메뉴 건너뛰기




Volumn 263, Issue , 2015, Pages 79-90

Delivery of therapeutic protein for prevention of neurodegenerative changes: Comparison of different CSF-delivery methods

Author keywords

Canine; Cerebrospinal fluid; Lysosomal storage disorder; Neurodegenerative disease; Recombinant protein; Treatment

Indexed keywords

GANGLIOSIDE; GLUTAMATE DECARBOXYLASE; RECOMBINANT PROTEIN; SULFAMIDASE; UNCLASSIFIED DRUG; HEPARAN SULFATE; HYDROLASE; N-SULFOGLUCOSAMINE SULFOHYDROLASE;

EID: 84908011876     PISSN: 00144886     EISSN: 10902430     Source Type: Journal    
DOI: 10.1016/j.expneurol.2014.09.008     Document Type: Article
Times cited : (26)

References (52)
  • 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, 99:132-141.
    • (2010) Mol. Genet. Metab. , vol.99 , pp. 132-141
    • 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
  • 5
    • 84862940707 scopus 로고    scopus 로고
    • CNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorder
    • Calias P., Papisov M., Pan J., Savioli N., Belov V., Huang Y., Lotterhand J., Alessandrini M., Liu N., Fischman A.J., et al. CNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorder. PLoS One 2012, 7:e30341.
    • (2012) PLoS One , vol.7 , pp. e30341
    • Calias, P.1    Papisov, M.2    Pan, J.3    Savioli, N.4    Belov, V.5    Huang, Y.6    Lotterhand, J.7    Alessandrini, M.8    Liu, N.9    Fischman, A.J.10
  • 6
    • 0018825881 scopus 로고
    • Sanfilippo A disease in the fetus-comparison with pre- and postnatal cases
    • Ceuterick C., Martin J.J., Libert J., Farriaux J.P. Sanfilippo A disease in the fetus-comparison with pre- and postnatal cases. Neuropadiatrie 1980, 11:176-185.
    • (1980) Neuropadiatrie , vol.11 , pp. 176-185
    • Ceuterick, C.1    Martin, J.J.2    Libert, J.3    Farriaux, J.P.4
  • 7
    • 41149092265 scopus 로고    scopus 로고
    • Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis
    • Chang M., Cooper J.D., Sleat D.E., Cheng S.H., Dodge J.C., Passini M.A., Lobel P., Davidson B.L. Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis. Mol. Ther. 2008, 16:649-656.
    • (2008) Mol. Ther. , vol.16 , pp. 649-656
    • Chang, M.1    Cooper, J.D.2    Sleat, D.E.3    Cheng, S.H.4    Dodge, J.C.5    Passini, M.A.6    Lobel, P.7    Davidson, B.L.8
  • 13
    • 79956259719 scopus 로고    scopus 로고
    • Intrathecal enzyme replacement therapy for mucopolysaccharidosis I: translating success in animal models to patients
    • Dickson P.I., Chen A.H. Intrathecal enzyme replacement therapy for mucopolysaccharidosis I: translating success in animal models to patients. Curr. Pharm. Biotechnol. 2011, 12:946-955.
    • (2011) Curr. Pharm. Biotechnol. , vol.12 , pp. 946-955
    • Dickson, P.I.1    Chen, A.H.2
  • 15
    • 70449158340 scopus 로고
    • A simple method for the isolation and purification of total lipids from animal tissues
    • Folch J., Lees M., Sloane Stanley G.H. A simple method for the isolation and purification of total lipids from animal tissues. J. Biol. Chem. 1957, 226:497-509.
    • (1957) J. Biol. Chem. , vol.226 , pp. 497-509
    • Folch, J.1    Lees, M.2    Sloane Stanley, G.H.3
  • 16
    • 35548941400 scopus 로고    scopus 로고
    • Functional correction of CNS lesions in a MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes
    • Fraldi A., Hemsley K., Crawley A., Lombardi A., Lau A., Sutherland L., Auricchio A., Ballabio A., Hopwood J. Functional correction of CNS lesions in a MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes. Hum. Mol. Genet. 2007, 16:2693-2702.
    • (2007) Hum. Mol. Genet. , vol.16 , pp. 2693-2702
