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Volumn 109, Issue 1, 2013, Pages 33-40

Enzyme replacement therapy started at birth improves outcome in difficult-to-treat organs in mucopolysaccharidosis I mice

Author keywords

Blood brain barrier; Enzyme replacement therapy; Hurler Syndrome; Mucopolysaccharidosis I

Indexed keywords

IMMUNOGLOBULIN G ANTIBODY; LARONIDASE; LEVO IDURONIDASE;

EID: 84876092659     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2013.03.005     Document Type: Article
Times cited : (58)

References (31)
  • 1
    • 0000869162 scopus 로고    scopus 로고
    • The mucopolysaccharidoses
    • McGraw-Hill, New York, C.R. Scriver, A.L. Beaudet, W.S. Sly, D. Valle (Eds.)
    • Neufeld E.F., Muenzer J. The mucopolysaccharidoses. The Metabolic and Molecular Bases of Inherited Disease 2001, 3421-3452 2. McGraw-Hill, New York. C.R. Scriver, A.L. Beaudet, W.S. Sly, D. Valle (Eds.).
    • (2001) The Metabolic and Molecular Bases of Inherited Disease
    • Neufeld, E.F.1    Muenzer, J.2
  • 3
    • 82955203807 scopus 로고    scopus 로고
    • Cell microencapsulation: a potential tool for the treatment of neuronopathic lysosomal storage diseases
    • Matte U., Lagranha V.L., de Carvalho T.G., Mayer F.Q., Giugliani R. Cell microencapsulation: a potential tool for the treatment of neuronopathic lysosomal storage diseases. J. Inherit. Metab. Dis. 2011, 34:983-990.
    • (2011) J. Inherit. Metab. Dis. , vol.34 , pp. 983-990
    • Matte, U.1    Lagranha, V.L.2    de Carvalho, T.G.3    Mayer, F.Q.4    Giugliani, R.5
  • 5
    • 34447121276 scopus 로고    scopus 로고
    • Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-l-iduronidase (laronidase)
    • Wraith J.E., Beck M., Lane R., Van der Ploeg A., Shapiro E., Xue Y., Kakkis E.D., Guffon N. Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-l-iduronidase (laronidase). Pediatrics 2007, 120:e37-e46.
    • (2007) Pediatrics , vol.120
    • Wraith, J.E.1    Beck, M.2    Lane, R.3    Van der Ploeg, A.4    Shapiro, E.5    Xue, Y.6    Kakkis, E.D.7    Guffon, N.8
  • 6
    • 74049159044 scopus 로고    scopus 로고
    • Enzyme replacement therapy in a 5-month-old boy with attenuated presymptomatic MPS I: 5-year follow-up
    • Gabrielli O., Clarke L.A., Bruni S., Coppa G.V. Enzyme replacement therapy in a 5-month-old boy with attenuated presymptomatic MPS I: 5-year follow-up. Pediatrics 2009, 125:e183-e187.
    • (2009) Pediatrics , vol.125
    • Gabrielli, O.1    Clarke, L.A.2    Bruni, S.3    Coppa, G.V.4
  • 7
    • 0033010571 scopus 로고    scopus 로고
    • Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy
    • Vogler C., Levy B., Galvin N.J., Thorpe C., Sands M.S., Barker J.E., Baty J., Birkenmeier E.H., Sly W.S. Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy. Pediatr. Res. 1999, 45:838-844.
    • (1999) Pediatr. Res. , vol.45 , pp. 838-844
    • Vogler, C.1    Levy, B.2    Galvin, N.J.3    Thorpe, C.4    Sands, M.S.5    Barker, J.E.6    Baty, J.7    Birkenmeier, E.H.8    Sly, W.S.9
  • 11
    • 84859004695 scopus 로고    scopus 로고
    • Recombinant encapsulated cells overexpressing alpha-l-iduronidase correct enzyme deficiency in human mucopolysaccharidosis type I cells
    • Baldo G., Quoos Mayer F., Burin M., Carrillo-Farga J., Matte U., Giugliani R. Recombinant encapsulated cells overexpressing alpha-l-iduronidase correct enzyme deficiency in human mucopolysaccharidosis type I cells. Cells Tissues Organs. 2012, 195:323-329.
    • (2012) Cells Tissues Organs. , vol.195 , pp. 323-329
    • Baldo, G.1    Quoos Mayer, F.2    Burin, M.3    Carrillo-Farga, J.4    Matte, U.5    Giugliani, R.6
  • 14
    • 78649449823 scopus 로고    scopus 로고
    • Bone marrow derived cells decrease inflammation but not oxidative stress in an experimental model of acute myocardial infarction
    • Tavares A.M., da Rosa Araújo A.S., Baldo G., Matte U., Khaper N., Belló-Klein A., Rohde L.E., Clausell N. Bone marrow derived cells decrease inflammation but not oxidative stress in an experimental model of acute myocardial infarction. Life Sci. 2010, 87:699-706.
    • (2010) Life Sci. , vol.87 , pp. 699-706
