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Volumn 12, Issue 5, 2005, Pages 985-990

Promoter dependence of plasmid-pluronics targeted α galactosidase A expression in skeletal muscle of fabry mice

Author keywords

galactosidase A; CMV promoter; Fabry disease gene therapy; Immune response; Immunosuppression; MCK promoter; Plasmid vector; Pluronic SP1017

Indexed keywords

ALPHA GALACTOSIDASE; PLASMID DNA; PLASMID VECTOR; POLOXAMER;

EID: 27744437341     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2005.02.032     Document Type: Article
Times cited : (20)

References (27)
  • 1
    • 0034766525 scopus 로고    scopus 로고
    • Anderson-Fabry disease: Clinical manifestations and impact of disease in a cohort of 98 hemizygous males
    • K.D. MacDermot A. Holmes and A.H. Miners Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males J. Med. Genet. 38 2001 750-760
    • (2001) J. Med. Genet. , vol.38 , pp. 750-760
    • MacDermot, K.D.1    Holmes, A.2    Miners, A.H.3
  • 2
    • 13044281563 scopus 로고    scopus 로고
    • Aging accentuates and bone marrow transplantation ameliorates metabolic defects in Fabry disease mice
    • T. Ohshima et al. Aging accentuates and bone marrow transplantation ameliorates metabolic defects in Fabry disease mice Proc. Natl. Acad. Sci. USA 96 1999 6423-6427
    • (1999) Proc. Natl. Acad. Sci. USA , vol.96 , pp. 6423-6427
    • Ohshima, T.1
  • 3
    • 0033166317 scopus 로고    scopus 로고
    • Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer
    • R.J. Ziegler et al. Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer Hum. Gene Ther. 10 1999 1667-1682
    • (1999) Hum. Gene Ther. , vol.10 , pp. 1667-1682
    • Ziegler, R.J.1
  • 4
    • 14844288273 scopus 로고    scopus 로고
    • Glycogen storage in multiple muscles of old GSD-II mice can be rapidly cleared after a single intravenous injection with a modified adenoviral vector expressing hGAA
    • F. Xu Glycogen storage in multiple muscles of old GSD-II mice can be rapidly cleared after a single intravenous injection with a modified adenoviral vector expressing hGAA J. Gene. Med. 7 2004 171-178
    • (2004) J. Gene. Med. , vol.7 , pp. 171-178
    • Xu, F.1
  • 5
    • 0035163539 scopus 로고    scopus 로고
    • Fabry disease: Preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice
    • Y.A. Ioannou K.M. Zeidner R.E. Gordon and R.J. Desnick Fabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice Am. J. Hum. Genet. 68 2001 14-25
    • (2001) Am. J. Hum. Genet. , vol.68 , pp. 14-25
    • Ioannou, Y.A.1    Zeidner, K.M.2    Gordon, R.E.3    Desnick, R.J.4
  • 6
    • 0035811624 scopus 로고    scopus 로고
    • Safety and efficacy of recombinant human alpha-galactosidase A-replacement therapy in Fabry's disease
    • C.M. Eng et al. Safety and efficacy of recombinant human alpha-galactosidase A-replacement therapy in Fabry's disease N. Engl. J. Med. 345 2001 9-16
    • (2001) N. Engl. J. Med. , vol.345 , pp. 9-16
    • Eng, C.M.1
  • 7
    • 12644284502 scopus 로고    scopus 로고
    • α-Galactosidase A deficient mice: A model of Fabry disease
    • T. Ohshima et al. α-Galactosidase A deficient mice: A model of Fabry disease Proc. Natl. Acad. Sci. USA 94 1997 2540-2544
    • (1997) Proc. Natl. Acad. Sci. USA , vol.94 , pp. 2540-2544
    • Ohshima, T.1
  • 8
    • 0034044578 scopus 로고    scopus 로고
    • A combination of poloxamers increases gene expression of plasmid DNA in skeletal muscle
