A Controlled Study of Adenoviral-Vector–Mediated Gene Transfer in the Nasal Epithelium of Patients with Cystic Fibrosis
Cystic fibrosis is a recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene. 1 – 4 The normal CFTR gene codes for a protein (CFTR) that plays a key part in epithelial transport of salt and water. 5 , 6 Mutations in CFTR result in abnorm...
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Veröffentlicht in: | The New England journal of medicine 1995-09, Vol.333 (13), p.823-831 |
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