메뉴 건너뛰기




Volumn 39, Issue 5, 2011, Pages 879-892

Safety evaluation of chronic intrathecal administration of idursulfase-IT in cynomolgus monkeys

Author keywords

cynomolgus monkey; Hunter syndrome; iduronate sulfatase; idursulfase; intrathecal; lysosomal storage disease; mucopolysaccharidosis II

Indexed keywords

IDURONATE 2 SULFATASE;

EID: 79961071835     PISSN: 01926233     EISSN: 15331601     Source Type: Journal    
DOI: 10.1177/0192623311409595     Document Type: Article
Times cited : (37)

References (34)
  • 1
    • 73749084892 scopus 로고    scopus 로고
    • Repeated intrathecal injections of recombinant human 4-sulphatase remove dural storage in mature mucopolysaccharidosis VI cats primed with a short-course tolerisation regimen
    • Auclair D., Finnie J., White J., Nielsen T., Fuller M., Kakkis E., Cheng A., O'Neill C. A., Hopwood J. J. Repeated intrathecal injections of recombinant human 4-sulphatase remove dural storage in mature mucopolysaccharidosis VI cats primed with a short-course tolerisation regimen. Mol Genet Metab. 2010 ; 99: 132-41
    • (2010) Mol Genet Metab , vol.99 , pp. 132-41
    • Auclair, D.1    Finnie, J.2    White, J.3    Nielsen, T.4    Fuller, M.5    Kakkis, E.6    Cheng, A.7    O'Neill, C.A.8    Hopwood, J.J.9
  • 2
    • 0006499259 scopus 로고
    • Cerebrospinal fluid infusion of alglucerase in the treatment of acute neuronopathic Gaucher's disease [abstract]
    • Bembi B., Ciana G., Bottega M., Pelos G., Gornati R., Berra B. Cerebrospinal fluid infusion of alglucerase in the treatment of acute neuronopathic Gaucher's disease [abstract]. Pediatr Res. 1995 ; 38: 425
    • (1995) Pediatr Res , vol.38 , pp. 425
    • Bembi, B.1    Ciana, G.2    Bottega, M.3    Pelos, G.4    Gornati, R.5    Berra, B.6
  • 3
    • 79959525794 scopus 로고    scopus 로고
    • Morphologic changes associated with intrathecal catheters for direct delivery to the central nervous system in preclinical studies
    • Butt M. T. Morphologic changes associated with intrathecal catheters for direct delivery to the central nervous system in preclinical studies. Toxicol Pathol. 2011 ; 39: 213-19
    • (2011) Toxicol Pathol , vol.39 , pp. 213-19
    • Butt, M.T.1
  • 6
    • 34248587050 scopus 로고    scopus 로고
    • Preclinical dose ranging studies for enzyme replacement therapy with idursulfase in a knock-out mouse model of MPS II
    • DOI 10.1016/j.ymgme.2007.03.003, PII S1096719207001011
    • Garcia A. R., DaCosta J. M., Pan J., Muenzer J., Lamsa J. C. Preclinical dose ranging studies for enzyme replacement therapy with idursulfase in a knock-out mouse model of MPS II. Mol Genet Metab. 2007 ; 91: 183-90 (Pubitemid 46765176)
    • (2007) Molecular Genetics and Metabolism , vol.91 , Issue.2 , pp. 183-190
    • Garcia, A.R.1    DaCosta, J.M.2    Pan, J.3    Muenzer, J.4    Lamsa, J.C.5
  • 7
    • 37249043762 scopus 로고    scopus 로고
    • The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome)
    • DOI 10.1007/s10545-007-0641-8
    • Garcia A. R., Pan J., Lamsa J. C., Muenzer J. The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome). J Inherit Metab Dis. 2007 ; 30: 924-34 (Pubitemid 350263133)
    • (2007) Journal of Inherited Metabolic Disease , vol.30 , Issue.6 , pp. 924-934
    • Garcia, A.R.1    Pan, J.2    Lamsa, J.C.3    Muenzer, J.4
  • 9
    • 77649195183 scopus 로고    scopus 로고
