Dong Yizhou, Dorkin J Robert, Wang Weiheng, Chang Philip H, Webber Matthew J, Tang Benjamin C, Yang Junghoon, Abutbul-Ionita Inbal, Danino Dganit, DeRosa Frank, Heartlein Michael, Langer Robert, Anderson Daniel G
David H. Koch Institute for Integrative Cancer Research, ‡Department of Chemical Engineering, §Department of Biology, and ∥Institute for Medical Engineering and Science, Massachusetts Institute of Technology , Cambridge, Massachusetts 02139, United States.
Department of Anesthesiology, Children's Hospital Boston, Harvard Medical School , Boston, Massachusetts 02115, United States.
Nano Lett. 2016 Feb 10;16(2):842-8. doi: 10.1021/acs.nanolett.5b02428. Epub 2016 Jan 13.
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.
为了发挥基于小干扰RNA（siRNA）和信使核糖核酸（mRNA）疗法的潜力，需要安全有效的递送方式。在此，我们报道了聚（糖酰胺胺）刷状纳米颗粒作为siRNA和mRNA递送载体的研发情况。这些聚合物能够在小鼠体内有效递送针对凝血因子VII的siRNA以及编码促红细胞生成素（EPO）的mRNA。重要的是，基于组织组织学、全身细胞因子水平和肝脏酶化学分析，这些纳米颗粒在有效剂量下具有良好的耐受性。本文报道的聚合物刷状纳米颗粒在治疗应用方面具有广阔前景。
