RNAi 和小干扰 RNA 在人类疾病治疗应用中的作用。
RNAi and small interfering RNAs in human disease therapeutic applications.
机构信息
Department of Molecular and Cellular Biology, Beckman Institute at City of Hope, 1500 East Duarte Road, Duarte, CA 91010, USA.
出版信息
Trends Biotechnol. 2010 Nov;28(11):570-9. doi: 10.1016/j.tibtech.2010.07.009. Epub 2010 Sep 15.
Abstract
Small interfering RNAs (siRNAs) have been shown to effectively downregulate gene expression in human cells, giving them potential to eradicate disease. Prospects for clinical applications are discussed in this review, along with an overview of recent history and our current understanding of siRNAs used for therapeutic application in human diseases, such as cancer and viral infections. Over recent years, progress has been made in lipids, ligands, nanoparticles, polymers and viral vectors as delivery agents and for gene-based expression of siRNA to enhance the efficacy and specificity of these methods while at the same time reducing toxicity. It has become apparent that given the recent advances in chemistry and delivery, RNAi will soon prove to be an important and widely used therapeutic modality.
摘要
小干扰 RNA（siRNA）已被证明可有效下调人类细胞中的基因表达，从而有潜力根除疾病。本文讨论了其临床应用的前景，概述了 siRNA 的历史沿革和我们目前对其在人类疾病（如癌症和病毒感染）治疗应用的理解。近年来，在脂质、配体、纳米颗粒、聚合物和病毒载体作为递药载体，以及基因表达 siRNA 以提高这些方法的疗效和特异性的同时降低毒性方面取得了进展。显然，鉴于化学和递药方面的最新进展，RNAi 很快将被证明是一种重要且广泛应用的治疗模式。
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