-
1
-
-
79959345818
-
New animal models of Parkinson's disease
-
Dehay B, Bezard E (2011) New animal models of Parkinson's disease. Mov Disord 26(7):1198-1205
-
(2011)
Mov Disord
, vol.26
, Issue.7
, pp. 1198-1205
-
-
Dehay, B.1
Bezard, E.2
-
2
-
-
34249941829
-
Nerve growth factor gene therapy in Alzheimer disease
-
Tuszynski MH (2007) Nerve growth factor gene therapy in Alzheimer disease. Alzheimer Dis Assoc Disord 21(2):179-189
-
(2007)
Alzheimer Dis Assoc Disord
, vol.21
, Issue.2
, pp. 179-189
-
-
Tuszynski, M.H.1
-
3
-
-
84868611681
-
PSD-95 expression controls L-DOPA dyskinesia through dopamine D1 receptor trafficking
-
Porras G et al (2012) PSD-95 expression controls L-DOPA dyskinesia through dopamine D1 receptor trafficking. J Clin Invest 122(11): 3977-3989
-
(2012)
J Clin Invest
, vol.122
, Issue.11
, pp. 3977-3989
-
-
Porras, G.1
-
4
-
-
77952964676
-
Lentiviral overexpression of GRK6 alleviates L-dopa-induced dyskinesia in experimental Parkinson's disease
-
Ahmed MR et al (2010) Lentiviral overexpression of GRK6 alleviates L-dopa-induced dyskinesia in experimental Parkinson's disease. Sci Transl Med 2(28):28ra28
-
(2010)
Sci Transl Med
, vol.2
, Issue.28
-
-
Ahmed, M.R.1
-
5
-
-
79955622718
-
Priorities in Parkinson's disease research
-
Meissner WG et al (2011) Priorities in Parkinson's disease research. Nat Rev Drug Discov 10(5):377-393
-
(2011)
Nat Rev Drug Discov
, vol.10
, Issue.5
, pp. 377-393
-
-
Meissner, W.G.1
-
6
-
-
21044458854
-
A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer disease
-
Tuszynski MH et al (2005) A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer disease. Nat Med 11(5):551-555
-
(2005)
Nat Med
, vol.11
, Issue.5
, pp. 551-555
-
-
Tuszynski, M.H.1
-
7
-
-
78349247631
-
Gene delivery of AAV2-neurturin for Parkinson's disease: A double-blind, randomised, controlled trial
-
Marks WJ Jr et al (2010) Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol 9(12):1164-1172
-
(2010)
Lancet Neurol
, vol.9
, Issue.12
, pp. 1164-1172
-
-
Marks, W.J.1
-
8
-
-
60649116702
-
Applications of lentiviral vectors for biology and gene therapy of neurological disorders
-
Lundberg C et al (2008) Applications of lentiviral vectors for biology and gene therapy of neurological disorders. Curr Gene Ther 8(6):461-473
-
(2008)
Curr Gene Ther
, vol.8
, Issue.6
, pp. 461-473
-
-
Lundberg, C.1
-
9
-
-
0032752994
-
Lentiviral gene transfer to the nonhuman primate brain
-
Kordower JH et al (1999) Lentiviral gene transfer to the nonhuman primate brain. Exp Neurol 160(1):1-16
-
(1999)
Exp Neurol
, vol.160
, Issue.1
, pp. 1-16
-
-
Kordower, J.H.1
-
10
-
-
0037378036
-
In vivo gene delivery of glial cell line-derived neurotrophic factor for Parkinson's disease
-
discussion S132-4
-
Kordower JH (2003) In vivo gene delivery of glial cell line-derived neurotrophic factor for Parkinson's disease. Ann Neurol 53(Suppl 3):S120-S132, discussion S132-4
-
(2003)
Ann Neurol
, vol.53
, pp. 120-132
-
-
Kordower, J.H.1
-
11
-
-
77956747059
-
Effects of glial cell line-derived neurotrophic factor on the nigrostriatal dopamine system in rodents and nonhuman primates
-
Gash DM, Gerhardt GA, Hoffer BJ (1998) Effects of glial cell line-derived neurotrophic factor on the nigrostriatal dopamine system in rodents and nonhuman primates. Adv Pharmacol 42:911-915
-
(1998)
Adv Pharmacol
, vol.42
, pp. 911-915
-
-
Gash, D.M.1
Gerhardt, G.A.2
Hoffer, B.J.3
-
12
-
-
0034721690
-
Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease
-
