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Lipid-based colloidal carriers for targeted siRNA delivery

机译:基于脂质的胶体载体,用于靶向性siRNA递送

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Since the groundbreaking and Nobel Prize-winning discovery of the naturally occurring RNAi cellular defense mechanism [1], there has been great expectations for the therapeutic exploitation of its mediator: siRNA [2]. In the cytoplasm of all eukaryotic cells, siRNA stimulates potent gene silencing upon incorporation into the RNA-induced silencing complex that catalyzes the cleavage of mRNA with complementary sequence, eventually resulting in down-regulation of target gene expression. RNAi-based therapeutics have a significant potential since any gene in the genome can, in principle, be targeted. Therefore, siRNA might be used ubiquitously for the treatment of a broad spectrum of diseases caused by the overexpression of one or multiple genes
机译:自从自然界中存在的RNAi细胞防御机制[1]破获诺贝尔奖以来,人们对其siRNA [2]的治疗方法有了很高的期望。在所有真核细胞的细胞质中,siRNA掺入RNA诱导的沉默复合物后会刺激有效的基因沉默,该复合物催化具有互补序列的mRNA裂解,最终导致靶基因表达的下调。基于RNAi的治疗方法具有巨大的潜力,因为原则上基因组中的任何基因都可以被靶向。因此,siRNA可能普遍用于治疗由一个或多个基因过表达引起的广泛疾病

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