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Volumn 17, Issue 7, 2009, Pages 1266-1273

Hepatocyte transplantation improves phenotype and extends survival in a murine model of intermediate maple syrup urine disease

Author keywords

[No Author keywords available]

Indexed keywords

2 OXOISOVALERATE DEHYDROGENASE (LIPOAMIDE); ALANINE; BRANCHED CHAIN AMINO ACID;

EID: 67649861071     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2009.99     Document Type: Article
Times cited : (29)

References (48)
  • 1
    • 0345735297 scopus 로고    scopus 로고
    • Mechanisms responsible for regulation of branched-chain amino acid catabolism
    • Harris, RA, Joshi, M and Jeoung, NH (2004). Mechanisms responsible for regulation of branched-chain amino acid catabolism. Biochem Biophys Res Commun 313: 391-396.
    • (2004) Biochem Biophys Res Commun , vol.313 , pp. 391-396
    • Harris, R.A.1    Joshi, M.2    Jeoung, N.H.3
  • 2
    • 0024578239 scopus 로고
    • The 2-oxo acid dehydrogenase complexes: Recent advances
    • Yeaman, S (1989). The 2-oxo acid dehydrogenase complexes: Recent advances. Biochem J 257: 625-632.
    • (1989) Biochem J , vol.257 , pp. 625-632
    • Yeaman, S.1
  • 3
    • 0002977005 scopus 로고    scopus 로고
    • Maple syrup urine disease (branched-chain ketoaciduria)
    • Scriver, CR, Beaudet, AL, Sly, WS, Valle, D eds, 8th edn. McGraw-Hill Medical Publishing: New York. pp
    • Chuang, DT and Shih, VE (2001). Maple syrup urine disease (branched-chain ketoaciduria). In: Scriver, CR, Beaudet, AL, Sly, WS, Valle, D (eds). The Metabolic and Molecular Basis of Inherited Disease, 8th edn. McGraw-Hill Medical Publishing: New York. pp. 1971-2005.
    • (2001) The Metabolic and Molecular Basis of Inherited Disease , pp. 1971-2005
    • Chuang, D.T.1    Shih, V.E.2
  • 4
    • 0001592465 scopus 로고
    • Metabolism of the white blood cells in maple syrup urine disease
    • Dancis, J, Hutzler, J and Levitz, M (1960). Metabolism of the white blood cells in maple syrup urine disease. Biochem Biophys Acta 43: 342-343.
    • (1960) Biochem Biophys Acta , vol.43 , pp. 342-343
    • Dancis, J.1    Hutzler, J.2    Levitz, M.3
  • 5
    • 0001468282 scopus 로고
    • A new syndrome: Progressive familial infantile cerebral dysfunction associated with an unusual urinary substance
    • Menkes, J, Hurst, P and Craig, J (1954). A new syndrome: Progressive familial infantile cerebral dysfunction associated with an unusual urinary substance. Pediatrics 14: 462-466.
    • (1954) Pediatrics , vol.14 , pp. 462-466
    • Menkes, J.1    Hurst, P.2    Craig, J.3
  • 6
    • 33646191736 scopus 로고
    • Maple syrup urine disease: Clinical and molecular genetic considerations
    • Rosenberg, R, Prusiner, S, DiMauro, R, Barchi, R and Kunkel, L eds, Butterworth-Heinemann: Boston. pp
    • Cox, RP and Chuang, DT (1993). Maple syrup urine disease: Clinical and molecular genetic considerations. In: Rosenberg, R, Prusiner, S, DiMauro, R, Barchi, R and Kunkel, L (eds). The Molecular and Genetic Basis for Neurological Disease. Butterworth-Heinemann: Boston. pp. 189-207.
    • (1993) The Molecular and Genetic Basis for Neurological Disease , pp. 189-207
    • Cox, R.P.1    Chuang, D.T.2
  • 9
    • 0034502503 scopus 로고    scopus 로고
    • Branched-chain L-amino acid metabolism in classical maple syrup urine disease after orthotopic liver transplantation
    • Bodner-Leidecker, A, Wendel, U, Saudubray, JM and Schadewaldt, P (2000). Branched-chain L-amino acid metabolism in classical maple syrup urine disease after orthotopic liver transplantation. J Inherit Metab Dis 23: 805-818.
