Optimization of continuous in vivo DOPA production and studies on ectopic da synthesis using rAAV5 vectors in Parkinsonian rats

Journal of Neurochemistry

Volumn 111, Issue 2, 2009, Pages 355-367

  Björklund, Tomas ^a   Hall, Hélène ^a   Breysse, Nathalie ^a,d   Soneson, Charlotte ^a,b   Carlsson, Thomas ^a,e   Mandel, Ronald J ^c   Carta, Manolo ^a   Kirik, Deniz ^a  

^a LUND UNIVERSITY   (Sweden)

^b LUND UNIVERSITY   (Sweden)

^c UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^d PFIZER INC   (United States)

^e PHILIPPS UNIVERSITY MARBURG   (Germany)

Author keywords

Dopamine; Enzyme replacement; GTP cyclohydrolase 1; Parkinson's disease; Recombinant adeno associated viral vector; Tyrosine hydroxylase

Indexed keywords

5,6,7,8 TETRAHYDRO LEVO BIOPTERIN; DOPA; GUANOSINE TRIPHOSPHATE CYCLOHYDROLASE I; TYROSINE; TYROSINE 3 MONOOXYGENASE; UNCLASSIFIED DRUG; VIRUS VECTOR;

ANIMAL EXPERIMENT; ARTICLE; CONTROLLED STUDY; CORPUS STRIATUM; ENZYME STABILITY; ENZYME SYNTHESIS; FEMALE; GENE THERAPY; IN VIVO STUDY; NONHUMAN; PARKINSON DISEASE; PRIORITY JOURNAL; RAT; STOICHIOMETRY;

ANIMALS; BIOPTERIN; CORPUS STRIATUM; DEPENDOVIRUS; DIHYDROXYPHENYLALANINE; DOPAMINE; FEMALE; GENE THERAPY; GTP CYCLOHYDROLASE; IMMUNOHISTOCHEMISTRY; OXIDOPAMINE; PARKINSONIAN DISORDERS; RATS; RATS, SPRAGUE-DAWLEY; SYMPATHOLYTICS; TYROSINE 3-MONOOXYGENASE;

ANIMALIA; PARKINSONIA; RATTUS;

EID: 70349319755     PISSN: 00223042     EISSN: 14714159     Source Type: Journal    
DOI: 10.1111/j.1471-4159.2009.06340.x     Document Type: Article

References (32)

