Cerebellar alterations and gait defects as therapeutic outcome measures for enzyme replacement therapy in alpha-mannosidosis

Damme M, Stroobants S, Walkley SU, Lüllmann-Rauch R, D'Hooge R, Fogh J, Saftig P, Lübke T, Blanz J (2011)
Journal of Neuropathology & Experimental Neurology 70(1): 83-94.

Zeitschriftenaufsatz | Veröffentlicht | Englisch
 
Download
Es wurden keine Dateien hochgeladen. Nur Publikationsnachweis!
DOI
Autor*in
Damme, MarkusUniBi; Stroobants, Stijn; Walkley, Steven U.; Lüllmann-Rauch, Renate; D'Hooge, Rudi; Fogh, Jens; Saftig, Paul; Lübke, TorbenUniBi ; Blanz, Judith
Einrichtung
Fakultät für Chemie > Biochemie I
Centrum für Biotechnologie > Institut für Biochemie und Biotechnik
Erscheinungsjahr
2011
Zeitschriftentitel
Journal of Neuropathology & Experimental Neurology
Band
70
Ausgabe
1
Seite(n)
83-94
ISSN
0022-3069
Page URI
https://pub.uni-bielefeld.de/record/1940515

Zitieren

Damme M, Stroobants S, Walkley SU, et al. Cerebellar alterations and gait defects as therapeutic outcome measures for enzyme replacement therapy in alpha-mannosidosis. Journal of Neuropathology & Experimental Neurology. 2011;70(1):83-94.
Damme, M., Stroobants, S., Walkley, S. U., Lüllmann-Rauch, R., D'Hooge, R., Fogh, J., Saftig, P., et al. (2011). Cerebellar alterations and gait defects as therapeutic outcome measures for enzyme replacement therapy in alpha-mannosidosis. Journal of Neuropathology & Experimental Neurology, 70(1), 83-94. https://doi.org/10.1097/NEN.0b013e31820428fa
Damme, Markus, Stroobants, Stijn, Walkley, Steven U., Lüllmann-Rauch, Renate, D'Hooge, Rudi, Fogh, Jens, Saftig, Paul, Lübke, Torben, and Blanz, Judith. 2011. “Cerebellar alterations and gait defects as therapeutic outcome measures for enzyme replacement therapy in alpha-mannosidosis”. Journal of Neuropathology & Experimental Neurology 70 (1): 83-94.
Damme, M., Stroobants, S., Walkley, S. U., Lüllmann-Rauch, R., D'Hooge, R., Fogh, J., Saftig, P., Lübke, T., and Blanz, J. (2011). Cerebellar alterations and gait defects as therapeutic outcome measures for enzyme replacement therapy in alpha-mannosidosis. Journal of Neuropathology & Experimental Neurology 70, 83-94.
Damme, M., et al., 2011. Cerebellar alterations and gait defects as therapeutic outcome measures for enzyme replacement therapy in alpha-mannosidosis. Journal of Neuropathology & Experimental Neurology, 70(1), p 83-94.
M. Damme, et al., “Cerebellar alterations and gait defects as therapeutic outcome measures for enzyme replacement therapy in alpha-mannosidosis”, Journal of Neuropathology & Experimental Neurology, vol. 70, 2011, pp. 83-94.
Damme, M., Stroobants, S., Walkley, S.U., Lüllmann-Rauch, R., D'Hooge, R., Fogh, J., Saftig, P., Lübke, T., Blanz, J.: Cerebellar alterations and gait defects as therapeutic outcome measures for enzyme replacement therapy in alpha-mannosidosis. Journal of Neuropathology & Experimental Neurology. 70, 83-94 (2011).
Damme, Markus, Stroobants, Stijn, Walkley, Steven U., Lüllmann-Rauch, Renate, D'Hooge, Rudi, Fogh, Jens, Saftig, Paul, Lübke, Torben, and Blanz, Judith. “Cerebellar alterations and gait defects as therapeutic outcome measures for enzyme replacement therapy in alpha-mannosidosis”. Journal of Neuropathology & Experimental Neurology 70.1 (2011): 83-94.

13 Zitationen in Europe PMC

Daten bereitgestellt von Europe PubMed Central.

