Strategies for gene therapy of Parkinson's disease using adeno-associated virus (AAV) vectors

Biogenic Amines

Volumn 15, Issue 1, 1999, Pages 21-37

  Ozawa, K ^a   Fan, D ^a   Ogawa, M ^a   Urabe, M ^a   Kume, A ^a   Monahan, J ^b   Nakano, I ^a  

^a JICHI MEDICAL UNIVERSITY   (Japan)

^b Avigen Inc   (United States)

Author keywords

6 OHDA lesioned rat; AAV vector; Aromatic L amino acid decarboxylase (AADC); Dopaminergic neurons; Gene therapy; Glial cell line derived neurotrophic factor (GDNF); Parkinson's disease; Tyrosine hydroxylase (TH)

Indexed keywords

AMINO ACID DECARBOXYLASE; DOPAMINE; ENZYME; LEVODOPA; NEUROTROPHIC FACTOR; TYROSINE; TYROSINE 3 MONOOXYGENASE; VIRUS VECTOR;

ADENOVIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; BEHAVIOR; BIOSYNTHESIS; CELL LINE; CORPUS STRIATUM; DENERVATION; DOPAMINE METABOLISM; DOPAMINERGIC NERVE CELL; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; GLIA CELL; HUMAN; MESENCEPHALON; NERVE CELL DEGENERATION; NEURITE; NONHUMAN; PARKINSON DISEASE; RAT; REVIEW; STEREOTAXIS; SUBSTANTIA NIGRA; TRANSGENE; VIRUS INFECTION;

EID: 0033063986     PISSN: 01688561     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (56)

