In vivo L-DOPA production by genetically modified primary rat fibroblast or 9L gliosarcoma cell grafts via coexpression of GTPcyclohydrolase I with tyrosine hydroxylase

Experimental Neurology

Volumn 151, Issue 2, 1998, Pages 249-264

  Leff, Stuart E ^a   Rendahl, Katherine G ^a   Spratt, S Kaye ^a   Kang, Un J ^b   Mandel, Ronald J ^a  

^a CELL GENESYS INC   (United States)

^b UNIVERSITY OF CHICAGO   (United States)

Author keywords

1 Dihydroxyphenylalanine; Dopamine; Gene therapy; Parkinson's disease; Rats; Tetrahydrobiopterin; Transgenes

Indexed keywords

APOMORPHINE; GUANOSINE TRIPHOSPHATE CYCLOHYDROLASE I; LEVODOPA; OXIDOPAMINE; TETRAHYDROBIOPTERIN; TYROSINE 3 MONOOXYGENASE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL CULTURE; CELL TRANSPLANTATION; CIRCLING BEHAVIOR; CONTROLLED STUDY; FIBROBLAST; GENE EXPRESSION; GENE THERAPY; GENETIC TRANSDUCTION; GLIOSARCOMA; HIGH PERFORMANCE LIQUID CHROMATOGRAPHY; HUMAN; HUMAN CELL; IMMUNOCYTOCHEMISTRY; MALE; MICRODIALYSIS; NONHUMAN; PARKINSON DISEASE; PRIORITY JOURNAL; RAT; TRANSGENE;

EID: 0032102936     PISSN: 00144886     EISSN: None     Source Type: Journal    
DOI: 10.1006/exnr.1998.6803     Document Type: Article

References (45)

