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Volumn 23, Issue 5, 2015, Pages 896-906

Controlled striatal DOPA production from a gene delivery system in a rodent model of parkinson's disease

Author keywords

[No Author keywords available]

Indexed keywords

3,4 DIHYDROXYPHENYLACETIC ACID; DIHYDROFOLATE REDUCTASE; DOPA; GUANOSINE TRIPHOSPHATE CYCLOHYDROLASE I; HOMOVANILLIC ACID; TRIMETHOPRIM; BIOLOGICAL MARKER; DOPAMINE; TYROSINE 3 MONOOXYGENASE;

EID: 84929049610     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.8     Document Type: Article
Times cited : (19)

References (43)
  • 1
    • 84897076939 scopus 로고    scopus 로고
    • Long-term safety and tolerability of prosavin a lentiviral vector-based gene therapy for parkinson's disease: A dose escalation open-label phase ' trial
    • Palfi S, Gurruchaga JM, Ralph GS, Lepetit H, Lavisse S, Buttery PC, et al. (2014). Long-term safety and tolerability of ProSavin a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation open-label phase ' trial. Lancet 383: 1138-1146
    • (2014) Lancet , vol.383 , pp. 1138-1146
    • Palfi, S.1    Gurruchaga, J.M.2    Ralph, G.S.3    Lepetit, H.4    Lavisse, S.5    Buttery, P.C.6
  • 2
    • 84879086501 scopus 로고    scopus 로고
    • Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients
    • Bartus RT, Baumann TL, Siffert J, Herzog CD, Alterman R, Boulis N, et al. (2013). Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients. Neurology 80: 1698-1701
    • (2013) Neurology , vol.80 , pp. 1698-1701
    • Bartus, R.T.1    Baumann, T.L.2    Siffert, J.3    Herzog, C.D.4    Alterman, R.5    Boulis, N.6
  • 3
    • 79952740079 scopus 로고    scopus 로고
    • AAV2-GAD gene therapy for advanced Parkinson's disease: A double-blind sham-surgery controlled randomised trial
    • LeWitt PA, Rezai AR, Leehey MA, Ojemann SG, Flaherty AW, Eskandar EN, et al. (2011). AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind sham-surgery controlled randomised trial. Lancet Neurol 10: 309-319
    • (2011) Lancet Neurol , vol.10 , pp. 309-319
    • Lewitt, P.A.1    Rezai, A.R.2    Leehey, M.A.3    Ojemann, S.G.4    Flaherty, A.W.5    Eskandar, E.N.6
  • 4
    • 78349247631 scopus 로고    scopus 로고
    • Gene delivery of AAV2-neurturin for Parkinson's disease: A double-blind randomised controlled trial
    • Marks WJ Jr, Bartus RT, Siffert J, Davis CS, Lozano A, Boulis N, et al. (2010). Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind randomised controlled trial. Lancet Neurol 9: 1164-1172
    • (2010) Lancet Neurol , vol.9 , pp. 1164-1172
    • Marks, W.J.1    Bartus, R.T.2    Siffert, J.3    Davis, C.S.4    Lozano, A.5    Boulis, N.6
  • 5
    • 77956262279 scopus 로고    scopus 로고
    • A phase i study of aromatic l-amino acid decarboxylase gene therapy for parkinson's disease
    • Muramatsu S-I, Fujimoto K-I, Kato S, Mizukami H, Asari S, Ikeguchi K, et al. (2010). A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease. Mol Ther 18: 1731-1735
    • (2010) Mol Ther , vol.18 , pp. 1731-1735
    • Muramatsu, S.-I.1    Fujimoto, K.-I.2    Kato, S.3    Mizukami, H.4    Asari, S.5    Ikeguchi, K.6
  • 7
    • 0024802672 scopus 로고
    • Experience with continuous enteral levodopa infusions in the treatment of 9 patients with advanced Parkinson's disease
