能的首选工具。《科学》杂志在2002年将其誉为“年度突破”，RNAi是一种天然存在的宿主防御机制，可介导靶mRNA转录本及其蛋白质产物的序列特异性降解。RNAi的特异性使其成为针对独特融合癌基因序列进行靶向治疗的理想工具。RNAi也可能对病毒介导的肿瘤发生有效，并有可能增强肿瘤对现有化疗的敏感性。目前对基于RNAi疗法成功的关注将取决于能够保护沉默装置免受内源性核酸酶破坏、维持小干扰RNA的组织特异性表达并防止破坏性非特异性宿主免疫反应激活的递送系统。
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