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Volumn 8, Issue 5, 1997, Pages 513-521

Adenovirus-mediated gene therapy in a mouse model of hereditary tyrosinemia type I

Author keywords

[No Author keywords available]

Indexed keywords

FUMARYLACETOACETASE;

EID: 0030917824     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1997.8.5-513     Document Type: Article
Times cited : (63)

References (38)
  • 1
    • 0028914687 scopus 로고
    • Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: Comparisons between immunocompetent and immunodeficient inbred strains
    • BARR, D., TUBB, J., FERGUSON, D., SCARIA, A., LIEBER, A., WILSON, C., PERKINS, J., and KAY, M.A. (1995). Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: comparisons between immunocompetent and immunodeficient inbred strains. Gene Ther. 2, 151-155.
    • (1995) Gene Ther. , vol.2 , pp. 151-155
    • Barr, D.1    Tubb, J.2    Ferguson, D.3    Scaria, A.4    Lieber, A.5    Wilson, C.6    Perkins, J.7    Kay, M.A.8
  • 2
    • 0017184389 scopus 로고
    • A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding
    • BRADFORD, M.M. (1976). A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding. Anal. Biochem. 72, 248-254.
    • (1976) Anal. Biochem. , vol.72 , pp. 248-254
    • Bradford, M.M.1
  • 3
    • 0026355641 scopus 로고
    • Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits
    • CHOWDHURY, J.R., GROSSMAN, M., GUPTA, S., CHOWDHURY, N.R., BAKER, J.R., JR., and WILSON, J.M. (1991). Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits. Science 254, 1802-1805.
    • (1991) Science , vol.254 , pp. 1802-1805
    • Chowdhury, J.R.1    Grossman, M.2    Gupta, S.3    Chowdhury, N.R.4    Baker Jr., J.R.5    Wilson, J.M.6
  • 4
    • 0029966514 scopus 로고    scopus 로고
    • Recombinant adenovirus deleted of all viral genes for gene therapy of cystic fibrosis
    • FISHER, K.J., CHOI, H., BURDA, J., CHEN, S.J., and WILSON, J.M. (1996). Recombinant adenovirus deleted of all viral genes for gene therapy of cystic fibrosis. Virology 217, 11-22.
    • (1996) Virology , vol.217 , pp. 11-22
    • Fisher, K.J.1    Choi, H.2    Burda, J.3    Chen, S.J.4    Wilson, J.M.5
  • 6
    • 0017710978 scopus 로고
    • Characteristics of a human cell line transformed by DNA from human adenovirus type 5
    • GRAHAM, F.L., SMILEY, J., RUSSELL, W.C., and NAIRN, R. (1977). Characteristics of a human cell line transformed by DNA from human adenovirus type 5. J. Gen. Virol. 36, 59-74.
    • (1977) J. Gen. Virol. , vol.36 , pp. 59-74
    • Graham, F.L.1    Smiley, J.2    Russell, W.C.3    Nairn, R.4
  • 7
    • 0001371293 scopus 로고
    • Tissue culture studies of the proliferative capacity of cervical carcinoma and normal epithelium
    • GRE, G.O., COFFMAN, W.D., and KUBICEK, M.T. (1952). Tissue culture studies of the proliferative capacity of cervical carcinoma and normal epithelium. Cancer Res. 12, 264-265.
    • (1952) Cancer Res. , vol.12 , pp. 264-265
    • Gre, G.O.1    Coffman, W.D.2    Kubicek, M.T.3
  • 8
    • 84966149718 scopus 로고
    • Succinylacetone
    • H. Bergmeyer, eds. (VCH Verlagsgesellschaft, Weinheim, Germany)
    • GRENIER, A., and LESCAULT, A. (1985). Succinylacetone. In Methods of Enzymatic Analysis. H. Bergmeyer, eds. (VCH Verlagsgesellschaft, Weinheim, Germany) p. 79.
