메뉴 건너뛰기




Volumn 142, Issue 4, 1998, Pages 169-174

Allogeneic bone marrow transplantation in the treatment of (lysosomal) storage diseases;Allogene beenmergtransplantatie bij de behandeling van (lysosomale) stapelingsziekten

Author keywords

[No Author keywords available]

Indexed keywords

ADRENOLEUKODYSTROPHY; ALLOGENIC BONE MARROW TRANSPLANTATION; ARTICLE; DISEASE COURSE; HUMAN; HURLER SYNDROME; LYSOSOME STORAGE DISEASE; METACHROMATIC LEUKODYSTROPHY; MORBIDITY; MORTALITY; NEUROLOGIC DISEASE; SKELETON MALFORMATION; TREATMENT INDICATION;

EID: 0032561969     PISSN: 00282162     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article
Times cited : (4)

References (35)
  • 1
    • 0028805224 scopus 로고
    • Therapeutic approaches to lysosomal storage diseases
    • Kayne EM. Therapeutic approaches to lysosomal storage diseases. Current Opinion in Pediatrics 1995;7:650-4.
    • (1995) Current Opinion in Pediatrics , vol.7 , pp. 650-654
    • Kayne, E.M.1
  • 2
    • 0025869216 scopus 로고
    • Replacement therapy for inherited enzyme deficiency - Macrophage-targeted glucocerebrosidase for Gaucher's disease
    • Barton NW, Brady RO, Dambrosia JM. Di Besceglie AM, Doppelt SH, Hill SC, et al. Replacement therapy for inherited enzyme deficiency - macrophage-targeted glucocerebrosidase for Gaucher's disease. N Engl J Med 1991;324:1464-70.
    • (1991) N Engl J Med , vol.324 , pp. 1464-1470
    • Barton, N.W.1    Brady, R.O.2    Dambrosia, J.M.3    Di Besceglie, A.M.4    Doppelt, S.H.5    Hill, S.C.6
  • 3
    • 0019466750 scopus 로고
    • Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation
    • Hobbs JR, Hugh-Jones K, Barrelt AG, Byrom N, Chambers DCO. Henry K, et al. Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation. Lancet 1981;ii:709-12.
    • (1981) Lancet , vol.2 , pp. 709-712
    • Hobbs, J.R.1    Hugh-Jones, K.2    Barrelt, A.G.3    Byrom, N.4    Chambers, D.C.O.5    Henry, K.6
  • 4
    • 0029634283 scopus 로고
    • Allogeneic bone marrow transplantation for lysosomal storage diseases. European Group for Bone Marrow Transplantation
    • Hoogerbrugge PM, Brouwer OF, Bordigoni P, Ringdén O, Kapaun P, Ortega JJ, et al. Allogeneic bone marrow transplantation for lysosomal storage diseases. European Group for Bone Marrow Transplantation. Lancet 1995;345:1398-402.
    • (1995) Lancet , vol.345 , pp. 1398-1402
    • Hoogerbrugge, P.M.1    Brouwer, O.F.2    Bordigoni, P.3    Ringdén, O.4    Kapaun, P.5    Ortega, J.J.6
  • 5
    • 0029114944 scopus 로고
    • The future for treatment by bone marrow transplantation for adrenoleukodystrophy. metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome
    • Krivit W, Lockman LA, Watkins PA, Hirsch J, Shapiro EG. The future for treatment by bone marrow transplantation for adrenoleukodystrophy. metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome. J Inherit Metab Dis 1995;18:398-412.
    • (1995) J Inherit Metab Dis , vol.18 , pp. 398-412
    • Krivit, W.1    Lockman, L.A.2    Watkins, P.A.3    Hirsch, J.4    Shapiro, E.G.5
  • 6
    • 0027179363 scopus 로고
    • Busulfan disposition below the age of three: Alteration in children with lysosomal storage disease
    • Vassal G, Fischer A, Challine D, Boland I. Ledheist F, Lemerle S, et al. Busulfan disposition below the age of three: alteration in children with lysosomal storage disease. Blood 1993;82:1030-4.
    • (1993) Blood , vol.82 , pp. 1030-1034
    • Vassal, G.1    Fischer, A.2    Challine, D.3    Boland, I.4    Ledheist, F.5    Lemerle, S.6
  • 8
    • 0031018301 scopus 로고    scopus 로고
    • Bone marrow transplantation for mucopolysaccharidosis type I: Experience of two British centres
    • Vellodi A, Young EP, Cooper A, Wraith JE, Winchester B. Meaney C, et al. Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres. Arch Dis Child 1997;76: 92-9.
    • (1997) Arch Dis Child , vol.76 , pp. 92-99
    • Vellodi, A.1    Young, E.P.2    Cooper, A.3    Wraith, J.E.4    Winchester, B.5    Meaney, C.6
  • 9
    • 0025022733 scopus 로고
    • Treatment of late infantile metachromatic leukodystrophy by bone marrow transplantation
    • Krivit W, Shapiro E, Kennedy W, Lipton M, Lockman L, Smith S, et al. Treatment of late infantile metachromatic leukodystrophy by bone marrow transplantation. N Engl J Med 1990;322:28-32.
