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Volumn 15, Issue 2, 2007, Pages 248-254

Infectious delivery and expression of a 135kb human FRDA genomic DNA locus complements friedreich's ataxia deficiency in human cells

Author keywords

[No Author keywords available]

Indexed keywords

FRATAXIN; GENOMIC DNA; HERPESVIRUS VECTOR;

EID: 33846308499     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/sj.mt.6300021     Document Type: Article
Times cited : (56)

References (28)
  • 1
    • 0036603021 scopus 로고    scopus 로고
    • Friedreich ataxia: A paradigm for mitochondrial diseases
    • Puccio, H and Koenig, M (2002). Friedreich ataxia: A paradigm for mitochondrial diseases. Curr Opin Genet Dev 12: 272-277.
    • (2002) Curr Opin Genet Dev , vol.12 , pp. 272-277
    • Puccio, H.1    Koenig, M.2
  • 2
    • 0037101845 scopus 로고    scopus 로고
    • The yeast frataxin homolog Yfh1p plays a specific role in the maturation of cellular Fe/S proteins
    • Muhlenhoff, U, Richhardt, N, Ristow, M, Kispal, G and Lill, R (2002). The yeast frataxin homolog Yfh1p plays a specific role in the maturation of cellular Fe/S proteins. Hum Mol Genet 11: 2025-2036.
    • (2002) Hum Mol Genet , vol.11 , pp. 2025-2036
    • Muhlenhoff, U.1    Richhardt, N.2    Ristow, M.3    Kispal, G.4    Lill, R.5
  • 3
    • 17144378216 scopus 로고    scopus 로고
    • Iron-sulphur cluster biogenesis and mitochondrial iron homeostasis
    • Rouault, TA and Tong, WH (2005). Iron-sulphur cluster biogenesis and mitochondrial iron homeostasis. Nat Rev Mol Cell Biol 6: 345-351.
    • (2005) Nat Rev Mol Cell Biol , vol.6 , pp. 345-351
    • Rouault, T.A.1    Tong, W.H.2
  • 5
    • 0036940427 scopus 로고    scopus 로고
    • The molecular basis of Friedreich ataxia
    • Pandolfo, M (2002). The molecular basis of Friedreich ataxia. Adv Exp Med Biol 516: 99-118.
    • (2002) Adv Exp Med Biol , vol.516 , pp. 99-118
    • Pandolfo, M.1
  • 6
    • 13344270899 scopus 로고    scopus 로고
    • Friedreich's ataxia: Autosomal recessive disease caused by an intronic GAA triplet repeat expansion
    • Campuzano, V et al. (1996). Friedreich's ataxia: Autosomal recessive disease caused by an intronic GAA triplet repeat expansion. Science 271: 1423-1427.
    • (1996) Science , vol.271 , pp. 1423-1427
    • Campuzano, V.1
  • 7
    • 0030846021 scopus 로고    scopus 로고
    • Regulation of mitochondrial iron accumulation by Yfh1p, a putative homolog of frataxin
    • Babcock, M et al. (1997). Regulation of mitochondrial iron accumulation by Yfh1p, a putative homolog of frataxin. Science 276: 1709-1712.
    • (1997) Science , vol.276 , pp. 1709-1712
    • Babcock, M.1
  • 8
    • 0033054177 scopus 로고    scopus 로고
    • The Friedreich's ataxia mutation confers cellular sensitivity to oxidant stress which is rescued by chelators of iron and calcium and inhibitors of apoptosis
    • Wong, A et al. (1999). The Friedreich's ataxia mutation confers cellular sensitivity to oxidant stress which is rescued by chelators of iron and calcium and inhibitors of apoptosis. Hum Mol Genet 8: 425-430.
    • (1999) Hum Mol Genet , vol.8 , pp. 425-430
    • Wong, A.1
  • 9
    • 0034778392 scopus 로고    scopus 로고
    • Disabled early recruitment of antioxidant defenses in Friedreich's ataxia
    • Chantrel-Groussard, K et al. (2001). Disabled early recruitment of antioxidant defenses in Friedreich's ataxia. Hum Mol Genet 10: 2061-2067.
    • (2001) Hum Mol Genet , vol.10 , pp. 2061-2067
    • Chantrel-Groussard, K.1
  • 10
    • 0035976855 scopus 로고    scopus 로고
    • Manganese superoxide dismutase induction by iron is impaired in Friedreich ataxia cells
    • Jiralerspong, S, Ge, B, Hudson, TJ and Pandolfo, M (2001). Manganese superoxide dismutase induction by iron is impaired in Friedreich ataxia cells. FEBS Lett 509: 101-105.