Long-term enzyme replacement therapy improves neurocognitive functioning and hippocampal synaptic plasticity in immune-tolerant alpha-mannosidosis mice.
Stroobants S, Damme M, Van der Jeugd A, Vermaercke B, Andersson C, Fogh J, Saftig P, Blanz J, D'Hooge R., Neurobiol Dis 106(), 2017
PMID: 28720484
GM2 Gangliosidosis in Shiba Inu Dogs with an In-Frame Deletion in HEXB.
Kolicheski A, Johnson GS, Villani NA, O'Brien DP, Mhlanga-Mutangadura T, Wenger DA, Mikoloski K, Eagleson JS, Taylor JF, Schnabel RD, Katz ML., J Vet Intern Med 31(5), 2017
PMID: 28833537
Diffusion Tensor Imaging for Assessing Brain Gray and White Matter Abnormalities in a Feline Model of α-Mannosidosis.
Kumar M, Duda JT, Yoon SY, Bagel J, O'Donnell P, Vite C, Pickup S, Gee JC, Wolfe JH, Poptani H., J Neuropathol Exp Neurol 75(1), 2016
PMID: 26671987
A mouse model for fucosidosis recapitulates storage pathology and neurological features of the milder form of the human disease.
Wolf H, Damme M, Stroobants S, D'Hooge R, Beck HC, Hermans-Borgmeyer I, Lüllmann-Rauch R, Dierks T, Lübke T., Dis Model Mech 9(9), 2016
PMID: 27491075
Chronic enzyme replacement therapy ameliorates neuropathology in alpha-mannosidosis mice.
Damme M, Stroobants S, Lüdemann M, Rothaug M, Lüllmann-Rauch R, Beck HC, Ericsson A, Andersson C, Fogh J, D'Hooge R, Saftig P, Blanz J., Ann Clin Transl Neurol 2(11), 2015
PMID: 26817023
Distribution and Severity of Neuropathology in β-Mannosidase-Deficient Mice is Strain Dependent.
Lovell KL, Zhu M, Drummond MC, Switzer RC, Friderici KH., JIMD Rep 13(), 2014
PMID: 24142277
Fast urinary screening of oligosaccharidoses by MALDI-TOF/TOF mass spectrometry.
Bonesso L, Piraud M, Caruba C, Van Obberghen E, Mengual R, Hinault C., Orphanet J Rare Dis 9(), 2014
PMID: 24502792
Increased gait variability in mice with small cerebellar cortex lesions and normal rotarod performance.
Stroobants S, Gantois I, Pooters T, D'Hooge R., Behav Brain Res 241(), 2013
PMID: 23219967
Natural history of alpha mannosidosis a longitudinal study.
Beck M, Olsen KJ, Wraith JE, Zeman J, Michalski JC, Saftig P, Fogh J, Malm D., Orphanet J Rare Dis 8(), 2013
PMID: 23786919
Disruption of the autophagy-lysosome pathway is involved in neuropathology of the nclf mouse model of neuronal ceroid lipofuscinosis.
Thelen M, Damme M, Schweizer M, Hagel C, Wong AM, Cooper JD, Braulke T, Galliciotti G., PLoS One 7(4), 2012
PMID: 22536393
Arylsulfatase G inactivation causes loss of heparan sulfate 3-O-sulfatase activity and mucopolysaccharidosis in mice.
Kowalewski B, Lamanna WC, Lawrence R, Damme M, Stroobants S, Padva M, Kalus I, Frese MA, Lübke T, Lüllmann-Rauch R, D'Hooge R, Esko JD, Dierks T., Proc Natl Acad Sci U S A 109(26), 2012
PMID: 22689975
Lysosomal dysfunction causes neurodegeneration in mucolipidosis II 'knock-in' mice.
Kollmann K, Damme M, Markmann S, Morelle W, Schweizer M, Hermans-Borgmeyer I, Röchert AK, Pohl S, Lübke T, Michalski JC, Käkelä R, Walkley SU, Braulke T., Brain 135(pt 9), 2012
PMID: 22961545
Loss of Niemann-Pick C1 or C2 protein results in similar biochemical changes suggesting that these proteins function in a common lysosomal pathway.
Dixit SS, Jadot M, Sohar I, Sleat DE, Stock AM, Lobel P., PLoS One 6(8), 2011
PMID: 21887293

54 References

Daten bereitgestellt von Europe PubMed Central.

