Abstract
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.
Publication types
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Research Support, Non-U.S. Gov't
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Research Support, U.S. Gov't, P.H.S.
MeSH terms
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Animals
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Base Sequence
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Carrier Proteins / genetics
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Chlorides / metabolism
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Colforsin / pharmacology
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Colon / chemistry
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Colon / pathology
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Cystic Fibrosis / genetics
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Cystic Fibrosis / metabolism
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Cystic Fibrosis / pathology
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Cystic Fibrosis / therapy*
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Cystic Fibrosis Transmembrane Conductance Regulator
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Disease Models, Animal
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Fatty Acid-Binding Protein 7
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Fatty Acid-Binding Proteins
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Gene Expression
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Genetic Therapy*
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Humans
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Intestinal Mucosa / chemistry
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Intestinal Mucosa / metabolism
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Intestinal Mucosa / pathology*
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Intestine, Small / chemistry
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Intestine, Small / pathology
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Membrane Proteins / analysis
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Membrane Proteins / genetics*
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Membrane Proteins / physiology
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Mice
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Mice, Transgenic
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Molecular Sequence Data
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Neoplasm Proteins*
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Nerve Tissue Proteins*
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Promoter Regions, Genetic
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RNA, Messenger / analysis
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RNA, Messenger / genetics
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Rats
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Recombinant Proteins / biosynthesis
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Tumor Suppressor Proteins*
Substances
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CFTR protein, human
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Carrier Proteins
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Chlorides
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FABP7 protein, human
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Fabp5 protein, mouse
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Fabp7 protein, mouse
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Fabp7 protein, rat
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Fatty Acid-Binding Protein 7
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Fatty Acid-Binding Proteins
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Membrane Proteins
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Neoplasm Proteins
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Nerve Tissue Proteins
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RNA, Messenger
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Recombinant Proteins
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Tumor Suppressor Proteins
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Cystic Fibrosis Transmembrane Conductance Regulator
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Colforsin