메뉴 건너뛰기




Volumn 55, Issue , 2003, Pages 271-296

Gene therapy for mucopolysaccharidosis

Author keywords

[No Author keywords available]

Indexed keywords

ANIMAL; GENE THERAPY; HUMAN; METHODOLOGY; MUCOPOLYSACCHARIDOSIS; REVIEW;

EID: 0141818018     PISSN: 00747742     EISSN: None     Source Type: Book Series    
DOI: 10.1016/S0074-7742(03)01012-2     Document Type: Article
Times cited : (9)

References (107)
  • 2
    • 0036166251 scopus 로고    scopus 로고
    • A deviant immune response to viral proteins and transgene product is generated on subretinal administration of adenovirus and adeno-associated virus
    • Anand, V., Duffy, B., Yang, Z., Dejneka, N. S., Maguire, A. M., and Bennett, J. (2002). A deviant immune response to viral proteins and transgene product is generated on subretinal administration of adenovirus and adeno-associated virus. Mol. Ther. 5, 125-132.
    • (2002) Mol. Ther. , vol.5 , pp. 125-132
    • Anand, V.1    Duffy, B.2    Yang, Z.3    Dejneka, N.S.4    Maguire, A.M.5    Bennett, J.6
  • 3
    • 0026904369 scopus 로고
    • Correction of mucopolysaccharidosis type I fibroblasts by retroviral-mediated transfer of the human alpha-L-iduronidase gene
    • Anson, D. S., Bielicki, J., and Hopwood, J. J. (1992). Correction of mucopolysaccharidosis type I fibroblasts by retroviral-mediated transfer of the human alpha-L-iduronidase gene. Hum. Gene Ther. 3, 371-379.
    • (1992) Hum. Gene Ther. , vol.3 , pp. 371-379
    • Anson, D.S.1    Bielicki, J.2    Hopwood, J.J.3
  • 4
    • 0035874875 scopus 로고    scopus 로고
    • Molecular basis of mucopolysaccharidosis type III B in emu (Dromaius novaenollandiae): An avian model of Sanfilippo syndrome type B
    • Aronovich, E. L., Johnston, J. M., Wang, P., Giger, U., and Whitley, C. B. (2001). Molecular basis of mucopolysaccharidosis type III B in emu (Dromaius novaenollandiae): an avian model of Sanfilippo syndrome type B. Genomics 74, 299-305.
    • (2001) Genomics , vol.74 , pp. 299-305
    • Aronovich, E.L.1    Johnston, J.M.2    Wang, P.3    Giger, U.4    Whitley, C.B.5
  • 5
    • 0026580688 scopus 로고
    • Immunoquantification and enzyme kinetics of alpha-L-iduronidase in cultured fibroblasts from normal controls and mucopolysaccharidosis type I patients
    • Ashton, L. J., Brooks, D. A., McCourt, P. A., Muller, V. J., Clements, P. R., and Hopwood, J. J. (1992). Immunoquantification and enzyme kinetics of alpha-L-iduronidase in cultured fibroblasts from normal controls and mucopolysaccharidosis type I patients. Am. J. Hum. Genet. 50, 787-794.
    • (1992) Am. J. Hum. Genet. , vol.50 , pp. 787-794
    • Ashton, L.J.1    Brooks, D.A.2    McCourt, P.A.3    Muller, V.J.4    Clements, P.R.5    Hopwood, J.J.6
  • 8
    • 0028070868 scopus 로고
    • Behavioral consequences of bone marrow transplantation in the treatment of murine mucopolysaccharidosis type VII
    • Bastedo, L., Sands, M. S., Lambert, D. T., Pisa, M. A., Birkenmeier, E., and Chang, P. L. (1994). Behavioral consequences of bone marrow transplantation in the treatment of murine mucopolysaccharidosis type VII. J. Clin. Invest. 94, 1180-1186.
    • (1994) J. Clin. Invest. , vol.94 , pp. 1180-1186
    • Bastedo, L.1    Sands, M.S.2    Lambert, D.T.3    Pisa, M.A.4    Birkenmeier, E.5    Chang, P.L.6
  • 10
    • 85011444686 scopus 로고
    • Correction of murine mucopolysaccharidosis type VII (MPS VII) by bone marrow transplantation and gene transfer therapy
    • Birkenmeier, E. H. (1991). Correction of murine mucopolysaccharidosis type VII (MPS VII) by bone marrow transplantation and gene transfer therapy. Hum. Gene Ther. 2, 113.
    • (1991) Hum. Gene Ther. , vol.2 , pp. 113
    • Birkenmeier, E.H.1
  • 12
    • 0033642895 scopus 로고    scopus 로고
    • Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors
    • Bosch, A., Perret, E., Desmaris, N., and Heard, J. M. (2000a). Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors. Mol. Ther. 1, 63-70.
    • (2000) Mol. Ther. , vol.1 , pp. 63-70
    • Bosch, A.1    Perret, E.2    Desmaris, N.3    Heard, J.M.4
  • 13
    • 0034690714 scopus 로고    scopus 로고
    • Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer
    • Bosch, A., Perret, E., Desmaris, N., Trono, D., and Heard, J. M. (2000b). Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer. Hum. Gene Ther. 11, 1139-1150.
