Dr. Jim Hu
Development of epithelial expression cassettes for Lung gene therapy. Development of vectors for efficient and tissue-specific expression of transgenes is critical to gene therapy research. Currently, the viral promoter-based expression cassettes, although very popular, do not offer sustained gene expression or cell-specificity in lung. We have taken a novel approach to the development of better expression cassettes for lung gene therapy by using human regulatory elements. Our first generation of vectors can direct efficient expression of reporter genes as well as the human CFTR gene in cultured lung epithelial cells. Our preliminary results showed that these human DNA regulatory elements can direct strong and epithelium-specific expression of the LacZ reporter gene in lungs of the transgenic mice.
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