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Volumn 11, Issue 5, 2005, Pages 745-753

Restoration of aspartoacylase activity in CNS neurons does not ameliorate motor deficits and demyelination in a model of Canavan disease

Author keywords

Adeno associated virus; Aspartoacylase; Canavan disease; Demyelination; Epilepsy; Gene therapy; Leukodystrophy; N acetylaspartate; Oligodendrocyte

Indexed keywords

ASPARTOACYLASE; CAPSID PROTEIN; N ACETYLASPARTIC ACID;

EID: 17644419965     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2005.01.006     Document Type: Article
Times cited : (47)

References (43)
  • 1
    • 0032919537 scopus 로고    scopus 로고
    • Biochemistry and molecular biology of Canavan disease
    • R. Matalon and K. Michals-Matalon Biochemistry and molecular biology of Canavan disease Neurochem. Res. 24 1999 507-513
    • (1999) Neurochem. Res. , vol.24 , pp. 507-513
    • Matalon, R.1    Michals-Matalon, K.2
  • 2
    • 0000824069 scopus 로고
    • Spongy degeneration of the central nervous system in infancy
    • B.Q. Banker J.T. Robertson and M. Victor Spongy degeneration of the central nervous system in infancy Neurology 14 1964 981
    • (1964) Neurology , vol.14 , pp. 981
    • Banker, B.Q.1    Robertson, J.T.2    Victor, M.3
  • 3
    • 0027362434 scopus 로고
    • Cloning of the human aspartoacylase cDNA and a common missense mutation in Canavan disease
    • R. Kaul G.P. Gao K. Balamurugan and R. Matalon Cloning of the human aspartoacylase cDNA and a common missense mutation in Canavan disease Nat. Genet. 5 1993 118-123
    • (1993) Nat. Genet. , vol.5 , pp. 118-123
    • Kaul, R.1    Gao, G.P.2    Balamurugan, K.3    Matalon, R.4
  • 4
    • 0034077081 scopus 로고    scopus 로고
    • Accumulation of N-acetyl-l-aspartate in the brain of the tremor rat, a mutant exhibiting absence-like seizure and spongiform degeneration in the central nervous system
    • K. Kitada et al. Accumulation of N-acetyl-l-aspartate in the brain of the tremor rat, a mutant exhibiting absence-like seizure and spongiform degeneration in the central nervous system J. Neurochem. 74 2000 2512-2519
    • (2000) J. Neurochem. , vol.74 , pp. 2512-2519
    • Kitada, K.1
  • 6
    • 0023088586 scopus 로고
    • A new model of petit mal epilepsy: Spontaneous spike and wave discharges in tremor rats
    • T. Serikawa Y. Ohno M. Sasa J. Yamada and S. Takaori A new model of petit mal epilepsy: Spontaneous spike and wave discharges in tremor rats Lab. Anim. 21 1987 68-71
    • (1987) Lab. Anim. , vol.21 , pp. 68-71
    • Serikawa, T.1    Ohno, Y.2    Sasa, M.3    Yamada, J.4    Takaori, S.5
  • 7
    • 0029086297 scopus 로고
    • Effect of antiepileptic drugs on absence-like seizures in the tremor rat
    • R. Hanaya et al. Effect of antiepileptic drugs on absence-like seizures in the tremor rat Epilepsia 36 1995 938-942
    • (1995) Epilepsia , vol.36 , pp. 938-942
    • Hanaya, R.1
  • 8
    • 0037409849 scopus 로고    scopus 로고
    • N-Acetylaspartate in the vertebrate brain: Metabolism and function
    • M.H. Baslow N-Acetylaspartate in the vertebrate brain: Metabolism and function Neurochem. Res. 28 2003 941-953
    • (2003) Neurochem. Res. , vol.28 , pp. 941-953
    • Baslow, M.H.1
  • 9
    • 0034890751 scopus 로고    scopus 로고