Kordower JH et al (2000) Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease. Science 290(5492):767-773
-
(2000)
Science
, vol.290
, Issue.5492
, pp. 767-773
-
-
Kordower, J.H.1
-
13
-
-
0037096359
-
Lentivirally delivered glial cell line-derived neurotrophic factor increases the number of striatal dopaminergic neurons in primate models of nigrostriatal degeneration
-
Palfi S et al (2002) Lentivirally delivered glial cell line-derived neurotrophic factor increases the number of striatal dopaminergic neurons in primate models of nigrostriatal degeneration. J Neurosci 22(12):4942-4954
-
(2002)
J Neurosci
, vol.22
, Issue.12
, pp. 4942-4954
-
-
Palfi, S.1
-
14
-
-
76949085430
-
Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia
-
Jarraya B et al (2009) Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia. Sci Transl Med 1(2):2ra4
-
(2009)
Sci Transl Med
, vol.1
, Issue.2
-
-
Jarraya, B.1
-
15
-
-
0035434121
-
Pathophysiology of levodopa-induced dyskinesia: Potential for new therapies
-
Bezard E, Brotchie JM, Gross CE (2001) Pathophysiology of levodopa-induced dyskinesia: potential for new therapies. Nat Rev Neurosci 2(8):577-588
-
(2001)
Nat Rev Neurosci
, vol.2
, Issue.8
, pp. 577-588
-
-
Bezard, E.1
Brotchie, J.M.2
Gross, C.E.3
-
16
-
-
34248575534
-
Levodopa in the treatment of Parkinson's disease
-
Fahn S (2006) Levodopa in the treatment of Parkinson's disease. J Neural Transm Suppl (71):1-15
-
(2006)
J Neural Transm Suppl
, Issue.71
, pp. 1-15
-
-
Fahn, S.1
-
17
-
-
34147170029
-
Altered D(1) dopamine receptor trafficking in parkinsonian and dyskinetic non-human primates
-
Guigoni C, Doudnikoff E, Li Q, Bloch B, Bezard E (2007) Altered D(1) dopamine receptor trafficking in parkinsonian and dyskinetic non-human primates. Neurobiol Dis 26(2):452-463
-
(2007)
Neurobiol Dis
, vol.26
, Issue.2
, pp. 452-463
-
-
Guigoni, C.1
Doudnikoff, E.2
Li, Q.3
Bloch, B.4
Bezard, E.5
-
18
-
-
15944407993
-
L-DOPA reverses the MPTP-induced elevation of the arrestin2 and GRK6 expression and enhanced ERK activation in monkey brain
-
Bezard E et al (2005) L-DOPA reverses the MPTP-induced elevation of the arrestin2 and GRK6 expression and enhanced ERK activation in monkey brain. Neurobiol Dis 18(2): 323-335
-
(2005)
Neurobiol Dis
, vol.18
, Issue.2
, pp. 323-335
-
-
Bezard, E.1
-
19
-
-
78650753240
-
Inhibition of Rasguanine nucleotide-releasing factor 1 (Ras- GRF1) signaling in the striatum reverts motor symptoms associated with L-dopa-induced dyskinesia
-
Fasano S et al (2010) Inhibition of Rasguanine nucleotide-releasing factor 1 (Ras- GRF1) signaling in the striatum reverts motor symptoms associated with L-dopa-induced dyskinesia. Proc Natl Acad Sci USA 107(50):21824-21829
-
(2010)
Proc Natl Acad Sci USA
, vol.107
, Issue.50
, pp. 21824-21829
-
-
Fasano, S.1
-
20
-
-
0029417121
-
Chronic L-DOPA administration induces dyskinesias in the 1-methyl-4- phenyl-1,2,3,6-tetrahydropyridine-treated common marmoset (Callithrix Jacchus)
-
Pearce RK, Jackson M, Smith L, Jenner P, Marsden CD (1995) Chronic L-DOPA administration induces dyskinesias in the 1-methyl-4- phenyl-1,2,3,6-tetrahydropyridine-treated common marmoset (Callithrix Jacchus). Mov Disord 10(6):731-740
-
(1995)
Mov Disord
, vol.10
, Issue.6
, pp. 731-740
-
-
Pearce, R.K.1
Jackson, M.2
Smith, L.3
Jenner, P.4
Marsden, C.D.5
-
21
-
-
37549025843
-
RGS9-2 negatively modulates L-3,4-dihydroxyphenylalanine-induced dyskinesia in experimental Parkinson's disease
-