    • (1985) Methods of Enzymatic Analysis , pp. 79
    • Grenier, A.1    Lescault, A.2
  • 9
    • 0027137419 scopus 로고
    • Loss of fumarylacetoacetate hydrolase is responsible for the neonatal hepatic dysfunction phenotype of lethal albino mice
    • GROMPE, M., AL-DHALIMY, M., FINEGOLD, M., OU, C.N., BURLINGAME, T., KENNAWAY, N.G., and SORIANO, P. (1993). Loss of fumarylacetoacetate hydrolase is responsible for the neonatal hepatic dysfunction phenotype of lethal albino mice. Genes & Dev. 7, 2298-2307.
    • (1993) Genes & Dev. , vol.7 , pp. 2298-2307
    • Grompe, M.1    Al-Dhalimy, M.2    Finegold, M.3    Ou, C.N.4    Burlingame, T.5    Kennaway, N.G.6    Soriano, P.7
  • 11
    • 0027499520 scopus 로고
    • Retroviruses: Delivery vehicle to the liver
    • GROSSMAN, M., and WILSON, J.M. (1993). Retroviruses: Delivery vehicle to the liver. Curr. Opin. Genet. Dev. 3, 110-114.
    • (1993) Curr. Opin. Genet. Dev. , vol.3 , pp. 110-114
    • Grossman, M.1    Wilson, J.M.2
  • 13
    • 0025822386 scopus 로고
    • Inherited hepatic enzyme defects as candidates for liver-directed gene therapy
    • HORWICH, A.L. (1991). Inherited hepatic enzyme defects as candidates for liver-directed gene therapy. Curr. Top. Microbiol. Immunol. 168, 185-200.
    • (1991) Curr. Top. Microbiol. Immunol. , vol.168 , pp. 185-200
    • Horwich, A.L.1
  • 15
    • 0027033259 scopus 로고
    • Hepatic gene therapy: Persistent expression of human alpha 1-antitrypsin in mice after direct gene delivery in vivo
    • KAY, M.A., LI, Q., LIU, T.J., LELAND, F., TOMAN, C., FINEGOLD, M., and WOO, S.L. (1992). Hepatic gene therapy: persistent expression of human alpha 1-antitrypsin in mice after direct gene delivery in vivo. Hum. Gene Ther. 3, 641-647.
    • (1992) Hum. Gene Ther. , vol.3 , pp. 641-647
    • Kay, M.A.1    Li, Q.2    Liu, T.J.3    Leland, F.4    Toman, C.5    Finegold, M.6    Woo, S.L.7
  • 17
    • 0029113869 scopus 로고
    • Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration
    • KAY, M.A., HOLTERMAN, A.X., MEUSE, L., GOWN, A., OCHS, H.D., LINSLEY, P.S., and WILSON, C.B. (1995). Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration. Nature Genet. 11, 191-197.
    • (1995) Nature Genet. , vol.11 , pp. 191-197
    • Kay, M.A.1    Holterman, A.X.2    Meuse, L.3    Gown, A.4    Ochs, H.D.5    Linsley, P.S.6    Wilson, C.B.7
  • 18
    • 0027131576 scopus 로고
    • Rescue of mice homozygous for lethal albino deletions: Implications for an animal model for the human liver disease tyrosinemia type I
    • KELSEY, G., RUPPERT, S., BEERMANN, F., GRUND, C., TANGUAY, R.M., and SCHÜTZ, G. (1993). Rescue of mice homozygous for lethal albino deletions: Implications for an animal model for the human liver disease tyrosinemia type I. Genes & Dev. 7, 2285-2297.
    • (1993) Genes & Dev. , vol.7 , pp. 2285-2297
    • Kelsey, G.1    Ruppert, S.2    Beermann, F.3    Grund, C.4    Tanguay, R.M.5    Schütz, G.6
  • 19
    • 77957012010 scopus 로고
    • Enzymes involved in conversion of tyrosine to acetoacetate
    • KNOX, W.E., and EDWARDS, S.W. (1995). Enzymes involved in conversion of tyrosine to acetoacetate. Methods Enzymol. 2, 287-300.
    • (1995) Methods Enzymol. , vol.2 , pp. 287-300
    • Knox, W.E.1    Edwards, S.W.2
  • 20
    • 0029943155 scopus 로고    scopus 로고
