Dual Function Lipid-Based Transfection Agents: Combination of Drug and DNA Produces Large Increase in Efficiency (22094)

INVENTION: Novel liposomes have been developed that are useful as non-viral vectors to deliver DNA to cells. The invention described herein focuses on the use of lipoplexes with incorporated microtubule disrupting agents as non-viral gene delivery vectors that can efficiently and non-toxically deliver genes to cells in vitro. Thus, these lipoplexes are useful as gene therapy and cellular transfection reagents with applications that include use as gene delivery vectors for gene therapy to treat a variety of diseases. Also, since this formulation is able to deliver a drug and a gene in a single agent and in a single administration, it has significant clinical advantages.

BACKGROUND:Incorporating microtubule disrupting agents such as vinblastine and vincristine into cationic lipids can increase the transfection efficiency of vascular smooth muscle endothelial cells 40-fold. The figure below summarizes the effects of vinblastine-lipoplexes on the transfection of b-galactosidase in vascular smooth muscle cells, which are traditionally difficult to transfect using the commercial transfection reagents currently available. Mechanistically, the microtubule disrupting agents appear to exert their effect by blocking trafficking of the lipoplexes to the lysosome and also by promoting transcription through NF-kB dependent mechanisms. The results depicted below, for vascular smooth muscle cells, demonstrate that the combination of cationic lipid with 1 part per hundred of vinblastine generates lipoplexes with an approximately 30-fold increase in transfection efficiency. Experiments are being planned to test the ability of these lipoplexes to deliver DNA in vivo. A Patent Application has been filed.

Inventor(s): Li Wang, Robert C. MacDonald

Type of Offer: Licensing

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