    • (1990) N Engl J Med , vol.322 , pp. 28-32
    • Krivit, W.1    Shapiro, E.2    Kennedy, W.3    Lipton, M.4    Lockman, L.5    Smith, S.6
  • 10
    • 0029146242 scopus 로고
    • Krivit W Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation
    • Shapiro EG, Lockman LA, Balthazor M. Krivit W Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation. J Inherit Metab Dis 1995;18:413-29.
    • (1995) J Inherit Metab Dis , vol.18 , pp. 413-429
    • Shapiro, E.G.1    Lockman, L.A.2    Balthazor, M.3
  • 11
    • 0029023990 scopus 로고
    • Clinical and therapeutic aspects of adrenoleukodystrophy and adrenomyeloneuropathy
    • Moser HW. Clinical and therapeutic aspects of adrenoleukodystrophy and adrenomyeloneuropathy. J Neuropathol Exp Neurol 1995; 54:740-5.
    • (1995) J Neuropathol Exp Neurol , vol.54 , pp. 740-745
    • Moser, H.W.1
  • 12
    • 0025329012 scopus 로고
    • Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation
    • Aubourg P, Blanche S, Jambaqué I, Rocchiccioli F, Kalifa G, Naud-Saudreau C, et al. Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation N Engi J Med 1990;322:1860-6.
    • (1990) N Engi J Med , vol.322 , pp. 1860-1866
    • Aubourg, P.1    Blanche, S.2    Jambaqué, I.3    Rocchiccioli, F.4    Kalifa, G.5    Naud-Saudreau, C.6
  • 13
    • 0012566412 scopus 로고
    • Bone marrow transplantation in children
    • Vossen JMJJ. Bone marrow transplantation in children. Ballieres Clin Paediatr 1994;1:1029-59.
    • (1994) Ballieres Clin Paediatr , vol.1 , pp. 1029-1059
    • Vossen, J.M.J.J.1
  • 14
    • 0026755999 scopus 로고
    • Bone marrow derived elements in the central nervous system: An immunohistochemical and ultrastructural survey of rat chimeras
    • Hickey WF, Vass K, Lassman NH. Bone marrow derived elements in the central nervous system: an immunohistochemical and ultrastructural survey of rat chimeras. J Neuropathol Exp Neurol 1992; 51:246-56.
    • (1992) J Neuropathol Exp Neurol , vol.51 , pp. 246-256
    • Hickey, W.F.1    Vass, K.2    Lassman, N.H.3
  • 15
    • 0027431164 scopus 로고
    • Bone marrow-derived elements in the peripheral nervous system. An immunohistochemical and ultrastructural investigation in chemeric rats
    • Vass K, Hickey WF, Schmidt RE, Lassmann H. Bone marrow-derived elements in the peripheral nervous system. An immunohistochemical and ultrastructural investigation in chemeric rats. Lab Invest 1993;69:275-82.
    • (1993) Lab Invest , vol.69 , pp. 275-282
    • Vass, K.1    Hickey, W.F.2    Schmidt, R.E.3    Lassmann, H.4
  • 16
    • 0027371977 scopus 로고
    • Cel en ziekte V. Pathologie van het lysosoom
    • Reuser AJJ. Cel en ziekte V. Pathologie van het lysosoom Ned Tijdschr Geneeskd 1993;137:2028-34.
    • (1993) Ned Tijdschr Geneeskd , vol.137 , pp. 2028-2034
    • Reuser, A.J.J.1
  • 18
    • 0028260714 scopus 로고
    • Bone marrow transplantation has a significant effect on enzyme levels and storage of glycosaminoglycans in tissues and in isolated hepatocytes of mucopolysaccharidosis type VII mice
    • Poorthuis BJHM, Romme AE, Willemsen R, Wagemaker G. Bone marrow transplantation has a significant effect on enzyme levels and storage of glycosaminoglycans in tissues and in isolated hepatocytes of mucopolysaccharidosis type VII mice. Pediatr Res 1994;36:187-93.
    • (1994) Pediatr Res , vol.36 , pp. 187-193
    • Poorthuis, B.J.H.M.1    Romme, A.E.2    Willemsen, R.3    Wagemaker, G.4
  • 19
    • 0028324026 scopus 로고
    • Bone marrow transplantation corrects the enzyme defect in neurons of the central nervous system in a lysosomal storage disease
    • Walkley SU, Thrall MA, Dobrenis K, Huang M, March PA, Siegel DA, et al. Bone marrow transplantation corrects the enzyme defect in neurons of the central nervous system in a lysosomal storage disease. Proc Natl Acad Sci USA 1994;91:2970-4.
    • (1994) Proc Natl Acad Sci USA , vol.91 , pp. 2970-2974
    • Walkley, S.U.1    Thrall, M.A.2    Dobrenis, K.3    Huang, M.4    March, P.A.5    Siegel, D.A.6
  • 21
    • 0017296773 scopus 로고
    • Solitary cells and enzyme exchange in tetraparental mice
    • Feder N. Solitary cells and enzyme exchange in tetraparental mice. Nature 1976;263:67-9.