    • Intraneuronal N-acetylaspartate supplies acetyl groups for myelin lipid synthesis: Evidence for myelin-associated aspartoacylase
    • G. Chakraborty P. Mekala D. Yahya G. Wu and R.W. Ledeen Intraneuronal N-acetylaspartate supplies acetyl groups for myelin lipid synthesis: evidence for myelin-associated aspartoacylase J. Neurochem. 78 2001 736-745
    • (2001) J. Neurochem. , vol.78 , pp. 736-745
    • Chakraborty, G.1    Mekala, P.2    Yahya, D.3    Wu, G.4    Ledeen, R.W.5
  • 10
    • 1342285559 scopus 로고    scopus 로고
    • N-Acetylaspartate synthase is bimodally expressed in microsomes and mitochondria of brain
    • Z.H. Lu G. Chakraborty R.W. Ledeen D. Yahya and G. Wu N-Acetylaspartate synthase is bimodally expressed in microsomes and mitochondria of brain Brain Res. Mol. Brain Res. 122 2004 71-78
    • (2004) Brain Res. Mol. Brain Res. , vol.122 , pp. 71-78
    • Lu, Z.H.1    Chakraborty, G.2    Ledeen, R.W.3    Yahya, D.4    Wu, G.5
  • 12
    • 0242643637 scopus 로고    scopus 로고
    • Identification and distribution of aspartoacylase in the postnatal rat brain
    • M. Klugmann et al. Identification and distribution of aspartoacylase in the postnatal rat brain Neuroreport 14 2003 1837-1840
    • (2003) Neuroreport , vol.14 , pp. 1837-1840
    • Klugmann, M.1
  • 13
    • 0033917280 scopus 로고    scopus 로고
    • Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease
    • P. Leone et al. Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease Ann. Neurol. 48 2000 27-38
    • (2000) Ann. Neurol. , vol.48 , pp. 27-38
    • Leone, P.1
  • 14
    • 0035576439 scopus 로고    scopus 로고
    • Viral-based gene transfer to the mammalian CNS for functional genomic studies
    • C.G. Janson S.W. McPhee P. Leone A. Freese and M.J. During Viral-based gene transfer to the mammalian CNS for functional genomic studies Trends Neurosci. 24 2001 706-712
    • (2001) Trends Neurosci. , vol.24 , pp. 706-712
    • Janson, C.G.1    McPhee, S.W.2    Leone, P.3    Freese, A.4    During, M.J.5
  • 15
    • 0037143294 scopus 로고    scopus 로고
    • Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain
    • C. Janson et al. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain Hum. Gene Ther. 13 2002 1391-1412
    • (2002) Hum. Gene Ther. , vol.13 , pp. 1391-1412
    • Janson, C.1
  • 16
    • 0038529698 scopus 로고    scopus 로고
    • Adeno-associated virus-mediated aspartoacylase gene transfer to the brain of knockout mouse for Canavan disease
    • R. Matalon et al. Adeno-associated virus-mediated aspartoacylase gene transfer to the brain of knockout mouse for Canavan disease Mol. Ther. 7 2003 580-587
    • (2003) Mol. Ther. , vol.7 , pp. 580-587
    • Matalon, R.1
  • 17
    • 0034724209 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system
    • B.L. Davidson et al. Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system Proc. Natl. Acad. Sci. USA 97 2000 3428-3432
    • (2000) Proc. Natl. Acad. Sci. USA , vol.97 , pp. 3428-3432
    • Davidson, B.L.1
  • 18
    • 0036225913 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus serotypes 2- and 5-mediated gene transfer in the mammalian brain: Quantitative analysis of heparin co-infusion
    • M.Y. Mastakov K. Baer R.M. Kotin and M.J. During Recombinant adeno-associated virus serotypes 2- and 5-mediated gene transfer in the mammalian brain: Quantitative analysis of heparin co-infusion Mol. Ther. 5 2002 371-380
