메뉴 건너뛰기




Volumn 7, Issue 1, 2012, Pages

CNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: Implications for neurological outcomes of lysosomal storage disorder

Author keywords

[No Author keywords available]

Indexed keywords

GLYCOSAMINOGLYCAN; IODINE 124; RECOMBINANT ENZYME; RECOMBINANT IDURONATE 2 SULFATASE; UNCLASSIFIED DRUG; IDURONATE 2 SULFATASE; RADIOACTIVE IODINE; RECOMBINANT PROTEIN;

EID: 84862940707     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0030341     Document Type: Article
Times cited : (107)

References (65)
  • 2
    • 9344226762 scopus 로고    scopus 로고
    • Molecular mechanisms of cerebrospinal fluid production
    • Brown PD, Davies SL, Speake T, Millar ID, (2004) Molecular mechanisms of cerebrospinal fluid production. Neuroscience 129: 957-970.
    • (2004) Neuroscience , vol.129 , pp. 957-970
    • Brown, P.D.1    Davies, S.L.2    Speake, T.3    Millar, I.D.4
  • 3
    • 0002354878 scopus 로고    scopus 로고
    • Ventricular organization of the cerebrospinal fluid: blood-brain barrier, brain edema, and hydrocephalus
    • In: Kandel E, Schwartz JH, Jessel TM, editors, New York, McGraw-Hill
    • Laterra J, Goldstein GW, (2000) Ventricular organization of the cerebrospinal fluid: blood-brain barrier, brain edema, and hydrocephalus In: Kandel E, Schwartz JH, Jessel TM, editors. Principles of neural science New York McGraw-Hill pp. 1288-1301.
    • (2000) Principles of Neural Science , pp. 1288-1301
    • Laterra, J.1    Goldstein, G.W.2
  • 4
    • 0042899083 scopus 로고    scopus 로고
    • Blood-brain barrier drug targeting: the future of brain drug development
    • Pardridge WM, (2003) Blood-brain barrier drug targeting: the future of brain drug development. Mol Interv 3: 90-105, 151.
    • (2003) Mol Interv , vol.3
    • Pardridge, W.M.1
  • 5
    • 77949405266 scopus 로고    scopus 로고
    • Genetic engineering of IgG-glucuronidase fusion proteins
    • Boado RJ, Pardridge WM, (2010) Genetic engineering of IgG-glucuronidase fusion proteins. J. Drug Target 18: 205-211.
    • (2010) J. Drug Target , vol.18 , pp. 205-211
    • Boado, R.J.1    Pardridge, W.M.2
  • 6
    • 70350236476 scopus 로고    scopus 로고
    • AGT-181: expression in CHO cells and pharmacokinetics, safety, and plasma iduronidase enzyme activity in Rhesus monkeys
    • Boado RJ, Hui EK, Lu JZ, Pardridge WM, (2009) AGT-181: expression in CHO cells and pharmacokinetics, safety, and plasma iduronidase enzyme activity in Rhesus monkeys. J Biotechnol 144: 135-141.
    • (2009) J Biotechnol , vol.144 , pp. 135-141
    • Boado, R.J.1    Hui, E.K.2    Lu, J.Z.3    Pardridge, W.M.4
  • 7
    • 60549101321 scopus 로고    scopus 로고
    • Engineering and expression of a chimeric transferrin receptor monoclonal antibody for blood-brain barrier delivery in the mouse
    • Boado RJ, Zhang Y, Wang Y, Pardridge WM, (2009) Engineering and expression of a chimeric transferrin receptor monoclonal antibody for blood-brain barrier delivery in the mouse. Biotechnol Bioeng 102: 1251-1258.
    • (2009) Biotechnol Bioeng , vol.102 , pp. 1251-1258
    • Boado, R.J.1    Zhang, Y.2    Wang, Y.3    Pardridge, W.M.4
  • 8
    • 76249087751 scopus 로고    scopus 로고
    • Pharmacokinetics and brain uptake of a genetically engineered bifunctional fusion antibody targeting the mouse transferrin receptor
    • Boado RJ, Zhou QH, Lu JZ, Hui EK, Pardridge WM, (2010) Pharmacokinetics and brain uptake of a genetically engineered bifunctional fusion antibody targeting the mouse transferrin receptor. Mol Pharm 7: 237-244.
