Publication Date:
1994-12-09
Description:
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.〈br /〉〈span class="detail_caption"〉Notes: 〈/span〉Zhou, L -- Dey, C R -- Wert, S E -- DuVall, M D -- Frizzell, R A -- Whitsett, J A -- DK38518/DK/NIDDK NIH HHS/ -- HL49004/HL/NHLBI NIH HHS/ -- HL51832/HL/NHLBI NIH HHS/ -- New York, N.Y. -- Science. 1994 Dec 9;266(5191):1705-8.〈br /〉〈span class="detail_caption"〉Author address: 〈/span〉Children's Hospital Medical Center, Division of Pulmonary Biology, Cincinnati, OH 45229-3039.〈br /〉〈span class="detail_caption"〉Record origin:〈/span〉 〈a href="http://www.ncbi.nlm.nih.gov/pubmed/7527588" target="_blank"〉PubMed〈/a〉
Keywords:
Animals
;
Base Sequence
;
Carrier Proteins/genetics
;
Chlorides/metabolism
;
Colforsin/pharmacology
;
Colon/chemistry/pathology
;
Cystic Fibrosis/genetics/metabolism/pathology/*therapy
;
Cystic Fibrosis Transmembrane Conductance Regulator
;
Disease Models, Animal
;
Fatty Acid-Binding Proteins
;
Gene Expression
;
*Genetic Therapy
;
Humans
;
Intestinal Mucosa/chemistry/*pathology/secretion
;
Intestine, Small/chemistry/pathology
;
Membrane Proteins/analysis/*genetics/physiology
;
Mice
;
Mice, Transgenic
;
Molecular Sequence Data
;
*Neoplasm Proteins
;
*Nerve Tissue Proteins
;
Promoter Regions, Genetic
;
RNA, Messenger/analysis/genetics
;
Rats
;
Recombinant Proteins/biosynthesis
;
*Tumor Suppressor Proteins
Print ISSN:
0036-8075
Electronic ISSN:
1095-9203
Topics:
Biology
,
Chemistry and Pharmacology
,
Computer Science
,
Medicine
,
Natural Sciences in General
,
Physics
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