메뉴 건너뛰기




Volumn 25, Issue 2, 2008, Pages 400-406

Lysosomal enzyme replacement of the brain with intravenous non-viral gene transfer

Author keywords

Blood brain barrier; Liposomes; Lysosomal storage disorders; Transferrin receptor

Indexed keywords

BETA GLUCURONIDASE; LIPOSOME; LYSOSOME ENZYME; MONOCLONAL ANTIBODY; PLASMID DNA; TRANSFERRIN RECEPTOR;

EID: 39149083089     PISSN: 07248741     EISSN: 1573904X     Source Type: Journal    
DOI: 10.1007/s11095-007-9357-6     Document Type: Article
Times cited : (51)

References (34)
  • 1
    • 0033925404 scopus 로고    scopus 로고
    • Gene therapy for lysosomal storage disorders with neuropathology
    • Y. A. Ioannou. Gene therapy for lysosomal storage disorders with neuropathology. J. Am. Soc. Nephrol. 11:1542-1547 (2000).
    • (2000) J. Am. Soc. Nephrol. , vol.11 , pp. 1542-1547
    • Ioannou, Y.A.1
  • 2
    • 0035845610 scopus 로고    scopus 로고
    • Intractable fever and cortical neuronal glycogen storage in glycogenosis type 2
    • C. Martini, G. Ciana, A. Benettoni, F. Katouzian, G. M. Severini, R. Bussani, et al. Intractable fever and cortical neuronal glycogen storage in glycogenosis type 2. Neurology 57:906-908 (2001).
    • (2001) Neurology , vol.57 , pp. 906-908
    • Martini, C.1    Ciana, G.2    Benettoni, A.3    Katouzian, F.4    Severini, G.M.5    Bussani, R.6
  • 3
    • 0032982480 scopus 로고    scopus 로고
    • Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector
    • S. S. Elliger, C. A. Elliger, C. P. Aguilar, N. R. Raju, and G. L. Watson. Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector. Gene Ther. 6:1175-1178 (1999).
    • (1999) Gene Ther. , vol.6 , pp. 1175-1178
    • Elliger, S.S.1    Elliger, C.A.2    Aguilar, C.P.3    Raju, N.R.4    Watson, G.L.5
  • 4
    • 0036932904 scopus 로고    scopus 로고
    • Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy
    • L. Xu, R. L. Mango, M. S. Sands, M. E. Haskins, N. M. Ellinwood, and K. P. Ponder. Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy. Mol. Ther. 6:745-758 (2002).
    • (2002) Mol. Ther. , vol.6 , pp. 745-758
    • Xu, L.1    Mango, R.L.2    Sands, M.S.3    Haskins, M.E.4    Ellinwood, N.M.5    Ponder, K.P.6
  • 5
    • 0035203152 scopus 로고    scopus 로고
    • Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector
    • M. A. Passini and J. H. Wolfe. Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector. J. Virol. 75:12382-12392 (2001).
    • (2001) J. Virol. , vol.75 , pp. 12382-12392
    • Passini, M.A.1    Wolfe, J.H.2
  • 6
    • 33745121193 scopus 로고    scopus 로고
    • Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII
    • B. A. Karolewski and J. H. Wolfe. Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII. Mol. Ther. 14:14-24 (2006).
    • (2006) Mol. Ther. , vol.14 , pp. 14-24
    • Karolewski, B.A.1    Wolfe, J.H.2
  • 7
    • 0032758906 scopus 로고    scopus 로고
    • Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice
    • A. F. Skorupa, K. J. Fisher, J. M. Wilson, M. K. Parente, and J. H. Wolfe. Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice. Exp. Neurol. 160:17-27 (1999).
    • (1999) Exp. Neurol. , vol.160 , pp. 17-27
    • Skorupa, A.F.1    Fisher, K.J.2    Wilson, J.M.3    Parente, M.K.4    Wolfe, J.H.5
  • 8
    • 0141996932 scopus 로고    scopus 로고
    • Adeno-associated virus vector-mediated transduction in the cat brain
    • C. H. Vite, M. A. Passini, M. E. Haskins, and J. H. Wolfe. Adeno-associated virus vector-mediated transduction in the cat brain. Gene Ther. 10:1874-1881 (2003).