1. Beck, K.D., Valverde, J., Alexi, T., Poulsen, K., Moffat, B., Vandlen, R.A., Rosenthal, A. and Hefti, F. (1995). Mesencephalic dopaminergic neurons protected by GDNF from axotomy-induced degeneration in the adult brain. Nature, 373: 339-341.
2. Bemheimer, H., Birkmayer, W., Homykiewicz, O., Jellinger, K. and Seitelberger, F. (1973). Brain dopamine and the syndromes of Parkinson and Huntington: clinical, morphological and neurochemical correlations. J. Neurol. Sci., 20: 415-455.
3. Bilang-Bleuel, A., Revah, F., Colin, P., Locquet, I., Robert, J.-J., Mallet, J. and Horellou, P. (1997). Intrastriatal injection of an adenoviral vector expressing glial-cell-line-derived neurotrophic factor prevents dopaminergic neuron degeneration and behavioral impairment in a rat model of Parkinson disease. Proc. Natl. Acad. Sci. USA, 94: 8818-8823.
4. Blömer, U., Naldini, L., Kafri, T., Trono, D., Verma, I.M. and Gage, F.H. (1997). Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector. J. Virol., 71: 6641-6649.
5. Choi-Lindberg, D.L., Lin, Q., Chang, Y.-N., Chiang, Y.L., Hay, C.M., Mohajeri, H., Davidson, B.L. and Bohn, M.C. (1997). Dopaminergic neurons protected from degeneration by GDNF gene therapy. Science, 275: 838-841.
6. Du, B., Wu, P., Boldt-Houle, D.M., and Terwilliger, E.F. (1996). Efficient transduction of human neurons with an adeno-associated virus vector. Gene Ther., 3: 254-261.
7. During, M.J., Naegele, J.R., O'Malley, K.L., and Geller, A.I. (1994). Long-term behavioral recovery in Parkinson's rats by an HSV vector expressing tyrosine hydroxylase. Science, 266: 1399-1403.
8. Fan, D., Ogawa, M., Ikeguchi, K., Fujimoto, K., Urabe, M., Kume, A., Nishizawa, M., Matsushita, N., Kiuchi, K., Ichinose, H., Nagatsu, T., Kurtzman, G.J., Nakano, I. and Ozawa, K. (1998a). Prevention of dopaminergic neuron death by adeno-associated virus vector-mediated GDNF gene transfer in rat mesencephalic cells in vitro. Neurosci. Lett., 248: 61-64.
9. Fan, D., Ogawa, M., Fujimoto, K., Ikeguchi, K., Ogasawara Y., Urabe, M., Nishizawa, M., Nakano, I., Yoshida, M., Nagatsu, I., Ichinose, H., Nagatsu, T., Kurtzman, G.J., and Ozawa, K. (1998b). Behavioral recovery in 6-OHDA-lesioned rats by cotransduction of striatum with tyrosine hydroxylase and aromatic L-amino acid decarboxylase genes using two separate AAV vectors. Hum. Gene Ther., 9: 2527-2535
10. Fisher, K.J., Jooss, K., Alston, J., Yang, Y., Haecker, S.E., High, K., Pathak, R., Raper, S.E. and Wilson, J.M. (1997). Recombinant adeno-associated virus for muscle directed gene therapy. Nat. Med., 3: 306-312.
11. Freed, C.R., Breeze, R.E., Rosenberg, N.L., Schneck, S.A., Kriek, E., Qi, J-X., Lone, T., Zhang, Y-B., Snyder, J.A., Wells, T.H., Ramig, L.O., Thompson, L., Mazziotta, J., Huang, S.C., Grafton, S., Brooks, D., Sawie, G., Schroter, G. and Ansari, A. (1992). Survival of implanted fetal dopamine cells and neurologic improvement 12 to 46 months after transplantation for Parkinson's disease. N. Engl. J. Med., 327: 1549-1555
12. Gash, D.M., Zhang, Z., Ovadia, A., Cass, W.A., Yi, A., Simmerman, L., Russell, D., Martin, D., Lapchak, P.A., Collins, F., Hoffer, B.J. and Gerhardt, D.A. (1996). Functional recovery in parkinsonian monkeys treated with GDNF. Nature, 380: 252-255.
13. Gearhart, D.A., Neafsey, E.J. and Collins, M.A. (1997). Characterization of brain beta-carboline-2-N-methyltransferase, an enzyme that may play a role in idiopathic Parkinson's disease. Neurochem. Res., 22: 113-121.
14. Geller, A.I., During, M.J., Oh, Y.J., Freese, A., and O'Malley, K. (1995). An HSV-1 vector expressing tyrosine hydroxylase cause production and release of 1-DOPA from cultured rat striatal cells. J. Neurochem., 64: 487-496.
15. Herzog, R.W., Hagstrom, J.N., Kung, S.H., Tai, S.J., Wilson, J.M., Fisher, K.J. and High, K.A. (1997). Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc. Natl. Acad. Sci. USA, 94: 5804-5809.
16. Ichinose, H., Kurosawa, Y., Titani, K., Fujita, K., and Nagatsu, T. (1989). Isolation and characterization of a cDNA clone encoding human aromatic 1-amino acid decarboxylase. Biochem. Biophys. Res. Commun., 164: 1024-1030.
