title: Synthesis and characterization of low molecular weight polyethyleneimine-terminated Poly(β-amino ester) for highly efficient gene delivery of minicircle DNA.
abstract: Gene therapy has held great promise for treating specific acquired and inherited diseases. However, the lack of safe and efficient gene delivery systems remains as the major challenge. Poly(β-amino ester)s (PBAEs) have attracted much attention due to their outstanding properties in biosafety, DNA delivery efficiency and convenience in synthesis. In this paper, we reported the further enhancement of the PBAE functions by increasing its positive charge through conjugating with low molecular weight polyethylenimine (LPEI). The resulted LPEI-PBAE polymer was able to condense minicircle DNA (mcDNA) forming nanoparticles with a diameter of 50-200nm. Furthermore, as compared to parental PBAE and a commercial transfection reagent very common in laboratory application, the LPEI-PBAE demonstrated significantly higher transfection efficiency with little cytotoxicity. These results suggested LPEI-PBAEs are worthy of further optimization for gene therapy applications.
authors.affiliation: Center for Gene and Cell Engineering, Institute of Biomedicine and Biotechnology, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, PR China.
authors.forename: Jing
authors.initials: J
authors.lastname: Zhao
authors.affiliation: Center for Gene and Cell Engineering, Institute of Biomedicine and Biotechnology, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, PR China.
authors.forename: Lei
authors.initials: L
authors.lastname: Yang
authors.affiliation: Center for Gene and Cell Engineering, Institute of Biomedicine and Biotechnology, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, PR China.
authors.forename: Ping
authors.initials: P
authors.lastname: Huang
authors.affiliation: Center for Gene and Cell Engineering, Institute of Biomedicine and Biotechnology, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, PR China; Paul C. Lauterbur Research Center for Biomedical Imaging, Shenzhen Key Laboratory for MRI, Institute of Biomedical and Health Engineering, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, PR China. Electronic address: zy.wang@siat.ac.cn.
authors.forename: ZhiYong
authors.initials: Z
authors.lastname: Wang
authors.affiliation: Paul C. Lauterbur Research Center for Biomedical Imaging, Shenzhen Key Laboratory for MRI, Institute of Biomedical and Health Engineering, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, PR China.
authors.forename: Yan
authors.initials: Y
authors.lastname: Tan
authors.affiliation: Paul C. Lauterbur Research Center for Biomedical Imaging, Shenzhen Key Laboratory for MRI, Institute of Biomedical and Health Engineering, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, PR China.
authors.forename: Hong
authors.initials: H
authors.lastname: Liu
authors.affiliation: Center for Gene and Cell Engineering, Institute of Biomedicine and Biotechnology, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, PR China.
authors.forename: JiaJia
authors.initials: J
authors.lastname: Pan
authors.affiliation: Center for Gene and Cell Engineering, Institute of Biomedicine and Biotechnology, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, PR China.
authors.forename: Cheng-Yi
authors.initials: CY
authors.lastname: He
authors.affiliation: Center for Gene and Cell Engineering, Institute of Biomedicine and Biotechnology, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, PR China. Electronic address: zy.chen1@siat.ac.cn.
authors.forename: Zhi-Ying
authors.initials: ZY
authors.lastname: Chen
mesh_terms: D000818:Animals; D002460:Cell Line; D004270:DNA, Circular / Q000008:administration & dosage* / Q000737:chemistry* / Q000235:genetics; D018014:Gene Transfer Techniques; D006801:Humans; D008970:Molecular Weight; D053758:Nanoparticles / Q000009:adverse effects / Q000737:chemistry; D010316:Particle Size; D011094:Polyethyleneimine / Q000009:adverse effects / Q000737:chemistry*; D011108:Polymers / Q000009:adverse effects / Q000138:chemical synthesis* / Q000737:chemistry*; D013499:Surface Properties; D013552:Swine
chemical_list: D004270:DNA, Circular; D011108:Polymers; C507253:poly(beta-amino ester); D011094:Polyethyleneimine
keywords: Gene delivery; Minicircle DNA; Non-viral vectors; Poly(β-amino ester)s; Polyethylenimine