메뉴 건너뛰기




Volumn 64, Issue 7, 2005, Pages 565-575

Hematopoietic cell transplantation ameliorates clinical phenotype and progression of the CNS pathology in the mouse model of late onset Krabbe disease

Author keywords

Demyelination; Globoid cell leukodystrophy; Green fluorescence protein; Hypertrophic neuropathy; Remyelination; Twitcher mouse

Indexed keywords

GALACTOSYLCERAMIDASE; GREEN FLUORESCENT PROTEIN; SAPOSIN A; SPHINGOLIPID ACTIVATOR PROTEIN; UNCLASSIFIED DRUG;

EID: 22244445463     PISSN: 00223069     EISSN: None     Source Type: Journal    
DOI: 10.1097/01.jnen.0000171646.01966.0c     Document Type: Article
Times cited : (21)

References (35)
  • 1
    • 0002054185 scopus 로고    scopus 로고
    • Galactosylceramide lipidosis: Globoid cell leukodystrophy (Krabbe disease)
    • Scriver CP, Beaudett AL, Sly WS, Valle D, eds. New York: McGraw-Hill
    • Wenger DA, Suzuki K, Suzuki Y, et al. Galactosylceramide lipidosis: Globoid cell leukodystrophy (Krabbe disease). In: Scriver CP, Beaudett AL, Sly WS, Valle D, eds. The Metabolic and Molecular Basis of Inherited Disease. Eighth Edition. New York: McGraw-Hill, 2001:3669-93
    • (2001) The Metabolic and Molecular Basis of Inherited Disease. Eighth Edition , pp. 3669-3693
    • Wenger, D.A.1    Suzuki, K.2    Suzuki, Y.3
  • 2
    • 0026410675 scopus 로고
    • Late-onset Krabbe disease (globoid cell leukodystrophy): Clinical and biochemical features of 15 cases
    • Kolodny EH, Raghavan S, Krivit W. Late-onset Krabbe disease (globoid cell leukodystrophy): Clinical and biochemical features of 15 cases. Dev Neurosci 1991;13:232-39
    • (1991) Dev Neurosci , vol.13 , pp. 232-239
    • Kolodny, E.H.1    Raghavan, S.2    Krivit, W.3
  • 3
    • 0026409894 scopus 로고
    • Symptomatology of late onset Krabbe's leukodystrophy: European experience
    • Lyon G, Hagberg B, Evrard E et al. Symptomatology of late onset Krabbe's leukodystrophy: European experience. Dev Neurosci 1991;13: 240-44
    • (1991) Dev Neurosci , vol.13 , pp. 240-244
    • Lyon, G.1    Hagberg, B.2    Evrard, E.3
  • 4
    • 0029804361 scopus 로고    scopus 로고
    • Peripheral neuropathy in late-onset Krabbe's disease: Histochemical and ultrastructural findings
    • Matsumoto R, Oka N, Nagahama Y, et al. Peripheral neuropathy in late-onset Krabbe's disease: Histochemical and ultrastructural findings. Acta Neuropathol 1996;92:635-39
    • (1996) Acta Neuropathol , vol.92 , pp. 635-639
    • Matsumoto, R.1    Oka, N.2    Nagahama, Y.3
  • 5
    • 0036226311 scopus 로고    scopus 로고
    • Peripheral neuropathy with hypomyelinating features in adult-onset Krabbe's disease
    • Sabatelli M, Quaranta L, Madia E et al. Peripheral neuropathy with hypomyelinating features in adult-onset Krabbe's disease. Neuromuscul Disord 2002;12:386-91
    • (2002) Neuromuscul Disord , vol.12 , pp. 386-391
    • Sabatelli, M.1    Quaranta, L.2    Madia, E.3
  • 7
    • 0032537062 scopus 로고    scopus 로고
    • Hematopoietic stem cell transplantation in globoid cell leukodystrophy
    • Krivit W, Shapiro EG, Peters C, et al. Hematopoietic stem cell transplantation in globoid cell leukodystrophy. N Engl J Med 1998;338:1119-26
    • (1998) N Engl J Med , vol.338 , pp. 1119-1126
    • Krivit, W.1    Shapiro, E.G.2    Peters, C.3
  • 8
    • 0032941197 scopus 로고    scopus 로고
    • Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler Maroteaux-Lamy, and Sly syndromes and Gaucher disease type III
    • Krivit W, Peters C, Shapiro EG. Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler Maroteaux-Lamy, and Sly syndromes and Gaucher disease type III. Curr Opin Neurol 1999;12:167-76
    • (1999) Curr Opin Neurol , vol.12 , pp. 167-176
    • Krivit, W.1    Peters, C.2    Shapiro, E.G.3
  • 9
    • 22244476458 scopus 로고    scopus 로고
    • Models of Krabbe disease
