疏水聚丙氨酸修饰的超支化聚乙烯亚胺作为高效的质粒DNA和小干扰RNA载体
Hydrophobic polyalanine modified hyperbranched polyethylenimine as high efficient pDNA and siRNA carrier.
作者信息
Guo Zhaopei, Tian Huayu, Lin Lin, Chen Jie, He Chaoliang, Tang Zhaohui, Chen Xuesi
机构信息
Key Laboratory of Polymer Ecomaterials, Changchun Institute of Applied Chemistry, Chinese Academy of Sciences, Changchun, 130022, China.
出版信息
Macromol Biosci. 2014 Oct;14(10):1406-14. doi: 10.1002/mabi.201400044. Epub 2014 Jun 20.
Successful gene therapy for cancer treatment and various human genetic diseases relies on high efficient gene carriers, and many carriers can efficiently deliver plasmid DNA to cells, while most of them are unefficient in siRNA delivery. In this study, a series of amphiphilic copolymers are synthesized for pDNA and siRNA delivery by grafting hydrophobic polyalanine to polyethylenimine (named PPAs). There are 12.5, 7.2, 12.3 and 11.3 times of gene expression more than PEI in HeLa, 293T, A549 and CHO cell lines for the optimal PPAs, and the highest luciferase knockdown efficiency in Huh7 and CT26 cells are investigated to be 90.4% and 89.0%, respectively. This meant the PPAs have the potential as efficient transfection reagents for pDNA and siRNA delivery in future gene therapy.
癌症治疗和各种人类遗传疾病的成功基因治疗依赖于高效的基因载体，许多载体可以有效地将质粒DNA递送至细胞，但其中大多数在递送小干扰RNA（siRNA）方面效率不高。在本研究中，通过将疏水性聚丙氨酸接枝到聚乙烯亚胺上（命名为PPA），合成了一系列用于递送质粒DNA和siRNA的两亲性共聚物。对于最佳的PPA，在HeLa、293T、A549和CHO细胞系中，基因表达分别比聚乙烯亚胺多12.5、7.2、12.3和11.3倍，并且在Huh7和CT26细胞中研究的最高荧光素酶敲低效率分别为90.4%和89.0%。这意味着PPA在未来基因治疗中具有作为高效转染试剂用于递送质粒DNA和siRNA的潜力。
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