메뉴 건너뛰기




Volumn 21, Issue 8, 2015, Pages 504-512

Erratum to 'Gene therapy for neurodegenerative diseases' [Trends in Molecular Medicine 21 (2015) 504-512] DOI: 10.1016/j.molmed.2015.06.001;Gene therapy for neurodegenerative diseases

Author keywords

Clinical trial; Gene therapy; Neurodegenerative disease

Indexed keywords

ALZHEIMER DISEASE; AMYOTROPHIC LATERAL SCLEROSIS; DEGENERATIVE DISEASE; GENE THERAPY; HUMAN; LYSOSOME STORAGE DISEASE; NONHUMAN; OUTCOME ASSESSMENT; PARKINSON DISEASE; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); REVIEW; SPINAL MUSCULAR ATROPHY; SPINOCEREBELLAR DEGENERATION; TREATMENT RESPONSE; UNIFIED PARKINSON DISEASE RATING SCALE; ADVERSE EFFECTS; ANIMAL; CLINICAL TRIAL (TOPIC); GENETICS; NEURODEGENERATIVE DISEASES; PROCEDURES; TREATMENT OUTCOME;

EID: 84938552922     PISSN: 14714914     EISSN: 1471499X     Source Type: Journal    
DOI: 10.1016/j.molmed.2015.12.008     Document Type: Erratum
Times cited : (105)

References (121)
  • 1
    • 84875240244 scopus 로고    scopus 로고
    • Adeno-associated virus (AAV) gene therapy for neurological disease
    • Weinberg M.S., et al. Adeno-associated virus (AAV) gene therapy for neurological disease. Neuropharmacology 2013, 69:82-88.
    • (2013) Neuropharmacology , vol.69 , pp. 82-88
    • Weinberg, M.S.1
  • 2
    • 67849124729 scopus 로고    scopus 로고
    • Gene therapy for neurodegenerative diseases based on lentiviral vectors
    • Nanou A., Azzouz M. Gene therapy for neurodegenerative diseases based on lentiviral vectors. Prog. Brain Res. 2009, 175:187-200.
    • (2009) Prog. Brain Res. , vol.175 , pp. 187-200
    • Nanou, A.1    Azzouz, M.2
  • 3
    • 67649861393 scopus 로고    scopus 로고
    • Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons
    • Duque S., et al. Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol. Ther. 2009, 17:1187-1196.
    • (2009) Mol. Ther. , vol.17 , pp. 1187-1196
    • Duque, S.1
  • 4
    • 84929237446 scopus 로고    scopus 로고
    • Intraspinal stem cell transplantation in amyotrophic lateral sclerosis: a phase I safety trial, technical note, and lumbar safety outcomes
    • Riley J., et al. Intraspinal stem cell transplantation in amyotrophic lateral sclerosis: a phase I safety trial, technical note, and lumbar safety outcomes. Neurosurgery 2012, 71:405-416.
    • (2012) Neurosurgery , vol.71 , pp. 405-416
    • Riley, J.1
  • 5
    • 84864877570 scopus 로고    scopus 로고
    • Robust spinal motor neuron transduction following intrathecal delivery of AAV9.GFP in pigs
    • Federici T., et al. Robust spinal motor neuron transduction following intrathecal delivery of AAV9.GFP in pigs. Gene Ther. 2012, 19:852-859.
    • (2012) Gene Ther. , vol.19 , pp. 852-859
    • Federici, T.1
  • 6
    • 41949110690 scopus 로고    scopus 로고
    • Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial
    • Marks W.J., et al. Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol. 2008, 7:400-408.
    • (2008) Lancet Neurol. , vol.7 , pp. 400-408
    • Marks, W.J.1
  • 7
    • 85027921807 scopus 로고    scopus 로고
    • A phase1 study of stereotactic gene delivery of AAV2-NGF for Alzheimer's disease
    • Rafii M.S., et al. A phase1 study of stereotactic gene delivery of AAV2-NGF for Alzheimer's disease. Alzheimers Dement. 2014, 10:571-581.
    • (2014) Alzheimers Dement. , vol.10 , pp. 571-581
    • Rafii, M.S.1
  • 8
    • 84871525461 scopus 로고    scopus 로고
    • Long-term follow-up after gene therapy for canavan disease
    • 165ra163
    • Leone P., et al. Long-term follow-up after gene therapy for canavan disease. Sci. Transl. Med. 2012, 4:165ra163.
    • (2012) Sci. Transl. Med. , vol.4
    • Leone, P.1
  • 9
    • 84926154769 scopus 로고    scopus 로고
    • Axial disability and deep brain stimulation in patients with Parkinson disease
    • Fasano A., et al. Axial disability and deep brain stimulation in patients with Parkinson disease. Nat. Rev. Neurol. 2015, 11:98-110.
    • (2015) Nat. Rev. Neurol. , vol.11 , pp. 98-110
    • Fasano, A.1
  • 10
    • 84895442417 scopus 로고    scopus 로고
    • Parkinson's disease gene therapy: success by design meets failure by efficacy
    • Bartus R.T., et al. Parkinson's disease gene therapy: success by design meets failure by efficacy. Mol. Ther. 2014, 22:487-497.
    • (2014) Mol. Ther. , vol.22 , pp. 487-497
    • Bartus, R.T.1
  • 11
    • 79952740079 scopus 로고    scopus 로고
    • AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial
    • LeWitt P.A., et al. AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial. Lancet Neurol. 2011, 10:309-319.
    • (2011) Lancet Neurol. , vol.10 , pp. 309-319
    • LeWitt, P.A.1
  • 12
    • 78349247631 scopus 로고    scopus 로고
    • Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial
    • Marks W.J., et al. Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol. 2010, 9:1164-1172.
    • (2010) Lancet Neurol. , vol.9 , pp. 1164-1172
    • Marks, W.J.1
  • 13
    • 79961126259 scopus 로고    scopus 로고
    • Properly scaled and targeted AAV2-NRTN (neurturin) to the substantia nigra is safe, effective and causes no weight loss: support for nigral targeting in Parkinson's disease
    • Bartus R.T., et al. Properly scaled and targeted AAV2-NRTN (neurturin) to the substantia nigra is safe, effective and causes no weight loss: support for nigral targeting in Parkinson's disease. Neurobiol. Dis. 2011, 44:38-52.
