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Volumn 2013, Issue , 2013, Pages

Systemic delivery of tyrosine-mutant AAV vectors results in robust transduction of neurons in adult mice

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; MICRORNA; TYROSINE;

EID: 84878689856     PISSN: 23146133     EISSN: 23146141     Source Type: Journal    
DOI: 10.1155/2013/974819     Document Type: Article
Times cited : (50)

References (43)
  • 1
    • 79956056971 scopus 로고    scopus 로고
    • Genetic therapy for the nervous system
    • 2-s2.0-79956056971 10.1093/hmg/ddr110
    • Bowers W. J., Breakefield X. O., Sena-Esteves M., Genetic therapy for the nervous system. Human Molecular Genetics 2011 20 R1 R28 R41 2-s2.0-79956056971 10.1093/hmg/ddr110
    • (2011) Human Molecular Genetics , vol.20
    • Bowers, W.J.1    Breakefield, X.O.2    Sena-Esteves, M.3
  • 3
    • 84875240244 scopus 로고    scopus 로고
    • Adeno-associated virus (AAV) gene therapy for neurological disease
    • 10.1016/j.neuropharm.2012.03.004 - 88
    • Weinberg M. S., Samulski R. J., McCown T. J., Adeno-associated virus (AAV) gene therapy for neurological disease. Neuropharmacology 2013 69 82- 88 10.1016/j.neuropharm.2012.03.004
    • (2013) Neuropharmacology , vol.69 , pp. 82
    • Weinberg, M.S.1    Samulski, R.J.2    McCown, T.J.3
  • 4
    • 77955175906 scopus 로고    scopus 로고
    • Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC
    • 2-s2.0-77955175906 10.1038/mt.2010.106
    • Hadaczek P., Eberling J. L., Pivirotto P., Bringas J., Forsayeth J., Bankiewicz K. S., Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC. Molecular Therapy 2010 18 8 1458 1461 2-s2.0-77955175906 10.1038/mt.2010.106
    • (2010) Molecular Therapy , vol.18 , Issue.8 , pp. 1458-1461
    • Hadaczek, P.1    Eberling, J.L.2    Pivirotto, P.3    Bringas, J.4    Forsayeth, J.5    Bankiewicz, K.S.6
  • 6
    • 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., Baumann T. L., Brown L., 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. Neurobiology of Aging 2012 34 1 35 61
    • (2012) Neurobiology of Aging , vol.34 , Issue.1 , pp. 35-61
    • Bartus, R.T.1    Baumann, T.L.2    Brown, L.3
  • 9
    • 80455173951 scopus 로고    scopus 로고
    • Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders
    • 10.1038/mt.2011.157
    • Bevan A. K., Duque S., Foust K. D., Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders. Molecular Therapy 2011 19 11 1971 1980 10.1038/mt.2011.157
    • (2011) Molecular Therapy , vol.19 , Issue.11 , pp. 1971-1980
    • Bevan, A.K.1    Duque, S.2    Foust, K.D.3
  • 10
    • 67649861393 scopus 로고    scopus 로고
    • Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons
    • 2-s2.0-67649861393 10.1038/mt.2009.71
    • Duque S., Joussemet B., Riviere C., Marais T., Dubreil L., Douar A. M., Fyfe J., Moullier P., Colle M. A., Barkats M., Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Molecular Therapy 2009 17 7 1187 1196 2-s2.0-67649861393 10.1038/mt.2009.71
    • (2009) Molecular Therapy , vol.17 , Issue.7 , pp. 1187-1196
    • Duque, S.1    Joussemet, B.2    Riviere, C.3    Marais, T.4    Dubreil, L.5    Douar, A.M.6    Fyfe, J.7    Moullier, P.8    Colle, M.A.9    Barkats, M.10
  • 11
    • 84864877570 scopus 로고    scopus 로고
    • Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs
    • 10.1038/gt.2011.130
    • Federici T., Taub J. S., Baum G. R., Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs. Gene Therapy 2012 19 8 852 859 10.1038/gt.2011.130
    • (2012) Gene Therapy , vol.19 , Issue.8 , pp. 852-859
    • Federici, T.1    Taub, J.S.2    Baum, G.R.3
  • 12
    • 60149106907 scopus 로고    scopus 로고
    • Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
    • 2-s2.0-60149106907 10.1038/nbt.1515
    • Foust K. D., Nurre E., Montgomery C. L., Hernandez A., Chan C. M., Kaspar B. K., Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nature Biotechnology 2009 27 1 59 65 2-s2.0-60149106907 10.1038/nbt.1515
