Targets for gene therapy of Parkinson's disease: Growth factors, signal transduction, and promoters

Experimental Neurology

Volumn 144, Issue 1, 1997, Pages 74-81

  During, Matthew J ^a,b   Leone, Paola ^a  

^a YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF AUCKLAND   (New Zealand)

Author keywords

[No Author keywords available]

Indexed keywords

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; DOPAMINERGIC NERVE CELL; GENE THERAPY; NIGRONEOSTRIATAL SYSTEM; NONHUMAN; PARKINSON DISEASE; PRIORITY JOURNAL; PROMOTER REGION; SIGNAL TRANSDUCTION;

EID: 0031104731     PISSN: 00144886     EISSN: None     Source Type: Journal    
DOI: 10.1006/exnr.1996.6391     Document Type: Article

References (65)

1. 1. ALEXI, T., AND F. HEFTI. 1993. Trophic actions of transforming growth factor alpha on mesencephalic dopaminergic neurons developing in culture. Neuroscience 55: 903-918.
2. 2. ALTAR, C. A., C. B. BOYLAN, M. FRITSCHE, AND B. E. JONES, et al. 1994. Efficacy of brain-derived neurotrophic factor and neurotrophin-3 on neurochemical and behavioral deficits associated with partial nigrostriatal dopamine lesions. J. Neurochem. 63: 1021-1032.
3. 3. ANDERSEN, J. K., D. A. GARBER, C. A. MEANEY, AND X. O. BREAKEFIELD. 1992. Gene transfer into mammalian central nervous system using herpes virus vectors: extended expression of bacterial lacZ in neurons using the neuron-specific enolase promoter. Hum. Gene Ther. 3:487-99.
4. 4. BANNON, M. J., M. S. POOSCH, Y. XIA, AND D. J. GOEBEL et al. 1992. Dopamine transporter mRNA content in human substantia nigra decreases precipitously with age. Proc. Natl. Acad. Sci. USA 89: 7095-7099.
5. 5. BENTLAGE, C. et al. 1993. Combined intranigral and intrastriatal microtransplantation of fetal dopamine neurons in 6-hydroxydopamine lesioned rats. Soc. Neurosci. Abstr. 19: 284.8.
6. 6. BHATT, M. H., B. J. SNOW, W. R. MARTIN, AND B. D. PATE et al. 1991. Positron emission tomography suggests that the rate of progression of idiopathic parkinsonism is slow. Ann. Neurol. 29: 673-677.
7. 7. BUGIANI, O. S., F. SALVARINI, G. PERDELLI, AND L. MANCARDI et al. 1978. Nerve cell loss with aging in the putamen. Eur. Neurol. 17: 286-291.
8. 8. CASPAR, D., C. MYTILINEOU, AND M. BLUM. 1991. EGF enhances the survival of dopamine neurons in rat embryonic mesencephalon primary culture. J. Neurosci. Res. 30: 372-381.
9. 9. CHASE, T. N., T. M. ENGBER, AND M. M. MOURADIAN. 1994. Palliative and prophylactic benefits of continuously administered dopaminomimetics in Parkinson's disease. Neurology 44(7 Suppl. 6): S15-8.
10. 10. CHOI-LUNDBERG, D. L., Q. LIN, H. MOHAJERI, AND Y. N. CHANG et al. 1996. GDNF gene therapy protects dopaminergic neurons from degeneration in a rat model of Parkinson's Disease. Cold Spring Harbor Meeting Meeting on Gene Therapy. [Abstract 61].
11. 11. DATE, I., M. F. NOTTER, S. Y. FELTEN, AND D. L. FELTEN. 1990. MPTP-treated young mice but not aging mice show partial recovery of the nigrostriatal dopaminergic system by stereotaxic injection of acidic fibroblast growth factor (aFGF). Brain Res. 526: 156-160.
12. 12. DRUCKER COLIN, R., AND F. GARCIA HERNANDEX. 1991. A new motor test sensitive to aging and dopaminergic function. J. Neurosci. Methods 39: 153-161.
13. 13. DURING, M. J., I. N. ACWORTH, AND R. J. WURTMAN. 1989 Dopamine release in rat striatum: Physiological coupling to tyrosine supply. J. Neurochem. 52: 1449-1454.
