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Non-viral nanosystems for systemic siRNA delivery.

机译:用于系统性siRNA递送的非病毒纳米系统。

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摘要

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.
机译:为了将siRNA(小的干扰核糖核酸)用于全身给药,通常需要一种输送系统来克服给药与靶位点之间的障碍。这些传送系统需要不同的属性才能有效。一方面,它们必须保护siRNA免受降解和/或失活,另一方面,它们本身必须在血液中稳定并具有隐形特性,以避免消除和降解。主动和/或被动靶向应有助于输送系统达到所需的细胞类型或组织,进行内在化,并将siRNA输送至细胞质,以便siRNA可以通过RNA干扰起作用并抑制蛋白质合成。这篇综述介绍了已在体内进行评估或已进入临床试验的不同非病毒递送系统的概述,重点是其理化性质,以帮助开发作为治疗解决方案的新型高效siRNA递送系统明天

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