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用于系统性 siRNA 递送的非病毒纳米系统。

Non-viral nanosystems for systemic siRNA delivery.

机构信息

INSERM U646, Université d'Angers, 49100 Angers, France.

出版信息

Pharmacol Res. 2010 Aug;62(2):100-14. doi: 10.1016/j.phrs.2009.11.013. Epub 2009 Dec 17.

Abstract

To use siRNA (small interfering ribonucleic acids) for systemic administration, a delivery system is often necessary to overcome barriers between administration and the target sites. These delivery systems require different properties to be efficient. On the one hand, they have to protect siRNA from degradation and/or inactivation and, on the other hand, they have themselves to be stable in blood and possess stealth properties to avoid elimination and degradation. Active and/or passive targeting should help the delivery system to reach the desired cell type or tissue, to be internalised, and to deliver siRNA to the cytoplasm so that siRNA can act by RNA interference and inhibit protein synthesis. This review presents an overview of different non-viral delivery systems, which have been evaluated in vivo or entered in clinical trials, with a focus on their physicochemical properties in order to help the development of new and efficient siRNA delivery systems, as the therapeutic solutions of tomorrow.

摘要

为了将 siRNA(小干扰核糖核酸)用于全身给药,通常需要一种输送系统来克服给药和靶位之间的障碍。这些输送系统需要不同的特性才能发挥作用。一方面,它们必须保护 siRNA 免受降解和/或失活,另一方面,它们在血液中必须稳定,并具有隐身特性以避免消除和降解。主动和/或被动靶向应有助于输送系统到达所需的细胞类型或组织,被内化,并将 siRNA 递送至细胞质,以便 siRNA 通过 RNA 干扰起作用并抑制蛋白质合成。本综述介绍了不同的非病毒输送系统的概述,这些系统已经在体内进行了评估或进入临床试验,重点介绍了它们的物理化学特性,以帮助开发新的有效的 siRNA 输送系统,作为明天的治疗解决方案。

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