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首页> 外文期刊>International Journal of Pharmaceutics >Novel hyperbranched polyamidoamine nanoparticle based gene delivery: Transfection, cytotoxicity and in vitro evaluation
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Novel hyperbranched polyamidoamine nanoparticle based gene delivery: Transfection, cytotoxicity and in vitro evaluation

机译:基于超支化聚酰胺胺纳米粒子的新型基因传递:转染,细胞毒性和体外评估

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In this study, hyperbranched polyamidoamine (hPAMAM) was developed as a novel non-viral gene vector for the first time. The hPAMAM was synthesized using a modified "one-pot" method. DNA was then bound to hPAMAM at different weight ratios (w hPAMAM/w DNA). The higher weight ratio could bring larger particle size and higher zeta potential of hPAMAM-DNA complexes. The encapsulated DNA was protected by hPAMAM from degradation for over 3 h. Under the optimal condition, high gene transfection efficiency could be achieved in COS7 (47.47 ± 1.42%) and HEK293 (40.8 ± 0.98%) cell lines. And hPAMAM showed rather minor cytotoxicity in vitro (cell viability = 91.38 ± 0.46% in COS7 and 92.38 ± 0.61% in HEK293). The hPAMAM mediated human vascular endothelial growth factor 165 (hVEGF 165) gene transfected cells could express hVEGF 165 stably for 14 days, with the peak expression at day 2. In conclusion, hPAMAM based gene delivery was economical, effective and biocompatible, and may serve as a promising non-viral vehicle for gene therapy.
机译:在这项研究中,超支化聚酰胺胺(hPAMAM)首次被开发为一种新型的非病毒基因载体。使用改良的“一锅法”合成hPAMAM。然后将DNA以不同的重量比(w hPAMAM / w DNA)与hPAMAM结合。较高的重量比可带来更大的粒径和更高的hPAMAM-DNA复合物的ζ电位。 hPAMAM保护封装的DNA超过3小时不被降解。在最佳条件下,COS7(47.47±1.42%)和HEK293(40.8±0.98%)细胞系可实现高基因转染效率。 hPAMAM在体外显示出较小的细胞毒性(COS7中的细胞活力= 91.38±0.46%,而HEK293中的细胞活力= 92.38±0.61%)。 hPAMAM介导的人血管内皮生长因子165(hVEGF 165)基因转染的细胞可以稳定表达hVEGF 165 14天,在第2天达到峰值。总之,基于hPAMAM的基因传递是经济,有效和生物相容的,可能有用作为有希望的基因治疗非病毒载体。

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