New paradigms on siRNA local application.
New paradigms on siRNA local application.
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DOI：
10.5483/bmbrep.2015.48.3.089
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发表时间：
2015-03
期刊：
影响因子：
3.8
通讯作者：
Duan X
中科院分区：
文献类型：
--
作者：
Pan M;Ni J;He H;Gao S;Duan X
小干扰RNA(SiRNA)通过与特定的mRNA序列配对而发挥作用，导致mRNA的降解。这是一种潜在的治疗方法，可用于许多由基因表达改变引起的疾病。由于siRNA在循环中迅速降解，它的传递仍然是一个主要问题。已经提出了各种策略来帮助细胞摄取siRNA和短或小发夹RNA(ShRNA)。在这里，我们回顾了最近发表的关于siRNA局部应用的数据。与全身递送方法相比，局部递送siRNA/shRNA具有许多优点，如靶向特定的组织或器官，模仿基因敲除效应，或建立特定的疾病模型。眼、脑和肿瘤组织是局部siRNA传递的“热门”目标组织/器官。SiRNA可以以裸露形式、经过化学修饰或以含有病毒或非病毒载体的制剂(如脂质体和纳米颗粒)在本地传递。本综述对RNAi局部给药及其在临床治疗中的潜在应用进行了全面综述。[BMB报告2015；48(3)：147-152]
Small interfering RNA (siRNA) functions through pairing with specific mRNA sequences and results in the mRNA’s degradation. It is a potential therapeutic approach for many diseases caused by altered gene expression. The delivery of siRNA is still a major problem due to its rapid degradation in the circulation. Various strategies have been proposed to help with the cellular uptake of siRNA and short or small hairpin RNA (shRNA). Here, we reviewed recently published data regarding local applications of siRNA. Compared with systemic delivery methods, local delivery of siRNA/shRNA has many advantages, such as targeting the specific tissues or organs, mimicking a gene knockout effect, or developing certain diseases models. The eye, brain, and tumor tissues are ‘hot’ target tissues/organs for local siRNA delivery. The siRNA can be delivered locally, in naked form, with chemical modifications, or in formulations with viral or non-viral vectors, such as liposomes and nanoparticles. This review provides a comprehensive overview of RNAi local administration and potential future applications in clinical treatment. [BMB Reports 2015; 48(3): 147-152]
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影响因子：
46.9
作者：
Dassie, Justin P.;Liu, Xiu-ying;Thomas, Gregory S.;Whitaker, Ryan M.;Thiel, Kristina W.;Stockdale, Katie R.;Meyerholz, David K.;McCaffrey, Anton P.;McNamara, James O., II;Giangrande, Paloma H.
通讯作者：
Giangrande, Paloma H.
影响因子：
16.1
作者：
Guo, Peixuan;Coban, Oana;Snead, Nicholas M.;Trebley, Joe;Hoeprich, Steve;Guo, Songchuan;Shu, Yi
通讯作者：
Shu, Yi
影响因子：
64.5
作者：
Lima, Walt F.;Prakash, Thazha P.;Crooke, Stanley T.
通讯作者：
Crooke, Stanley T.
DOI：
10.1016/j.jconrel.2014.01.012
发表时间：
2014-03-28
期刊：
Journal of controlled release : official journal of the Controlled Release Society
影响因子：
--
作者：
Wang J;Lu Z;Yeung BZ;Wientjes MG;Cole DJ;Au JL
通讯作者：
Au JL
影响因子：
17.1
作者：
Brock A;Krause S;Li H;Kowalski M;Goldberg MS;Collins JJ;Ingber DE
通讯作者：
Ingber DE