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PMID: 7527588 Published · ppublish English Journal Article Research Support, Non-U.S. Gov't Research Support, U.S. Gov't, P.H.S.

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

Science (New York, N.Y.) ·Vol. 266 ·No. 5191 ·1994-12-09 ·Pages 1705-8

Zhou L, Dey CR, Wert SE, DuVall MD, Frizzell RA, Whitsett JA

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.

Related Genes

CFTR

MeSH Terms

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 Protein 7 Fatty Acid-Binding Proteins Gene Expression Genetic Therapy Humans Intestinal Mucosa/chemistry,metabolism,pathology 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

Chemicals

CFTR protein, human Carrier Proteins Chlorides FABP7 protein, human Fabp5 protein, mouse Fabp7 protein, mouse Fabp7 protein, rat Fatty Acid-Binding Protein 7 Fatty Acid-Binding Proteins Membrane Proteins Neoplasm Proteins Nerve Tissue Proteins RNA, Messenger Recombinant Proteins Tumor Suppressor Proteins Cystic Fibrosis Transmembrane Conductance Regulator Colforsin

Authors & Affiliations

6 authors, click to expand affiliations / ORCID

Zhou L

Children's Hospital Medical Center, Division of Pulmonary Biology, Cincinnati, OH 45229-3039.

Dey C R

Wert S E

DuVall M D

Frizzell R A

Whitsett J A

Article Info

Journal

Science (New York, N.Y.)

Abbr.

Science

ISSN

0036-8075

DOI

10.1126/science.7527588

Published

1994-12-09

Pages

1705-8

Language

English

Region

United States

NLM ID

0404511

Subset

Grants

NIDDK NIH HHS · DK38518 · United States

NHLBI NIH HHS · HL49004 · United States

NHLBI NIH HHS · HL51832 · United States

External Links

PubMed source DOI: 10.1126/science.7527588 View journal details

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

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