메뉴 건너뛰기




Volumn 13, Issue SUPPL. 3, 2007, Pages

Issues regarding gene therapy products for Parkinson's disease: The development of CERE-120 (AAV-NTN) as one reference point

Author keywords

Disease progression; Neuronal restoration; Neuroprotection; Neurotrophic factors; Neurturin

Indexed keywords

CERE 120; GENE PRODUCT; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; NEURTURIN; UNCLASSIFIED DRUG;

EID: 38949196405     PISSN: 13538020     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1353-8020(08)70052-X     Document Type: Article
Times cited : (28)

References (36)
  • 1
    • 0032850453 scopus 로고    scopus 로고
    • Clinicopathological findings following intraventricular glial-derived neurotrophic factor treatment in a patient with Parkinson's disease
    • Kordower J., Palfi S., Chen E., Ma S., Sendera T., Cochran E., et al. Clinicopathological findings following intraventricular glial-derived neurotrophic factor treatment in a patient with Parkinson's disease. Ann Neurol 46 (1999) 419-424
    • (1999) Ann Neurol , vol.46 , pp. 419-424
    • Kordower, J.1    Palfi, S.2    Chen, E.3    Ma, S.4    Sendera, T.5    Cochran, E.6
  • 2
    • 0037435511 scopus 로고    scopus 로고
    • Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD
    • Nutt J., Burchiel K., Comella C., Jankovic J., Lang A., Laws Jr. E., et al. Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD. Neurology 60 (2003) 69-73
    • (2003) Neurology , vol.60 , pp. 69-73
    • Nutt, J.1    Burchiel, K.2    Comella, C.3    Jankovic, J.4    Lang, A.5    Laws Jr., E.6
  • 4
    • 0031149479 scopus 로고    scopus 로고
    • Hyperplastic changes within the leptomeninges of the rat and monkey in response to chronic intracerebroventricular infusion of nerve growth factor
    • Day-Lollini P., Stewart G., Taylor M., Johnson R., and Chellman G. Hyperplastic changes within the leptomeninges of the rat and monkey in response to chronic intracerebroventricular infusion of nerve growth factor. Exp Neurol 145 (1997) 24-37
    • (1997) Exp Neurol , vol.145 , pp. 24-37
    • Day-Lollini, P.1    Stewart, G.2    Taylor, M.3    Johnson, R.4    Chellman, G.5
  • 5
    • 35748939368 scopus 로고    scopus 로고
    • Six-month continuous intraputamenal infusion toxicity study of recombinant methionyl human glial cell line-derived neurotrophic factor (r-metHuGDNF) in rhesus monkeys
    • Hovland Jr. D., Boyd R., Butt M., Engelhardt J., Moxness M., Ma M., et al. Six-month continuous intraputamenal infusion toxicity study of recombinant methionyl human glial cell line-derived neurotrophic factor (r-metHuGDNF) in rhesus monkeys. Toxicol Pathol 35 (2007) 676-692
    • (2007) Toxicol Pathol , vol.35 , pp. 676-692
    • Hovland Jr., D.1    Boyd, R.2    Butt, M.3    Engelhardt, J.4    Moxness, M.5    Ma, M.6
  • 6
    • 33750609664 scopus 로고    scopus 로고
    • Point source concentration of GDNF may explain failure of phase II clinical trial
    • Salvatore M., Ai Y., Fischer B., Zhang A., Grondin R., Zhang Z., et al. Point source concentration of GDNF may explain failure of phase II clinical trial. Exp Neurol 202 (2006) 497-505
    • (2006) Exp Neurol , vol.202 , pp. 497-505
    • Salvatore, M.1    Ai, Y.2    Fischer, B.3    Zhang, A.4    Grondin, R.5    Zhang, Z.6
  • 7
    • 0142089747 scopus 로고    scopus 로고
    • Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
    • Raper S., Chirmule N., Lee F., Wivel N., Bagg A., Gao G., et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Gen Metab 80 (2003) 148-158
    • (2003) Mol Gen Metab , vol.80 , pp. 148-158
    • Raper, S.1    Chirmule, N.2    Lee, F.3    Wivel, N.4    Bagg, A.5    Gao, G.6
  • 8
    • 0042738935 scopus 로고    scopus 로고
    • Occurrence of leukaemia following gene therapy of X-linked SCID
    • Kohn D., Sadelain M., and Glorioso J. Occurrence of leukaemia following gene therapy of X-linked SCID. Nat Rev Cancer 3 (2003) 477-488
    • (2003) Nat Rev Cancer , vol.3 , pp. 477-488
