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… - Science, 1994 - science.org
L Zhou, CR Dey, SE Wert, MD DuVall, RA Frizzell, JA Whitsett
Science, 1994•science.orgCystic 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 (h CFTR) 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 …
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 (h CFTR) 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 …
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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