    • (2003) Gene Ther. , vol.10 , pp. 1874-1881
    • Vite, C.H.1    Passini, M.A.2    Haskins, M.E.3    Wolfe, J.H.4
  • 9
    • 0037079071 scopus 로고    scopus 로고
    • Drug and gene delivery to the brain: The vascular route
    • W. M. Pardridge. Drug and gene delivery to the brain: the vascular route. Neuron 36:555-558 (2002).
    • (2002) Neuron , vol.36 , pp. 555-558
    • Pardridge, W.M.1
  • 11
    • 0034815482 scopus 로고    scopus 로고
    • Receptor-mediated gene targeting to tissues in vivo following intravenous administration of pegylated immunoliposomes
    • N. Shi, R. J. Boado, and W. M. Pardridge. Receptor-mediated gene targeting to tissues in vivo following intravenous administration of pegylated immunoliposomes. Pharm. Res. 18:1091-1095 (2001).
    • (2001) Pharm. Res. , vol.18 , pp. 1091-1095
    • Shi, N.1    Boado, R.J.2    Pardridge, W.M.3
  • 12
    • 0037222013 scopus 로고    scopus 로고
    • Global non-viral gene transfer to the primate brain following intravenous administration
    • Y. Zhang, F. Schlachetzki, and W.M. Pardridge. Global non-viral gene transfer to the primate brain following intravenous administration. Mol. Ther. 7:11-18 (2003).
    • (2003) Mol. Ther. , vol.7 , pp. 11-18
    • Zhang, Y.1    Schlachetzki, F.2    Pardridge, W.M.3
  • 13
    • 0025633324 scopus 로고
    • Characterization and distribution of transferrin receptors in the rat brain
    • D. C. Mash, J. Pablo, D. D. Flynn, S. M. Efange, and W. J. Weiner. Characterization and distribution of transferrin receptors in the rat brain. J. Neurochem. 55:1972-1979 (1990).
    • (1990) J. Neurochem. , vol.55 , pp. 1972-1979
    • Mash, D.C.1    Pablo, J.2    Flynn, D.D.3    Efange, S.M.4    Weiner, W.J.5
  • 14
    • 0033521130 scopus 로고    scopus 로고
    • Brain insulin receptors and spatial memory. Correlated changes in gene expression, tyrosine phosphorylation, and signaling molecules in the hippocampus of water maze trained rats
    • W. Zhao, H. Chen, H. Xu, E. Moore, N. Meiri, M. J. Quon, et al. Brain insulin receptors and spatial memory. Correlated changes in gene expression, tyrosine phosphorylation, and signaling molecules in the hippocampus of water maze trained rats. J. Biol. Chem. 274:34893-34902 (1999).
    • (1999) J. Biol. Chem. , vol.274 , pp. 34893-34902
    • Zhao, W.1    Chen, H.2    Xu, H.3    Moore, E.4    Meiri, N.5    Quon, M.J.6
  • 15
    • 0024524979 scopus 로고
    • Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency
    • E. H. Birkenmeier, M. T. Davisson, W. G. Beamer, R. E. Ganschow, C. A. Vogler, B. Gwynn, et al. Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency. J. Clin. Invest. 83:1258-1266 (1989).
    • (1989) J. Clin. Invest. , vol.83 , pp. 1258-1266
    • Birkenmeier, E.H.1    Davisson, M.T.2    Beamer, W.G.3    Ganschow, R.E.4    Vogler, C.A.5    Gwynn, B.6
  • 17
    • 13044258426 scopus 로고    scopus 로고
    • Pardridge. Selective expression of the large neutral amino acid transporter at the blood-brain barrier
    • R. J. Boado, J. Y. Li, M. Nagaya, C. Zhang, and W. M. Pardridge. Selective expression of the large neutral amino acid transporter at the blood-brain barrier. Proc. Natl. Acad. Sci. U.S.A. 96:12079-12084 (1999).
    • (1999) Proc. Natl. Acad. Sci. U.S.A. , vol.96 , pp. 12079-12084
    • Boado, R.J.1    Li, J.Y.2    Nagaya, M.3    Zhang, C.4    Pardridge, W.M.5
  • 18
    • 24044468014 scopus 로고    scopus 로고
    • Site-directed mutagenesis of cysteine residues of large neutral amino acid transporter LAT1
