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Science 9 December 1994:
Vol. 266. no. 5191, pp. 1705 - 1708
DOI: 10.1126/science.7527588
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Articles

Science, Vol 266, Issue 5191, 1705-1708
Copyright © 1994 by American Association for the Advancement of Science

articles

Correction of lethal intestinal defect in a mouse model of cystic fibrosis by human CFTR

L Zhou, CR Dey, SE Wert, MD DuVall, RA Frizzell, and JA Whitsett

Children's Hospital Medical Center, Division of Pulmonary Biology, Cincinnati, OH 45229-3039.

Cystic fibrosis (CF) is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR). A potential animal model of CF, the CFTR-/- mouse, has had limited utility because most mice die from intestinal obstruction during the first month of life. Human CFTR (hCFTR) was expressed in CFTR-/- mice under the control of the rat intestinal fatty acid-binding protein gene promoter. The mice survived and showed functional correction of ileal goblet cell and crypt cell hyperplasia and cyclic adenosine monophosphate-stimulated chloride secretion. These results support the concept that transfer of the hCFTR gene may be a useful strategy for correcting physiologic defects in patients with CF.


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