Strategies for targeted nonviral delivery of siRNAs in vivo.
Strategies for targeted nonviral delivery of siRNAs in vivo.
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DOI：
10.1016/j.molmed.2009.09.001
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发表时间：
2009-11
影响因子：
13.6
通讯作者：
Manjunath N
中科院分区：
文献类型：
--
作者：
Kim SS;Garg H;Joshi A;Manjunath N
通过RNA干扰（RNAi）沉默特定基因表达已迅速成为基因功能反向遗传分析的标准工具。它在治疗目前没有有效治疗方法或治疗效果欠佳的疾病方面也具有巨大潜力。然而，合成的小干扰RNA（siRNA）的细胞摄取差是其临床应用的主要障碍。近年来在克服这一障碍方面取得了很大进展，并且已经描述了几种用于体内递送siRNA的方法。此外，最新进展集中在实现限制于体内相关组织和细胞类型的靶向siRNA递送。这些方法有望降低剂量需求，并使siRNA诱导的毒性最小化，从而将siRNA治疗领域推向临床应用。
Silencing specific gene expression by RNA interference (RNAi) has rapidly become a standard tool for reverse-genetic analysis of gene functions. It also has a tremendous potential in the treatment of diseases for which currently effective treatment is not available or is suboptimal. However, the poor cellular uptake of synthetic small interfering RNAs (siRNAs) is a major impediment for their clinical use. Great progress has been made in recent years to overcome this barrier and several methods have been described for in vivo delivery of siRNA. Moreover, latest advances have focused on achieving targeted siRNA delivery restricted to relevant tissues and cell types in vivo. These approaches are expected to reduce the dose requirement as well as minimize siRNA-induced toxicities, thereby advancing the field of siRNA therapy towards clinical use.
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影响因子：
12.4
作者：
Howard, Kenneth A.;Rahbek, Ulrik L.;Kjems, Jorgen
通讯作者：
Kjems, Jorgen
影响因子：
--
作者：
Chiu, YL;Ali, A;Rana, TM
通讯作者：
Rana, TM
影响因子：
10.5
作者：
Babiarz, Joshua E.;Ruby, J. Graham;Blelloch, Robert
通讯作者：
Blelloch, Robert
影响因子：
64.8
作者：
Castanotto, Daniela;Rossi, John J.
通讯作者：
Rossi, John J.
DOI：
10.1038/nrd2310
发表时间：
2007-06
期刊：
Nature reviews. Drug discovery
影响因子：
--
作者：
de Fougerolles A;Vornlocher HP;Maraganore J;Lieberman J
通讯作者：
Lieberman J