    • A new adenoviral vector: Replacement of all viral coding sequences with 28 Kb of DNA independently expressing both full length dystrophin and beta-galactosidase
    • KOCHANEK, S., CLEMENS, P.R., MITANI, K., CHEN, H.H., CHAN, S., and CASKEY, C.T. (1996). A new adenoviral vector: Replacement of all viral coding sequences with 28 Kb of DNA independently expressing both full length dystrophin and beta-galactosidase. Proc. Natl. Acad. Sci. USA 83, 5731-5736.
    • (1996) Proc. Natl. Acad. Sci. USA , vol.83 , pp. 5731-5736
    • Kochanek, S.1    Clemens, P.R.2    Mitani, K.3    Chen, H.H.4    Chan, S.5    Caskey, C.T.6
  • 21
    • 0027723616 scopus 로고
    • Adenovirus-mediated correction of the genetic defect in hepatocytes from patients with familial hypercholesterolemia
    • KOZARSKY, K., GROSSMAN, M., and WILSON, J.M. (1993). Adenovirus-mediated correction of the genetic defect in hepatocytes from patients with familial hypercholesterolemia. Somat. Cell Mol. Genet. 19, 449-458.
    • (1993) Somat. Cell Mol. Genet. , vol.19 , pp. 449-458
    • Kozarsky, K.1    Grossman, M.2    Wilson, J.M.3
  • 22
    • 0027316082 scopus 로고
    • Hereditary tyrosinemia type I. Self-induced correction of the fumarylacetoacetase defect
    • KVITTINGEN, E.A., ROOTWELT, H., BRANDTZAEG, P., BERGAN, A., and BERGER, R. (1993). Hereditary tyrosinemia type I. Self-induced correction of the fumarylacetoacetase defect. J. Clin. Invest. 91, 1816-1821.
    • (1993) J. Clin. Invest. , vol.91 , pp. 1816-1821
    • Kvittingen, E.A.1    Rootwelt, H.2    Brandtzaeg, P.3    Bergan, A.4    Berger, R.5
  • 23
    • 0028146998 scopus 로고
    • Self-induced correction of the genetic defect in tyrosinemia type I
    • KVITTINGEN, E.A., ROOTWELT, H., BERGER, R., and BRANDTZAEG, P. (1994). Self-induced correction of the genetic defect in tyrosinemia type I. J. Clin. Invest. 94, 1657-1661.
    • (1994) J. Clin. Invest. , vol.94 , pp. 1657-1661
    • Kvittingen, E.A.1    Rootwelt, H.2    Berger, R.3    Brandtzaeg, P.4
  • 24
    • 0027404488 scopus 로고
    • Localization of cells in the rat brain expressing fumarylacetoacetate hydrolase, the deficient enzyme in hereditary tyrosinemia type 1
    • LABELLE, Y., PUYMIRAT, J., and TANGUAY, R.M. (1993). Localization of cells in the rat brain expressing fumarylacetoacetate hydrolase, the deficient enzyme in hereditary tyrosinemia type 1. Biochim. Biophys. Acta 1180, 250-256.
    • (1993) Biochim. Biophys. Acta , vol.1180 , pp. 250-256
    • Labelle, Y.1    Puymirat, J.2    Tanguay, R.M.3
  • 26
    • 0026675589 scopus 로고
    • Treatment of hereditary tyrosinaemia type I by inhibition of 4-hydroxyphenylpyruvate dioxygenase
    • LINDSTEDT, S., HOLME, E., LOCK, E.A., HJALMARSON, O., and STRANDVIK, B. (1992). Treatment of hereditary tyrosinaemia type I by inhibition of 4-hydroxyphenylpyruvate dioxygenase. Lancet 340, 813-817.
    • (1992) Lancet , vol.340 , pp. 813-817
    • Lindstedt, S.1    Holme, E.2    Lock, E.A.3    Hjalmarson, O.4    Strandvik, B.5
  • 27
    • 0000362736 scopus 로고
    • Hypertyrosinemia
    • C.R. Scriver, A.L. Beaudet, W. Sly, and D. Valle, eds. (MacGraw-Hill, New York)
    • MITCHELL, G.A., LAMBERT, M., and TANGUAY, R.M. (1994). Hypertyrosinemia. In The Metabolic Basis of Inherited Disease. C.R. Scriver, A.L. Beaudet, W. Sly, and D. Valle, eds. (MacGraw-Hill, New York) pp. 1077-1106.