    • discussion 72
    • Sage JI, Trooskin S, Sonsalla PK and Heikkila RE. (1989). Experience with continuous enteral levodopa infusions in the treatment of 9 patients with advanced Parkinson's disease. Neurology 39(11 Suppl 2): 60-3; discussion 72
    • (1989) Neurology , vol.39 , Issue.11 SUPPL2 , pp. 60-63
    • Sage, J.I.1    Trooskin, S.2    Sonsalla, P.K.3    Heikkila, R.E.4
  • 8
    • 0023937322 scopus 로고
    • Long-term duodenal infusion of levodopa for motor fluctuations in parkinsonism
    • Sage JI, Trooskin S, Sonsalla PK, Heikkila R and Duvoisin RC. (1988). Long-term duodenal infusion of levodopa for motor fluctuations in parkinsonism. Ann Neurol 24: 87-89
    • (1988) Ann Neurol , vol.24 , pp. 87-89
    • Sage, J.I.1    Trooskin, S.2    Sonsalla, P.K.3    Heikkila, R.4    Duvoisin, R.C.5
  • 9
    • 0023627787 scopus 로고
    • Motor fluctuations in Parkinson's disease: Pathogenetic and therapeutic studies
    • Mouradian MM, Juncos JL, Fabbrini G and Chase TN. (1987). Motor fluctuations in Parkinson's disease: pathogenetic and therapeutic studies. Ann Neurol 22: 475-479
    • (1987) Ann Neurol , vol.22 , pp. 475-479
    • Mouradian, M.M.1    Juncos, J.L.2    Fabbrini, G.3    Chase, T.N.4
  • 10
    • 0016587362 scopus 로고
    • On-off response clinical and biochemical correlations during oral and intravenous levodopa administration in parkinsonian patients
    • Shoulson I, Glaubiger GA and Chase TN. (1975). On-off response. Clinical and biochemical correlations during oral and intravenous levodopa administration in parkinsonian patients. Neurology 25: 1144-1148
    • (1975) Neurology , vol.25 , pp. 1144-1148
    • Shoulson, I.1    Glaubiger, G.A.2    Chase, T.N.3
  • 11
    • 84865551695 scopus 로고    scopus 로고
    • Key factors determining the efficacy of gene therapy for continuous dopa delivery in the parkinsonian brain
    • Cederfjäll E, Sahin G and Kirik D. (2012). Key factors determining the efficacy of gene therapy for continuous DOPA delivery in the Parkinsonian brain. Neurobiol Dis 48: 222-227
    • (2012) Neurobiol Dis , vol.48 , pp. 222-227
    • Cederfjäll, E.1    Sahin, G.2    Kirik, D.3
  • 12
    • 15044365742 scopus 로고    scopus 로고
    • Reversal of dyskinesias in an animal model of parkinson's disease by continuous l-dopa delivery using raav vectors
    • Carlsson T, Winkler C, Burger C, Muzyczka N, Mandel RJ, Cenci A, et al. (2005). Reversal of dyskinesias in an animal model of Parkinson's disease by continuous L-DOPA delivery using rAAV vectors. Brain 128(Pt 3): 559-569
    • (2005) Brain , vol.128 , Issue.PT3 , pp. 559-569
    • Carlsson, T.1    Winkler, C.2    Burger, C.3    Muzyczka, N.4    Mandel, R.J.5    Cenci, A.6
  • 13
    • 0037007106 scopus 로고    scopus 로고
    • Reversal of motor impairments in parkinsonian rats by continuous intrastriatal delivery of L-dopa using rAAV-mediated gene transfer
    • Kirik D, Georgievska B, Burger C, Winkler C, Muzyczka N, Mandel RJ, et al. (2002). Reversal of motor impairments in parkinsonian rats by continuous intrastriatal delivery of L-dopa using rAAV-mediated gene transfer. Proc Natl Acad Sci USA 99: 4708-4713
    • (2002) Proc Natl Acad Sci USA , vol.99 , pp. 4708-4713
