메뉴 건너뛰기




Volumn 11, Issue 6, 2009, Pages 498-505

Global diffuse distribution in the brain and efficient gene delivery to the dorsal root ganglia by intrathecal injection of adeno-associated viral vector serotype 1

Author keywords

Adeno associated viral vector serotype 1 (AAV1); Arylsulfatase A (ASA); Brain; Dorsal root ganglia; Intrathecal (IT) Injection; Metachromatic leukodystrophy (MLD)

Indexed keywords

CEREBROSIDE SULFATASE; GREEN FLUORESCENT PROTEIN; PARVOVIRUS VECTOR; SULFATIDE;

EID: 65949115945     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1325     Document Type: Article
Times cited : (31)

References (37)
  • 2
    • 10544235699 scopus 로고    scopus 로고
    • Phenotype of arylsulfatase A-deficient mice: Relationship to human metachromatic leukodystrophy
    • Hess B, Saftig P, Hartmann D, et al. Phenotype of arylsulfatase A-deficient mice: relationship to human metachromatic leukodystrophy. Proc Natl Acad Sci USA 1996; 93: 14821-14826.
    • (1996) Proc Natl Acad Sci USA , vol.93 , pp. 14821-14826
    • Hess, B.1    Saftig, P.2    Hartmann, D.3
  • 3
    • 25144474418 scopus 로고    scopus 로고
    • Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery
    • Luca T, Givogri MI, Perani L, et al. Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery. Mol Ther 2005; 12: 669-679.
    • (2005) Mol Ther , vol.12 , pp. 669-679
    • Luca, T.1    Givogri, M.I.2    Perani, L.3
  • 4
    • 33847238458 scopus 로고    scopus 로고
    • Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer
    • Sevin C, Verot L, Benraiss A, et al. Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer. Gene Ther 2007; 14: 405-414.
    • (2007) Gene Ther , vol.14 , pp. 405-414
    • Sevin, C.1    Verot, L.2    Benraiss, A.3
  • 5
    • 33846022746 scopus 로고    scopus 로고
    • AAV1 Mediated Co-expression of formylglycine-generating enzyme and arylsulfatase A efficiently corrects sulfatide storage in a mouse model of metachromatic leukodystrophy
    • Kurai T, Hisayasu S, Kitagawa R, et al. AAV1 Mediated Co-expression of formylglycine-generating enzyme and arylsulfatase A efficiently corrects sulfatide storage in a mouse model of metachromatic leukodystrophy. Mol Ther 2007; 15: 38-43.
    • (2007) Mol Ther , vol.15 , pp. 38-43
    • Kurai, T.1    Hisayasu, S.2    Kitagawa, R.3
  • 6
    • 33750597616 scopus 로고    scopus 로고
    • Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice
    • Biffi A, Capotondo A, Fasano S, et al. Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. J Clin Invest 2006; 116: 3070-3082.
    • (2006) J Clin Invest , vol.116 , pp. 3070-3082
    • Biffi, A.1    Capotondo, A.2    Fasano, S.3
  • 7
    • 11144354757 scopus 로고    scopus 로고
    • Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified haematopoietic stem cells
    • Biffi A, De Palma M, Quattrini A, et al. Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified haematopoietic stem cells. J Clin Invest 2004; 113: 1118-1129.
    • (2004) J Clin Invest , vol.113 , pp. 1118-1129
    • Biffi, A.1    De Palma, M.2    Quattrini, A.3
  • 8
    • 20944444685 scopus 로고    scopus 로고
    • Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy
    • Matzner U, Herbst E, Hedayati KK, et al. Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy. Hum Mol Genet 2005; 14: 1139-1152.
    • (2005) Hum Mol Genet , vol.14 , pp. 1139-1152
    • Matzner, U.1    Herbst, E.2    Hedayati, K.K.3
  • 9
    • 33645452310 scopus 로고    scopus 로고
    • Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain
    • Hadaczek P, Kohutnicka M, Krauze MT, et al. Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain. Hum Gene Ther 2006; 17: 291-302.
    • (2006) Hum Gene Ther , vol.17 , pp. 291-302
    • Hadaczek, P.1    Kohutnicka, M.2    Krauze, M.T.3
  • 10
    • 45749146197 scopus 로고    scopus 로고