Gold SJ et al (2007) RGS9-2 negatively modulates L-3,4-dihydroxyphenylalanine-induced dyskinesia in experimental Parkinson's disease. J Neurosci 27(52):14338-14348
-
(2007)
J Neurosci
, vol.27
, Issue.52
, pp. 14338-14348
-
-
Gold, S.J.1
-
22
-
-
16344390018
-
Subcellular redistribution of the synapse-associated proteins PSD-95 and SAP97 in animal models of Parkinson's disease and L-DOPA-induced dyskinesia
-
Nash JE, Johnston TH, Collingridge GL, Garner CC, Brotchie JM (2005) Subcellular redistribution of the synapse-associated proteins PSD-95 and SAP97 in animal models of Parkinson's disease and L-DOPA-induced dyskinesia. FASEB J 19(6):583-585
-
(2005)
FASEB J
, vol.19
, Issue.6
, pp. 583-585
-
-
Nash, J.E.1
Johnston, T.H.2
Collingridge, G.L.3
Garner, C.C.4
Brotchie, J.M.5
-
23
-
-
34547136437
-
Expression of mutated huntingtin fragment in the putamen is sufficient to produce abnormal movement in non- human primates
-
Palfi S et al (2007) Expression of mutated huntingtin fragment in the putamen is sufficient to produce abnormal movement in non- human primates. Mol Ther 15(8): 1444-1451
-
(2007)
Mol Ther
, vol.15
, Issue.8
, pp. 1444-1451
-
-
Palfi, S.1
-
24
-
-
40549119871
-
Designer gene delivery vectors: Molecular engineering and evolution of adeno-associated viral vectors for enhanced gene transfer
-
Kwon I, Schaffer DV (2008) Designer gene delivery vectors: molecular engineering and evolution of adeno-associated viral vectors for enhanced gene transfer. Pharm Res 25(3): 489-499
-
(2008)
Pharm Res
, vol.25
, Issue.3
, pp. 489-499
-
-
Kwon, I.1
Schaffer, D.V.2
-
25
-
-
70249151690
-
Adenovirus-adeno-associated virus hybrid for large-scale recombinant adenoassociated virus production
-
Zhang H, Xie J, Xie Q, Wilson JM, Gao G (2009) Adenovirus-adeno-associated virus hybrid for large-scale recombinant adenoassociated virus production. Hum Gene Ther 20(9):922-929
-
(2009)
Hum Gene Ther
, vol.20
, Issue.9
, pp. 922-929
-
-
Zhang, H.1
Xie, J.2
Xie, Q.3
Wilson, J.M.4
Gao, G.5
-
27
-
-
0037015049
-
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
-
Gao GP et al (2002) Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 99(18):11854-11859
-
(2002)
Proc Natl Acad Sci USA
, vol.99
, Issue.18
, pp. 11854-11859
-
-
Gao, G.P.1
-
28
-
-
62549159967
-
Tailoring the AAV vector capsid for gene therapy
-
Vandenberghe LH, Wilson JM, Gao G (2009) Tailoring the AAV vector capsid for gene therapy. Gene Ther 16(3):311-319
-
(2009)
Gene Ther
, vol.16
, Issue.3
, pp. 311-319
-
-
Vandenberghe, L.H.1
Wilson, J.M.2
Gao, G.3
-
29
-
-
0033942230
-
Convectionenhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach
-
Bankiewicz KS et al (2000) Convectionenhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach. Exp Neurol 164(1):2-14
-
(2000)
Exp Neurol
, vol.164
, Issue.1
, pp. 2-14
-
-
Bankiewicz, K.S.1
-
30
-
-
33748083041
-
Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC
-
Bankiewicz KS et al (2006) Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC. Mol Ther 14(4):564-570
-
(2006)
Mol Ther
, vol.14
, Issue.4
, pp. 564-570
-
-
Bankiewicz, K.S.1
-
31
-
-
33748074480
-
A dose-ranging study of AAV-hAADC therapy in Parkinsonian monkeys
-
Forsayeth JR et al (2006) A dose-ranging study of AAV-hAADC therapy in Parkinsonian monkeys. Mol Ther 14(4):571-577
-
(2006)
Mol Ther
, vol.14
, Issue.4
, pp. 571-577
-
-
Forsayeth, J.R.1
-
32
-
-
77955175906
-
Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC
-
Hadaczek P et al (2010) Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC. Mol Ther 18(8):1458-1461
-
(2010)
Mol Ther
, vol.18
, Issue.8
