Article
Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770.
Proceedings of the National Academy of Sciences of the United States of America - 3 Nov 2009
Van Goor Fredrick, Hadida Sabine, Grootenhuis Peter D J, Burton Bill, Cao Dong, Neuberger Tim, Turnbull Amanda, Singh Ashvani, Joubran John, Hazlewood Anna, Zhou Jinglan, McCartney Jason, Arumugam Vijayalaksmi, Decker Caroline, Yang Jennifer, Young Chris, Olson Eric R, Wine Jeffery J, Frizzell Raymond A, Ashlock Melissa, Negulescu Paul
Abstract excerpt
Cystic fibrosis (CF) is a fatal genetic disease caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR), a protein kinase A (PKA)-activated epithelial anion channel involved in salt and fluid transport in multiple organs, including the lung. Most CF mutations either reduce the number of CFTR channels at the cell surface (e.g., synthesis or processing mutations) or impair channel...
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