    • Biodistribution of adeno-associated virus type-2 in nonhuman primates after convection-enhanced delivery to brain
    • Cunningham J, Pivirotto P, Bringas J, et al. Biodistribution of adeno-associated virus type-2 in nonhuman primates after convection-enhanced delivery to brain. Mol Ther 2008; 16: 1267-1275.
    • (2008) Mol Ther , vol.16 , pp. 1267-1275
    • Cunningham, J.1    Pivirotto, P.2    Bringas, J.3
  • 11
    • 34447098114 scopus 로고    scopus 로고
    • Significantly increased lifespan and improved behavioural performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice
    • Fu H, Kang L, Jennings JS, et al. Significantly increased lifespan and improved behavioural performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice. Gene Ther 2007; 14: 1065-1077.
    • (2007) Gene Ther , vol.14 , pp. 1065-1077
    • Fu, H.1    Kang, L.2    Jennings, J.S.3
  • 12
    • 34047267343 scopus 로고    scopus 로고
    • Intrathecal enzyme replacement therapy: Successful treatment of brain disease via the cerebrospinal fluid
    • Dickson P, McEntee M, Vogler C, et al. Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid. Mol Genet Metab 2007; 91: 61-68.
    • (2007) Mol Genet Metab , vol.91 , pp. 61-68
    • Dickson, P.1    McEntee, M.2    Vogler, C.3
  • 13
    • 33646808142 scopus 로고    scopus 로고
    • Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice
    • Watson G, Bastacky J, Belichenko P, et al. Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice. Gene Ther 2006; 13: 917-925.
    • (2006) Gene Ther , vol.13 , pp. 917-925
    • Watson, G.1    Bastacky, J.2    Belichenko, P.3
  • 14
    • 0024362504 scopus 로고
    • Expression vector system based on the chicken beta-actin promoter directs efficient production of interleukin-5
    • Miyazaki J, Takaki S, Araki K, et al. Expression vector system based on the chicken beta-actin promoter directs efficient production of interleukin-5. Gene 1989; 79: 269-277.
    • (1989) Gene , vol.79 , pp. 269-277
    • Miyazaki, J.1    Takaki, S.2    Araki, K.3
  • 15
    • 0025796875 scopus 로고
    • Indiscriminate activity from the B19 parvovirus p6 promoter in nonpermissive cells
    • Liu JM, Fujii H, Green SW, et al. Indiscriminate activity from the B19 parvovirus p6 promoter in nonpermissive cells. Virology 1991; 182: 361-364.
    • (1991) Virology , vol.182 , pp. 361-364
    • Liu, J.M.1    Fujii, H.2    Green, S.W.3
  • 16
    • 2442737801 scopus 로고    scopus 로고
    • Purification of recombinant adeno-associated virus by iodixanol gradient ultracentrifugation allows rapid and reproducible preparation of vector stocks for gene transfer in the nervous system
    • Hermens WT, ter Brake O, Dijkhuizen PA, et al. Purification of recombinant adeno-associated virus by iodixanol gradient ultracentrifugation allows rapid and reproducible preparation of vector stocks for gene transfer in the nervous system. Hum Gene Ther 1999; 10: 1885-1891.
    • (1999) Hum Gene Ther , vol.10 , pp. 1885-1891
    • Hermens, W.T.1    ter Brake, O.2    Dijkhuizen, P.A.3
  • 17
    • 0035099437 scopus 로고    scopus 로고
    • In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: Correction of neuropathology and protection against learning impairments in affected mice
    • Consiglio A, Quattrini A,Martino S, et al. In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice. Nat Med 2001; 7: 310-316.
    • (2001) Nat Med , vol.7 , pp. 310-316
    • Consiglio, A.1    Quattrini, A.2    Martino, S.3
  • 18
    • 0347286956 scopus 로고    scopus 로고
    • Self-complementary adeno-associated virus serotype 2 vector: Global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain
    • Fu H, Muenzer J, Samulski RJ, et al. Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain. Mol Ther 2003; 8: 911-917.
    • (2003) Mol Ther , vol.8 , pp. 911-917
    • Fu, H.1    Muenzer, J.2    Samulski, R.J.3
