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小干扰RNA:未来乳腺癌治疗的潜在工具?

siRNA: a potential tool for future breast cancer therapy?

作者信息

Størvold Gro Leite, Andersen Tone Ikdahl, Perou Charles M, Frengen Eirik

机构信息

Institute of Medical Genetics, University of Oslo, Norway.

出版信息

Crit Rev Oncog. 2006 Jul;12(1-2):127-50. doi: 10.1615/critrevoncog.v12.i1-2.70.

Abstract

The siRNA technology is a promising research tool for use in functional genomics, and it also shows potential for use in future therapy. Thus far, siRNAs have been used to specifically silence disease-associated alleles in animal models. The technology is still quite new, and the biological mechanisms underlying RNAi and siRNA-mediated knockdown of gene expression are not yet fully understood. The main issues when siRNAs are designed are efficiency and specificity, and it is of great importance to consider possible off-target effects in the siRNA design. One major challenge in siRNA-based therapy is the development of systems for efficient delivery to the target cells. A large number of tools have, over the last few years, been designed for the delivery of DNA and RNA for gene therapy, and extensive efforts are now placed into developing clinical applications of siRNAs in a range of human diseases, including breast cancer.

摘要

小干扰RNA(siRNA)技术是功能基因组学中一种很有前景的研究工具,在未来治疗中也显示出应用潜力。到目前为止,siRNA已被用于在动物模型中特异性沉默与疾病相关的等位基因。该技术仍然相当新,RNA干扰(RNAi)和siRNA介导的基因表达敲低的生物学机制尚未完全了解。设计siRNA时的主要问题是效率和特异性,在siRNA设计中考虑可能的脱靶效应非常重要。基于siRNA的治疗的一个主要挑战是开发高效递送至靶细胞的系统。在过去几年中,已经设计了大量用于基因治疗的DNA和RNA递送工具,目前正在广泛努力开发siRNA在包括乳腺癌在内的一系列人类疾病中的临床应用。

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