Secondary accumulation of gangliosides in lysosomal storage disorders.
Walkley SU., Semin. Cell Dev. Biol. 15(4), 2004
PMID: 15207833
Postnatal development of inflammation in a murine model of Niemann-Pick type C disease: immunohistochemical observations of microglia and astroglia.
Baudry M, Yao Y, Simmons D, Liu J, Bi X., Exp. Neurol. 184(2), 2003
PMID: 14769381
Neuronal localization and association of Niemann Pick C2 protein (HE1/NPC2) with the postsynaptic density.
Ong WY, Sundaram RK, Huang E, Ghoshal S, Kumar U, Pentchev PG, Patel SC., Neuroscience 128(3), 2004
PMID: 15381285
Deficiency of alpha-mannosidase in Angus cattle. An inherited lysosomal storage disease.
Hocking JD, Jolly RD, Batt RD., Biochem. J. 128(1), 1972
PMID: 4673577
Mannosidosis: patterns of storage and urinary excretion of oligosaccharides in the bovine model.
Jolly RD, Slack PM, Winter PJ, Murphy CE., Aust J Exp Biol Med Sci 58(4), 1980
PMID: 7436888
The catabolism of mammalian glycoproteins. Comparison of the storage products in bovine, feline and human mannosidosis.
Abraham D, Blakemore WF, Jolly RD, Sidebotham R, Winchester B., Biochem. J. 215(3), 1983
PMID: 6661184
An immunohistochemical study on the ontogeny of cells immunoreactive for spot 35 protein, a novel Purkinje cell-specific protein, in the rat cerebellum.
Takahashi-Iwanaga H, Kondo H, Yamakuni T, Takahashi Y., Brain Res. 394(2), 1986
PMID: 3533215
Lysosomal degradation of Asn-linked glycoproteins.
Aronson NN Jr, Kuranda MJ., FASEB J. 3(14), 1989
PMID: 2531691
MR findings in mannosidosis.
Dietemann JL, Filippi de la Palavesa MM, Tranchant C, Kastler B., Neuroradiology 32(6), 1990
PMID: 2287376
The substrate-specificity of human lysosomal alpha-D-mannosidase in relation to genetic alpha-mannosidosis.
al Daher S, de Gasperi R, Daniel P, Hall N, Warren CD, Winchester B., Biochem. J. 277 ( Pt 3)(), 1991
PMID: 1872811
Neuroaxonal dystrophy in neuronal storage disorders: evidence for major GABAergic neuron involvement.
Walkley SU, Baker HJ, Rattazzi MC, Haskins ME, Wu JY., J. Neurol. Sci. 104(1), 1991
PMID: 1919594
Ectopic dendrites occur only on cortical pyramidal cells containing elevated GM2 ganglioside in alpha-mannosidosis.
Goodman LA, Livingston PO, Walkley SU., Proc. Natl. Acad. Sci. U.S.A. 88(24), 1991
PMID: 1763046
A human lysosomal alpha(1----6)-mannosidase active on the branched trimannosyl core of complex glycans.
Daniel PF, Evans JE, De Gasperi R, Winchester B, Warren CD., Glycobiology 2(4), 1992
PMID: 1421754
Automatic analysis of altered gait in arylsulphatase A-deficient mice in the open field.
Leroy T, Stroobants S, Aerts JM, D'Hooge R, Berckmans D., Behav Res Methods 41(3), 2009
PMID: 19587193
Macrosialin, a mouse macrophage-restricted glycoprotein, is a member of the lamp/lgp family.
Holness CL, da Silva RP, Fawcett J, Gordon S, Simmons DL., J. Biol. Chem. 268(13), 1993
PMID: 8486654
Mammalian alpha-mannosidases--multiple forms but a common purpose?
Daniel PF, Winchester B, Warren CD., Glycobiology 4(5), 1994
PMID: 7881169
GM2 ganglioside and pyramidal neuron dendritogenesis.
Walkley SU, Siegel DA, Dobrenis K., Neurochem. Res. 20(11), 1995
PMID: 8786814
Neurology of adult alpha-mannosidosis.
Niemann S, Beck M, Seidel G, Spranger J, Vieregge P., J. Neurol. Neurosurg. Psychiatry 61(1), 1996
PMID: 8676143
Impaired motor skills on static and mobile beams in lurcher mutant mice.
Le Marec N, Caston J, Lalonde R., Exp Brain Res 116(1), 1997
PMID: 9305822
Reduction of lipofuscin-like autofluorescence in fluorescently labeled tissue.
Schnell SA, Staines WA, Wessendorf MW., J. Histochem. Cytochem. 47(6), 1999