    • Kirik, D.1    Georgievska, B.2    Burger, C.3    Winkler, C.4    Muzyczka, N.5    Mandel, R.J.6
  • 14
    • 0032101756 scopus 로고    scopus 로고
    • Characterization of intrastriatal recombinant adeno-associated virus-mediated gene transfer of human tyrosine hydroxylase and human gtp-cyclohydrolase i in a rat model of parkinson's disease
    • Mandel RJ, Rendahl KG, Spratt SK, Snyder RO, Cohen LK and Leff SE. (1998). Characterization of intrastriatal recombinant adeno-associated virus-mediated gene transfer of human tyrosine hydroxylase and human GTP-cyclohydrolase I in a rat model of Parkinson's disease. J Neurosci 18: 4271-4284
    • (1998) J Neurosci , vol.18 , pp. 4271-4284
    • Mandel, R.J.1    Rendahl, K.G.2    Spratt, S.K.3    Snyder, R.O.4    Cohen, L.K.5    Leff, S.E.6
  • 15
    • 0026483508 scopus 로고
    • Dopa-decarboxylation in the striata of rats with unilateral substantia nigra lesions
    • Kang UJ, Park DH, Wessel T, Baker H and Joh TH. (1992). Dopa-decarboxylation in the striata of rats with unilateral substantia nigra lesions. Neurosci Lett 147: 53-57
    • (1992) Neurosci Lett , vol.147 , pp. 53-57
    • Kang, U.J.1    Park, D.H.2    Wessel, T.3    Baker, H.4    Joh, T.H.5
  • 16
    • 0015922157 scopus 로고
    • Immunohistochemical localization of aromatic l-amino acid decarboxylase (dopa decarboxylase) in central dopamine and 5-hydroxytryptamine nerve cell bodies of the rat
    • Hökfelt T, Fuxe K and Goldstein M. (1973). Immunohistochemical localization of aromatic L-amino acid decarboxylase (DOPA decarboxylase) in central dopamine and 5-hydroxytryptamine nerve cell bodies of the rat. Brain Res 53: 175-180
    • (1973) Brain Res , vol.53 , pp. 175-180
    • Hökfelt, T.1    Fuxe, K.2    Goldstein, M.3
  • 17
    • 77249179185 scopus 로고    scopus 로고
    • Optimized adeno-associated viral vector-mediated striatal DOPA delivery restores sensorimotor function and prevents dyskinesias in a model of advanced Parkinson's disease
    • Björklund T, Carlsson T, Cederfjäll EA, Carta M and Kirik D. (2010). Optimized adeno-associated viral vector-mediated striatal DOPA delivery restores sensorimotor function and prevents dyskinesias in a model of advanced Parkinson's disease. Brain 133(Pt 2): 496-511
    • (2010) Brain , vol.133 , Issue.PT2 , pp. 496-511
    • Björklund, T.1    Carlsson, T.2    Cederfjäll, E.A.3    Carta, M.4    Kirik, D.5
  • 18
    • 84863494448 scopus 로고    scopus 로고
    • Design of a single aav vector for coexpression of th and gch1 to establish continuous dopa synthesis in a rat model of parkinson's disease
    • Cederfjäll E, Sahin G, Kirik D and Björklund T. (2012). Design of a single AAV vector for coexpression of TH and GCH1 to establish continuous DOPA synthesis in a rat model of Parkinson's disease. Mol Ther 20: 1315-1326
    • (2012) Mol Ther , vol.20 , pp. 1315-1326
    • Cederfjäll, E.1    Sahin, G.2    Kirik, D.3    Björklund, T.4
  • 19
    • 33748195107 scopus 로고    scopus 로고
    • A rapid reversible and tunable method to regulate protein function in living cells using synthetic small molecules
    • Banaszynski LA, Chen LC, Maynard-Smith LA, Ooi AG and Wandless TJ. (2006). A rapid reversible and tunable method to regulate protein function in living cells using synthetic small molecules. Cell 126: 995-1004