    • (2011) Neurobiol. Dis. , vol.44 , pp. 38-52
    • Bartus, R.T.1
  • 14
    • 79951476995 scopus 로고    scopus 로고
    • Bioactivity of AAV2-neurturin gene therapy (CERE-120): differences between Parkinson's disease and nonhuman primate brains
    • Bartus R.T., et al. Bioactivity of AAV2-neurturin gene therapy (CERE-120): differences between Parkinson's disease and nonhuman primate brains. Mov. Disord. 2011, 26:27-36.
    • (2011) Mov. Disord. , vol.26 , pp. 27-36
    • Bartus, R.T.1
  • 15
    • 84879394186 scopus 로고    scopus 로고
    • Enhanced neurotrophic distribution, cell signaling and neuroprotection following substantia nigral versus striatal delivery of AAV2-NRTN (CERE-120)
    • Herzog C.D., et al. Enhanced neurotrophic distribution, cell signaling and neuroprotection following substantia nigral versus striatal delivery of AAV2-NRTN (CERE-120). Neurobiol. Dis. 2013, 58:38-48.
    • (2013) Neurobiol. Dis. , vol.58 , pp. 38-48
    • Herzog, C.D.1
  • 16
    • 84868156476 scopus 로고    scopus 로고
    • Advancing neurotrophic factors as treatments for age-related neurodegenerative diseases: developing and demonstrating 'clinical proof-of-concept' for AAV-neurturin (CERE-120) in Parkinson's disease
    • Bartus R.T., et al. Advancing neurotrophic factors as treatments for age-related neurodegenerative diseases: developing and demonstrating 'clinical proof-of-concept' for AAV-neurturin (CERE-120) in Parkinson's disease. Neurobiol. Aging 2013, 34:35-61.
    • (2013) Neurobiol. Aging , vol.34 , pp. 35-61
    • Bartus, R.T.1
  • 17
    • 0036897235 scopus 로고    scopus 로고
    • Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induces sustained transgene expression, dopamine production, and functional improvement in a rat model of Parkinson's disease
    • Azzouz M., et al. Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induces sustained transgene expression, dopamine production, and functional improvement in a rat model of Parkinson's disease. J. Neurosci. 2002, 22:10302-10312.
    • (2002) J. Neurosci. , vol.22 , pp. 10302-10312
    • Azzouz, M.1
  • 18
    • 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 1/2 trial
    • Palfi S., et al. Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial. Lancet 2014, 383:1138-1146.
    • (2014) Lancet , vol.383 , pp. 1138-1146
    • Palfi, S.1
  • 19
    • 77956262279 scopus 로고    scopus 로고
    • A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease
    • Muramatsu S., et al. A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease. Mol. Ther. 2010, 18:1731-1735.
    • (2010) Mol. Ther. , vol.18 , pp. 1731-1735
    • Muramatsu, S.1
  • 20
    • 73449128979 scopus 로고    scopus 로고
    • Safety and tolerability of putaminal AADC gene therapy for Parkinson disease
    • Christine C.W., et al. Safety and tolerability of putaminal AADC gene therapy for Parkinson disease. Neurology 2009, 73:1662-1669.
    • (2009) Neurology , vol.73 , pp. 1662-1669
    • Christine, C.W.1
  • 21
    • 84860130544 scopus 로고    scopus 로고
    • Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease
    • Mittermeyer G., et al. Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease. Hum. Gene Ther. 2012, 23:377-381.
    • (2012) Hum. Gene Ther. , vol.23 , pp. 377-381
    • Mittermeyer, G.1
  • 22
    • 84856921721 scopus 로고    scopus 로고
    • Safety and tolerability of magnetic resonance imaging-guided convection-enhanced delivery of AAV2-hAADC with a novel delivery platform in nonhuman primate striatum
    • San Sebastian W., et al. Safety and tolerability of magnetic resonance imaging-guided convection-enhanced delivery of AAV2-hAADC with a novel delivery platform in nonhuman primate striatum. Hum. Gene Ther. 2012, 23:210-217.
    • (2012) Hum. Gene Ther. , vol.23 , pp. 210-217
    • San Sebastian, W.1
  • 23
    • 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
    • Cederfjall E., et al. 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. 2012, 20:1315-1326.
    • (2012) Mol. Ther. , vol.20 , pp. 1315-1326
    • Cederfjall, E.1
  • 24
    • 84880302312 scopus 로고    scopus 로고
    • Continuous DOPA synthesis from a single AAV: dosing and efficacy in models of Parkinson's disease
    • Cederfjall E., et al. Continuous DOPA synthesis from a single AAV: dosing and efficacy in models of Parkinson's disease. Sci. Rep. 2013, 3:2157.
    • (2013) Sci. Rep. , vol.3 , pp. 2157
    • Cederfjall, E.1
  • 25
    • 84880006379 scopus 로고    scopus 로고
    • AAV2-mediated striatum delivery of human CDNF prevents the deterioration of midbrain dopamine neurons in a 6-hydroxydopamine induced parkinsonian rat model
    • Ren X., et al. AAV2-mediated striatum delivery of human CDNF prevents the deterioration of midbrain dopamine neurons in a 6-hydroxydopamine induced parkinsonian rat model. Exp. Neurol. 2013, 248:148-156.
    • (2013) Exp. Neurol. , vol.248 , pp. 148-156
    • Ren, X.1
  • 26
    • 84892477651 scopus 로고    scopus 로고
    • Gene therapy with AAV2-CDNF provides functional benefits in a rat model of Parkinson's disease
    • Back S., et al. Gene therapy with AAV2-CDNF provides functional benefits in a rat model of Parkinson's disease. Brain Behav. 2013, 3:75-88.
    • (2013) Brain Behav. , vol.3 , pp. 75-88
    • Back, S.1
  • 27
    • 0036434879 scopus 로고    scopus 로고
    • Aberrant sprouting and downregulation of tyrosine hydroxylase in lesioned nigrostriatal dopamine neurons induced by long-lasting overexpression of glial cell line derived neurotrophic factor in the striatum by lentiviral gene transfer
    • Georgievska B., et al. Aberrant sprouting and downregulation of tyrosine hydroxylase in lesioned nigrostriatal dopamine neurons induced by long-lasting overexpression of glial cell line derived neurotrophic factor in the striatum by lentiviral gene transfer. Exp. Neurol. 2002, 177:461-474.