    • (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
  • 15
    • 0025779840 scopus 로고
    • Analysis of N-acetylgalactosamine-4-sulfatase protein and kinetics in mucopolysaccharidosis type VI patients
    • Brooks, D. A., McCourt, P. A., Gibson, G. J., Ashton, L. J., Shutter, M., and Hopwood, J. J. (1991). Analysis of N-acetylgalactosamine-4-sulfatase protein and kinetics in mucopolysaccharidosis type VI patients. Am. J. Hum. Genet. 48, 710-719.
    • (1991) Am. J. Hum. Genet. , vol.48 , pp. 710-719
    • Brooks, D.A.1    McCourt, P.A.2    Gibson, G.J.3    Ashton, L.J.4    Shutter, M.5    Hopwood, J.J.6
  • 16
    • 0031927424 scopus 로고    scopus 로고
    • Immune response to enzyme replacement therapy: Clinical signs of hypersensitivity reactions and altered enzyme distribution in a high titre rat model
    • Brooks, D. A., Hopwood, J. J., and King, B. M. (1998). Immune response to enzyme replacement therapy: Clinical signs of hypersensitivity reactions and altered enzyme distribution in a high titre rat model. Biochim. Biophys. Acta 1407, 163-172.
    • (1998) Biochim. Biophys. Acta , vol.1407 , pp. 163-172
    • Brooks, D.A.1    Hopwood, J.J.2    King, B.M.3
  • 17
    • 0033831002 scopus 로고    scopus 로고
    • Lentiviral vector transduction of hematopoietic stem cells that mediate long-term reconstitution of lethally irradiated mice
    • Chen, W., Wu, X., Levasseur, D. N., Liu, H., Lai, L., Kappes, J. C., and Townes, T. M. (2000). Lentiviral vector transduction of hematopoietic stem cells that mediate long-term reconstitution of lethally irradiated mice. Stem Cells 18, 352-359.
    • (2000) Stem Cells , vol.18 , pp. 352-359
    • Chen, W.1    Wu, X.2    Levasseur, D.N.3    Liu, H.4    Lai, L.5    Kappes, J.C.6    Townes, T.M.7
  • 19
    • 0032906299 scopus 로고    scopus 로고
    • Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice
    • Daly, T. M., Okuyama, T., Vogler, C., Haskins, M. E., Muzyczka, N., and Sands, M. S. (1999a). Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice. Hum. Gene Ther. 10, 85-94.
    • (1999) Hum. Gene Ther. , vol.10 , pp. 85-94
    • Daly, T.M.1    Okuyama, T.2    Vogler, C.3    Haskins, M.E.4    Muzyczka, N.5    Sands, M.S.6
  • 20
    • 0033515007 scopus 로고    scopus 로고
    • Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease
    • Daly, T. M., Vogler, C., Levy, B., Haskins, M. E., and Sands, M. S. (1999b). Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease. Proc. Natl. Acad. Sci. USA 96, 2296-2300.
    • (1999) Proc. Natl. Acad. Sci. USA , vol.96 , pp. 2296-2300
    • Daly, T.M.1    Vogler, C.2    Levy, B.3    Haskins, M.E.4    Sands, M.S.5
  • 21
    • 0034824239 scopus 로고    scopus 로고
    • Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer
    • Daly, T. M., Ohlemiller, K. K., Roberts, M. S., Vogler, C. A., and Sands, M. S. (2001). Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer. Gene Ther. 8, 1291-1298.
    • (2001) Gene Ther. , vol.8 , pp. 1291-1298
    • Daly, T.M.1    Ohlemiller, K.K.2    Roberts, M.S.3    Vogler, C.A.4    Sands, M.S.5
  • 22
    • 0034724209 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system
    • Davidson, B. L., Stein, C. S., Heth, J. A., Martins, I., Kotin, R. M., Derksen, T. A., Zabner, J., Ghodsi, A., and Chiorini, J. A. (2000). Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system. Proc. Natl. Acad. Sci. USA 97, 3428-3432.
    • (2000) Proc. Natl. Acad. Sci. USA , vol.97 , pp. 3428-3432
    • Davidson, B.L.1    Stein, C.S.2    Heth, J.A.3    Martins, I.4    Kotin, R.M.5    Derksen, T.A.6    Zabner, J.7    Ghodsi, A.8    Chiorini, J.A.9
  • 23
    • 0017185722 scopus 로고
    • Enzyme replacement therapy by fibroblast transplantation in a case of Hunter syndrome
    • Dean, M. F., Muir, H., Benson, P. F., Button, L. R., Boylston, A., and Mowbray, J. (1976). Enzyme replacement therapy by fibroblast transplantation in a case of Hunter syndrome. Nature 261, 323-325.
    • (1976) Nature , vol.261 , pp. 323-325
    • Dean, M.F.1    Muir, H.2    Benson, P.F.3    Button, L.R.4    Boylston, A.5    Mowbray, J.6
  • 24
    • 0029846012 scopus 로고    scopus 로고
    • Central nervous system delivery of recombinant ciliary neurotrophic factor by polymer encapsulated differentiated C2C12 myoblasts
    • Déglon, N., Heyd, B., Tan, S. A., Joseph, J. M., Zum, A. D., and Aebischer, P. (1996). Central nervous system delivery of recombinant ciliary neurotrophic factor by polymer encapsulated differentiated C2C12 myoblasts. Hum. Gent Ther. 7, 2135-2146.
