WS09.04 Enhancement of channel activity of the CFTR protein delivered via gene therapy vectors

Objectives: The advancements in developing CFTR-targeted drugs offer effective treatments for most cystic fibrosis patients. However, there is still a small portion of patients who do not respond to the new drugs. In addition, it unclear whether can maintain lung function long term. Thus, other novel therapeutic strategies should be investigated. Using gene therapy express copy functional CFTR CF cells an attractive strategy. Currently, one major challenges how deliver enough protein lungs achieve effects. Our group has been using expression cassette based on human cytokeratin 18 (K18) promoter gene. Since K18 reported enhanced by sodium butyrate, here we are examining further following vector delivery cells. Methods: or LacZ reporter was delivered cultured epithelial helper-dependent adenoviral vectors. transduced with without treatment butyrate were harvested. assessed chemiluminescent assays. enhancement at both mRNA level real-time PCR and Western blotting; determined FLiPR Results: Cultured treated showed increase from least one-fold. Consistent analysis, show same channel function. Conclusion: vectors significantly if used driving expression.