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Science. 1994 Dec 9;266(5191):1705-8.

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

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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.

[Indexed for MEDLINE]

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