Polyethylenimine as a Promising Vector for Targeted siRNA Delivery
Polyethylenimine as a Promising Vector for Targeted siRNA Delivery
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DOI：
10.2174/157488412800228857
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发表时间：
2012-01-01
影响因子：
3.2
通讯作者：
Nimesh, Surendra
中科院分区：
文献类型：
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作者：
Nimesh, Surendra
最近发现的用于基因治疗的RNA干扰(RNAi)技术引发了爆炸性的研究努力，开发小干扰RNA(SiRNA)作为基因沉默的治疗手段。由于其大分子量(约13 kDa)、多阴离子性质(类似于40个负磷酸基团)和快速的酶降解，siRNA的传递仍然是一个悬而未决的问题。因此，需要一种合适的递送载体来克服固有的、细胞内摄取差和体内外稳定性有限的问题。在各种非病毒载体中，以聚乙烯亚胺(PEI)或其衍生物为代表的聚合物载体因其高效、易操作而备受关注。PEI在DNA传递方面已经得到了广泛的研究，直到最近这种聚合物才被用于siRNA传递。这篇综述将集中在PEI传递siRNA方面的研究，重点是靶向、自组装的聚合物纳米粒子，这些纳米粒子有望成为基因治疗的治疗工具。
Recent discovery of RNA interference (RNAi) technology for gene therapy has triggered explosive research efforts towards development of small interfering RNA (siRNA) as therapeutic modality for gene silencing. Owing to its large molecular weight (similar to 13 kDa), polyanionic nature (similar to 40 negative phosphate groups) and rapid enzymatic degradation, delivery of siRNA remains an unresolved issue. Hence, there arises a need of an appropriate delivery vector to overcome the intrinsic, poor intracellular uptake and limited in vitro and in vivo stability. Amongst the various non-viral delivery vectors, the application of polymeric vectors such as polyethylenimine (PEI) or its derivatives has attracted much attention due to its high transfection efficiency and ease of manipulation. PEI has been extensively investigated for DNA delivery, only recently this polymer has been employed for siRNA delivery. This review will focus on studies done on PEI to deliver siRNA, with emphasis on the targeted, self-assembled polymeric nanoparticles with promising potential to evolve as therapeutic tool in gene therapy.