EFFICIENT ADENOVIRAL-MEDIATED ORNITHINE TRANSCARBAMYLASE EXPRESSION IN DEFICIENT MOUSE AND HUMAN HEPATOCYTES

被引:89
作者
MORSY, MA
ALFORD, EL
BETT, A
GRAHAM, FL
CASKEY, CT
机构
[1] BAYLOR COLL MED,INST MOLEC GENET,1 BAYLOR PLAZA,HOUSTON,TX 77030
[2] BAYLOR COLL MED,DEPT OTORHINOLARYNGOL,HOUSTON,TX 77030
[3] BAYLOR COLL MED,HOWARD HUGHES MED INST,HOUSTON,TX 77030
[4] MCMASTER UNIV,DEPT BIOL,HAMILTON L8S 4K1,ONTARIO,CANADA
[5] MCMASTER UNIV,DEPT PATHOL,HAMILTON L8S 4K1,ONTARIO,CANADA
关键词
ORNITHINE TRANSCARBAMYLASE; HUMAN; LIVER; GENE THERAPY; BETA-GALACTOSIDASE;
D O I
10.1172/JCI116739
中图分类号
R-3 [医学研究方法]; R3 [基础医学];
学科分类号
1001 ;
摘要
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.
引用
收藏
页码:1580 / 1586
页数:7
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