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Volumn 318, Issue 2, 2004, Pages 381-390

Expression and secretion of human glucocerebrosidase mediated by recombinant lentivirus vectors in vitro and in vivo: Implications for gene therapy of Gaucher disease

Author keywords

Gaucher disease; Gene therapy; Glucocerebrosidase; Lentivirus; Liver; Mouse

Indexed keywords

GLUCOSYLCERAMIDASE; LENTIVIRUS VECTOR;

EID: 2142713043     PISSN: 0006291X     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.bbrc.2004.04.040     Document Type: Article
Times cited : (24)

References (46)
  • 1
    • 0013927537 scopus 로고
    • Demonstration of a deficiency of glucocerebroside-cleaving enzyme in Gaucher's disease
    • Brady R.O., Kanfer J.N., Bradley R.M., Shapiro D. Demonstration of a deficiency of glucocerebroside-cleaving enzyme in Gaucher's disease. J. Clin. Invest. 45:1966;1112-1115
    • (1966) J. Clin. Invest. , vol.45 , pp. 1112-1115
    • Brady, R.O.1    Kanfer, J.N.2    Bradley, R.M.3    Shapiro, D.4
  • 3
    • 0027444631 scopus 로고
    • The role of neurogenetics in Gaucher disease
    • Brady R.O., Barton N.W., Grabowski G.A. The role of neurogenetics in Gaucher disease. Arch. Neurol. 50:1993;1212-1224
    • (1993) Arch. Neurol. , vol.50 , pp. 1212-1224
    • Brady, R.O.1    Barton, N.W.2    Grabowski, G.A.3
  • 5
    • 0025236339 scopus 로고
    • Therapeutic response to intravenous infusion of glucocerebrosidase in a patient with Gaucher disease
    • Barton N.W., Furbish F.S., Murray G.J., Garfield M., Brady R.O. Therapeutic response to intravenous infusion of glucocerebrosidase in a patient with Gaucher disease. Proc. Natl. Acad. Sci. USA. 87:1990;1913-1916
    • (1990) Proc. Natl. Acad. Sci. USA , vol.87 , pp. 1913-1916
    • Barton, N.W.1    Furbish, F.S.2    Murray, G.J.3    Garfield, M.4    Brady, R.O.5
  • 9
    • 0025219491 scopus 로고
    • Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease
    • Pink J.K., Correll P.H., Perry L.K., Brady R.O., Karlsson S. Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease. Proc. Natl. Acad. Sci. USA. 87:1990;2334-2338
    • (1990) Proc. Natl. Acad. Sci. USA , vol.87 , pp. 2334-2338
    • Pink, J.K.1    Correll, P.H.2    Perry, L.K.3    Brady, R.O.4    Karlsson, S.5
  • 11
    • 0025099115 scopus 로고
    • Expression of human glucocerebrosidase in murine long-term bone marrow cultures after retroviral vector-mediated transfer
    • Nolta J.A., Sender L.S., Barranger J.A., Kohn D.B. Expression of human glucocerebrosidase in murine long-term bone marrow cultures after retroviral vector-mediated transfer. Blood. 75:1990;787-798
    • (1990) Blood , vol.75 , pp. 787-798
    • Nolta, J.A.1    Sender, L.S.2    Barranger, J.A.3    Kohn, D.B.4
  • 12
    • 0026652473 scopus 로고
    • High levels of human glucocerebrosidase activity in macrophages of long-term reconstituted mice after retroviral infection of hematopoietic stem cells
    • Correll P.H., Colilla S., Dave H.P., Karlsson S. High levels of human glucocerebrosidase activity in macrophages of long-term reconstituted mice after retroviral infection of hematopoietic stem cells. Blood. 80:1992;331-336
    • (1992) Blood , vol.80 , pp. 331-336
    • Correll, P.H.1    Colilla, S.2    Dave, H.P.3    Karlsson, S.4
  • 17
    • 0034120890 scopus 로고    scopus 로고
    • Gene transfer into stimulated and unstimulated T lymphocytes by HIV-1-derived lentiviral vectors
    • Costello E., Munoz M., Buetti E., Meylan P.R., Diggelmann H., Thali M.E. Gene transfer into stimulated and unstimulated T lymphocytes by HIV-1-derived lentiviral vectors. Gene Ther. 7:2000;596-604
    • (2000) Gene Ther. , vol.7 , pp. 596-604
    • Costello, E.1    Munoz, M.2    Buetti, E.3    Meylan, P.R.4    Diggelmann, H.5    Thali, M.E.6
  • 18
    • 0033613827 scopus 로고    scopus 로고
    • Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors
    • Miyoshi H., Smith K.A., Mosierm D.E., Verma I.M., Torbett B.E. Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science. 283:1999;682-686
    • (1999) Science , vol.283 , pp. 682-686
    • Miyoshi, H.1    Smith, K.A.2    Mosierm, D.E.3    Verma, I.M.4    Torbett, B.E.5
  • 20
    • 0029993858 scopus 로고    scopus 로고
    • Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains infected with a lentiviral vector
    • Naldini L., Blomer U., Gage F.H., Trono D., Verma I.M. Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains infected with a lentiviral vector. Proc. Natl. Acad. Sci. USA. 93:1996;11382-11388
    • (1996) Proc. Natl. Acad. Sci. USA , vol.93 , pp. 11382-11388
    • Naldini, L.1    Blomer, U.2    Gage, F.H.3    Trono, D.4    Verma, I.M.5
  • 21
    • 0036469861 scopus 로고    scopus 로고
    • Gene transfer into the central nervous system in vivo using a recombinant lentivirus vector
    • Zhennan L., Brady R.O. Gene transfer into the central nervous system in vivo using a recombinant lentivirus vector. J. Neurosci. Res. 67:2002;363-371
    • (2002) J. Neurosci. Res. , vol.67 , pp. 363-371
    • Zhennan, L.1    Brady, R.O.2
  • 22
    • 0030716959 scopus 로고    scopus 로고
    • Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors
    • Kafri T., Blomer U., Peterson D.A., Gage F.H., Verma I.M. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat. Genet. 17:1997;314-317
    • (1997) Nat. Genet. , vol.17 , pp. 314-317
    • Kafri, T.1    Blomer, U.2    Peterson, D.A.3    Gage, F.H.4    Verma, I.M.5
  • 23
    • 0031686754 scopus 로고    scopus 로고
    • High-titer human immunodeficiency virus type 1-based vector systems for gene delivery into nondividing cells
    • Mochizuki H., Schwartz J.P., Tanaka K., Brady R.O., Reiser J. High-titer human immunodeficiency virus type 1-based vector systems for gene delivery into nondividing cells. J. Virol. 72:1998;8873-8883
    • (1998) J. Virol. , vol.72 , pp. 8873-8883
    • Mochizuki, H.1    Schwartz, J.P.2    Tanaka, K.3    Brady, R.O.4    Reiser, J.5
  • 24
    • 0034633662 scopus 로고    scopus 로고
    • Intercellular delivery of a herpes simplex virus VP22 fusion protein from cells infected with lentiviral vectors
    • Lai Z., Han I., Zirzow G., Brady R.O., Reiser J. Intercellular delivery of a herpes simplex virus VP22 fusion protein from cells infected with lentiviral vectors. Proc. Natl. Acad. Sci. USA. 97:2000;11297-11302
    • (2000) Proc. Natl. Acad. Sci. USA , vol.97 , pp. 11297-11302
    • Lai, Z.1    Han, I.2    Zirzow, G.3    Brady, R.O.4    Reiser, J.5
  • 25
    • 0027236954 scopus 로고
    • Production of high-titer helper-free retroviruses by transient transfection
    • Pear W.S., Nolan G.P., Scott M.L., Baltimore D. Production of high-titer helper-free retroviruses by transient transfection. Proc. Natl. Acad. Sci. USA. 90:1993;8392-8396
    • (1993) Proc. Natl. Acad. Sci. USA , vol.90 , pp. 8392-8396
    • Pear, W.S.1    Nolan, G.P.2    Scott, M.L.3    Baltimore, D.4
  • 26
    • 0037097043 scopus 로고    scopus 로고
    • Correction of mucopolysaccharidosis type IIIb fibroblasts by lentivirus vector-mediated gene transfer
    • Villani G.R.D., Follenzi A., Vanacore B., Domenico C.D., Naldini L., Natale P.D. Correction of mucopolysaccharidosis type IIIb fibroblasts by lentivirus vector-mediated gene transfer. Biochem. J. 364:2002;747-753
    • (2002) Biochem. J. , vol.364 , pp. 747-753
    • Villani, G.R.D.1    Follenzi, A.2    Vanacore, B.3    Domenico, C.D.4    Naldini, L.5    Natale, P.D.6
  • 27
    • 0021320439 scopus 로고
    • Synthesis of a fluorescent derivative of glucosyl ceramide for the sensitive determination of glucocerebrosidase activity
    • Dinur T., Grabowski G.A., Desnick R.J., Gatt S. Synthesis of a fluorescent derivative of glucosyl ceramide for the sensitive determination of glucocerebrosidase activity. Anal. Biochem. 136:1984;223-234
    • (1984) Anal. Biochem. , vol.136 , pp. 223-234
    • Dinur, T.1    Grabowski, G.A.2    Desnick, R.J.3    Gatt, S.4
  • 29
  • 30
    • 0036664451 scopus 로고    scopus 로고
    • Highly efficient lentiviral vector-mediated transduction of nondividing, fully reimplantable primary hepatocytes
    • Nguyen T.H., Oberholzer J., Birraux J., Majno P., Morel P., Trono D. Highly efficient lentiviral vector-mediated transduction of nondividing, fully reimplantable primary hepatocytes. Mol. Ther. 6:2002;199-209
    • (2002) Mol. Ther. , vol.6 , pp. 199-209
    • Nguyen, T.H.1    Oberholzer, J.2    Birraux, J.3    Majno, P.4    Morel, P.5    Trono, D.6
  • 32
    • 0019475525 scopus 로고