    • (1994) The Metabolic Basis of Inherited Disease , pp. 1077-1106
    • Mitchell, G.A.1    Lambert, M.2    Tanguay, R.M.3
  • 28
    • 0027272371 scopus 로고
    • Efficient adenoviral-mediated ornithine transcarbamylase expression in deficient mouse and human hepatocytes
    • MORSY, M.A., ALFORD, E.L., BETT, A., GRAHAM, F.L., and CASKEY, C.T. (1993). Efficient adenoviral-mediated ornithine transcarbamylase expression in deficient mouse and human hepatocytes. J. Clin. Invest. 92, 1580-1586.
    • (1993) J. Clin. Invest. , vol.92 , pp. 1580-1586
    • Morsy, M.A.1    Alford, E.L.2    Bett, A.3    Graham, F.L.4    Caskey, C.T.5
  • 30
    • 0029875835 scopus 로고    scopus 로고
    • Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinemia type I
    • OVERTURF, K., AL-DHALIMY, M., TANGUAY, R., BRANTLY, M., OU, C.N., FINEGOLD, M., and GROMPE, M. (1996). Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinemia type I. Nature Genet. 12, 266-273.
    • (1996) Nature Genet. , vol.12 , pp. 266-273
    • Overturf, K.1    Al-Dhalimy, M.2    Tanguay, R.3    Brantly, M.4    Ou, C.N.5    Finegold, M.6    Grompe, M.7
  • 32
    • 0025977277 scopus 로고
    • Cloning and expression of the cDNA encoding human fumarylacetoacetae hydrolase, the enzyme deficient in hereditary tyrosinemia: Assignment of the gene to chromosome 15
    • PHANEUF, D., LABELLE, Y., BÉRUBÉ, D., ARDEN, K., CAVENEE, W., GAGNÉ, R., and TANGUAY, R.M. (1991). Cloning and expression of the cDNA encoding human fumarylacetoacetae hydrolase, the enzyme deficient in hereditary tyrosinemia: Assignment of the gene to chromosome 15. Am. J. Hum. Genet. 48, 525-535.
    • (1991) Am. J. Hum. Genet. , vol.48 , pp. 525-535
    • Phaneuf, D.1    Labelle, Y.2    Bérubé, D.3    Arden, K.4    Cavenee, W.5    Gagné, R.6    Tanguay, R.M.7
  • 35
    • 0023122779 scopus 로고
    • Effects of succinylacetone on amino acid uptake in the rat kidney
    • SPENCER, P.D., and ROTH, K.S. (1987). Effects of succinylacetone on amino acid uptake in the rat kidney. Biochem. Med. Metab. Biol. 37, 101-109.
    • (1987) Biochem. Med. Metab. Biol. , vol.37 , pp. 101-109
    • Spencer, P.D.1    Roth, K.S.2
  • 36
    • 0025487393 scopus 로고
    • Evaluation of the transfer and expression in mice of an enzyme-encoding gene using a human adenovirus vector
    • STRATFORD-PERRICAUDET, L.D., LEVRERO, M., CHASSE, J.F., PERRICAUDET, M., and BRIAND, P. (1990). Evaluation of the transfer and expression in mice of an enzyme-encoding gene using a human adenovirus vector. Hum. Gene Ther. 1, 241-256.
    • (1990) Hum. Gene Ther. , vol.1 , pp. 241-256
    • Stratford-Perricaudet, L.D.1    Levrero, M.2    Chasse, J.F.3    Perricaudet, M.4    Briand, P.5
  • 37
    • 0026751864 scopus 로고
    • Physiological basis for an animal model of the renal Fanconi syndrome: Use of succinylacetone in the rat
    • WYSS, P.A., BOYNTON, S.B., CHU, J., SPENCER, R.F., and ROTH, K.S. (1992). Physiological basis for an animal model of the renal Fanconi syndrome: Use of succinylacetone in the rat. Clin. Sci. 83, 81-87.
    • (1992) Clin. Sci. , vol.83 , pp. 81-87
    • Wyss, P.A.1    Boynton, S.B.2    Chu, J.3    Spencer, R.F.4    Roth, K.S.5


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