1. Alterio J., Ravassard P., Haavik J., Le Caer J. P., Biguet N. F., Waksman G. Mallet J. (1998) Human tyrosine hydroxylase isoforms. Inhibition by excess tetrahydropterin and unusual behavior of isoform 3 after cAMP-dependent protein kinase phosphorylation. J. Biol. Chem. 273, 10196 10201.
2. Bencsics C., Wachtel S. R., Milstien S., Hatakeyama K., Becker J. B. Kang U. J. (1996) Double transduction with GTP cyclohydrolase I and tyrosine hydroxylase is necessary for spontaneous synthesis of l-DOPA by primary fibroblasts. J. Neurosci. 16, 4449 4456.
3. Betto P., Ricciarello G., Giambenedetti M., Lucarelli C., Ruggeri S. Stocchi F. (1988) Improved high-performance liquid chromatographic analysis with double detection system for l-dopa, its metabolites and carbidopa in plasma of parkinsonian patients under l-dopa therapy. J. Chromatogr. 459, 341 349.
4. Carlsson A., Davis J. N., Kehr W., Lindqvist M. Atack C. V. (1972) Simultaneous measurement of tyrosine and tryptophan hydroxylase activities in brain in vivo using an inhibitor of the aromatic amino acid decarboxylase. Naunyn Schmiedebergs Arch. Pharmacol. 275, 153 168.
5. Carlsson T., Winkler C., Burger C., Muzyczka N., Mandel R. J., Cenci A., Bjorklund A. Kirik D. (2005) Reversal of dyskinesias in an animal model of Parkinson's disease by continuous l-DOPA delivery using rAAV vectors. Brain 128, 559 569.
6. Carlsson T., Carta M., Winkler C., Björklund A. Kirik D. (2007) Serotonin neuron transplants exacerbate l-DOPA-induced dyskinesias in a rat model of Parkinson's disease. J. Neurosci. 27, 8011 8022.
7. Carlsson T., Carta M., Muñoz A., Mattsson B., Winkler C., Kirik D. Björklund A. (2009) Impact of grafted serotonin and dopamine neurons on development of l-DOPA-induced dyskinesias in parkinsonian rats is determined by the extent of dopamine neuron degeneration. Brain 132, 319 335.
8. Carta M., Carlsson T., Kirik D. Björklund A. (2007) Dopamine released from 5-HT terminals is the cause of l-DOPA-induced dyskinesia in parkinsonian rats. Brain 130, 1819 1833.
9. Corti O., Sánchez-Capelo A., Colin P., Hanoun N., Hamon M. Mallet J. (1999) Long-term doxycycline-controlled expression of human tyrosine hydroxylase after direct adenovirus-mediated gene transfer to a rat model of Parkinson's disease. Proc. Natl Acad. Sci. USA 96, 12120 12125.
10. During M. J., Naegele J. R., O'Malley K. L. Geller A. I. (1994) Long-term behavioral recovery in parkinsonian rats by an HSV vector expressing tyrosine hydroxylase. Science 266, 1399 1403.
11. Everett G. M. Borcherding J. W. (1970) l-Dopa: effect on concentrations of dopamine, norepinephrine, and serotonin in brains of mice. Science 168, 849 850.
12. Harper C. G., Kril J. J. Daly J. M. (1987) The specific gravity of the brains of alcoholic and control patients: a pathological study. Br. J. Addict. 82, 1349 1354.
13. Hornykiewicz O. (1962) Dopamine (3-hydroxytyramine) in the central nervous system and its relation to the Parkinson syndrome in man. Dtsch. Med. Wochenschr. 87, 1807 1810.
14. Ichinose H. Nagatsu T. (1997) Molecular genetics of hereditary dystonia: mutations in the GTP cyclohydrolase I gene. Brain Res. Bull. 43, 35 38. (Pubitemid 27265788)
15. Ichinose H., Suzuki T., Inagaki H., Ohye T. Nagatsu T. (1999) Molecular genetics of dopa-responsive dystonia. Biol. Chem. 380, 1355 1364.
16. Isacson O. (1995) Behavioral effects and gene delivery in a rat model of Parkinson's disease. Science 269, 856 857.
17. Kaplitt M. G., Leone P., Samulski R. J., Xiao X., Pfaff D. W., O'Malley K. L. During M. J. (1994) Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat. Genet. 8, 148 154. (Pubitemid 24308362)
18. Kirik D., Georgievska B., Burger C., Winkler C., Muzyczka N., Mandel R. J. Bjorklund A. (2002) Reversal of motor impairments in parkinsonian rats by continuous intrastriatal delivery of l-dopa using rAAV-mediated gene transfer. Proc. Natl Acad. Sci. USA 99, 4708 4713.
19. Leff S. E., Rendahl K. G., Spratt S. K., Kang U. J. Mandel R. J. (1998) In vivol-DOPA production by genetically modified primary rat fibroblast or 9L gliosarcoma cell grafts via coexpression of GTPcyclohydrolase I with tyrosine hydroxylase. Exp. Neurol. 151, 249 264.
20. Leriche L., Björklund T., Breysse N. et al. (2009) Positron emission tomography imaging demonstrates correlation between behavioral recovery and correction of dopamine neurotransmission after gene therapy. J. Neurosci. 29, 1544 1553.
21. Lundberg C., Björklund T., Carlsson T., Jakobsson J., Hantraye P., Déglon N. Kirik D. (2008) Applications of lentiviral vectors for biology and gene therapy of neurological disorders. Curr. Gene Ther. 8, 461 473.
22. Mandel R. J., Rendahl K. G., Spratt S. K., Snyder R. O., Cohen L. K. Leff S. E. (1998) Characterization of intrastriatal recombinant adeno-associated virus-mediated gene transfer of human tyrosine hydroxylase and human GTP-cyclohydrolase I in a rat model of Parkinson's disease. J. Neurosci. 18, 4271 4284.
23. Mann S. P. Gordon J. I. (1979) Inhibition of guinea-pig brain tyrosine hydroxylase by catechols and biopterin. J. Neurochem. 33, 133 138.
24. Morón J. A., Brockington A., Wise R. A., Rocha B. A. Hope B. T. (2002) Dopamine uptake through the norepinephrine transporter in brain regions with low levels of the dopamine transporter: evidence from knock-out mouse lines. J. Neurosci. 22, 389 395.
25. Muñoz A., Li Q., Gardoni F. et al. (2008) Combined 5-HT1A and 5-HT1B receptor agonists for the treatment of l-DOPA-induced dyskinesia. Brain 131, 3380 3394.
26. Nagatsu T., Levitt M. Udenfriend S. (1964) Tyrosine hydroxylase. The initial step in norepinephrine biosynthesis. J. Biol. Chem. 239, 2910 2917.
27. Ng K. Y., Chase T. N., Colburn R. W. Kopin I. J. (1970) l-Dopa-induced release of cerebral monoamines. Science 170, 76 77.
28. Nyholm D., Askmark H., Gomes-Trolin C., Knutson T., Lennernäs H., Nyström C. Aquilonius S. M. (2003) Optimizing levodopa pharmacokinetics: intestinal infusion versus oral sustained-release tablets. Clin. Neuropharmacol. 26, 156 163.
29. Obeso J. A., Olanow C. W. Nutt J. G. (2000) Levodopa motor complications in Parkinson's disease. Trends Neurosci. 23, S2 S7.
30. Oorschot D. E. (1994) Are you using neuronal densities, synaptic densities or neurochemical densities as your definitive data? There is a better way to go. Prog. Neurobiol. 44, 233 247.
31. Romero-Ramos M., Venero J. L., Cano J. Machado A. (2000) Low selenium diet induces tyrosine hydroxylase enzyme in nigrostriatal system of the rat. Brain Res. Mol. Brain Res. 84, 7 16.
32. Ungerstedt U. Arbuthnott G. W. (1970) Quantitative recording of rotational behavior in rats after 6-hydroxy-dopamine lesions of the nigrostriatal dopamine system. Brain Res. 24, 485 493.