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Cystic Fibrosis

Mini-proteins used to repair defect in model cell systems

Published in Gene Therapy Weekly, April 18th, 2002

Cystic fibrosis (CF) is caused by mutations in a gene that encodes the cystic fibrosis transmembrane regulator (CFTR) protein. Normal CFTR protein forms channels for chloride ions to leave cells. The controlled outflow of chloride ions maintains cells' electrolyte and fluid balance and allows bacteria to be cleared from the surface of airway cells. This action prevents infection, which is a leading cause of death in people with CF.

University of Iowa researchers have shown that shortened versions of the normal CFTR protein can function like the normal full-length protein in experimental models of CF disease. These findings may point the way toward strategies that...

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