Hydrophobic polyalanine modified hyperbranched polyethylenimine as high efficient pDNA and siRNA carrier.

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.