    • (2001) FEBS Lett , vol.509 , pp. 101-105
    • Jiralerspong, S.1    Ge, B.2    Hudson, T.J.3    Pandolfo, M.4
  • 11
    • 0348136710 scopus 로고    scopus 로고
    • Mitochondria-targeted antioxidants protect Friedreich Ataxia fibroblasts from endogenous oxidative stress more effectively than untargeted antioxidants
    • Jauslin, ML, Wirth, T, Meier, T and Schoumacher, F (2003). Mitochondria-targeted antioxidants protect Friedreich Ataxia fibroblasts from endogenous oxidative stress more effectively than untargeted antioxidants. FASEB J 17: 1972-1974.
    • (2003) FASEB J , vol.17 , pp. 1972-1974
    • Jauslin, M.L.1    Wirth, T.2    Meier, T.3    Schoumacher, F.4
  • 12
    • 0038448961 scopus 로고    scopus 로고
    • Upregulation of expression from the FRDA genomic locus for the therapy of Friedreich ataxia
    • Sarsero, JP et al. (2003). Upregulation of expression from the FRDA genomic locus for the therapy of Friedreich ataxia. J Gene Med 5: 72-81.
    • (2003) J Gene Med , vol.5 , pp. 72-81
    • Sarsero, J.P.1
  • 13
    • 2542556601 scopus 로고    scopus 로고
    • Idebenone delays the onset of cardiac functional alteration without correction of Fe-S enzymes deficit in a mouse model for Friedreich ataxia
    • Seznec, H et al. (2004). Idebenone delays the onset of cardiac functional alteration without correction of Fe-S enzymes deficit in a mouse model for Friedreich ataxia. Hum Mol Genet 13: 1017-1024.
    • (2004) Hum Mol Genet , vol.13 , pp. 1017-1024
    • Seznec, H.1
  • 14
    • 0036221156 scopus 로고    scopus 로고
    • Idebenone and reduced cardiac hypertrophy in Friedreich's ataxia
    • Hausse, AO et al. (2002). Idebenone and reduced cardiac hypertrophy in Friedreich's ataxia. Heart 87: 346-349.
    • (2002) Heart , vol.87 , pp. 346-349
    • Hausse, A.O.1
  • 15
    • 0035865087 scopus 로고    scopus 로고
    • A physical map of the human genome
    • McPherson, JD et al. (2001). A physical map of the human genome. Nature 409: 934-941.
    • (2001) Nature , vol.409 , pp. 934-941
    • McPherson, J.D.1
  • 16
    • 0020451027 scopus 로고
    • The herpes simplex virus amplicon: A new eucaryotic defective-virus cloning-amplifying vector
    • Spaete, RR and Frenkel, N (1982). The herpes simplex virus amplicon: A new eucaryotic defective-virus cloning-amplifying vector. Cell 30: 295-304.
    • (1982) Cell , vol.30 , pp. 295-304
    • Spaete, R.R.1    Frenkel, N.2
  • 18
    • 4644362951 scopus 로고    scopus 로고
    • Update on herpes virus amplicon vectors
    • Oehmig, A, Fraefel, C and Breakefield, XO (2004). Update on herpes virus amplicon vectors. Mol Ther 10: 630-643.
    • (2004) Mol Ther , vol.10 , pp. 630-643
    • Oehmig, A.1    Fraefel, C.2    Breakefield, X.O.3
  • 19
    • 0036202348 scopus 로고    scopus 로고
    • Highly efficient and specific gene transfer to Purkinje cells in vivo using a herpes simplex virus I amplicon
    • Agudo, M et al. (2002). Highly efficient and specific gene transfer to Purkinje cells in vivo using a herpes simplex virus I amplicon. Hum Gene Ther 13: 665-674.
    • (2002) Hum Gene Ther , vol.13 , pp. 665-674
    • Agudo, M.1
  • 20
    • 33746217565 scopus 로고    scopus 로고
    • Bioluminescence imaging after HSV amplicon vector delivery into brain
    • Pike, L, Petravicz, J and Wang, S (2006). Bioluminescence imaging after HSV amplicon vector delivery into brain. J Gene Med 8: 804-813.
    • (2006) J Gene Med , vol.8 , pp. 804-813
    • Pike, L.1    Petravicz, J.2    Wang, S.3
  • 21
    • 0034756520 scopus 로고    scopus 로고
    • An infectious transfer and expression system for genomic DNA loci in human and mouse cells
    • Wade-Martins, R, Smith, ER, Tyminski, E, Chiocca, EA and Saeki, Y (2001). An infectious transfer and expression system for genomic DNA loci in human and mouse cells. Nat Biotechnol 19: 1067-1070.
