Abstract
Gene therapy has the ability to treat diseases by delivering exogenous DNA into the nuclei of target cells to express a therapeutic protein. In intravenous gene delivery, free oligonucleotides and DNA are rapidly degraded by serum nucleases in the blood before they can reach the target site. Therefore, the main goal for gene therapy is not just to obtain cellular expression of an exogenous gene per se, but also to develop non-toxic and efficient carriers. Because of its abundant positive surface charges, PEI is able to condense the negatively charged DNA into PEI/DNA complexes with a net positive charge. PEG facilitates the formation of polyplexes with improved solubility, reduced aggregation, lower cytotoxicity, and possibly decreases opsonization with serum proteins in the bloodstream. Also, dexamethasone is the potent ligand of the glucocorticoid receptor which facilitates the transfer into nucleus, and it is known to enlarge the nuclear pore complexes. In this study, PEG-Glu-PEI-Dexa was synthesized as a kind of biodegradable polycation for gene delivery.
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