    • (2000) J Inherit Metab Dis , vol.23 , pp. 805-818
    • Bodner-Leidecker, A.1    Wendel, U.2    Saudubray, J.M.3    Schadewaldt, P.4
  • 10
    • 0028065296 scopus 로고
    • Mid-term outcome of 2 cases with maple syrup urine disease: Role of liver transplantation in the treatment
    • Netter, JC, Cossarizza, G, Narcy, C, Hubert, P, Ogier, H, Revillon, Y et al. (1994). Mid-term outcome of 2 cases with maple syrup urine disease: Role of liver transplantation in the treatment. Arch Pediatr 1: 730-734.
    • (1994) Arch Pediatr , vol.1 , pp. 730-734
    • Netter, J.C.1    Cossarizza, G.2    Narcy, C.3    Hubert, P.4    Ogier, H.5    Revillon, Y.6
  • 11
    • 33745219691 scopus 로고    scopus 로고
    • Hepatocyte transplantation: Clinical experience and potential for future use
    • Strom, SC, Bruzzone, P, Cai, H, Ellis, E, Lehmann, T, Mitamura, K et al. (2006). Hepatocyte transplantation: Clinical experience and potential for future use. Cell Transplant 15 Suppl 1: S105-S110.
    • (2006) Cell Transplant , vol.15 , Issue.SUPPL. 1
    • Strom, S.C.1    Bruzzone, P.2    Cai, H.3    Ellis, E.4    Lehmann, T.5    Mitamura, K.6
  • 14
    • 0017471623 scopus 로고
    • Correction of hyperbilirubinemia in the glucuronyltransferase-deficient rat by intraportal hepatocyte transplantation
    • Groth, C, Arborgh, B, Bjorken, C, Sundberg, B and Lundgren, G (1977). Correction of hyperbilirubinemia in the glucuronyltransferase-deficient rat by intraportal hepatocyte transplantation. Transplant Proc 9: 313-316.
    • (1977) Transplant Proc , vol.9 , pp. 313-316
    • Groth, C.1    Arborgh, B.2    Bjorken, C.3    Sundberg, B.4    Lundgren, G.5
  • 16
    • 0017062465 scopus 로고
    • Hepatocellular transplantation for metabolic deficiencies: Decrease of plasma bilirubin in Gunn rats
    • Matas, AJ, Sutherland, DE, Steffes, MW, Mauer, SM, Sowe, A, Simmons, RL et al. (1976). Hepatocellular transplantation for metabolic deficiencies: Decrease of plasma bilirubin in Gunn rats. Science 192: 892-894.
    • (1976) Science , vol.192 , pp. 892-894
    • Matas, A.J.1    Sutherland, D.E.2    Steffes, M.W.3    Mauer, S.M.4    Sowe, A.5    Simmons, R.L.6
  • 18
    • 0037176516 scopus 로고    scopus 로고
    • Hepatocyte transplantation as a treatment for glycogen storage disease type 1a
    • Muraca, M, Gerunda, G, Neri, D, Vilei, MT, Granato, A, Feltracco, P et al. (2002). Hepatocyte transplantation as a treatment for glycogen storage disease type 1a. Lancet 359: 317-318.
    • (2002) Lancet , vol.359 , pp. 317-318
    • Muraca, M.1    Gerunda, G.2    Neri, D.3    Vilei, M.T.4    Granato, A.5    Feltracco, P.6
  • 19
    • 0034981021 scopus 로고    scopus 로고
    • Correction of liver disease following transplantation of normal rat hepatocytes into Long-Evans Cinnamon rats modeling Wilson's disease
    • Irani, AN, Malhi, H, Slehria, S, Gorla, GR, Volenberg, I, Schilsky, ML et al. (2001). Correction of liver disease following transplantation of normal rat hepatocytes into Long-Evans Cinnamon rats modeling Wilson's disease. Mol Ther 3: 302-309.
