Lai Wing-Fu, Jung Han-Sung
Division in Anatomy and Developmental Biology, Department of Oral Biology, Oral Science Research Center, BK21 PLUS Project, Yonsei University College of Dentistry, Seoul, Korea.
Division in Anatomy and Developmental Biology, Department of Oral Biology, Oral Science Research Center, BK21 PLUS Project, Yonsei University College of Dentistry, Seoul, Korea; Oral Biosciences, Faculty of Dentistry, The University of Hong Kong, Hong Kong SAR.
PLoS One. 2014 Jun 23;9(6):e100258. doi: 10.1371/journal.pone.0100258. eCollection 2014.
Successful gene therapy necessitates safe and efficient gene transfer. This article describes the use of a cationic polymer, which was synthesized by cross-linking low molecular weight branched poly(ethylenimine) (PEI) with both β-cyclodextrin and propane-1,2,3-triol, for efficient and safe non-viral gene delivery. Experimentation demonstrated that the polymer had a pH buffering capacity and DNA condensing ability comparable to those of PEI 25 kDa. In B16-F0 cells, the polymer increased the transfection efficiency of naked DNA by 700-fold and yielded better transfection efficiencies than Fugene HD (threefold higher) and PEI 25 kDa (fivefold higher). The high transfection efficiency of the polymer was not affected by the presence of serum during transfection. In addition to B16-F0 cells, the polymer enabled efficient transfection of HepG2 and U87 cells with low cytotoxicity. Our results indicated that our polymer is a safe and efficient transfection reagent that warrants further development for in vitro, in vivo and clinical applications.
成功的基因治疗需要安全有效的基因传递。本文描述了一种阳离子聚合物的应用,该聚合物是通过将低分子量支链聚(乙烯亚胺)(PEI)与β-环糊精和1,2,3-丙三醇交联合成的,用于高效安全的非病毒基因递送。实验表明,该聚合物具有与25 kDa PEI相当的pH缓冲能力和DNA凝聚能力。在B16-F0细胞中,该聚合物使裸DNA的转染效率提高了700倍,并且产生了比Fugene HD(高三倍)和25 kDa PEI(高五倍)更好的转染效率。该聚合物的高转染效率不受转染过程中血清存在的影响。除了B16-F0细胞外,该聚合物还能使HepG2和U87细胞高效转染,且细胞毒性低。我们的结果表明,我们的聚合物是一种安全有效的转染试剂,值得在体外、体内和临床应用中进一步开发。