    • (2006) Cell , vol.126 , pp. 995-1004
    • Banaszynski, L.A.1    Chen, L.C.2    Maynard-Smith, L.A.3    Ooi, A.G.4    Wandless, T.J.5
  • 20
    • 77956972260 scopus 로고    scopus 로고
    • A general chemical method to regulate protein stability in the mammalian central nervous system
    • Iwamoto M, Björklund T, Lundberg C, Kirik D and Wandless TJ. (2010). A general chemical method to regulate protein stability in the mammalian central nervous system. Chem Biol 17: 981-988
    • (2010) Chem Biol , vol.17 , pp. 981-988
    • Iwamoto, M.1    Björklund, T.2    Lundberg, C.3    Kirik, D.4    Wandless, T.J.5
  • 21
    • 84866995180 scopus 로고    scopus 로고
    • Destabilizing domains mediate reversible transgene expression in the brain
    • Tai K, Quintino L, Isaksson C, Gussing F and Lundberg C. (2012). Destabilizing domains mediate reversible transgene expression in the brain. PLoS One 7: e46269
    • (2012) Plos One , vol.7 , pp. e46269
    • Tai, K.1    Quintino, L.2    Isaksson, C.3    Gussing, F.4    Lundberg, C.5
  • 22
    • 84880302312 scopus 로고    scopus 로고
    • Continuous dopa synthesis from a single aav: Dosing and efficacy in models of parkinson's disease
    • Cederfjäll E, Nilsson N, Sahin G, Chu Y, Nikitidou E, Björklund T, et al. (2013). Continuous DOPA synthesis from a single AAV: dosing and efficacy in models of Parkinson's disease. Sci Rep 3: 2157
    • (2013) Sci Rep , vol.3 , pp. 2157
    • Cederfjäll, E.1    Nilsson, N.2    Sahin, G.3    Chu, Y.4    Nikitidou, E.5    Björklund, T.6
  • 23
    • 84865567760 scopus 로고    scopus 로고
    • Regulated protein expression for in vivo gene therapy for neurological disorders: Progress strategies and issues
    • Manfredsson FP, Bloom DC and Mandel RJ. (2012). Regulated protein expression for in vivo gene therapy for neurological disorders: progress strategies and issues. Neurobiol Dis 48: 212-221
    • (2012) Neurobiol Dis , vol.48 , pp. 212-221
    • Manfredsson, F.P.1    Bloom, D.C.2    Mandel, R.J.3
  • 24
    • 0026720075 scopus 로고
    • Tight control of gene expression in mammalian cells by tetracycline-responsive promoters
    • Gossen M and Bujard H. (1992). Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc Natl Acad Sci USA 89: 5547-5551
    • (1992) Proc Natl Acad Sci USA , vol.89 , pp. 5547-5551
    • Gossen, M.1    Bujard, H.2
  • 25
    • 0033740246 scopus 로고    scopus 로고
    • Tetracycline-regulated gene expression in the brain
    • Mansuy IM and Bujard H. (2000). Tetracycline-regulated gene expression in the brain. Curr Opin Neurobiol 10: 593-596
    • (2000) Curr Opin Neurobiol , vol.10 , pp. 593-596
    • Mansuy, I.M.1    Bujard, H.2
  • 27
    • 0032716172 scopus 로고    scopus 로고
    • Long-term doxycycline-controlled expression of human tyrosine hydroxylase after direct adenovirus-mediated gene transfer to a rat model of Parkinson's disease
    • Corti O, Sánchez-Capelo A, Colin P, Hanoun N, Hamon M and Mallet J. (1999). Long-term doxycycline-controlled expression of human tyrosine hydroxylase after direct adenovirus-mediated gene transfer to a rat model of Parkinson's disease. Proc Natl Acad Sci USA 96: 12120-12125
