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Volumn 23, Issue 1, 2015, Pages 53-62

Widespread and efficient transduction of spinal cord and brain following neonatal AAV injection and potential disease modifying effect in ALS mice

Author keywords

[No Author keywords available]

Indexed keywords

ANTIVIRUS AGENT; INTERLEUKIN 10; RAAV2/1; RAAV2/5; RAAV2/8; RAAV2/9; RECOMBINANT ADENO ASSOCIATED VIRUS VECTOR; UNCLASSIFIED DRUG; VIRUS VECTOR; ENHANCED GREEN FLUORESCENT PROTEIN; GREEN FLUORESCENT PROTEIN; IL10 PROTEIN, MOUSE; SOD1 G93A PROTEIN; SUPEROXIDE DISMUTASE;

EID: 84920561391     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2014.180     Document Type: Article
Times cited : (50)

References (43)
  • 1
    • 84877103526 scopus 로고    scopus 로고
    • Thinking laterally about neurodegenerative proteinopathies
    • Golde, TE, Borchelt, DR, Giasson, BI and Lewis, J (2013). Thinking laterally about neurodegenerative proteinopathies. J Clin Invest 123: 1847-1855.
    • (2013) J Clin Invest , vol.123 , pp. 1847-1855
    • Golde, T.E.1    Borchelt, D.R.2    Giasson, B.I.3    Lewis, J.4
  • 2
    • 84880302312 scopus 로고    scopus 로고
    • Continuous DOPA synthesis from a single AAV: Dosing and effcacy in models of Parkinson's disease
    • Cederfjäll, E, Nilsson, N, Sahin, G, Chu, Y, Nikitidou, E, Björklund, T et al. (2013). Continuous DOPA synthesis from a single AAV: dosing and effcacy in models of Parkinson's disease. Sci Rep 3: 2157.
    • (2013) Sci Rep , vol.3 , pp. 2157
    • Cederfjäll, E.1    Nilsson, N.2    Sahin, G.3    Chu, Y.4    Nikitidou, E.5    Björklund, T.6
  • 3
    • 84881314432 scopus 로고    scopus 로고
    • Acat1 knockdown gene therapy decreases amyloid-β in a mouse model of Alzheimer's disease
    • Murphy, SR, Chang, CC, Dogbevia, G, Bryleva, EY, Bowen, Z, Hasan, MT et al. (2013). Acat1 knockdown gene therapy decreases amyloid-β in a mouse model of Alzheimer's disease. Mol Ther 21: 1497-1506.
    • (2013) Mol Ther , vol.21 , pp. 1497-1506
    • Murphy, S.R.1    Chang, C.C.2    Dogbevia, G.3    Bryleva, E.Y.4    Bowen, Z.5    Hasan, M.T.6
  • 4
    • 84869016768 scopus 로고    scopus 로고
    • Targeting astrocytes ameliorates neurologic changes in a mouse model of Alzheimer's disease
    • Furman, JL, Sama, DM, Gant, JC, Beckett, TL, Murphy, M P, Bachstetter, AD et al. (2012). Targeting astrocytes ameliorates neurologic changes in a mouse model of Alzheimer's disease. J Neurosci 32: 16129-16140.
    • (2012) J Neurosci , vol.32 , pp. 16129-16140
    • Furman, J.L.1    Sama, D.M.2    Gant, J.C.3    Beckett, T.L.4    Murphy, M.P.5    Bachstetter, A.D.6
  • 5
    • 84866984026 scopus 로고    scopus 로고
    • Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type A
    • Bu, J, Ashe, KM, Bringas, J, Marshall, J, Dodge, JC, Cabrera-Salazar, MA et al. (2012). Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type A. Mol Ther 20: 1893-1901.
    • (2012) Mol Ther , vol.20 , pp. 1893-1901
    • Bu, J.1    Ashe, K.M.2    Bringas, J.3    Marshall, J.4    Dodge, J.C.5    Cabrera-Salazar, M.A.6
  • 6
    • 54749126318 scopus 로고    scopus 로고
    • Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain
    • Cearley, CN, Vandenberghe, LH, Parente, MK, Carnish, ER, Wilson, JM and Wolfe, JH (2008). Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain. Mol Ther 16: 1710-1718.
