Approach to ex vivo gene therapy in the treatment of Parkinson's disease

Brain and Development

Volumn 22, Issue SUPPL. 1, 2000, Pages

  Ishida, Akihiko ^a   Yasuzumi, Fumioki ^a  

^a UNIVERSITY OF THE RYUKYUS   (Japan)

Author keywords

Dopamine; Ex vivo; Fibroblast; Gene therapy; Neurodegenerative diseases; Neurotrophic factor; Parkinson's disease; Vector

Indexed keywords

BASIC FIBROBLAST GROWTH FACTOR; BRAIN DERIVED NEUROTROPHIC FACTOR; CILIARY NEUROTROPHIC FACTOR; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; LEVODOPA; NEUROTROPHIC FACTOR; TYROSINE 3 MONOOXYGENASE; VIRUS VECTOR;

ANIMAL CELL; ANIMAL MODEL; AUTOTRANSPLANTATION; CELL TRANSPLANTATION; CELL TYPE; CLONING VECTOR; CONFERENCE PAPER; CONTROLLED STUDY; CORPUS STRIATUM; DEGENERATIVE DISEASE; DISEASE MODEL; DRUG DELIVERY SYSTEM; FETUS CELL; GENE THERAPY; NEUROPROTECTION; NONHUMAN; PARKINSON DISEASE; PROTEIN EXPRESSION; RETROVIRUS; SKIN FIBROBLAST; TECHNIQUE;

ANIMALS; BRAIN TISSUE TRANSPLANTATION; FIBROBLASTS; GENE THERAPY; GLIAL CELL LINE-DERIVED NEUROTROPHIC FACTOR; HUMANS; NEOSTRIATUM; NERVE GROWTH FACTORS; NERVE TISSUE PROTEINS; PARKINSON DISEASE; TRANSPLANTATION, AUTOLOGOUS;

EID: 0033839543     PISSN: 03877604     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Conference Paper

References (30)

