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Volumn 17, Issue 1, 2010, Pages

Characterization of Fabry mice treated with recombinant adeno-associated virus 2/8-mediated gene transfer

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; ALPHA GALACTOSIDASE; GLOBOTRIAOSYLCERAMIDE; CERAMIDE TRIHEXOSIDE;

EID: 77950807425     PISSN: 10217770     EISSN: 14230127     Source Type: Journal    
DOI: 10.1186/1423-0127-17-26     Document Type: Article
Times cited : (20)

References (36)
  • 2
    • 32944476769 scopus 로고    scopus 로고
    • Enzyme replacement for lysosomal diseases
    • 10.1146/annurev.med.57.110104.115650. 16409150
    • Enzyme replacement for lysosomal diseases. RO Brady, Annu Rev Med 2006 57 283 296 10.1146/annurev.med.57.110104.115650 16409150
    • (2006) Annu Rev Med , vol.57 , pp. 283-296
    • Brady, R.O.1
  • 3
    • 2342524106 scopus 로고    scopus 로고
    • Fabry disease in childhood
    • 10.1016/j.jpeds.2004.01.051. 15126980
    • Fabry disease in childhood. RJ Desnick RO Brady, J Pediatr 2004 144 20 26 10.1016/j.jpeds.2004.01.051 15126980
    • (2004) J Pediatr , vol.144 , pp. 1920-26
    • Desnick, R.J.1    Brady, R.O.2
  • 4
    • 0026751048 scopus 로고
    • Fabry disease: Immune-cytochemical characterization of neuronal involvement
    • 10.1002/ana.410310410. 1375013
    • Fabry disease: immune-cytochemical characterization of neuronal involvement. GA DeVeber GA Schwarting EH Kolodny NW Kowall, Ann Neurol 1992 31 409 415 10.1002/ana.410310410 1375013
    • (1992) Ann Neurol , vol.31 , pp. 409-415
    • Deveber, G.A.1    Schwarting, G.A.2    Kolodny, E.H.3    Kowall, N.W.4
  • 6
  • 8
    • 0035816007 scopus 로고    scopus 로고
    • Enzyme replacement therapy in Fabry disease: A randomized controlled trial
    • 10.1001/jama.285.21.2743. 11386930
    • Enzyme replacement therapy in Fabry disease: a randomized controlled trial. R Schiffmann JB Kopp HA Austin S Sabnis DF Moore T Weibel JE Balow RO Brady, JAMA 2001 285 2743 2749 10.1001/jama.285.21.2743 11386930
    • (2001) JAMA , vol.285 , pp. 2743-2749
    • Schiffmann, R.1    Kopp, J.B.2    Austin, H.A.3    Sabnis, S.4    Moore, D.F.5    Weibel, T.6    Balow, J.E.7    Brady, R.O.8
  • 10
    • 0033543108 scopus 로고    scopus 로고
    • Circulating alpha-galactosidase A derived from transduced bone marrow cells: Relevance for corrective gene transfer for Fabry disease
    • 10.1089/10430349950017293. 10466627
    • Circulating alpha-galactosidase A derived from transduced bone marrow cells: relevance for corrective gene transfer for Fabry disease. T Takenaka G Qin RO Brady JA Medin, Hum Gene Ther 1999 10 1931 1939 10.1089/10430349950017293 10466627
    • (1999) Hum Gene Ther , vol.10 , pp. 1931-1939
    • Takenaka, T.1    Qin, G.2    Brady, R.O.3    Medin, J.A.4
  • 13
    • 33847213924 scopus 로고    scopus 로고
    • Correction of the biochemical and functional deficits in Fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A
    • 10.1038/sj.mt.6300066. 17191071
    • Correction of the biochemical and functional deficits in Fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A. RJ Ziegler M Cherry CM Barbon C Li SD Bercury D Armentano RJ Desnick SH Cheng, Mol Ther 2007 15 492 500 10.1038/sj.mt.6300066 17191071
    • (2007) Mol Ther , vol.15 , pp. 492-500
    • Ziegler, R.J.1    Cherry, M.2    Barbon, C.M.3    Li, C.4    Bercury, S.D.5    Armentano, D.6    Desnick, R.J.7    Cheng, S.H.8
  • 14
    • 28844452008 scopus 로고    scopus 로고
    • Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates
    • 10.1016/j.ymthe.2005.08.017. 16219492
    • Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates. G Gao Y Lu R Calcedo RL Grant P Bell L Wang J Figueredo M Lock JM Wilson, Mol Ther 2006 13 77 87 10.1016/j.ymthe.2005.08.017 16219492