    • (2008) Mol Ther , vol.16 , pp. 1710-1718
    • Cearley, C.N.1    Vandenberghe, L.H.2    Parente, M.K.3    Carnish, E.R.4    Wilson, J.M.5    Wolfe, J.H.6
  • 7
    • 77954495173 scopus 로고    scopus 로고
    • IFN-gamma promotes complement expression and attenuates amyloid plaque deposition in amyloid beta precursor protein transgenic mice
    • Chakrabarty, P, Ceballos-Diaz, C, Beccard, A, Janus, C, Dickson, D, Golde, TE et al. (2010). IFN-gamma promotes complement expression and attenuates amyloid plaque deposition in amyloid beta precursor protein transgenic mice. J Immunol 184: 5333-5343.
    • (2010) J Immunol , vol.184 , pp. 5333-5343
    • Chakrabarty, P.1    Ceballos-Diaz, C.2    Beccard, A.3    Janus, C.4    Dickson, D.5    Golde, T.E.6
  • 8
    • 33751111439 scopus 로고    scopus 로고
    • Intracranial adeno-associated virus-mediated delivery of anti-pan amyloid beta, amyloid beta40, and amyloid beta42 single-chain variable fragments attenuates plaque pathology in amyloid precursor protein mice
    • Levites, Y, Jansen, K, Smithson, LA, Dakin, R, Holloway, VM, Das, P et al. (2006). Intracranial adeno-associated virus-mediated delivery of anti-pan amyloid beta, amyloid beta40, and amyloid beta42 single-chain variable fragments attenuates plaque pathology in amyloid precursor protein mice. J Neurosci 26: 11923-11928.
    • (2006) J Neurosci , vol.26 , pp. 11923-11928
    • Levites, Y.1    Jansen, K.2    Smithson, L.A.3    Dakin, R.4    Holloway, V.M.5    Das, P.6
  • 9
    • 32944470807 scopus 로고    scopus 로고
    • Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders
    • Mandel, RJ, Manfredsson, F P, Foust, KD, Rising, A, Reimsnider, S, Nash, K et al. (2006). Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders. Mol Ther 13: 463-483.
    • (2006) Mol Ther , vol.13 , pp. 463-483
    • Mandel, R.J.1    Manfredsson, F.P.2    Foust, K.D.3    Rising, A.4    Reimsnider, S.5    Nash, K.6
  • 10
    • 79954622209 scopus 로고    scopus 로고
    • Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges
    • Mingozzi, F and High, KA (2011). Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet 12: 341-355.
    • (2011) Nat Rev Genet , vol.12 , pp. 341-355
    • Mingozzi, F.1    High, K.A.2
  • 11
    • 84859440283 scopus 로고    scopus 로고
    • The AAV vector toolkit: Poised at the clinical crossroads
    • Asokan, A, Schaffer, DV and Samulski, RJ (2012). The AAV vector toolkit: poised at the clinical crossroads. Mol Ther 20: 699-708.
    • (2012) Mol Ther , vol.20 , pp. 699-708
    • Asokan, A.1    Schaffer, D.V.2    Samulski, R.J.3
  • 12
    • 84879371255 scopus 로고    scopus 로고
    • Capsid serotype and timing of injection determines AAV transduction in the neonatal mice brain
    • Chakrabarty, P, Rosario, A, Cruz, P, Siemienski, Z, Ceballos-Diaz, C, Crosby, K et al. (2013). Capsid serotype and timing of injection determines AAV transduction in the neonatal mice brain. PLoS One 8: e67680.
    • (2013) PLoS One , vol.8 , pp. e67680
    • Chakrabarty, P.1    Rosario, A.2    Cruz, P.3    Siemienski, Z.4    Ceballos-Diaz, C.5    Crosby, K.6
  • 14
    • 84873410199 scopus 로고    scopus 로고
    • Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice
    • Benkhelifa-Ziyyat, S, Besse, A, Roda, M, Duque, S, Astord, S, Carcenac, R et al. (2013). Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice. Mol Ther 21: 282-290.
    • (2013) Mol Ther , vol.21 , pp. 282-290
    • Benkhelifa-Ziyyat, S.1    Besse, A.2    Roda, M.3    Duque, S.4    Astord, S.5    Carcenac, R.6
  • 15
    • 84880338159 scopus 로고    scopus 로고
    • Intraspinal AAV injections immediately rostral to a thoracic spinal cord injury site effciently transduces neurons in spinal cord and brain
    • Klaw, MC, Xu, C and Tom, VJ (2013). Intraspinal AAV injections immediately rostral to a thoracic spinal cord injury site effciently transduces neurons in spinal cord and brain. Mol Ther Nucleic Acids 2: e108.