    • (2002) Exp. Neurol. , vol.177 , pp. 461-474
    • Georgievska, B.1
  • 28
    • 37549031788 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 D.N., 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. 2007, 35:676-692.
    • (2007) Toxicol. Pathol. , vol.35 , pp. 676-692
    • Hovland, D.N.1
  • 29
    • 70449122321 scopus 로고    scopus 로고
    • Tight long-term dynamic doxycycline responsive nigrostriatal GDNF using a single rAAV vector
    • Manfredsson F.P., et al. Tight long-term dynamic doxycycline responsive nigrostriatal GDNF using a single rAAV vector. Mol. Ther. 2009, 17:1857-1867.
    • (2009) Mol. Ther. , vol.17 , pp. 1857-1867
    • Manfredsson, F.P.1
  • 30
    • 0037267170 scopus 로고    scopus 로고
    • Long-term striatal overexpression of GDNF selectively downregulates tyrosine hydroxylase in the intact nigrostriatal dopamine system
    • Rosenblad C., et al. Long-term striatal overexpression of GDNF selectively downregulates tyrosine hydroxylase in the intact nigrostriatal dopamine system. Eur. J. Neurosci. 2003, 17:260-270.
    • (2003) Eur. J. Neurosci. , vol.17 , pp. 260-270
    • Rosenblad, C.1
  • 31
    • 84890118525 scopus 로고    scopus 로고
    • Functional neuroprotection and efficient regulation of GDNF using destabilizing domains in a rodent model of Parkinson's disease
    • Quintino L., et al. Functional neuroprotection and efficient regulation of GDNF using destabilizing domains in a rodent model of Parkinson's disease. Mol. Ther. 2013, 21:2169-2180.
    • (2013) Mol. Ther. , vol.21 , pp. 2169-2180
    • Quintino, L.1
  • 32
    • 77956972260 scopus 로고    scopus 로고
    • A general chemical method to regulate protein stability in the mammalian central nervous system
    • Iwamoto M., et al. A general chemical method to regulate protein stability in the mammalian central nervous system. Chem. Biol. 2010, 17:981-988.
    • (2010) Chem. Biol. , vol.17 , pp. 981-988
    • Iwamoto, M.1
  • 33
    • 84922481787 scopus 로고    scopus 로고
    • Dissection of genetic factors associated with amyotrophic lateral sclerosis
    • Leblond C.S., et al. Dissection of genetic factors associated with amyotrophic lateral sclerosis. Exp. Neurol. 2014, 262:91-101.
    • (2014) Exp. Neurol. , vol.262 , pp. 91-101
    • Leblond, C.S.1
  • 34
    • 84876466100 scopus 로고    scopus 로고
    • An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study
    • Miller T.M., et al. An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Lancet Neurol. 2013, 12:435-442.
    • (2013) Lancet Neurol. , vol.12 , pp. 435-442
    • Miller, T.M.1
  • 35
    • 84911408927 scopus 로고    scopus 로고
    • Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortex
    • Thomsen G.M., et al. Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortex. J. Neurosci. 2014, 34:15587-15600.
    • (2014) J. Neurosci. , vol.34 , pp. 15587-15600
    • Thomsen, G.M.1
  • 36
    • 84890119907 scopus 로고    scopus 로고
    • Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS
    • Foust K.D., et al. Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS. Mol. Ther. 2013, 21:2148-2159.
    • (2013) Mol. Ther. , vol.21 , pp. 2148-2159
    • Foust, K.D.1
  • 37
    • 84895456487 scopus 로고    scopus 로고
    • Adeno-associated virus-mediated delivery of a recombinant single-chain antibody against misfolded superoxide dismutase for treatment of amyotrophic lateral sclerosis
    • Patel P., et al. Adeno-associated virus-mediated delivery of a recombinant single-chain antibody against misfolded superoxide dismutase for treatment of amyotrophic lateral sclerosis. Mol. Ther. 2014, 22:498-510.
    • (2014) Mol. Ther. , vol.22 , pp. 498-510
    • Patel, P.1
  • 38
    • 84892370972 scopus 로고    scopus 로고
    • Gene and protein therapies utilizing VEGF for ALS
    • Keifer O.P., et al. Gene and protein therapies utilizing VEGF for ALS. Pharmacol. Ther. 2014, 141:261-271.
    • (2014) Pharmacol. Ther. , vol.141 , pp. 261-271
    • Keifer, O.P.1
  • 39
    • 0034978562 scopus 로고    scopus 로고
    • Deletion of the hypoxia-response element in the vascular endothelial growth factor promoter causes motor neuron degeneration
    • Oosthuyse B., et al. Deletion of the hypoxia-response element in the vascular endothelial growth factor promoter causes motor neuron degeneration. Nat. Genet. 2001, 28:131-138.
    • (2001) Nat. Genet. , vol.28 , pp. 131-138
    • Oosthuyse, B.1
  • 40
    • 78650918245 scopus 로고    scopus 로고
    • AAV4-mediated expression of IGF-1 and VEGF within cellular components of the ventricular system improves survival outcome in familial ALS mice
    • Dodge J.C., et al. AAV4-mediated expression of IGF-1 and VEGF within cellular components of the ventricular system improves survival outcome in familial ALS mice. Mol. Ther. 2010, 18:2075-2084.
    • (2010) Mol. Ther. , vol.18 , pp. 2075-2084
    • Dodge, J.C.1
  • 41
    • 80053972328 scopus 로고    scopus 로고
    • Intramuscular administration of a VEGF zinc finger transcription factor activator (VEGF-ZFP-TF) improves functional outcomes in SOD1 rats
    • Kliem M.A., et al. Intramuscular administration of a VEGF zinc finger transcription factor activator (VEGF-ZFP-TF) improves functional outcomes in SOD1 rats. Amyotroph. Lateral Scler. 2011, 12:331-339.
    • (2011) Amyotroph. Lateral Scler. , vol.12 , pp. 331-339
    • Kliem, M.A.1
  • 42
    • 79551631827 scopus 로고    scopus 로고
    • CNS-targeted viral delivery of G-CSF in an animal model for ALS: improved efficacy and preservation of the neuromuscular unit
    • Henriques A., et al. CNS-targeted viral delivery of G-CSF in an animal model for ALS: improved efficacy and preservation of the neuromuscular unit. Mol. Ther. 2011, 19:284-292.