    • (2006) Mol Ther , vol.13 , pp. 77-87
    • Gao, G.1    Lu, Y.2    Calcedo, R.3    Grant, R.L.4    Bell, P.5    Wang, L.6    Figueredo, J.7    Lock, M.8    Wilson, J.M.9
  • 17
    • 0035163539 scopus 로고    scopus 로고
    • Fabry disease: Preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice
    • 10.1086/316953. 11115376
    • Fabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice. YA Ioannou KM Zeidner RE Gordon RJ Desnick, Am J Hum Genet 2001 68 14 25 10.1086/316953 11115376
    • (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
  • 18
    • 0018264552 scopus 로고
    • Purification and properties of the two major isozymes of -galactosidase from human placenta
    • 201618
    • Purification and properties of the two major isozymes of -galactosidase from human placenta. JW Kusiak JM Quirk RO Brady, J Biol Chem 1978 253 184 190 201618
    • (1978) J Biol Chem , vol.253 , pp. 184-190
    • Kusiak, J.W.1    Quirk, J.M.2    Brady, R.O.3
  • 19
    • 0344683171 scopus 로고
    • Improved procedure for the extraction of lipids from human erythrocytes
    • 14336214
    • Improved procedure for the extraction of lipids from human erythrocytes. HG Rose M Oklander, J Lipid Res 1965 6 428 431 14336214
    • (1965) J Lipid Res , vol.6 , pp. 428-431
    • Rose, H.G.1    Oklander, M.2
  • 21
    • 10644289281 scopus 로고    scopus 로고
    • Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice
    • 10.1128/JVI.79.1.214-224.2005. 15596817
    • Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. H Nakai S Fuess TA Storm S Muramatsu Y Nara MA Kay, J Virol 2005 79 214 224 10.1128/JVI.79.1.214-224.2005 15596817
    • (2005) J Virol , vol.79 , pp. 214-224
    • Nakai, H.1    Fuess, S.2    Storm, T.A.3    Muramatsu, S.4    Nara, Y.5    Kay, M.A.6
  • 23
    • 0036263495 scopus 로고    scopus 로고
    • Renal pathology in Fabry disease
    • 12068025
    • Renal pathology in Fabry disease. J Alroy S Sabnis JB Kopp, J Am Soc Nephrol 2002 Suppl 2 134 138 12068025
    • (2002) J Am Soc Nephrol , Issue.SUPPL 2 , pp. 19134-138
    • Alroy, J.1    Sabnis, S.2    Kopp, J.B.3
  • 24
    • 0033814178 scopus 로고    scopus 로고
    • Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo
    • 10.1128/JVI.74.20.9451-9463.2000. 11000214
    • Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo. H Nakai TA Storm MA Kay, J Virol 2000 74 9451 9463 10.1128/JVI.74.20.9451-9463.2000 11000214
    • (2000) J Virol , vol.74 , pp. 9451-9463
    • Nakai, H.1    Storm, T.A.2    Kay, M.A.3
  • 25
    • 54249126862 scopus 로고    scopus 로고
    • Gene therapy using adeno-associated virus vectors
    • 10.1128/CMR.00008-08. 18854481
    • Gene therapy using adeno-associated virus vectors. S Daya KI Berns, Clin Microbiol Rev 2008 21 583 593 10.1128/CMR.00008-08 18854481
    • (2008) Clin Microbiol Rev , vol.21 , pp. 583-593
    • Daya, S.1    Berns, K.I.2
  • 27
    • 77951939632 scopus 로고    scopus 로고
    • Generation and characterization of transgenic mice expressing a human mutant alpha-galactosidase with an R301Q substitution causing a variant form of Fabry disease
    • Generation and characterization of transgenic mice expressing a human mutant alpha-galactosidase with an R301Q substitution causing a variant form of Fabry disease. M Shimmoto R Kase K Itoh K Utsumi S Ishii C Taya H Yonekawa H Sakuraba, Vision Res 1997 48 353 359
    • (1997) Vision Res , vol.48 , pp. 353-359
    • Shimmoto, M.1    Kase, R.2    Itoh, K.3    Utsumi, K.4    Ishii, S.5    Taya, C.6    Yonekawa, H.7    Sakuraba, H.8
  • 28
    • 0031809296 scopus 로고    scopus 로고
    • Immunohistochemical characterization of transgenic mice highly expressing human lysosomal alpha-galactosidase
    • 9630664