    • (1976) Nature , vol.263 , pp. 67-69
    • Feder, N.1
  • 22
    • 0019197887 scopus 로고
    • The uptake of enzymes into lysosomes: An overview
    • Neufeld EF. The uptake of enzymes into lysosomes: an overview. Birth Defects 1980;16.77-84.
    • (1980) Birth Defects , vol.16 , pp. 77-84
    • Neufeld, E.F.1
  • 23
    • 0021719194 scopus 로고
    • Marrow transplantation in genetic disease
    • Barranger JA Marrow transplantation in genetic disease N Engl J Med 1984;311:1629-31.
    • (1984) N Engl J Med , vol.311 , pp. 1629-1631
    • Barranger, J.A.1
  • 24
    • 0023948528 scopus 로고
    • Donor-derived cells in the central nervous system of twitcher mice after bone marrow transplantation
    • Hoogerbrugge PM, Suzuki K, Suzuki K. Poorthuis BJHM, Kobayashi T, Wagemaker G, et al. Donor-derived cells in the central nervous system of twitcher mice after bone marrow transplantation. Science 1988;239:1035-8.
    • (1988) Science , vol.239 , pp. 1035-1038
    • Hoogerbrugge, P.M.1    Suzuki, K.2    Suzuki, K.3    Poorthuis, B.J.H.M.4    Kobayashi, T.5    Wagemaker, G.6
  • 25
    • 0029143678 scopus 로고
    • Microglia: The effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases
    • Krivit W, Sung JH, Shapiro EG, Lockman LA. Microglia: the effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases. Cell Transplant 1995;4:385-92.
    • (1995) Cell Transplant , vol.4 , pp. 385-392
    • Krivit, W.1    Sung, J.H.2    Shapiro, E.G.3    Lockman, L.A.4
  • 26
    • 0029634358 scopus 로고
    • Bone marrow transplantation for lysosomal diseases
    • Walkley SU, Dobrenis K. Bone marrow transplantation for lysosomal diseases. Lancet 1995;345:1382-3.
    • (1995) Lancet , vol.345 , pp. 1382-1383
    • Walkley, S.U.1    Dobrenis, K.2
  • 29
    • 0027944127 scopus 로고
    • Long-term follow-up of a patient transplanted for Hunter's disease type IIB: A case report and literature review
    • Bergstrom SK, Quinn JJ, Greenstein R, Ascensao J. Long-term follow-up of a patient transplanted for Hunter's disease type IIB: a case report and literature review. Bone Marrow Transplant 1994;14: 653-8.
    • (1994) Bone Marrow Transplant , vol.14 , pp. 653-658
    • Bergstrom, S.K.1    Quinn, J.J.2    Greenstein, R.3    Ascensao, J.4
  • 30
    • 0029013710 scopus 로고
    • Poor cognitive outcome of eleven children with Sanfilippo syndrome after bone marrow transplantation and successful engraftment
    • Klein KA, Krivit W, Whitley CB, Peters C, Cool VA, Fuhrman, et al. Poor cognitive outcome of eleven children with Sanfilippo syndrome after bone marrow transplantation and successful engraftment. Bone Marrow Transplant 1994;15:176-81.
    • (1994) Bone Marrow Transplant , vol.15 , pp. 176-181
    • Klein, K.A.1    Krivit, W.2    Whitley, C.B.3    Peters, C.4    Cool, V.A.5    Fuhrman6
  • 32
    • 0002776718 scopus 로고
    • Bone marrow transplantation for storage diseases
    • Desnick RJ, editor. New York: Churchill Livingstone
    • Krivit W, Shapiro E. Bone marrow transplantation for storage diseases. In: Desnick RJ, editor. Treatment of genetic diseases. New York: Churchill Livingstone, 1991:203-21.
    • (1991) Treatment of Genetic Diseases , pp. 203-221
    • Krivit, W.1    Shapiro, E.2
  • 33
    • 0026540519 scopus 로고
    • Maroteaux-Lamy syndrome: Treatment by allogenic bone marrow transplantation in 6 patients and potential for autotransplantation bone marrow gene insertion
    • Krivit W. Maroteaux-Lamy syndrome: treatment by allogenic bone marrow transplantation in 6 patients and potential for autotransplantation bone marrow gene insertion. Int Pediatr 1992;7:47-52
    • (1992) Int Pediatr , vol.7 , pp. 47-52
    • Krivit, W.1
  • 35
    • 0023221342 scopus 로고
    • Beneficial effect of pre-transplant splenectomy on displacement bone marrow transplantation for Gaucher's syndrome
    • Hobbs JR, Jones KH, Shaw PJ, Lindsay I, Hancock M. Beneficial effect of pre-transplant splenectomy on displacement bone marrow transplantation for Gaucher's syndrome. Lancet 1987;i:1111-5
    • (1987) Lancet , vol.1 , pp. 1111-1115
    • Hobbs, J.R.1    Jones, K.H.2    Shaw, P.J.3    Lindsay, I.4    Hancock, M.5


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.