메뉴 건너뛰기




Volumn 38, Issue 2, 2008, Pages 109-119

Naked plasmid DNA-based α-galactosidase a gene transfer partially reduces systemic accumulation of globotriaosylceramide in Fabry mice

Author keywords

CAG promoter; Catheter based gene transfer; Fabry disease; Hydrodynamics based transfection; Naked plasmid DNA; Renal vein injection

Indexed keywords

DNA; FOOD ADDITIVES; GENES; GENETIC ENGINEERING; HEALTH; LIVER; NUCLEIC ACIDS; ORGANIC ACIDS; VECTORS;

EID: 42449097323     PISSN: 10736085     EISSN: None     Source Type: Journal    
DOI: 10.1007/s12033-007-9008-5     Document Type: Article
Times cited : (19)

References (40)
  • 7
    • 0028919110 scopus 로고
    • The functional role of glutamine-280 and threonine-282 in human alpha-galactosidase
    • S. Ishii R. Kase H. Sakuraba 1995 The functional role of glutamine-280 and threonine-282 in human alpha-galactosidase Biochimica et Biophysica Acta 1270 163 167
    • (1995) Biochimica et Biophysica Acta , vol.1270 , pp. 163-167
    • Ishii, S.1    Kase, R.2    Sakuraba, H.3
  • 11
    • 0035989018 scopus 로고    scopus 로고
    • Adenovirus-transduced lung as a portal for delivering alpha-galactosidase a into systemic circulation for Fabry disease
    • C. Li R. J. Ziegler M. Cherry R. J. Desnick N. S. Yew S. H. Cheng 2002 Adenovirus-transduced lung as a portal for delivering alpha-galactosidase A into systemic circulation for Fabry disease Molecular Therapy 5 745 754
    • (2002) Molecular Therapy , vol.5 , pp. 745-754
    • Li, C.1    Ziegler, R.J.2    Cherry, M.3    Desnick, R.J.4    Yew, N.S.5    Cheng, S.H.6
  • 21
    • 0025884056 scopus 로고
    • Efficient selection for high-expression transfectants with a novel eukaryotic vector
    • H. Niwa K. Yamamura J. Miyazaki 1991 Efficient selection for high-expression transfectants with a novel eukaryotic vector Gene 108 193 199
    • (1991) Gene , vol.108 , pp. 193-199
    • Niwa, H.1    Yamamura, K.2    Miyazaki, J.3
  • 25
    • 7044284796 scopus 로고    scopus 로고
    • Transgenic mouse expressing human mutant alpha-galactosidase a in an endogenous enzyme deficient background: A biochemical animal model for studying active-site specific chaperone therapy for Fabry disease
    • S. Ishii H. Yoshioka K. Mannen A. B. Kulkarni J. Q. Fan 2004 Transgenic mouse expressing human mutant alpha-galactosidase A in an endogenous enzyme deficient background: A biochemical animal model for studying active-site specific chaperone therapy for Fabry disease Biochimica et Biophysica Acta 1690 250 257
    • (2004) Biochimica et Biophysica Acta , vol.1690 , pp. 250-257
    • Ishii, S.1    Yoshioka, H.2    Mannen, K.3    Kulkarni, A.B.4    Fan, J.Q.5
  • 26
    • 0033018496 scopus 로고    scopus 로고
    • Accelerated transport and maturation of lysosomal alpha-galactosidase a in Fabry lymphoblasts by an enzyme inhibitor
    • J. Q. Fan S. Ishii N. Asano Y. Suzuki 1999 Accelerated transport and maturation of lysosomal alpha-galactosidase A in Fabry lymphoblasts by an enzyme inhibitor Nature Medicine 5 112 115
    • (1999) Nature Medicine , vol.5 , pp. 112-115
    • Fan, J.Q.1    Ishii, S.2    Asano, N.3    Suzuki, Y.4
  • 27
    • 0019464277 scopus 로고
    • Differential assay for lysosomal alpha-galactosidases in human tissues and its application to Fabry's disease
    • J. S. Mayes J. B. Scheerer R. N. Sifers M.L. Donaldson 1981 Differential assay for lysosomal alpha-galactosidases in human tissues and its application to Fabry's disease Clinica Chimica Acta. 112 245 251
    • (1981) Clinica Chimica Acta. , vol.112 , pp. 245-251
    • Mayes, J.S.1    Scheerer, J.B.2    Sifers, R.N.3    Donaldson, M.L.4
  • 28
    • 0019767282 scopus 로고
    • Testosterone effects on the induction and urinary excretion of mouse kidney glycosphingolipids associated with lysosomes
    • R. H. McCluer M. A. Williams S. K. Gross M. H. Meisler 1981 Testosterone effects on the induction and urinary excretion of mouse kidney glycosphingolipids associated with lysosomes Journal of Biological Chemistry 256 13112 13120
    • (1981) Journal of Biological Chemistry , vol.256 , pp. 13112-13120
    • McCluer, R.H.1    Williams, M.A.2    Gross, S.K.3    Meisler, M.H.4
  • 30
    • 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 R. J. Desnick 2001 Fabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice American Journal of Human Genetics 68 14 25
    • (2001) American Journal of Human Genetics , vol.68 , pp. 14-25
    • Ioannou, Y.A.1    Zeidner, K.M.2    Gordon, R.E.3    Desnick, R.J.4
  • 33
    • 0033761804 scopus 로고    scopus 로고
    • Adeno-associated virus vectors for gene therapy: More pros than cons?
    • P. E. R. J. Monahan Samulski 2000 Adeno-associated virus vectors for gene therapy: More pros than cons? Molecular Medicine Today 6 433 440
    • (2000) Molecular Medicine Today , vol.6 , pp. 433-440
    • Monahan Samulski, P.E.R.J.1
  • 35
    • 1442299241 scopus 로고    scopus 로고
    • The molecular defect leading to Fabry disease: Structure of human alpha-galactosidase
    • S. C. D. N. Garman Garboczi 2004 The molecular defect leading to Fabry disease: structure of human alpha-galactosidase Journal of Molecular Biology 337 319 335
    • (2004) Journal of Molecular Biology , vol.337 , pp. 319-335
    • Garman Garboczi, S.C.D.N.1
  • 39
    • 4444333780 scopus 로고    scopus 로고
    • Glomerular ultrafiltration, in Elements of normal renal structure and function, vol. 1
    • WB Saunders Philadelphia
    • Maddox D. A. & Brenner, B. M. (2000). Glomerular ultrafiltration, in Elements of normal renal structure and function, vol. 1. In Brenner B. M. (Ed.), Brenner & Rector's The kidney 7th ed. (pp. 353-412). Philadelphia: WB Saunders.
    • (2000) Brenner & Rector's the Kidney 7th Ed , pp. 353-412
    • Maddox Brenner, D.A.B.M.1    Brenner, B.M.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.