Efficient adenoviral-mediated ornithine transcarbamylase expression in deficient mouse and human hepatocytes

Manal A. Morsy, Eugene L. Alford, Andrew Bett, Frank L. Graham, C. Thomas Caskey

Research output: Contribution to journalArticlepeer-review

89 Scopus citations

Abstract

100% of primary human hepatocytes infected with an adenoviral vector carrying β-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.

Original languageEnglish (US)
Pages (from-to)1580-1586
Number of pages7
JournalJournal of Clinical Investigation
Volume92
Issue number3
DOIs
StatePublished - Sep 1993

Keywords

  • β-galactosidase
  • Gene therapy
  • Human
  • Liver
  • Ornithine transcarbamylase

ASJC Scopus subject areas

  • Medicine(all)

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