Skip to main content

Somatic gene therapy in animal models of Parkinson’s disease

  • Conference paper
  • 78 Accesses

Part of the book series: 6th International Winter Conference on N eurodegeneration ((NEURAL SUPPL,volume 55))

Summary

Gene therapy in Parkinson’s disease (PD) emerged about 10 years ago but until now, no clinical trials are under way, because most approaches have failed to show long-term therapeutic effects in PD animal models and because safety concerns precluded the use in humans so far. This review tries to give an overview on the development of different strategies in gene therapy in PD animal models and point out new and possibly more successful directions, including the transplantation of neural precursor cells and pig tissue.

This is a preview of subscription content, log in via an institution.

Buying options

Chapter
USD   29.95
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
eBook
USD   39.99
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book
USD   54.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Purchases are for personal use only

Learn about institutional subscriptions

Preview

Unable to display preview. Download preview PDF.

Unable to display preview. Download preview PDF.

References

  • Afione SA, Conrad CK, Kearns WG, Chunduru S, Adams R, Reynolds TC, Guggino WB, Cutting GR, Carter BJ, Flotte TR (1996) In vivo model of adeno-associated virus vector persistence and rescue. J Virol 70: 3235–3241.

    PubMed  CAS  Google Scholar 

  • Akli S, Caillaud C, Vigne E, Startford-Perricaudet LD, Poenaru L, Perricaudet M, Kahn A, Peschanski MR (1993) Transfer of foreign gene into the brain using adenovirus vectors. Nat Gen 3: 224–228.

    Article  CAS  Google Scholar 

  • Anderson WF (1998) Human gene therapy. Nature 392: S25–S30.

    Article  Google Scholar 

  • Anton R, Kordower JH, Maidment NT, Manaster JS, Kane DJ, Rabizadeh S, Schueller SB, Yang J, Edwards RH, Markham CH, Bredesen DE (1994) Neural-targeted gene therapy for rodent and primate hemiparkinsonism. Exp Neurol 127: 207–218.

    Article  PubMed  CAS  Google Scholar 

  • Bencsics C, Wachtel RS, Milstien S, Kang UJ (1996) Double transduction with GTP cyclohydrolase 1 and tyrosine hydroxylase is necessary for spontaneous synthesis of L-dopa by primary fibroblasts. J Neurosci 16: 4449–4456.

    PubMed  CAS  Google Scholar 

  • Berns KI, Bohenzky RA (1987) Adeno-associated viruses: an update. Adv Virus Res 32:243–306.

    Article  PubMed  CAS  Google Scholar 

  • Bilang-Bleuel A, Revah F, Colin P, Locquet I, Robert JJ, Mallet J, Horellou P (1997) Intrastriatal injection of an adenoviral vector expressing glial-cell-line-derived neurotrophic factor prevents dopaminergic neuron degeneration and behavioural impairment in a rat model of Parkinson disease. Proc Natl Acad Sci USA 94: 8818–8823.

    Article  PubMed  CAS  Google Scholar 

  • Björklund A, Rosenblad C, Winkler C, Kirik D (1997) Studies on neuroprotective and regenerative effects of GDNF in a partial lesion model of Parkinson’s disease. Neurobiol Dis 4: 186–200.

    Article  PubMed  Google Scholar 

  • Buc-Caron MH (1995) Neuroepithelial progenitor cells explanted from human fetal brain proliferate and differentiate in vitro. Neurobiol Dis 2: 37–47.

    Article  PubMed  CAS  Google Scholar 

  • Bukrinsky MI, Haggerty S, Dempsey MP, Sharova N, Adzhubei A, Spitz L, Lewis P, Goldfarb D, Emerman M, Stevenson M (1993) A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells. Nature 365: 666–669.

    Article  PubMed  CAS  Google Scholar 

  • Choi-Lundberg D, Lin Q, Chang Y, Chiang YL, Hay CM, Mohajeri H, Davidson BL, Bohn MC (1997) Dopaminergic neurons protected from degeneration by GDNF gene therapy. Science 275: 838–841.

    Article  PubMed  CAS  Google Scholar 

  • Cunningham LA, Hansen JT, Short MP, Bohn MC (1991) The use of genetically altered astrocytes to provide nerve growth factor to adrenal chromaffin cells grafted into the striatum. Brain Res 561: 192–202.

