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Cationic lipid nanoemulsions for RNAi screening

机译:用于RNAi筛选的阳离子脂质纳米乳剂

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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:自从发现RNA干扰以来,在研究和临床工作中已经做出了许多努力来拓宽小干扰RNA(siRNA)的视野。 siRNA通过互补信使RNA的序列特异性下调显示出特定而有效的基因沉默活性,以及​​作为治疗多种疾病的治疗剂的巨大潜力。但是,siRNA介导的基因敲低要求siRNA可以到达发生mRNA干扰机制的细胞质。不幸的是,由于存在降解酶,siRNA在细胞外环境中相对脆弱,并且其高分子量与其阴离子电荷相关,从而大大限制了其细胞跨质膜的吸收。为了克服这些障碍,包含成像造影剂的多功能纳米粒子作为改进和监测siRNA传递的一种原始且有前途的方法正在出现。

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