Poly(glycoamidoamine) Brushes Formulated Nanomaterials for Systemic siRNA and mRNA Delivery in Vivo.
Poly(glycoamidoamine) Brushes Formulated Nanomaterials for Systemic siRNA and mRNA Delivery in Vivo.
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聚（糖氨基胺）刷子在体内制定了用于全身siRNA和mRNA递送的纳米材料。
DOI：
10.1021/acs.nanolett.5b02428
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发表时间：
2016-02-10
期刊：
影响因子：
10.8
通讯作者：
Anderson DG
中科院分区：
文献类型：
--
作者：
Dong Y;Dorkin JR;Wang W;Chang PH;Webber MJ;Tang BC;Yang J;Abutbul-Ionita I;Danino D;DeRosa F;Heartlein M;Langer R;Anderson DG
需要安全有效地提供siRNA和基于信使核糖核酸的疗法，才能发挥其潜力。在这里，我们报道了聚糖胺胺刷状纳米颗粒作为siRNA和mRNAs载体的研究进展。这些聚合物能够在小鼠体内显著传递针对FVII的siRNA和编码促红细胞生成素(EPO)的mRNA。重要的是，根据组织组织学、全身细胞因子水平和肝酶化学的分析，这些纳米粒在其有效剂量下耐受性良好。本文报道的聚合物刷子纳米粒在治疗应用方面很有前途。
Safe and effective delivery is required for siRNA and mRNA-based therapeutics to reach their potential. Here, we report on the development of poly(glycoamidoamine) brush nanoparticles as delivery vehicles for siRNA and mRNA. These polymers were capable of significant delivery of siRNA against FVII and mRNA-encoding erythropoietin (EPO) in mice. Importantly, these nanoparticles were well-tolerated at their effective dose based on analysis of tissue histology, systemic cytokine levels, and liver enzyme chemistry. The polymer brush nanoparticles reported here are promising for therapeutic applications.