    • Kohn, D.1    Sadelain, M.2    Glorioso, J.3
  • 9
    • 0024722595 scopus 로고
    • Neurotrophic factors: can the degenerating brain be induced to heal itself?
    • Bartus R. Neurotrophic factors: can the degenerating brain be induced to heal itself?. Neurobiol Aging 10 (1989) 513
    • (1989) Neurobiol Aging , vol.10 , pp. 513
    • Bartus, R.1
  • 10
    • 0024394332 scopus 로고
    • Function of neurotrophic factors in the adult and aging brain and their possible use in the treatment of neurodegenerative diseases
    • Hefti F., Hartikka J., and Knusel B. Function of neurotrophic factors in the adult and aging brain and their possible use in the treatment of neurodegenerative diseases. Neurobiol Aging 10 (1989) 515-533
    • (1989) Neurobiol Aging , vol.10 , pp. 515-533
    • Hefti, F.1    Hartikka, J.2    Knusel, B.3
  • 11
    • 0033773237 scopus 로고    scopus 로고
    • Preservation of a functional nigrostriatal dopamine pathway by GDNF in the intrastriatal 6-OHDA lesion model depends on the site of administration of the trophic factor
    • Kirik D., Rosenblad C., and Bjorklund A. Preservation of a functional nigrostriatal dopamine pathway by GDNF in the intrastriatal 6-OHDA lesion model depends on the site of administration of the trophic factor. Eur J Neurosci. 12 (2000) 3871-3882
    • (2000) Eur J Neurosci. , vol.12 , pp. 3871-3882
    • Kirik, D.1    Rosenblad, C.2    Bjorklund, A.3
  • 12
    • 0029614333 scopus 로고
    • Morphological and functional effects of intranigrally administered GDNF in normal rhesus monkeys
    • Gash D., Zhang Z., Cass W., Ovadia A., Simmerman L., Martin D., et al. Morphological and functional effects of intranigrally administered GDNF in normal rhesus monkeys. J Comp Neurol 363 (1995) 345-358
    • (1995) J Comp Neurol , vol.363 , pp. 345-358
    • Gash, D.1    Zhang, Z.2    Cass, W.3    Ovadia, A.4    Simmerman, L.5    Martin, D.6
  • 13
    • 0036751973 scopus 로고    scopus 로고
    • Effects of chronic intraputamenal infusion of glial cell line-derived neurotrophic factor (GDNF) in aged Rhesus monkeys
    • Maswood N., Grondin R., Zhang Z., Stanford J., Surgener S., Gash D., et al. Effects of chronic intraputamenal infusion of glial cell line-derived neurotrophic factor (GDNF) in aged Rhesus monkeys. Neurobiol Aging 23 (2002) 881-889
    • (2002) Neurobiol Aging , vol.23 , pp. 881-889
    • Maswood, N.1    Grondin, R.2    Zhang, Z.3    Stanford, J.4    Surgener, S.5    Gash, D.6
  • 14
    • 0034659835 scopus 로고    scopus 로고
    • Long-term rAAV- mediated gene transfer of GDNF in the rat Parkinson's model: intrastriatal but not intranigral transduction promotes functional regeneration in the lesioned nigrostriatal system
    • Kirik D., Rosenblad C., Bjorklund A., and Mandel R. Long-term rAAV- mediated gene transfer of GDNF in the rat Parkinson's model: intrastriatal but not intranigral transduction promotes functional regeneration in the lesioned nigrostriatal system. J Neurosci 20 (2000) 4686-4700
    • (2000) J Neurosci , vol.20 , pp. 4686-4700
    • Kirik, D.1    Rosenblad, C.2    Bjorklund, A.3    Mandel, R.4
  • 15
    • 0032423612 scopus 로고    scopus 로고
    • Behavioral and cellular protection of rat dopaminergic neurons by an adenoviral vector encoding glial cell line-derived neurotrophic factor
    • Choi-Lundberg D., Lin Q., Schallert T., Crippens D., Davidson B., Chang Y., et al. Behavioral and cellular protection of rat dopaminergic neurons by an adenoviral vector encoding glial cell line-derived neurotrophic factor. Exp Neurol 154 (1998) 261-275
    • (1998) Exp Neurol , vol.154 , pp. 261-275
    • Choi-Lundberg, D.1    Lin, Q.2    Schallert, T.3    Crippens, D.4    Davidson, B.5    Chang, Y.6
  • 16
    • 0034721690 scopus 로고    scopus 로고
    • Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease
    • Kordower J., Emborg M., Bloch J., Ma S., Chu Y., Leventhal L., et al. Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease. Science 290 (2000) 767-773