    • (2002) Mol. Ther. , vol.5 , pp. 371-380
    • Mastakov, M.Y.1    Baer, K.2    Kotin, R.M.3    During, M.J.4
  • 19
    • 0042525989 scopus 로고    scopus 로고
    • Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain
    • C. Wang C.M. Wang K.R. Clark and T.J. Sferra Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain Gene Ther. 10 2003 1528-1534
    • (2003) Gene Ther. , vol.10 , pp. 1528-1534
    • Wang, C.1    Wang, C.M.2    Clark, K.R.3    Sferra, T.J.4
  • 20
    • 0037119206 scopus 로고    scopus 로고
    • Adenoviral gene transfer of aspartoacylase into the tremor rat, a genetic model of epilepsy, as a trial of gene therapy for inherited epileptic disorder
    • T. Seki et al. Adenoviral gene transfer of aspartoacylase into the tremor rat, a genetic model of epilepsy, as a trial of gene therapy for inherited epileptic disorder Neurosci. Lett. 328 2002 249-252
    • (2002) Neurosci. Lett. , vol.328 , pp. 249-252
    • Seki, T.1
  • 21
    • 12144291522 scopus 로고    scopus 로고
    • Anticonvulsant and antiepileptogenic effects mediated by adeno-associated virus vector neuropeptide Y expression in the rat hippocampus
    • C. Richichi et al. Anticonvulsant and antiepileptogenic effects mediated by adeno-associated virus vector neuropeptide Y expression in the rat hippocampus J. Neurosci. 24 2004 3051-3059
    • (2004) J. Neurosci. , vol.24 , pp. 3051-3059
    • Richichi, C.1
  • 22
    • 0037368410 scopus 로고    scopus 로고
    • Generation and characterization of chimeric recombinant AAV vectors
    • B. Hauck L. Chen and W. Xiao Generation and characterization of chimeric recombinant AAV vectors Mol. Ther. 7 2003 419-425
    • (2003) Mol. Ther. , vol.7 , pp. 419-425
    • Hauck, B.1    Chen, L.2    Xiao, W.3
  • 23
    • 4344566457 scopus 로고    scopus 로고
    • Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system
    • C. Burger et al. Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system Mol. Ther. 10 2004 302-317
    • (2004) Mol. Ther. , vol.10 , pp. 302-317
    • Burger, C.1
  • 24
    • 17944378442 scopus 로고    scopus 로고
    • Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes
    • R. Xu et al. Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes Gene Ther. 8 2001 1323-1332
    • (2001) Gene Ther. , vol.8 , pp. 1323-1332
    • Xu, R.1
  • 25
    • 0036162622 scopus 로고    scopus 로고
    • Evidence supporting a role for N-acetyl-l-aspartate as a molecular water pump in myelinated neurons in the central nervous system: An analytical review
    • M.H. Baslow Evidence supporting a role for N-acetyl-l-aspartate as a molecular water pump in myelinated neurons in the central nervous system: An analytical review Neurochem. Int. 40 2002 295-300
    • (2002) Neurochem. Int. , vol.40 , pp. 295-300
    • Baslow, M.H.1
  • 26
    • 0034616077 scopus 로고    scopus 로고
    • Epileptic seizures induced by N-acetyl-l-aspartate in rats: In vivo and in vitro studies
    • T. Akimitsu et al. Epileptic seizures induced by N-acetyl-l-aspartate in rats: In vivo and in vitro studies Brain Res. 861 2000 143-150
    • (2000) Brain Res. , vol.861 , pp. 143-150
    • Akimitsu, T.1
  • 27
    • 0025728364 scopus 로고
    • CNS pathology in the neurological mutant rats zitter, tremor and zitter-tremor double mutant (spontaneously epileptic rat, SER)
