Article
Molecular pathophysiology of cystic fibrosis based on the rescued knockout mouse model.
Molecular genetics and metabolism - 1 Jun 1998
Cohen J C, Morrow S L, Cork R J, Delcarpio J B, Larson J E
Abstract excerpt
Cystic fibrosis transmembrane conductance regulator (cftr) gene mutations are thought to result in cystic fibrosis due to an absence of the protein's chloride channel. Recently, the lethal intestinal blockage in the cftr knockout mouse was reversed by a single in utero dose of a recombinant adenovirus containing the human cftr gene. The rescue of these animals did not require continuous expression of the gene and...
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