    • (2000) Science , vol.290 , pp. 767-773
    • Kordower, J.1    Emborg, M.2    Bloch, J.3    Ma, S.4    Chu, Y.5    Leventhal, L.6
  • 17
    • 33644833272 scopus 로고    scopus 로고
    • Randomized controlled trial of intraputamenal glial cell line-derived neurotrophic factor infusion in Parkinson disease
    • Lang A., Gill S., Patel N., Lozano A., Nutt J., Penn R., et al. Randomized controlled trial of intraputamenal glial cell line-derived neurotrophic factor infusion in Parkinson disease. Ann Neurol 59 (2006) 459-466
    • (2006) Ann Neurol , vol.59 , pp. 459-466
    • Lang, A.1    Gill, S.2    Patel, N.3    Lozano, A.4    Nutt, J.5    Penn, R.6
  • 18
    • 0038249170 scopus 로고    scopus 로고
    • Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease
    • Gill S., Patel N., Hotton G., O'Sullivan K., McCarter R., Bunnage M., et al. Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease. Nat Med 9 (2003) 589-595
    • (2003) Nat Med , vol.9 , pp. 589-595
    • Gill, S.1    Patel, N.2    Hotton, G.3    O'Sullivan, K.4    McCarter, R.5    Bunnage, M.6
  • 19
    • 14844311296 scopus 로고    scopus 로고
    • Improvement of bilateral motor functions in patients with Parkinson disease through the unilateral intraputaminal infusion of glial cell line- derived neurotrophic factor
    • Slevin J., Gerhardt G., Smith C., Gash D., Kryscio R., and Young B. Improvement of bilateral motor functions in patients with Parkinson disease through the unilateral intraputaminal infusion of glial cell line- derived neurotrophic factor. J Neurosurgery 102 (2005) 216-222
    • (2005) J Neurosurgery , vol.102 , pp. 216-222
    • Slevin, J.1    Gerhardt, G.2    Smith, C.3    Gash, D.4    Kryscio, R.5    Young, B.6
  • 21
    • 34347403884 scopus 로고    scopus 로고
    • Convective delivery of glial cell line-derived neurotrophic factor in the human putamen
    • Morrison P., Lonser R., and Oldfield E. Convective delivery of glial cell line-derived neurotrophic factor in the human putamen. J Neurosurg 107 (2007) 74-83
    • (2007) J Neurosurg , vol.107 , pp. 74-83
    • Morrison, P.1    Lonser, R.2    Oldfield, E.3
  • 22
    • 33646447596 scopus 로고
    • Drug delivery to the brain: The problem lurking behind the problem
    • Bartus R. Drug delivery to the brain: The problem lurking behind the problem. Neurobiol Aging 10 (1989) 621
    • (1989) Neurobiol Aging , vol.10 , pp. 621
    • Bartus, R.1
  • 23
    • 0032918148 scopus 로고    scopus 로고
    • The blood-brain barrier as a target for pharmacological modulation
    • Bartus R. The blood-brain barrier as a target for pharmacological modulation. Curr Opin Drug Disc Dev 2 (1999) 152-157
    • (1999) Curr Opin Drug Disc Dev , vol.2 , pp. 152-157
    • Bartus, R.1
  • 24
    • 20844452391 scopus 로고    scopus 로고
    • Neurotrophin receptor-based strategies for Alzheimer's disease
    • Longo F., and Massa S. Neurotrophin receptor-based strategies for Alzheimer's disease. Curr Alzheimer Res 2 (2005) 167-169
    • (2005) Curr Alzheimer Res , vol.2 , pp. 167-169
    • Longo, F.1    Massa, S.2
  • 25
    • 33846011117 scopus 로고    scopus 로고
    • Striatal delivery of neurturin by CERE-120, an AAV2 vector for the treatment of dopaminergic neuron degeneration in Parkinson's disease
    • Gasmi M., Herzog C., Brandon E., Cunningham J., Ramirez G., Ketchum E., et al. Striatal delivery of neurturin by CERE-120, an AAV2 vector for the treatment of dopaminergic neuron degeneration in Parkinson's disease. Mol Ther 15 (2007) 62-68
    • (2007) Mol Ther , vol.15 , pp. 62-68
    • Gasmi, M.1    Herzog, C.2    Brandon, E.3    Cunningham, J.4    Ramirez, G.5    Ketchum, E.6
  • 26
    • 34250683023 scopus 로고    scopus 로고
    • Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial
    • Kaplitt M., Feigin A., Tang C., Fitzsimons H., Mattis P., Lawlor P., et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 369 (2007) 2097-2105
