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A Controlled Study of Adenoviral-Vector–Mediated Gene Transfer in the Nasal Epithelium of Patients with Cystic Fibrosis
Journal article   Open access   Peer reviewed

A Controlled Study of Adenoviral-Vector–Mediated Gene Transfer in the Nasal Epithelium of Patients with Cystic Fibrosis

Michael R Knowles, Kathy W Hohneker, Zhaoqing Zhou, John C Olsen, Terry L Noah, Ping-Chuan Hu, Margaret W Leigh, John F Engelhardt, Lloyd J Edwards, Kim R Jones, …
The New England journal of medicine, Vol.333(13), pp.823-831
09/28/1995
DOI: 10.1056/NEJM199509283331302
PMID: 7544439
url
https://doi.org/10.1056/NEJM199509283331302View
Published (Version of record) Open Access

Abstract

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 abnormal secretions that obstruct and ultimately damage epithelium in many areas of the body. 7 The principal cause of death among patients with cystic fibrosis is lung disease. Patients who are homozygous for mutations in the CFTR gene have defective cyclic AMP (cAMP)–regulated secretion of chloride 8 , 9 and elevated absorption of sodium . . .

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