    • (2009) Nature Biotechnology , vol.27 , Issue.1 , pp. 59-65
    • Foust, K.D.1    Nurre, E.2    Montgomery, C.L.3    Hernandez, A.4    Chan, C.M.5    Kaspar, B.K.6
  • 13
    • 80053923197 scopus 로고    scopus 로고
    • Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system
    • 10.1096/fj.11-182311
    • Rahim A. A., Wong A. M., Hoefer K., Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system. The FASEB Journal 2011 25 10 3505 3518 10.1096/fj.11-182311
    • (2011) The FASEB Journal , vol.25 , Issue.10 , pp. 3505-3518
    • Rahim, A.A.1    Wong, A.M.2    Hoefer, K.3
  • 14
    • 84860171925 scopus 로고    scopus 로고
    • Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates
    • 10.1089/hum.2011.200
    • Samaranch L., Salegio E. A., San Sebastian W., Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates. Human Gene Therapy 2012 23 4 382 389 10.1089/hum.2011.200
    • (2012) Human Gene Therapy , vol.23 , Issue.4 , pp. 382-389
    • Samaranch, L.1    Salegio, E.A.2    San Sebastian, W.3
  • 15
    • 84858026947 scopus 로고    scopus 로고
    • Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery
    • 10.1089/hum.2011.166
    • Glascock J. J., Osman E. Y., Wetz M. J., Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery. Human Gene Therapy 2012 23 3 330 335 10.1089/hum.2011.166
    • (2012) Human Gene Therapy , vol.23 , Issue.3 , pp. 330-335
    • Glascock, J.J.1    Osman, E.Y.2    Wetz, M.J.3
  • 17
    • 54849162100 scopus 로고    scopus 로고
    • Self-complementary AAV vectors; Advances and applications
    • 2-s2.0-54849162100 10.1038/mt.2008.171
    • McCarty D. M., Self-complementary AAV vectors; advances and applications. Molecular Therapy 2008 16 10 1648 1656 2-s2.0-54849162100 10.1038/mt.2008.171
    • (2008) Molecular Therapy , vol.16 , Issue.10 , pp. 1648-1656
    • McCarty, D.M.1
  • 18
    • 79957888016 scopus 로고    scopus 로고
    • Preclinical differences of intravascular AAV9 delivery to neurons and glia: A comparative study of adult mice and nonhuman primates
    • 2-s2.0-79957888016 10.1038/mt.2011.72
    • Gray S. J., Matagne V., Bachaboina L., Yadav S., Ojeda S. R., Samulski R. J., Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. Molecular Therapy 2011 19 6 1058 1069 2-s2.0-79957888016 10.1038/mt.2011.72
    • (2011) Molecular Therapy , vol.19 , Issue.6 , pp. 1058-1069
    • Gray, S.J.1    Matagne, V.2    Bachaboina, L.3    Yadav, S.4    Ojeda, S.R.5    Samulski, R.J.6
  • 19
    • 84871922836 scopus 로고    scopus 로고
    • Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses
    • Ciesielska A., Hadaczek P., Mittermeyer G., Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses. Molecular Therapy 2012 21 1 158 166
    • (2012) Molecular Therapy , vol.21 , Issue.1 , pp. 158-166
    • Ciesielska, A.1    Hadaczek, P.2    Mittermeyer, G.3
  • 20
    • 84872241791 scopus 로고    scopus 로고
    • Restricted transgene expression in the brain with cell-type specific neuronal promoters
    • Delzor A., Dufour N., Petit F., Restricted transgene expression in the brain with cell-type specific neuronal promoters. Human Gene Therapy Methods 2012 23 4 242 254
    • (2012) Human Gene Therapy Methods , vol.23 , Issue.4 , pp. 242-254
    • Delzor, A.1    Dufour, N.2    Petit, F.3
  • 21
    • 81255169334 scopus 로고    scopus 로고
    • Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis
    • 10.1093/hmg/ddr391
    • Ku C. A., Chiodo V. A., Boye S. L., Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis. Human Molecular Genetics 2011 20 23 4569 4581 10.1093/hmg/ddr391
    • (2011) Human Molecular Genetics , vol.20 , Issue.23 , pp. 4569-4581
    • Ku, C.A.1    Chiodo, V.A.2    Boye, S.L.3
  • 22
    • 78649375117 scopus 로고    scopus 로고