    • (2013) Mol Ther Nucleic Acids , vol.2 , pp. e108
    • Klaw, M.C.1    Xu, C.2    Tom, V.J.3
  • 16
    • 0042528664 scopus 로고    scopus 로고
    • Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model
    • Kaspar, BK, Lladó, J, Sherkat, N, Rothstein, JD and Gage, FH (2003). Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science 301: 839-842.
    • (2003) Science , vol.301 , pp. 839-842
    • Kaspar, B.K.1    Lladó, J.2    Sherkat, N.3    Rothstein, J.D.4    Gage, F.H.5
  • 18
    • 84861326894 scopus 로고    scopus 로고
    • The advent of AAV9 expands applications for brain and spinal cord gene delivery
    • Dayton, RD, Wang, DB and Klein, RL (2012). The advent of AAV9 expands applications for brain and spinal cord gene delivery. Expert Opin Biol Ther 12: 757-766.
    • (2012) Expert Opin Biol Ther , vol.12 , pp. 757-766
    • Dayton, R.D.1    Wang, D.B.2    Klein, R.L.3
  • 19
    • 77749249680 scopus 로고    scopus 로고
    • Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
    • Foust, KD, Wang, X, McGovern, VL, Braun, L, Bevan, AK, Haidet, AM et al. (2010). Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol 28: 271-274.
    • (2010) Nat Biotechnol , vol.28 , pp. 271-274
    • Foust, K.D.1    Wang, X.2    McGovern, V.L.3    Braun, L.4    Bevan, A.K.5    Haidet, A.M.6
  • 20
    • 38649139370 scopus 로고    scopus 로고
    • Effcient retrograde neuronal transduction utilizing self-complementary AAV1
    • Hollis, ER 2nd, Kadoya, K, Hirsch, M, Samulski, RJ and Tuszynski, MH (2008). Effcient retrograde neuronal transduction utilizing self-complementary AAV1. Mol Ther 16: 296-301.
    • (2008) Mol Ther , vol.16 , pp. 296-301
    • Hollis, E.R.1    Kadoya, K.2    Hirsch, M.3    Samulski, R.J.4    Tuszynski, M.H.5
  • 21
    • 84880593182 scopus 로고    scopus 로고
    • ScAAV9 intracisternal delivery results in effcient gene transfer to the central nervous system of a feline model of motor neuron disease
    • Bucher, T, Colle, MA, Wakeling, E, Dubreil, L, Fyfe, J, Briot-Nivard, D et al. (2013). scAAV9 intracisternal delivery results in effcient gene transfer to the central nervous system of a feline model of motor neuron disease. Hum Gene Ther 24: 670-682.
    • (2013) Hum Gene Ther , vol.24 , pp. 670-682
    • Bucher, T.1    Colle, M.A.2    Wakeling, E.3    Dubreil, L.4    Fyfe, J.5    Briot-Nivard, D.6
  • 22
    • 77949629188 scopus 로고    scopus 로고
    • Evaluation of a rapamycin-regulated serotype 2 adeno-associated viral vector in macaque parotid glands
    • Zheng, C, Voutetakis, A, Metzger, M, Afone, S, Cotrim, AP, Eckhaus, MA et al. (2010). Evaluation of a rapamycin-regulated serotype 2 adeno-associated viral vector in macaque parotid glands. Oral Dis 16: 269-277.
    • (2010) Oral Dis , vol.16 , pp. 269-277
    • Zheng, C.1    Voutetakis, A.2    Metzger, M.3    Afone, S.4    Cotrim, A.P.5    Eckhaus, M.A.6
  • 23
    • 79551641133 scopus 로고    scopus 로고
    • Neuroprotection by gene therapy targeting mutant SOD1 in individual pools of motor neurons does not translate into therapeutic beneft in fALS mice
    • Towne, C, Setola, V, Schneider, BL and Aebischer, P (2011). Neuroprotection by gene therapy targeting mutant SOD1 in individual pools of motor neurons does not translate into therapeutic beneft in fALS mice. Mol Ther 19: 274-283.
    • (2011) Mol Ther , vol.19 , pp. 274-283
    • Towne, C.1    Setola, V.2    Schneider, B.L.3    Aebischer, P.4
  • 24
    • 84904412918 scopus 로고    scopus 로고
    • Translational fdelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy
    • Passini, MA, Bu, J, Richards, AM, Treleaven, CM, Sullivan, JA, O'Riordan, CR et al. (2014). Translational fdelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy. Hum Gene Ther 25: 619-630.