    • Immunohistochemical characterization of transgenic mice highly expressing human lysosomal alpha-galactosidase. R Kase M Shimmoto K Itoh K Utsumi M Kotani C Taya H Yonekawa H Sakuraba, Biochim Biophys Acta 1998 1406 260 266 9630664
    • (1998) Biochim Biophys Acta , vol.1406 , pp. 260-266
    • Kase, R.1    Shimmoto, M.2    Itoh, K.3    Utsumi, K.4    Kotani, M.5    Taya, C.6    Yonekawa, H.7    Sakuraba, H.8
  • 29
    • 33947201045 scopus 로고    scopus 로고
    • Distribution of alpha-galactosidase A in normal human kidney and renal accumulation and distribution of recombinant alpha-galactosidase A in Fabry mice
    • 10.1681/ASN.2006080822. 17287429
    • Distribution of alpha-galactosidase A in normal human kidney and renal accumulation and distribution of recombinant alpha-galactosidase A in Fabry mice. EI Christensen Q Zhou SS Sørensen AK Rasmussen C Jacobsen U Feldt-Rasmussen R Nielsen, J Am Soc Nephrol 2007 18 698 706 10.1681/ASN. 2006080822 17287429
    • (2007) J Am Soc Nephrol , vol.18 , pp. 698-706
    • Christensen, E.I.1    Zhou, Q.2    Sørensen, S.S.3    Rasmussen, A.K.4    Jacobsen, C.5    Feldt-Rasmussen, U.6    Nielsen, R.7
  • 30
    • 0025826050 scopus 로고
    • Lysosomal storage diseases
    • 10.1146/annurev.bi.60.070191.001353. 1883197
    • Lysosomal storage diseases. EF Neufeld, Annu Rev Biochem 1991 60 257 280 10.1146/annurev.bi.60.070191.001353 1883197
    • (1991) Annu Rev Biochem , vol.60 , pp. 257-280
    • Neufeld, E.F.1
  • 31
    • 0036266877 scopus 로고    scopus 로고
    • Natural history and treatment of renal involvement in Fabry disease
    • 12068026
    • Natural history and treatment of renal involvement in Fabry disease. M Branton R Schiffmann JB Kopp, J Am Soc Nephrol 2002 13 139 143 12068026
    • (2002) J Am Soc Nephrol , vol.13 , pp. 19139-143
    • Branton, M.1    Schiffmann, R.2    Kopp, J.B.3
  • 32
    • 44449115633 scopus 로고    scopus 로고
    • Recommendations and guidelines for the diagnosis and treatment of Fabry nephropathy in adults
    • 10.1038/ncpneph0806. 18431378
    • Recommendations and guidelines for the diagnosis and treatment of Fabry nephropathy in adults. A Ortiz JP Oliveira C Wanner, Nat Clin Pract Nephrol 2008 4 327 336 10.1038/ncpneph0806 18431378
    • (2008) Nat Clin Pract Nephrol , vol.4 , pp. 327-336
    • Ortiz, A.1    Oliveira, J.P.2    Wanner, C.3
  • 33
    • 67651096945 scopus 로고    scopus 로고
    • Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease
    • 19707461
    • Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease. FC Fervenza R Torra DG Warnock, Biologics 2008 2 823 843 19707461
    • (2008) Biologics , vol.2 , pp. 823-843
    • Fervenza, F.C.1    Torra, R.2    Warnock, D.G.3
  • 35
    • 34548474768 scopus 로고    scopus 로고
    • Antiproteinuric therapy and fabry nephropathy: Sustained reduction of proteinuria in patients receiving enzyme replacement therapy with agalsidase-beta
    • 10.1681/ASN.2006121400. 17656478
    • Antiproteinuric therapy and fabry nephropathy: sustained reduction of proteinuria in patients receiving enzyme replacement therapy with agalsidase-beta. H Tahir LL Jackson DG Warnock, J Am Soc Nephrol 2007 18 2609 2617 10.1681/ASN.2006121400 17656478
    • (2007) J Am Soc Nephrol , vol.18 , pp. 2609-2617
    • Tahir, H.1    Jackson, L.L.2    Warnock, D.G.3
  • 36
    • 68949210394 scopus 로고    scopus 로고
    • Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies
    • 10.1089/hum.2009.094. 19569968
    • Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies. N Clément DR Knop BJ Byrne, Hum Gene Ther 2009 20 796 806 10.1089/hum.2009.094 19569968
    • (2009) Hum Gene Ther , vol.20 , pp. 796-806
    • Clément, N.1    Knop, D.R.2    Byrne, B.J.3


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