    Article  PubMed  CAS  Google Scholar 

  • Deacon T, Schumacher J, Dinsmore J, Thomas C, Palmer P, Isacson O (1997) Histological evidence of fetal pig neural cell survival after transplantation into a patient with Parkinson’s disease. Nat Med 3: 350–353.

    Article  PubMed  CAS  Google Scholar 

  • Dorling A, Riesbeck K, Warrens A, Lechler R (1997) Clinical xenotransplantation of solid organs. Lancet 349: 867–871.

    Article  PubMed  CAS  Google Scholar 

  • Dörig RE, Marcil A, Chopra A, Richardson CD (1993) The human CD46 molecule is a receptor for measles virus (Edmonston Strain). Cell 75: 295–305.

    Article  PubMed  Google Scholar 

  • During MJ, Naegele JR, O’Malley KL, Geller AI (1994) Long-term behavioral recovery in parkinsonian rats by an HSV vector expressing tyrosine hydroxylase. Science 266: 1399–1403.

    Article  PubMed  CAS  Google Scholar 

  • During MJ, Samulski RJ, Elsworth JD, Kaplitt MG, Leone P, Xiao X, Li J, Freese A (1998) In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector. Gene Ther 5: 820–827.

    Article  PubMed  CAS  Google Scholar 

  • Duvoisin R (1992) Overview of Parkinson’s disease. Ann N Y Acad Sci 648: 187–193.

    Article  PubMed  CAS  Google Scholar 

  • Fearnley JM, Lees AJ (1991) Aging and Parkinson’s disease: Substantia nigra regional specificity. Brain 114: 2283–2301.

    Article  PubMed  Google Scholar 

  • Ferrari FK, Samulski T, Shenk T, Samulski RJ (1996) Second-strand synthesis is a ratelimiting step for efficient transduction by recombinant adeno-associated virus vectors. J Virol 70: 3227–3234.

    PubMed  CAS  Google Scholar 

  • Fink DJ, Sternberg PC, Weber M, Mata M, Goins WF, Glorioso JC (1992) In vivo expression of beta-galactosidase in hippocampal neurons by HSV-mediated gene transfer. Hum Gene Ther 3: 11–19.

    Article  PubMed  CAS  Google Scholar 

  • Fisher L, Jinnah H, Kale L, Higgins G, Gage F (1991) Survival and function of intrastriatally grafted primary fibroblasts genetically modified to produce L-DOPA. Neuron 6: 371–380.

    Article  PubMed  CAS  Google Scholar 

  • Forno LS (1996) Neuropathology of Parkinson’s disease. J Neuropath Exp Neurol 55: 259–272.

    Article  PubMed  CAS  Google Scholar 

  • Frederiksen K, Jat PS, Valtz N, Levy D, McKay R (1988) Immortalization of precursor cells from the mammalian CNS. Neuron 1: 439–448.

    Article  PubMed  CAS  Google Scholar 

  • Freed CR, Breeze RE, Rosenberg NL, Schneck SA, Kriek E, Qi JX, Lone T, Zhang YB, Snyder JA, Wells TH, et al (1992) Survival of implanted fetal dopamine cells and neurological improvement 12 to 46 months after transplantation for Parkinson’s disease. N Engl J Med 327: 1549–1555.

    Article  PubMed  CAS  Google Scholar 

  • Freese A, Stern M, Kaplitt MG, O’Connor WM, Abbey MV, O’Connor MJ, During MJ (1996) Prospects for gene therapy in Parkinson’s disease. Mov Disord 11: 469–488.

    Article  PubMed  CAS  Google Scholar 

  • Frim DM, Uhler TA, Galpern WR, Beal MF, Breakfield XO, Isacson O (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.

    Article  PubMed  CAS  Google Scholar 

  • Gage F (1998) Cell therapy. Nature 392: S18–S24.

    Google Scholar 

  • Gage F, Coates PW, Palmer TD, Kuhn HG, Fisher L, Suhonen JO, Peterson DA, Suhr ST, Ray J (1995) Survival and differentation of adult neuronal progenitor cells transplanted to the adult brain. Proc Natl Acad Sci USA 92: 11879–11883.

    Article  PubMed  CAS  Google Scholar 

  • Geller AI, Freese A (1990) Infection of cultured central nervous system neurons with a defective herpes simplex virus 1 vector results in stable expression of E. coli betagalactosidase. Proc Natl Acad Sci USA 87: 1149–1153.