    • (2001) Nat Biotechnol , vol.19 , pp. 1067-1070
    • Wade-Martins, R.1    Smith, E.R.2    Tyminski, E.3    Chiocca, E.A.4    Saeki, Y.5
  • 22
    • 0038237520 scopus 로고    scopus 로고
    • Infectious delivery of a 135-kb LDLR genomic locus leads to regulated complementation of low-density lipoprotein receptor deficiency in human cells
    • Wade-Martins, R, Saeki, Y and Chiocca, EA (2003). Infectious delivery of a 135-kb LDLR genomic locus leads to regulated complementation of low-density lipoprotein receptor deficiency in human cells. Mol Ther 7: 604-612.
    • (2003) Mol Ther , vol.7 , pp. 604-612
    • Wade-Martins, R.1    Saeki, Y.2    Chiocca, E.A.3
  • 23
    • 4243175966 scopus 로고    scopus 로고
    • Infectious delivery of the 132 kb CDKN2A/CDKN2B genomic DNA region results in correctly spliced gene expression and growth suppression in glioma cells
    • Inoue, R, Moghaddam, KA, Ranasinghe, M, Saeki, Y, Chiocca, EA and Wade-Martins, R (2004). Infectious delivery of the 132 kb CDKN2A/CDKN2B genomic DNA region results in correctly spliced gene expression and growth suppression in glioma cells. Gene Therapy 11: 1195-1204.
    • (2004) Gene Therapy , vol.11 , pp. 1195-1204
    • Inoue, R.1    Moghaddam, K.A.2    Ranasinghe, M.3    Saeki, Y.4    Chiocca, E.A.5    Wade-Martins, R.6
  • 24
    • 23344450226 scopus 로고    scopus 로고
    • Herpes simplex virus type 1 amplicon vectors for the infectious delivery and expression of genomic DNA loci
    • Senior, SL and Wade-Martins, R (2005). Herpes simplex virus type 1 amplicon vectors for the infectious delivery and expression of genomic DNA loci. Cur Opin Mol Ther 7: 337-345.
    • (2005) Cur Opin Mol Ther , vol.7 , pp. 337-345
    • Senior, S.L.1    Wade-Martins, R.2
  • 25
    • 0034991897 scopus 로고    scopus 로고
    • Improved helper virus-free packaging system for HSV amplicon vectors using an ICP27-deleted, oversized HSV-1 DNA in a bacterial artificial chromosome
    • Saeki, Y, Fraefel, C, Ichikawa, T, Breakefield, XO and Chiocca, EA (2001). Improved helper virus-free packaging system for HSV amplicon vectors using an ICP27-deleted, oversized HSV-1 DNA in a bacterial artificial chromosome. Mol Ther 3: 591-601.
    • (2001) Mol Ther , vol.3 , pp. 591-601
    • Saeki, Y.1    Fraefel, C.2    Ichikawa, T.3    Breakefield, X.O.4    Chiocca, E.A.5
  • 26
    • 9844222853 scopus 로고    scopus 로고
    • Frataxin is reduced in Friedreich ataxia patients and is associated with mitochondrial membranes
    • Campuzano, V et al. (1997). Frataxin is reduced in Friedreich ataxia patients and is associated with mitochondrial membranes. Hum Mol Genet 6: 1771-1780.
    • (1997) Hum Mol Genet , vol.6 , pp. 1771-1780
    • Campuzano, V.1
  • 27
    • 0033669478 scopus 로고    scopus 로고
    • Stable correction of a genetic deficiency in human cells by an episome carrying a 115 kb genomic transgene
    • Wade-Martins, R, White, RE, Kimura, H, Cook, P and James, MR (2000). Stable correction of a genetic deficiency in human cells by an episome carrying a 115 kb genomic transgene. Nat Biotechnol 18: 1311-1314.
    • (2000) Nat Biotechnol , vol.18 , pp. 1311-1314
    • Wade-Martins, R.1    White, R.E.2    Kimura, H.3    Cook, P.4    James, M.R.5
  • 28
    • 23844514701 scopus 로고    scopus 로고
    • Partial correction of sensitivity to oxidant stress in Friedreich's ataxia patient fibroblasts by frataxin-encoding adeno-associated virus and lentivirus vectors
    • Fleming, J et al. (2005). Partial correction of sensitivity to oxidant stress in Friedreich's ataxia patient fibroblasts by frataxin-encoding adeno-associated virus and lentivirus vectors. Hum Gene Ther 16: 1-10.
    • (2005) Hum Gene Ther , vol.16 , pp. 1-10
    • Fleming, J.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.