    • Tavares, A.M.1    da Rosa Araújo, A.S.2    Baldo, G.3    Matte, U.4    Khaper, N.5    Belló-Klein, A.6    Rohde, L.E.7    Clausell, N.8
  • 16
    • 84876080111 scopus 로고    scopus 로고
    • Retroviral Vector-mediated Gene Therapy to Mucopolysaccharidosis I Mice Improves Sensorimotor Impairments and Other Behavior Deficits
    • in press.
    • G. Baldo, D. Wozniak, K. Ohlemiller, Y. Zhang, R. Giugliani R, K.P. Ponder, Retroviral Vector-mediated Gene Therapy to Mucopolysaccharidosis I Mice Improves Sensorimotor Impairments and Other Behavior Deficits, J Inherit Metab Dis. in press.
    • J Inherit Metab Dis
    • Baldo, G.1    Wozniak, D.2    Ohlemiller, K.3    Zhang, Y.4    Giugliani, R.R.5    Ponder, K.P.6
  • 17
    • 37349103032 scopus 로고    scopus 로고
    • Progression of multiple behavioral deficits with various ages of onset in a murine model of Hurler syndrome
    • Pan D., Sciascia A., Vorhees C.V., Williams M.T. Progression of multiple behavioral deficits with various ages of onset in a murine model of Hurler syndrome. Brain Res. 2008, 1188:241-253.
    • (2008) Brain Res. , vol.1188 , pp. 241-253
    • Pan, D.1    Sciascia, A.2    Vorhees, C.V.3    Williams, M.T.4
  • 18
    • 84863951229 scopus 로고    scopus 로고
    • Intraperitoneal implant of recombinant encapsulated cells overexpressing alpha-l-iduronidase partially corrects visceral pathology in Mucopolysaccharidosis type I mice
    • Baldo G., Mayer F.Q., Martinelli B., Meyer F., Burin M., Meurer L., Tavares A.M., Giugliani R., Matte U. Intraperitoneal implant of recombinant encapsulated cells overexpressing alpha-l-iduronidase partially corrects visceral pathology in Mucopolysaccharidosis type I mice. Cytotherapy 2012, 14:860-867.
    • (2012) Cytotherapy , vol.14 , pp. 860-867
    • Baldo, G.1    Mayer, F.Q.2    Martinelli, B.3    Meyer, F.4    Burin, M.5    Meurer, L.6    Tavares, A.M.7    Giugliani, R.8    Matte, U.9
  • 23
    • 33745943855 scopus 로고    scopus 로고
    • Cardiac findings after enzyme replacement therapy for mucopolysaccharidosis type I
    • Braunlin E.A., Berry I.M., Whitley C.B. Cardiac findings after enzyme replacement therapy for mucopolysaccharidosis type I. Am. J. Cardiol. 2006, 98:416-418.
    • (2006) Am. J. Cardiol. , vol.98 , pp. 416-418
    • Braunlin, E.A.1    Berry, I.M.2    Whitley, C.B.3
  • 25
    • 79956012320 scopus 로고    scopus 로고
    • Aortic stenosis in a patient with Hurler's syndrome after bone marrow transplantation
    • Watanabe N., Anagnostopoulos P.V., Azakie A. Aortic stenosis in a patient with Hurler's syndrome after bone marrow transplantation. Cardiol. Young 2011, 21:349-350.
    • (2011) Cardiol. Young , vol.21 , pp. 349-350
    • Watanabe, N.1    Anagnostopoulos, P.V.2    Azakie, A.3
  • 29
    • 77953232381 scopus 로고    scopus 로고
    • Efficacy of recombinant human alpha-l-iduronidase (laronidase) on restricted range of motion of upper extremities in mucopolysaccharidosis type I patients
    • Tylki-Szymanska A., Marucha J., Jurecka A., Syczewska M., Czartoryska B. Efficacy of recombinant human alpha-l-iduronidase (laronidase) on restricted range of motion of upper extremities in mucopolysaccharidosis type I patients. J. Inherit. Metab. Dis. 2010, 33:151-157.
    • (2010) J. Inherit. Metab. Dis. , vol.33 , pp. 151-157
    • Tylki-Szymanska, A.1    Marucha, J.2    Jurecka, A.3    Syczewska, M.4    Czartoryska, B.5
  • 30
    • 0037906571 scopus 로고    scopus 로고
    • Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I
    • Kakavanos R., Turner C.T., Hopwood J.J., Kakkis E.D., Brooks D.A. Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I. Lancet 2003, 361:1608-1613.
    • (2003) Lancet , vol.361 , pp. 1608-1613
    • Kakavanos, R.1    Turner, C.T.2    Hopwood, J.J.3    Kakkis, E.D.4    Brooks, D.A.5
  • 31
    • 48749084652 scopus 로고    scopus 로고
    • Immune response hinders therapy for lysosomal storage diseases
    • Ponder K.P. Immune response hinders therapy for lysosomal storage diseases. J. Clin. Invest. 2008, 118:2686-2689.
    • (2008) J. Clin. Invest. , vol.118 , pp. 2686-2689
    • Ponder, K.P.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.