    • (2011) Mol. Ther. , vol.19 , pp. 284-292
    • Henriques, A.1
  • 43
    • 84865987121 scopus 로고    scopus 로고
    • Modulating inflammatory monocytes with a unique microRNA gene signature ameliorates murine ALS
    • Butovsky O., et al. Modulating inflammatory monocytes with a unique microRNA gene signature ameliorates murine ALS. J. Clin. Invest. 2012, 122:3063-3087.
    • (2012) J. Clin. Invest. , vol.122 , pp. 3063-3087
    • Butovsky, O.1
  • 44
    • 84884712642 scopus 로고    scopus 로고
    • Method for widespread microRNA-155 inhibition prolongs survival in ALS-model mice
    • Koval E.D., et al. Method for widespread microRNA-155 inhibition prolongs survival in ALS-model mice. Hum. Mol. Genet. 2013, 22:4127-4135.
    • (2013) Hum. Mol. Genet. , vol.22 , pp. 4127-4135
    • Koval, E.D.1
  • 45
    • 84919687395 scopus 로고    scopus 로고
    • Nonsense-mediated decay in genetic disease: friend or foe? Mutation research
    • Miller J.N., Pearce D.A. Nonsense-mediated decay in genetic disease: friend or foe? Mutation research. Rev. Mutat. Res. 2014, 762:52-64.
    • (2014) Rev. Mutat. Res. , vol.762 , pp. 52-64
    • Miller, J.N.1    Pearce, D.A.2
  • 46
    • 84920541142 scopus 로고    scopus 로고
    • Preservation of forelimb function by UPF1 gene therapy in a rat model of TDP-43-induced motor paralysis
    • Jackson K.L., et al. Preservation of forelimb function by UPF1 gene therapy in a rat model of TDP-43-induced motor paralysis. Gene Ther. 2015, 22:20-28.
    • (2015) Gene Ther. , vol.22 , pp. 20-28
    • Jackson, K.L.1
  • 47
    • 84859492616 scopus 로고    scopus 로고
    • Atrophy of the cholinergic basal forebrain over the adult age range and in early stages of Alzheimer's disease
    • Grothe M., et al. Atrophy of the cholinergic basal forebrain over the adult age range and in early stages of Alzheimer's disease. Biol. Psychiatry 2012, 71:805-813.
    • (2012) Biol. Psychiatry , vol.71 , pp. 805-813
    • Grothe, M.1
  • 48
    • 84880759156 scopus 로고    scopus 로고
    • Neurotoxicity of amyloid beta-protein: synaptic and network dysfunction
    • Mucke L., Selkoe D.J. Neurotoxicity of amyloid beta-protein: synaptic and network dysfunction. Cold Spring Harb. Perspect. Med. 2012, 2:a006338.
    • (2012) Cold Spring Harb. Perspect. Med. , vol.2 , pp. a006338
    • Mucke, L.1    Selkoe, D.J.2
  • 49
    • 84929703757 scopus 로고    scopus 로고
    • Current and future implications of basic and translational research on amyloid-beta peptide production and removal pathways
    • Bohm C., et al. Current and future implications of basic and translational research on amyloid-beta peptide production and removal pathways. Mol. Cell. Neurosci. 2015, 66:3-11.
    • (2015) Mol. Cell. Neurosci. , vol.66 , pp. 3-11
    • Bohm, C.1
  • 50
    • 84919435450 scopus 로고    scopus 로고
    • Long-term efficacy and toxicity of cholinesterase inhibitors in the treatment of Alzheimer disease
    • Hogan D.B., et al. Long-term efficacy and toxicity of cholinesterase inhibitors in the treatment of Alzheimer disease. Can. J. Psychiatry 2014, 59:618-623.
    • (2014) Can. J. Psychiatry , vol.59 , pp. 618-623
    • Hogan, D.B.1
  • 51
    • 0042697305 scopus 로고    scopus 로고
    • Triple-transgenic model of Alzheimer's disease with plaques and tangles: intracellular Abeta and synaptic dysfunction
    • Oddo S., et al. Triple-transgenic model of Alzheimer's disease with plaques and tangles: intracellular Abeta and synaptic dysfunction. Neuron 2003, 39:409-421.
    • (2003) Neuron , vol.39 , pp. 409-421
    • Oddo, S.1
  • 52
    • 84881314432 scopus 로고    scopus 로고
    • Acat1 knockdown gene therapy decreases amyloid-beta in a mouse model of Alzheimer's disease
    • Murphy S.R., et al. Acat1 knockdown gene therapy decreases amyloid-beta in a mouse model of Alzheimer's disease. Mol. Ther. 2013, 21:1497-1506.
    • (2013) Mol. Ther. , vol.21 , pp. 1497-1506
    • Murphy, S.R.1
  • 53
    • 84905270419 scopus 로고    scopus 로고
    • Antisense-mediated exon skipping decreases tau protein expression: a potential therapy for tauopathies
    • Sud R., et al. Antisense-mediated exon skipping decreases tau protein expression: a potential therapy for tauopathies. Mol. Ther. Nucleic Acids 2014, 3:e180.
    • (2014) Mol. Ther. Nucleic Acids , vol.3 , pp. e180
    • Sud, R.1
  • 54
    • 27344441173 scopus 로고    scopus 로고
    • Metabolism of amyloid-beta peptide and Alzheimer's disease
    • Iwata N., et al. Metabolism of amyloid-beta peptide and Alzheimer's disease. Pharmacol. Ther. 2005, 108:129-148.
    • (2005) Pharmacol. Ther. , vol.108 , pp. 129-148
    • Iwata, N.1
  • 55
    • 84875866006 scopus 로고    scopus 로고
    • Global brain delivery of neprilysin gene by intravascular administration of AAV vector in mice
    • Iwata N., et al. Global brain delivery of neprilysin gene by intravascular administration of AAV vector in mice. Sci. Rep. 2013, 3:1472.
    • (2013) Sci. Rep. , vol.3 , pp. 1472
    • Iwata, N.1
  • 56
    • 84875499318 scopus 로고    scopus 로고
    • Intracranial injection of AAV expressing NEP but not IDE reduces amyloid pathology in APP+PS1 transgenic mice
    • Carty N., et al. Intracranial injection of AAV expressing NEP but not IDE reduces amyloid pathology in APP+PS1 transgenic mice. PLoS ONE 2013, 8:e59626.
