Successful targeting of the mouse cystic fibrosis transmembrane conductance regulator gene in embryonal stem cells.

Journal article

We wish to construct a mouse model for the human inherited disease cystic fibrosis. We describe here the successful targeting in embryonal stem cells of the murine homologue (Cftr) of the cystic fibrosis transmembrane conductance regulator gene, as the first critical step towards this end. The targeting event precisely disrupts exon 10, the site of the major mutation in patients with cystic fibrosis. The targeted cells are pluripotent and competent to form chimaeras.

Authors: Dorin, JR; Dickinson, P; Emslie, E; Clarke, A; Dobbie, L

• Publisher: Kluwer Academic Publishers
• Journal: Transgenic research
• Volume: 1
• Issue: 2
• Pages: 101-105
• Publication date: 1992-03-01
• DOI: 10.1007/bf02513027
• EISSN: 1573-9368
• ISSN: 0962-8819
• Language: English
• Keywords: Disease Models, Animal; Exons; Stem Cells; Humans; Restriction Mapping; Embryo, Mammalian; Cystic Fibrosis; Membrane Proteins; Animals; Cystic Fibrosis Transmembrane Conductance Regulator; Mice, Transgenic; Mice