17. Imaoka, T., Date, I., Ohmoto, T. and Nagatsu, T. (1998). Significant behavioral recovery in Parkinson's disease model by direct intracerebral gene transfer using continuous injection of a plasmid DNA-liposome complex. Hum. Gene Ther., 9: 1093-1102.
18. Jeager, C.B., Ruggiero, D.A., Albert, V.R., Joh, T.H. and Reis, D.J. (1984). Immunocytochemical localization of aromatic 1-amino acid decarboxylase. In: Handbook of Chemical Neuroanatomy, A. Björklund and T. Hokfelt (Eds.), Elsevier, Amsterdam, Vol 2, pp. 384-408.
19. Jiao, S., Gurevich, V., and Wolff, J.A (1993). Long-term correction of rat model of Parkinson's disease by gene therapy. Nature, 362: 450-453.
20. Jiao, S., Hogan, K., and Wolff, J.A. (1994). Gene therapy for neuromuscular disorders. Cur. Neurol., 14: 1-28.
21. Jinnah, H.A. and Friedmann, T. (1995). Gene therapy and the brain. Bri. Med. Bull., 51: 138-148.
22. Kamegai, M., Niijima, K., Kunishita, T., Nishizawa, M., Ogawa, M., Araki, M., Ueki, A., Konishi, Y. and Tabira, T. (1990). Interleukin 3 as a trophic factor for central cholinergic neurons in vitro and in vivo. Neuron, 2: 429-436.
23. Kang, U.J., Park, D.H., Wessel, T., Baker, H., and Joh, T.H. (1992). DOPA-decarboxylation in the striata of rats with unilateral substantia nigra lesions. Neurosci. Lett., 147: 53-57.
24. Kang, U.J., Fisher, L.J., Joh, T.H., O'Malley, K.L., and Gage, F.H. (1993). Regulation of dopamine production by genetically modified primary fibroblasts. J. Neurosci., 13: 5203-5211.
25. Kaplitt, M.G., Leone, P., Samulski, R.J., Xiao, X., Pfaff, D.W., O'Malley, K.L. and 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.
26. Kaneda, N., Kobayashi, K., Ichinose, H., Kishi, F., Nakazawa, A., Kurosawa, Y., Fujita, K., and Nagatsu, T. (1987). Isolation of a novel cDNA clone for human tyrosine hydroxylase: alternative RNA splicing produces four kinds of mRNA from a single gene. Biochem. Biophys. Res. Commun., 146: 971-975.
27. Kessler, P.D, Podsakoff, G.M., Chen, X., McQuiston, S.A., Colosi, P.C., Matelis, L.A., Kurtzman, G.J. and Byrne, B.J. (1996). Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc. Natl. Acad. Sci. USA, 93: 14082-14087.
28. Kotin, R.M. (1994). Prospects for the use of adeno-associated virus as a vector for human gene therapy. Hum. Gene Ther., 5: 793-801.
29. Le Gal La Salle, G,. Robert, J.J., Bernard, S., Ridoux, V., Stratford-Perricaudet, L.D., Perricaudet, M., and Mallet, J. (1993). An adenovirus vector for gene transfer into neurons and glia in the brain. Science, 259: 988-990.
30. Lin, L.-F.H., Doherty, D.H., Lile, J.D., Bektesh, S. and Collins, F. (1993). GDNF: a glial cell line-derived neurotrophic factor for midbrain dopaminergic neurons. Science, 260: 1130-1132.
31. Lundberg, C., Horellou, P., Mallet, J. and Björklund, A. (1996). Generation of DOPA-producing astrocytes by retroviral transduction of the human tyrosine hydroxylase gene: in vitro characterization and in vivo effects in the rat Parkinson model. Exp. Neurol., 139: 39-53.
32. Mandel, R.J., Spratt, S.K., Snyder, R.O. and Leff, S.E. (1997). Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson's disease in rats. Proc. Natl. Acad. Sci. USA, 94: 14083-14088.
33. Marcel, T. and Grausz, J.D. (1997). The TMC worldwide gene therapy enrollment report, end 1996. Hum. Gene Ther., 8: 775-800.
34. Martinez, A., Haavik, J., Flatmark, T., Arrondo, J.L.R. and Muga, A. (1996). Conformational properties and stability of tyrosine hydroxylase studied by infrared spectroscopy. J. Biol. Chem., 271: 19737-19742.
35. Martinez-Serrano, A., Lundberg, C., and Björklund, A. (1997). Use of conditionally immortalized neural progenitors for transplantation and gene transfer to the CNS. In: Isolation, Characterization and Utilization of CNS Stem Cells, F. Gage and Y. Christen (Eds), Springer-Verlag, Berlin, pp. 151-169.
36. Matsushita, N., Fujita, Y., Tanaka, N., Nagatsu, T. and Kiuchi. K. (1997). Cloning and structural organization of the gene encoding the mouse glial cell-line-derived neurotrophic factor, GDNF. Gene, 203: 149-157.
37. Matsushita, T., Elliger, S., Elliger, C., Podsakoff, G., Villarreal, L., Kurtzman, G.J., Iwaki, Y. and Colosi, P. (1998). Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther., 5: 938-945.
