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- Title
Global non-viral gene transfer to the primate brain following intravenous administration
- Authors
Zhang, Yun; Schlachetzki, Felix; Pardridge, William M.
- Abstract
Expression plasmids encoding either luciferase or β-galactosidase were encapsulated in the interior of an “artificial virus” comprised of an 85 nm pegylated immunoliposome, which was targeted to the rhesus monkey brain in vivo with a monoclonal antibody (MAb) to the human insulin receptor (HIR). The HIRMAb enables the liposome carrying the exogenous gene to undergo transcytosis across the blood-brain barrier and endocytosis across the neuronal plasma membrane following intravenous injection. The level of luciferase gene expression in the brain was 50-fold higher in the rhesus monkey as compared to the rat. Widespread neuronal expression of the β-galactosidase gene in primate brain was demonstrated by both histochemistry and confocal microscopy. This approach makes feasible reversible adult transgenics in 24 hours.
- Subjects
GENE therapy; INSULIN receptors
- Publication
Molecular Therapy, 2003, Vol 7, Issue 1, p11
- ISSN
1525-0016
- Publication type
Article
- DOI
10.1016/S1525-0016(02)00018-7