1. Bankiewicz K. S., Leff S. E., Nagy D., Jungles S., Rokovich J., Spratt K., Cohen L., Libonati M., Snyder R. O., Mandel R. J. Practical aspects of the development of ex vivo and in vivo gene therapy for Parkinson's disease. Exp. Neurol. 144:1997;147-156.
2. Barker R., Dunnett S. B. Ibotenic acid lesions of the striatum reduce drug-induced rotation in the 6-hydroxydopamine-lesioned rat. Exp. Brain Res. 101:1994;365-374.
3. Bencsics C., Wachtel S. R., Milstien S., Hatakeyama K., Becker J. B., Kang U. J. Double transduction with GTP cyclohydrolase I and tyrosine hydroxylase is necessary for spontaneous synthesis of L-DOPA by primary fibroblasts. J. Neurosci. 16:1996;4449-4456.
4. Chase T. N., Baronti F., Fabbrini G., Heuser I. J., Juncos J. L., Mouradian M. M. Rationale for continuous dopaminomimetic therapy of Parkinson's disease. Neurology. 39:1989;7-10.
5. Chen K. S., Gage F. H. Somatic gene transfer of NGF to the aged brain: behavioral and morphological amelioration. J. Neurosci. 15:1995;2819-2825.
6. Choi-Lundberg D. L., Lin Q., Chang L.-N., Chiang L., Hay C. M., Mohajeri H., Davidson B. L., Bohn M. C. Dopaminergic neurons protected from degeneration by GDNF gene therapy. Science. 275:1997;838-841.
7. Danos O., Mulligan R. C. Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges. Proc. Natl. Acad. Sci. USA. 85:1988;6460-6464.
8. Davis J. L., Witt R. M., Gross P. R., Hokanson C. A., Jungles S., Cohen L. K., Danos O., Spratt S. K. Retroviral particles produced from a stable human-derived packaging cell line transduce target cells with very high efficiencies. Human Gene Ther. 8:1997;1459-1467.
9. Dunnett S. B., Bunch S. T., Gage F. H., Björklund A. Dopamine-rich transplants in rats with 6-OHDA lesions of the ventral tegmental area: Effects on spontaneous and drug-induced locomotor activity. Behav. Brain Res. 13:1984;71-82.
10. During M. J., Naegele J. R., O'Malley K. L., Geller A. I. Long-term behavioral recovery in parkinsonian rats by an HSV vector expressing tyrosine hydroxylase. Science. 266:1994;1399-1403.
11. Fisher L. J., Jinnah H. A., Kale L. C., Higgins G. A., Gage F. H. Survival and function of intrastriatally grafted primary fibroblasts genetically modified to produce L-DOPA. Neuron. 6:1991;371-380.
12. Gage F. H., Wolff J. A., Rosenberg M. B., Xu L., Yee J.-K., Shults C., Friedmann T. Grafting genetically modified cells to the brain: Possibilities for the future. Neurosci. 23:1987;795-807.
13. Gossen M., Bujard H. Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc. Natl. Acad. Sci. USA. 89:1992;5547-5557.
14. Gutlich M., Jaeger E., Rucknagel K. P., Werner T., Rodl W., Ziegler I., Bacher A. Human GTP cyclohydrolase I: Only one out of three cDNA isoforms gives rise to the active enzyme. Biochem. J. 302:1994;215-2212.
15. Harms J. S., Splitter G. A. Interferon-gamma inhibits transgene expression drive by SV40 or CMV promoters but augments expression driven by the mammalian MHC I promoter. Hum. Gene Ther. 6:1995;1291-1297.
16. Horellou P., Marlier L., Privat A., Mallet J. Behavioural effect of engineered cells that synthesize L-DOPA or dopamine after grafting into the rat neostriatum. Eur. J. Neurosci. 2:1990;116-119.
17. Horellou P., Vigne E., Castel M. N., Barneoud P., Colin P., Perricaudet M., Delaere P., Mallet J. Direct intracerebral gene transfer of an adenoviral vector expressing tyrosine hydroxylase in a rat model of Parkinson's disease. NeuroReport. 6:1994;49-53.
18. Horellou P., Vigne E., Castel M. N., Barneoud P., Perricaudet M., Delaire P., Mallet J. Transplantation of cells genetically modified and direct intracerebral gene transfer with an adenoviral vector expressing tyrosine hydroxylase in a rat model of parkinson's disease. Restor. Neurol. Neurosci. 8:1995;63.
19. Hurn B. A., Chantler S. M. Production of reagent antibodies. Methods Enzymol. 70:1980;104-142.
20. Isacson O. Behavioral effects and gene delivery in a rat model of Parkinson's disease. Science. 269:1995;856-857.
21. Kaplitt M. G., Leone P., Samulski R. J., Xiao X., Pfaff D. W., O'Malley K. L., During M. J. Long term gene expression and phenotypic correction using adeno associated virus vectors in the mammalian brain. Nat. Genet. 8:1994;148-153.
22. Kawaja M. D., Fagan A. M., Firestein B. L., Gage F. H. Intracerebral grafting of cultured autologous skin fibroblasts into the rat striatum: An assessment of graft size and ultrastructure. J. Comp. Neurol. 307:1991;695-706.
23. Kirk R. E. Experimental Design: Procedures for the Behavioral Sciences. 1968;Brooks/Cole, Belmont.