14. 14. DURING, M. J., A. FREESE, P. KIBAT, AND A. Y. DEUTCH. et al. 1992. Biochemical and behavioral recovery in a rodent model of Parkinson's disease following stereotactic implantation of dopamine-containing liposomes. Exp. Neurol. 115: 193-199.
15. 15. DURING, M. J. 1992. Genetic manipulation of signal transduction pathways in the adult mammalian brain. Soc. Neurosci. Abstr. 18: 453.3.
16. 16. DURING, M. J., J. R. NAEGELE, K. L. O'MALLEY, AND A. I. GELLER. 1994. Long-term behavioral recovery in Parkinsonian rats by a HSV vector expressing tyrosine hydroxylase. Science 266: 1399-1403.
17. 17. DURING, M. J. et al. 1995. Neuroprotection and neuroregeneration in rodent and primate parkinsonian models using AAV vectors expressing GDNF. Soc. Neurosci. Abstr. 21: 225.9.
18. 18. EMERICH, D. F., P. MCDERMOTT, P. KRUEGER, AND M. BANKS et al. 1993. Locomotion of aged rats: Relationship to neurochemical but not morphological changes in nigrostriatal dopaminergic neurons. Brain Res. Bull. 32: 477-486.
19. 19. ENGELE, J., AND M. C. BOHN. 1991. The neurotrophic effects of fibroblast growth factors on dopaminergic neuros in vitro are mediated by mesencephalic glia. J. Neurosci. 11:3070-3078.
20. 20. FELTEN, D. L., S. Y. FELTEN, K. STEECE COLLIER, AND I. DATE et al. 1992. Age-related decline in the dopaminergic nigrostriatal system: The oxidative hypothesis and protective strategies. Ann. Neurol. 32: S133-S136.
21. 21. FINCH, C. E., P. K. RANDALL, AND J. F. MARSHALL. 1981. Aging and basal ganglia functions. Annu. Rev. Gerontol. Geriat. 2: 47-49.
22. 22. FERRARI, G., AND L. A. GREENE. 1994. Proliferative inhibition by dominant-negative Ras rescues naive and neuronally differentiated PC12 cells from apoptotic death. EMBO J. 13: 5922-5928.
23. 23. FRIM, D. M., T. A. UHLER, W. R. GALPERN, AND M. F. BEAL et al. 1994. Implanted fibroblasts genetically engineered to produce brain-derived neurotrophic factor prevent MPTP toxicity to dopaminergic neurons in the rat. Proc. Natl. Acad. Sci. USA 91: 5104-5108.
24. 24. GELLER, A. I., M. J. DURING, A. FREESE, AND J. R. HAYCOCK et al. 1993. Stable modification of rat sympathetic neuron physiology and neurotransmitter release by expression of adenylate cyclase from an HSV-1 vector. Proc. Natl. Acad. Sci. USA 90: 7603-7607.
25. 25. HADJICONSANTINOU, M., J. G. FIKIN, A. DALIA, AND N. H. NEFF. 1991. Epidermal growth factor enhances striatal dopaminergic parameters in the MPTP-treated mouse. J. Neurochem. 57: 479-482.
26. 26. HAGG, T., AND S. VARON. 1993. Ciliary neurotrophic factor prevents degeneration of adult rat substantia nigra dopaminergic neurons in vivo. Proc. Natl. Acad. Sci. USA 90: 6315-6319.
27. 27. HARDEN, D. G., AND A. A. GRACE. 1995. Activation of dopamine cell firing by repeated L-DOPA administration to dopamine-depleted rats: its potential role in mediating the therapeutic response to L-DOPA treatment. J. Neurosci. 15: 6157-6166.
28. 28. HEFTI, F., E. MELAMED, B. J. SAHAKIAN, AND R. J. WURTMAN. 1980. Circling behavior in rats with partial, unilateral nigrostriatal lesions: effects of amphetamine, apomorphine, and DOPA. Pharmacol. Biochem. Behav. 12: 185-188.
29. 29. Ho, D. Y., J. R. MCLAUGHLIN, AND R. M. SAPOLSKY. 1996. Inducible gene expression from defective Herpes Simplex virus vectors using the tetracycline-responsive prometer system. Mol. Brain Res. 41(1-2): 200-209.
30. 30. HORELLOU, P., E. VIGNE, M. N. CASTEL, AND P. BARNEOUD et al. 1994. Direct intracerebral gene transfer of an adenoviral vector expressing tyrosine hydroxylase in a rat model of Parkinson's disease. Neuroreport 6: 49-53.