    Article  PubMed  CAS  Google Scholar 

  • Geller AI, Freese A, During MJ, O’Malley KL (1995) A HSV-1 vector expressing tyrosine hydroxylase causes production and release of L-dopa from cultured rat striatal cells. J Neurochem 64: 487–496.

    Article  PubMed  CAS  Google Scholar 

  • Giulian D, Lachman LB (1985) Interleukin-1 stimulation of astroglial proliferation after brain injury. Science 228: 497–499.

    Article  PubMed  CAS  Google Scholar 

  • Glorioso JC, Goins WF, Meaney CA, Fink DJ, DeLuca NA (1994) Gene transfer to brain using herpes simplex virus vectors. Ann Neurol 35: S28–S34.

    Article  PubMed  CAS  Google Scholar 

  • Graham FL, Smiley J, Russell WC, Nairn R (1977) Characteristics of a human cell line transformed by DNA from human adenovirus 5. J Gen Virol 36: 59–72.

    Article  PubMed  CAS  Google Scholar 

  • Hagg T, Varon S (1993) Ciliary neurotrophic factor prevents degeneration of adult rat substantia nigra dopaminergic neurons in vivo. Proc Natl Acad Sci USA 90: 6315–6319.

    Article  PubMed  CAS  Google Scholar 

  • Heckl-Ostreicher B, Binder R, Kirschfink M (1995) Functional activity of the membranassociated complement inhibitor CD59 in a pig to human in vitro model for hyperacute xenograft rejection. Clin Exp Immunol 102: 589–595.

    Article  PubMed  CAS  Google Scholar 

  • Hefti F, Hartikka J, Schlumpf M (1985) Implantation of PC12 cells into the corpus striatum of rats with lesions of the dopaminergic nigrostriatal neurons. Brain Res 348: 283–288.

    Article  PubMed  CAS  Google Scholar 

  • Horellou P, Guibert B (1989) Retroviral transfer of a human tyrosine hydroxylase cDNA in various cell lines: regulated release of dopamine in mouse anterior pituitary AtT-20 cells. Proc Natl Acad Sci USA 86: 7233–7237.

    Article  PubMed  CAS  Google Scholar 

  • Horellou P, Brundin P, Kalen P, Mallet J, Björklund A (1990a) In vivo release of DOPA and dopamine from genetically engineered cells grafted to the denervated rat striatum. Neuron 5: 393–402.

    Article  PubMed  CAS  Google Scholar 

  • Horellou P, Marlier L, Privat A, Mallet J (1990b) Behavioural effect of engineered cells that synthesize L-DOPA or dopamine after grafting into the rat neostriatum. Eur J Neurosci 2: 116–119.

    Article  PubMed  Google Scholar 

  • Horellou P, Marlier L, Privat A, Mallet J (1990c) Exogenous expression of L-dopa and dopamine in various cell lines following transfer of rat and human tyrosine hydroxylase cDNA: Grafting in an animal model of Parkinson’s disease. Prog Brain Res 82: 23–32.

    Article  PubMed  CAS  Google Scholar 

  • Horellou P, Vigne E, Castel MN, Barneoud P, Colin P, Perricaudet M, Delaere P, Mallet J (1994) Direct intracerebral gene transfer of an adenoviral vector expressing tyrosine hydroxylase in a rat model of Parkinson’s disease. Neuroreport 6: 49–53.

    Article  PubMed  CAS  Google Scholar 

  • Horellou P, Sabate O, Buc-Caron MH, Mallet J (1997) Adenovirus-mediated gene transfer to the central nervous system for Parkinson’s disease. Exp Neurol 144: 131–138.

    Article  PubMed  CAS  Google Scholar 

  • Hulspas R, Tiarks C, Reilly J, Hsieh CC, Recht L, Quesenberry PJ (1997) In vitro cell density-dependent clonal growth of EGF-responsive murine neural progenitor cells under serum-free conditions. Exp Neurol 148:147–156.

    Article  PubMed  CAS  Google Scholar 

  • Hyman C, Hofer M, Barde YA, Juhasz M, Yancopoulos GD, Squinto SP, Lindsay RM (1991) BDNF is an neurotrophic factor for dopaminergic neurons of the substantia nigra. Nature 350: 230–232.

    Article  PubMed  CAS  Google Scholar 

  • Hyman C, Juhasz C, Jackson C, Wright P, Ip NY, Lindsay RM (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.

