Zhao Wenfang, Liu Kehai, Chen Shunsheng, Zhu Man Man, Lv Hui, Hu Jing, Mao Yuan
Department of Biopharmaceutics, College of Food Science and Technology, Shanghai Ocean University, Shanghai 201306, China.
Biomed Mater Eng. 2014;24(6):1933-9. doi: 10.3233/BME-141002.
To solve the contradiction between the cell toxicity and transfection efficiency of polyethylenimine (PEI) derivate in non-viral gene therapy, a novel gene vector, P123-PEI-R18 was synthesized by using biodegradable PEI derivate conjugated with trifunctional peptide RGD-TAT-NLS. The particle size of P123-PEI-R18/DNA was around 100-250 nm. The gene vector could condense DNA at the weight ratio of 2 and protect plasmid DNA from being dissolved in the blood circulation. Importantly, the complexes exhibited lower cell toxicity and higher transfection efficiency contrasted with PEI 25 kDa in vitro. P123-PEI-R18 holds high potential as a safe and efficient gene vector.
为了解决聚乙烯亚胺（PEI）衍生物在非病毒基因治疗中细胞毒性与转染效率之间的矛盾，通过将可生物降解的PEI衍生物与三功能肽RGD-TAT-NLS偶联，合成了一种新型基因载体P123-PEI-R18。P123-PEI-R18/DNA的粒径约为100-250nm。该基因载体能够以2的重量比凝聚DNA，并保护质粒DNA在血液循环中不被溶解。重要的是，与25kDa的PEI相比，该复合物在体外表现出较低的细胞毒性和较高的转染效率。P123-PEI-R18作为一种安全高效的基因载体具有很高的潜力。
