Lentiviral-mediated delivery of siRNAs for antiviral therapy.
Lentiviral-mediated delivery of siRNAs for antiviral therapy.
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慢病毒介导的siRNA用于抗病毒疗法的递送。
DOI：
10.1038/sj.gt.3302688
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发表时间：
2006-03
期刊：
影响因子：
5.1
通讯作者：
Rossi JJ
中科院分区：
文献类型：
--
作者：
Morris KV;Rossi JJ
慢病毒载体预示着一个有前途的系统，提供抗病毒基因治疗病毒感染，如HIV-1，因为他们能够稳定转导分裂和非分裂细胞。最近，小干扰RNA（siRNA）已被证明在沉默靶基因方面非常有效。RNA干扰是一种天然机制，从酵母到人类在本质上是保守的，通过这种机制，siRNA在转录（靶向DNA）或转录后（靶向mRNA）特异性和有效地下调靶基因的表达。siRNA设计的特异性和相对简单性暗示siRNA将被证明是有利的治疗剂。由于siRNA是一种小的核酸试剂，它们不太可能引起免疫应答，并且编码这些siRNA的基因可以容易地被操纵并通过慢病毒载体递送到靶细胞。因此，表达siRNA的慢病毒载体代表了治疗病毒感染如HIV-1的潜在治疗方法。本文将重点介绍siRNAs的发展、慢病毒载体的构建及其在病毒感染治疗中的应用。
Lentiviral vectors portend a promising system to deliver antiviral genes for treating viral infections such as HIV-1 as they are capable of stably transducing both dividing and nondividing cells. Recently, small interfering RNAs (siRNAs) have been shown to be quite efficacious in silencing target genes. RNA interference is a natural mechanism, conserved in nature from Yeast to Humans, by which siRNAs operate to specifically and potently downregulate the expression of a target gene either transcriptionally (targeted to DNA) or post-transcriptionally (targeted to mRNA). The specificity and relative simplicity of siRNA design insinuate that siRNAs will prove to be favorable therapeutic agents. Since siRNAs are a small nucleic acid reagents, they are unlikely to elicit an immune response and genes encoding these siRNAs can be easily manipulated and delivered by lentiviral vectors to target cells. As such, lentiviral vectors expressing siRNAs represent a potential therapeutic approach for the treatment of viral infections such as HIV-1. This review will focus on the development, lentiviral based delivery, and the potential therapeutic use of siRNAs in treating viral infections.
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