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Hoffmann, R. A wiki for the life sciences where authorship matters. Nature Genetics (2008)
 

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

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.[1]

References

  1. Correction of lethal intestinal defect in a mouse model of cystic fibrosis by human CFTR. Zhou, L., Dey, C.R., Wert, S.E., DuVall, M.D., Frizzell, R.A., Whitsett, J.A. Science (1994) [Pubmed]
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