  • 19
    • 4844221895 scopus 로고    scopus 로고
    • Lysosomal sulfatide storage in the brain of arylsulfatase A-deficient mice: Cellular alterations and topographic distribution
    • Wittke D, Hartmann D, Gieselmann V, et al. Lysosomal sulfatide storage in the brain of arylsulfatase A-deficient mice: cellular alterations and topographic distribution. Acta Neuropathol (Berl) 2004; 108: 261-271.
    • (2004) Acta Neuropathol (Berl) , vol.108 , pp. 261-271
    • Wittke, D.1    Hartmann, D.2    Gieselmann, V.3
  • 20
    • 70449158340 scopus 로고
    • A simple method for the isolation and purification of total lipides from animal tissues
    • Folch J, Lees M, Sloane Stanley GH. A simple method for the isolation and purification of total lipides from animal tissues. J Biol Chem 1957; 226: 497-509.
    • (1957) J Biol Chem , vol.226 , pp. 497-509
    • Folch, J.1    Lees, M.2    Sloane Stanley, G.H.3
  • 21
    • 22644438595 scopus 로고    scopus 로고
    • Gene transfer to dorsal root ganglia by intrathecal injection: Effects on regeneration of peripheral nerves
    • Wang X, Wang C, Zeng J, et al. Gene transfer to dorsal root ganglia by intrathecal injection: effects on regeneration of peripheral nerves. Mol Ther 2005; 12: 314-320.
    • (2005) Mol Ther , vol.12 , pp. 314-320
    • Wang, X.1    Wang, C.2    Zeng, J.3
  • 22
    • 0038618928 scopus 로고    scopus 로고
    • Efficiencies of transgene expression in nociceptive neurons through different routes of delivery of adeno-associated viral vectors
    • Xu Y, Gu Y, Wu P, et al. Efficiencies of transgene expression in nociceptive neurons through different routes of delivery of adeno-associated viral vectors. Hum Gene Ther 2003; 14: 897-906.
    • (2003) Hum Gene Ther , vol.14 , pp. 897-906
    • Xu, Y.1    Gu, Y.2    Wu, P.3
  • 23
    • 0030077356 scopus 로고    scopus 로고
    • Function of the two mannose 6-phosphate receptors in lysosomal enzyme transport
    • Munier-Lehmann H, Mauxion F, Hoflack B. Function of the two mannose 6-phosphate receptors in lysosomal enzyme transport. Biochem Soc Trans 1996; 24: 133-136.
    • (1996) Biochem Soc Trans , vol.24 , pp. 133-136
    • Munier-Lehmann, H.1    Mauxion, F.2    Hoflack, B.3
  • 24
    • 0032516810 scopus 로고    scopus 로고
    • Protein transport from the secretory to the endocytic pathway in mammalian cells
    • Le Borgne R, Hoflack B. Protein transport from the secretory to the endocytic pathway in mammalian cells. Biochim Biophys Acta 1998; 1404: 195-209.
    • (1998) Biochim Biophys Acta , vol.1404 , pp. 195-209
    • Le Borgne, R.1    Hoflack, B.2
  • 25
    • 0026637316 scopus 로고
    • Structure and function of the mannose 6-phosphate/ insulinlike growth factor II receptors
    • Kornfeld S. Structure and function of the mannose 6-phosphate/ insulinlike growth factor II receptors. Annu Rev Biochem 1992; 61: 307-330.
    • (1992) Annu Rev Biochem , vol.61 , pp. 307-330
    • Kornfeld, S.1
  • 26
    • 0036566195 scopus 로고    scopus 로고
    • Functional amelioration of murine galactosialidosis by genetically modified bone marrow haematopoietic progenitor cells
    • Leimig T, Mann L, Martin Mdel P, et al. Functional amelioration of murine galactosialidosis by genetically modified bone marrow haematopoietic progenitor cells. Blood 2002; 99: 3169-3178.
    • (2002) Blood , vol.99 , pp. 3169-3178
    • Leimig, T.1    Mann, L.2    Martin Mdel, P.3
  • 27
    • 33846873893 scopus 로고    scopus 로고
    • Lentiviral-mediated gene correction of mucopolysaccharidosis type IIIA
    • Anson DS, McIntyre C, Thomas B, et al. Lentiviral-mediated gene correction of mucopolysaccharidosis type IIIA. Genet Vaccines Ther 2007; 5: 1.
    • (2007) Genet Vaccines Ther , vol.5 , pp. 1
    • Anson, D.S.1    McIntyre, C.2    Thomas, B.3
  • 28
    • 0015262499 scopus 로고