    • (2010) Mol Pharm , vol.7 , pp. 237-244
    • Boado, R.J.1    Zhou, Q.H.2    Lu, J.Z.3    Hui, E.K.4    Pardridge, W.M.5
  • 9
    • 0036518450 scopus 로고    scopus 로고
    • Receptor-mediated delivery of an antisense gene to human brain cancer cells
    • Zhang Y, Jeong Lee H, Boado RJ, Pardridge WM, (2002) Receptor-mediated delivery of an antisense gene to human brain cancer cells. J Gene Med 4: 183-194.
    • (2002) J Gene Med , vol.4 , pp. 183-194
    • Zhang, Y.1    Jeong Lee, H.2    Boado, R.J.3    Pardridge, W.M.4
  • 10
    • 0033996507 scopus 로고    scopus 로고
    • Transport across the primate blood-brain barrier of a genetically engineered chimeric monoclonal antibody to the human insulin receptor
    • Coloma MJ, Lee HJ, Kurihara A, Landaw EM, Boado RJ, et al. (2000) Transport across the primate blood-brain barrier of a genetically engineered chimeric monoclonal antibody to the human insulin receptor. Pharm Res 17: 266-274.
    • (2000) Pharm Res , vol.17 , pp. 266-274
    • Coloma, M.J.1    Lee, H.J.2    Kurihara, A.3    Landaw, E.M.4    Boado, R.J.5
  • 11
    • 67649205803 scopus 로고    scopus 로고
    • The potential for nanoparticle-based drug delivery to the brain: overcoming the blood-brain barrier
    • Barbu E, Molnar E, Tsibouklis J, Gorecki DC, (2009) The potential for nanoparticle-based drug delivery to the brain: overcoming the blood-brain barrier. Expert Opin Drug Deliv 6: 553-565.
    • (2009) Expert Opin Drug Deliv , vol.6 , pp. 553-565
    • Barbu, E.1    Molnar, E.2    Tsibouklis, J.3    Gorecki, D.C.4
  • 14
    • 77749283336 scopus 로고    scopus 로고
    • Central nervous system delivery of large molecules: challenges and new frontiers for intrathecally administered therapeutics
    • Soderquist RG, Mahoney MJ, (2010) Central nervous system delivery of large molecules: challenges and new frontiers for intrathecally administered therapeutics. Expert Opin Drug Deliv 7: 285-293.
    • (2010) Expert Opin Drug Deliv , vol.7 , pp. 285-293
    • Soderquist, R.G.1    Mahoney, M.J.2
  • 15
    • 33748434958 scopus 로고    scopus 로고
    • Intrathecal baclofen in the treatment of adult spasticity
    • Hsieh JC, Penn RD, (2006) Intrathecal baclofen in the treatment of adult spasticity. Neurosurg Focus 21: 1-6.
    • (2006) Neurosurg Focus , vol.21 , pp. 1-6
    • Hsieh, J.C.1    Penn, R.D.2
  • 17
    • 0037435511 scopus 로고    scopus 로고
    • Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD
    • Nutt JG, Burchiel KJ, Comella CL, Jankovic J, Lang AE, et al. (2003) Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD. Neurology 60: 69-73.
    • (2003) Neurology , vol.60 , pp. 69-73
    • Nutt, J.G.1    Burchiel, K.J.2    Comella, C.L.3    Jankovic, J.4    Lang, A.E.5
  • 18
    • 85052608961 scopus 로고    scopus 로고
    • GDNF delivery for Parkinson's disease
    • Patel NK, Gill SS, (2007) GDNF delivery for Parkinson's disease. Acta Neurochir 97 (Suppl): 135-154.
    • (2007) Acta Neurochir , vol.97 , Issue.SUPPL. , pp. 135-154
    • Patel, N.K.1    Gill, S.S.2
  • 19
    • 84921706429 scopus 로고    scopus 로고
    • Comparative efficacy of epidural, subarachnoid, and intracerebroventricular opioids in patients with pain due to cancer
    • CD005178
    • Ballantyne JC, Carwood CM, (2005) Comparative efficacy of epidural, subarachnoid, and intracerebroventricular opioids in patients with pain due to cancer. Cochrane Database Syst Rev CD005178.
