Nonviral delivery of synthetic siRNAs in vivo

被引：389

作者：

Akhtar, Saghir

Benter, Ibrahim F.

机构：

[1] Kuwait Univ, Hlth Sci Ctr, Fac Med, Dept Pharmacol & Toxicol, Safat 13110, Kuwait

[2] SA Pharma, Sutton Coldfield, W Midlands, England

来源：

JOURNAL OF CLINICAL INVESTIGATION | 2007年 / 117卷 / 12期

关键词：

D O I：

10.1172/JCI33494

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

Sequence-specific gene silencing using small interfering RNA (siRNA) is a Nobel prize-winning technology that is now being evaluated in clinical trials as a potentially novel therapeutic strategy. This article provides an overview of the major pharmaceutical challenges facing siRNA therapeutics, focusing on the delivery strategies for synthetic siRNA duplexes in vivo, as this remains one of the most important issues to be resolved. This article also highlights the importance of understanding the genocompatibility/toxicogenomics of siRNA delivery reagents in terms of their impact on gene-silencing activity and specificity. Collectively, this information is essential for the selection of optimally acting siRNA delivery system combinations for the many proposed applications of RNA interference.

引用

收藏

页码：3623 / 3632

页数：10

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