    • High-efficiency transduction of fibroblasts and mesenchymal stem cells by tyrosine-mutant AAV2 vectors for their potential use in cellular therapy
    • 2-s2.0-78649375117 10.1089/hum.2010.005
    • Li M., Jayandharan G. R., Li B., Ling C., Ma W., Srivastava A., Zhong L., High-efficiency transduction of fibroblasts and mesenchymal stem cells by tyrosine-mutant AAV2 vectors for their potential use in cellular therapy. Human Gene Therapy 2010 21 11 1527 1543 2-s2.0-78649375117 10.1089/hum.2010.005
    • (2010) Human Gene Therapy , vol.21 , Issue.11 , pp. 1527-1543
    • Li, M.1    Jayandharan, G.R.2    Li, B.3    Ling, C.4    Ma, W.5    Srivastava, A.6    Zhong, L.7
  • 24
    • 77958102381 scopus 로고    scopus 로고
    • Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle
    • 2-s2.0-77958102381 10.1089/hum.2010.003
    • Qiao C., Zhang W., Yuan Z., Shin J. H., Li J., Jayandharan G. R., Zhong L., Srivastava A., Xiao X., Duan D., Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle. Human Gene Therapy 2010 21 10 1343 1348 2-s2.0-77958102381 10.1089/hum.2010.003
    • (2010) Human Gene Therapy , vol.21 , Issue.10 , pp. 1343-1348
    • Qiao, C.1    Zhang, W.2    Yuan, Z.3    Shin, J.H.4    Li, J.5    Jayandharan, G.R.6    Zhong, L.7    Srivastava, A.8    Xiao, X.9    Duan, D.10
  • 26
    • 84856975718 scopus 로고    scopus 로고
    • Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9
    • 10.1038/gt.2011.163
    • Dalkara D., Byrne L. C., Lee T., Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9. Gene Therapy 2012 19 2 176 181 10.1038/gt.2011.163
    • (2012) Gene Therapy , vol.19 , Issue.2 , pp. 176-181
    • Dalkara, D.1    Byrne, L.C.2    Lee, T.3
  • 27
    • 84862908306 scopus 로고    scopus 로고
    • Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma
    • 10.1089/hum.2011.131
    • Zhang Y., Duan D., Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma. Human Gene Therapy 2012 23 1 98 103 10.1089/hum.2011.131
    • (2012) Human Gene Therapy , vol.23 , Issue.1 , pp. 98-103
    • Zhang, Y.1    Duan, D.2
  • 29
    • 0025368830 scopus 로고
    • The mouse ribosomal protein L7 gene. Its primary structure and functional analysis of the promoter region
    • 2-s2.0-0025368830
    • Meyuhas O., Klein A., The mouse ribosomal protein L7 gene. Its primary structure and functional analysis of the promoter region. Journal of Biological Chemistry 1990 265 20 11465 11473 2-s2.0-0025368830
    • (1990) Journal of Biological Chemistry , vol.265 , Issue.20 , pp. 11465-11473
    • Meyuhas, O.1    Klein, A.2
  • 30
    • 2642512201 scopus 로고    scopus 로고
    • Clades of adeno-associated viruses are widely disseminated in human tissues
    • DOI 10.1128/JVI.78.12.6381-6388.2004
    • Gao G., Vandenberghe L. H., Alvira M. R., Lu Y., Calcedo R., Zhou X., Wilson J. M., Clades of adeno-associated viruses are widely disseminated in human tissues. Journal of Virology 2004 78 12 6381 6388 2-s2.0-2642512201 10.1128/JVI.78.12.6381-6388.2004 (Pubitemid 38715936)
    • (2004) Journal of Virology , vol.78 , Issue.12 , pp. 6381-6388
    • Gao, G.1    Vandenberghe, L.H.2    Alvira, M.R.3    Lu, Y.4    Calcedo, R.5    Zhou, X.6    Wilson, J.M.7
  • 32
    • 80051584095 scopus 로고    scopus 로고
    • An efficient method for the long-term and specific expression of exogenous cDNAs in cultured Purkinje neurons
    • Wagner W., McCroskery S., Hammer J. A. III, An efficient method for the long-term and specific expression of exogenous cDNAs in cultured Purkinje neurons. Journal of Neuroscience Methods 2011 200 2 95 105
    • (2011) Journal of Neuroscience Methods , vol.200 , Issue.2 , pp. 95-105
    • Wagner, W.1    McCroskery, S.2    Hammer III, J.A.3
  • 33
    • 10644289281 scopus 로고    scopus 로고
    • Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice
    • DOI 10.1128/JVI.79.1.214-224.2005