    • (2014) Hum Gene Ther , vol.25 , pp. 619-630
    • Passini, M.A.1    Bu, J.2    Richards, A.M.3    Treleaven, C.M.4    Sullivan, J.A.5    O'Riordan, C.R.6
  • 25
    • 84890119907 scopus 로고    scopus 로고
    • Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS
    • Foust, KD, Salazar, DL, Likhite, S, Ferraiuolo, L, Ditsworth, D, Ilieva, H et al. (2013). Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS. Mol Ther 21: 2148-2159.
    • (2013) Mol Ther , vol.21 , pp. 2148-2159
    • Foust, K.D.1    Salazar, D.L.2    Likhite, S.3    Ferraiuolo, L.4    Ditsworth, D.5    Ilieva, H.6
  • 26
    • 84893307092 scopus 로고    scopus 로고
    • AAV9-mediated expression of a non-self protein in nonhuman primate central nervous system triggers widespread neuroinfammation driven by antigen-presenting cell transduction
    • Samaranch, L, San Sebastian, W, Kells, AP, Salegio, EA, Heller, G, Bringas, JR et al. (2014). AAV9-mediated expression of a non-self protein in nonhuman primate central nervous system triggers widespread neuroinfammation driven by antigen-presenting cell transduction. Mol Ther 22: 329-337.
    • (2014) Mol Ther , vol.22 , pp. 329-337
    • Samaranch, L.1    San Sebastian, W.2    Kells, A.P.3    Salegio, E.A.4    Heller, G.5    Bringas, J.R.6
  • 27
    • 84875925195 scopus 로고    scopus 로고
    • 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 and 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: 450-459.
    • (2013) Gene Ther , vol.20 , pp. 450-459
    • Gray, S.J.1    Nagabhushan Kalburgi, S.2    McCown, T.J.3    Jude Samulski, R.4
  • 28
    • 80053009250 scopus 로고    scopus 로고
    • Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery
    • Snyder, BR, Gray, SJ, Quach, ET, Huang, JW, Leung, CH, Samulski, RJ et al. (2011). Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery. Hum Gene Ther 22: 1129-1135.
    • (2011) Hum Gene Ther , vol.22 , pp. 1129-1135
    • Snyder, B.R.1    Gray, S.J.2    Quach, E.T.3    Huang, J.W.4    Leung, C.H.5    Samulski, R.J.6
  • 30
    • 76149092520 scopus 로고    scopus 로고
    • Massive gliosis induced by interleukin-6 suppresses Abeta deposition in vivo: Evidence against infammation as a driving force for amyloid deposition
    • Chakrabarty, P, Jansen-West, K, Beccard, A, Ceballos-Diaz, C, Levites, Y, Verbeeck, C et al. (2010). Massive gliosis induced by interleukin-6 suppresses Abeta deposition in vivo: evidence against infammation as a driving force for amyloid deposition. FASEB J 24: 548-559.
    • (2010) FASEB J , vol.24 , pp. 548-559
    • Chakrabarty, P.1    Jansen-West, K.2    Beccard, A.3    Ceballos-Diaz, C.4    Levites, Y.5    Verbeeck, C.6
  • 32
    • 79951551206 scopus 로고    scopus 로고
    • Hippocampal expression of murine TNFα results in attenuation of amyloid deposition in vivo
    • Chakrabarty, P, Herring, A, Ceballos-Diaz, C, Das, P and Golde, TE (2011). Hippocampal expression of murine TNFα results in attenuation of amyloid deposition in vivo. Mol Neurodegener 6: 16.
    • (2011) Mol Neurodegener , vol.6 , pp. 16
    • Chakrabarty, P.1    Herring, A.2    Ceballos-Diaz, C.3    Das, P.4    Golde, T.E.5
  • 33
    • 84885969062 scopus 로고    scopus 로고
    • The host environment is responsible for aging-related functional NK cell defciency
    • Chiu, BC, Martin, BE, Stolberg, VR and Chensue, SW (2013). The host environment is responsible for aging-related functional NK cell defciency. J Immunol 191: 4688-4698.
    • (2013) J Immunol , vol.191 , pp. 4688-4698
    • Chiu, B.C.1    Martin, B.E.2    Stolberg, V.R.3    Chensue, S.W.4
  • 34
    • 79957528028 scopus 로고    scopus 로고
    • Endogenous regulatory T lymphocytes ameliorate amyotrophic lateral sclerosis in mice and correlate with disease progression in patients with amyotrophic lateral sclerosis
    • Beers, DR, Henkel, JS, Zhao, W, Wang, J, Huang, A, Wen, S et al. (2011). Endogenous regulatory T lymphocytes ameliorate amyotrophic lateral sclerosis in mice and correlate with disease progression in patients with amyotrophic lateral sclerosis. Brain 134(Pt 5): 1293-1314.