    • (2007) Lancet , vol.369 , pp. 2097-2105
    • Kaplitt, M.1    Feigin, A.2    Tang, C.3    Fitzsimons, H.4    Mattis, P.5    Lawlor, P.6
  • 27
    • 38949151767 scopus 로고    scopus 로고
    • Arvanitakis Z, Tuszynski MH, Potkin S, Bartus RT, Bennett D. A Phase I Clinical trial of CERE-110 (AAV-NGF) gene delivery in Alzheimer's disease. American Academy of Neurology Annual Meeting, 2007, Boston, MA.
    • Arvanitakis Z, Tuszynski MH, Potkin S, Bartus RT, Bennett D. A Phase I Clinical trial of CERE-110 (AAV-NGF) gene delivery in Alzheimer's disease. American Academy of Neurology Annual Meeting, 2007, Boston, MA.
  • 28
    • 38949188117 scopus 로고    scopus 로고
    • Herzog CD, Brown L, Gammon D, Kruegel B, Lin R, Wilson A, et al. Expression, bioactivity, and safety 1 year following AAV2- mediated delivery of neurturin to the monkey nigrostriatal system support CERE-120 for Parkinson's disease. J Neurosurg, in press.
    • Herzog CD, Brown L, Gammon D, Kruegel B, Lin R, Wilson A, et al. Expression, bioactivity, and safety 1 year following AAV2- mediated delivery of neurturin to the monkey nigrostriatal system support CERE-120 for Parkinson's disease. J Neurosurg, in press.
  • 29
    • 38949115094 scopus 로고    scopus 로고
    • Herzog CD, Dass B, Gasmi M, Bakay RA, Stansell 3rd J, Tuszynski MH, et al. Transgene expression, bioactivity, and safety of CERE-120 (AAV2-Neurturin) following delivery to the monkey striatum. Mol Ther, in press.
    • Herzog CD, Dass B, Gasmi M, Bakay RA, Stansell 3rd J, Tuszynski MH, et al. Transgene expression, bioactivity, and safety of CERE-120 (AAV2-Neurturin) following delivery to the monkey striatum. Mol Ther, in press.
  • 30
    • 34250336916 scopus 로고    scopus 로고
    • AAV2-mediated delivery of human neurturin to the rat nigrostriatal system: Long-term efficacy and tolerability of CERE- 120 for Parkinson's disease
    • Gasmi M., Brandon E., Herzog C., Wilson A., Bishop K., Hofer E., et al. AAV2-mediated delivery of human neurturin to the rat nigrostriatal system: Long-term efficacy and tolerability of CERE- 120 for Parkinson's disease. Neurobiol Dis 27 (2007) 67-76
    • (2007) Neurobiol Dis , vol.27 , pp. 67-76
    • Gasmi, M.1    Brandon, E.2    Herzog, C.3    Wilson, A.4    Bishop, K.5    Hofer, E.6
  • 31
    • 34447572589 scopus 로고    scopus 로고
    • Striatal delivery of CERE-120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys
    • Herzog C., Dass B., Holden J., Stansell III J., Gasmi M., Tuszynski M., et al. Striatal delivery of CERE-120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys. Mov Disord 22 (2007) 1124-1132
    • (2007) Mov Disord , vol.22 , pp. 1124-1132
    • Herzog, C.1    Dass, B.2    Holden, J.3    Stansell III, J.4    Gasmi, M.5    Tuszynski, M.6
  • 32
    • 38949113901 scopus 로고    scopus 로고
    • Wilson A, Hofer EK, Goulart A, Gasmi M, Bishop KM, Herzog CD, et al. Bioactivity and safety of intrastriatal CERE-120 (AAV2-NTN) in aged Fischer 344 rats. Neurobiol Aging, in press.
    • Wilson A, Hofer EK, Goulart A, Gasmi M, Bishop KM, Herzog CD, et al. Bioactivity and safety of intrastriatal CERE-120 (AAV2-NTN) in aged Fischer 344 rats. Neurobiol Aging, in press.
  • 33
    • 33845993229 scopus 로고    scopus 로고
    • Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys
    • Kordower J., Herzog C., Dass B., Bakay R., Stansell III J., Gasmi M., et al. Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys. Ann Neurol 60 (2006) 706-715
    • (2006) Ann Neurol , vol.60 , pp. 706-715
    • Kordower, J.1    Herzog, C.2    Dass, B.3    Bakay, R.4    Stansell III, J.5    Gasmi, M.6
  • 34
    • 38949150956 scopus 로고    scopus 로고
    • st Annual Meeting, 2006, Chicago, IL.
    • st Annual Meeting, 2006, Chicago, IL.
  • 35
    • 38949168312 scopus 로고    scopus 로고
    • th Annual Meeting, 2007, Washington, DC.
    • th Annual Meeting, 2007, Washington, DC.


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.