Efficient adenoviral-mediated ornithine transcarbamylase expression in deficient mouse and human hepatocytes.
Open Access
- 1 September 1993
- journal article
- Published by American Society for Clinical Investigation in Journal of Clinical Investigation
- Vol. 92 (3) , 1580-1586
- https://doi.org/10.1172/jci116739
Abstract
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.Keywords
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