    • (1996) Hum. Gent Ther. , vol.7 , pp. 2135-2146
    • Déglon, N.1    Heyd, B.2    Tan, S.A.3    Joseph, J.M.4    Zum, A.D.5    Aebischer, P.6
  • 27
    • 0032982480 scopus 로고    scopus 로고
    • Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adenoassociated virus vector
    • Elliger, S. S., Eiliger, C. A., Aguilar, C. P., Raju, N. R., and Watson, G. L. (1999). Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adenoassociated virus vector. Gene Ther. 6, 1175-1178.
    • (1999) Gene Ther. , vol.6 , pp. 1175-1178
    • Elliger, S.S.1    Eiliger, C.A.2    Aguilar, C.P.3    Raju, N.R.4    Watson, G.L.5
  • 32
    • 0014352329 scopus 로고
    • Hurler and Hunter syndromes: Mutual correction of the defect in cultured fibroblasts
    • Fratantoni, J. C., Hall, C. W., and Neufeld, E. F. (1968). Hurler and Hunter syndromes: Mutual correction of the defect in cultured fibroblasts. Science 162, 570-572.
    • (1968) Science , vol.162 , pp. 570-572
    • Fratantoni, J.C.1    Hall, C.W.2    Neufeld, E.F.3
  • 33
    • 0033567908 scopus 로고    scopus 로고
    • Behavior and therapeutic efficacy of beta-glucuronidase-positive mononuclear phagocytes in a murine model of mucopolysaccharidosis type VII
    • Freeman, B. J., Roberts, M. S., Vogler, C. A., Nicholes, A., Hofling, A. A., and Sands, M. S. (1999). Behavior and therapeutic efficacy of beta-glucuronidase-positive mononuclear phagocytes in a murine model of mucopolysaccharidosis type VII. Blood 94, 2142-2150.
    • (1999) Blood , vol.94 , pp. 2142-2150
    • Freeman, B.J.1    Roberts, M.S.2    Vogler, C.A.3    Nicholes, A.4    Hofling, A.A.5    Sands, M.S.6
  • 34
    • 0034986419 scopus 로고    scopus 로고
    • Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII
    • Frisella, W. A., O'Connor, L. H., Vogler, C. A., Roberts, M., Walkley, S., Levy, B., Daly, T. M., and Sands, M. S. (2001). Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII. Mol. Ther. 3, 351-358.
    • (2001) Mol. Ther. , vol.3 , pp. 351-358
    • Frisella, W.A.1    O'Connor, L.H.2    Vogler, C.A.3    Roberts, M.4    Walkley, S.5    Levy, B.6    Daly, T.M.7    Sands, M.S.8
  • 36
    • 0032213413 scopus 로고    scopus 로고
    • Extensive beta-glucuronidase activity in murine central nervous system after adenovirus-mediated gene transfer to brain
    • Ghodsi, A., Stein, C., Derksen, T., Yang, G., Anderson, R. D., and Davidson, B. L. (1998). Extensive beta-glucuronidase activity in murine central nervous system after adenovirus-mediated gene transfer to brain. Hum. Gene Ther. 9, 2331-2340.
    • (1998) Hum. Gene Ther. , vol.9 , pp. 2331-2340
    • Ghodsi, A.1    Stein, C.2    Derksen, T.3    Yang, G.4    Anderson, R.D.5    Davidson, B.L.6
  • 37
    • 0032728371 scopus 로고    scopus 로고
    • Systemic hyperosmolality improves beta-glucuronidase distribution and pathology in murine MPS VII brain following intraventricular gene transfer
    • Ghodsi, A., Stein, C., Derksen, T., Martins, I., Anderson, R. D., and Davidson, B. L. (1999). Systemic hyperosmolality improves beta-glucuronidase distribution and pathology in murine MPS VII brain following intraventricular gene transfer. Exp. Neurol. 160, 109-116.
    • (1999) Exp. Neurol. , vol.160 , pp. 109-116
    • Ghodsi, A.1    Stein, C.2    Derksen, T.3    Martins, I.4    Anderson, R.D.5    Davidson, B.L.6
  • 38
    • 0031868229 scopus 로고    scopus 로고
    • Enzyme therapy for Gaucher disease: The first 5 years
    • Grabowski, G. A., Leslie, N., and Wenstrup, R. (1998). Enzyme therapy for Gaucher disease: The first 5 years. Blood Rev. 12, 115-133.
    • (1998) Blood Rev. , vol.12 , pp. 115-133
    • Grabowski, G.A.1    Leslie, N.2    Wenstrup, R.3
  • 40
    • 0021213609 scopus 로고
    • Beta-glucuronidase deficiency in a dog: A model of human mucopolysaccharidosis VII
    • Haskins, M. E., Desnick, R. J., DiFerrante, N., Jezyk, P. F., and Patterson, D. F. (1984). Beta-glucuronidase deficiency in a dog: A model of human mucopolysaccharidosis VII. Pediatr. Res. 18, 980-984.
