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.
Morsy
M A
MA
Institute of Molecular Genetics, Baylor College of Medicine, Houston, Texas 77030.
Alford
E L
EL
Bett
A
A
Graham
F L
FL
Caskey
C T
CT
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