    • Lazzarini RA, Griffin JW, Lassman H, Nave K-A, Miller RH, Trapp BD, eds. Amsterdam: Elsevier
    • Suzuki K. Models of Krabbe disease. In: Lazzarini RA, Griffin JW, Lassman H, Nave K-A, Miller RH, Trapp BD, eds. Myelin Biology and Disease. Amsterdam: Elsevier, 2004, Vol. 2, 1101-13
    • (2004) Myelin Biology and Disease , vol.2 , pp. 1101-1113
    • Suzuki, K.1
  • 10
    • 0021249707 scopus 로고
    • Prolonged survival and remyelination after hematopoietic cell transplantation in the twitcher mouse
    • Yeager AM, Brennan S, Tiffany C, et al. Prolonged survival and remyelination after hematopoietic cell transplantation in the twitcher mouse. Science 1984;225:1052-54
    • (1984) Science , vol.225 , pp. 1052-1054
    • Yeager, A.M.1    Brennan, S.2    Tiffany, C.3
  • 11
    • 0023212302 scopus 로고
    • Hematopoietic cell transplantation in murine globoid cell leukodystrophy (the twitcher mouse): Effects on levels of galactosylceramidase, psychosine, and galactocerebrosides
    • Ichioka T, Kishimoto Y, Bennan S, et al. Hematopoietic cell transplantation in murine globoid cell leukodystrophy (the twitcher mouse): Effects on levels of galactosylceramidase, psychosine, and galactocerebrosides. Proc Natl Acad Sci USA 1987;84:4259-63
    • (1987) Proc Natl Acad Sci USA , vol.84 , pp. 4259-4263
    • Ichioka, T.1    Kishimoto, Y.2    Bennan, S.3
  • 12
    • 0023948528 scopus 로고
    • Donor-derived cells in the central nervous system of twitcher mice after bone marrow transplantation
    • Hoogerbrugge PM, Suzuki K, Suzuki K, et al. Donor-derived cells in the central nervous system of twitcher mice after bone marrow transplantation. Science 1988;239:1035-38
    • (1988) Science , vol.239 , pp. 1035-1038
    • Hoogerbrugge, P.M.1    Suzuki, K.2    Suzuki, K.3
  • 13
    • 0034799137 scopus 로고    scopus 로고
    • Intraventricular administration of recombinant adenovirus to neonatal twitcher mouse leads to clinicopathological improvements
    • Shen JS, Watabe K, Ohashi T, et al. Intraventricular administration of recombinant adenovirus to neonatal twitcher mouse leads to clinicopathological improvements. Gene Ther 2001;8:1081-87
    • (2001) Gene Ther , vol.8 , pp. 1081-1087
    • Shen, J.S.1    Watabe, K.2    Ohashi, T.3
  • 14
    • 0034656333 scopus 로고    scopus 로고
    • L-cycloserine slows the clinical and pathological course in mice with globoid cell leukodystrophy (Twitcher mice)
    • LeVine SM, Pedchenko TV, Bronshteyn IG, et al. L-cycloserine slows the clinical and pathological course in mice with globoid cell leukodystrophy (Twitcher mice). J Neurosci Res 2000;60:231-36
    • (2000) J Neurosci Res , vol.60 , pp. 231-236
    • Levine, S.M.1    Pedchenko, T.V.2    Bronshteyn, I.G.3
  • 15
    • 0031400601 scopus 로고    scopus 로고
    • Transgenic introduction of human galactosylceramidase into twitcher mouse: Significant phenotype improvement with a minimal expression
    • Matsumoto A, Vanier M, Oya Y, et al. Transgenic introduction of human galactosylceramidase into twitcher mouse: Significant phenotype improvement with a minimal expression. Develop Brain Dysfunction 1997; 10:142-54
    • (1997) Develop Brain Dysfunction , vol.10 , pp. 142-154
    • Matsumoto, A.1    Vanier, M.2    Oya, Y.3
  • 16
    • 4243064037 scopus 로고    scopus 로고
    • Transgenic rescue of Krabbe disease in the twitcher mouse
    • De Gasperi R, Friedrich VL, Perez GM, et al. Transgenic rescue of Krabbe disease in the twitcher mouse. Gene Ther 2004;11:1188-94
    • (2004) Gene Ther , vol.11 , pp. 1188-1194
    • De Gasperi, R.1    Friedrich, V.L.2    Perez, G.M.3
  • 17
    • 0035873272 scopus 로고    scopus 로고
    • A mutation in the saposin a domain of the sphingolipid activator protein (prosaposin) gene results in a late onset, chronic form of globoid cell leukodystrophy in the mouse