    • (2013) PLoS ONE , vol.8 , pp. e59626
    • Carty, N.1
  • 57
    • 77149145513 scopus 로고    scopus 로고
    • Leptin reduces the accumulation of Abeta and phosphorylated tau induced by 27-hydroxycholesterol in rabbit organotypic slices
    • Marwarha G., et al. Leptin reduces the accumulation of Abeta and phosphorylated tau induced by 27-hydroxycholesterol in rabbit organotypic slices. J. Alzheimers Dis. 2010, 19:1007-1019.
    • (2010) J. Alzheimers Dis. , vol.19 , pp. 1007-1019
    • Marwarha, G.1
  • 58
    • 84868141733 scopus 로고    scopus 로고
    • Leptin prevents hippocampal synaptic disruption and neuronal cell death induced by amyloid beta
    • Doherty G.H., et al. Leptin prevents hippocampal synaptic disruption and neuronal cell death induced by amyloid beta. Neurobiol. Aging 2013, 34:226-237.
    • (2013) Neurobiol. Aging , vol.34 , pp. 226-237
    • Doherty, G.H.1
  • 59
    • 84924041624 scopus 로고    scopus 로고
    • Leptin and the brain: influences on brain development, cognitive functioning and psychiatric disorders
    • Farr O.M., et al. Leptin and the brain: influences on brain development, cognitive functioning and psychiatric disorders. Metab. Clin. Exp. 2015, 64:114-130.
    • (2015) Metab. Clin. Exp. , vol.64 , pp. 114-130
    • Farr, O.M.1
  • 60
    • 84896732087 scopus 로고    scopus 로고
    • Leptin gene therapy attenuates neuronal damages evoked by amyloid-beta and rescues memory deficits in APP/PS1 mice
    • Perez-Gonzalez R., et al. Leptin gene therapy attenuates neuronal damages evoked by amyloid-beta and rescues memory deficits in APP/PS1 mice. Gene Ther. 2014, 21:298-308.
    • (2014) Gene Ther. , vol.21 , pp. 298-308
    • Perez-Gonzalez, R.1
  • 61
    • 77954203843 scopus 로고    scopus 로고
    • Reelin-mediated signaling in neuropsychiatric and neurodegenerative diseases
    • Knuesel I., et al. Reelin-mediated signaling in neuropsychiatric and neurodegenerative diseases. Prog. Neurobiol. 2010, 91:257-274.
    • (2010) Prog. Neurobiol. , vol.91 , pp. 257-274
    • Knuesel, I.1
  • 62
    • 62649098662 scopus 로고    scopus 로고
    • Age-related accumulation of Reelin in amyloid-like deposits
    • Knuesel I., et al. Age-related accumulation of Reelin in amyloid-like deposits. Neurobiol. Aging 2009, 30:697-716.
    • (2009) Neurobiol. Aging , vol.30 , pp. 697-716
    • Knuesel, I.1
  • 63
    • 77954517681 scopus 로고    scopus 로고
    • Reduced Reelin expression accelerates amyloid-beta plaque formation and tau pathology in transgenic Alzheimer's disease mice
    • Kocherhans S., et al. Reduced Reelin expression accelerates amyloid-beta plaque formation and tau pathology in transgenic Alzheimer's disease mice. J. Neurosci. 2010, 30:9228-9240.
    • (2010) J. Neurosci. , vol.30 , pp. 9228-9240
    • Kocherhans, S.1
  • 64
    • 84862980208 scopus 로고    scopus 로고
    • Reelin depletion is an early phenomenon of Alzheimer's pathology
    • Herring A., et al. Reelin depletion is an early phenomenon of Alzheimer's pathology. J. Alzheimers Dis. 2012, 30:963-979.
    • (2012) J. Alzheimers Dis. , vol.30 , pp. 963-979
    • Herring, A.1
  • 65
    • 84866483826 scopus 로고    scopus 로고
    • F-spondin gene transfer improves memory performance and reduces amyloid-beta levels in mice
    • Hafez D.M., et al. F-spondin gene transfer improves memory performance and reduces amyloid-beta levels in mice. Neuroscience 2012, 223:465-472.
    • (2012) Neuroscience , vol.223 , pp. 465-472
    • Hafez, D.M.1
  • 66
    • 11144353586 scopus 로고    scopus 로고
    • ABAD directly links Abeta to mitochondrial toxicity in Alzheimer's disease
    • Lustbader J.W., et al. ABAD directly links Abeta to mitochondrial toxicity in Alzheimer's disease. Science 2004, 304:448-452.
    • (2004) Science , vol.304 , pp. 448-452
    • Lustbader, J.W.1
  • 67
    • 84919631298 scopus 로고    scopus 로고
    • Caspase-3 short hairpin RNAs: a potential therapeutic agent in neurodegeneration of aluminum-exposed animal model
    • Zhang Q., et al. Caspase-3 short hairpin RNAs: a potential therapeutic agent in neurodegeneration of aluminum-exposed animal model. Curr. Alzheimer Res. 2014, 11:961-970.
    • (2014) Curr. Alzheimer Res. , vol.11 , pp. 961-970
    • Zhang, Q.1
  • 68
    • 84904265385 scopus 로고    scopus 로고
    • A viral vector expressing hypoxia-inducible factor 1 alpha inhibits hippocampal neuronal apoptosis
    • Chai X., et al. A viral vector expressing hypoxia-inducible factor 1 alpha inhibits hippocampal neuronal apoptosis. Neural Regen. Res. 2014, 9:1145-1153.
    • (2014) Neural Regen. Res. , vol.9 , pp. 1145-1153
    • Chai, X.1
  • 69
    • 84911982234 scopus 로고    scopus 로고
    • Lenti-GDNF gene therapy protects against Alzheimer's disease-like neuropathology in 3×Tg-AD mice and MC65 cells
    • Revilla S., et al. Lenti-GDNF gene therapy protects against Alzheimer's disease-like neuropathology in 3×Tg-AD mice and MC65 cells. CNS Neurosci. Therapeutics 2014, 20:961-972.
    • (2014) CNS Neurosci. Therapeutics , vol.20 , pp. 961-972
    • Revilla, S.1
  • 70
    • 84908135739 scopus 로고    scopus 로고
    • Insulin-like growth factor 2 reverses memory and synaptic deficits in APP transgenic mice
    • Pascual-Lucas M., et al. Insulin-like growth factor 2 reverses memory and synaptic deficits in APP transgenic mice. EMBO Mol. Med. 2014, 6:1246-1262.