, pp. 1458-1461
-
-
Hadaczek, P.1
-
33
-
-
0036210202
-
Behavioral recovery in a primate model of Parkinson's disease by triple transduction of striatal cells with adenoassociated viral vectors expressing dopaminesynthesizing enzymes
-
Muramatsu S et al (2002) Behavioral recovery in a primate model of Parkinson's disease by triple transduction of striatal cells with adenoassociated viral vectors expressing dopaminesynthesizing enzymes. Hum Gene Ther 13(3):345-354
-
(2002)
Hum Gene Ther
, vol.13
, Issue.3
, pp. 345-354
-
-
Muramatsu, S.1
-
34
-
-
0011087583
-
Deep brain stimulation for Parkinson's disease: Disrupting the disruption
-
Lozano AM, Dostrovsky J, Chen R, Ashby P (2002) Deep brain stimulation for Parkinson's disease: disrupting the disruption. Lancet Neurol 1(4):225-231
-
(2002)
Lancet Neurol
, vol.1
, Issue.4
, pp. 225-231
-
-
Lozano, A.M.1
Dostrovsky, J.2
Chen, R.3
Ashby, P.4
-
35
-
-
33847198228
-
Subthalamic glutamic acid decarboxylase gene therapy: Changes in motor function and cortical metabolism
-
Emborg ME et al (2007) Subthalamic glutamic acid decarboxylase gene therapy: changes in motor function and cortical metabolism. J Cereb Blood Flow Metab 27(3):501-509
-
(2007)
J Cereb Blood Flow Metab
, vol.27
, Issue.3
, pp. 501-509
-
-
Emborg, M.E.1
-
36
-
-
37349011012
-
Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's disease
-
Feigin A et al (2007) Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's disease. Proc Natl Acad Sci USA 104(49):19559-19564
-
(2007)
Proc Natl Acad Sci USA
, vol.104
, Issue.49
, pp. 19559-19564
-
-
Feigin, A.1
-
37
-
-
79952740079
-
AAV2-GAD gene therapy for advanced Parkinson's disease: A doubleblind, sham-surgery controlled, randomised trial
-
LeWitt PA et al (2011) AAV2-GAD gene therapy for advanced Parkinson's disease: a doubleblind, sham-surgery controlled, randomised trial. Lancet Neurol 10(4):309-319
-
(2011)
Lancet Neurol
, vol.10
, Issue.4
, pp. 309-319
-
-
LeWitt, P.A.1
-
38
-
-
12844287442
-
Continuous low-level glial cell line-derived neurotrophic factor delivery using recombinant adeno-associated viral vectors provides neuroprotection and induces behavioral recovery in a primate model of Parkinson's disease
-
Eslamboli A et al (2005) Continuous low-level glial cell line-derived neurotrophic factor delivery using recombinant adeno-associated viral vectors provides neuroprotection and induces behavioral recovery in a primate model of Parkinson's disease. J Neurosci 25(4):769-777
-
(2005)
J Neurosci
, vol.25
, Issue.4
, pp. 769-777
-
-
Eslamboli, A.1
-
39
-
-
77954692657
-
Regeneration of the MPTP-lesioned dopaminergic system after convection-enhanced delivery of AAV2- GDNF
-
Kells AP et al (2010) Regeneration of the MPTP-lesioned dopaminergic system after convection-enhanced delivery of AAV2- GDNF. J Neurosci 30(28):9567-9577
-
(2010)
J Neurosci
, vol.30
, Issue.28
, pp. 9567-9577
-
-
Kells, A.P.1
-
40
-
-
33845993229
-
Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTPtreated monkeys
-
Kordower JH et al (2006) Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTPtreated monkeys. Ann Neurol 60(6):706-715
-
(2006)
Ann Neurol
, vol.60
, Issue.6
, pp. 706-715
-
-
Kordower, J.H.1
-
41
-
-
68949175520
-
Striatal overexpression of DeltaJunD resets L-DOPA-induced dyskinesia in a primate model of Parkinson disease
-
Berton O et al (2009) Striatal overexpression of DeltaJunD resets L-DOPA-induced dyskinesia in a primate model of Parkinson disease. Biol Psychiatry 66(6):554-561
-
(2009)
