Micro- and nanoscale delivery systems for macromolecules: gene delivery

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One of the biggest challenges in gene therapy -- inserting genes into an individual's cells and tissues to treat a disease -- is successfully delivering DNA into cells, followed by the DNA being expressed. In our search for suitable materials to package DNA, we have identified a group of compounds known as gemini surfactants that can condense, protect and transport DNA into cells. Using these electrically charged compounds, we have developed a non-viral nanoparticle-based technology to deliver genes through the skin. Non-viral delivery systems have significant advantages relative to viral systems, as they are safer, cheaper and easier to prepare. Their major disadvantage, however, is that they are not as efficient, a deficiency this proposed research will address.