    • (2001) Mol Ther , vol.3 , pp. 302-309
    • Irani, A.N.1    Malhi, H.2    Slehria, S.3    Gorla, G.R.4    Volenberg, I.5    Schilsky, M.L.6
  • 20
    • 0037978092 scopus 로고    scopus 로고
    • Isolated hepatocyte transplantation in an infant with a severe urea cycle disorder
    • Horslen, SP, McCowan, TC, Goertzen, TC, Warkentin, PI, Cai, HB, Strom, SC et al. (2003). Isolated hepatocyte transplantation in an infant with a severe urea cycle disorder. Pediatrics 111 (6 Pt 1): 1262-1267.
    • (2003) Pediatrics , vol.111 , Issue.6 PART 1 , pp. 1262-1267
    • Horslen, S.P.1    McCowan, T.C.2    Goertzen, T.C.3    Warkentin, P.I.4    Cai, H.B.5    Strom, S.C.6
  • 22
    • 43449122185 scopus 로고    scopus 로고
    • Correction of hyperoxaluria by liver repopulation with hepatocytes in a mouse model of primary hyperoxaluria type-1
    • Jiang, J, Salido, EC, Guha, C, Wang, X, Moitra, R, Liu, L et al. (2008). Correction of hyperoxaluria by liver repopulation with hepatocytes in a mouse model of primary hyperoxaluria type-1. Transplantation 85: 1253-1260.
    • (2008) Transplantation , vol.85 , pp. 1253-1260
    • Jiang, J.1    Salido, E.C.2    Guha, C.3    Wang, X.4    Moitra, R.5    Liu, L.6
  • 24
    • 0032898238 scopus 로고    scopus 로고
    • Oren, R, Dabeva, MD, Petkov, PM, Hurston, E, Laconi, E and Shafritz, DA (1999). Restoration of serum albumin levels in nagase analbuminemic rats by hepatocyte transplantation. Hepatology 29 b: 75-81.
    • Oren, R, Dabeva, MD, Petkov, PM, Hurston, E, Laconi, E and Shafritz, DA (1999). Restoration of serum albumin levels in nagase analbuminemic rats by hepatocyte transplantation. Hepatology 29 b: 75-81.
  • 25
    • 0029875835 scopus 로고    scopus 로고
    • Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type I
    • Overturf, K, Al-Dhalimy, M, Tanguay, R, Brantly, M, Ou, CN, Finegold, M et al. (1996). Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type I. Nat Genet 12: 266-273.
    • (1996) Nat 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
  • 26
    • 0031056013 scopus 로고    scopus 로고
    • Hepatocyte transplantation as a bridge to orthotopic liver transplantation in terminal liver failure
    • Strom, SC, Fisher, RA, Thompson, MT, Sanyal, AJ, Cole, PE, Ham, JM et al. (1997). Hepatocyte transplantation as a bridge to orthotopic liver transplantation in terminal liver failure. Transplantation 63: 559-569.
    • (1997) Transplantation , vol.63 , pp. 559-569
    • Strom, S.C.1    Fisher, R.A.2    Thompson, M.T.3    Sanyal, A.J.4    Cole, P.E.5    Ham, J.M.6
  • 27
    • 0344142526 scopus 로고    scopus 로고
    • Hepatocyte transplantation for the treatment of human disease
    • Strom, SC, Chowdhury, J and Fox, IJ (1999). Hepatocyte transplantation for the treatment of human disease. Semin Liver Dis 19: 39-48.
    • (1999) Semin Liver Dis , vol.19 , pp. 39-48
    • Strom, S.C.1    Chowdhury, J.2    Fox, I.J.3
  • 28
    • 0034719362 scopus 로고    scopus 로고
    • Defining hepatocellular chimerism in a liver failure patient bridged with hepatocyte infusion
    • Fisher, RA, Bu, D, Thompson, M, Tisnado, J, Prasad, U, Stirling, R et al. (2000). Defining hepatocellular chimerism in a liver failure patient bridged with hepatocyte infusion. Transplantation 69 303-307.
