ABSTRACT
The rationale for using genetic techniques to treat diseases that are a consequence of an inherited defect in a single gene is straightforward. If an appropriate normal gene can be delivered in a functioning form in an adequate dose to the right cells, the disease should be cured. To use gene therapy to treat acute lung disease, acquired rather than inherited, the rationale is more complex, and the strategies, in some cases, are more convoluted. The therapeutic gene need not qualitatively alter the host response but, like more conventional drugs, simply tilt the host-toxin interaction in the host’s favor. The ultimate goal for gene therapy of inherited diseases would be to deliver the normal gene in a manner that assured its permanent expression. Cationic liposome/plasmid delivery systems have some significant advantages for therapy of acute diseases. Cationic liposome/plasmid complexes can deliver functioning transgenes to the lungs following either intravenous or airway administration.