1. Duvoisin R.C. Overview of Parkinson's disease. Ann NY Acad Sci. 648:1992;187-193.
2. Suhr S.T., Gage F.H. Gene therapy for neurologic disease. Arch Neurol. 50:1993;1252-1268.
3. Fisher L.J., Gage F.H. Novel therapeutic directions for Parkinson's disease. Mol Med Today. 1:1995;181-187.
4. Schofield J.P., Caskey C.T. Non-viral approaches to gene therapy. Br Med Bull. 51:1995;56-71.
5. Vile R.G., Russell S.J. Retroviruses as vector. Br Med Bull. 51:1995;12-30.
6. Naldini L., Blomer U., Gallay P., Ory D., Mulligan R., Gage F.H., et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science. 272:1996;263-267.
7. Palmer T.D., Rosman G.J., Osborne W.R.A., Miller A.D. Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced gene. Proc Natl Acad Sci USA. 88:1991;1330-1334.
8. Hock R.A., Miller A.D., Osborne W.R. Expression of human adenosine deaminase from various strong promoters after gene transfer into human hematopoietic cell line. Blood. 74:1989;876-881.
9. Fred W.J., Geller H.M., Poltorak M., Cannon-Spoor H.E., Cottingham S.L., LaMarca M.E., et al. Genetically altered and defined cell lines for transplantation in animal models of Parkinson's disease. Prog Brain Res. 82:1990;11-21.
10. Partridge T.A., Davies K.E. Myoblast-based gene therapies. Br Med Bull. 51:1995;123-137.
11. Ray J., Gage F.H. Gene transfer into established and primary fibroblast cell lines. BioTechniques. 13:1992;598-603.
12. Freed C.R., Breeze R.E., Rosenberg N.L., Schneck S.A., Kriek E., Qi J.X., et al. Survival of implanted fetal dopamine cells and neurologic improvement 12 to 46 months after transplantation for Parkinson's disease. N Engl J Med. 327:1992;1549-1555.
13. Madrazo I., Francobourland R., Ostroskysolis F., Augilera M., Cuevas C., Zamorano C., et al. Fetal homotransplants to the striatum of Parkinsonian subjects. Arch Neurol. 47:1990;1281-1285.
14. Isacson O., Deacon T.W., Pakzaban P., Galpern W.R., Dinsmore J., Burns L.H. Transplanted xenogeneic neural cells in neurodegenerative disease models exhibit remarkable axonal target specificity and distinct growth patterns of glial and axonal fibers. Nature Med. 1:1995;1189-1194.
15. Ungerstedt U., Arbuthnott W.A. Quantitative recording of rotational behavior in rats after 6-hydroxydopamine lesions of the nigrostriatal dopamine system. Brain Res. 24:1970;485-493.
16. 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 produced L-dopa. Nature. 6:1991;371-380.
17. Lundberg C., Horellou P., Mallet J., Bjorklund A. Generation of dopa-producing astrocytes by retroviral transduction of the human tyrosine hydroxylase. Exp Neurol. 139:1996;39-53.
18. Zhou H., Lund R.D. Effects of the age donor or host tissue on astrocyte migration from intracerebral xenografts of corpus callosum. Exp Neurol. 122:1993;155-164.
19. Spina M.B., Squinto S.P., Miller J., Lindsay R.M., Hyman C. Brain-derived neurotrophic factor protects dopamine neurons against 6-hydroxydopamine and N-methyl-4-phenylpyridinium ion toxicity. J Neurochem. 59:1992;99-106.
20. Hagg T., Varon S. Ciliary neurotrophic factor prevents degeneration of adult rat substantia nigra dopaminergic neurons in vivo. Proc Natl Acad Sci USA. 90:1993;6135-6139.
21. Otto D., Unsicker K. FGF-2 mediated protection of cultured mesencephalic dopaminergic neurons against MPTP and MPP+. J Neurosci. 34:1993;382-393.
22. Beck K.D., Valverde J., Alexi T., Poulsen K., Moffat B., Vandlen R.A., et al. Mesencephalic dopaminergic neurons protected by GDNF from axotomy induced degeneration in the adult brain. Nature. 373:1995;339-341.
23. Hou J.G., Lin L.F., Mytilineou C. Glial cell line-derived neurotrophic factor exerts neurotrophic effects on dopaminergic neurons in vitro and promotes their survival and regrowth after damage by 1-methyl-4-phenylpyridinium. J Neurochem. 66:1996;74-82.
24. Schinstine M., Gage F.H. Factors affecting proviral expression in primary cells grafted into the CNS. Waxman S.G. Molecular and cellular approaches to the treatment of neurological disease. 1993;311-323 Raven, New York.
25. Ishida A., Yamashiro K., Mukawa J., Hasegawa M. Regulation of L-DOPA production by genetically modified primary fibroblasts transfected with retrovirus vector system. Cell Trans. 5:1996;5-7.
26. Ishida A., Yamashiro K., Mukawa J., Hasegawa M. Tetrahydrobiopterin-dependent production of L-DOPA in genetically modified primary fibroblasts. Pteridine. 7:1996;151-153.
27. Kawaja M.D., Fisher L.J., Shinstine M., Jinnah H.A., Ray J., Chen L.S., et al. Grating genetically modified cells within the rat central nervous system. Dunnett S.B., Bjorklund A., et al. Neural transplantation. 1992;21-55 IRL, New York.
28. Bankiewicz K.S., Leff S.E., Nagy D., Jungles S., Rokovich J., Spratt K., et al. Practical aspects of the development of ex vivo and in vivo gene therapy for Parkinson's disease. Exp Neurol. 144:1997;147-156.
29. Ishida A., Yamashiro K., Mukawa J. Intracerebral gene therapy in the treatment of Parkinson's disease. Biochem Clin. 12:1997;61-65.
30. Leff S.E., Rendahl K.G., Spratt S.K., Kang U.J., Mandel R.J. In vivo L-dopa production by genetically modified primary rat fibroblast or 9L gliosarcoma cell grafts via coexpression of GTP cyclohydrolase I with tyrosine hydroxylase. Exp Neurol. 151:1996;249-264.