    • (1999) Proc Natl Acad Sci USA , vol.96 , pp. 12120-12125
    • Corti, O.1    Sánchez-Capelo, A.2    Colin, P.3    Hanoun, N.4    Hamon, M.5    Mallet, J.6
  • 28
    • 5644254312 scopus 로고    scopus 로고
    • Regulated delivery of glial cell line-derived neurotrophic factor into rat striatum using a tetracycline-dependent lentiviral vector
    • Georgievska B, Jakobsson J, Persson E, Ericson C, Kirik D and Lundberg C. (2004). Regulated delivery of glial cell line-derived neurotrophic factor into rat striatum using a tetracycline-dependent lentiviral vector. Hum Gene Ther 15: 934-944
    • (2004) Hum Gene Ther , vol.15 , pp. 934-944
    • Georgievska, B.1    Jakobsson, J.2    Persson, E.3    Ericson, C.4    Kirik, D.5    Lundberg, C.6
  • 29
  • 30
    • 77952425513 scopus 로고    scopus 로고
    • Lack of humoral immune response to the tetracycline (tet) activator in rats injected intracranially with tet-off raav vectors
    • Han Y, Chang QA, Virag T, West NC, George D, Castro MG, et al. (2010). Lack of humoral immune response to the tetracycline (Tet) activator in rats injected intracranially with Tet-off rAAV vectors. Gene Ther 17: 616-625
    • (2010) Gene Ther , vol.17 , pp. 616-625
    • Han, Y.1    Chang, Q.A.2    Virag, T.3    West, N.C.4    George, D.5    Castro, M.G.6
  • 31
    • 33747627183 scopus 로고    scopus 로고
    • Trends in herpes simplex virus type 1 and type 2 seroprevalence in the united states
    • Xu, F, Sternberg MR, Kottiri BJ, McQuillan GM, Lee FK, Nahmias AJ, et al. (2006). Trends in herpes simplex virus type 1 and type 2 seroprevalence in the United States. JAMA 296: 964-973
    • (2006) Jama , vol.296 , pp. 964-973
    • Xu, F.1    Sternberg, M.R.2    Kottiri, B.J.3    McQuillan, G.M.4    Lee, F.K.5    Nahmias, A.J.6
  • 32
    • 84877017257 scopus 로고    scopus 로고
    • Pharmacokinetics of antibacterial agents in the CSF of children and adolescents
    • Sullins AK and Abdel-Rahman SM. (2013). Pharmacokinetics of antibacterial agents in the CSF of children and adolescents. Paediatr Drugs 15: 93-117
    • (2013) Paediatr Drugs , vol.15 , pp. 93-117
    • Sullins, A.K.1    Abdel-Rahman, S.M.2
  • 33
    • 37249038595 scopus 로고    scopus 로고
    • A comparative study examining the cytotoxicity of inducible gene expression system ligands in different cell types
    • Xie J, Nair A and Hermiston TW. (2008). A comparative study examining the cytotoxicity of inducible gene expression system ligands in different cell types. Toxicol In Vitro 22: 261-266
    • (2008) Toxicol in Vitro , vol.22 , pp. 261-266
    • Xie, J.1    Nair, A.2    Hermiston, T.W.3
  • 35
    • 0025129116 scopus 로고
    • Modification of central dopaminergic mechanisms by continuous levodopa therapy for advanced Parkinson's disease
    • Mouradian MM, Heuser IJ, Baronti F and Chase TN. (1990). Modification of central dopaminergic mechanisms by continuous levodopa therapy for advanced Parkinson's disease. Ann Neurol 27: 18-23
    • (1990) Ann Neurol , vol.27 , pp. 18-23
    • Mouradian, M.M.1    Heuser, I.J.2    Baronti, F.3    Chase, T.N.4
  • 37
    • 0033022953 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield
    • Zolotukhin S, Byrne BJ, Mason E, Zolotukhin I, Potter M, Chesnut K, et al. (1999). Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther 6: 973-985