38. Mizuno, Y., Ikebe, S., Hattori, N., Mochizuki, H., Nishi, K., Yoshino, H. and Kondo, T. (1995). Pathogenesis of nigral cell death in Parkinson's disease. In: Alzheimer's and Parkinson's Disease, I. Hanin, M. Yoshida and A. Fisher (Eds.), Plemun, New York, pp. 211-216.
39. Muzyczka, N. (1992). Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr. Top. Microbiol. Immunol., 158: 97-129.
40. Nagatsu, I., Kondo, Y., Inagaki, S., Kojima, H. and Nagatsu, T. (1979). Immunofluorescent and biochemical studies on tyrosine hydroxylase and dopamine-beta-hydroxylase of the bullfrog sciatic nerves. Histochemistry, 61: 103-109.
41. Nagatsu, T. (1992). Molecular biology of dopamine systems. In: Controversies in the Treatment of Parkinson's Disease, U.K. Rinne and N. Yanagisawa (Eds), PMSI, Tokyo, pp. 15-26.
42. Nagatsu, T. (1997). Isoquinoline neurotoxins in the brain and Parkinson's disease. Neurosci. Res., 29: 99-111.
43. Naldini, L., Blömer, U., Gallay, P., Ory, D., Mulligan, R., Gage, F.H., Verma, I.M., and Trono, D. (1996). In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science, 272: 263-267.
44. Ogawa, M., Araki, M., Niijima, K., Naito, M. and Yoshida, M. (1992). Microglial reactions in the inferior olive and its tract induced by intraperitoneal injection of 3-acetylpyridine in the rat. Neurodegeneration, 1: 37-43.
45. Ogawa, M., Ogura, K., Nagatsuka, Y., Ohta, S., Imai, I., Nagatsu, I., Nagatsu, T. and Yoshida, M. (1995). Effects of chronic administration of 1,2,3,4-tetrahydroisoquinoline and its derivatives on the monkey: immunohistochemical study. In: Alzheimer's and Parkinson's Disease, I. Hanin, M. Yoshida and A. Fisher (Eds.), Plemun, New York, pp. 583-587.
46. Ross, G., Erickson R., Knorr, D., Motulsky, A.G., Parkman, R., Samulski, J., Straus, S.E. and Smith, B.R. (1996). Gene therapy in the United States: a five-year status report. Hum. Gene Ther., 7: 1781-1790.
47. Sauer, H., Rosenblad, C. and Björklund, A. (1995). Glial cell line-derived neurotrophic factor but not transforming growth factor b3 prevents delayed degeneration of nigral dopaminergic neurons following striatal 6-hydroxydopamine lesion. Proc. Natl. Acad. Sci. USA, 92: 8935-8939.
48. Snyder, R.O., Miao, C.H., Patijn, G.A., Spratt, S.K., Danos, O., Nagy, D., Gown, A.M., Winther, B., Meuse, L., Cohen, L.K., Thompson, A.R. and Kay, M.A. (1997). Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat. Genet., 16: 270-276.
49. Spencer, D.D., Robbins, R.J., Naftolin, F., Marek, K.L., Vollmer, T., Leranth, C., Roth, R.H., Price, L.H., Gjedde, A., Bunney, B.S., Sass, K.J., Elsworth, J.D., Kier, E.L., Makuch, R., Horrer, P.B. and Redmone, D.E.Jr. (1992). Unilateral transplantation of human fetal mesencephalic tissue into the caudate nucleus of patients with Parkinson's disease. N. Engl. J. Med., 327: 1541-1548.
50. Tashiro, T., Kaneko, T., Sugimoto, T., Nagatsu, I., Kikuchi, H., and Mizuno, N. (1989). Striatal neurons with aromatic 1-amino acid decarboxylase-like immunoreactivity in the rat. Neurosci. Lett., 100: 29-34.
51. Tomac, A., Lindqvist, E., Lin, L.-F.H., Ogren, S., Young, D., Hoffer, B.J. and Olson, L. (1995). Protection and repair of the nigrostriatal dopaminergic system by GDNF in vivo. Nature, 373: 335-339.
52. Unsicker, K. (1996). GDNF: a cytokine at the interface of TGF-b and neurotrophins. Cell Tissue Res., 286: 175-178.
53. Verma, I.M. and Somia, N. (1997). Gene therapy - promises, problems and prospects. Nature, 389: 239-242.
54. Wachtel, S.R., Bencsics, C. and Kang, U.J. (1997). Role of aromatic 1-amino acid decarboxylase for dopamine replacement by genetically modified fibroblasts in a rat model of Parkinson's disease. J. Neurochem., 69: 2055-2063.
55. Yoshida, M., Niwa, T. and Nagatsu, T. (1990). Parkinsonism in monkeys produced by chronic administration of an endogenous substance of the brain, tetrahydroisoquinoline: The behavioral and biochemical changes. Neurosci. Lett., 119: 109-113.
56. Zufferey, R., Nagy, D., Mandel, R.J., Naldini, L. and Trono. D. (1997). Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat. Biotechnol., 15: 871-875.