| First Author | Zhou L | Year | 1994 |
| Journal | Science | Volume | 266 |
| Issue | 5191 | Pages | 1705-8 |
| PubMed ID | 7527588 | Mgi Jnum | J:21934 |
| Mgi Id | MGI:69834 | Doi | 10.1126/science.7527588 |
| Citation | Zhou L, et al. (1994) Correction of lethal intestinal defect in a mouse model of cystic fibrosis by human CFTR. Science 266(5191):1705-8 |
| abstractText | 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. |