24. Lentz S. I., Kapatos G. Tetrahydrobiopterin biosynthesis in the rat brain: heterogeneity of GTP cyclohydrolase i mRNA expression in monoamine-containing neurons. Neurochem. Int. 28:1996;569-582.
25. Levine R. A., Kuhn D. M., Lovenberg W. The regional distribution of hydroxylase cofactor in rat brain. J. Neurochem. 13107:1979;12592.
26. Levine R. A., Miller L. P., Lovenberg W. Tetrahydrobiopterin in striatum: Localization in dopamine nerve terminals and role in catecholamine synthesis. Science. 214:1981;919-921.
27. Lundberg C., Horellou P., Mallet J., Bjorklund A. Generation of DOPA-producing astrocytes by retroviral transduction of the human tyrosine hydroxylase gene: Exp. Neurol. 139:1996;39-53.
28. Mandel R. J., Clevenger D. G., Nagy D., Jaret T. M., Morten M., Leff S. E. Bioactivity of syngeneic rat fibroblasts genetically modified to express human nerve growth factor as demonstrated by enhanced survival of medial septal cholinergic neurons after fimbria-fornix transection. Exp. Neurol. 135:1995;167.
29. Naldini L., Blömer U., Gallay P., Ory D., Mulligan R., Gage F. H., Verma I. M., Trono D. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science. 272:1996;263-267.
30. Naviaux R. K., Verma I. M. Retroviral vectors for persistent expression in vivo. Curr. Opin. Biotech. 3:1992;54-547.
31. Nichol C. A., Smith G. K., Duch D. S. Biosynthesis and metabolism of tetrahydrobiopterin and molybdopterin. Annu. Rev. Biochem. 54:1985;729-764.
32. No D., Yao T.-P., Evans R. M. Ecdysone-inducible gene expression in mammalian cells and transgenic mice. Proc. Nat. Acad. Sci. USA. 93:1996;3346-3351.
33. Obeso J. A., Grandas F., Herrero M. T., Horowski R. The role of pulsatile versus continuous dopamine receptor stimulation for functional recovery in parkinson's disease. Eur. J. Neurosci. 6:1994;889-897.
34. Paxinos G., Watson C. The Rat Brain in Stereotaxic Coordinates. 1987;Academic Press, San Diego.
35. Qin L., Ding Y., Pahud D. R., Chang E., Imperiale M. J., Bromberg J. S. Promoter attenuation in gene therapy: interferon-ξ and tumor necrosis factor-α inhibit transgene expression. Hum. Gene Ther. 8:1997;2019-2029.
36. Rivera V. M., Clackson T., Natesan S., Pollock R., Amara J. F., Keenan T., Magari S. R., Phillips T., Courage N. L., Cerasoli F. Jr., Holt D. A., Gilman M. A humanized system for pharmacologic control of gene expression. Nature Med. 2:1996;1028-1032.
37. Schmidt R. H., Björklund A., Stenevi U., Dunnett S. B., Gage F. H. Intracerebral grafting of neuronal cell suspensions III. Activity of intrastriatal nigral suspension implants as assessed by measurements of dopamine synthesis and metabolism. Acta Physiol Scand. 522:1983;19-28.
38. Schuh L. A., Bennett J. P. Suppression of dyskinesias in advanced parkinson's disease. 1. Continuous intravenous levodopa shifts dose response for production of dyskinesias but not for relief of parkinsonism in patients with advanced parkinson's disease. Neurology. 43:1993;1545-1550.
39. Sigel M. B., Sinha Y. N., Vanderlaan W. P. Production of antibodies by inoculation into lymph nodes. Methods Enzymol. 93:1983;3-12.
40. Sternberger L. A., Hardy P. H., Cuculis J. J., Meyer H. G. The unlabelled antibody-enzyme method of immunohistochemistry. Preparation and properties of soluble antigen-antibody complex (horseradish peroxidase-antihorseradish peroxidase) and its use in the identification of spirochetes. J. Histochem. Cytochem. 18:1970;315-333.
41. Uchida K., Ishii A., Kaneda N., Toya S., Nagatsu T., Kohsaka S. Tetrahydrobiopterin-dependent production of L-dopa in NRK fibroblasts transfected with tyrosine hydroxylase cDNA: Future use for intracerebral grafting. Neurosci. Lett. 109:1990;282-286.
42. Ungerstedt U., Arbuthnott G. W. Quantitative recording of rotational behavior in rats after 6-hydroxy-dopamine lesions of the nigrostriatal dopamine system. Brain Res. 24:1970;485-493.
43. Werner E. R., Werner-Felmayer G., Fuchs D., Hausen A., Reibnegger G., Yim J. J., Pfeiderer W., Wachter H. Tetrahydrobiopterin biosynthetic activities in human macrophages, fibroblasts, THP-1, and T 24 cells. J. Biol. Chem. 265:1990;3189-3192.
44. Wolff J. A., Fisher L. J., Xu L., Jinnah H. A., Langlais P. J., Iuvone P. M., O'Malley K. L., Rosenberg M. B., Shimohama S., Friedmann T., Gage F. H. Grafting fibroblasts genetically modified to produce L-dopa in a rat model of Parkinson's disease. Proc. Natl. Acad. Sci. USA. 86:1989;9011-9014.
45. Wu D. K., Cepko C. L. The stability of endogenous tyrosine hydroxylase protein in PC-12 cells differs from that expressed in mouse fibroblasts by gene transfer. J. Neurochem. 62:1994;863-872.