    PubMed  CAS  Google Scholar 

  • Jiao S, Schultz E, Wolff J (1992a) Intracerebral transplants of primary muscle cells: A potential “platform” for transgene expression in the brain. Brain Res 575:143–147.

    Article  PubMed  CAS  Google Scholar 

  • Jiao S, Wolff J (1992b) Long-term survival of autologous muscle grafts in rat brain. Neurosci Lett 137: 207–210.

    Article  PubMed  CAS  Google Scholar 

  • Jiao S, Gurevich V, Wolff J (1993) Long-term correction of rat model of Parkinson’s disease by gene therapy. Nature 362: 450–453.

    Article  PubMed  CAS  Google Scholar 

  • Kaplitt MG, Leone P, Samulski RJ, Xiao X, Pfaff DW, O’Malley KL, During MJ (1994) Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Gen 8: 148–154.

    Article  CAS  Google Scholar 

  • Karpati G, Lochmüller H, Nalbantoglu J, Durham H (1996) The principles of gene therapy for the nervous system. Trends Neurosci 19: 49–54.

    Article  PubMed  CAS  Google Scholar 

  • Kawaja M, Gage F (1992) Morphological and neurochemical features of cultured primary skin fibroblasts of Fischer 344 rats following striatal implantation. J Comp Neurol 317: 102–116.

    Article  PubMed  CAS  Google Scholar 

  • Kawaja M, Fagan AM, Firestein BL, Gage F (1991) Intracerebral grafting of cultured autologous skin fibroblasts into rat striatum: An assessment of graft size and ultrastructure. J Comp Neurol 307: 695–706.

    Article  PubMed  CAS  Google Scholar 

  • Kelly PH, Seviour PW, Iversen SD (1975) Amphetamine and apomorphine responses in the rat following 6-OHDA lesions of the nucleus accumbens septi and corpus striatum. Brain Res 94: 507–522.

    Article  PubMed  CAS  Google Scholar 

  • Kim VN, Mitrophanous K, Kingsman SM, Kingsman AJ (1998) Minimal requirement for a lentivirus vector based on human immundeficiency virus type 1. J Virol 72: 811–816.

    PubMed  CAS  Google Scholar 

  • Kordower JH, Goetz CG, Freeman TB, Olanow CW (1997) Dopaminergic transplants in patients with Parkinson’s disease: Neuroanatomical correlates of clinical recovery. Exp Neurol 144: 41–46.

    Article  PubMed  CAS  Google Scholar 

  • Lange K, Youdim M, Riederer P (1992) Neurotoxicity and neuroprotection in Parkinson’s disease. J Neural Transm 38: 27–44.

    CAS  Google Scholar 

  • Le Gal La Salle G, Robert JJ, Berrard S, Ridoux V, Startford-Perricaudet LD, Perricaudet M, Mallet J (1993) An adenovirus vector for gene transfer into neurons and glia in the brain. Science 259: 988–990.

    Article  PubMed  CAS  Google Scholar 

  • Levine RA, Miller LP, Lovenberg W (1981) Tetrahydrobiopterin in striatum: localization in dopamine nerve terminals and role in catecholamine synthesis. Science 213: 349–350.

    Article  Google Scholar 

  • Levivier M, Przedborski S, Bencsics C, Kang UJ (1995) Intrastriatal implantation of fibroblasts genetically engineered to produce brain-derived neurotrophic factor prevents degeneration of dopaminergic neurons in rat model of Parkinson’s disease. J Neurosci 15: 7810–7820.

    PubMed  CAS  Google Scholar 

  • Lin LF, Doherty DH, Lile JD, Bektesh S, Collins F (1993) GDNF: A glial cell line-derived neurotrophic factor for midbrain dopaminergic neurons. Science 260: 1130–1132.

    Article  PubMed  CAS  Google Scholar 

  • Lundberg C, Horellou P, Mallet J, Björklund A (1996) Generation of dopa-producing astrocytes by retro viral transduction of the human tyrosine hydroxylase gene: in vitro characterization and in vivo effects in the rat Parkinson model. Exp Neurol 139: 39–53.

    Article  PubMed  CAS  Google Scholar 

  • Mayer E, Dunnett SB, Fawcett JW (1993) Mitogenic effect of basic fibroblast growth factor on embryonic ventral mesencephalic dopaminergic neuron precursors. Brain Res Dev Brain Res 72: 253–258.

