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Jaarsma D., Haasdijk E.D., Grashorn J.A., Hawkins R., van Duijn W., Verspaget H.W., London J., and Holstege J.C. Human Cu/Zn superoxide dismutase (SOD1) overexpression in mice causes mitochondrial vacuolization, axonal degeneration, and premature motoneuron death and accelerates motoneuron disease in mice expressing a familial amyotrophic lateral sclerosis mutant SOD1. Neurobiol Dis 7 (2000) 623-643
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Gonzalez de Aguilar J.L., Echaniz-Laguna A., Fergani A., Rene F., Meininger V., Loeffler J.P., and Dupuis L. Amyotrophic lateral sclerosis: all roads lead to Rome. J Neurochem 101 (2007) 1153-1160
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Fischer L.R., Culver D.G., Tennant P., Davis A.A., Wang M., Castellano-Sanchez A., Khan J., Polak M.A., and Glass J.D. Amyotrophic lateral sclerosis is a distal axonopathy: evidence in mice and man. Exp Neurol 185 (2004) 232-240
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34147131311
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Inhibition of p38 mitogen activated protein kinase activation and mutant SOD1(G93A)-induced motor neuron death
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••], shows that the complete rescue of motor neuron cell bodies is not sufficient to delay significantly the pathology, strongly suggesting that NMJ destruction is the pathogenic event.
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••], shows that the complete rescue of motor neuron cell bodies is not sufficient to delay significantly the pathology, strongly suggesting that NMJ destruction is the pathogenic event.
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Accumulation of SOD1 mutants in postnatal motoneurons does not cause motoneuron pathology or motoneuron disease
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Lino M.M., Schneider C., and Caroni P. Accumulation of SOD1 mutants in postnatal motoneurons does not cause motoneuron pathology or motoneuron disease. J Neurosci 22 (2002) 4825-4832
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Neuron-specific expression of mutant superoxide dismutase 1 in transgenic mice does not lead to motor impairment
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Pramatarova A., Laganiere J., Roussel J., Brisebois K., and Rouleau G.A. Neuron-specific expression of mutant superoxide dismutase 1 in transgenic mice does not lead to motor impairment. J Neurosci 21 (2001) 3369-3374
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39849103473
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Neuron-specific expression of mutant superoxide dismutase is sufficient to induce amyotrophic lateral sclerosis in transgenic mice
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Jaarsma D., Teuling E., Haasdijk E.D., De Zeeuw C.I., and Hoogenraad C.C. Neuron-specific expression of mutant superoxide dismutase is sufficient to induce amyotrophic lateral sclerosis in transgenic mice. J Neurosci 28 (2008) 2075-2088
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Jaarsma, D.1
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16
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Onset and progression in inherited ALS determined by motor neurons and microglia
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Boillee S., Yamanaka K., Lobsiger C.S., Copeland N.G., Jenkins N.A., Kassiotis G., Kollias G., and Cleveland D.W. Onset and progression in inherited ALS determined by motor neurons and microglia. Science 312 (2006) 1389-1392
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Boillee, S.1
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Kassiotis, G.6
Kollias, G.7
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Clement A.M., Nguyen M.D., Roberts E.A., Garcia M.L., Boillee S., Rule M., McMahon A.P., Doucette W., Siwek D., Ferrante R.J., et al. Wild-type nonneuronal cells extend survival of SOD1 mutant motor neurons in ALS mice. Science 302 (2003) 113-117
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Siwek, D.9
Ferrante, R.J.10
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21
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39749188753
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Astrocytes as determinants of disease progression in inherited amyotrophic lateral sclerosis
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This work is the first in vivo demonstration of an active role of astrocytic mSOD1 expression in mSOD1-mediated pathogenesis.
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Yamanaka K., Chun S.J., Boillee S., Fujimori-Tonou N., Yamashita H., Gutmann D.H., Takahashi R., Misawa H., and Cleveland D.W. Astrocytes as determinants of disease progression in inherited amyotrophic lateral sclerosis. Nat Neurosci 11 (2008) 251-253. This work is the first in vivo demonstration of an active role of astrocytic mSOD1 expression in mSOD1-mediated pathogenesis.
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Yamanaka, K.1
Chun, S.J.2
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Yamashita, H.5
Gutmann, D.H.6
Takahashi, R.7
Misawa, H.8
Cleveland, D.W.9
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22
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33750478657
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Wild-type microglia extend survival in PU.1 knockout mice with familial amyotrophic lateral sclerosis
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Beers D.R., Henkel J.S., Xiao Q., Zhao W., Wang J., Yen A.A., Siklos L., McKercher S.R., and Appel S.H. Wild-type microglia extend survival in PU.1 knockout mice with familial amyotrophic lateral sclerosis. Proc Natl Acad Sci U S A 103 (2006) 16021-16026
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Proc Natl Acad Sci U S A
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Beers, D.R.1
Henkel, J.S.2
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Wang, J.5
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Siklos, L.7
McKercher, S.R.8
Appel, S.H.9
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23
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44349169012
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Systemic AAV6 delivery mediating RNA interference against SOD1: neuromuscular transduction does not alter disease progression in fALS mice
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The authors describe that knocking down mSOD1 in multiple tissues, including muscles, does not modify the pathogenesis, suggesting that decreasing mSOD1 expression in muscle might not be a valuable therapeutic strategy.