Biol Psychiatry
, vol.66
, Issue.6
, pp. 554-561
-
-
Berton, O.1
-
42
-
-
64049083111
-
Human alpha- iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: Vector diffusion and biodistribution
-
Ciron C et al (2009) Human alpha- iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: vector diffusion and biodistribution. Hum Gene Ther 20(4):350-360
-
(2009)
Hum Gene Ther
, vol.20
, Issue.4
, pp. 350-360
-
-
Ciron, C.1
-
43
-
-
64149106830
-
Comparison of transduction efficiency of recombinant AAV serotypes 1, 2, 5, and 8 in the rat nigrostriatal system
-
McFarland NR, Lee JS, Hyman BT, McLean PJ (2009) Comparison of transduction efficiency of recombinant AAV serotypes 1, 2, 5, and 8 in the rat nigrostriatal system. J Neurochem 109(3):838-845
-
(2009)
J Neurochem
, vol.109
, Issue.3
, pp. 838-845
-
-
McFarland, N.R.1
Lee, J.S.2
Hyman, B.T.3
McLean, P.J.4
-
44
-
-
34247271439
-
Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain
-
Taymans JM et al (2007) Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain. Hum Gene Ther 18(3):195-206
-
(2007)
Hum Gene Ther
, vol.18
, Issue.3
, pp. 195-206
-
-
Taymans, J.M.1
-
45
-
-
77649331498
-
Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate
-
Colle MA et al (2010) Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate. Hum Mol Genet 19(1):147-158
-
(2010)
Hum Mol Genet
, vol.19
, Issue.1
, pp. 147-158
-
-
Colle, M.A.1
-
46
-
-
77649270699
-
Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates
-
Dodiya HB et al (2010) Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates. Mol Ther 18(3):579-587
-
(2010)
Mol Ther
, vol.18
, Issue.3
, pp. 579-587
-
-
Dodiya, H.B.1
-
47
-
-
62549094869
-
Transduction of nonhuman primate brain with adeno-associated virus serotype 1: Vector trafficking and immune response
-
Hadaczek P et al (2009) Transduction of nonhuman primate brain with adeno-associated virus serotype 1: vector trafficking and immune response. Hum Gene Ther 20(3):225-237
-
(2009)
Hum Gene Ther
, vol.20
, Issue.3
, pp. 225-237
-
-
Hadaczek, P.1
-
48
-
-
77649271981
-
Comparative transduction efficiency of AAV vector serotypes 1-6 in the substantia nigra and striatum of the primate brain
-
Markakis EA et al (2010) Comparative transduction efficiency of AAV vector serotypes 1-6 in the substantia nigra and striatum of the primate brain. Mol Ther 18(3):588-593
-
(2010)
Mol Ther
, vol.18
, Issue.3
, pp. 588-593
-
-
Markakis, E.A.1
-
49
-
-
79952258871
-
Recombinant adenoassociated virus type 2 pseudotypes: Comparing safety, specificity, and transduction efficiency in the primate striatum. Laboratory investigation
-
Sanchez CE et al (2011) Recombinant adenoassociated virus type 2 pseudotypes: comparing safety, specificity, and transduction efficiency in the primate striatum. Laboratory investigation. J Neurosurg 114(3):672-680
-
(2011)
J Neurosurg
, vol.114
, Issue.3
, pp. 672-680
-
-
Sanchez, C.E.1
-
50
-
-
33845196246
-
Neuronal specificity of alpha-synuclein toxicity and effect of Parkin co-expression in primates
-
Yasuda T et al (2007) Neuronal specificity of alpha-synuclein toxicity and effect of Parkin co-expression in primates. Neuroscience 144(2):743-753
-
(2007)
Neuroscience
, vol.144
, Issue.2
, pp. 743-753
-
-
Yasuda, T.1
-
51
-
-
35148872604
-
DNA palindromes with a modest arm length of greater, similar 20 base pairs are a significant target for recombinant adeno-associated virus vector integration in the liver, muscles, and heart in mice
-