    • (1984) Pediatr. Res. , vol.18 , pp. 980-984
    • Haskins, M.E.1    Desnick, R.J.2    Diferrante, N.3    Jezyk, P.F.4    Patterson, D.F.5
  • 41
    • 0023240272 scopus 로고
    • Correction of lysosomal enzyme deficiency in various organs of beta-glucuronidase-deficient mice by allogeneic bone marrow transplantation
    • Hoogerbrugge, P. M., Poorthuis, B. J., Mulder, A. H., Wagemaker, G., Dooren, L. J., Vossen, J. M., and van Bekkum, D. W. (1987). Correction of lysosomal enzyme deficiency in various organs of beta-glucuronidase-deficient mice by allogeneic bone marrow transplantation. Transplantation 43, 609-614.
    • (1987) Transplantation , vol.43 , pp. 609-614
    • Hoogerbrugge, P.M.1    Poorthuis, B.J.2    Mulder, A.H.3    Wagemaker, G.4    Dooren, L.J.5    Vossen, J.M.6    Van Bekkum, D.W.7
  • 43
    • 0036157645 scopus 로고    scopus 로고
    • Viral-mediated gene transfer to mouse primary neural progenitor cells
    • Hughes, S. M., Moussavi-Harami, F., Sauter, S. L., and Davidson, B. L. (2002). Viral-mediated gene transfer to mouse primary neural progenitor cells. Mol. Ther. 5, 16-24.
    • (2002) Mol. Ther. , vol.5 , pp. 16-24
    • Hughes, S.M.1    Moussavi-Harami, F.2    Sauter, S.L.3    Davidson, B.L.4
  • 44
    • 0017758232 scopus 로고
    • Mucopolysaccharidosis in a cat with arylsulfatase B deficiency: A model of Maroteaux-Lamy syndrome
    • Jezyk, P. F., Haskins, M. E., Patterson, D. F., Mellman, W. J., and Greenstein, M. (1977). Mucopolysaccharidosis in a cat with arylsulfatase B deficiency: A model of Maroteaux-Lamy syndrome. Science 198, 834-836.
    • (1977) Science , vol.198 , pp. 834-836
    • Jezyk, P.F.1    Haskins, M.E.2    Patterson, D.F.3    Mellman, W.J.4    Greenstein, M.5
  • 48
    • 0034756448 scopus 로고    scopus 로고
    • Adenovirus-mediated gene therapy for corneal clouding in mice with mucopolysaccharidosis type VII
    • Kamata, Y., Okuyama, T., Kosuga, M., O'Hira, A., Kanaji, A., Sasaki, K., Yamada, M., and Azuma, N. (2001). Adenovirus-mediated gene therapy for corneal clouding in mice with mucopolysaccharidosis type VII. Mol. Ther. 4, 307-312.
    • (2001) Mol. Ther. , vol.4 , pp. 307-312
    • Kamata, Y.1    Okuyama, T.2    Kosuga, M.3    O'Hira, A.4    Kanaji, A.5    Sasaki, K.6    Yamada, M.7    Azuma, N.8
  • 49
    • 0011596655 scopus 로고
    • Phosphohexosyl components of a lysosomal enzyme are recognized by pinocytosis receptors on human fibroblasts
    • Kaplan, A., Achord, D. T., and Sly, W. S. (1977). Phosphohexosyl components of a lysosomal enzyme are recognized by pinocytosis receptors on human fibroblasts. Proc. Natl. Acad. Sci. USA 74, 2026-2030.
    • (1977) Proc. Natl. Acad. Sci. USA , vol.74 , pp. 2026-2030
    • Kaplan, A.1    Achord, D.T.2    Sly, W.S.3
  • 53
    • 0029114944 scopus 로고
    • The future for treatment by bone marrow transplantation for adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome
    • Krivit, W., Lockman, L. A., Watkins, P. A., Hirsch, J., and Shapiro, E. G. (1995). The future for treatment by bone marrow transplantation for adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome. J. Inherit. Metab. Dis. 18, 398-112.
    • (1995) J. Inherit. Metab. Dis. , vol.18 , pp. 398-1112
    • Krivit, W.1    Lockman, L.A.2    Watkins, P.A.3    Hirsch, J.4    Shapiro, E.G.5
  • 55
    • 0029166226 scopus 로고
    • Phenotype correction in retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer
    • Li, T., and Davidson, B. L. (1995). Phenotype correction in retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer. Proc. Natl. Acad. Sci. USA. 92, 7700-7704.
    • (1995) Proc. Natl. Acad. Sci. USA , vol.92 , pp. 7700-7704
    • Li, T.1    Davidson, B.L.2
  • 56
    • 0027178735 scopus 로고
    • Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically modified bone marrow cells
    • Maréchal, V., Naffakh, N., Danos, O., and Heard, J. M. (1993). Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically modified bone marrow cells. Blood 82, 1358-1365.
    • (1993) Blood , vol.82 , pp. 1358-1365
    • Maréchal, V.1    Naffakh, N.2    Danos, O.3    Heard, J.M.4
  • 58
    • 0027231473 scopus 로고
    • Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts
    • Moullier, P., Bohl, D., Heard, J. M., and Danos, O. (1993a). Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts. Nat. Genet. 4, 154-159.