    • Fraldi, A.1    Hemsley, K.2    Crawley, A.3    Lombardi, A.4    Lau, A.5    Sutherland, L.6    Auricchio, A.7    Ballabio, A.8    Hopwood, J.9
  • 17
    • 3042647298 scopus 로고    scopus 로고
    • Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice
    • Gliddon B.L., Hopwood J.J. Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice. Pediatr. Res. 2004, 56:65-72.
    • (2004) Pediatr. Res. , vol.56 , pp. 65-72
    • Gliddon, B.L.1    Hopwood, J.J.2
  • 19
    • 33846904009 scopus 로고    scopus 로고
    • Injection of recombinant human sulfamidase into the CSF via the cerebellomedullary cistern in MPS IIIA mice
    • 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, 90:313-328.
    • (2007) Mol. Genet. Metab. , vol.90 , pp. 313-328
    • Hemsley, K.M.1    King, B.2    Hopwood, J.J.3
  • 20
    • 52949100951 scopus 로고    scopus 로고
    • Effect of high dose, repeated intra-CSF injection of sulphamidase on neuropathology in MPS IIIA mice
    • Hemsley K.M., Beard H., King B.M., Hopwood J.J. Effect of high dose, repeated intra-CSF injection of sulphamidase on neuropathology in MPS IIIA mice. Genes Brain Behav. 2008, 7:740-753.
    • (2008) Genes Brain Behav. , vol.7 , pp. 740-753
    • Hemsley, K.M.1    Beard, H.2    King, B.M.3    Hopwood, J.J.4
  • 23
    • 0020030423 scopus 로고
    • Diagnosis of Sanfilippo type A syndrome by estimation of sulfamidase activity using a radiolabelled tetrasaccharide substrate
    • Hopwood J.J., Elliott H. Diagnosis of Sanfilippo type A syndrome by estimation of sulfamidase activity using a radiolabelled tetrasaccharide substrate. Clin. Chim. Acta 1982, 123:241-250.
    • (1982) Clin. Chim. Acta , vol.123 , pp. 241-250
    • Hopwood, J.J.1    Elliott, H.2
  • 24
    • 58149492631 scopus 로고    scopus 로고
    • Targeted analysis of ganglioside and sulphatide molecular species by LC/ESI-MS/MS with theoretically expanded multiple reaction monitoring
    • Ikeda K., Shimizu T., Taguchi R. Targeted analysis of ganglioside and sulphatide molecular species by LC/ESI-MS/MS with theoretically expanded multiple reaction monitoring. J. Lipid Res. 2008, 49:2678-2689.
    • (2008) J. Lipid Res. , vol.49 , pp. 2678-2689
    • Ikeda, K.1    Shimizu, T.2    Taguchi, R.3
  • 25
  • 27
    • 84883652395 scopus 로고    scopus 로고
    • Intracisternal enzyme replacement therapy in lysosomal storage diseases: dispersal pathways, regional enzyme concentrations and the effect of post-treatment posture
    • Jolly R., Marshall N., Marshall J., Hartman A., Hemsley K., Winner L. Intracisternal enzyme replacement therapy in lysosomal storage diseases: dispersal pathways, regional enzyme concentrations and the effect of post-treatment posture. Neuropathol. Appl. Neurobiol. 2013, 39:681-692.
    • (2013) Neuropathol. Appl. Neurobiol. , vol.39 , pp. 681-692
    • Jolly, R.1    Marshall, N.2    Marshall, J.3    Hartman, A.4    Hemsley, K.5    Winner, L.6
  • 31
    • 0347301644 scopus 로고    scopus 로고
    • In vitro characterization of genetically modified embryonic stem cells as a therapy for murine mucopolysaccharidosis type IIIA
    • Lau A.A., Hemsley K.M., Meedeniya A., Hopwood J.J. In vitro characterization of genetically modified embryonic stem cells as a therapy for murine mucopolysaccharidosis type IIIA. Mol. Genet. Metab. 2004, 81:86-95.
    • (2004) Mol. Genet. Metab. , vol.81 , pp. 86-95
    • Lau, A.A.1    Hemsley, K.M.2    Meedeniya, A.3    Hopwood, J.J.4
  • 32
    • 77951976097 scopus 로고    scopus 로고
    • SGSH gene transfer in mucopolysaccharidosis type IIIA mice using canine adenovirus vectors
