메뉴 건너뛰기




Volumn 159, Issue 2, 2011, Pages

Early clinical markers of central nervous system involvement in mucopolysaccharidosis type II

Author keywords

[No Author keywords available]

Indexed keywords

ADOLESCENT; ADULT; ARTICLE; BLADDER AND BOWEL MANAGEMENT; BONE MARROW TRANSPLANTATION; CENTRAL NERVOUS SYSTEM; CHILD; CLINICAL ARTICLE; COGNITION; COGNITIVE DEFECT; DISEASE MARKER; DISEASE SEVERITY; ENZYME REPLACEMENT; HUMAN; HUNTER SYNDROME; INFANT; MASTICATION; MENTAL DETERIORATION; NEUROLOGIC EXAMINATION; PRESCHOOL CHILD; PRIORITY JOURNAL; RETROSPECTIVE STUDY; SCHOOL CHILD; SEIZURE; SLEEP DISORDER;

EID: 79960467875     PISSN: 00223476     EISSN: 10976833     Source Type: Journal    
DOI: 10.1016/j.jpeds.2011.03.019     Document Type: Article
Times cited : (43)

References (29)
  • 1
    • 0027944127 scopus 로고
    • Long-term follow-up of a patient transplanted for Hunter's disease type IIB: A case report and literature review
    • S.K. Bergstrom, J.J. Quinn, R. Greenstein, and J. Ascensao Long-term follow-up of a patient transplanted for Hunter's disease type IIB: a case report and literature review Bone Marrow Transplant 14 1994 653 658 (Pubitemid 24338498)
    • (1994) Bone Marrow Transplantation , vol.14 , Issue.4 , pp. 653-658
    • Bergstrom, S.K.1    Quinn, J.J.2    Greenstein, R.3    Ascensao, J.4
  • 3
    • 0018350224 scopus 로고
    • Enzyme replacement therapy by fibroblast transplantation. Long-term biochemical study in three cases of Hunter's syndrome
    • M.F. Dean, R.L. Stevens, H. Muir, P.F. Benson, L.R. Button, and R.L. Anderson Enzyme replacement therapy by fibroblast transplantation: long-term biochemical study in three cases of Hunter's syndrome J Clin Invest 63 1979 138 146 (Pubitemid 9085090)
    • (1979) Journal of Clinical Investigation , vol.63 , Issue.1 , pp. 138-146
    • Dean, M.F.1    Stevens, R.L.2    Muir, H.3
  • 4
    • 0015101540 scopus 로고
    • Effect of leukocyte transfusion in a child with type II mucopolysaccharidosis
    • A.G. Knudson Jr., N. Di Ferrante, and J.E. Curtis Effect of leukocyte transfusion in a child with type II mucopolysaccharidosis Proc Natl Acad Sci USA 68 1971 1738 1741
    • (1971) Proc Natl Acad Sci USA , vol.68 , pp. 1738-1741
    • Knudson, Jr.A.G.1    Di Ferrante, N.2    Curtis, J.E.3
  • 5
    • 0016744226 scopus 로고
    • A therapeutic trial of fresh plasma infusions over a period of 22 months in two siblings with Hunter's syndrome
    • S. Yatziv, M. Statter, P. Abeliuk, M. Meshulam, and A. Russel A therapeutic trial of fresh plasma infusions over a period of 22 months in two siblings with Hunter's syndrome Isr J Med Sci 11 1975 802 808
    • (1975) Isr J Med Sci , vol.11 , pp. 802-808
    • Yatziv, S.1    Statter, M.2    Abeliuk, P.3    Meshulam, M.4    Russel, A.5
  • 6
    • 33645110474 scopus 로고    scopus 로고
    • Correction of Hunter syndrome in the MPS II mouse model by AAV2/8-mediated gene delivery
    • M. Cardone, V.A. Polito, S. Pepe, L. Mann, A. D'Azzo, and A. Auricchio Correction of Hunter syndrome in the MPS II mouse model by AAV2/8-mediated gene delivery Hum Mol Genet 15 2006 1225 1236
    • (2006) Hum Mol Genet , vol.15 , pp. 1225-1236
    • Cardone, M.1    Polito, V.A.2    Pepe, S.3    Mann, L.4    D'Azzo, A.5    Auricchio, A.6
  • 8
    • 0032909039 scopus 로고    scopus 로고
    • Retroviral transduction and expansion of peripheral blood lymphocytes for the treatment of mucopolysaccharidosis type II, Hunter's syndrome
    • D.F. Stroncek, A. Hubel, R.A. Shankar, S.R. Burger, D. Pan, and J. McCullough Retroviral transduction and expansion of peripheral blood lymphocytes for the treatment of mucopolysaccharidosis type II, Hunter's syndrome Transfusion 39 1999 343 350 (Pubitemid 29181676)
    • (1999) Transfusion , vol.39 , Issue.4 , pp. 343-350
    • Stroncek, D.F.1    Hubel, A.2    Shankar, R.A.3    Burger, S.R.4    Pan, D.5    McCullough, J.6    Whitley, C.B.7
  • 10
    • 33846899175 scopus 로고    scopus 로고
    • A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome)
    • J. Muenzer, M. Gucsavas-Calikoglu, S. McCandless, T. Schuetz, and A. Kimura A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome) Mol Genet Metab 90 2007 325 355
    • (2007) Mol Genet Metab , vol.90 , pp. 325-355