    • A. Kondo H. Nagara K. Akazawa J. Tateishi T. Serikawa and J. Yamada CNS pathology in the neurological mutant rats zitter, tremor and zitter-tremor double mutant (spontaneously epileptic rat, SER) Brain 114 1991 979-999
    • (1991) Brain , vol.114 , pp. 979-999
    • Kondo, A.1    Nagara, H.2    Akazawa, K.3    Tateishi, J.4    Serikawa, T.5    Yamada, J.6
  • 28
    • 0015857284 scopus 로고
    • Myelination in rat brain: Method of myelin isolation
    • W.T. Norton and S.E. Poduslo Myelination in rat brain: Method of myelin isolation J. Neurochem. 21 1973 749-757
    • (1973) J. Neurochem. , vol.21 , pp. 749-757
    • Norton, W.T.1    Poduslo, S.E.2
  • 29
    • 0024985260 scopus 로고
    • Delayed postnatal behavioral development in spontaneously epileptic rats and tremor rats, and poor operant performance in spontaneously epileptic rats
    • Y. Asano A. Okaniwa T. Serikawa and J. Yamada Delayed postnatal behavioral development in spontaneously epileptic rats and tremor rats, and poor operant performance in spontaneously epileptic rats Jikken Dobutsu 39 1990 69-74
    • (1990) Jikken Dobutsu , vol.39 , pp. 69-74
    • Asano, Y.1    Okaniwa, A.2    Serikawa, T.3    Yamada, J.4
  • 30
    • 0032824161 scopus 로고    scopus 로고
    • Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease
    • P. Leone C.G. Janson S.J. McPhee and M.J. During Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease Curr. Opin. Mol. Ther. 1 1999 487-492
    • (1999) Curr. Opin. Mol. Ther. , vol.1 , pp. 487-492
    • Leone, P.1    Janson, C.G.2    McPhee, S.J.3    During, M.J.4
  • 31
    • 0036428796 scopus 로고    scopus 로고
    • Promoters and regulatory elements that improve adeno-associated virus transgene expression in the brain
    • H.L. Fitzsimons R.J. Bland and M.J. During Promoters and regulatory elements that improve adeno-associated virus transgene expression in the brain Methods 28 2002 227-236
    • (2002) Methods , vol.28 , pp. 227-236
    • Fitzsimons, H.L.1    Bland, R.J.2    During, M.J.3
  • 32
    • 0036202298 scopus 로고    scopus 로고
    • Critical issues in gene therapy for neurologic disease
    • G. Hsich M. Sena-Esteves and X.O. Breakefield Critical issues in gene therapy for neurologic disease Hum. Gene Ther. 13 2002 579-604
    • (2002) Hum. Gene Ther. , vol.13 , pp. 579-604
    • Hsich, G.1    Sena-Esteves, M.2    Breakefield, X.O.3
  • 33
    • 0026633564 scopus 로고
    • Specific expression of N-acetylaspartate in neurons, oligodendrocyte-type-2 astrocyte progenitors, and immature oligodendrocytes in vitro
    • J. Urenjak S.R. Williams D.G. Gadian and M. Noble Specific expression of N-acetylaspartate in neurons, oligodendrocyte-type-2 astrocyte progenitors, and immature oligodendrocytes in vitro J. Neurochem. 59 1992 55-61
    • (1992) J. Neurochem. , vol.59 , pp. 55-61
    • Urenjak, J.1    Williams, S.R.2    Gadian, D.G.3    Noble, M.4
  • 34
    • 0027450301 scopus 로고
    • Proton nuclear magnetic resonance spectroscopy unambiguously identifies different neural cell types
    • J. Urenjak S.R. Williams D.G. Gadian and M. Noble Proton nuclear magnetic resonance spectroscopy unambiguously identifies different neural cell types J. Neurosci. 13 1993 981-989
    • (1993) J. Neurosci. , vol.13 , pp. 981-989
    • Urenjak, J.1    Williams, S.R.2    Gadian, D.G.3    Noble, M.4
