Gene therapy in cystic fibrosis.

Abstract excerpt

Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane regulator (CFTR) gene and is the most common life-shortening genetic defect in Caucasians. Life expectancy in CF has improved substantially over the last 75 years because of treatments aimed at end-organ complications. Since the CFTR gene was discovered in 1989 more than 1900 mutations have been...

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