    • Delivery of recombinant proteins via the cerebrospinal fluid as a therapy option for neurodegenerative lysosomal storage diseases
    • Hemsley K. M., Hopwood J. J. Delivery of recombinant proteins via the cerebrospinal fluid as a therapy option for neurodegenerative lysosomal storage diseases. Int J Clin Pharmacol Ther. 2009 ; 47 Suppl 1: S118-23
    • (2009) Int J Clin Pharmacol Ther , vol.471 , pp. 118-23
    • Hemsley, K.M.1    Hopwood, J.J.2
  • 10
    • 52949100951 scopus 로고    scopus 로고
    • Effect of high dose, repeated intra-cerebrospinal fluid injection of sulphamidase on neuropathology in mucopolysaccharidosis type IIIA mice
    • Hemsley K. M., Beard H., King B. M., Hopwood J. J. Effect of high dose, repeated intra-cerebrospinal fluid injection of sulphamidase on neuropathology in mucopolysaccharidosis type IIIA mice. Genes Brain Behav. 2008 ; 7: 740-53
    • (2008) Genes Brain Behav , vol.7 , pp. 740-53
    • Hemsley, K.M.1    Beard, H.2    King, B.M.3    Hopwood, J.J.4
  • 11
    • 33846904009 scopus 로고    scopus 로고
    • Injection of recombinant human sulfamidase into the CSF via the cerebellomedullary cistern in MPS IIIA mice
    • DOI 10.1016/j.ymgme.2006.10.005, PII S109671920600326X
    • Hemsley K. M., King B., Hopwood J. J. Injection of recombinant human sulfamidase into the CSF via the cerebellomedullary cistern in MPS IIIA mice. Mol Genet Metab. 2007 ; 90: 313-28 (Pubitemid 46241904)
    • (2007) Molecular Genetics and Metabolism , vol.90 , Issue.3 SPEC. ISS. , pp. 313-328
    • Hemsley, K.M.1    King, B.2    Hopwood, J.J.3
  • 15
    • 4944239910 scopus 로고    scopus 로고
    • Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I
    • DOI 10.1016/j.ymgme.2004.07.003, PII S1096719204001763, ASHG 2004 Meeting Toronto
    • Kakkis E., McEntee M., Vogler C., Le S., Levy B., Belichenko P., Mobley W., Dickson P., Hanson S., Passage M. Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I. Mol Genet Metab. 2004 ; 83: 163-74 (Pubitemid 39328075)
    • (2004) Molecular Genetics and Metabolism , vol.83 , Issue.1-2 , pp. 163-174
    • Kakkis, E.1    McEntee, M.2    Vogler, C.3    Le, S.4    Levy, B.5    Belichenko, P.6    Mobley, W.7    Dickson, P.8    Hanson, S.9    Passage, M.10
  • 16
    • 0035999743 scopus 로고    scopus 로고
    • Enzyme replacement therapy for the mucopolysaccharide storage disorders
    • DOI 10.1517/13543784.11.5.675
    • Kakkis E. D. Enzyme replacement therapy for the mucopolysaccharide storage disorders. Expert Opin Investig Drugs. 2002 ; 11: 675-85 (Pubitemid 34520617)
    • (2002) Expert Opinion on Investigational Drugs , vol.11 , Issue.5 , pp. 675-685
    • Kakkis, E.D.1
  • 23
    • 0031028248 scopus 로고    scopus 로고
    • Intrathecal ciliary neurotrophic factor delivery for treatment of amyotrophic lateral sclerosis (phase i trial)
    • Penn R. D., Kroin J. S., York M. M., Cedarbaum J. M. Intrathecal ciliary neurotrophic factor delivery for treatment of amyotrophic lateral sclerosis (phase I trial). Neurosurgery. 1997 ; 40: 94-99
    • (1997) Neurosurgery , vol.40 , pp. 94-99
    • Penn, R.D.1    Kroin, J.S.2    York, M.M.3    Cedarbaum, J.M.4
  • 24
    • 33846882646 scopus 로고    scopus 로고
    • Brain oedema in focal ischaemia: Molecular pathophysiology and theoretical implications
    • DOI 10.1016/S1474-4422(07)70055-8, PII S1474442207700558
    • Simard J. M., Kent T. A., Chen M., Tarasov K. V., Gerzanich V. Brain oedema in focal ischaemia: molecular pathophysiology and theoretical implications. Lancet Neurol. 2007 ; 6: 258-68 (Pubitemid 46228081)
