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Volumn 807, Issue , 2011, Pages 159-177

Recombinant AAV Delivery to the Central Nervous System

Author keywords

Confocal microscopy; GDNF; GFP; Hirt DNA; Immunohistochemistry; Intravenous injection; mRNA; Quantitative PCR; Retrograde transport; Stereotaxy

Indexed keywords

ADENO ASSOCIATED VIRUS; ANIMAL; ARTICLE; CENTRAL NERVOUS SYSTEM; CONFOCAL MICROSCOPY; GENE VECTOR; GENETICS; IMMUNOHISTOCHEMISTRY; METABOLISM; MOTONEURON; MOUSE; POLYMERASE CHAIN REACTION;

EID: 83455225632     PISSN: 10643745     EISSN: 19406029     Source Type: Book Series    
DOI: 10.1007/978-1-61779-370-7_7     Document Type: Chapter
Times cited : (10)

References (33)
  • 3
    • 29144442863 scopus 로고    scopus 로고
    • Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease
    • Dodge JC, Clarke J, Song A, Bu J, Yang W, Taksir TV, et al. (2005) Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease. Proc Natl Acad Sci USA 102, 17822–7.
    • (2005) Proc Natl Acad Sci USA , vol.102 , pp. 17822-17827
    • Dodge, J.C.1    Clarke, J.2    Song, A.3    Bu, J.4    Yang, W.5    Taksir, T.V.6
  • 4
    • 0036154761 scopus 로고    scopus 로고
    • Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery
    • Fu H, Samulski RJ, McCown TJ, Picornell YJ, Fletcher D, Muenzer J. (2002) Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery. Mol Ther 5, 42–49.
    • (2002) Mol Ther , vol.5 , pp. 42-49
    • Fu, H.1    Samulski, R.J.2    McCown, T.J.3    Picornell, Y.J.4    Fletcher, D.5    Muenzer, J.6
  • 5
    • 33846312858 scopus 로고    scopus 로고
    • Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNS
    • Liu G, Chen YH, He X, Martins I, Heth JA, Chiorini JA, et al. (2007) Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNS. Mol Ther 15, 242–7.
    • (2007) Mol Ther , vol.15 , pp. 242-247
    • Liu, G.1    Chen, Y.H.2    He, X.3    Martins, I.4    Heth, J.A.5    Chiorini, J.A.6
  • 6
    • 0034659835 scopus 로고    scopus 로고
    • Long-term rAAV-mediated gene transfer of GDNF in the rat Parkinson’s model: Intrastriatal but not intranigral transduction promotes functional regeneration in the lesioned nigros-triatal system
    • Jun 15
    • Kirik D, Rosenblad C, Bjorklund A, Mandel RJ. Long-term rAAV-mediated gene transfer of GDNF in the rat Parkinson’s model: intrastriatal but not intranigral transduction promotes functional regeneration in the lesioned nigros-triatal system. J Neurosci 2000 Jun 15;20(12): 4686–4700.
    • (2000) J Neurosci , vol.20 , Issue.12 , pp. 4686-4700
    • Kirik, D.1    Rosenblad, C.2    Bjorklund, A.3    Mandel, R.J.4
  • 7
    • 0031456051 scopus 로고    scopus 로고
    • Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson’s disease in rats
    • Mandel RJ, Spratt SK, Snyder RO, Leff SE. (1997) Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson’s disease in rats. Proc Natl Acad Sci USA 94, 14083–8.
    • (1997) Proc Natl Acad Sci USA , vol.94 , pp. 14083-14088
    • Mandel, R.J.1    Spratt, S.K.2    Snyder, R.O.3    Leff, S.E.4
  • 8
    • 72449188768 scopus 로고    scopus 로고
    • Reversible neurochemical changes mediated by delayed intrastriatal glial cell line-derived neurotrophic factor gene delivery in a partial Parkinson’s disease rat model
    • Yang X, Mertens B, Lehtonen E, Vercammen L, Bockstael O, Chtarto A, et al. (2009) Reversible neurochemical changes mediated by delayed intrastriatal glial cell line-derived neurotrophic factor gene delivery in a partial Parkinson’s disease rat model. J Gene Med 11, 899–912.
    • (2009) J Gene Med , vol.11 , pp. 899-912
    • Yang, X.1    Mertens, B.2    Lehtonen, E.3    Vercammen, L.4    Bockstael, O.5    Chtarto, A.6
  • 9
    • 45749153812 scopus 로고    scopus 로고
    • AAV-BDNF mediated attenuation of quinolinic acid-induced neuropathology and motor function impairment
    • Kells AP, Henry RA, Connor B. (2008) AAV-BDNF mediated attenuation of quinolinic acid-induced neuropathology and motor function impairment. Gene Ther 15, 966–977.
    • (2008) Gene Ther , vol.15 , pp. 966-977
    • Kells, A.P.1    Henry, R.A.2    Connor, B.3
  • 11
    • 0041464695 scopus 로고    scopus 로고
    • Attenuation of seizures and neuronal death by adeno-associated virus vector galanin expression and secretion
    • Haberman RP, Samulski RJ, McCown TJ. (2003) Attenuation of seizures and neuronal death by adeno-associated virus vector galanin expression and secretion. Nat Med 9, 1076–80.