    • Lau A.A., Hopwood J.J., Kremer E.J., Hemsley K.M. SGSH gene transfer in mucopolysaccharidosis type IIIA mice using canine adenovirus vectors. Mol. Genet. Metab. 2010, 100:168-175.
    • (2010) Mol. Genet. Metab. , vol.100 , pp. 168-175
    • Lau, A.A.1    Hopwood, J.J.2    Kremer, E.J.3    Hemsley, K.M.4
  • 33
    • 77956428075 scopus 로고    scopus 로고
    • Allogeneic stem cell transplantation does not improve neurological deficits in mucopolysaccharidosis type IIIA mice
    • Lau A.A., Hannouche H., Rozaklis T., Hassiotis S., Hopwood J.J., Hemsley K.M. Allogeneic stem cell transplantation does not improve neurological deficits in mucopolysaccharidosis type IIIA mice. Exp. Neurol. 2010, 225:445-454.
    • (2010) Exp. Neurol. , vol.225 , pp. 445-454
    • Lau, A.A.1    Hannouche, H.2    Rozaklis, T.3    Hassiotis, S.4    Hopwood, J.J.5    Hemsley, K.M.6
  • 34
    • 34547747963 scopus 로고    scopus 로고
    • Single-dose intracerebroventricular administration of galactocerebrosidase improves survival in a mouse model of globoid cell leukodystrophy
    • Lee W.C., Tsoi Y.K., Troendle F.J., DeLucia M.W., Ahmed Z., Dicky C.A., Dickson D.W., Eckman C.B. Single-dose intracerebroventricular administration of galactocerebrosidase improves survival in a mouse model of globoid cell leukodystrophy. FASEB J. 2007, 21:2520-2527.
    • (2007) FASEB J. , vol.21 , pp. 2520-2527
    • Lee, W.C.1    Tsoi, Y.K.2    Troendle, F.J.3    DeLucia, M.W.4    Ahmed, Z.5    Dicky, C.A.6    Dickson, D.W.7    Eckman, C.B.8
  • 36
    • 79957890647 scopus 로고    scopus 로고
    • Therapeutic potential of intracerebroventricular replacement of modified human beta-hexosaminidase B for GM2 gangliosidosis
    • Matsuoka K., Tamura T., Tsuji D., Dohzono Y., Kitakaze K., Ohno K., Saito S., Sakuraba H., Itoh K. Therapeutic potential of intracerebroventricular replacement of modified human beta-hexosaminidase B for GM2 gangliosidosis. Mol. Ther. 2011, 19:1017-1024.
    • (2011) Mol. Ther. , vol.19 , pp. 1017-1024
    • Matsuoka, K.1    Tamura, T.2    Tsuji, D.3    Dohzono, Y.4    Kitakaze, K.5    Ohno, K.6    Saito, S.7    Sakuraba, H.8    Itoh, K.9
  • 37
    • 9744229206 scopus 로고    scopus 로고
    • Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders
    • McGlynn R., Dobrenis K., Walkley S.U. Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders. J. Comp. Neurol. 2004, 480:415-426.
    • (2004) J. Comp. Neurol. , vol.480 , pp. 415-426
    • McGlynn, R.1    Dobrenis, K.2    Walkley, S.U.3
  • 40
    • 34548411851 scopus 로고    scopus 로고
    • Improvement in behaviour after substrate deprivation therapy with rhodamine B in a mouse model of MPS IIIA
    • Roberts A.L., Rees M.H., Klebe S., Fletcher J.M., Byers S. Improvement in behaviour after substrate deprivation therapy with rhodamine B in a mouse model of MPS IIIA. Mol. Genet. Metab. 2007, 92:115-121.
    • (2007) Mol. Genet. Metab. , vol.92 , pp. 115-121
    • Roberts, A.L.1    Rees, M.H.2    Klebe, S.3    Fletcher, J.M.4    Byers, S.5
  • 42
    • 84871243601 scopus 로고    scopus 로고
    • Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPS IIIA mice through systemic AAV9 gene transfer
    • Ruzo A., Marcó S., García M., Villacampa P., Ribera A., Ayuso E., Maggioni L., Mingozzi F., Haurigot V., Bosch F. Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPS IIIA mice through systemic AAV9 gene transfer. Hum. Gene Ther. 2012, 23:1237-1246.
    • (2012) Hum. Gene Ther. , vol.23 , pp. 1237-1246
    • Ruzo, A.1    Marcó, S.2    García, M.3    Villacampa, P.4    Ribera, A.5    Ayuso, E.6    Maggioni, L.7    Mingozzi, F.8    Haurigot, V.9    Bosch, F.10