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Towne C., Raoul C., Schneider B.L., and Aebischer P. Systemic AAV6 delivery mediating RNA interference against SOD1: neuromuscular transduction does not alter disease progression in fALS mice. Mol Ther 16 (2008) 1018-1025. The authors describe that knocking down mSOD1 in multiple tissues, including muscles, does not modify the pathogenesis, suggesting that decreasing mSOD1 expression in muscle might not be a valuable therapeutic strategy.
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Mol Ther
, vol.16
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Towne, C.1
Raoul, C.2
Schneider, B.L.3
Aebischer, P.4
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24
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33845876647
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Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis
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Miller T.M., Kim S.H., Yamanaka K., Hester M., Umapathi P., Arnson H., Rizo L., Mendell J.R., Gage F.H., Cleveland D.W., et al. Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis. Proc Natl Acad Sci U S A 103 (2006) 19546-19551
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Proc Natl Acad Sci U S A
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Miller, T.M.1
Kim, S.H.2
Yamanaka, K.3
Hester, M.4
Umapathi, P.5
Arnson, H.6
Rizo, L.7
Mendell, J.R.8
Gage, F.H.9
Cleveland, D.W.10
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25
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20144389664
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Nogo expression in muscle correlates with amyotrophic lateral sclerosis severity
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Jokic N., Gonzalez de Aguilar J.L., Pradat P.F., Dupuis L., Echaniz-Laguna A., Muller A., Dubourg O., Seilhean D., Hauw J.J., Loeffler J.P., et al. Nogo expression in muscle correlates with amyotrophic lateral sclerosis severity. Ann Neurol 57 (2005) 553-556
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Ann Neurol
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Jokic, N.1
Gonzalez de Aguilar, J.L.2
Pradat, P.F.3
Dupuis, L.4
Echaniz-Laguna, A.5
Muller, A.6
Dubourg, O.7
Seilhean, D.8
Hauw, J.J.9
Loeffler, J.P.10
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26
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0036403767
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Nogo provides a molecular marker for diagnosis of amyotrophic lateral sclerosis
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Dupuis L., Gonzalez de Aguilar J.L., di Scala F., Rene F., de Tapia M., Pradat P.F., Lacomblez L., Seihlan D., Prinjha R., Walsh F.S., et al. Nogo provides a molecular marker for diagnosis of amyotrophic lateral sclerosis. Neurobiol Dis 10 (2002) 358-365
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Neurobiol Dis
, vol.10
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Dupuis, L.1
Gonzalez de Aguilar, J.L.2
di Scala, F.3
Rene, F.4
de Tapia, M.5
Pradat, P.F.6
Lacomblez, L.7
Seihlan, D.8
Prinjha, R.9
Walsh, F.S.10
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27
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34547765317
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Muscle Nogo-A expression is a prognostic marker in lower motor neuron syndromes
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Pradat P.F., Bruneteau G., Gonzalez de Aguilar J.L., Dupuis L., Jokic N., Salachas F., Le Forestier N., Echaniz-Laguna A., Dubourg O., Hauw J.J., et al. Muscle Nogo-A expression is a prognostic marker in lower motor neuron syndromes. Ann Neurol 62 (2007) 15-20
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Ann Neurol
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Pradat, P.F.1
Bruneteau, G.2
Gonzalez de Aguilar, J.L.3
Dupuis, L.4
Jokic, N.5
Salachas, F.6
Le Forestier, N.7
Echaniz-Laguna, A.8
Dubourg, O.9
Hauw, J.J.10
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28
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33750527780
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The neurite outgrowth inhibitor Nogo-A promotes denervation in an amyotrophic lateral sclerosis model
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Jokic N., Gonzalez de Aguilar J.L., Dimou L., Lin S., Fergani A., Ruegg M.A., Schwab M.E., Dupuis L., and Loeffler J.P. The neurite outgrowth inhibitor Nogo-A promotes denervation in an amyotrophic lateral sclerosis model. EMBO Rep 7 (2006) 1162-1167
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EMBO Rep
, vol.7
, pp. 1162-1167
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Jokic, N.1
Gonzalez de Aguilar, J.L.2
Dimou, L.3
Lin, S.4
Fergani, A.5
Ruegg, M.A.6
Schwab, M.E.7
Dupuis, L.8
Loeffler, J.P.9
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29
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13844253540
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Muscle expression of a local Igf-1 isoform protects motor neurons in an ALS mouse model
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Dobrowolny G., Giacinti C., Pelosi L., Nicoletti C., Winn N., Barberi L., Molinaro M., Rosenthal N., and Musaro A. Muscle expression of a local Igf-1 isoform protects motor neurons in an ALS mouse model. J Cell Biol 168 (2005) 193-199
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J Cell Biol
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Dobrowolny, G.1
Giacinti, C.2
Pelosi, L.3
Nicoletti, C.4
Winn, N.5
Barberi, L.6
Molinaro, M.7
Rosenthal, N.8
Musaro, A.9
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30
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54849404282
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Skeletal muscle is a primary target of SOD1G93A-mediated toxicity
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This is the first demonstration of an active role of skeletal muscle mSOD1 expression in mSOD1-mediated pathogenesis.