    • Muenzer, J.1    Gucsavas-Calikoglu, M.2    McCandless, S.3    Schuetz, T.4    Kimura, A.5
  • 11
    • 33747209013 scopus 로고    scopus 로고
    • A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)
    • Corr 8:599
    • J. Muenzer, J.E. Wraith, M. Beck, R. Giugliani, P. Harmatz, and C.M. Eng A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome) Genet Med 8 2006 465 473 Corr 8:599
    • (2006) Genet Med , vol.8 , pp. 465-473
    • Muenzer, J.1    Wraith, J.E.2    Beck, M.3    Giugliani, R.4    Harmatz, P.5    Eng, C.M.6
  • 12
    • 77649219162 scopus 로고    scopus 로고
    • Novel treatments and future perspectives: Outcomes of intrathecal drug delivery
    • P. Dickson Novel treatments and future perspectives: outcomes of intrathecal drug delivery Int J Clin Pharmacol Ther 47 Suppl 1 2009 S124 S127
    • (2009) Int J Clin Pharmacol Ther , vol.47 , Issue.SUPPL. 1
    • Dickson, P.1
  • 15
    • 33646808142 scopus 로고    scopus 로고
    • Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS i mice
    • G. Watson, J. Bastacky, P. Belichenko, M. Buddhikot, S. Jungles, and M. Vellard Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice Gene Ther 13 2006 917 925
    • (2006) Gene Ther , vol.13 , pp. 917-925
    • Watson, G.1    Bastacky, J.2    Belichenko, P.3    Buddhikot, M.4    Jungles, S.5    Vellard, M.6
  • 16
    • 0036098944 scopus 로고    scopus 로고
    • Enhanced secretion and uptake of β-glucuronidase improves adeno-associated viral-mediated gene therapy of mucopolysaccharidosis type VII mice
    • DOI 10.1006/mthe.2002.0594
    • S. Elliger, C. Elliger, C. Lang, and G. Watson Enhanced secretion and uptake of beta-glucuronidase improves adeno-associated viral-mediated gene therapy of mucopolysaccharidosis type VII mice Mol Ther 5 2002 617 626 (Pubitemid 34546092)
    • (2002) Molecular Therapy , vol.5 , Issue.5 , pp. 617-626
    • Elliger, S.S.1    Elliger, C.A.2    Lang, C.3    Watson, G.L.4
  • 18
    • 50149108962 scopus 로고    scopus 로고
    • Neurological findings in Hunter disease: Pathology and possible therapeutic effects reviewed
    • S. Sawaf, E. Mayatepek, and B. Hoffman Neurological findings in Hunter disease: pathology and possible therapeutic effects reviewed J Inherit Metab Dis 31 2008 473 480
    • (2008) J Inherit Metab Dis , vol.31 , pp. 473-480
    • Sawaf, S.1    Mayatepek, E.2    Hoffman, B.3
  • 19
    • 21344455562 scopus 로고    scopus 로고
    • TNF-α levels and TNF-α gene polymorphism in type I Gaucher disease
    • DOI 10.1016/j.cyto.2005.03.006, PII S1043466605001432
    • G. Altarescu, A. Zimran, H. Michelakakis, and D. Elstein TNF-α levels and TNF-α gene polymorphism in type I Gaucher disease Cytokine 31 2005 149 152 (Pubitemid 40910231)
    • (2005) Cytokine , vol.31 , Issue.2 , pp. 149-152
    • Altarescu, G.1    Zimran, A.2    Michelakakis, H.3    Elstein, D.4
  • 22
    • 0000646543 scopus 로고
    • Mental retardation associated with acid mucopolysacchariduria (heparitin sulfate type)
    • S. Sanfilippo, R. Podosin, L. Langer, and R. Good Mental retardation associated with acid mucopolysacchariduria (heparitin sulfate type) J Pediatr 63 1963 837 838
    • (1963) J Pediatr , vol.63 , pp. 837-838
    • Sanfilippo, S.1    Podosin, R.2    Langer, L.3    Good, R.4
  • 24
    • 28444479825 scopus 로고    scopus 로고
    • Sleep disturbance in Sanfilippo syndrome: A parental questionnaire study
    • DOI 10.1136/adc.2004.065482
    • J. Fraser, A. Gason, J. Wraith, and M. Delatycki Sleep disturbances in Sanfilippo syndrome: a parental questionnaire Arch Dis Child 90 2005 1239 1242 (Pubitemid 41736308)
    • (2005) Archives of Disease in Childhood , vol.90 , Issue.12 , pp. 1239-1242
    • Fraser, J.1    Gason, A.A.2    Wraith, J.E.3    Delatycki, M.B.4
  • 27
    • 0020419764 scopus 로고
    • A clinical and genetic study of Hunter's syndrome. 2. Differences between the mild and severe forms
    • I.D. Young, P.S. Harper, R.G. Newcombe, and I.M. Archer A clinical and genetic study of Hunter's syndrome: two differences between the mild and severe forms J Med Genet 19 1982 408 411 (Pubitemid 13167537)
    • (1982) Journal of Medical Genetics , vol.19 , Issue.6 , pp. 408-411
    • Young, I.D.1    Harper, P.S.2    Newcombe, R.G.3    Archer, I.M.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.