Barik Sailen
Department of Biochemistry and Molecular Biology, College of Medicine, MSB2370, University of South Alabama, Mobile, 36688-0002, USA.
J Mol Med (Berl). 2005 Oct;83(10):764-73. doi: 10.1007/s00109-005-0690-0. Epub 2005 Jul 19.
Silencing of gene expression by ribonucleic acid (RNA), known as RNA interference (RNAi), is now recognized as a major means of gene regulation in biology. In this mechanism, small noncoding double-stranded RNA molecules knock down gene expression through a variety of mechanisms that include messenger RNA (mRNA) degradation, inhibition of mRNA translation, or chromatin remodeling. The posttranscriptional mechanism of RNAi has been embraced by researchers as a powerful tool for generating deficient phenotypes without mutating the gene. In parallel, exciting recent results have promised its application in disease therapy. This review aims to summarize the current knowledge in this area and provide a roadmap that may eventually launch RNAi from the research bench to the medicine chest.
核糖核酸（RNA）介导的基因表达沉默，即RNA干扰（RNAi），如今被公认为生物学中基因调控的主要方式。在这一机制中，小型非编码双链RNA分子通过多种机制降低基因表达，这些机制包括信使核糖核酸（mRNA）降解、mRNA翻译抑制或染色质重塑。RNAi的转录后机制已被研究人员视为一种强大工具，可用于在不使基因发生突变的情况下产生缺陷表型。与此同时，近期令人振奋的研究成果预示了其在疾病治疗中的应用前景。本综述旨在总结该领域的现有知识，并提供一份路线图，最终可能会将RNAi从研究台推向药箱。