    • (2005) Cochrane Database Syst Rev
    • Ballantyne, J.C.1    Carwood, C.M.2
  • 22
    • 0030297788 scopus 로고    scopus 로고
    • Rapid distribution of intraventricularly administered sucrose into cerebrospinal fluid cisterns via subarachnoid velae in rat
    • Ghersi-Egea JF, Finnegan W, Chen JL, Fenstermacher JD, (1996) Rapid distribution of intraventricularly administered sucrose into cerebrospinal fluid cisterns via subarachnoid velae in rat. Neuroscience 75: 1271-1288.
    • (1996) Neuroscience , vol.75 , pp. 1271-1288
    • Ghersi-Egea, J.F.1    Finnegan, W.2    Chen, J.L.3    Fenstermacher, J.D.4
  • 23
    • 0032759087 scopus 로고    scopus 로고
    • Studies of cerebrospinal fluid flow and penetration into brain following lateral ventricle and cisterna magna injections of the tracer [14C]inulin in rat
    • Proescholdt MG, Hutto B, Brady LS, Herkenham M, (2000) Studies of cerebrospinal fluid flow and penetration into brain following lateral ventricle and cisterna magna injections of the tracer [14C]inulin in rat. Neuroscience 95: 577-592.
    • (2000) Neuroscience , vol.95 , pp. 577-592
    • Proescholdt, M.G.1    Hutto, B.2    Brady, L.S.3    Herkenham, M.4
  • 24
    • 0025103546 scopus 로고
    • Rapid solute transport throughout the brain via paravascular fluid pathways
    • Rennels ML, Blaumanis OR, Grady PA, (1990) Rapid solute transport throughout the brain via paravascular fluid pathways. Adv Neurol 52: 431-439.
    • (1990) Adv Neurol , vol.52 , pp. 431-439
    • Rennels, M.L.1    Blaumanis, O.R.2    Grady, P.A.3
  • 25
    • 0021959647 scopus 로고
    • Evidence for a 'paravascular' fluid circulation in the mammalian central nervous system, provided by the rapid distribution of tracer protein throughout the brain from the subarachnoid space
    • Rennels ML, Gregory TF, Blaumanis OR, Fujimoto K, Grady PA, (1985) Evidence for a 'paravascular' fluid circulation in the mammalian central nervous system, provided by the rapid distribution of tracer protein throughout the brain from the subarachnoid space. Brain Res 326: 47-63.
    • (1985) Brain Res , vol.326 , pp. 47-63
    • Rennels, M.L.1    Gregory, T.F.2    Blaumanis, O.R.3    Fujimoto, K.4    Grady, P.A.5
  • 27
    • 38849176942 scopus 로고    scopus 로고
    • Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome)
    • Martin R, Beck M, Eng C, Giugliani R, Harmatz P, et al. (2008) Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome). Pediatrics 121: e377-386.
    • (2008) Pediatrics , vol.121
    • Martin, R.1    Beck, M.2    Eng, C.3    Giugliani, R.4    Harmatz, P.5
  • 28
    • 84862967821 scopus 로고    scopus 로고
    • [package insert], Cambridge, MA, Shire Human Genetic Therapies, Inc
    • Elaprase [package insert] (2010) Cambridge, MA Shire Human Genetic Therapies, Inc.
    • (2010) Elaprase
  • 29
    • 48949117579 scopus 로고    scopus 로고
    • Lysosomal storage diseases and the blood-brain barrier
    • Begley DJ, Pontikis CC, Scarpa M, (2008) Lysosomal storage diseases and the blood-brain barrier. Curr Pharm Des 14: 1566-1580.
    • (2008) Curr Pharm Des , vol.14 , pp. 1566-1580
    • Begley, D.J.1    Pontikis, C.C.2    Scarpa, M.3
  • 30
    • 36849001356 scopus 로고    scopus 로고
    • Intrathecal treatment with anti-Nogo-A antibody improves functional recovery in adult rats after stroke
    • Tsai SY, Markus TM, Andrews EM, Cheatwood JL, Emerick AJ, et al. (2007) Intrathecal treatment with anti-Nogo-A antibody improves functional recovery in adult rats after stroke. Exp Brain Res182: 261-266.