    • Matsuda J, Vanier MT, Saito Y, et al. A mutation in the saposin A domain of the sphingolipid activator protein (prosaposin) gene results in a late onset, chronic form of globoid cell leukodystrophy in the mouse. Hum Mol Genet 2001;10:1191-99
    • (2001) Hum Mol Genet , vol.10 , pp. 1191-1199
    • Matsuda, J.1    Vanier, M.T.2    Saito, Y.3
  • 19
    • 0028169142 scopus 로고
    • Spacio-temporal progression of demyelination in twitcher mouse: With clinico-pathological correlation
    • Taniike M, Suzuki K. Spacio-temporal progression of demyelination in twitcher mouse: With clinico-pathological correlation. Acta Neuropathol 1994;88:228-36
    • (1994) Acta Neuropathol , vol.88 , pp. 228-236
    • Taniike, M.1    Suzuki, K.2
  • 20
    • 0030904130 scopus 로고    scopus 로고
    • 'Greenmice' as a source of ubiquitous green cells
    • Okabe M, Ikawa M, Kominami K, et al. 'Greenmice' as a source of ubiquitous green cells. FEBS Lett 1997;407:313-19
    • (1997) FEBS Lett , vol.407 , pp. 313-319
    • Okabe, M.1    Ikawa, M.2    Kominami, K.3
  • 21
    • 0034759421 scopus 로고    scopus 로고
    • Expression of immune-related molecules was down regulated n twitcher mice following bone marrow transplantation
    • 2l. Wu Y-P, McMahon E J, Matsuda J, et al. Expression of immune-related molecules was down regulated n twitcher mice following bone marrow transplantation. J Neuropathol Exp Neurol 2001;60:1062-74
    • (2001) J Neuropathol Exp Neurol , vol.60 , pp. 1062-1074
    • Wu, Y.-P.1    McMahon, E.J.2    Matsuda, J.3
  • 22
    • 0033882996 scopus 로고    scopus 로고
    • + hematogenous cells in twitcher mice following bone marrow transplantation
    • + hematogenous cells in twitcher mice following bone marrow transplantation. Am J Pathol 2000;156:1849-54
    • (2000) Am J Pathol , vol.156 , pp. 1849-1854
    • Wu, Y.-P.1    McMahon, E.J.2    Kraine, M.R.3
  • 24
    • 0036709433 scopus 로고    scopus 로고
    • Peripheral macrophage recruitment in cuprizone-induced CNS demyelination despite an intact blood brain barrier
    • McMahorn EJ, Suzuki K, Matsushima GK. Peripheral macrophage recruitment in cuprizone-induced CNS demyelination despite an intact blood brain barrier. J Neuroimmunol 2002;130:32-45
    • (2002) J Neuroimmunol , vol.130 , pp. 32-45
    • McMahorn, E.J.1    Suzuki, K.2    Matsushima, G.K.3
  • 25
    • 3442886765 scopus 로고    scopus 로고
    • Genetic background markedly influences vulnerability of the hippocampal neuronal organization in the twitcher mouse model of globoid cell leukodystrophy
    • Tominaga K, Matsuda J, Kido M, et al. Genetic background markedly influences vulnerability of the hippocampal neuronal organization in the twitcher mouse model of globoid cell leukodystrophy. J Neurosci Res 2004;77:507-16
    • (2004) J Neurosci Res , vol.77 , pp. 507-516
    • Tominaga, K.1    Matsuda, J.2    Kido, M.3
  • 26
    • 0033065014 scopus 로고    scopus 로고
    • An apoptotic depletion of oligodendrocytes in the twitcher, a nmrine model of globoid cell leukodystrophy
    • Taniike M, Mohri I, Eguchi N, et al. An apoptotic depletion of oligodendrocytes in the twitcher, a nmrine model of globoid cell leukodystrophy. J Neuropathol Exp Neurol 1999;58:644-53
    • (1999) J Neuropathol Exp Neurol , vol.58 , pp. 644-653
    • Taniike, M.1    Mohri, I.2    Eguchi, N.3
  • 27
    • 0030614992 scopus 로고    scopus 로고
    • IL-6 and TNF alpha expression in brains of twitcher, quaking and normal mice
    • LeVine SM, Brown DC. IL-6 and TNF alpha expression in brains of twitcher, quaking and normal mice. J Neuroimmunol 1997;73:47-56
    • (1997) J Neuroimmunol , vol.73 , pp. 47-56
    • LeVine, S.M.1    Brown, D.C.2
  • 28
    • 0033936785 scopus 로고    scopus 로고
    • Infiltration of hematogenous lineage cells into the demyelinating central nervous system of twitcher mice
    • Wu Y-P, Matsuda J, Kubota A, et al. Infiltration of hematogenous lineage cells into the demyelinating central nervous system of twitcher mice. J Neuropathol Exp Neurol 2000;59:628-39