    • R. J. Boado, J. Y. Li, C. Chu, F. Ogoshi, P. Wise, and W. M. Pardridge. Site-directed mutagenesis of cysteine residues of large neutral amino acid transporter LAT1. Biochim. Biophys. Acta 1715:104-110 (2005).
    • (2005) Biochim. Biophys. Acta , vol.1715 , pp. 104-110
    • Boado, R.J.1    Li, J.Y.2    Chu, C.3    Ogoshi, F.4    Wise, P.5    Pardridge, W.M.6
  • 19
    • 1342332987 scopus 로고    scopus 로고
    • Gene targeting in vivo with pegylated immunoliposomes
    • W. M. Pardridge. Gene targeting in vivo with pegylated immunoliposomes. Methods Enzymol. 373:507-528 (2003).
    • (2003) Methods Enzymol. , vol.373 , pp. 507-528
    • Pardridge, W.M.1
  • 20
    • 0003312375 scopus 로고    scopus 로고
    • Marked enhancement in gene expression by targeting the human insulin receptor
    • Y. Zhang, R. J. Boado, and W. M. Pardridge. Marked enhancement in gene expression by targeting the human insulin receptor. J. Genet. Med. 5:157-163 (2003).
    • (2003) J. Genet. Med. , vol.5 , pp. 157-163
    • Zhang, Y.1    Boado, R.J.2    Pardridge, W.M.3
  • 21
    • 0027505029 scopus 로고
    • C-terminal processing of human beta-glucuronidase. the propeptide is required for full expression of catalytic activity, intracellular retention, and proper phosphorylation
    • M. R. Islam, J. H. Grubb, and W. S. Sly. C-terminal processing of human beta-glucuronidase. The propeptide is required for full expression of catalytic activity, intracellular retention, and proper phosphorylation. J. Biol. Chem. 268:22627-22633 (1993).
    • (1993) J. Biol. Chem. , vol.268 , pp. 22627-22633
    • Islam, M.R.1    Grubb, J.H.2    Sly, W.S.3
  • 22
    • 0028837306 scopus 로고
    • High level expression and export of beta-glucuronidase from murine mucopolysaccharidosis VII cells corrected by a double-copy retrovirus vector
    • J. H. Wolfe, J. W. Kyle, M. S. Sands, W. S. Sly, D. G. Markowitz, and M. K. Parente. High level expression and export of beta-glucuronidase from murine mucopolysaccharidosis VII cells corrected by a double-copy retrovirus vector. Gene Ther. 2:70-78 (1995).
    • (1995) Gene Ther. , vol.2 , pp. 70-78
    • Wolfe, J.H.1    Kyle, J.W.2    Sands, M.S.3    Sly, W.S.4    Markowitz, D.G.5    Parente, M.K.6
  • 23
    • 0033551773 scopus 로고    scopus 로고
    • Active site residues of human beta-glucuronidase. Evidence for Glu(540) as the nucleophile and Glu(451) as the acid-base residue
    • M. R. Islam, S. Tomatsu, G. N. Shah, J. H. Grubb, S. Jain, and W. S. Sly. Active site residues of human beta-glucuronidase. Evidence for Glu(540) as the nucleophile and Glu(451) as the acid-base residue. J. Biol. Chem. 274:23451-23455 (1999).
    • (1999) J. Biol. Chem. , vol.274 , pp. 23451-23455
    • Islam, M.R.1    Tomatsu, S.2    Shah, G.N.3    Grubb, J.H.4    Jain, S.5    Sly, W.S.6
  • 25
    • 0028093249 scopus 로고
    • Overexpression rescues the mutant phenotype of L176F mutation causing β-glucuronidase deficiency mucopolysaccharidosis in two Mennonite siblings
    • B. J. Wu, S. Tomatsu, S. Fukuda, K. Sukegawa, T. Orii, and W. S. Sly. Overexpression rescues the mutant phenotype of L176F mutation causing β-glucuronidase deficiency mucopolysaccharidosis in two Mennonite siblings. J. Biol. Chem. 269:23681-23688 (1994).
    • (1994) J. Biol. Chem. , vol.269 , pp. 23681-23688
    • Wu, B.J.1    Tomatsu, S.2    Fukuda, S.3    Sukegawa, K.4    Orii, T.5    Sly, W.S.6
  • 26
  • 27
    • 0029000058 scopus 로고
    • Targeting of an anti-CR3 (CD11b/CD18) monoclonal antibody to spleen, but not brain, in vivo in mice