  • 35
    • 0041413364 scopus 로고    scopus 로고
    • Activation by N-acetyl-l-aspartate of acutely dissociated hippocampal neurons in rats via metabotropic glutamate receptors
    • H.D. Yan K. Ishihara T. Serikawa and M. Sasa Activation by N-acetyl-l-aspartate of acutely dissociated hippocampal neurons in rats via metabotropic glutamate receptors Epilepsia 44 2003 1153-1159
    • (2003) Epilepsia , vol.44 , pp. 1153-1159
    • Yan, H.D.1    Ishihara, K.2    Serikawa, T.3    Sasa, M.4
  • 37
    • 0037370398 scopus 로고    scopus 로고
    • Myelin: Not just a conduit for conduction
    • B. Popko Myelin: Not just a conduit for conduction Nat. Genet. 33 2003 327-328
    • (2003) Nat. Genet. , vol.33 , pp. 327-328
    • Popko, B.1
  • 38
    • 13444292151 scopus 로고    scopus 로고
    • AAV-mediated hippocampal expression of short and long Homer 1 proteins differentially affects cognition and seizure activity in adult rats
    • M. Klugmann et al. AAV-mediated hippocampal expression of short and long Homer 1 proteins differentially affects cognition and seizure activity in adult rats Mol. Cell. Neurosci. 28 2005 343-356
    • (2005) Mol. Cell. Neurosci. , vol.28 , pp. 343-356
    • Klugmann, M.1
  • 39
    • 0034980351 scopus 로고    scopus 로고
    • Combined injection of rAAV with mannitol enhances gene expression in the rat brain
    • M.Y. Mastakov K. Baer R. Xu H. Fitzsimons and M.J. During Combined injection of rAAV with mannitol enhances gene expression in the rat brain Mol. Ther. 3 2001 225-232
    • (2001) Mol. Ther. , vol.3 , pp. 225-232
    • Mastakov, M.Y.1    Baer, K.2    Xu, R.3    Fitzsimons, H.4    During, M.J.5
  • 40
    • 0034790687 scopus 로고    scopus 로고
    • Developmental and regional distribution of aspartoacylase in rat brain tissue
    • K.K. Bhakoo T.J. Craig and P. Styles Developmental and regional distribution of aspartoacylase in rat brain tissue J. Neurochem. 79 2001 211-220
    • (2001) J. Neurochem. , vol.79 , pp. 211-220
    • Bhakoo, K.K.1    Craig, T.J.2    Styles, P.3
  • 41
    • 0032957382 scopus 로고    scopus 로고
    • Environmental enrichment inhibits spontaneous apoptosis, prevents seizures and is neuroprotective
    • D. Young P.A. Lawlor P. Leone M. Dragunow and M.J. During Environmental enrichment inhibits spontaneous apoptosis, prevents seizures and is neuroprotective Nat. Med. 5 1999 448-453
    • (1999) Nat. Med. , vol.5 , pp. 448-453
    • Young, D.1    Lawlor, P.A.2    Leone, P.3    Dragunow, M.4    During, M.J.5
  • 42
    • 0035898261 scopus 로고    scopus 로고
    • Behavioural correlates of striatal glial fibrillary acidic protein in the 3-nitropropionic acid rat model: Disturbed walking pattern and spatial orientation
    • C.E. Teunissen et al. Behavioural correlates of striatal glial fibrillary acidic protein in the 3-nitropropionic acid rat model: disturbed walking pattern and spatial orientation Neuroscience 105 2001 153-167
    • (2001) Neuroscience , vol.105 , pp. 153-167
    • Teunissen, C.E.1
  • 43
    • 0031037761 scopus 로고    scopus 로고
    • Assembly of CNS myelin in the absence of proteolipid protein
    • M. Klugmann et al. Assembly of CNS myelin in the absence of proteolipid protein Neuron 18 1997 59-70
    • (1997) Neuron , vol.18 , pp. 59-70
    • Klugmann, M.1


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