    • (1993) Nat. Genet. , vol.4 , pp. 154-159
    • Moullier, P.1    Bohl, D.2    Heard, J.M.3    Danos, O.4
  • 59
    • 0027201986 scopus 로고
    • Continuous systemic secretion of a lysosomal enzyme by genetically modified mouse skin fibroblasts
    • Moullier, P., Maréchal, V., Danos, O., and Heard, J. M. (1993b). Continuous systemic secretion of a lysosomal enzyme by genetically modified mouse skin fibroblasts. Transplantation 56, 427-132.
    • (1993) Transplantation , vol.56 , pp. 427-1132
    • Moullier, P.1    Maréchal, V.2    Danos, O.3    Heard, J.M.4
  • 60
    • 0028915224 scopus 로고
    • Long-term delivery of a lysosomal enzyme by genetically modified fibroblasts in dogs
    • Moullier, P., Bohl, D., Cardoso, J., Heard, J. M., and Danos, O. (1995). Long-term delivery of a lysosomal enzyme by genetically modified fibroblasts in dogs. Nat. Med. 1, 353-357.
    • (1995) Nat. Med. , vol.1 , pp. 353-357
    • Moullier, P.1    Bohl, D.2    Cardoso, J.3    Heard, J.M.4    Danos, O.5
  • 61
    • 0000561621 scopus 로고    scopus 로고
    • Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function
    • O'Connor, L. H., Erway, L. C., Vogler, C. A., Sly, W. S., Nicholes, A., Grubb, J., Holmberg, S. W., Levy, B., and Sands, M. S. (1998). Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function. J. Clin. Invest. 101, 1394-1400.
    • (1998) J. Clin. Invest. , vol.101 , pp. 1394-1400
    • O'Connor, L.H.1    Erway, L.C.2    Vogler, C.A.3    Sly, W.S.4    Nicholes, A.5    Grubb, J.6    Holmberg, S.W.7    Levy, B.8    Sands, M.S.9
  • 62
    • 0036155063 scopus 로고    scopus 로고
    • Neurotropism and retrograde axonal transport of a canine adenoviral vector: A tool for targeting key structures undergoing neurodegenerative processes
    • Peltékian, E., Garcia, L., and Danos, O. (2002). Neurotropism and retrograde axonal transport of a canine adenoviral vector: A tool for targeting key structures undergoing neurodegenerative processes. Mol. Ther. 5, 25-32.
    • (2002) Mol. Ther. , vol.5 , pp. 25-32
    • Peltékian, E.1    Garcia, L.2    Danos, O.3
  • 63
    • 0025797623 scopus 로고
    • Restoration of arylsulphatase B activity in human mucopolysaccharidosis- type-VI fibroblasts by retroviral-vector-mediated gene transfer
    • Peters, C., Rommerskirch, W., Modaressi, S., and von Figura, K. (1991). Restoration of arylsulphatase B activity in human mucopolysaccharidosis-type-VI fibroblasts by retroviral-vector-mediated gene transfer. Biochem. J. 276, 499-504.
    • (1991) Biochem. J. , vol.276 , pp. 499-504
    • Peters, C.1    Rommerskirch, W.2    Modaressi, S.3    Von Figura, K.4
  • 66
    • 0026095924 scopus 로고
    • Targeting of proteins to the lysosome
    • Pfeffer, S. R. (1991). Targeting of proteins to the lysosome. Curr. Top. Microbiol. Immunol. 70, 43-65.
    • (1991) Curr. Top. Microbiol. Immunol. , vol.70 , pp. 43-65
    • Pfeffer, S.R.1
  • 67
    • 0028260714 scopus 로고
    • Bone marrow transplantation has a significant effect on enzyme levels and storage of glycosaminoglycans in tissues and in isolated hepatocytes of mucopolysaccharidosis type VII mice
    • Poorthuis, B. J., Romme, A. E., Willemsen, R., and Wagemaker, G. (1994). Bone marrow transplantation has a significant effect on enzyme levels and storage of glycosaminoglycans in tissues and in isolated hepatocytes of mucopolysaccharidosis type VII mice. Pediatr. Res. 36, 187-193.
    • (1994) Pediatr. Res. , vol.36 , pp. 187-193
    • Poorthuis, B.J.1    Romme, A.E.2    Willemsen, R.3    Wagemaker, G.4
  • 69
    • 0032928402 scopus 로고    scopus 로고
    • Delivery of recombinant gene products to the central nervous system with nonautologous cells in alginate microcapsules
    • Ross, C. J., Ralph, M., and Chang, P. L. (1999). Delivery of recombinant gene products to the central nervous system with nonautologous cells in alginate microcapsules. Hum. Gene Ther. 10, 49-59.
    • (1999) Hum. Gene Ther. , vol.10 , pp. 49-59
    • Ross, C.J.1    Ralph, M.2    Chang, P.L.3
  • 70
    • 0034633677 scopus 로고    scopus 로고
    • Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells
    • Ross, C. J., Bastedo, L., Maier, S. A., Sands, M. S., and Chang, P. L. (2000a). Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells. Hum. Gene Ther. 11, 2117-2127.
    • (2000) Hum. Gene Ther. , vol.11 , pp. 2117-2127
    • Ross, C.J.1    Bastedo, L.2    Maier, S.A.3    Sands, M.S.4    Chang, P.L.5
  • 71
    • 0033670210 scopus 로고    scopus 로고
    • Somatic gene therapy for a neurodegenerative disease using microencapsulated recombinant cells
    • Ross, C. J., Ralph, M., and Chang, P. L. (2000b). Somatic gene therapy for a neurodegenerative disease using microencapsulated recombinant cells. Exp. Neurol. 166, 276-286.
