Cystic fibrosis is a genetic disease in which a chloride ion channel called CFTR is mutated, resulting defective mucus clearance at the epithelial cells lining the airways of the lungs. A paper published last year reported a successful step towards gene therapy for cystic fibrosis patients. A normal copy of CFTR was applied to cultured airway epithelial cells from a cystic fibrosis patient, and these cells were able to recover normal mucus transport. Image above shows these airway epithelial cells after CFTR gene delivery, expressing CFTR (red) on their lumenal side.
Reference: Liqun Zhang, cover image for PLoS Biology in July 2009. Image accompanies a PLoS Biology paper found here, by Liqun Zhang, Brian Button, Sherif E. Gabriel, Susan Burkett, Yu Yan, Mario H. Skiadopoulos, Yan Li Dang, Leatrice N. Vogel, Tristan McKay, April Mengos, Richard C. Boucher, Peter L. Collins, and Raymond J. Pickles.
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