Inagaki K et al (2007) DNA palindromes with a modest arm length of greater, similar 20 base pairs are a significant target for recombinant adeno-associated virus vector integration in the liver, muscles, and heart in mice. J Virol 81(20):11290-11303
-
(2007)
J Virol
, vol.81
, Issue.20
, pp. 11290-11303
-
-
Inagaki, K.1
-
52
-
-
79961032418
-
Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system
-
Zhang H et al (2011) Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system. Mol Ther 19(8):1440-1448
-
(2011)
Mol Ther
, vol.19
, Issue.8
, pp. 1440-1448
-
-
Zhang, H.1
-
53
-
-
60149106907
-
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
-
Foust KD et al (2009) Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol 27(1):59-65
-
(2009)
Nat Biotechnol
, vol.27
, Issue.1
, pp. 59-65
-
-
Foust, K.D.1
-
54
-
-
67649861393
-
Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons
-
Duque S et al (2009) Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol Ther 17(7):1187-1196
-
(2009)
Mol Ther
, vol.17
, Issue.7
, pp. 1187-1196
-
-
Duque, S.1
-
55
-
-
78751700314
-
Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice
-
Dominguez E et al (2011) Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice. Hum Mol Genet 20(4):681-693
-
(2011)
Hum Mol Genet
, vol.20
, Issue.4
, pp. 681-693
-
-
Dominguez, E.1
-
56
-
-
77749249680
-
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
-
Foust KD et al (2010) Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol 28(3):271-274
-
(2010)
Nat Biotechnol
, vol.28
, Issue.3
, pp. 271-274
-
-
Foust, K.D.1
-
57
-
-
79955574241
-
Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder
-
Spampanato C et al (2011) Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder. Mol Ther 19(5):860-869
-
(2011)
Mol Ther
, vol.19
, Issue.5
, pp. 860-869
-
-
Spampanato, C.1
-
58
-
-
79957888016
-
Preclinical differences of intravascular AAV9 delivery to neurons and glia: A comparative study of adult mice and nonhuman primates
-
Gray SJ et al (2011) Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. Mol Ther 19(6):1058-1069
-
(2011)
Mol Ther
, vol.19
, Issue.6
, pp. 1058-1069
-
-
Gray, S.J.1
-
59
-
-
80455173951
-
Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders
-
Bevan AK et al (2011) Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders. Mol Ther 19(11):1971-1980
-
(2011)
Mol Ther
, vol.19
, Issue.11
, pp. 1971-1980
-
-
Bevan, A.K.1
-
60
-
-
84860171925
-
Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates
-
Samaranch L et al (2012) Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates. Hum Gene Ther 23(4):382-389
-
(2012)
Hum Gene Ther
, vol.23
, Issue.4
, pp. 382-389
-
-
Samaranch, L.1
-
61
-
-
84859743388
-
Systemic scAAV9 variant mediates brain transduction in newborn rhesus macaques
-
Dehay B, Dalkara D, Dovero S, Li Q, Bezard E (2012) Systemic scAAV9 variant mediates brain transduction in newborn rhesus macaques. Sci Rep 2:253
-
(2012)
Sci Rep
, vol.2
, pp. 253
-
-
Dehay, B.1
Dalkara, D.2
Dovero, S.3
Li, Q.4
Bezard, E.5
-
62
-
-
84871994994
-
Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems
-
Mattar CN et al (2013) Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems. Gene Ther 20(1):69-83
-
(2013)
Gene Ther
, vol.20
, Issue.1
, pp. 69-83
-
-
Mattar, C.N.1
-
63
-
-
84864877570
-
Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs
-
Federici T et al (2012) Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs. Gene Ther 19(8):852-859
-
(2012)
Gene Ther
, vol.19
, Issue.8
, pp. 852-859
-
-
Federici, T.1
-
64
-
-
79952193362
-
