Article
New and emerging targeted therapies for cystic fibrosis.
BMJ (Clinical research ed.) - 30 Mar 2016
Quon Bradley S, Rowe Steven M
Abstract excerpt
Cystic fibrosis (CF) is a monogenic autosomal recessive disorder that affects about 70,000 people worldwide. The clinical manifestations of the disease are caused by defects in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The discovery of the CFTR gene in 1989 has led to a sophisticated understanding of how thousands of mutations in the CFTR gene affect the structure and function of the...
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