메뉴 건너뛰기




Volumn 111, Issue 2, 2014, Pages 116-122

High-dose enzyme replacement therapy in murine Hurler syndrome

Author keywords

Blood brain barrier; Enzyme replacement therapy; High dose; Hurler syndrome; Mucopolysaccharidosis I

Indexed keywords

GANGLIOSIDE GM3; LARONIDASE; LEVO IDURONIDASE; WATER;

EID: 84893687476     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2013.09.008     Document Type: Article
Times cited : (42)

References (32)
  • 2
    • 4344630950 scopus 로고    scopus 로고
    • Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier
    • Urayama A., Grubb J.H., Sly W.S., Banks W.A. Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier. Proc. Natl. Acad. Sci. U. S. A. 2004, 101:12658-12663.
    • (2004) Proc. Natl. Acad. Sci. U. S. A. , vol.101 , pp. 12658-12663
    • Urayama, A.1    Grubb, J.H.2    Sly, W.S.3    Banks, W.A.4
  • 3
    • 0030954670 scopus 로고    scopus 로고
    • Carbohydrate structures of recombinant human alpha-l-iduronidase secreted by Chinese hamster ovary cells
    • Zhao K.W., Faull K.F., Kakkis E.D., Neufeld E.F. Carbohydrate structures of recombinant human alpha-l-iduronidase secreted by Chinese hamster ovary cells. J. Biol. Chem. 1997, 272:22758-22765.
    • (1997) J. Biol. Chem. , vol.272 , pp. 22758-22765
    • Zhao, K.W.1    Faull, K.F.2    Kakkis, E.D.3    Neufeld, E.F.4
  • 4
    • 48949117579 scopus 로고    scopus 로고
    • Lysosomal storage diseases and the blood-brain barrier
    • Begley D.J., Pontikis C.C., Scarpa M. Lysosomal storage diseases and the blood-brain barrier. Curr. Pharm. Des. 2008, 14:1566-1580.
    • (2008) Curr. Pharm. Des. , vol.14 , pp. 1566-1580
    • Begley, D.J.1    Pontikis, C.C.2    Scarpa, M.3
  • 5
    • 42149113096 scopus 로고    scopus 로고
    • Central nervous system therapy for lysosomal storage disorders
    • Enns G.M., Huhn S.L. Central nervous system therapy for lysosomal storage disorders. Neurosurg. Focus. 2008, 24:3-4.
    • (2008) Neurosurg. Focus. , vol.24 , pp. 3-4
    • Enns, G.M.1    Huhn, S.L.2
  • 7
    • 24644516577 scopus 로고    scopus 로고
    • Enzyme replacement therapy results in substantial improvements in early clinical phenotype in a mouse model of globoid cell leukodystrophy
    • Lee W.C., Courtenay A., Troendle F.J., Stallings-Mann M.L., Dickey C.A., DeLucia M.W., Dickson D.W., Eckman C.B. Enzyme replacement therapy results in substantial improvements in early clinical phenotype in a mouse model of globoid cell leukodystrophy. FASEB J. 2005, 19:1549-1551.
    • (2005) FASEB J. , vol.19 , pp. 1549-1551
    • Lee, W.C.1    Courtenay, A.2    Troendle, F.J.3    Stallings-Mann, M.L.4    Dickey, C.A.5    DeLucia, M.W.6    Dickson, D.W.7    Eckman, C.B.8
  • 16
    • 34848831191 scopus 로고    scopus 로고
    • Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy
    • Garcia-Rivera M.F., Colvin-Wanshura L.E., Nelson M.S., Nan Z., Khan S.A., Rogers T.B., Maitra I., Low W.C., Gupta P. Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy. Brain Res. Bull. 2007, 74:429-438.
    • (2007) Brain Res. Bull. , vol.74 , pp. 429-438
    • Garcia-Rivera, M.F.1    Colvin-Wanshura, L.E.2    Nelson, M.S.3    Nan, Z.4    Khan, S.A.5    Rogers, T.B.6    Maitra, I.7    Low, W.C.8    Gupta, P.9