    • (2007) Exp Brain Res , vol.182 , pp. 261-266
    • Tsai, S.Y.1    Markus, T.M.2    Andrews, E.M.3    Cheatwood, J.L.4    Emerick, A.J.5
  • 31
    • 4944239910 scopus 로고    scopus 로고
    • Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I
    • Kakkis E, McEntee M, Vogler C, Le S, Levy B, et al. (2004) Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I. Mol Genet Metab 83: 163-174.
    • (2004) Mol Genet Metab , vol.83 , pp. 163-174
    • Kakkis, E.1    McEntee, M.2    Vogler, C.3    Le, S.4    Levy, B.5
  • 32
    • 0023731412 scopus 로고
    • Drug "diffusion" within the brain
    • Fenstermacher J, Kaye T, (1988) Drug "diffusion" within the brain. Ann N Y Acad Sci 531: 29-39.
    • (1988) Ann N Y Acad Sci , vol.531 , pp. 29-39
    • Fenstermacher, J.1    Kaye, T.2
  • 33
    • 0017295213 scopus 로고
    • Spinal descent of cerebrospinal fluid in man
    • Chiro GD, Hammock MK, Bleyer WA, (1976) Spinal descent of cerebrospinal fluid in man. Neurology 26: 1-8.
    • (1976) Neurology , vol.26 , pp. 1-8
    • Chiro, G.D.1    Hammock, M.K.2    Bleyer, W.A.3
  • 34
    • 33947166059 scopus 로고    scopus 로고
    • Fluid compartments of the central nervous system
    • In: Zheng W, Chodobski A, editors, Boca Raton, Chapman & Hall/CRC
    • Segal MB, (2005) Fluid compartments of the central nervous system. In: Zheng W, Chodobski A, editors. The blood-cerebrospinal fluid barrier Boca Raton, Chapman & Hall/CRC pp. 83-99.
    • (2005) The Blood-Cerebrospinal Fluid Barrier , pp. 83-99
    • Segal, M.B.1
  • 35
    • 79961071835 scopus 로고    scopus 로고
    • Safety evaluation of chronic intrathecal administration of idursulfase-IT in cynomolgus monkeys
    • Felice BR, Wright TL, Boyd RB, Butt MT, Pfeifer RW, et al. (2011) Safety evaluation of chronic intrathecal administration of idursulfase-IT in cynomolgus monkeys. Toxicol Pathol 39: 879-892.
    • (2011) Toxicol Pathol , vol.39 , pp. 879-892
    • Felice, B.R.1    Wright, T.L.2    Boyd, R.B.3    Butt, M.T.4    Pfeifer, R.W.5
  • 36
    • 37249043762 scopus 로고    scopus 로고
    • The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome)
    • Garcia AR, Pan J, Lamsa JC, Muenzer J, (2007) The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome). J Inherit Metab Dis 30: 924-934.
    • (2007) J Inherit Metab Dis , vol.30 , pp. 924-934
    • Garcia, A.R.1    Pan, J.2    Lamsa, J.C.3    Muenzer, J.4
  • 37
    • 0035999743 scopus 로고    scopus 로고
    • Enzyme replacement therapy for the mucopolysaccharide storage disorders
    • Kakkis ED, (2002) Enzyme replacement therapy for the mucopolysaccharide storage disorders. Expert Opin Investig Drugs 11: 675-685.
    • (2002) Expert Opin Investig Drugs , vol.11 , pp. 675-685
    • Kakkis, E.D.1
  • 38
    • 77649195183 scopus 로고    scopus 로고
    • Delivery of recombinant proteins via the cerebrospinal fluid as a therapy option for neurodegenerative lysosomal storage diseases
    • Hemsley KM, Hopwood JJ, (2009) Delivery of recombinant proteins via the cerebrospinal fluid as a therapy option for neurodegenerative lysosomal storage diseases. Int J Clin Pharmacol. Ther 47 (Suppl 1): S118-123.
