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Cationic cyclodextrin amphiphiles as gene delivery vectors

2004; Elsevier BV; Volume: 14; Issue: 1 Linguagem: Inglês

10.1016/s1773-2247(04)50006-0

2588-8943

Sally‐Ann Cryan, Ruth Donohue, Bart Jan Ravoo, R. Darcy, Caitríona M. O’Driscoll,

Virus-based gene therapy research

Amphiphilic polycationic cyclodextrins, heptakis[2-(m-amino-oligoethyleneglycol)-6-deoxy-6-hexylthio]-p-cyclodextrin and heptakis[2-(m- amino-oligoethyleneglycol)-6-deoxy-6-hexadecylthio]-p-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 104 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.