    Article  PubMed  CAS  Google Scholar 

  • Mehler MF, Rozental R, Dougherty M, Spray DC, Kessler JA (1993) Cytokine regulation of neural differentiation of hippocampal progenitor cells. Science 362: 62–65.

    CAS  Google Scholar 

  • Meignier B, Longnecker R, Mavromara-Nazos P, Sears A, Roizman B (1988) Virulence of and establishment of latency by genetically engineered deletion mutants of herpes simplex virus type 1. Virology 162: 251–254.

    Article  PubMed  CAS  Google Scholar 

  • Mytilineou C, Park TH, Shen J (1992) Epidermal growth factor-induced survival and proliferation of neuronal precursor cells from embryonic rat mesencephalon. Neurosci Lett 135: 62–66.

    Article  PubMed  CAS  Google Scholar 

  • Naldini L, Blomer U, Gage F, Trono D, Verma IM (1996a) Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc Natl Acad Sci USA 93: 11382–11388.

    Article  PubMed  CAS  Google Scholar 

  • Naldini L, Blömer U, Gallay P, Ory D, Mulligan R, Gage F, Verma IM, Trono D (1996b) In vivo gene delivery and stable transduction of nondividing cells by lentiviral vector. Science 272: 263–267.

    Article  PubMed  CAS  Google Scholar 

  • Onifer S, Whittemore SR, Holets VR (1993) Variable morphological differentation of a rapid-derived neuronal cell line following transplantation into the adult rat CNS. Exp Neurol 122:130–142.

    Article  PubMed  CAS  Google Scholar 

  • Partridge TA, Davies KE (1995) Myoblast-based gene therapies. Br Med Bull 51:123–137.

    PubMed  CAS  Google Scholar 

  • Patience C, Takeuchi Y, Weiss RA (1997) Infection of human cells by an endogenous retrovirus of pigs. Nat Med 3: 282–286.

    Article  PubMed  CAS  Google Scholar 

  • Platt JL (1994) A perspective on xenograft rejection and accommodation. Immunol Rev 141: 127–149.

    Article  PubMed  CAS  Google Scholar 

  • Platt JL (1998) New directions for organ transplantation. Nature 392: S11–S17.

    Article  Google Scholar 

  • Ramakrishnan R, Fink DJ, Guihua J, Desai P, Glorioso JC, Levine M (1994) Competitive quantitative Polymerase chain reaction (PCR) analysis of herpes simplex virus typ 1 DNA and LAT RNA in latently infected cells of the rat brain. J Virol 68: 1864–1870.

    PubMed  CAS  Google Scholar 

  • Raymon HK, Thode S, Gage F (1997) Application of ex vivo gene therapy in the treatment of Parkinson’s disease. Exp Neurol 144: 82–91.

    Article  PubMed  CAS  Google Scholar 

  • Reynolds BA, Weiss S (1992) Generation of neurons and astrocytes from isolated cells of the adult mammalian central nervous system. Science 255: 1707–1710.

    Article  PubMed  CAS  Google Scholar 

  • Reynolds BA, Weiss RA (1996) Clonal and population analyses demonstrate that an EGF-responsive mammalian embryonic CNS precursor is a stem cell. Dev Biol 175: 1–13.

    Article  PubMed  CAS  Google Scholar 

  • Richards LJ, Kilpatrick TJ, Bartlett PF (1992) De novo generation of neuronal cells from the adult mouse brain. Proc Natl Acad Sci USA 89: 8591–8595.

    Article  PubMed  CAS  Google Scholar 

  • Rother RP, Fodor WL, Springhorn JP, Birks CW, Setter E, Sandrin MS, Squinto SP, Rollins SA (1995) A novel mechanism of retrovirus inactivation in human serum mediated by anti-alpha galactosyl natural antibody. J Exp Med 182: 1345–1355.

    Article  PubMed  CAS  Google Scholar 

  • Sabate O, Horellou P, Vigne E, Colin P, Perricaudet M, Buc-Caron MH, Mallet J (1995) Transplantation to the rat brain of human neural progenitors that were genetically modified using adenoviruses. Nat Gen 9: 256–260.

    Article  CAS  Google Scholar 

  • Sachs DH, Bach FH (1990) Immunology of xenograft rejection. Hum Immunol 28: 245–251.

    Article  PubMed  CAS  Google Scholar 

  • Samulski JS, Chang LS, Shenk T (1989) Helper-free stocks of adeno-associated viruses: normal integration does not require viral gene expression. J Virol 63: 3822–3828.