    • (2011) Brain , vol.134 , pp. 1293-1314
    • Beers, D.R.1    Henkel, J.S.2    Zhao, W.3    Wang, J.4    Huang, A.5    Wen, S.6
  • 35
    • 84865987121 scopus 로고    scopus 로고
    • Modulating infammatory monocytes with a unique microRNA gene signature ameliorates murine ALS
    • Butovsky, O, Siddiqui, S, Gabriely, G, Lanser, AJ, Dake, B, Murugaiyan, G et al. (2012). Modulating infammatory monocytes with a unique microRNA gene signature ameliorates murine ALS. J Clin Invest 122: 3063-3087.
    • (2012) J Clin Invest , vol.122 , pp. 3063-3087
    • Butovsky, O.1    Siddiqui, S.2    Gabriely, G.3    Lanser, A.J.4    Dake, B.5    Murugaiyan, G.6
  • 36
    • 84864928300 scopus 로고    scopus 로고
    • Regulatory T lymphocytes from ALS mice suppress microglia and effector T lymphocytes through different cytokine-mediated mechanisms
    • Zhao, W, Beers, DR, Liao, B, Henkel, JS and Appel, SH (2012). Regulatory T lymphocytes from ALS mice suppress microglia and effector T lymphocytes through different cytokine-mediated mechanisms. Neurobiol Dis 48: 418-428.
    • (2012) Neurobiol Dis , vol.48 , pp. 418-428
    • Zhao, W.1    Beers, D.R.2    Liao, B.3    Henkel, J.S.4    Appel, S.H.5
  • 38
    • 33750478657 scopus 로고    scopus 로고
    • Wild-type microglia extend survival in PU.1 knockout mice with familial amyotrophic lateral sclerosis
    • Beers, DR, Henkel, JS, Xiao, Q, Zhao, W, Wang, J, Yen, AA et al. (2006). Wild-type microglia extend survival in PU.1 knockout mice with familial amyotrophic lateral sclerosis. Proc Natl Acad Sci USA 103: 16021-16026.
    • (2006) Proc Natl Acad Sci USA , vol.103 , pp. 16021-16026
    • Beers, D.R.1    Henkel, J.S.2    Xiao, Q.3    Zhao, W.4    Wang, J.5    Yen, A.A.6
  • 40
    • 15444376598 scopus 로고    scopus 로고
    • Mouse model of multiple system atrophy alpha-synuclein expression in oligodendrocytes causes glial and neuronal degeneration
    • Yazawa, I, Giasson, BI, Sasaki, R, Zhang, B, Joyce, S, Uryu, K et al. (2005). Mouse model of multiple system atrophy alpha-synuclein expression in oligodendrocytes causes glial and neuronal degeneration. Neuron 45: 847-859.
    • (2005) Neuron , vol.45 , pp. 847-859
    • Yazawa, I.1    Giasson, B.I.2    Sasaki, R.3    Zhang, B.4    Joyce, S.5    Uryu, K.6
  • 41
    • 33749010065 scopus 로고    scopus 로고
    • Bergmann glia expression of polyglutamine-expanded ataxin-7 produces neurodegeneration by impairing glutamate transport
    • Custer, SK, Garden, GA, Gill, N, Rueb, U, Libby, RT, Schultz, C et al. (2006). Bergmann glia expression of polyglutamine-expanded ataxin-7 produces neurodegeneration by impairing glutamate transport. Nat Neurosci 9: 1302-1311.
    • (2006) Nat Neurosci , vol.9 , pp. 1302-1311
    • Custer, S.K.1    Garden, G.A.2    Gill, N.3    Rueb, U.4    Libby, R.T.5    Schultz, C.6
  • 42
    • 0036428801 scopus 로고    scopus 로고
    • Production and purifcation of serotype 1, 2, and 5 recombinant adeno-associated viral vectors
    • Zolotukhin, S, Potter, M, Zolotukhin, I, Sakai, Y, Loiler, S, Fraites, TJ Jr et al. (2002). Production and purifcation of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods 28: 158-167.
    • (2002) Methods , vol.28 , pp. 158-167
    • Zolotukhin, S.1    Potter, M.2    Zolotukhin, I.3    Sakai, Y.4    Loiler, S.5    Fraites, T.J.J.R.6


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