    • (2009) Int J Clin Pharmacol Ther , vol.47 , Issue.SUPPL. 1
    • Hemsley, K.M.1    Hopwood, J.J.2
  • 39
    • 41949126259 scopus 로고    scopus 로고
    • Mechanisms of neurodegeneration in mucopolysaccharidoses II and IIIB: analysis of human brain tissue
    • Hamano K, Hayashi M, Shioda K, Fukatsu R, Mizutani S, (2008) Mechanisms of neurodegeneration in mucopolysaccharidoses II and IIIB: analysis of human brain tissue. Acta Neuropathol 115: 547-559.
    • (2008) Acta Neuropathol , vol.115 , pp. 547-559
    • Hamano, K.1    Hayashi, M.2    Shioda, K.3    Fukatsu, R.4    Mizutani, S.5
  • 40
    • 34250822221 scopus 로고    scopus 로고
    • Evaluation of chronic intrathecal administration of idursulfase-IT in cynomolgus monkeys Am J Neuroradiol
    • Vedolin L, Schwartz IVD, Komlos M, Schuch A, Puga AC, et al. (2007) Evaluation of chronic intrathecal administration of idursulfase-IT in cynomolgus monkeys Am J Neuroradiol 28: 1029-1033.
    • (2007) , vol.28 , pp. 1029-1033
    • Vedolin, L.1    Schwartz, I.V.D.2    Komlos, M.3    Schuch, A.4    Puga, A.C.5
  • 41
    • 4444287601 scopus 로고    scopus 로고
    • Brain MRI findings in patients with mucopolysaccharidosis types I and II and mild clinical presentation
    • Matheus MG, Castillo M, Smith JK, Armao D, Towle D, et al. (2004) Brain MRI findings in patients with mucopolysaccharidosis types I and II and mild clinical presentation. Neuroradiology 46: 666-672.
    • (2004) Neuroradiology , vol.46 , pp. 666-672
    • Matheus, M.G.1    Castillo, M.2    Smith, J.K.3    Armao, D.4    Towle, D.5
  • 42
    • 64549141555 scopus 로고    scopus 로고
    • Bone marrow transplantation in children with Hunter syndrome: outcome after 7 to 17 years
    • Guffon N, Bertrand Y, Forest I, Fouilhoux A, Froissart R, (2009) Bone marrow transplantation in children with Hunter syndrome: outcome after 7 to 17 years. J Pediatr 154: 733-737.
    • (2009) J Pediatr , vol.154 , pp. 733-737
    • Guffon, N.1    Bertrand, Y.2    Forest, I.3    Fouilhoux, A.4    Froissart, R.5
  • 43
    • 77649342578 scopus 로고    scopus 로고
    • Stem cell transplantation for neurometabolic and neurodegenerative diseases
    • Shihabuddin LS, Aubert I, (2010) Stem cell transplantation for neurometabolic and neurodegenerative diseases. Neuropharmacology 58: 845-854.
    • (2010) Neuropharmacology , vol.58 , pp. 845-854
    • Shihabuddin, L.S.1    Aubert, I.2
  • 44
    • 0036703479 scopus 로고    scopus 로고
    • Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream
    • Passini MA, Lee EB, Heuer GG, Wolfe JH, (2002) Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream. J Neurosci 22: 6437-6446.
    • (2002) J Neurosci , vol.22 , pp. 6437-6446
    • Passini, M.A.1    Lee, E.B.2    Heuer, G.G.3    Wolfe, J.H.4
  • 45
    • 34548610909 scopus 로고    scopus 로고
    • A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease
    • Cearley CN, Wolfe JH, (2007) A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease. J Neurosci 27: 9928-9940.
    • (2007) J Neurosci , vol.27 , pp. 9928-9940
    • Cearley, C.N.1    Wolfe, J.H.2
  • 46
    • 25144474418 scopus 로고    scopus 로고
    • Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery
    • Luca T, Givogri MI, Perani L, Galbiati F, Follenzi A, et al. (2005) Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery. Mol Ther 12: 669-679.