MicroRNA-regulated, systemically delivered rAAV9: A step closer to CNS-restricted transgene expression
-
Xie J et al (2011) MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression. Mol Ther 19(3):526-535
-
(2011)
Mol Ther
, vol.19
, Issue.3
, pp. 526-535
-
-
Xie, J.1
-
65
-
-
84859440283
-
The AAV vector toolkit: Poised at the clinical crossroads
-
Asokan A, Schaffer DV, Samulski RJ (2012) The AAV vector toolkit: poised at the clinical crossroads. Mol Ther 20(4):699-708
-
(2012)
Mol Ther
, vol.20
, Issue.4
, pp. 699-708
-
-
Asokan, A.1
Schaffer, D.V.2
Samulski, R.J.3
-
66
-
-
77649273563
-
Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB)
-
Gray SJ et al (2010) Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB). Mol Ther 18(3):570-578
-
(2010)
Mol Ther
, vol.18
, Issue.3
, pp. 570-578
-
-
Gray, S.J.1
-
67
-
-
74049120342
-
Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle
-
Asokan A et al (2010) Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle. Nat Biotechnol 28(1):79-82
-
(2010)
Nat Biotechnol
, vol.28
, Issue.1
, pp. 79-82
-
-
Asokan, A.1
-
68
-
-
84875925195
-
Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
-
Gray SJ, Nagabhushan Kalburgi S, McCown TJ, Jude Samulski R (2013) Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates. Gene Ther 20(4):450-459
-
(2013)
Gene Ther
, vol.20
, Issue.4
, pp. 450-459
-
-
Gray, S.J.1
Nagabhushan Kalburgi, S.2
McCown, T.J.3
Jude Samulski, R.4
-
69
-
-
78649325447
-
Comparison of ventricular and intravenous lentiviral-mediated gene therapy for murine MPS VII
-
Bielicki J, McIntyre C, Anson DS (2010) Comparison of ventricular and intravenous lentiviral-mediated gene therapy for murine MPS VII. Mol Genet Metab 101(4):370-382
-
(2010)
Mol Genet Metab
, vol.101
, Issue.4
, pp. 370-382
-
-
Bielicki, J.1
McIntyre, C.2
Anson, D.S.3
-
70
-
-
0038618702
-
Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidasedeficient mice
-
Passini MA et al (2003) Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidasedeficient mice. J Virol 77(12):7034-7040
-
(2003)
J Virol
, vol.77
, Issue.12
, pp. 7034-7040
-
-
Passini, M.A.1
-
71
-
-
77749292139
-
Genetic approach for intracerebroventricular delivery
-
Regev L, Ezrielev E, Gershon E, Gil S, Chen A (2010) Genetic approach for intracerebroventricular delivery. Proc Natl Acad Sci USA 107(9):4424-4429
-
(2010)
Proc Natl Acad Sci USA
, vol.107
, Issue.9
, pp. 4424-4429
-
-
Regev, L.1
Ezrielev, E.2
Gershon, E.3
Gil, S.4
Chen, A.5
-
72
-
-
79961013057
-
Neurosurgical convection-enhanced delivery of treatments for Parkinson's disease
-
Lam MF, Thomas MG, Lind CR (2011) Neurosurgical convection-enhanced delivery of treatments for Parkinson's disease. J Clin Neurosci 18(9):1163-1167
-
(2011)
J Clin Neurosci
, vol.18
, Issue.9
, pp. 1163-1167
-
-
Lam, M.F.1
Thomas, M.G.2
Lind, C.R.3
-
73
-
-
23444437213
-
Convection-enhanced delivery of macromolecules in the brain
-
Bobo RH et al (1994) Convection-enhanced delivery of macromolecules in the brain. Proc Natl Acad Sci USA 91(6):2076-2080
-
(1994)
Proc Natl Acad Sci USA
, vol.91
, Issue.6
, pp. 2076-2080
-
-
Bobo, R.H.1
-
74
-
-
45749146197
-
Biodistribution of adeno-associated virus type-2 in nonhuman primates after convection-enhanced delivery to brain
-
Cunningham J et al (2008) Biodistribution of adeno-associated virus type-2 in nonhuman primates after convection-enhanced delivery to brain. Mol Ther 16(7):1267-1275
-
(2008)
Mol Ther
, vol.16
, Issue.7
, pp. 1267-1275
-
-
Cunningham, J.1
-
75
-
-
37349081554