    • (2014) EMBO Mol. Med. , vol.6 , pp. 1246-1262
    • Pascual-Lucas, M.1
  • 71
    • 84930573237 scopus 로고    scopus 로고
    • Spinal muscular atrophy - recent therapeutic advances for an old challenge
    • Published online May 19, 2015
    • Faravelli I., et al. Spinal muscular atrophy - recent therapeutic advances for an old challenge. Nat. Rev. Neurol. 2015, Published online May 19, 2015. 10.1038/nrneurol.2015.77.
    • (2015) Nat. Rev. Neurol.
    • Faravelli, I.1
  • 72
    • 84867326136 scopus 로고    scopus 로고
    • Update on gene and stem cell therapy approaches for spinal muscular atrophy
    • Donnelly E.M., Boulis N.M. Update on gene and stem cell therapy approaches for spinal muscular atrophy. Expert Opin. Biol. Ther. 2012, 12:1463-1471.
    • (2012) Expert Opin. Biol. Ther. , vol.12 , pp. 1463-1471
    • Donnelly, E.M.1    Boulis, N.M.2
  • 73
    • 84904412918 scopus 로고    scopus 로고
    • Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy
    • Passini M.A., et al. Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy. Hum. Gene Ther. 2014, 25:619-630.
    • (2014) Hum. Gene Ther. , vol.25 , pp. 619-630
    • Passini, M.A.1
  • 74
    • 84924066675 scopus 로고    scopus 로고
    • Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates
    • Meyer K., et al. Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates. Mol. Ther. 2015, 23:477-487.
    • (2015) Mol. Ther. , vol.23 , pp. 477-487
    • Meyer, K.1
  • 75
    • 77749249680 scopus 로고    scopus 로고
    • Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
    • Foust K.D., et al. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat. Biotechnol. 2010, 28:271-274.
    • (2010) Nat. Biotechnol. , vol.28 , pp. 271-274
    • Foust, K.D.1
  • 76
    • 77957741150 scopus 로고    scopus 로고
    • Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery
    • Bevan A.K., et al. Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery. Hum. Mol. Genet. 2010, 19:3895-3905.
    • (2010) Hum. Mol. Genet. , vol.19 , pp. 3895-3905
    • Bevan, A.K.1
  • 77
    • 84923771867 scopus 로고    scopus 로고
    • A large animal model of spinal muscular atrophy and correction of phenotype
    • Duque S.I., et al. A large animal model of spinal muscular atrophy and correction of phenotype. Ann. Neurol. 2015, 77:399-414.
    • (2015) Ann. Neurol. , vol.77 , pp. 399-414
    • Duque, S.I.1
  • 78
    • 79952348568 scopus 로고    scopus 로고
    • Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy
    • 72ra18
    • Passini M.A., et al. Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy. Sci. Transl. Med. 2011, 3:72ra18.
    • (2011) Sci. Transl. Med. , vol.3
    • Passini, M.A.1
  • 79
    • 84924527302 scopus 로고    scopus 로고
    • Rescue of gene-expression changes in an induced mouse model of spinal muscular atrophy by an antisense oligonucleotide that promotes inclusion of SMN2 exon 7
    • Staropoli J.F., et al. Rescue of gene-expression changes in an induced mouse model of spinal muscular atrophy by an antisense oligonucleotide that promotes inclusion of SMN2 exon 7. Genomics 2015, 105:220-228.
    • (2015) Genomics , vol.105 , pp. 220-228
    • Staropoli, J.F.1
  • 80
    • 84886425853 scopus 로고    scopus 로고
    • Clinical neurogenetics: autosomal dominant spinocerebellar ataxia
    • Shakkottai V.G., Fogel B.L. Clinical neurogenetics: autosomal dominant spinocerebellar ataxia. Neurol. Clin. 2013, 31:987-1007.
    • (2013) Neurol. Clin. , vol.31 , pp. 987-1007
    • Shakkottai, V.G.1    Fogel, B.L.2
  • 81
    • 84872774379 scopus 로고    scopus 로고
    • Silencing mutant ataxin-3 rescues motor deficits and neuropathology in Machado-Joseph disease transgenic mice
    • Nobrega C., et al. Silencing mutant ataxin-3 rescues motor deficits and neuropathology in Machado-Joseph disease transgenic mice. PLoS ONE 2013, 8:e52396.
    • (2013) PLoS ONE , vol.8 , pp. e52396
    • Nobrega, C.1
  • 82
    • 84885023525 scopus 로고    scopus 로고
    • Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice
    • Rodriguez-Lebron E., et al. Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice. Mol. Ther. 2013, 21:1909-1918.
    • (2013) Mol. Ther. , vol.21 , pp. 1909-1918
    • Rodriguez-Lebron, E.1
  • 83
    • 77955290123 scopus 로고    scopus 로고
    • Silencing ataxin-3 mitigates degeneration in a rat model of Machado-Joseph disease: no role for wild-type ataxin-3?
    • Alves S., et al. Silencing ataxin-3 mitigates degeneration in a rat model of Machado-Joseph disease: no role for wild-type ataxin-3?. Hum. Mol. Genet. 2010, 19:2380-2394.
    • (2010) Hum. Mol. Genet. , vol.19 , pp. 2380-2394
    • Alves, S.1
  • 84
    • 84895468010 scopus 로고    scopus 로고
    • Broad therapeutic benefit after RNAi expression vector delivery to deep cerebellar nuclei: implications for spinocerebellar ataxia type 1 therapy
    • Keiser M.S., et al. Broad therapeutic benefit after RNAi expression vector delivery to deep cerebellar nuclei: implications for spinocerebellar ataxia type 1 therapy. Mol. Ther. 2014, 22:588-595.
    • (2014) Mol. Ther. , vol.22 , pp. 588-595
    • Keiser, M.S.1
  • 85
    • 84899769824 scopus 로고    scopus 로고
    • RNA interference-based therapy for spinocerebellar ataxia type 7 retinal degeneration
    • Ramachandran P.S., et al. RNA interference-based therapy for spinocerebellar ataxia type 7 retinal degeneration. PLoS ONE 2014, 9:e95362.
    • (2014) PLoS ONE , vol.9 , pp. e95362
    • Ramachandran, P.S.1
  • 86
    • 84965083268 scopus 로고    scopus 로고
    • A review of gene therapy in canine and feline models of lysosomal storage disorders
    • Bradbury A.M., et al. A review of gene therapy in canine and feline models of lysosomal storage disorders. Hum. Gene Ther. Clin. Dev. 2015, 26:27-37.