    • (1999) Gene Ther , vol.6 , pp. 973-985
    • Zolotukhin, S.1    Byrne, B.J.2    Mason, E.3    Zolotukhin, I.4    Potter, M.5    Chesnut, K.6
  • 38
    • 84871997054 scopus 로고    scopus 로고
    • Universal real-time pcr for the detection and quantification of adenoassociated virus serotype 2-derived inverted terminal repeat sequences
    • Aurnhammer C, Haase M, Muether N, Hausl M, Rauschhuber C, Huber I, et al. (2012). Universal real-time PCR for the detection and quantification of adenoassociated virus serotype 2-derived inverted terminal repeat sequences. Hum Gene Ther Methods 23: 18-28
    • (2012) Hum Gene Ther Methods , vol.23 , pp. 18-28
    • Aurnhammer, C.1    Haase, M.2    Muether, N.3    Hausl, M.4    Rauschhuber, C.5    Huber, I.6
  • 39
    • 28144440866 scopus 로고    scopus 로고
    • The corridor task: A simple test of lateralised response selection sensitive to unilateral dopamine deafferentation and graft-derived dopamine replacement in the striatum
    • Dowd E, Monville C, Torres EM and Dunnett SB. (2005). The Corridor Task: a simple test of lateralised response selection sensitive to unilateral dopamine deafferentation and graft-derived dopamine replacement in the striatum. Brain Res Bull 68: 24-30
    • (2005) Brain Res Bull , vol.68 , pp. 24-30
    • Dowd, E.1    Monville, C.2    Torres, E.M.3    Dunnett, S.B.4
  • 40
    • 0018421663 scopus 로고
    • Excessive bracing reactions and their control by atropine and l-dopa in an animal analog of parkinsonism
    • Schallert T, De Ryck M, Whishaw IQ, Ramirez VD and Teitelbaum P. (1979). Excessive bracing reactions and their control by atropine and L-DOPA in an animal analog of Parkinsonism. Exp Neurol 64: 33-43
    • (1979) Exp Neurol , vol.64 , pp. 33-43
    • Schallert, T.1    De Ryck, M.2    Whishaw, I.Q.3    Ramirez, V.D.4    Teitelbaum, P.5
  • 41
    • 0029064091 scopus 로고
    • Forelimb akinesia in the rat Parkinson model: Differential effects of dopamine agonists and nigral transplants as assessed by a new stepping test
    • Olsson M, Nikkhah G, Bentlage C and Björklund A. (1995). Forelimb akinesia in the rat Parkinson model: differential effects of dopamine agonists and nigral transplants as assessed by a new stepping test. J Neurosci 15(5 Pt 2): 3863-3875
    • (1995) J Neurosci , vol.15 , Issue.5 PT2 , pp. 3863-3875
    • Olsson, M.1    Nikkhah, G.2    Bentlage, C.3    Björklund, A.4
  • 42
    • 84985280160 scopus 로고
    • The efficiency of systematic sampling in stereology and its prediction
    • Gundersen HJ and Jensen EB. (1987). The efficiency of systematic sampling in stereology and its prediction. J Microsc 147(Pt 3): 229-263
    • (1987) J Microsc , vol.147 , Issue.PT3 , pp. 229-263
    • Gundersen, H.J.1    Jensen, E.B.2
  • 43
    • 0033083082 scopus 로고    scopus 로고
    • Stereological methods for estimating the total number of neurons and synapses: Issues of precision and bias
    • West, MJ. (1999). Stereological methods for estimating the total number of neurons and synapses: issues of precision and bias. Trends Neurosci 22: 51-61
    • (1999) Trends Neurosci , vol.22 , pp. 51-61
    • West, M.J.1


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