Multivalent cationic dendrofullerenes for gene transfer: synthesis and DNA complexation.

ABSTRACT

Non-viral nucleic acid vectors able to display high transfection efficiencies with low toxicity and overcoming the multiple biological barriers are needed to further develop the clinical applications of gene therapy. The synthesis of hexakis-adducts of [60]fullerene endowed with 12, 24 and 36 positive ammonium groups and a tridecafullerene appended with 120 positive charges has been performed. The delivery of a plasmid containing the green fluorescent protein (EGFP) gene into HEK293 (Human Embryonic Kidney) cells resulting in effective gene expression has demonstrated the efficacy of these compounds to form polyplexes with DNA. Particularly, giant tridecafullerene macromolecules have shown higher efficiency in the complexation and transfection of DNA. Thus, they can be considered as promising non-viral vectors for transfection purposes.