    PubMed  CAS  Google Scholar 

  • Sauer H, Oertel WH (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: 401–415.

    Article  PubMed  CAS  Google Scholar 

  • Sautter J, Tseng JL, Braguglia D, Aebischer P, Spenger C, Seiler RW, Widmer HR, Zurn AD (1998) Implants of polymer-encapsulated genetically modified cells releasing glial cell line-derived neurotrophic factor improve survival, growth and function of dopaminergic grafts. Exp Neurol 149: 230–236.

    Article  PubMed  CAS  Google Scholar 

  • Shetty AK, Turner DA (1998) In vitro survival and differentation of neurons derived from epidermal growth factor-responsive postnatal hippocampal stem cells: inducing effects of brain-derived neurotrophic factor. J Neurobiol 35: 395–425.

    Article  PubMed  CAS  Google Scholar 

  • Snyder EY, Deitcher DL, Walsh C, Arnold-Aldea S, Hartwieg EA, Cepko CL (1992) Multipotent neural cell lines can engraft and participate in development of mouse cerebellum. Cell 68: 33–51.

    Article  PubMed  CAS  Google Scholar 

  • Spaete R, Frenkel N (1982) The herpes simplex virus amplicon: A new eucaryotic defective-virus cloning amplifying vector. Cell 30: 295–304.

    Article  PubMed  CAS  Google Scholar 

  • Speck PG, Simmons A (1991) Divergent molecular pathways of productive and latent infection with a virulent strain of herpes simplex virus type 1. J Virol 65: 4004–4005.

    Google Scholar 

  • Steele DJ, Auchincloss HJ (1995) Xenotransplantation. Annu Rev Med 46: 345–360.

    Article  PubMed  CAS  Google Scholar 

  • Svendsen CN, Fawcett JW, Bentlage C, Dunnett SB (1995) Increased survival of rat EGF-generated CNS precursor cells using B27 supplemented medium. Exp Brain Res 102: 407–414.

    Article  PubMed  CAS  Google Scholar 

  • Svendsen CN, Clarke DJ, Rosser AE, Dunnett SB (1996) Survival and differentiation of rat and human epidermal growth factor-responsive precursor cells following grafting into the lesioned adult central nervous system. Exp Neurol 137: 376–388.

    Article  PubMed  CAS  Google Scholar 

  • Svendsen CN, Caldwell MA, Shen J, ter Borg MG, Rosser AE, Tyers P, Karmiol S, Dunnett SB (1997) Long-term survival of human central nervous system progenitor cells transplanted into rat model of Parkinson’s disease. Exp Neurol 148: 135–146.

    Article  PubMed  CAS  Google Scholar 

  • Takayama H, Ray J, Raymon HK, Baird A, Hogg J, Fisher L, Gage F (1995) Basic fibroblast growth factor increases dopaminergic graft survival and function in a rat model of Parkinson’s disease. Nat Med 1: 53–58.

    Article  PubMed  CAS  Google Scholar 

  • Takeuchi Y, Porter CD, Strahan KM, Preece AF, Gustafsson K, Cosset FL, Weiss RA, Collins MKL (1996) Sensitization of cells and retroviruses to human serum by (alphal-3) galactosyltransferase. Nature 379: 85–88.

    Article  PubMed  CAS  Google Scholar 

  • Tearle RG, Tange MJ, Zannettino ZL, Katerelos M, Shinkel TA, Van-Denderen BJ, Lonie AJ, Lyons I, Nottle MB, Cox MB, Becker MB, et al (1996) The alpha-1,3-galactosyltransferase knockout mouse: implications for xenotransplatation. Transplantation 61: 13–19.

    Article  PubMed  CAS  Google Scholar 

  • Wang J, Bankiewics KS, Plunkett RJ, Oldfield EH (1994) Intrastriatal implantation of interleukin-1. J Neurosurg 80: 484–490.

    Article  PubMed  CAS  Google Scholar 

  • Ward T, Pipkin PA, Clarkson NA, Stone DM, Minor PD, Almond JW (1994) Decayaccelerating factor CD55 is identified as the receptor for echovirus 7 using CELICS, a rapid immuno-focal cloning method. EMBO 13: 5070–5074.

    CAS  Google Scholar 

  • Weiss RA (1998) Transgenic pigs and virus adaptation. Nature 391: 327–328.