31. 31. HORNEKIEWICZ, O. 1992. The primary site of dopamine neuron damage in Parkinson's Disease: Substantia nigra or striatum. Mov. Disorders 7: 288.
32. 32. HYMAN, C. et al. 1994. BDNF is a neurotrophic factor for dopaminergic neurons of the substantia nigra. Nature 350: 230-232.
33. 33. HYMAN, C., M. JUHASZ, C. JACKSON, AND P. WRIGHT et al. 1994. Overlapping and distinct actions of the neurotrophins BDNF, NT-3, and NT-4/5 on cultured dopaminergic and GABAergic neurons of the ventral mesencephalon. J. Neurosci. 14: 335-347.
34. 34. JOLICOEUR, F. B., R. RIVEST, AND A. DRUMHELLER. 1991. Hypokinesia, rigidity, and tremor induced by intrahypothalamic 6-OHDA lesions in the rat. Brain Res. Bull. 26: 317-320.
35. 35. JONES, B. E. et al. 1993. The effects of BDNF and NT-3 in a selective striatal dopamine lesion produced by chronic intrastriatal infusion of 6-OHDA. Soc. Neurosci. Abstr. 19: 662.
36. 36. JONGEN RELO, A. L., G. J. DOCTER, A. J. JONKER, AND P. VOORN. 1995. Differential localization of mRNAs encoding dopamine D1 or D2 receptors in cholinergic neurons in the core and shell of the rat nucleus accumbens. Mol. Brain Res. 28: 169-74.
37. 37. KAPLITT, M. G., A. D. KWONG, S. P. KLEOPOULOS, AND C. V. MOBBS et al. 1994. Preproenkephalin promoter yields region-specific and long-term expression in adult brain following direct in vivo gene transfer via a defective herpes simplex viral vector. Proc. Natl. Acad. Sci. USA 91: 8979-8983.
38. 38. KAPLITT, M. G., P. LEONE, R. J. SAMULSKI, AND X. XIAO et al. 1994. Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nature Genet. 8: 148-154.
39. 39. KNUSEL, B., P. P. MICHEL, J. S. SCHHWABER, AND F. HEFTI. 1990. Selective and non-selective stimulation of central cholinergic and dopaminergic development in vitro by nerve growth factor, basic fibroblast growth factor, epidermal growth factor, insulin and the insulin-like growth factors I and II. J. Neurosci. 10: 558-570.
40. 40. KOH, J. Y., B. J. GWAG, D. LOBNER, AND D. W. CHOI. 1995. Potentiated necrosis of cultured cortical neurons by neurotrophins. Science 268: 573-575.
41. 41. KOJIMA, H. et al. 1993. Isolation of a subclonal cell line of PC12 transfected with dexamethasone-regulated ras oncogene: morphological differentiation, biochemical properties, and tumorigenicity. J. Biochem. 114: 194-202.
42. 42. KRIEGLSTEIN, K., C. SUTER CRAZZOLARA, W. H. FISCHER, AND K. UNSICKER. 1995. TGF-beta superfamily members promote survival of midbrain dopaminergic neurons and protect them against MPP+ toxicity. EMBO J. 14(4): 736-742.
43. 43. LIN, L. F., D. H. DOHERTY, J. D. LILE, AND S. BEKTESH et al. 1993. GDNF: A glial cell line-derived neurotrophic factor for midbrain dopaminergic neurons. Science 260: 1130-1132.
44. 44. LINDNER, M. D., S. R. WINN, E. E. BAETGE, AND J. P. HAMMANG et al. 1995. Implantation of encapsulated catecholamine and GDNF-producing cells in rats with unilateral dopamine depletions and parkinsonian symptoms. Exp. Neurol. 132: 62-76.
45. 45. LUCIDI-PHILLIPI, C. et al. 1995. Brain-derived neurotrophic factor-transduced fibroblasts: Production of BDNF and effects of grafting to the adult rat brain. J. Comp. Neurol. 354: 361-76.
46. 46. LUCIDI-PHILLIPI, C. A., D. O. CLARY, L. F. REICHARDT, AND F. H. GAGE. 1996. TrkA activation is sufficient to rescue axotomized cholinergic neurons. Neuron 16: 653-63.