  • 45
    • 79959813959 scopus 로고    scopus 로고
    • Intracerebroventricular enzyme infusion corrects central nervous system pathology and dysfunction in a mouse model of metachromatic leukodystrophy
    • Stroobants S., Gerlach D., Matthes F., Hartmann D., Fogh J., Gieselmann V., D'Hooge R., Matzner U. Intracerebroventricular enzyme infusion corrects central nervous system pathology and dysfunction in a mouse model of metachromatic leukodystrophy. Hum. Mol. Genet. 2011, 20:2760-2769.
    • (2011) Hum. Mol. Genet. , vol.20 , pp. 2760-2769
    • Stroobants, S.1    Gerlach, D.2    Matthes, F.3    Hartmann, D.4    Fogh, J.5    Gieselmann, V.6    D'Hooge, R.7    Matzner, U.8
  • 46
    • 84896106760 scopus 로고    scopus 로고
    • A simple method for early age phenotype confirmation using toe tissue from a mouse model of MPS IIIA
    • Trim P.J., Lau A.A., Hopwood J.J., Snel M.F. A simple method for early age phenotype confirmation using toe tissue from a mouse model of MPS IIIA. Rapid Commun. Mass Spectrom. 2014, 28:933-938.
    • (2014) Rapid Commun. Mass Spectrom. , vol.28 , pp. 933-938
    • Trim, P.J.1    Lau, A.A.2    Hopwood, J.J.3    Snel, M.F.4
  • 49
    • 79958064659 scopus 로고    scopus 로고
    • Biodistribution and pharmacodynamics of recombinant human alpha-L-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations
    • Vite C.H., Wang P., Patel R.T., Walton R.M., Walkley S.U., Sellers R.S., Ellinwood N.M., Cheng A.S., White J.T., O'Neill C.A., et al. Biodistribution and pharmacodynamics of recombinant human alpha-L-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations. Mol. Genet. Metab. 2011, 103:268-274.
    • (2011) Mol. Genet. Metab. , vol.103 , pp. 268-274
    • Vite, C.H.1    Wang, P.2    Patel, R.T.3    Walton, R.M.4    Walkley, S.U.5    Sellers, R.S.6    Ellinwood, N.M.7    Cheng, A.S.8    White, J.T.9    O'Neill, C.A.10
  • 50
    • 0031667073 scopus 로고    scopus 로고
    • Method for long-term cerebrospinal fluid collection in the conscious dog
    • Wilsson-Rahmberg M., Olovson S.G., Forshult E. Method for long-term cerebrospinal fluid collection in the conscious dog. J. Investig. Surg. 1998, 11:207-214.
    • (1998) J. Investig. Surg. , vol.11 , pp. 207-214
    • Wilsson-Rahmberg, M.1    Olovson, S.G.2    Forshult, E.3
  • 51
    • 34548801983 scopus 로고    scopus 로고
    • Intraparenchymal injections of acid sphingomyelinase results in regional correction of lysosomal storage pathology in the Niemann-Pick A mouse
    • Yang W.W., Dodge J.C., Passini M.A., Taksir T.V., Griffiths D., Schuchman E.H., Cheng S.H., Shihabuddin L.S. Intraparenchymal injections of acid sphingomyelinase results in regional correction of lysosomal storage pathology in the Niemann-Pick A mouse. Exp. Neurol. 2007, 207:258-626.
    • (2007) Exp. Neurol. , vol.207 , pp. 258-626
    • Yang, W.W.1    Dodge, J.C.2    Passini, M.A.3    Taksir, T.V.4    Griffiths, D.5    Schuchman, E.H.6    Cheng, S.H.7    Shihabuddin, L.S.8
  • 52
    • 0035708854 scopus 로고    scopus 로고
    • Identification of a mutation causing mucopolysaccharidosis type IIIA in New Zealand Huntaway dogs
    • Yogalingam G., Pollard T., Gliddon B., Jolly R.D., Hopwood J.J. Identification of a mutation causing mucopolysaccharidosis type IIIA in New Zealand Huntaway dogs. Genomics 2002, 79:150-153.
    • (2002) Genomics , vol.79 , pp. 150-153
    • Yogalingam, G.1    Pollard, T.2    Gliddon, B.3    Jolly, R.D.4    Hopwood, J.J.5


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.