Summary
Recent studies have identified the underlying molecular defect in cystic fibrosis (CF). Reduced or absent cAMP-mediated chloride transport in epithelial-lined organs characterizes this disease. With the identification of the CF gene, gene therapy has become a potential novel form of treatment for this disease. This article reviews the rapid progress in CF research from the understanding of the bioelectric defect to the recently begun human gene therapy trials.
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References
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Alton, E.W.F.W. Gene therapy for cystic fibrosis. J Inherit Metab Dis 18, 501–507 (1995). https://doi.org/10.1007/BF00710061
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DOI: https://doi.org/10.1007/BF00710061