    • (2015) Hum. Gene Ther. Clin. Dev. , vol.26 , pp. 27-37
    • Bradbury, A.M.1
  • 87
    • 84929508366 scopus 로고    scopus 로고
    • Non-neuronopathic lysosomal storage disorders: disease spectrum and treatments
    • Pastores G.M., Hughes D.A. Non-neuronopathic lysosomal storage disorders: disease spectrum and treatments. Best Pract. Res. Clin. Endocrinol. Metab. 2015, 29:173-182.
    • (2015) Best Pract. Res. Clin. Endocrinol. Metab. , vol.29 , pp. 173-182
    • Pastores, G.M.1    Hughes, D.A.2
  • 88
    • 71649099089 scopus 로고    scopus 로고
    • Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants
    • Kishnani P.S., et al. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. Mol. Genet. Metab. 2010, 99:26-33.
    • (2010) Mol. Genet. Metab. , vol.99 , pp. 26-33
    • Kishnani, P.S.1
  • 89
    • 33847206112 scopus 로고    scopus 로고
    • Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors
    • Mah C., et al. Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors. Mol. Ther. 2007, 15:501-507.
    • (2007) Mol. Ther. , vol.15 , pp. 501-507
    • Mah, C.1
  • 90
    • 77649271590 scopus 로고    scopus 로고
    • Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease
    • Mah C.S., et al. Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease. Mol. Ther. 2010, 18:502-510.
    • (2010) Mol. Ther. , vol.18 , pp. 502-510
    • Mah, C.S.1
  • 91
    • 84879161464 scopus 로고    scopus 로고
    • Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes
    • Smith B.K., et al. Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes. Hum. Gene Ther. 2013, 24:630-640.
    • (2013) Hum. Gene Ther. , vol.24 , pp. 630-640
    • Smith, B.K.1
  • 92
    • 84897573702 scopus 로고    scopus 로고
    • Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice
    • Elmallah M.K., et al. Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice. Mol. Ther. 2014, 22:702-712.
    • (2014) Mol. Ther. , vol.22 , pp. 702-712
    • Elmallah, M.K.1
  • 93
    • 84883743826 scopus 로고    scopus 로고
    • Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease
    • Falk D.J., et al. Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease. Mol. Ther. 2013, 21:1661-1667.
    • (2013) Mol. Ther. , vol.21 , pp. 1661-1667
    • Falk, D.J.1
  • 94
    • 84856950003 scopus 로고    scopus 로고
    • Spinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice
    • Qiu K., et al. Spinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice. Mol. Ther. 2012, 20:21-27.
    • (2012) Mol. Ther. , vol.20 , pp. 21-27
    • Qiu, K.1
  • 95
    • 84921448380 scopus 로고    scopus 로고
    • Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade
    • Han S.O., et al. Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade. Hum. Gene Ther. 2015, 26:26-35.
    • (2015) Hum. Gene Ther. , vol.26 , pp. 26-35
    • Han, S.O.1
  • 96
    • 84929507063 scopus 로고    scopus 로고
    • Metachromatic leukodystrophy: disease spectrum and approaches for treatment
    • van Rappard D.F., et al. Metachromatic leukodystrophy: disease spectrum and approaches for treatment. Best Pract. Res. Clin. Endocrinol. Metab. 2015, 29:261-273.
    • (2015) Best Pract. Res. Clin. Endocrinol. Metab. , vol.29 , pp. 261-273
    • van Rappard, D.F.1
  • 97
    • 84865270609 scopus 로고    scopus 로고
    • Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice
    • Piguet F., et al. Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice. Hum. Gene Ther. 2012, 23:903-914.
    • (2012) Hum. Gene Ther. , vol.23 , pp. 903-914
    • Piguet, F.1
  • 98
    • 84897577867 scopus 로고    scopus 로고
    • Long-term correction of biochemical and neurological abnormalities in MLD mice model by neonatal systemic injection of an AAV serotype 9 vector
    • Miyake N., et al. Long-term correction of biochemical and neurological abnormalities in MLD mice model by neonatal systemic injection of an AAV serotype 9 vector. Gene Ther. 2014, 21:427-433.
    • (2014) Gene Ther. , vol.21 , pp. 427-433
    • Miyake, N.1
  • 99
    • 78650905961 scopus 로고    scopus 로고
    • Mucopolysaccharidosis type IIIA: clinical spectrum and genotype-phenotype correlations
    • Valstar M.J., et al. Mucopolysaccharidosis type IIIA: clinical spectrum and genotype-phenotype correlations. Ann. Neurol. 2010, 68:876-887.
    • (2010) Ann. Neurol. , vol.68 , pp. 876-887
    • Valstar, M.J.1
  • 100
    • 84902952909 scopus 로고    scopus 로고
    • Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial
    • Tardieu M., et al. Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial. Hum. Gene Ther. 2014, 25:506-516.
    • (2014) Hum. Gene Ther. , vol.25 , pp. 506-516
    • Tardieu, M.1
  • 101
    • 84927081397 scopus 로고    scopus 로고
    • Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice
    • Duncan F.J., et al. Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice. Mol. Ther. 2015, 23:638-647.
    • (2015) Mol. Ther. , vol.23 , pp. 638-647
    • Duncan, F.J.1
  • 102
    • 84881228888 scopus 로고    scopus 로고
    • Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
    • Haurigot V., et al. Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy. J. Clin. Invest. 2013, 123:3254-3271.
    • (2013) J. Clin. Invest. , vol.123 , pp. 3254-3271
    • Haurigot, V.1
  • 103
    • 84871243601 scopus 로고    scopus 로고
    • Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer
    • Ruzo A., et al. Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer. Hum. Gene Ther. 2012, 23:1237-1246.
    • (2012) Hum. Gene Ther. , vol.23 , pp. 1237-1246
    • Ruzo, A.1
  • 104
    • 77950521638 scopus 로고    scopus 로고
    • Identification and characterization of a novel homozygous deletion in the alpha-N-acetylglucosaminidase gene in a patient with Sanfilippo type B syndrome (mucopolysaccharidosis IIIB)
    • Champion K.J., et al. Identification and characterization of a novel homozygous deletion in the alpha-N-acetylglucosaminidase gene in a patient with Sanfilippo type B syndrome (mucopolysaccharidosis IIIB). Mol. Genet. Metab. 2010, 100:51-56.
