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小干扰RNA:作为治疗药物的潜力——第一部分。小干扰RNA的设计

siRNAs: their potential as therapeutic agents--Part I. Designing of siRNAs.

作者信息

Hajeri Praveensingh B, Singh Sunit Kumar

机构信息

Section of Infectious Diseases & Immunobiology, Centre for Cellular and Molecular Biology, Uppal Road, Hyderabad-500007, AP, India.

出版信息

Drug Discov Today. 2009 Sep;14(17-18):851-8. doi: 10.1016/j.drudis.2009.06.001. Epub 2009 Jun 21.

DOI:10.1016/j.drudis.2009.06.001
PMID:19540928
Abstract

RNA interference (RNAi) is a novel and essential biological process, as well as a powerful experimental tool with the potential to be used in therapeutic development. RNAi-based strategies have the capability of being able to be driven from bench to bedside. It is very important to develop the precise tools for designing the siRNAs to get the most efficient knockdown of the target genes and to reduce any off-target effects. In this review we have discussed the strategies and parameters required for effective siRNA designing and synthesis, based on already published literature.

摘要

RNA干扰(RNAi)是一种新颖且重要的生物学过程,也是一种有潜力用于治疗开发的强大实验工具。基于RNAi的策略有能力从实验室走向临床应用。开发精确的工具来设计小干扰RNA(siRNA)以最有效地敲低靶基因并减少任何脱靶效应非常重要。在本综述中,我们根据已发表的文献讨论了有效设计和合成siRNA所需的策略和参数。

相似文献

1
siRNAs: their potential as therapeutic agents--Part I. Designing of siRNAs.小干扰RNA:作为治疗药物的潜力——第一部分。小干扰RNA的设计
Drug Discov Today. 2009 Sep;14(17-18):851-8. doi: 10.1016/j.drudis.2009.06.001. Epub 2009 Jun 21.
2
On the art of identifying effective and specific siRNAs.论有效且特异的小干扰RNA的鉴定技术
Nat Methods. 2006 Sep;3(9):670-6. doi: 10.1038/nmeth911.
3
siRNAs: their potential as therapeutic agents--Part II. Methods of delivery.小干扰RNA:作为治疗药物的潜力——第二部分。递送方法。
Drug Discov Today. 2009 Sep;14(17-18):859-65. doi: 10.1016/j.drudis.2009.06.002. Epub 2009 Jun 21.
4
The impact of target site accessibility on the design of effective siRNAs.靶位点可及性对有效小干扰RNA设计的影响。
Nat Biotechnol. 2008 May;26(5):578-83. doi: 10.1038/nbt1404. Epub 2008 Apr 27.
5
Short interfering RNA (siRNA): tool or therapeutic?小干扰RNA(siRNA):工具还是疗法?
Clin Sci (Lond). 2006 Jan;110(1):47-58. doi: 10.1042/CS20050162.
6
In silico selection of active siRNA.活性小干扰RNA的计算机筛选
Drug Discov Today. 2007 Feb;12(3-4):139-48. doi: 10.1016/j.drudis.2006.11.015. Epub 2006 Dec 13.
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Computational siRNA design considering alternative splicing.考虑可变剪接的计算性小干扰RNA设计
Methods Mol Biol. 2010;623:81-92. doi: 10.1007/978-1-60761-588-0_5.
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The silent treatment: siRNAs as small molecule drugs.沉默疗法:作为小分子药物的小干扰RNA
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Designing optimal siRNA based on target site accessibility.基于靶位点可及性设计最佳小干扰RNA
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A protocol for designing siRNAs with high functionality and specificity.一种用于设计具有高功能性和特异性的小干扰RNA(siRNA)的方案。
Nat Protoc. 2007;2(9):2068-78. doi: 10.1038/nprot.2007.278.

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Inhibition of Respiratory Syncytial Virus Replication by Simultaneous Targeting of mRNA and Genomic RNA Using Dual-Targeting siRNAs.使用双靶点小干扰RNA同时靶向mRNA和基因组RNA抑制呼吸道合胞病毒复制
Mol Biotechnol. 2016 Nov;58(11):767-775. doi: 10.1007/s12033-016-9976-4.
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Preclinical and clinical development of siRNA-based therapeutics.基于小干扰RNA的治疗药物的临床前和临床开发。
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