    Article  PubMed  CAS  Google Scholar 

  • Weiss RA, Reynolds BA, Vescovi AL, Morshead C, Craig CG, van der Kooy D (1996) Is there a neural stem cell in the mammalian forebrain? Trends Neurosci 19: 387–393.

    Article  PubMed  CAS  Google Scholar 

  • Wenning GK, Odin P, Morrish P, Rehncrona S, Widner H, Brundin P, Rothwell JC, Brown R, et al (1997) Short-and long-term survival and function of unilateral intrastriatal dopaminergic grafts in Parkinson’s disease. Ann Neurol 42: 95–107.

    Article  PubMed  CAS  Google Scholar 

  • Werner ER, Werner-Felmayer G, Fuchs D, Hausen A, Reibnegger D, Yim JJ, Pfleiderer W, Wachter H (1990) Tetrahydrobiopterin biosynthetic activities in human macrophages, fibroblasts, THP-1, and T24 cells. J Biol Chem 265: 3189–3192.

    PubMed  CAS  Google Scholar 

  • Wolff J (1996) Retraction of Jiao S, Gurevich V, Wolff JA, in Nature 1993 Apr 1;362 (6419):450-453. Nature 380: 734.

    Article  PubMed  Google Scholar 

  • Wolff J, Fisher L, Xu L, Gage F (1989a) Grafting fibroblasts genetically modified to produce L-dopa in a rat model of Parkinson disease. Proc Natl Acad Sci USA 86: 9011–9014.

    Article  PubMed  CAS  Google Scholar 

  • Wolff J, Fisher L, Xu L, Hyder A, Jinnah H (1989b) Grafting fibroblasts genetically modified to produce L-dopa in a rat model of Parkinson disease. Proc Natl Acad Sci USA 86: 9011–9014.

    Article  PubMed  CAS  Google Scholar 

  • Xiao X, Li J, Samulski JS (1996) Long-term and efficient in vivo gene transfer into muscle tissue of immunocompetent mice with an rAAV vector. J Virol 70: 8098–8108.

    PubMed  CAS  Google Scholar 

  • Xiao X, Li J, McCown TJ, Samulski JS (1997) Gene transfer by adeno-associated virus vectors into the central nervous system. Exp Neurol 144: 113–124.

    Article  PubMed  CAS  Google Scholar 

  • Yoshimoto Y, Lin Q, Collier TJ, Frim DM, Breakfield XO, Bohn MC (1995) Astrocyts retrovirally transduced with BDNF elicit behavioural improvement in a rat model of Parkinson’s disease. Brain Res 691: 25–36.

    Article  PubMed  CAS  Google Scholar 

  • Zawada WM, Cibelli JB, Choi PK, Clarkson ED, Golueke PJ, Witta SE, Bell KP, Kane J, Ponce De Leon FA, Jerry DJ, Robl JM, Freed CR, Stice SL (1998) Somatic cell cloned transgenic bovine neurons for transplantation in parkinsonian rats. Nat Med 4: 569–574.

    Article  PubMed  CAS  Google Scholar 

  • Zhu SM, Kujirai K, Dollison A, Angulo J, Fahn S, Cadet JL (1992) Implantation of genetically modified mesencephalic fetal cells into rat striatum. Brain Res Bull 29:81–93.

    Article  PubMed  CAS  Google Scholar 

  • Zufferey R, Nagy D, Mandel RJ, Naldini L, Trono D (1997) Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat Biotech 15: 871–875.

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Editor information

Editors and Affiliations

Rights and permissions

Reprints and permissions

Copyright information

© 1999 Springer-Verlag/Wien

About this paper

Cite this paper

Bauer, M., Ueffing, M., Meitinger, T., Gasser, T. (1999). Somatic gene therapy in animal models of Parkinson’s disease. In: Poewe, W., Ransmayr, G. (eds) Advances in Research on Neurodegeneration. 6th International Winter Conference on N eurodegeneration, vol 55. Springer, Vienna. https://doi.org/10.1007/978-3-7091-6369-6_12

Download citation

  • DOI: https://doi.org/10.1007/978-3-7091-6369-6_12

  • Publisher Name: Springer, Vienna

  • Print ISBN: 978-3-211-83261-5

  • Online ISBN: 978-3-7091-6369-6

  • eBook Packages: Springer Book Archive

Publish with us

Policies and ethics