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A Fabricated siRNA Nanoparticle for Ultralong Gene Silencing In Vivo

机译：体内可用于超长基因沉默的siRNA纳米颗粒。

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摘要

Persistent gene silencing is crucially required for the successful therapeutics of short interfering RNA (siRNA). Here, a nanoparticle-based delivery system is presented which assembles by layering siRNAs between protease degra-dable polypeptides to extend the therapeutic window. These tightly packed nanoparticles are efficiently taken up by cells by endocytosis, and the fabricated siRNAs are gradually released following intracellular degradation of the polypeptide layers. During cell division, the particles are distributed to the daughter cells. Due to the slow degradation through the multiple layers, the particles continuously release siRNA in all cells. Using this controlled release construct, the in vivo gene silencing effect of siRNA is consistent for an ultralong period of time (＞3 weeks) with only a single treatment.
机译：持久的基因沉默对于短干扰RNA（siRNA）的成功治疗至关重要。在此，提出了一种基于纳米颗粒的递送系统，该系统通过在可蛋白酶降解的多肽之间分层siRNA进行组装以扩展治疗范围。这些紧密堆积的纳米颗粒通过细胞内吞作用被细胞有效吸收，并且在多肽层的细胞内降解后，所制造的siRNA逐渐释放。在细胞分裂期间，颗粒被分布到子细胞。由于穿过多层的缓慢降解，颗粒在所有细胞中连续释放siRNA。使用这种控释构建体，只需一次治疗，siRNA的体内基因沉默效果在超长的时间段（＞ 3周）内是一致的。

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  • 来源
    《Advanced Functional Materials》 |2013年第28期|3488-3493|共6页
  • 作者

    Seung Koo Lee; Ching-Hsuan Tung;

  • 作者单位

    Department of Translational Imaging The Methodist Hospital Research Institute Weill Cornell Medical College, Houston, TX 77030, USA;

    Department of Translational Imaging The Methodist Hospital Research Institute Weill Cornell Medical College, Houston, TX 77030, USA;

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