Advances in Systemic siRNA Delivery.
Advances in Systemic siRNA Delivery.
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DOI：
10.1358/dof.2009.034.09.1413267
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发表时间：
2009-09
影响因子：
0.2
通讯作者：
Mixson AJ
中科院分区：
文献类型：
--
作者：
Leng Q;Woodle MC;Lu PY;Mixson AJ
小干扰RNA（siRNA）的序列特异性基因沉默已经改变了基础科学研究，目前正在临床前和临床试验中评估siRNA治疗多种疾病的疗效。尽管具有潜在价值，但高度带负电荷的SiRNA具有典型的递送问题，即需要跨细胞膜转运到胞质溶胶。因此，siRNA递送的载体开发是siRNA实现广泛临床应用之前要解决的最重要问题之一。正在评估包括脂质体、肽、聚合物和适体在内的各种非病毒载体将siRNA引导至靶组织并穿过质膜屏障进入细胞的能力。对于基于siRNA的治疗，已经出现了几种具有低毒性和对疾病靶点的特异性增加的有前途的载体。这篇综述将讨论siRNA治疗的非病毒方法，特别关注用于siRNA体内系统递送的合成载体。
Sequence-specific gene silencing with small interfering RNA (siRNA) has transformed basic science research, and the efficacy of siRNA therapeutics toward a variety of diseases is now being evaluated in pre-clinical and clinical trials. Despite its potential value, the highly negatively charged siRNA has the classic delivery problem of requiring transport across cell membranes to the cytosol. Consequently, carrier development for siRNA delivery is one of the most important problems to solve before siRNA can achieve widespread clinical use. An assortment of non-viral carriers including liposomes, peptides, polymers, and aptamers are being evaluated for their ability to shepherd siRNA to the target tissue and cross the plasma membrane barrier into the cell. Several promising carriers with low toxicity and increased specificity for disease targets have emerged for siRNA-based therapeutics. This review will discuss non-viral approaches for siRNA therapeutics, with particular focus on synthetic carriers for in vivo systemic delivery of siRNA.
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影响因子：
14.9
作者：
Bartlett DW;Davis ME
通讯作者：
Davis ME
影响因子：
10.8
作者：
Dobrovoiskaia, Marina A.;Clogston, Jeffrey D.;Neun, Barry W.;Hall, Jennifer B.;Patri, Anil K.;McNeil, Scott E.
通讯作者：
McNeil, Scott E.
影响因子：
14.9
作者：
De Souza AT;Dai X;Spencer AG;Reppen T;Menzie A;Roesch PL;He Y;Caguyong MJ;Bloomer S;Herweijer H;Wolff JA;Hagstrom JE;Lewis DL;Linsley PS;Ulrich RG
通讯作者：
Ulrich RG
影响因子：
5.3
作者：
Davidson, TJ;Harel, S;Troy, CM
通讯作者：
Troy, CM
影响因子：
--
作者：
Chiu, YL;Ali, A;Rana, TM
通讯作者：
Rana, TM