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Cystic fibrosis

Cystic fibrosis is caused by mutations in cystic fibrosis transmembrane conductance regulator (CFTR), which encodes a transmembrane ion channel on epithelial cells. Many therapies are under investigation to treat patients with cystic fibrosis, including ion channel modulators and gene therapy.

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Cystic fibrosis. Nat Rev Dis Primers 1, 15049 (2015).

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