在体内将治疗性rna递送到靶细胞

M. Ng, T. Hagen
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引用次数: 0

摘要

基于rna的疗法是治疗人类疾病最有前途的方法之一。具体来说,使用短干扰RNA (siRNA) siRNA和microRNA (miRNA)模拟物进行体内RNA干扰具有巨大的潜力,因为它可以直接降低治疗靶蛋白的表达。然而,在临床上成功实施siRNA和其他基于RNA的疗法存在许多主要障碍。其中包括RNA在体内的不稳定性和难以有效地将RNA递送到靶细胞中。因此,多年来已经采取了各种创新方法来开发有效的RNA递送方法。这些方法包括脂质体,聚合物纳米颗粒和肽介导的细胞递送。在最近的一项创新研究中,生物工程细菌外膜囊泡被用作siRNA有效递送到体内细胞的载体。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
DELIVERY OF THERAPEUTIC RNAs INTO TARGET CELLS IN VIVO
RNA-based therapy is one of the most promising approaches to treat human diseases. Specifically, the use of short interfering RNA (siRNA) siRNA and microRNA (miRNA) mimics for in vivo RNA interference has immense potential as it directly lowers the expression of the therapeutic target protein. However, there are a number of major roadblocks to the successful implementation of siRNA and other RNA based therapies in the clinic. These include the instability of RNAs in vivo and the difficulty to efficiently deliver the RNA into the target cells. Hence, various innovative approaches have been taken over the years to develop effective RNA delivery methods. These methods include liposome-, polymeric nanoparticle- and peptide-mediated cellular delivery. In a recent innovative study, bioengineered bacterial outer membrane vesicles were used as vehicles for effective delivery of siRNA into cells in vivo.
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