    • (2003) Nat Med , vol.9 , pp. 1076-1080
    • Haberman, R.P.1    Samulski, R.J.2    McCown, T.J.3
  • 12
    • 0034691506 scopus 로고    scopus 로고
    • Triple transduction with adeno-associated virus vectors expressing tyrosine hydroxylase, aromatic-L-amino-acid decarboxylase, and GTP cyclohydrolase I for gene therapy of Parkinson’s disease
    • Shen Y, Muramatsu SI, Ikeguchi K, Fujimoto KI, Fan DS, Ogawa M, et al. (2000) Triple transduction with adeno-associated virus vectors expressing tyrosine hydroxylase, aromatic-L-amino-acid decarboxylase, and GTP cyclohydrolase I for gene therapy of Parkinson’s disease. Hum Gene Ther 11, 1509–19.
    • (2000) Hum Gene Ther , vol.11 , pp. 1509-1519
    • Shen, Y.1    Muramatsu, S.I.2    Ikeguchi, K.3    Fujimoto, K.I.4    Fan, D.S.5    Ogawa, M.6
  • 13
    • 15044365742 scopus 로고    scopus 로고
    • Reversal of dyskine-sias in an animal model of Parkinson’s disease by continuous L-DOPA delivery using rAAV vectors
    • Carlsson T, Winkler C, Burger C, Muzyczka N, Mandel RJ, Cenci A, et al. Reversal of dyskine-sias in an animal model of Parkinson’s disease by continuous L-DOPA delivery using rAAV vectors. (2005) Brain 128, 559–69.
    • (2005) Brain , vol.128 , pp. 559-569
    • Carlsson, T.1    Winkler, C.2    Burger, C.3    Muzyczka, N.4    Mandel, R.J.5    Cenci, A.6
  • 14
    • 4043057946 scopus 로고    scopus 로고
    • RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia
    • Xia H, Mao Q, Eliason SL, Harper SQ, Martins IH, Orr HT, et al. (2004) RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. Nat Med 10, 816–20.
    • (2004) Nat Med , vol.10 , pp. 816-820
    • Xia, H.1    Mao, Q.2    Eliason, S.L.3    Harper, S.Q.4    Martins, I.H.5    Orr, H.T.6
  • 15
    • 25144464388 scopus 로고    scopus 로고
    • Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington’s disease transgenic mice
    • Rodriguez-Lebron E, Denovan-Wright EM, Nash K, Lewin AS, Mandel RJ. (2005) Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington’s disease transgenic mice. Mol Ther 12, 618–33.
    • (2005) Mol Ther , vol.12 , pp. 618-633
    • Rodriguez-Lebron, E.1    Denovan-Wright, E.M.2    Nash, K.3    Lewin, A.S.4    Mandel, R.J.5
  • 16
    • 48349146711 scopus 로고    scopus 로고
    • Human interleukin-10 gene transfer is protective in a rat model of Parkinson’s disease
    • Johnston LC, Su X, Maguire-Zeiss K, Horovitz K, Ankoudinova I, Guschin D, et al. (2008) Human interleukin-10 gene transfer is protective in a rat model of Parkinson’s disease. Mol Ther 16, 1392–9.
    • (2008) Mol Ther , vol.16 , pp. 1392-1399
    • Johnston, L.C.1    Su, X.2    Maguire-Zeiss, K.3    Horovitz, K.4    Ankoudinova, I.5    Guschin, D.6
  • 17
    • 77249179125 scopus 로고    scopus 로고
    • Optimized cannula design and placement for convection-enhanced delivery in rat striatum
    • Yin D, Forsayeth J, Bankiewicz KS. (2009) Optimized cannula design and placement for convection-enhanced delivery in rat striatum. J Neurosci Methods 187, 46–51.
    • (2009) J Neurosci Methods , vol.187 , pp. 46-51
    • Yin, D.1    Forsayeth, J.2    Bankiewicz, K.S.3
  • 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
    • Mastakov MY, Baer K, Kotin RM, During MJ. (2002) Recombinant adeno-associated virus serotypes 2-and 5-mediated gene transfer in the mammalian brain: quantitative analysis of heparin co-infusion. Mol Ther 5, 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
    • 0034980351 scopus 로고    scopus 로고
    • Combined injection of rAAV with mannitol enhances gene expression in the rat brain
    • Mastakov MY, Baer K, Xu R, Fitzsimons H, During MJ. (2001) Combined injection of rAAV with mannitol enhances gene expression in the rat brain. Mol Ther 3, 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
  • 20
    • 85139042821 scopus 로고    scopus 로고
    • Paxinos, G. and Watson, 1997. “The rat brain in stereotaxic coordinates”, 3rd compact edition, Academic Press, Orlando, FLA
    • Paxinos, G. and Watson, 1997. “The rat brain in stereotaxic coordinates”, 3rd compact edition, Academic Press, Orlando, FLA
  • 22
    • 2642537522 scopus 로고    scopus 로고
    • Circulating anti-wild-type adeno-associated virus type 2 (AAV2) antibodies inhibit recombinant AAV2 (rAAV2)-mediated, but not rAAV5-mediated, gene transfer in the brain
