Cationic cyclodextrin amphiphiles as gene delivery vectors

Amphiphilic polycationic cyclodextrins, heptakis[2-(omega-amino-oligoethyleneglycol)-6-deoxy-6-hexylthio]-beta-cyclodextrin and heptakis[2-(omega-amino-oligoethyleneglycol)-6-deoxy-6-hexadecylthio]-beta-cyclodextrin, were assessed for condensation of plasmid DNA and cell transfection. These cyclodextrins can self-assemble into cationic vesicles or nanoparticles and, unlike their amphiphilic non-aminated analogues, form DNA lipoplexes which efficiently transfected COS-7 cells. Levels of luciferase expression with the hexadecylthiopolyamino-CD vector were 2 x 10(4) times greater than for DNA alone. These levels are a significant improvement on non-amphiphilic polyamino CDs as vectors and are of the same order as the widely used transfection agent Lipofectin.