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Short interfering RNA therapeutics: nanocarriers, prospects and limitations

机译:短干扰RNA疗法:纳米载体,前景和局限性

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摘要

Since the first experiment depicting gene inhibition using RNA interference mechanism, extensive research has been carried out to design targeted delivery systems that use short interfering RNAs (siRNAs) for gene expression regulation. Although several siRNAs loaded nanoparticle systems have reached clinical trial stage, cellular uptake, reticuloendothelial entrapment and endosomal escape still limit the efficacy of these drugs considerably. This review discusses about the RNA interference mechanism, nanostructures being used as non-viral vectors for targeted delivery, limitations of the common delivery systems and the current siRNA-loaded nanoparticle formulations undergoing clinical testing.
机译:自从使用RNA干扰机制描述基因抑制的第一个实验以来,已经进行了广泛的研究来设计使用短干扰RNA(siRNA)进行基因表达调控的靶向递送系统。尽管几种载有siRNA的纳米颗粒系统已进入临床试验阶段,但细胞摄取,网状内皮捕获和内体逃逸仍大大限制了这些药物的疗效。这篇综述讨论了RNA干扰机制,用作非病毒载体进行靶向递送的纳米结构,常见递送系统的局限性以及目前正在接受临床测试的载有siRNA的纳米颗粒制剂。

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