    • (2000) Exp. Neurol. , vol.166 , pp. 276-286
    • Ross, C.J.1    Ralph, M.2    Chang, P.L.3
  • 73
    • 0017752809 scopus 로고
    • Recognition and receptor-mediated uptake of a lysosomal enzyme, alpha-1-iduronidase, by cultured human fibroblasts
    • Sando, G. N., and Neufeld, E. F. (1977). Recognition and receptor-mediated uptake of a lysosomal enzyme, alpha-1-iduronidase, by cultured human fibroblasts. Cell 12, 619-627.
    • (1977) Cell , vol.12 , pp. 619-627
    • Sando, G.N.1    Neufeld, E.F.2
  • 74
    • 0027258694 scopus 로고
    • Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates
    • Sands, M. S., Barker, J. E., Vogler, C., Levy, B., Gwynn, B., Galvin, N., Sly, W. S., and Birkenmeier, E. (1993). Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates. Lab. Invest. 68, 676-686.
    • (1993) Lab. Invest. , vol.68 , pp. 676-686
    • Sands, M.S.1    Barker, J.E.2    Vogler, C.3    Levy, B.4    Gwynn, B.5    Galvin, N.6    Sly, W.S.7    Birkenmeier, E.8
  • 76
    • 0029130027 scopus 로고
    • Syngeneic bone marrow transplantation reduces the hearing loss associated with murine mucopolysaccharidosis type VII
    • Sands, M. S., Erway, L. C., Vogler, C., Sly, W. S., and Birkenmeier, E. H. (1995). Syngeneic bone marrow transplantation reduces the hearing loss associated with murine mucopolysaccharidosis type VII. Blood 86, 2033-2040.
    • (1995) Blood , vol.86 , pp. 2033-2040
    • Sands, M.S.1    Erway, L.C.2    Vogler, C.3    Sly, W.S.4    Birkenmeier, E.H.5
  • 78
    • 0033520487 scopus 로고    scopus 로고
    • In vivo protein transduction: Delivery of a biologically active protein into the mouse
    • Schwarze, S. R., Ho, A., Vocero-Akbani, A., and Dowdy, S. F. (1999). In vivo protein transduction: Delivery of a biologically active protein into the mouse. Science 285, 1569-1572.
    • (1999) Science , vol.285 , pp. 1569-1572
    • Schwarze, S.R.1    Ho, A.2    Vocero-Akbani, A.3    Dowdy, S.F.4
  • 79
    • 0034964121 scopus 로고    scopus 로고
    • Primary adult human astrocytes as an ex vivo vehicle for beta-glucuronidase delivery in the brain
    • Serguera, C., Sarkis, C., Ridet, J. L., Colin, P., Moullier, P., and Mallet, J. (2001). Primary adult human astrocytes as an ex vivo vehicle for beta-glucuronidase delivery in the brain. Mol. Ther. 3, 875-881.
    • (2001) Mol. Ther. , vol.3 , pp. 875-881
    • Serguera, C.1    Sarkis, C.2    Ridet, J.L.3    Colin, P.4    Moullier, P.5    Mallet, J.6
  • 80
    • 0034088881 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysaccharidosis type VII mouse
    • Sferra, T. J., Qu, G., McNeely, D., Rennard, R., Clark, K. R., Lo, W. D., and Johnson, P. R. (2000). Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysaccharidosis type VII mouse. Hum. Gene Ther. 11, 507-519.
    • (2000) Hum. Gene Ther. , vol.11 , pp. 507-519
    • Sferra, T.J.1    Qu, G.2    McNeely, D.3    Rennard, R.4    Clark, K.R.5    Lo, W.D.6    Johnson, P.R.7
  • 82
    • 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
    • Skorupa, A. F., Fisher, K. J., Wilson, J. M., Parente, M. K., and Wolfe, J. H. (1999). 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) 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
  • 83
    • 0019277147 scopus 로고
    • Correction of enzyme deficiency in mice by allogeneic bone marrow transplantation with total lymphoid irradiation
    • Slavin, S., and Yatziv, S. (1980). Correction of enzyme deficiency in mice by allogeneic bone marrow transplantation with total lymphoid irradiation. Science 210, 1150-1152.
    • (1980) Science , vol.210 , pp. 1150-1152
    • Slavin, S.1    Yatziv, S.2
  • 84
    • 0035956963 scopus 로고    scopus 로고
    • Active site mutant transgene confers tolerance to human beta-glucuronidase without affecting the phenotype of MPS VII mice
    • Sly, W. S., Vogler, C., Grubb, J. H., Zhou, M., Jiang, J., Zhou, X. Y., Tomatsu, S., Bi, Y., and Snella, E. M. (2001). Active site mutant transgene confers tolerance to human beta-glucuronidase without affecting the phenotype of MPS VII mice. Proc. Natl. Acad. Sci. USA 98, 2205-2210.
