Comparison of cDNA and genomic forms of tyrosine hydroxylase gene therapy of the brain with Trojan horse liposomes

Journal of Gene Medicine

Volumn 9, Issue 7, 2007, Pages 605-612

  Xia, Chun Fang ^a   Chu, Chun ^a   Li, Jianyi ^a   Wang, Yuntao ^a   Zhang, Yun ^a   Boado, Ruben J ^a   Pardridge, William M ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Blood brain barrier; Brain gene therapy; Non viral gene transfer; Tyrosine hydroxylase

Indexed keywords

COMPLEMENTARY DNA; GENOMIC DNA; LIPOSOME; MEMBRANE RECEPTOR; PLASMID DNA; TYROSINE 3 MONOOXYGENASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL CULTURE; COMBINATION CHEMOTHERAPY; CONTROLLED STUDY; CORPUS STRIATUM; DOSE TIME EFFECT RELATION; DRUG POTENTIATION; ENZYME ACTIVITY; ENZYME LOCALIZATION; ENZYME REPLACEMENT; EUKARYOTE; EXPERIMENTAL STUDY; GENE ACTIVITY; GENE CONSTRUCT; GENE EXPRESSION; GENE LOCATION; GENE STRUCTURE; GENE TARGETING; HUMAN; HUMAN CELL; IN VIVO STUDY; INTERMETHOD COMPARISON; LIPOSOMAL GENE DELIVERY SYSTEM; MALE; MONOTHERAPY; NONHUMAN; NONVIRAL GENE THERAPY; PARKINSON DISEASE; PRIORITY JOURNAL; RAT;

ANIMALS; BRAIN; CELLS, CULTURED; CORPUS STRIATUM; DNA, COMPLEMENTARY; GENE THERAPY; GENOME; HUMANS; LIPOSOMES; NEUROGLIA; PARKINSON DISEASE; PLASMIDS; RATS; RESTRICTION MAPPING; TYROSINE 3-MONOOXYGENASE;

EQUIDAE; EUKARYOTA; RATTUS;

EID: 34447571799     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1046     Document Type: Article

References (21)

1. Palmiter RD, Sandgren EP, Avarbock MR, Allen DD, Brinster RL. Heterologous introns can enhance expression of transgenes in mice. Proc Natl Acad Sci U S A 1991; 88: 478-482.
2. Choi T, Huang M, Gorman C, Jaenisch R. A generic intron increases gene expression in transgenic mice. Mol Cell Biol 1991; 11: 3070-3074.
3. Petitclerc D, Attal J, Theron MC, et al. The effect of various introns and transcription terminators on the efficiency of expression vectors in various cultured cell lines and in the mammary gland of transgenic mice. J Biotechnol 1995; 40: 169-178.
4. Lipps HJ, Jenke AC, Nehlsen K, Scinteie MF, Stehle IM, Bode J. Chromosome-based vectors for gene therapy. Gene 2003; 304: 23-33.
5. Chu C, Zhang Y, Boado RJ, Pardridge WM. Decline in exogenous gene expression in primate brain following intravenous administration is due to plasmid degradation. Pharm Res 2006; 23: 1586-1590.
6. Zhang Y, Calon F, Zhu C, Boado RJ, Pardridge WM. Intravenous nonviral gene therapy causes normalization of striatal tyrosine hydroxylase and reversal of motor impairment in experimental parkinsonism. Hum Gene Ther 2003; 14: 1-12.
7. Zhang Y, Schlachetzki F, Zhang YF, Boado RJ, Pardridge WM. Normalization of striatal tyrosine hydroxylase and reversal of motor impairment in experimental parkinsonism with intravenous nonviral gene therapy and a brain-specific promoter. Hum Gene Ther 2004; 15: 339-350.
8. Boado RJ, Pardridge WM. Molecular cloning of the bovine blood-brain barrier glucose transporter cDNA and demonstration of phylogenetic conservation of the 5′-untranslated region. Mol Cell Neurosci 1990; 1: 224-232.
9. Brown ER, Coker GT 3rd, O'Malley KL. Organization and evolution of the rat tyrosine hydroxylase gene. Biochemistry 1987; 26:5208-5212.
10. Min N, Joh TH, Kim KS, Peng C, Son JH. 5′ upstream DNA sequence of the rat tyrosine hydroxylase gene directs high-level and tissue-specific expression to catecholaminergic neurons in the central nervous system of transgenic mice. Brain Res Mol Brain Res 1994; 27: 281-289.
11. Dwyer KJ, Boado RJ, Pardridge WM. Cis-element/cytoplasmic protein interaction within the 3′-untranslated region of the GLUT1 glucose transporter mRNA. J Neurochem 1996; 66: 449-458.
12. Boado RJ, Li JY, Chu C, Ogoshi F, Wise P, Pardridge WM. Site-directed mutagenesis of cysteine residues of large neutral amino acid transporter LAT1. Biochim Biophys Acta 2005; 1715: 104-110.
13. Pardridge WM. Gene targeting in vivo with pegylated immunoliposomes. Methods Enzymol 2003; 373: 507-528.
14. Pardridge WM. Non-viral gene transfer across the blood-brain barrier with Trojan horse liposomes. In Delivery and Expression of DNA and RNA, Friedmann T, Rossi J (eds). Cold Spring Harbor Laboratory Press: Cold Spring Harbor, 2007; 701-710.
15. Zhang Y, Schlachetzki F, Pardridge WM. Global non-viral gene transfer to the primate brain following intravenous administration. Mol Ther 2003; 7: 11-18.
16. Hwang O, Baker H, Gross S, Joh TH. Localization of GTP cyclohydrolase in monoaminergic but not nitric oxide-producing cells. Synapse 1998; 28: 140-153.
17. Vann LR, Payne SG, Edsall LC, Twitty S, Spiegel S, Milstien S. Involvement of sphingosine kinase in TNF-alpha-stimulated tetrahydrobiopterin biosynthesis in C6 glioma cells. J Biol Chem 2002; 277: 12649-12656.
18. Kim TE, Park MJ, Choi EJ, et al. Cloning and cell type-specific regulation of the human tyrosine hydroxylase gene promoter. Biochem Biophys Res Commun 2003; 312: 1123-1131.
19. Kessler MA, Yang M, Gollomp KL, Jin H, Iacovitti L. The human tyrosine hydroxylase gene promoter. Brain Res Mol Brain Res 2003; 112: 8-23.
20. Wang Y, Yu L, Geller AI. Diverse stabilities of expression in the rat brain from different cellular promoters in a helper virus-free herpes simplex virus type 1 vector system. Hum Gene Ther 1999, 10: 1763-1771.
21. Chun HS, Yoo MS, DeGiorgio LA, et al. Marked dopaminergic cell loss subsequent to developmental, intranigral expression of glial cell line-derived neurotrophic factor. Exp Neurol 2002; 173: 235-244.