47. 47. MARTILLA, R. J. 1992. Parkinson's Disease Incidence. Handbook of Parkinson's Disease. W C. Koller, D. Marcel: New York.
48. 48. MITSUMOTO, H., K. IKEDA, B. KLINKOSZ, AND J. M. CEDARBAUM et al. 1994. Arrest of motor neuron disease in wobbler mice cotreated with CNTF and BDNF. Science 265: 1107-1110.
49. 49. NISHI, R. 1994. Neurotrophic factors: Two are better than one. Science 265: 1052-1053.
50. 50. No, D., T. P. YAO, AND R. M. EVANS. 1996. Ecdysone-inducible gene expression in mammalian cells and transgenic. Proc. Natl. Acad. Sci. USA 93(8): 3346-3351.
51. 51. NORTON, D., D. JAMES, AND T. SCHALLERT. 1993. Forelimb motor function, neostriatal dendritic spine asymmetries, and effects of intranigral muscimol after unilateral neustriatal dopamine depletion in adult rats. Soc. Neurosci. Abstr. 19: 165.9.
52. 52. PEZZOLI, G., S. FAHN, AND A. DWORK et al. 1988. Non-chromaffin tissue plus nerve growth factor reduces experimental parkinsonism in aged rats. Brain Res. 459: 398-103.
53. 53. PRASAD, C., H. IKEGAMI, I. SHIMIZU, AND E. S. ONAIVI. 1994. Chronic nicotine intake decelerates aging of the nigrostriatal dopaminergic neurons. Life Sci. 54: 169-1184.
54. 54. PUJOL, J., C. JUNQUE, P. VENDRELL, AND J. M. GRAU et al. 1992. Reduction of substantia nigra width and motor decline in aging and Parkinson's Disease. Arch. Neurol. 49: 144-148.
55. 55. SAUER, H., W. FISHER, G. NIKKHAH, AND S. J. WIEGAND et al. 1993. Brain-derived neurotrophic factor enhances function rather than survival of intrastriatal dopamine cell-rich grafts. Brain Res. 626: 37-44.
56. 56. SAUER, H., AND W. H. OERTEL. 1994. Progressive degeneration of nigrostriatal dopamine neurons following intrastriatal terminal lesions with 6-hydroxydopamine: A combined retrograde tracing and immunocytochemical study in the rat. Neuroscience 59: 401-115.
57. 57. SCHULIGOI, R., J. FERNANDEZ, R. P. HEAVENS, AND D. J. SIRINATHSINGHI. 1993. Decreased tyrosine hydroxylase mRNA but not cholecystokinin mRNA in the pars compacta of the substantia nigra and ventral tegmental area of aged rats. Mol. Brain Res. 19: 333-338.
58. 58. SEVERSON, J. A., J. MARCUSSDON, B. WINBLAND, AND C. E. FINCH. 1982. Age-related loss of dopaminergic sites in human basal ganglia. J. Neurochem. 39: 1623-1632.
59. 59. STEINBUSCH, H. M., R. J. VERMEULEN, AND J. A. TONNAER. 1990. Basic fibroblast growth factor enhances survival and sprouting of fetal dopaminergic cells implanted in the denervated rat caudate-putamen: Preliminary observations. Prog. Brain Res. 82: 81-86.
60. 60. UNGERSTEDT, U., AND G. ARBUTHNOTT. 1970. Quantitative recording of rotational behavior in rats after 6-hydroxydopamine lesions of the nigrostriatal system. Brain Res. 24: 485-493.
61. 61. WALLACE, D. C. 1992. Mitochondrial genetics: A paradigm for aging and degenerative diseases? Science 256: 628-632.
62. 62. WANG, Y., B. W. O'MALLEY JR, S. Y. TSAI, AND B. W. O'MALLEY. 1994. A regulatory system for use in gene transfer. Proc. Natl. Acad. Sci. USA. 91(17): 8180-8184.
63. 63. WOLFF, J. A. 1993. Postnatal gene transfer into the central nervous system. Curr. Opin. Neurobiol. 3: 743-748.
64. 64. XU, H., H. FEDEROFF, J. MARAGOS, L. F. PARADA, AND J. A. KESSLER. 1994. Viral transduction of trkA into cultured nodose and spinal motor neurons conveys NGF responsiveness. Dev. Biol. 163: 152-161.
65. 65. YOSHIMOTO, Y., Q. LIN, T. J. COLLIER, D. M. FRIM et al. 1995. Astrocytes retrovirally with BDNF elicit behavioral improvements in a rat model of Parkinson's Disease. Brain Res. 691: 25-36.