[Cystic fibrosis: From gene discovery to precision medicine].

Abstract excerpt

The discovery in 1989 that cystic fibrosis, the most common life-shortening hereditary disease in Caucasians, was caused by mutations in cystic fibrosis transmembrane conductance regulator (CFTR) gene, put in motion whole new areas of research, diagnosis, and therapeutic development. In this review, we focus on the most important advances in our understanding of the molecular basis of CFTR dysfunction. To date,...

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