    • Nakai H., Fuess S., Storm T. A., Muramatsu S. I., Nara Y., Kay M. A., Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. Journal of Virology 2005 79 1 214 224 2-s2.0-10644289281 10.1128/JVI.79.1.214-224.2005 (Pubitemid 39650967)
    • (2005) Journal of Virology , vol.79 , Issue.1 , pp. 214-224
    • Nakai, H.1    Fuess, S.2    Storm, T.A.3    Muramatsu, S.-I.4    Nara, Y.5    Kay, M.A.6
  • 34
    • 84872233239 scopus 로고    scopus 로고
    • Limitations of encapsidation of recombinant self-complementary adeno-associated viral genomes in different serotype capsids and their quantitation
    • Wang Y., Ling C., Song L., Limitations of encapsidation of recombinant self-complementary adeno-associated viral genomes in different serotype capsids and their quantitation. Human Gene Therapy Methods 2012 23 4 225 233
    • (2012) Human Gene Therapy Methods , vol.23 , Issue.4 , pp. 225-233
    • Wang, Y.1    Ling, C.2    Song, L.3
  • 35
    • 79961032418 scopus 로고    scopus 로고
    • Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system
    • 2-s2.0-79961032418 10.1038/mt.2011.98
    • Zhang H., Yang B., Mu X., Ahmed S. S., Su Q., He R., Wang H., Mueller C., Sena-Esteves M., Brown R., Xu Z., Gao G., Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system. Molecular Therapy 2011 19 8 1440 1448 2-s2.0-79961032418 10.1038/mt.2011.98
    • (2011) Molecular Therapy , vol.19 , Issue.8 , pp. 1440-1448
    • Zhang, H.1    Yang, B.2    Mu, X.3    Ahmed, S.S.4    Su, Q.5    He, R.6    Wang, H.7    Mueller, C.8    Sena-Esteves, M.9    Brown, R.10    Xu, Z.11    Gao, G.12
  • 36
    • 79951704433 scopus 로고    scopus 로고
    • Neuroinflammation in amyotrophic lateral sclerosis: Role of glial activation in motor neuron disease
    • 2-s2.0-79951704433 10.1016/S1474-4422(11)70015-1
    • Philips T., Robberecht W., Neuroinflammation in amyotrophic lateral sclerosis: role of glial activation in motor neuron disease. The Lancet Neurology 2011 10 3 253 263 2-s2.0-79951704433 10.1016/S1474-4422(11)70015-1
    • (2011) The Lancet Neurology , vol.10 , Issue.3 , pp. 253-263
    • Philips, T.1    Robberecht, W.2
  • 37
    • 77957048035 scopus 로고    scopus 로고
    • Astrocytes and therapeutics for Parkinson's disease
    • 2-s2.0-77957048035 10.1016/j.nurt.2010.07.001
    • Rappold P. M., Tieu K., Astrocytes and therapeutics for Parkinson's disease. Neurotherapeutics 2010 7 4 413 423 2-s2.0-77957048035 10.1016/j.nurt.2010.07.001
    • (2010) Neurotherapeutics , vol.7 , Issue.4 , pp. 413-423
    • Rappold, P.M.1    Tieu, K.2
  • 38
    • 84862526741 scopus 로고    scopus 로고
    • Tyrosine cross-linking reveals interfacial dynamics in adeno-associated viral capsids during infection
    • 10.1021/cb3000265
    • Horowitz E. D., Finn M. G., Asokan A., Tyrosine cross-linking reveals interfacial dynamics in adeno-associated viral capsids during infection. ACS Chemical Biology 2012 7 6 1059 1066 10.1021/cb3000265
    • (2012) ACS Chemical Biology , vol.7 , Issue.6 , pp. 1059-1066
    • Horowitz, E.D.1    Finn, M.G.2    Asokan, A.3
  • 41
    • 84863714144 scopus 로고    scopus 로고
    • Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF2
    • 10.1038/nm.2791
    • Miyazaki Y., Adachi H., Katsuno M., Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF2. Nature Medicine 2012 18 7 1136 1141 10.1038/nm.2791
    • (2012) Nature Medicine , vol.18 , Issue.7 , pp. 1136-1141
    • Miyazaki, Y.1    Adachi, H.2    Katsuno, M.3
  • 43
    • 84869208042 scopus 로고    scopus 로고
    • Properties of the adeno-associated virus assembly activating protein
    • 10.1128/JVI.01675-12
    • Naumer M., Sonntag F., Schmidt K., Properties of the adeno-associated virus assembly activating protein. Journal of Virology 2012 86 23 13038 13048 10.1128/JVI.01675-12
    • (2012) Journal of Virology , vol.86 , Issue.23 , pp. 13038-13048
    • Naumer, M.1    Sonntag, F.2    Schmidt, K.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.