  • 17
    • 33845287687 scopus 로고    scopus 로고
    • Stress, prefrontal cortex and environmental enrichment: studies on dopamine and acetylcholine release and working memory performance in rats
    • Del Arco A., Segovia G., Garrido P., de Blas M., Mora F. Stress, prefrontal cortex and environmental enrichment: studies on dopamine and acetylcholine release and working memory performance in rats. Behav. Brain Res. 2007, 176:267-273.
    • (2007) Behav. Brain Res. , vol.176 , pp. 267-273
    • Del Arco, A.1    Segovia, G.2    Garrido, P.3    de Blas, M.4    Mora, F.5
  • 18
    • 79955965084 scopus 로고    scopus 로고
    • Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I
    • Wolf D.A., Lenander A.W., Nan Z., Belur L.R., Whitley C.B., Gupta P., Low W.C., McIvor R.S. Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I. Neurobiol. Dis. 2011, 43:123-133.
    • (2011) Neurobiol. Dis. , vol.43 , pp. 123-133
    • Wolf, D.A.1    Lenander, A.W.2    Nan, Z.3    Belur, L.R.4    Whitley, C.B.5    Gupta, P.6    Low, W.C.7    McIvor, R.S.8
  • 22
    • 0031926575 scopus 로고    scopus 로고
    • Follow-up of nine patients with Hurler syndrome after bone marrow transplantation
    • Guffon N., Souillet G., Maire I., Straczek J., Guibaud P. Follow-up of nine patients with Hurler syndrome after bone marrow transplantation. J. Pediatr. 1998, 133:119-125.
    • (1998) J. Pediatr. , vol.133 , pp. 119-125
    • Guffon, N.1    Souillet, G.2    Maire, I.3    Straczek, J.4    Guibaud, P.5
  • 25
    • 2442643119 scopus 로고    scopus 로고
    • Are the extracellular [correction of extracelluar] pathways a conduit for the delivery of therapeutics to the brain?
    • Banks W.A. Are the extracellular [correction of extracelluar] pathways a conduit for the delivery of therapeutics to the brain?. Curr. Pharm. Des. 2004, 10:1365-1370.
    • (2004) Curr. Pharm. Des. , vol.10 , pp. 1365-1370
    • Banks, W.A.1
  • 28
    • 40449086665 scopus 로고    scopus 로고
    • Dose translation from animal to human studies revisited
    • Reagan-Shaw S., Nihal M., Ahmad N. Dose translation from animal to human studies revisited. FASEB J. 2008, 22:659-661.
    • (2008) FASEB J. , vol.22 , pp. 659-661
    • Reagan-Shaw, S.1    Nihal, M.2    Ahmad, N.3
  • 29
    • 33044491354 scopus 로고    scopus 로고
    • Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, Rockville, Maryland, USA
    • Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research Estimating the Safe Starting Dose in Clinical Trials for Therapeutics in Adult Healthy Volunteers 2002, U.S. Food and Drug Administration, Rockville, Maryland, USA.
    • (2002) Estimating the Safe Starting Dose in Clinical Trials for Therapeutics in Adult Healthy Volunteers
  • 30
    • 84860138173 scopus 로고    scopus 로고
    • Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration
    • Wolf D.A., Hanson L.R., Aronovich E.L., Nan Z., Low W.C., Frey W.H., McIvor R.S. Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration. Mol. Genet. Metab. 2012, 106:131-134.
    • (2012) Mol. Genet. Metab. , vol.106 , pp. 131-134
    • Wolf, D.A.1    Hanson, L.R.2    Aronovich, E.L.3    Nan, Z.4    Low, W.C.5    Frey, W.H.6    McIvor, R.S.7


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.