    • Peden CS, Burger C, Muzyczka N, Mandel RJ. (2004) Circulating anti-wild-type adeno-associated virus type 2 (AAV2) antibodies inhibit recombinant AAV2 (rAAV2)-mediated, but not rAAV5-mediated, gene transfer in the brain. J Virol 78, 6344–59.
    • (2004) J Virol , vol.78 , pp. 6344-6359
    • Peden, C.S.1    Burger, C.2    Muzyczka, N.3    Mandel, R.J.4
  • 23
    • 61649120100 scopus 로고    scopus 로고
    • Striatal readministration of rAAV vectors reveals an immune response against AAV2 capsids that can be circumvented
    • Peden CS, Manfredsson FP, Reimsnider SK, Poirier AE, Burger C, Muzyczka N, et al. (2009) Striatal readministration of rAAV vectors reveals an immune response against AAV2 capsids that can be circumvented. Mol Ther 17, 524–537.
    • (2009) Mol Ther , vol.17 , pp. 524-537
    • Peden, C.S.1    Manfredsson, F.P.2    Reimsnider, S.K.3    Poirier, A.E.4    Burger, C.5    Muzyczka, N.6
  • 24
    • 67649861393 scopus 로고    scopus 로고
    • Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons
    • Duque S, Joussemet B, Riviere C, Marais T, Dubreil L, Douar AM, et al. (2009) Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol Ther 17, 1187–96.
    • (2009) Mol Ther , vol.17 , pp. 1187-1196
    • Duque, S.1    Joussemet, B.2    Riviere, C.3    Marais, T.4    Dubreil, L.5    Douar, A.M.6
  • 26
    • 70249131513 scopus 로고    scopus 로고
    • The neonatal blood-brain barrier is functionally effective, and immaturity does not explain differential targeting of AAV9
    • Saunders NR, Joakim EC, Dziegielewska KM. (2009) The neonatal blood-brain barrier is functionally effective, and immaturity does not explain differential targeting of AAV9. Nat Biotechnol 27, 804–5.
    • (2009) Nat Biotechnol , vol.27 , pp. 804-805
    • Saunders, N.R.1    Joakim, E.C.2    Dziegielewska, K.M.3
  • 27
    • 74349088517 scopus 로고    scopus 로고
    • Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6
    • Towne C, Schneider BL, Kieran D, Redmond DE, Jr., Aebischer P. (2010) Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6. Gene Ther 17, 141–6.
    • (2010) Gene Ther , vol.17 , pp. 141-146
    • Towne, C.1    Schneider, B.L.2    Kieran, D.3    Redmond, D.E.4    Aebischer, P.5
  • 28
    • 0042528664 scopus 로고    scopus 로고
    • Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model
    • Kaspar BK, Llado J, Sherkat N, Rothstein JD, 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    Llado, J.2    Sherkat, N.3    Rothstein, J.D.4    Gage, F.H.5
  • 29
    • 74949140217 scopus 로고    scopus 로고
    • Efficient retrograde transport of adeno-associated virus type 8 to spinal cord and dorsal root ganglion after vector delivery in muscle
    • Zheng H, Qiao C, Wang CH, Li J, Li J, Yuan Z, et al. (2010) Efficient retrograde transport of adeno-associated virus type 8 to spinal cord and dorsal root ganglion after vector delivery in muscle. Hum Gene Ther 21, 87–97.
    • (2010) Hum Gene Ther , vol.21 , pp. 87-97
    • Zheng, H.1    Qiao, C.2    Wang, C.H.3    Li, J.4    Li, J.5    Yuan, Z.6
  • 30
    • 38649139370 scopus 로고    scopus 로고
    • Efficient retrograde neuronal transduction utilizing self-complementary AAV1
    • Hollis ER, Kadoya K, Hirsch M, Samulski RJ, Tuszynski MH. (2008) Efficient 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
  • 31
    • 0021234096 scopus 로고
    • The unbiased estimation of number and sizes of arbitrary particles using the disector
    • Sterio DC. (1984) The unbiased estimation of number and sizes of arbitrary particles using the disector. J Microsc 134, 127–36.
    • (1984) J Microsc , vol.134 , pp. 127-136
    • Sterio, D.C.1
  • 32
    • 0014202537 scopus 로고
    • Selective extraction of polyoma DNA from infected mouse cell cultures
    • Hirt B. (1967) Selective extraction of polyoma DNA from infected mouse cell cultures. J Mol Biol 26: 365–9.
    • (1967) J Mol Biol , vol.26 , pp. 365-369
    • Hirt, B.1
  • 33
    • 0030848357 scopus 로고    scopus 로고
    • An acid-treatment method for the enhanced detection of GDNF in biological samples
    • Okragly AJ, Haak-Frendscho M. (1997) An acid-treatment method for the enhanced detection of GDNF in biological samples. Exp Neurol 145, 592–6.
    • (1997) Exp Neurol , vol.145 , pp. 592-596
    • Okragly, A.J.1    Haak-Frendscho, M.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.