    • (2007) Lancet Neurology , vol.6 , Issue.3 , pp. 258-268
    • Simard, J.M.1    Kent, T.A.2    Chen, M.3    Tarasov, K.V.4    Gerzanich, V.5
  • 25
    • 53249138093 scopus 로고    scopus 로고
    • Brain endothelial cell-cell junctions: How to "open" the blood brain barrier
    • Stamatovic S. M., Keep R. F., Andjelkovic A. V. Brain endothelial cell-cell junctions: How to "open" the blood brain barrier. Curr Neuropharmacol. 2008 ; 6: 179-92
    • (2008) Curr Neuropharmacol , vol.6 , pp. 179-92
    • Stamatovic, S.M.1    Keep, R.F.2    Andjelkovic, A.V.3
  • 26
    • 84856309590 scopus 로고    scopus 로고
    • United States Food and Drug Administration Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research. Elaprase (idursulfase) injection, application no. 125151
    • United States Food and Drug Administration Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research. Elaprase (idursulfase) injection, application no. 125151. Pharmacology Review(s); 2006. http://www.accessdata.fda.gov/drugsatfda-docs/nda/2006/125151s0000-Elaprase- PharmR.pdf
    • (2006) Pharmacology Review(s)
  • 27
    • 34547896622 scopus 로고    scopus 로고
    • Epinephrine enhances lysosomal enzyme delivery across the blood brain barrier by up-regulation of the mannose 6-phosphate receptor
    • Urayama A., Grubb J. H., Banks W. A., Sly W. S. Epinephrine enhances lysosomal enzyme delivery across the blood brain barrier by up-regulation of the mannose 6-phosphate receptor. Proc Natl Acad Sci U S A. 2007 ; 104: 12873-78
    • (2007) Proc Natl Acad Sci U S A , vol.104 , pp. 12873-78
    • Urayama, A.1    Grubb, J.H.2    Banks, W.A.3    Sly, W.S.4
  • 31
    • 0017570202 scopus 로고
    • Hunter syndrome presenting as macrocephaly and hydrocephalus
    • Yatziv S., Epstein C. J. Hunter syndrome presenting as macrocephaly and hydrocephalus. J Med Genet. 1977 ; 14: 445-47 (Pubitemid 8262660)
    • (1977) Journal of Medical Genetics , vol.14 , Issue.6 , pp. 445-447
    • Yatziv, S.1    Epstein, C.J.2
  • 32
    • 0019990520 scopus 로고
    • Incidence of Hunter's Syndrome
    • Young I.D., Harper P.S. Incidence of Hunter's Syndrome. Hum Genet. 1982 ; 60 391 :
    • (1982) Hum Genet , pp. 60391
    • Young, I.D.1    Harper, P.S.2
  • 33
    • 0020613358 scopus 로고
    • The natural history of the severe form of Hunter's syndrome: A study based on 52 cases
    • Young I. D., Harper P. S. The natural history of the severe form of Hunter's syndrome: A study based on 52 cases. Dev Med Child Neurol. 1983 ; 25: 481-89 (Pubitemid 13030892)
    • (1983) Developmental Medicine and Child Neurology , vol.25 , Issue.4 , pp. 481-489
    • Young, I.D.1    Harper, P.S.2
  • 34
    • 0032904141 scopus 로고    scopus 로고
    • Delivery, distribution, and neuronal uptake of exogenous mannose- terminal glucocerebrosidase in the intact rat brain
    • DOI 10.1023/A:1022578424693
    • Zirzow G. C., Sanchez O. A., Murray G. J., Brady R. O., Oldfield E. H. Delivery, distribution, and neuronal uptake of exogenous mannose-terminal glucocerebrosidase in the intact rat brain. Neurochem Res. 1999 ; 24: 301-5 (Pubitemid 29067974)
    • (1999) Neurochemical Research , vol.24 , Issue.2 , pp. 301-305
    • Zirzow, G.C.1    Sanchez, O.A.2    Murray, G.J.3    Brady, R.O.4    Oldfield, E.H.5


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.