    • The fine structure of cellular layers and connective tissue space at spinal nerve root attachments in the rat
    • Haller FR, Haller C, Low FN. The fine structure of cellular layers and connective tissue space at spinal nerve root attachments in the rat. Am J Anat 1972; 133: 109-123.
    • (1972) Am J Anat , vol.133 , pp. 109-123
    • Haller, F.R.1    Haller, C.2    Low, F.N.3
  • 29
    • 0015124885 scopus 로고
    • The fine structure of a lateral recess of the subarachnoid space in the rat
    • Himango WA, Low FN. The fine structure of a lateral recess of the subarachnoid space in the rat. Anat Rec 1971; 171: 1-19.
    • (1971) Anat Rec , vol.171 , pp. 1-19
    • Himango, W.A.1    Low, F.N.2
  • 30
    • 0016188838 scopus 로고
    • Scanning electron microscopy of the subarachnoid space in the dog. II. Spinal nerve exits
    • Malloy JJ, Low FN. Scanning electron microscopy of the subarachnoid space in the dog. II. Spinal nerve exits. J Comp Neurol 1974; 157: 87-107.
    • (1974) J Comp Neurol , vol.157 , pp. 87-107
    • Malloy, J.J.1    Low, F.N.2
  • 31
    • 0028353348 scopus 로고
    • Morphological indications for considerable diffuse reabsorption of cerebrospinal fluid in spinal meninges particularly in the areas of meningeal funnels. An electronmicroscopical study including tracing experiments in rats
    • Zenker W, Bankoul S, Braun JS. Morphological indications for considerable diffuse reabsorption of cerebrospinal fluid in spinal meninges particularly in the areas of meningeal funnels. An electronmicroscopical study including tracing experiments in rats. Anat Embryol (Berl) 1994; 189: 243-258.
    • (1994) Anat Embryol (Berl) , vol.189 , pp. 243-258
    • Zenker, W.1    Bankoul, S.2    Braun, J.S.3
  • 33
    • 27144545459 scopus 로고    scopus 로고
    • Intrathecal gene transfer by adeno-associated virus for pain
    • Beutler AS, Banck MS, Walsh CE, et al. Intrathecal gene transfer by adeno-associated virus for pain. Curr Opin Mol Ther 2005; 7: 431-439.
    • (2005) Curr Opin Mol Ther , vol.7 , pp. 431-439
    • Beutler, A.S.1    Banck, M.S.2    Walsh, C.E.3
  • 34
    • 30744436835 scopus 로고    scopus 로고
    • HSV-mediated gene transfer of vascular endothelial growth factor to dorsal root ganglia prevents diabetic neuropathy
    • Chattopadhyay M, Krisky D, Wolfe D, et al. HSV-mediated gene transfer of vascular endothelial growth factor to dorsal root ganglia prevents diabetic neuropathy. Gene Ther 2005; 12: 1377-1384.
    • (2005) Gene Ther , vol.12 , pp. 1377-1384
    • Chattopadhyay, M.1    Krisky, D.2    Wolfe, D.3
  • 35
    • 9244259139 scopus 로고    scopus 로고
    • Herpes vector-mediated gene transfer in treatment of diseases of the nervous system
    • Glorioso JC, Fink DJ. Herpes vector-mediated gene transfer in treatment of diseases of the nervous system. Annu Rev Microbiol 2004; 58: 253-271.
    • (2004) Annu Rev Microbiol , vol.58 , pp. 253-271
    • Glorioso, J.C.1    Fink, D.J.2
  • 36
    • 43049157976 scopus 로고    scopus 로고
    • Gene therapy directed at the neuroimmune component of chronic pain with particular attention to the role of TNFalpha
    • Mata M, Hao S, Fink DJ. Gene therapy directed at the neuroimmune component of chronic pain with particular attention to the role of TNFalpha. Neurosci Lett 2008; 437: 209-213.
    • (2008) Neurosci Lett , vol.437 , pp. 209-213
    • Mata, M.1    Hao, S.2    Fink, D.J.3
  • 37
    • 38649139370 scopus 로고    scopus 로고
    • Efficient retrograde neuronal transduction utilizing self-complementary AAV1
    • Hollis ER II, Kadoya K, Hirsch M, et al. Efficient retrograde neuronal transduction utilizing self-complementary AAV1. Mol Ther 2008; 16: 296-301.
    • (2008) Mol Ther , vol.16 , pp. 296-301
    • Hollis II, E.R.1    Kadoya, K.2    Hirsch, M.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.