-
Image-guided convection- enhanced delivery platform in the treatment of neurological diseases
-
Fiandaca MS, Forsayeth JR, Dickinson PJ, Bankiewicz KS (2008) Image-guided convection- enhanced delivery platform in the treatment of neurological diseases. Neurotherapeutics 5(1):123-127
-
(2008)
Neurotherapeutics
, vol.5
, Issue.1
, pp. 123-127
-
-
Fiandaca, M.S.1
Forsayeth, J.R.2
Dickinson, P.J.3
Bankiewicz, K.S.4
-
76
-
-
84860001035
-
Guided delivery of adeno-associated viral vectors into the primate brain
-
Salegio EA, Samaranch L, Kells AP, Forsayeth J, Bankiewicz K (2012) Guided delivery of adeno-associated viral vectors into the primate brain. Adv Drug Deliv Rev 64(7):598-604
-
(2012)
Adv Drug Deliv Rev
, vol.64
, Issue.7
, pp. 598-604
-
-
Salegio, E.A.1
Samaranch, L.2
Kells, A.P.3
Forsayeth, J.4
Bankiewicz, K.5
-
77
-
-
3242747428
-
A realistic brain tissue phantom for intraparenchymal infusion studies
-
Chen ZJ et al (2004) A realistic brain tissue phantom for intraparenchymal infusion studies. J Neurosurg 101(2):314-322
-
(2004)
J Neurosurg
, vol.101
, Issue.2
, pp. 314-322
-
-
Chen, Z.J.1
-
78
-
-
33747614013
-
In vitro and in vivo imaging studies of a new endohedral metallofullerene nanoparticle
-
Fatouros PP et al (2006) In vitro and in vivo imaging studies of a new endohedral metallofullerene nanoparticle. Radiology 240(3):756-764
-
(2006)
Radiology
, vol.240
, Issue.3
, pp. 756-764
-
-
Fatouros, P.P.1
-
79
-
-
32044440778
-
Reflux-free cannula for convection-enhanced high-speed delivery of therapeutic agents
-
Krauze MT et al (2005) Reflux-free cannula for convection-enhanced high-speed delivery of therapeutic agents. J Neurosurg 103(5): 923-929
-
(2005)
J Neurosurg
, vol.103
, Issue.5
, pp. 923-929
-
-
Krauze, M.T.1
-
80
-
-
79951557738
-
Design of an indwelling cannula for convection-enhanced delivery
-
Rosenbluth KH et al (2011) Design of an indwelling cannula for convection-enhanced delivery. J Neurosci Methods 196(1):118-123
-
(2011)
J Neurosci Methods
, vol.196
, Issue.1
, pp. 118-123
-
-
Rosenbluth, K.H.1
-
81
-
-
22044450632
-
AAV2-mediated gene delivery to monkey putamen: Evaluation of an infusion device and delivery parameters
-
Sanftner LM et al (2005) AAV2-mediated gene delivery to monkey putamen: evaluation of an infusion device and delivery parameters. Exp Neurol 194(2):476-483
-
(2005)
Exp Neurol
, vol.194
, Issue.2
, pp. 476-483
-
-
Sanftner, L.M.1
-
82
-
-
27744517790
-
Gadolinium-loaded liposomes allow for real-time magnetic resonance imaging of convection-enhanced delivery in the primate brain
-
Saito R et al (2005) Gadolinium-loaded liposomes allow for real-time magnetic resonance imaging of convection-enhanced delivery in the primate brain. Exp Neurol 196(2):381-389
-
(2005)
Exp Neurol
, vol.196
, Issue.2
, pp. 381-389
-
-
Saito, R.1
-
83
-
-
78650813431
-
Optimal region of the putamen for image-guided convection-enhanced delivery of therapeutics in human and nonhuman primates
-
Yin D et al (2011) Optimal region of the putamen for image-guided convection-enhanced delivery of therapeutics in human and nonhuman primates. Neuroimage 54(Suppl 1):S196-S203
-
(2011)
Neuroimage
, vol.54
, pp. 196-203
-
-
Yin, D.1
-
84
-
-
44949222522
-
Results from a phase I safety trial of hAADC gene therapy for Parkinson disease
-
Eberling JL et al (2008) Results from a phase I safety trial of hAADC gene therapy for Parkinson disease. Neurology 70(21):1980-1983
-
(2008)
Neurology
, vol.70
, Issue.21
, pp. 1980-1983
-
-
Eberling, J.L.1
|