PMID: 10330448
Targeted disruption of the lysosomal alpha-mannosidase gene results in mice resembling a mild form of human alpha-mannosidosis.
Stinchi S, Lullmann-Rauch R, Hartmann D, Coenen R, Beccari T, Orlacchio A, von Figura K, Saftig P., Hum. Mol. Genet. 8(8), 1999
PMID: 10400983
Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders.
McGlynn R, Dobrenis K, Walkley SU., J. Comp. Neurol. 480(4), 2004
PMID: 15558784
Lysosomal storage disorders.
Vellodi A., Br. J. Haematol. 128(4), 2005
PMID: 15686451
Neurocognitive and psychotiform behavioral alterations and enhanced hippocampal long-term potentiation in transgenic mice displaying neuropathological features of human alpha-mannosidosis.
D'Hooge R, Lullmann-Rauch R, Beckers T, Balschun D, Schwake M, Reiss K, von Figura K, Saftig P., J. Neurosci. 25(28), 2005
PMID: 16014715
Storage solutions: treating lysosomal disorders of the brain.
Jeyakumar M, Dwek RA, Butters TD, Platt FM., Nat. Rev. Neurosci. 6(9), 2005
PMID: 16049428
Gangliosides trigger inflammatory responses via TLR4 in brain glia.
Jou I, Lee JH, Park SY, Yoon HJ, Joe EH, Park EJ., Am. J. Pathol. 168(5), 2006
PMID: 16651628
A comparison analysis of hindlimb kinematics during overground and treadmill locomotion in rats.
Pereira JE, Cabrita AM, Filipe VM, Bulas-Cruz J, Couto PA, Melo-Pinto P, Costa LM, Geuna S, Mauricio AC, Varejao AS., Behav. Brain Res. 172(2), 2006
PMID: 16777243
Multivariate neurocognitive and emotional profile of a mannosidosis murine model for therapy assessment.
Caeyenberghs K, Balschun D, Roces DP, Schwake M, Saftig P, D'Hooge R., Neurobiol. Dis. 23(2), 2006
PMID: 16766199
Mechanism of cholesterol transfer from the Niemann-Pick type C2 protein to model membranes supports a role in lysosomal cholesterol transport.
Cheruku SR, Xu Z, Dutia R, Lobel P, Storch J., J. Biol. Chem. 281(42), 2006
PMID: 16606609
Systemic infections and inflammation affect chronic neurodegeneration.
Perry VH, Cunningham C, Holmes C., Nat. Rev. Immunol. 7(2), 2007
PMID: 17220915
Gait parameters of treadmill versus overground locomotion in mouse.
Herbin M, Hackert R, Gasc JP, Renous S., Behav. Brain Res. 181(2), 2007
PMID: 17521749
Developmental analysis of CNS pathology in the lysosomal storage disease alpha-mannosidosis.
Crawley AC, Walkley SU., J. Neuropathol. Exp. Neurol. 66(8), 2007
PMID: 17882013
Induction of tolerance to human arylsulfatase A in a mouse model of metachromatic leukodystrophy.
Matzner U, Matthes F, Herbst E, Lullmann-Rauch R, Callaerts-Vegh Z, D'Hooge R, Weigelt C, Eistrup C, Fogh J, Gieselmann V., Mol. Med. 13(9-10), 2007
PMID: 17660863
Behavioural characterisation of the alpha-mannosidosis guinea pig.
Robinson AJ, Crawley AC, Auclair D, Weston PF, Hirte C, Hemsley KM, Hopwood JJ., Behav. Brain Res. 186(2), 2007
PMID: 17889945
Contribution of TLR2 to the initiation of ganglioside-triggered inflammatory signaling.
Yoon HJ, Jeon SB, Suk K, Choi DK, Hong YJ, Park EJ., Mol. Cells 25(1), 2008
PMID: 18319620
Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis.
Chang M, Cooper JD, Sleat DE, Cheng SH, Dodge JC, Passini MA, Lobel P, Davidson BL., Mol. Ther. 16(4), 2008
PMID: 18362923
Early signs of neurolipidosis-related behavioural alterations in a murine model of metachromatic leukodystrophy.
Stroobants S, Leroy T, Eckhardt M, Aerts JM, Berckmans D, D'Hooge R., Behav. Brain Res. 189(2), 2008
PMID: 18336930
Alpha-mannosidosis.
Malm D, Nilssen O., Orphanet J Rare Dis 3(), 2008
PMID: 18651971
Reversal of peripheral and central neural storage and ataxia after recombinant enzyme replacement therapy in alpha-mannosidosis mice.