    • (2000) Transplantation , vol.69 , pp. 303-307
    • Fisher, R.A.1    Bu, D.2    Thompson, M.3    Tisnado, J.4    Prasad, U.5    Stirling, R.6
  • 30
    • 33646195697 scopus 로고    scopus 로고
    • Production and characterization of murine models of classic and intermediate maple syrup urine disease
    • Homanics, GE, Skvorak, K, Ferguson, C, Watkins, S and Paul, HS (2006). Production and characterization of murine models of classic and intermediate maple syrup urine disease. BMC Med Gen 7: 33.
    • (2006) BMC Med Gen , vol.7 , pp. 33
    • Homanics, G.E.1    Skvorak, K.2    Ferguson, C.3    Watkins, S.4    Paul, H.S.5
  • 31
    • 0028895728 scopus 로고
    • Rapid diagnosis of maple syrup urine disease in blood spots from newborns by tandem mass spectrometry
    • Chace, DH, Hillman, SL, Millington, DS, Kahler, SG, Roe, CR and Naylor, EW (1995). Rapid diagnosis of maple syrup urine disease in blood spots from newborns by tandem mass spectrometry. Clin Chem 41: 62-68.
    • (1995) Clin Chem , vol.41 , pp. 62-68
    • Chace, D.H.1    Hillman, S.L.2    Millington, D.S.3    Kahler, S.G.4    Roe, C.R.5    Naylor, E.W.6
  • 32
    • 0001783517 scopus 로고
    • Amino acid analysis of physiological samples
    • Hommes, FA ed, 1st edn, Wiley-Liss: New York. pp
    • Slocum, RH and Cummings, JG (1991). Amino acid analysis of physiological samples. In: Hommes, FA (ed). Techniques in Diagnostic Human Biochemical Genetics, 1st edn., Wiley-Liss: New York. pp. 87-126.
    • (1991) Techniques in Diagnostic Human Biochemical Genetics , pp. 87-126
    • Slocum, R.H.1    Cummings, J.G.2
  • 33
    • 64449083415 scopus 로고    scopus 로고
    • Animal models of maple syrup urine disease
    • Skvorak, KJ (2009). Animal models of maple syrup urine disease. J Inherit Metab Dis 32: 229-246.
    • (2009) J Inherit Metab Dis , vol.32 , pp. 229-246
    • Skvorak, K.J.1
  • 34
    • 64449086258 scopus 로고    scopus 로고
    • Dual mechanisms of brain injury and novel treatment strategy in maple syrup urine disease
    • Zinnanti, WJ, Lazovic, J, Griffin, K, Skvorak, KJ, Paul, HS, Homanics, GE et al. (2009). Dual mechanisms of brain injury and novel treatment strategy in maple syrup urine disease. Brain 132 (Pt 4): 903-918.
    • (2009) Brain , vol.132 , Issue.PART 4 , pp. 903-918
    • Zinnanti, W.J.1    Lazovic, J.2    Griffin, K.3    Skvorak, K.J.4    Paul, H.S.5    Homanics, G.E.6
  • 35
    • 15244341687 scopus 로고    scopus 로고
    • Branched chain amino acids as a parameter for catabolism in treated phenylketonuria
    • Illsinger S, LT, Meyer, U, Vaske B and Das AM (2005). Branched chain amino acids as a parameter for catabolism in treated phenylketonuria. Amino Acids 28: 45-50.
    • (2005) Amino Acids , vol.28 , pp. 45-50
    • Illsinger, S.L.1    Meyer, U.2    Vaske, B.3    Das, A.M.4
  • 36
    • 0018611755 scopus 로고
    • Morphology and function of isolated hepatocytes transplanted into rat spleen
    • Mito, M, Ebata, H, Kusano, M, Onishi, T, Saito, T and Sakamoto, S (1979). Morphology and function of isolated hepatocytes transplanted into rat spleen. Transplatation 28: 499-505.
    • (1979) Transplatation , vol.28 , pp. 499-505
    • Mito, M.1    Ebata, H.2    Kusano, M.3    Onishi, T.4    Saito, T.5    Sakamoto, S.6
  • 38
    • 0026031297 scopus 로고
    • Mouse hepatocytes migrate to liver parenchyma and function indefinitely after intrasplenic transplantation
    • Ponder, KP, Gupta, S, Leland, F, Darlington, G, Finegold, M, DeMayo, J et al. (1991). Mouse hepatocytes migrate to liver parenchyma and function indefinitely after intrasplenic transplantation. Proc Natl Acad Sci USA 88: 1217-1221.