    • (2010) Mol. Genet. Metab. , vol.100 , pp. 51-56
    • Champion, K.J.1
  • 105
    • 79957882063 scopus 로고    scopus 로고
    • Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery
    • Fu H., et al. Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery. Mol. Ther. 2011, 19:1025-1033.
    • (2011) Mol. Ther. , vol.19 , pp. 1025-1033
    • Fu, H.1
  • 106
    • 84926453574 scopus 로고    scopus 로고
    • Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy
    • Ribera A., et al. Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy. Hum. Mol. Genet. 2015, 24:2078-2095.
    • (2015) Hum. Mol. Genet. , vol.24 , pp. 2078-2095
    • Ribera, A.1
  • 107
    • 84902676801 scopus 로고    scopus 로고
    • Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates
    • Murrey D.A., et al. Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates. Hum. Gene Ther. Clin. Dev. 2014, 25:72-84.
    • (2014) Hum. Gene Ther. Clin. Dev. , vol.25 , pp. 72-84
    • Murrey, D.A.1
  • 108
    • 0035905889 scopus 로고    scopus 로고
    • Enzyme-replacement therapy in mucopolysaccharidosis I
    • Kakkis E.D., et al. Enzyme-replacement therapy in mucopolysaccharidosis I. N. Engl. J. Med. 2001, 344:182-188.
    • (2001) N. Engl. J. Med. , vol.344 , pp. 182-188
    • Kakkis, E.D.1
  • 109
    • 84865976292 scopus 로고    scopus 로고
    • Increased longevity and metabolic correction following syngeneic BMT in a murine model of mucopolysaccharidosis type I
    • Wolf D.A., et al. Increased longevity and metabolic correction following syngeneic BMT in a murine model of mucopolysaccharidosis type I. Bone Marrow Transplant. 2012, 47:1235-1240.
    • (2012) Bone Marrow Transplant. , vol.47 , pp. 1235-1240
    • Wolf, D.A.1
  • 110
    • 84891829278 scopus 로고    scopus 로고
    • Comparison of endovascular and intraventricular gene therapy with adeno-associated virus-alpha-l-iduronidase for Hurler disease
    • Janson C.G., et al. Comparison of endovascular and intraventricular gene therapy with adeno-associated virus-alpha-l-iduronidase for Hurler disease. Neurosurgery 2014, 74:99-111.
    • (2014) Neurosurgery , vol.74 , pp. 99-111
    • Janson, C.G.1
  • 111
    • 84927155708 scopus 로고    scopus 로고
    • Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I
    • Hinderer C., et al. Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I. Mol. Ther. 2014, 22:2018-2027.
    • (2014) Mol. Ther. , vol.22 , pp. 2018-2027
    • Hinderer, C.1
  • 112
    • 33645864671 scopus 로고    scopus 로고
    • Influence of caloric restriction on motor behavior, longevity, and brain lipid composition in Sandhoff disease mice
    • Denny C.A., et al. Influence of caloric restriction on motor behavior, longevity, and brain lipid composition in Sandhoff disease mice. J. Neurosci. Res. 2006, 83:1028-1038.
    • (2006) J. Neurosci. Res. , vol.83 , pp. 1028-1038
    • Denny, C.A.1
  • 113
    • 80054916282 scopus 로고    scopus 로고
    • Adeno-associated virus-mediated expression of beta-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain
    • Sargeant T.J., et al. Adeno-associated virus-mediated expression of beta-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain. Hum. Mol. Genet. 2011, 20:4371-4380.
    • (2011) Hum. Mol. Genet. , vol.20 , pp. 4371-4380
    • Sargeant, T.J.1
  • 114
    • 84977515780 scopus 로고    scopus 로고
    • AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system
    • Published online April 13, 2015
    • Rockwell H.E., et al. AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system. ASN Neuro. 2015, 7. Published online April 13, 2015. 10.1177/1759091415569908.
    • (2015) ASN Neuro. , vol.7
    • Rockwell, H.E.1
  • 115
    • 84922409352 scopus 로고    scopus 로고
    • Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease
    • McCurdy V.J., et al. Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease. Gene Ther. 2015, 22:181-189.
    • (2015) Gene Ther. , vol.22 , pp. 181-189
    • McCurdy, V.J.1
  • 116
    • 84879086501 scopus 로고    scopus 로고
    • Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients
    • Bartus R.T., et al. Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients. Neurology 2013, 80:1698-1701.
    • (2013) Neurology , vol.80 , pp. 1698-1701
    • Bartus, R.T.1
  • 117
    • 60149106907 scopus 로고    scopus 로고
    • Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
    • Foust K.D., et al. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat. Biotechnol. 2009, 27:59-65.
    • (2009) Nat. Biotechnol. , vol.27 , pp. 59-65
    • Foust, K.D.1
  • 118
    • 84920911585 scopus 로고    scopus 로고
    • Biomarkers in the diagnosis and management of Alzheimer's disease
    • Wurtman R. Biomarkers in the diagnosis and management of Alzheimer's disease. Metab. Clin. Exp. 2015, 64(Suppl. 1):S47-S50.
    • (2015) Metab. Clin. Exp. , vol.64 , pp. S47-S50
    • Wurtman, R.1
  • 119
    • 84926176151 scopus 로고    scopus 로고
    • Use of biomarkers in ALS drug development and clinical trials
    • Bakkar N., et al. Use of biomarkers in ALS drug development and clinical trials. Brain Res. 2015, 1607:94-107.
    • (2015) Brain Res. , vol.1607 , pp. 94-107
    • Bakkar, N.1
  • 120
    • 84937758712 scopus 로고    scopus 로고
    • Amyloid biomarkers in Alzheimer's disease
    • Blennow K., et al. Amyloid biomarkers in Alzheimer's disease. Trends Pharmacol. Sci. 2015, 36:297-309.
    • (2015) Trends Pharmacol. Sci. , vol.36 , pp. 297-309
    • Blennow, K.1
  • 121
    • 84908351208 scopus 로고    scopus 로고
    • Cerebrospinal fluid biomarkers in parkinsonian conditions: an update and future directions
    • Magdalinou N., et al. Cerebrospinal fluid biomarkers in parkinsonian conditions: an update and future directions. J. Neurol. Neurosurg. Psychiatry 2014, 85:1065-1075.
    • (2014) J. Neurol. Neurosurg. Psychiatry , vol.85 , pp. 1065-1075
    • Magdalinou, N.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.