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Cationic lipid nanoemulsions for RNAi screening

机译:RNAi筛选的阳离子脂纳米乳液

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Since the discovery of RNA interference, many efforts have been made to broaden perspectives of small interfering RNA (siRNA) in research and clinical work. siRNA shows a specific and effective gene silencing activity through a sequence specific down-regulation of the complementary messenger RNA as well as a tremendous potential as therapeutic agent for treatment of numerous diseases. However, the siRNA-mediated gene knock down requires that siRNA can reach the cytoplasm, where mRNA interference mechanism occurs. Unfortunately, the siRNA is relatively vulnerable in the extracellular environment due to the presence of degradation enzymes and its high molecular weight associated to its anionic charge limit considerably its cell incorporation across the. plasma membrane. To overcome these obstacles, multifunctional nanoparticles comprising an imaging contrast agent are emerging as an original and promising approach in the improved and monitored delivery of siRNA.
机译:由于发现RNA干扰,因此已经促进了许多努力来扩大小干扰RNA(siRNA)在研究和临床工作中的视角。 siRNA通过互补信使RNA的序列特异性下调以及作为治疗许多疾病治疗的治疗剂的巨大潜力,显示特异性和有效的基因沉默活性。然而,siRNA介导的基因崩溃要求siRNA可以到达mRNA干扰机理的细胞质。遗憾的是,由于存在降解酶的存在,并且其与其阴离子电荷限制的高分子量相当于其细胞掺入,SiRNA在细胞外环境中相对容易受到伤害。质膜。为了克服这些障碍,包含成像造影剂的多功能纳米颗粒在改进和监测的siRNA递送中被出现为原始和有前途的方法。

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