Efficient adenoviral-mediated ornithine transcarbamylase expression in deficient mouse and human hepatocytes.
100% of primary human hepatocytes infected with an adenoviral vector carrying beta-galactosidase expressed the exogenous gene. Expression was also achieved in > 40% of adult mouse hepatocytes in vivo. Normal levels of activity were achieved in mouse ornithine transcarbamylase (OTC)-deficient primary hepatocytes using another adenoviral vector carrying human OTC cDNA. Study of OTC-deficient primary human hepatocytes from a single patient confirmed the utility of adenoviral delivery of OTC. We describe adenoviral-mediated exogenous gene expression in human and mouse hepatocytes in vitro and in mouse liver in vivo. Data suggest that adenoviral vectors may be useful for correcting OTC deficiency.
Institute of Molecular Genetics, Baylor College of Medicine, Houston, Texas 77030.
M A
MA
Morsy
E L
EL
Alford
A
A
Bett
F L
FL
Graham
C T
CT
Caskey
D000256:Adenoviridae / Q000235:genetics*; D000818:Animals; D015870:Gene Expression; D005822:Genetic Vectors; D006801:Humans; D066298:In Vitro Techniques; D008099:Liver / Q000201:enzymology; D051379:Mice; D009954:Ornithine Carbamoyltransferase / Q000235:genetics*; D014162:Transfection
D009954:Ornithine Carbamoyltransferase