Successful gene therapy to treat lung disease will require development of effective gene transfer tools. While molecular tools (called vectors) have been developed to escort a foreign gene into cells, technologies to administer these vectors to the lungs by inhalation has remained largely unchanged and are inefficient. GeneRx+, inc. is a research and development company focused on developing plasmid/liposome gene expression systems to treat lung disease. Inspired Medical Products, Inc. designs efficient aerosol devices which intelligently adapt to the individual subject's breathing pattern and which are tailored for the specific drug to be inhaled. The purpose of this application is to combine the unique expertise of these two companies to develop and test in vitro an improved aerosol device designed to deliver functional plasmid/liposome complexes to either the conducting airways or to the distal airspaces of humans. The investigators propose to: 1) determine in vitro physical characteristics of plasmid/lipid aerosols generated by our new device; 2) using simulated breath traces-actuated delivery in vitro, calculate the amount likely to be administered in vivo to a sheep (whose lung size approximates that of humans); and 3) demonstrate that aerosolization of the plasmid/liposome complexes does not alter transfection efficacy in cultured human lung cells.
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