    • (1991) Proc Natl Acad Sci USA , vol.88 , pp. 1217-1221
    • Ponder, K.P.1    Gupta, S.2    Leland, F.3    Darlington, G.4    Finegold, M.5    DeMayo, J.6
  • 39
    • 0035406246 scopus 로고    scopus 로고
    • Amplification of engrafted hepatocytes by preparative manipulation of the host liver
    • Guha, C, Deb, N, Sappal, B, Ghosh, S, Roy-Chowdhury, N and Roy-Chowdhury, J (2001). Amplification of engrafted hepatocytes by preparative manipulation of the host liver. Artif Organs 25: 522-528.
    • (2001) Artif Organs , vol.25 , pp. 522-528
    • Guha, C.1    Deb, N.2    Sappal, B.3    Ghosh, S.4    Roy-Chowdhury, N.5    Roy-Chowdhury, J.6
  • 40
    • 0031870235 scopus 로고    scopus 로고
    • Long-term, near-total liver replacement by transplantation of isolated hepatocytes in rats treated with retrorsine
    • Laconi, E, Oren, R, Mukhopadhyay, DK, Hurston, E, Laconi, S, Pani, P et al. (1998). Long-term, near-total liver replacement by transplantation of isolated hepatocytes in rats treated with retrorsine. Am J Path 153: 319-329.
    • (1998) Am J Path , vol.153 , pp. 319-329
    • Laconi, E.1    Oren, R.2    Mukhopadhyay, D.K.3    Hurston, E.4    Laconi, S.5    Pani, P.6
  • 41
    • 0037096654 scopus 로고    scopus 로고
    • Liver repopulation after cell transplantation in mice treated with retrorsine and carbon tetrachloride
    • Guo, D, Fu, T, Nelson, JA, Superina, RA and Soriano, HE (2002). Liver repopulation after cell transplantation in mice treated with retrorsine and carbon tetrachloride. Transplantation 73: 1818-1824.
    • (2002) Transplantation , vol.73 , pp. 1818-1824
    • Guo, D.1    Fu, T.2    Nelson, J.A.3    Superina, R.A.4    Soriano, H.E.5
  • 43
    • 33645098770 scopus 로고    scopus 로고
    • Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer
    • Ding, Z, Georgiev, P and Thöny, B (2006). Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer. Gene Ther 13: 587-593.
    • (2006) Gene Ther , vol.13 , pp. 587-593
    • Ding, Z.1    Georgiev, P.2    Thöny, B.3
  • 44
    • 0025992469 scopus 로고
    • Promoter traps in embryonic stem cells: A genetic screen to identify and mutate developmental genes in mice
    • Friedrich, G and Soriano, P (1991). Promoter traps in embryonic stem cells: A genetic screen to identify and mutate developmental genes in mice. Genes Dev 5: 1513-1523.
    • (1991) Genes Dev , vol.5 , pp. 1513-1523
    • Friedrich, G.1    Soriano, P.2
  • 45
    • 67649835657 scopus 로고    scopus 로고
    • Gt(ROSA)26Sor, Gt(ROSA)26Sortm1Sho
    • JAX , Version 1, The Jackson Laboratory: Bar Harbor, ME
    • JAX (2008). Gt(ROSA)26Sor, Gt(ROSA)26Sortm1Sho, Version 1 Genotyping Protocol. The Jackson Laboratory: Bar Harbor, ME.
    • (2008) Genotyping Protocol
  • 47
    • 0026774112 scopus 로고
    • Mechanism of increased conversion of branched chain keto acid dehydrogenase from inactive to active form by a medium chain fatty acid (octanoate) in skeletal muscle
    • Paul, HS and Adibi, SA (1992). Mechanism of increased conversion of branched chain keto acid dehydrogenase from inactive to active form by a medium chain fatty acid (octanoate) in skeletal muscle. J Biol Chem 267: 11208-11214.
    • (1992) J Biol Chem , vol.267 , pp. 11208-11214
    • Paul, H.S.1    Adibi, S.A.2


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