    • (2001) Proc. Natl. Acad. Sci. USA , vol.98 , pp. 2205-2210
    • Sly, W.S.1    Vogler, C.2    Grubb, J.H.3    Zhou, M.4    Jiang, J.5    Zhou, X.Y.6    Tomatsu, S.7    Bi, Y.8    Snella, E.M.9
  • 85
    • 0028951119 scopus 로고
    • Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain
    • Snyder, E. Y., Taylor, R. M., and Wolfe, J. H. (1995). Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain. Nature 374, 367-370.
    • (1995) Nature , vol.374 , pp. 367-370
    • Snyder, E.Y.1    Taylor, R.M.2    Wolfe, J.H.3
  • 86
    • 0035283136 scopus 로고    scopus 로고
    • Nonablative neonatal marrow transplantation attenuates functional and physical defects of beta-glucuronidase deficiency
    • Soper, B. W., Lessard, M. D., Vogler, C. A., Levy, B., Beamer, W. G., Sly, W. S., and Barker, J. E. (2001). Nonablative neonatal marrow transplantation attenuates functional and physical defects of beta-glucuronidase deficiency. Blood 97, 1498-1504.
    • (2001) Blood , vol.97 , pp. 1498-1504
    • Soper, B.W.1    Lessard, M.D.2    Vogler, C.A.3    Levy, B.4    Beamer, W.G.5    Sly, W.S.6    Barker, J.E.7
  • 88
    • 0032976576 scopus 로고    scopus 로고
    • Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII mice
    • Stein, C. S., Ghodsi, A., Derksen, T., and Davidson, B. L. (1999). Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII mice. J. Virol. 73, 3424-3429.
    • (1999) J. Virol. , vol.73 , pp. 3424-3429
    • Stein, C.S.1    Ghodsi, A.2    Derksen, T.3    Davidson, B.L.4
  • 90
    • 0030854815 scopus 로고    scopus 로고
    • Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase
    • Taylor, R. M., and Wolfe, J. H. (1997). Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase. Nat. Med. 3, 771-774.
    • (1997) Nat. Med. , vol.3 , pp. 771-774
    • Taylor, R.M.1    Wolfe, J.H.2
  • 91
    • 0026712987 scopus 로고
    • N-Acetylglucosamine 6-sulphatase deficiency in a Nubian goat: A model of Sanfilippo syndrome type D (mucopolysaccharidosis IIID)
    • Thompson, J. N., Jones, M. Z., Dawson, G., and Huffman, P. S. (1992). N-Acetylglucosamine 6-sulphatase deficiency in a Nubian goat: A model of Sanfilippo syndrome type D (mucopolysaccharidosis IIID). J. Inherit. Metab. Dis. 15, 760-768.
    • (1992) J. Inherit. Metab. Dis. , vol.15 , pp. 760-768
    • Thompson, J.N.1    Jones, M.Z.2    Dawson, G.3    Huffman, P.S.4
  • 93
    • 0029954652 scopus 로고    scopus 로고
    • Enzyme replacement with recombinant beta-glucuronidase in murine mucopolysaccharidosis type VII: Impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth, and survival
    • Vogler, C., Sands, M. S., Levy, B., Galvin, N., Birkcnmeier, E. H., and Sly, W. S. (1996). Enzyme replacement with recombinant beta-glucuronidase in murine mucopolysaccharidosis type VII: Impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth, and survival. Pediatr. Res. 39, 1050-1054.
    • (1996) Pediatr. Res. , vol.39 , pp. 1050-1054
    • Vogler, C.1    Sands, M.S.2    Levy, B.3    Galvin, N.4    Birkcnmeier, E.H.5    Sly, W.S.6
  • 94
    • 0033010571 scopus 로고    scopus 로고
    • Enzyme replacement in murine mucopolysaccharidosis type VII: Neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy
    • Vogler, C., Levy, B., Galvin, N. J., Thorpe, C., Sands, M. S., Barker, J. E., Baty, J., Birkenmeier, E. H., and Sly, W. S. (1999). Enzyme replacement in murine mucopolysaccharidosis type VII: Neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy. Pediatr. Res. 45, 838-844.
    • (1999) Pediatr. Res. , vol.45 , pp. 838-844
    • Vogler, C.1    Levy, B.2    Galvin, N.J.3    Thorpe, C.4    Sands, M.S.5    Barker, J.E.6    Baty, J.7    Birkenmeier, E.H.8    Sly, W.S.9
  • 97
    • 0032066332 scopus 로고    scopus 로고
    • Clinical and morphologic features of mucopolysaccharidosis type II in a dog: Naturally occurring model of Hunter syndrome
    • Wilkerson, M. J., Lewis, D. C., Marks, S. L., and Prieur, D. J. (1998). Clinical and morphologic features of mucopolysaccharidosis type II in a dog: naturally occurring model of Hunter syndrome. Vet. Pathol. 35, 230-233.
    • (1998) Vet. Pathol. , vol.35 , pp. 230-233
    • Wilkerson, M.J.1    Lewis, D.C.2    Marks, S.L.3    Prieur, D.J.4
  • 99
    • 0027051181 scopus 로고
    • Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer
    • Wolfe, J. H., Sands, M. S., Barker, J. E., Gwynn, B., Rowe, L. B., Vogler, C. A., and Birkenmeier, E. H. (1992). Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer. Nature 360, 749-753.
