Cystic Fibrosis
Small molecules rescue Delta F508-CFTR trafficking and gating
Published in Biotech Business Week, July 3rd, 2006
"CF is a fatal genetic disease caused by mutations in cftr, a gene encoding a PKA-regulated Cl-channel. The most common mutation results in a deletion of phenylAla at position 508 (Delta F508-CFTR) that impairs protein folding, trafficking, and channel gating in epithelial cells," wrote F. Vangoor and colleagues, Vertex Pharmaceutical.
They continued, "In the airway, these defects alter salt and fluid transport, leading to chronic infection, inflammation, and loss of lung function. There are...
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