The safe and effective gene delivery vector remains the key step for gene therapy. Hydrophobic-modified Phe-PEI (PP80) was exhibited in advantage with biocompatibility and gene delivery with smaller size and easier penetration into cells and tissues. PP80 delivery of rev-casp-3 gene was demonstrated effectively to inhibit HeLa xenograft growth in our previous work. However, it was necessary to evaluate its applicability in other cells or tissues as gene carrier. Here, we quantitatively optimized the complex ratio of PP80 and plasmid DNA (pDNA) and evaluated the potential pyrogenicity by rabbit pyrogen test. In addition, PP80-mediated expression of scFv1C9 gene blocked HepG2 cell cycle progress in vitro. Subsequently, PP80-scFv1C9 was injected into HepG2 xenograft and significantly inhibited the xenograft growth in nude mice. Further investigation indicated that PP80 was an effective gene carrier and possible for entering hepatic xenograft. These features of PP80 made it attractive as a potential gene carrier for cancer therapy.
Keywords: PP80; gene therapy; scFv1C9; xenograft.