    • P. Lemieux et al. A combination of poloxamers increases gene expression of plasmid DNA in skeletal muscle Gene Ther. 7 2000 986-991
    • (2000) Gene Ther. , vol.7 , pp. 986-991
    • Lemieux, P.1
  • 9
    • 0024261447 scopus 로고
    • A muscle-specific enhancer is located at the 3′ end of the myosin light-chain 1/3 gene locus
    • M. Donoghue H. Ernst B. Wentworth B. Nadal-Ginard and N. Rosenthal A muscle-specific enhancer is located at the 3′ end of the myosin light-chain 1/3 gene locus Genes Dev. 2 1988 1779-1790
    • (1988) Genes Dev. , vol.2 , pp. 1779-1790
    • Donoghue, M.1    Ernst, H.2    Wentworth, B.3    Nadal-Ginard, B.4    Rosenthal, N.5
  • 10
    • 0022727888 scopus 로고
    • Nuclear factor 1 interacts with five DNA elements in the promoter region of the human cytomegalovirus major immediate early gene
    • L. Hennighausen and B. Fleckenstein Nuclear factor 1 interacts with five DNA elements in the promoter region of the human cytomegalovirus major immediate early gene EMBO J. 5 1986 1367-1371
    • (1986) EMBO J. , vol.5 , pp. 1367-1371
    • Hennighausen, L.1    Fleckenstein, B.2
  • 11
    • 8244264752 scopus 로고    scopus 로고
    • Efficient muscle-specific transgene expression after adenovirus-mediated gene transfer in mice using a 1.35 kb muscle creatine kinase promoter/ enhancer
    • N. Larochelle et al. Efficient muscle-specific transgene expression after adenovirus-mediated gene transfer in mice using a 1.35 kb muscle creatine kinase promoter/enhancer Gene Ther. 4 1997 465-472
    • (1997) Gene Ther. , vol.4 , pp. 465-472
    • Larochelle, N.1
  • 12
    • 0034091578 scopus 로고    scopus 로고
    • Reduction of globotriaosylceramide in Fabry disease mice by substrate deprivation
    • A. Abe et al. Reduction of globotriaosylceramide in Fabry disease mice by substrate deprivation J. Clin. Invest. 105 2000 1563-1571
    • (2000) J. Clin. Invest. , vol.105 , pp. 1563-1571
    • Abe, A.1
  • 13
    • 0037109052 scopus 로고    scopus 로고
    • Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer
    • H. Takahashi et al. Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer Proc. Natl. Acad. Sci. USA 99 2002 13777-13782
    • (2002) Proc. Natl. Acad. Sci. USA , vol.99 , pp. 13777-13782
    • Takahashi, H.1
  • 14
    • 0037453003 scopus 로고    scopus 로고
    • Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer
    • J. Park et al. Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer Proc. Natl. Acad. Sci. USA 100 2003 3450-3454
    • (2003) Proc. Natl. Acad. Sci. USA , vol.100 , pp. 3450-3454
    • Park, J.1
  • 15
    • 0037135675 scopus 로고    scopus 로고
    • Role of transfection in the priming of cytotoxic T-cells by DNA-mediated immunization
    • H. Sbai J. Schneider A.V. Hill and R.G. Whalen Role of transfection in the priming of cytotoxic T-cells by DNA-mediated immunization Vaccine 20 2002 3137-3147
    • (2002) Vaccine , vol.20 , pp. 3137-3147
    • Sbai, H.1    Schneider, J.2    Hill, A.V.3    Whalen, R.G.4
  • 17
    • 85047691459 scopus 로고    scopus 로고
    • Developing DNA vaccines that call to dendritic cells
    • M.A. Kutzler and D.B. Weiner Developing DNA vaccines that call to dendritic cells J. Clin. Invest. 114 2004 1241-1244
    • (2004) J. Clin. Invest. , vol.114 , pp. 1241-1244
    • Kutzler, M.A.1    Weiner, D.B.2
  • 18
    • 12944265457 scopus 로고    scopus 로고
    • Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease
    • R. Schiffmann et al. Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease Proc. Natl. Acad. Sci. USA 97 2000 365-370
    • (2000) Proc. Natl. Acad. Sci. USA , vol.97 , pp. 365-370
    • Schiffmann, R.1
  • 19
    • 0037029879 scopus 로고    scopus 로고
    • Muscle specific versus ubiquitous expression of Gag based HIV-1 DNA vaccines: A comparative analysis
    • A. Bojak D. Hammer H. Wolf and R. Wagner Muscle specific versus ubiquitous expression of Gag based HIV-1 DNA vaccines: A comparative analysis Vaccine 20 2002 1975-1979
    • (2002) Vaccine , vol.20 , pp. 1975-1979
    • Bojak, A.1    Hammer, D.2    Wolf, H.3    Wagner, R.4
  • 20
    • 0033695299 scopus 로고    scopus 로고
    • Proteolysis in MHC class II antigen presentation: Who's in charge?
    • J.A. Villadangos and H.L. Ploegh Proteolysis in MHC class II antigen presentation: Who's in charge? Immunity 12 2000 233-239
    • (2000) Immunity , vol.12 , pp. 233-239
    • Villadangos, J.A.1    Ploegh, H.L.2
  • 21
    • 0037713660 scopus 로고    scopus 로고
    • Fabry disease: Novel alpha-galactosidase A 3′-terminal mutations result in multiple transcripts due to aberrant 3′-end formation
    • M. Yasuda J. Shabbeer M. Osawa and R.J. Desnick Fabry disease: Novel alpha-galactosidase A 3′-terminal mutations result in multiple transcripts due to aberrant 3′-end formation Am. J. Hum. Genet. 73 2003 162-173
    • (2003) Am. J. Hum. Genet. , vol.73 , pp. 162-173
    • Yasuda, M.1    Shabbeer, J.2    Osawa, M.3    Desnick, R.J.4
  • 22
    • 0028990407 scopus 로고
    • Alpha-galactosidase gene mutations in Fabry disease: Heterogeneous expressions of mutant enzyme proteins
    • T. Okumiya S. Ishii R. Kase S. Kamei H. Sakuraba and Y. Suzuki Alpha-galactosidase gene mutations in Fabry disease: Heterogeneous expressions of mutant enzyme proteins Hum. Genet. 95 1995 557-561
    • (1995) Hum. Genet. , vol.95 , pp. 557-561
    • Okumiya, T.1    Ishii, S.2    Kase, R.3    Kamei, S.4    Sakuraba, H.5    Suzuki, Y.6
  • 25
    • 0030914425 scopus 로고    scopus 로고
    • Gene transfer and expression of human alpha-galactosidase from mouse muscle in vitro and in vivo
    • F.J. Novo D.C. Gorecki G. Goldspink and K.D. MacDermot Gene transfer and expression of human alpha-galactosidase from mouse muscle in vitro and in vivo Gene Ther. 4 1997 488-492
    • (1997) Gene Ther. , vol.4 , pp. 488-492
    • Novo, F.J.1    Gorecki, D.C.2    Goldspink, G.3    MacDermot, K.D.4
  • 26
    • 0031589164 scopus 로고    scopus 로고
    • Immune responses, not promoter inactivation, are responsible for decreased long-term expression following plasmid gene transfer into skeletal muscle
    • K.E. Wells et al. Immune responses, not promoter inactivation, are responsible for decreased long-term expression following plasmid gene transfer into skeletal muscle FEBS Lett. 407 1997 164-168
    • (1997) FEBS Lett. , vol.407 , pp. 164-168
    • Wells, K.E.1
  • 27
    • 0033623151 scopus 로고    scopus 로고
    • Immune responses to dystropin: Implications for gene therapy of Duchenne muscular dystrophy
    • A. Ferrer K.E. Wells and D.J. Wells Immune responses to dystropin: implications for gene therapy of Duchenne muscular dystrophy Gene Ther. 7 2000 1439-1446
    • (2000) Gene Ther. , vol.7 , pp. 1439-1446
    • Ferrer, A.1    Wells, K.E.2    Wells, D.J.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.