Abstract RNA interference is an effective and naturally occurring post-transcriptional gene regulatory mechanism. This mechanism involves the degradation of a target messenger RNA (mRNA) through the introduction of short interfering RNA (siRNA) that is complementary to the target mRNA. The application of siRNA-based therapeutics is limited by the development of an effective delivery system, as naked siRNA is unstable and cannot penetrate the cell membrane. In this study, we investigated the use of cationic niosomes (CN) prepared by microfluidic mixing for siRNA delivery. In an in vitro model, these vesicles were able to deliver anti-luciferase siRNA and effectively suppress luciferase expression in B16-F10 mouse melanoma cells. More importantly, in an in vivo mouse model, intratumoral administration of CN-carrying anti-luciferase siRNA led to significant suppression of luciferase expression compared with naked siRNA. Thus, we have established a novel and effective system for the delivery of siRNA both in vitro and in vivo, which shows high potential for future application of gene therapeutics.

中文翻译：

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通过非离子表面活性剂囊泡传递 siRNA 的概念研究证明：蛋白质敲低的体外和体内评估

摘要 RNA 干扰是一种有效且自然发生的转录后基因调控机制。该机制涉及通过引入与目标 mRNA 互补的短干扰 RNA (siRNA) 来降解目标信使 RNA (mRNA)。基于 siRNA 的疗法的应用受到有效递送系统开发的限制，因为裸 siRNA 不稳定且不能穿透细胞膜。在这项研究中，我们研究了通过微流体混合制备的阳离子 niosomes (CN) 用于 siRNA 递送的用途。在体外模型中，这些囊泡能够传递抗荧光素酶 siRNA 并有效抑制 B16-F10 小鼠黑色素瘤细胞中的荧光素酶表达。更重要的是，在体内小鼠模型中，与裸 siRNA 相比，携带 CN 的抗荧光素酶 siRNA 的瘤内给药导致荧光素酶表达的显着抑制。因此，我们已经建立了一种新的、有效的 siRNA 体外和体内递送系统，这显示了基因治疗未来应用的巨大潜力。