    • (2000) J Neuropathol Exp Neurol , vol.59 , pp. 628-639
    • Wu, Y.-P.1    Matsuda, J.2    Kubota, A.3
  • 29
    • 85039397609 scopus 로고    scopus 로고
    • Enzyme replacement therapy results in substantial improvements in early but not late clinical phenotype in a mouse model of globoid cell leukodystrophy
    • San Diego, October 25
    • Lee WC, Courtenay AI, Troendle F J, et al. Enzyme replacement therapy results in substantial improvements in early but not late clinical phenotype in a mouse model of globoid cell leukodystrophy. Abstract (#582.17) for the 34th Annual Meeting of the Society for Neuroscience, San Diego, October 25, 2004
    • (2004) Abstract (#582.17) for the 34th Annual Meeting of the Society for Neuroscience
    • Lee, W.C.1    Courtenay, A.I.2    Troendle, F.J.3
  • 30
    • 0002852974 scopus 로고
    • Interactions between axons and Schwann cells
    • Dyck PJ, Thomas PK, Griffin JW, Low PA, Poduslo JF, eds. Philadelphia: W.B. Saunders Co.
    • Griffin JW, Kidd G, Trapp BD. Interactions between axons and Schwann cells. In: Dyck PJ, Thomas PK, Griffin JW, Low PA, Poduslo JF, eds. Peripheral Neuropathy. Third Edition, Philadelphia: W.B. Saunders Co.; 1993, 317-30
    • (1993) Peripheral Neuropathy. Third Edition , pp. 317-330
    • Griffin, J.W.1    Kidd, G.2    Trapp, B.D.3
  • 31
    • 0030250873 scopus 로고    scopus 로고
    • Onion bulb cells in mice deficient for myelin genes share molecular properties with immature, differentiated non-myelinating, and denervated Schwann cells
    • Guenard V, Montag D, Schachner D, et al. Onion bulb cells in mice deficient for myelin genes share molecular properties with immature, differentiated non-myelinating, and denervated Schwann cells. Glia 1996;18:27-38
    • (1996) Glia , vol.18 , pp. 27-38
    • Guenard, V.1    Montag, D.2    Schachner, D.3
  • 32
    • 0042237909 scopus 로고    scopus 로고
    • Hypertrophic neuropathies and malignant peripheral nerve sheath tumors in transgenic mice overexpressing glial growth factor β3 in myelinating Schwann cells
    • Huijbregts R PH, Roth KA, Schmidt RE, et al. Hypertrophic neuropathies and malignant peripheral nerve sheath tumors in transgenic mice overexpressing glial growth factor β3 in myelinating Schwann cells. J Neurosci 2003;23:7269-80
    • (2003) J Neurosci , vol.23 , pp. 7269-7280
    • Huijbregts, R.P.H.1    Roth, K.A.2    Schmidt, R.E.3
  • 33
    • 2542495972 scopus 로고    scopus 로고
    • Pathology of a mouse mutation in peripheral myelin protein P0 is characteristic of severe and early onset form of human Charcot-Marie-Tooth type 1B disorder
    • Rünker AE, Kobsar I, Fink T, et al. Pathology of a mouse mutation in peripheral myelin protein P0 is characteristic of severe and early onset form of human Charcot-Marie-Tooth type 1B disorder. J Cell Biol 2004;165: 565-73
    • (2004) J Cell Biol , vol.165 , pp. 565-573
    • Rünker, A.E.1    Kobsar, I.2    Fink, T.3
  • 34
    • 0028006109 scopus 로고
    • Progressive dysfunction of twitcher Schwann cells is evaluated better in vitro than in vivo
    • Komiyama A, Suzuki K. Progressive dysfunction of twitcher Schwann cells is evaluated better in vitro than in vivo. Brain Res 1994;637:106-13
    • (1994) Brain Res , vol.637 , pp. 106-113
    • Komiyama, A.1    Suzuki, K.2
  • 35
    • 20844453744 scopus 로고    scopus 로고
    • Outcomes of babies with infantile Krabbe Disease after umbilical cord blood transplantation
    • Escolar ML, Poe MD, Provenzale JM, et al. Outcomes of babies with infantile Krabbe Disease after umbilical cord blood transplantation. N Engl J Med 2005;352:2069-81
    • (2005) N Engl J Med , vol.352 , pp. 2069-2081
    • Escolar, M.L.1    Poe, M.D.2    Provenzale, J.M.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.