Blanz J, Stroobants S, Lullmann-Rauch R, Morelle W, Ludemann M, D'Hooge R, Reuterwall H, Michalski JC, Fogh J, Andersson C, Saftig P., Hum. Mol. Genet. 17(22), 2008
PMID: 18713755
GM1 induces p38 and microtubule dependent ramification of rat primary microglia in vitro.
Park JY, Kim HY, Jou I, Park SM., Brain Res. 1244(), 2008
PMID: 18930716
Enzyme replacement improves ataxic gait and central nervous system histopathology in a mouse model of metachromatic leukodystrophy.
Matzner U, Lullmann-Rauch R, Stroobants S, Andersson C, Weigelt C, Eistrup C, Fogh J, D'Hooge R, Gieselmann V., Mol. Ther. 17(4), 2009
PMID: 19174759
Cerebellar defects in a mouse model of juvenile neuronal ceroid lipofuscinosis.
Weimer JM, Benedict JW, Getty AL, Pontikis CC, Lim MJ, Cooper JD, Pearce DA., Brain Res. 1266(), 2009
PMID: 19230832
Impaired lysosomal trimming of N-linked oligosaccharides leads to hyperglycosylation of native lysosomal proteins in mice with alpha-mannosidosis.
Damme M, Morelle W, Schmidt B, Andersson C, Fogh J, Michalski JC, Lubke T., Mol. Cell. Biol. 30(1), 2010
PMID: 19884343
Cerebellar pathology and motor deficits in the palmitoyl protein thioesterase 1-deficient mouse.
Macauley SL, Wozniak DF, Kielar C, Tan Y, Cooper JD, Sands MS., Exp. Neurol. 217(1), 2009
PMID: 19416667
Alpha-mannosidosis in the guinea pig: a new animal model for lysosomal storage disorders.
Crawley AC, Jones MZ, Bonning LE, Finnie JW, Hopwood JJ., Pediatr. Res. 46(5), 1999
PMID: 10541310
Glycoprotein lysosomal storage disorders: alpha- and beta-mannosidosis, fucosidosis and alpha-N-acetylgalactosaminidase deficiency.
Michalski JC, Klein A., Biochim. Biophys. Acta 1455(2-3), 1999
PMID: 10571005
Treadmill performance of mice with cerebellar lesions: 2. Lurcher mutant mice.
Le Marec N, Lalonde R., Neurobiol Learn Mem 73(3), 2000
PMID: 10775492
Microglial activation precedes acute neurodegeneration in Sandhoff disease and is suppressed by bone marrow transplantation.
Wada R, Tifft CJ, Proia RL., Proc. Natl. Acad. Sci. U.S.A. 97(20), 2000
PMID: 11005868
Histopathology, electrodiagnostic testing, and magnetic resonance imaging show significant peripheral and central nervous system myelin abnormalities in the cat model of alpha-mannosidosis.
Vite CH, McGowan JC, Braund KG, Drobatz KJ, Glickson JD, Wolfe JH, Haskins ME., J. Neuropathol. Exp. Neurol. 60(8), 2001
PMID: 11487056
Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII.
Sands MS, Vogler CA, Ohlemiller KK, Roberts MS, Grubb JH, Levy B, Sly WS., J. Biol. Chem. 276(46), 2001
PMID: 11562370
Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB.
Ohmi K, Greenberg DS, Rajavel KS, Ryazantsev S, Li HH, Neufeld EF., Proc. Natl. Acad. Sci. U.S.A. 100(4), 2003
PMID: 12576554
Central nervous system inflammation is a hallmark of pathogenesis in mouse models of GM1 and GM2 gangliosidosis.
Jeyakumar M, Thomas R, Elliot-Smith E, Smith DA, van der Spoel AC, d'Azzo A, Perry VH, Butters TD, Dwek RA, Platt FM., Brain 126(Pt 4), 2003
PMID: 12615653
Neurobiology and cellular pathogenesis of glycolipid storage diseases.
Walkley SU., Philos. Trans. R. Soc. Lond., B, Biol. Sci. 358(1433), 2003
PMID: 12803923
Efficacy of enzyme replacement therapy in alpha-mannosidosis mice: a preclinical animal study.
Roces DP, Lullmann-Rauch R, Peng J, Balducci C, Andersson C, Tollersrud O, Fogh J, Orlacchio A, Beccari T, Saftig P, von Figura K., Hum. Mol. Genet. 13(18), 2004
PMID: 15269179
Export

Markieren/ Markierung löschen
Markierte Publikationen

Open Data PUB

Web of Science

Dieser Datensatz im Web of Science®
Quellen

PMID: 21157375
PubMed | Europe PMC

Suchen in

Google Scholar