    • S. Skarlatos, and W. M. Pardridge. Targeting of an anti-CR3 (CD11b/CD18) monoclonal antibody to spleen, but not brain, in vivo in mice. J. Drug Target. 3:9-14 (1995).
    • (1995) J. Drug Target. , vol.3 , pp. 9-14
    • Skarlatos, S.1    Pardridge, W.M.2
  • 28
    • 26844431562 scopus 로고    scopus 로고
    • Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors
    • G. Liu, I. Martins, J. A. Wemmie, J. A. Chiorini, and B. L. Davidson. Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors. J. Neurosci. 25:9321-9327 (2005).
    • (2005) J. Neurosci. , vol.25 , pp. 9321-9327
    • Liu, G.1    Martins, I.2    Wemmie, J.A.3    Chiorini, J.A.4    Davidson, B.L.5
  • 29
    • 0034690714 scopus 로고    scopus 로고
    • Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer
    • A. Bosch, E. Perret, N. Desmaris, D. Trono, and J. M. Heard. Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer. Hum. Gene Ther. 11:1139-1150 (2000).
    • (2000) Hum. Gene Ther. , vol.11 , pp. 1139-1150
    • Bosch, A.1    Perret, E.2    Desmaris, N.3    Trono, D.4    Heard, J.M.5
  • 30
    • 28444483094 scopus 로고    scopus 로고
    • Stable levels of long-term transgene expression driven by the latency-associated transcript promoter in a herpes simplex virus type 1 vector
    • B. K. Berges, J. H. Wolfe, and N. W. Fraser. Stable levels of long-term transgene expression driven by the latency-associated transcript promoter in a herpes simplex virus type 1 vector. Mol. Ther. 12:1111-1119 (2005).
    • (2005) Mol. Ther. , vol.12 , pp. 1111-1119
    • Berges, B.K.1    Wolfe, J.H.2    Fraser, N.W.3
  • 31
    • 0035900762 scopus 로고    scopus 로고
    • Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII
    • M. S. Sands, C. A. Vogler, K. K. Ohlemiller, M. S. Roberts, J. H. Grubb, B. Levy, et al. Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII. J. Biol. Chem. 276:43160-43165 (2001).
    • (2001) J. Biol. Chem. , vol.276 , pp. 43160-43165
    • Sands, M.S.1    Vogler, C.A.2    Ohlemiller, K.K.3    Roberts, M.S.4    Grubb, J.H.5    Levy, B.6
  • 32
    • 4344630950 scopus 로고    scopus 로고
    • Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier
    • A. Urayama, J. H. Grubb, W. S. Sly, and W. A. Banks. Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier. Proc. Natl. Acad. Sci. U.S.A. 101:12658-12663 (2004).
    • (2004) Proc. Natl. Acad. Sci. U.S.A. , vol.101 , pp. 12658-12663
    • Urayama, A.1    Grubb, J.H.2    Sly, W.S.3    Banks, W.A.4
  • 33
    • 0037418258 scopus 로고    scopus 로고
    • Transgene produces massive overexpression of human beta-glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors
    • C. Vogler, N. Galvin, B. Levy, J. Grubb, J. Jiang, X. Y. Zhou, and W. S Sly. Transgene produces massive overexpression of human beta-glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors. Proc. Natl. Acad. Sci. U.S.A. 100:2669-2673 (2003).
    • (2003) Proc. Natl. Acad. Sci. U.S.A. , vol.100 , pp. 2669-2673
    • Vogler, C.1    Galvin, N.2    Levy, B.3    Grubb, J.4    Jiang, J.5    Zhou, X.Y.6    Sly, W.S.7
  • 34
    • 33745591896 scopus 로고    scopus 로고
    • Decline in exogenous gene expression in primate brain following intravenous administration is due to plasmid degradation
    • C. Chu, Y. Zhang, R. J. Boado, and W. M. Pardridge. Decline in exogenous gene expression in primate brain following intravenous administration is due to plasmid degradation. Pharm. Res. 23:1586-1590 (2006).
    • (2006) Pharm. Res. , vol.23 , pp. 1586-1590
    • Chu, C.1    Zhang, Y.2    Boado, R.J.3    Pardridge, W.M.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.