    • (2005) Mol Ther , vol.12 , pp. 669-679
    • Luca, T.1    Givogri, M.I.2    Perani, L.3    Galbiati, F.4    Follenzi, A.5
  • 47
    • 0035099437 scopus 로고    scopus 로고
    • In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice
    • Consiglio A, Quattrini A, Martino S, Bensdadoun JC, Dolcetta D, et al. (2001) In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice. Nat Med 7: 310-316.
    • (2001) Nat Med , vol.7 , pp. 310-316
    • Consiglio, A.1    Quattrini, A.2    Martino, S.3    Bensdadoun, J.C.4    Dolcetta, D.5
  • 48
    • 77953522837 scopus 로고    scopus 로고
    • Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models
    • Lattanzi A, Neri M, Maderna C, di Girolamo I, Martino S, et al. (2010) Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models. Hum Mol Genet 19: 2208-2227.
    • (2010) Hum Mol Genet , vol.19 , pp. 2208-2227
    • Lattanzi, A.1    Neri, M.2    Maderna, C.3    Di Girolamo, I.4    Martino, S.5
  • 49
    • 0037868094 scopus 로고    scopus 로고
    • Viral vectors for gene delivery to the nervous system
    • Davidson BL, Breakefield XO, (2003) Viral vectors for gene delivery to the nervous system. Nat Rev Neurosci 4: 353-364.
    • (2003) Nat Rev Neurosci , vol.4 , pp. 353-364
    • Davidson, B.L.1    Breakefield, X.O.2
  • 50
    • 0141996932 scopus 로고    scopus 로고
    • Adeno-associated virus vector-mediated transduction in the cat brain
    • Vite CH, Passini MA, Haskins ME, Wolfe JH, (2003) Adeno-associated virus vector-mediated transduction in the cat brain. Gene Ther 10: 1874-1881.
    • (2003) Gene Ther , vol.10 , pp. 1874-1881
    • Vite, C.H.1    Passini, M.A.2    Haskins, M.E.3    Wolfe, J.H.4
  • 51
    • 9744251004 scopus 로고    scopus 로고
    • Integration target site selection for retroviruses and transposable elements
    • Wu X, Burgess SM, (2004) Integration target site selection for retroviruses and transposable elements. Cell Mol Life Sci 61: 2588-2596.
    • (2004) Cell Mol Life Sci , vol.61 , pp. 2588-2596
    • Wu, X.1    Burgess, S.M.2
  • 52
    • 0037441587 scopus 로고    scopus 로고
    • Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis
    • Woods NB, Muessig A, Schmidt M, Flygare J, Olsson K, et al. (2003) Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis. Blood 101: 1284-1289.
    • (2003) Blood , vol.101 , pp. 1284-1289
    • Woods, N.B.1    Muessig, A.2    Schmidt, M.3    Flygare, J.4    Olsson, K.5
  • 53
    • 20044396722 scopus 로고    scopus 로고
    • Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells
    • Hematti P, Hong BK, Ferguson C, Adler R, Hanawa H, et al. (2004) Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells. PLoS Biol 2: 2183-2190.
    • (2004) PLoS Biol , vol.2 , pp. 2183-2190
    • Hematti, P.1    Hong, B.K.2    Ferguson, C.3    Adler, R.4    Hanawa, H.5
  • 54
    • 0038054338 scopus 로고    scopus 로고
    • AAV serotype 2 vectors preferentially integrate into active genes in mice
    • Nakai H, Montini E, Fuess S, Storm TA, Grompe M, et al. (2003) AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat Genet 34: 297-302.
    • (2003) Nat Genet , vol.34 , pp. 297-302
    • Nakai, H.1    Montini, E.2    Fuess, S.3    Storm, T.A.4    Grompe, M.5
  • 55
    • 29844439691 scopus 로고    scopus 로고
    • Cellular distribution of insulin-like growth factor-II/mannose-6-phosphate receptor in normal human brain and its alteration in Alzheimer's disease pathology
    • Kar S, Poirier J, Guevara J, Dea D, Hawkes C, et al. (2006) Cellular distribution of insulin-like growth factor-II/mannose-6-phosphate receptor in normal human brain and its alteration in Alzheimer's disease pathology. Neurobiol Aging 27: 199-210.