    • Uptake and distribution of placental glucocerebrosidase in rat hepatic cells and effects of sequential deglycosylation
    • Furbish F.S., Steer C.J., Krett N.L., Barranger J.A. Uptake and distribution of placental glucocerebrosidase in rat hepatic cells and effects of sequential deglycosylation. Biochim. Biophys. Acta. 673:1981;425-434
    • (1981) Biochim. Biophys. Acta , vol.673 , pp. 425-434
    • Furbish, F.S.1    Steer, C.J.2    Krett, N.L.3    Barranger, J.A.4
  • 33
    • 0021164336 scopus 로고
    • Structure of the N-asparagine-linked oligosaccharide units of human placental beta-glucocerebrosidase
    • Takasaki S., Murray G.J., Furbish F.S., Brady R.O., Barranger J.A., Kobata A. Structure of the N-asparagine-linked oligosaccharide units of human placental beta-glucocerebrosidase. J. Biol. Chem. 259:1984;10112-10117
    • (1984) J. Biol. Chem. , vol.259 , pp. 10112-10117
    • Takasaki, S.1    Murray, G.J.2    Furbish, F.S.3    Brady, R.O.4    Barranger, J.A.5    Kobata, A.6
  • 34
    • 0022386744 scopus 로고
    • Biosynthesis of the lysosomal enzyme glucocerebrosidase
    • Erickson A.H., Ginns E.I., Barranger J.A. Biosynthesis of the lysosomal enzyme glucocerebrosidase. J. Biol. Chem. 260:1985;14319-14324
    • (1985) J. Biol. Chem. , vol.260 , pp. 14319-14324
    • Erickson, A.H.1    Ginns, E.I.2    Barranger, J.A.3
  • 38
    • 0036171327 scopus 로고    scopus 로고
    • Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors
    • Follenzi A., Sabatino G., Lombardo A., Boccaccio C., Naldini L. Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors. Hum. Gene Ther. 13:2002;243-260
    • (2002) Hum. Gene Ther. , vol.13 , pp. 243-260
    • Follenzi, A.1    Sabatino, G.2    Lombardo, A.3    Boccaccio, C.4    Naldini, L.5
  • 39
    • 0041854301 scopus 로고    scopus 로고
    • The effect of age on hepatic gene transfer with self-inactivating lentiviral vectors in vivo
    • Park F., Ohashi K., Kay M.A. The effect of age on hepatic gene transfer with self-inactivating lentiviral vectors in vivo. Mol. Ther. 8:2003;314-323
    • (2003) Mol. Ther. , vol.8 , pp. 314-323
    • Park, F.1    Ohashi, K.2    Kay, M.A.3
  • 40
    • 0029845416 scopus 로고    scopus 로고
    • Therapeutic delivery of proteins to macrophages: Implications for treatment of Gaucher's disease
    • Mistry P.K., Wraight E.P., Cox T.M. Therapeutic delivery of proteins to macrophages: implications for treatment of Gaucher's disease. Lancet. 348:1996;1555-1559
    • (1996) Lancet , vol.348 , pp. 1555-1559
    • Mistry, P.K.1    Wraight, E.P.2    Cox, T.M.3
  • 42
    • 0035025888 scopus 로고    scopus 로고
    • A quantitative assay for HIV cDNA integration in vivo
    • Butler S.L., Hansen M.S., Bushman F.D. A quantitative assay for HIV cDNA integration in vivo. Nat. Med. 7:2001;631-634
    • (2001) Nat. Med. , vol.7 , pp. 631-634
    • Butler, S.L.1    Hansen, M.S.2    Bushman, F.D.3
  • 44
    • 0034898078 scopus 로고    scopus 로고
    • Accumulation and intranuclear distribution of unintegrated human immunodeficiency virus type 1 DNA
    • Bell P., Montaner L.J., Maul G.G. Accumulation and intranuclear distribution of unintegrated human immunodeficiency virus type 1 DNA. J. Virol. 75:2001;7683-7691
    • (2001) J. Virol. , vol.75 , pp. 7683-7691
    • Bell, P.1    Montaner, L.J.2    Maul, G.G.3
  • 45
    • 0142150948 scopus 로고    scopus 로고
    • Global analysis of cellular transcription following infection with an HIV-based vector
    • Mitchell R., Chiang C.Y., Berry C., Bushman F. Global analysis of cellular transcription following infection with an HIV-based vector. Mol. Ther. 8:2003;674-687
    • (2003) Mol. Ther. , vol.8 , pp. 674-687
    • Mitchell, R.1    Chiang, C.Y.2    Berry, C.3    Bushman, F.4
  • 46
    • 0036665573 scopus 로고    scopus 로고
    • Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow
    • Pan D., Gunther R., Duan W., Wendell S., Kaemmerer W., Kafri T., Verma I.M., Whitley C.B. Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow. Mol. Ther. 6:2002;19-29
    • (2002) Mol. Ther. , vol.6 , pp. 19-29
    • Pan, D.1    Gunther, R.2    Duan, W.3    Wendell, S.4    Kaemmerer, W.5    Kafri, T.6    Verma, I.M.7    Whitley, C.B.8


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