    • (1992) Nature , vol.360 , pp. 749-753
    • Wolfe, J.H.1    Sands, M.S.2    Barker, J.E.3    Gwynn, B.4    Rowe, L.B.5    Vogler, C.A.6    Birkenmeier, E.H.7
  • 100
    • 0030450135 scopus 로고    scopus 로고
    • Increased susceptibility to the pathogenic effects of wild-type and recombinant herpesviruses in MPS VII mice compared to normal siblings
    • Wolfe, J. H., Martin, C. E., Deshmane, S. L., Reilly, J. J., Kesari, S., and Fraser, N. W. (1996). Increased susceptibility to the pathogenic effects of wild-type and recombinant herpesviruses in MPS VII mice compared to normal siblings. J. Neurovirol. 2, 417-422.
    • (1996) J. Neurovirol. , vol.2 , pp. 417-422
    • Wolfe, J.H.1    Martin, C.E.2    Deshmane, S.L.3    Reilly, J.J.4    Kesari, S.5    Fraser, N.W.6
  • 101
    • 0034567124 scopus 로고    scopus 로고
    • Gene transfer of low levels of beta-glucuronidase corrects hepatic lysosomal storage in a large animal model of mucopolysaccharidosis VII
    • Wolfe, J. H., Sands, M. S., Harel, N., Weil, M. A., Parente, M. K., Polesky, A. C., Reilly, J. J., Hasson, C., Weimelt, S., and Haskins, M. E. (2000). Gene transfer of low levels of beta-glucuronidase corrects hepatic lysosomal storage in a large animal model of mucopolysaccharidosis VII. Mol. Ther. 2, 552-561.
    • (2000) Mol. Ther. , vol.2 , pp. 552-561
    • Wolfe, J.H.1    Sands, M.S.2    Harel, N.3    Weil, M.A.4    Parente, M.K.5    Polesky, A.C.6    Reilly, J.J.7    Hasson, C.8    Weimelt, S.9    Haskins, M.E.10
  • 102
    • 0034941801 scopus 로고    scopus 로고
    • The HIV Tat protein transduction domain improves the biodistribution of beta-glucuronidase expressed from recombinant viral vectors
    • Xia, H., Mao, Q., and Davidson, B. L. (2001). The HIV Tat protein transduction domain improves the biodistribution of beta-glucuronidase expressed from recombinant viral vectors. Nat. Biotechnol. 19, 640-644.
    • (2001) Nat. Biotechnol. , vol.19 , pp. 640-644
    • Xia, H.1    Mao, Q.2    Davidson, B.L.3
  • 103
    • 0036168867 scopus 로고    scopus 로고
    • Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs
    • Xu, L, Haskins, M. E., Melniczek, J. R., Gao, C., Weil, M. A., O'Malley, T. M., O'Donnell, P. A., Mazrier, H., Ellinwood, N. M., Zweigle, J., Wolfe, J. H., and Ponder, K. P. (2002). Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs. Mol. Ther. 5, 141-153.
    • (2002) Mol. Ther. , vol.5 , pp. 141-153
    • Xu, L.1    Haskins, M.E.2    Melniczek, J.R.3    Gao, C.4    Weil, M.A.5    O'Malley, T.M.6    O'Donnell, P.A.7    Mazrier, H.8    Ellinwood, N.M.9    Zweigle, J.10    Wolfe, J.H.11    Ponder, K.P.12
  • 104
    • 0019957039 scopus 로고
    • Long-term enzyme replacement therapy in beta-glucuronidase-defficient mice by allogeneic bone marrow transplantation
    • Yatziv, S., Weiss, L., Morecki, S., Fuks, Z., and Slavin, S. (1982). Long-term enzyme replacement therapy in beta-glucuronidase-defficient mice by allogeneic bone marrow transplantation. J. Lab. Clin. Med. 99, 792-797.
    • (1982) J. Lab. Clin. Med. , vol.99 , pp. 792-797
    • Yatziv, S.1    Weiss, L.2    Morecki, S.3    Fuks, Z.4    Slavin, S.5
  • 106
    • 0034527294 scopus 로고    scopus 로고
    • Short-term enzyme replacement in the murine model of Sanfilippo syndrome type B
    • Yu, W. H., Zhao, K. W., Ryazantsev, S., Rozengurt, N., and Neufeld, E. F. (2000). Short-term enzyme replacement in the murine model of Sanfilippo syndrome type B. Mol. Genet. Metab. 71, 573-580.
    • (2000) Mol. Genet. Metab. , vol.71 , pp. 573-580
    • Yu, W.H.1    Zhao, K.W.2    Ryazantsev, S.3    Rozengurt, N.4    Neufeld, E.F.5
  • 107
    • 0034218620 scopus 로고    scopus 로고
    • Significantly increased expression of beta-glucuronidase in the central nervous system of mucopolysaccharidosis type VII mice from the latency-associated transcript promoter in a nonpathogenic herpes simplex virus type 1 vector
    • Zhu, J., Kang, W., Wolfe, J. H., and Fraser, N. W. (2000). Significantly increased expression of beta-glucuronidase in the central nervous system of mucopolysaccharidosis type VII mice from the latency-associated transcript promoter in a nonpathogenic herpes simplex virus type 1 vector. Mol. Ther. 2, 82-94.
    • (2000) Mol. Ther. , vol.2 , pp. 82-94
    • Zhu, J.1    Kang, W.2    Wolfe, J.H.3    Fraser, N.W.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.