    • (2006) Neurobiol Aging , vol.27 , pp. 199-210
    • Kar, S.1    Poirier, J.2    Guevara, J.3    Dea, D.4    Hawkes, C.5
  • 56
    • 0036460724 scopus 로고    scopus 로고
    • Insulin-like growth factor-II/Mannose-6-phosphate receptor in the spinal cord and dorsal root ganglia of the adult rat
    • Hawkes C, Kar S, (2002) Insulin-like growth factor-II/Mannose-6-phosphate receptor in the spinal cord and dorsal root ganglia of the adult rat. Eur J Neurosci 15: 33-39.
    • (2002) Eur J Neurosci , vol.15 , pp. 33-39
    • Hawkes, C.1    Kar, S.2
  • 58
    • 33747209013 scopus 로고    scopus 로고
    • A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter Syndrome)
    • Muenzer J, Wraith JE, Beck M, Guigliani R, Harmatz P, et al. (2006) A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter Syndrome). Genet Med 8: 465-473.
    • (2006) Genet Med , vol.8 , pp. 465-473
    • Muenzer, J.1    Wraith, J.E.2    Beck, M.3    Guigliani, R.4    Harmatz, P.5
  • 59
    • 0242409499 scopus 로고    scopus 로고
    • AMIDE: a free software tool for multimodality medical image analysis
    • Loening AM, Gambhir SS, (2003) AMIDE: a free software tool for multimodality medical image analysis. Mol Imaging 2: 131-137.
    • (2003) Mol Imaging , vol.2 , pp. 131-137
    • Loening, A.M.1    Gambhir, S.S.2
  • 60
    • 0028700063 scopus 로고
    • Accelerated image reconstruction using ordered subsets of projection data
    • Hudson HM, Larkin RS, (1994) Accelerated image reconstruction using ordered subsets of projection data. IEEE Trans Med Imaging 13: 601-609.
    • (1994) IEEE Trans Med Imaging , vol.13 , pp. 601-609
    • Hudson, H.M.1    Larkin, R.S.2
  • 61
    • 0031981253 scopus 로고    scopus 로고
    • High-resolution 3D Bayesian image reconstruction using the microPET small-animal scanner
    • Qi J, Leahy RM, Cherry SR, Chatziioannou A, Farquhar TH, (1998) High-resolution 3D Bayesian image reconstruction using the microPET small-animal scanner. Phys Med Biol 43: 1001-1013.
    • (1998) Phys Med Biol , vol.43 , pp. 1001-1013
    • Qi, J.1    Leahy, R.M.2    Cherry, S.R.3    Chatziioannou, A.4    Farquhar, T.H.5
  • 63
    • 84862957075 scopus 로고    scopus 로고
    • PET imaging of protein pharmacokinetics in the CSF of rats and monkeys with I-124
    • Papisov M, Belov V, Fischman A, Bonab A, Titus J, et al. (2010) PET imaging of protein pharmacokinetics in the CSF of rats and monkeys with I-124. J Nucl Med 51 (Suppl 2): 1751.
    • (2010) J Nucl Med , vol.51 , Issue.SUPPL. 2 , pp. 1751
    • Papisov, M.1    Belov, V.2    Fischman, A.3    Bonab, A.4    Titus, J.5
  • 64
    • 0035194407 scopus 로고    scopus 로고
    • A fluorimetric enzyme assay for the diagnosis of MPS II (Hunter disease)
    • Voznyi YV, Keulemans JL, van Diggelen OP, (2001) A fluorimetric enzyme assay for the diagnosis of MPS II (Hunter disease). J Inherit Metab Dis 24: 675-680.
    • (2001) J Inherit Metab Dis , vol.24 , pp. 675-680
    • Voznyi, Y.V.1    Keulemans, J.L.2    van Diggelen, O.P.3
  • 65
    • 37249043762 scopus 로고    scopus 로고
    • The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome)
    • Garcia AR, Pan J, Lamsa JC, Muenzer J, (2007) The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome). J Inherit Metab Dis 30: 924-934.
    • (2007) J Inherit Metab Dis , vol.30 , pp. 924-934
    • Garcia, A.R.1    Pan, J.2    Lamsa, J.C.3    Muenzer, J.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.