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Dobrowolny G., Aucello M., Rizzuto E., Beccafico S., Mammucari C., Bonconpagni S., Belia S., Wannenes F., Nicoletti C., Del Prete Z., et al. Skeletal muscle is a primary target of SOD1G93A-mediated toxicity. Cell Metab 8 (2008) 425-436. This is the first demonstration of an active role of skeletal muscle mSOD1 expression in mSOD1-mediated pathogenesis.
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Cell Metab
, vol.8
, pp. 425-436
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Dobrowolny, G.1
Aucello, M.2
Rizzuto, E.3
Beccafico, S.4
Mammucari, C.5
Bonconpagni, S.6
Belia, S.7
Wannenes, F.8
Nicoletti, C.9
Del Prete, Z.10
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31
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3343020674
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Evidence for defective energy homeostasis in amyotrophic lateral sclerosis: benefit of a high-energy diet in a transgenic mouse model
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Dupuis L., Oudart H., Rene F., Gonzalez de Aguilar J.L., and Loeffler J.P. Evidence for defective energy homeostasis in amyotrophic lateral sclerosis: benefit of a high-energy diet in a transgenic mouse model. Proc Natl Acad Sci U S A 101 (2004) 11159-11164
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Proc Natl Acad Sci U S A
, vol.101
, pp. 11159-11164
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Dupuis, L.1
Oudart, H.2
Rene, F.3
Gonzalez de Aguilar, J.L.4
Loeffler, J.P.5
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33
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34548145662
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Increased peripheral lipid clearance in an animal model of amyotrophic lateral sclerosis
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Fergani A., Oudart H., Gonzalez De Aguilar J.L., Fricker B., Rene F., Hocquette J.F., Meininger V., Dupuis L., and Loeffler J.P. Increased peripheral lipid clearance in an animal model of amyotrophic lateral sclerosis. J Lipid Res 48 (2007) 1571-1580
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(2007)
J Lipid Res
, vol.48
, pp. 1571-1580
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Fergani, A.1
Oudart, H.2
Gonzalez De Aguilar, J.L.3
Fricker, B.4
Rene, F.5
Hocquette, J.F.6
Meininger, V.7
Dupuis, L.8
Loeffler, J.P.9
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34
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41349122697
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Dyslipidemia is a protective factor in amyotrophic lateral sclerosis
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This paper describes systemic alterations of lipidemia in ALS patients and their correlation with survival.
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Dupuis L., Corcia P., Fergani A., Gonzalez De Aguilar J.L., Bonnefont-Roussel D., Bittar R., Seilhean D., Hauw J.J., Lacomblez L., Loeffler J.P., et al. Dyslipidemia is a protective factor in amyotrophic lateral sclerosis. Neurology 70 (2008) 1004-1009. This paper describes systemic alterations of lipidemia in ALS patients and their correlation with survival.
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(2008)
Neurology
, vol.70
, pp. 1004-1009
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Dupuis, L.1
Corcia, P.2
Fergani, A.3
Gonzalez De Aguilar, J.L.4
Bonnefont-Roussel, D.5
Bittar, R.6
Seilhean, D.7
Hauw, J.J.8
Lacomblez, L.9
Loeffler, J.P.10
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35
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33947323759
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Lost in translation: treatment trials in the SOD1 mouse and in human ALS
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Benatar M. Lost in translation: treatment trials in the SOD1 mouse and in human ALS. Neurobiol Dis 26 (2007) 1-13
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(2007)
Neurobiol Dis
, vol.26
, pp. 1-13
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Benatar, M.1
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36
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39349107014
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Design, power, and interpretation of studies in the standard murine model of ALS
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The authors failed to replicate a number of published positive effects in mSOD1 mice and conclude that most of these effects are experimental noise.
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Scott S., Kranz J.E., Cole J., Lincecum J.M., Thompson K., Kelly N., Bostrom A., Theodoss J., Al-Nakhala B.M., Vieira F.G., et al. Design, power, and interpretation of studies in the standard murine model of ALS. Amyotroph Lateral Scler 9 (2008) 4-15. The authors failed to replicate a number of published positive effects in mSOD1 mice and conclude that most of these effects are experimental noise.
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(2008)
Amyotroph Lateral Scler
, vol.9
, pp. 4-15
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Scott, S.1
Kranz, J.E.2
Cole, J.3
Lincecum, J.M.4
Thompson, K.5
Kelly, N.6
Bostrom, A.7
Theodoss, J.8
Al-Nakhala, B.M.9
Vieira, F.G.10
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37
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34547587021
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Guidelines for the preclinical in vivo evaluation of pharmacological active drugs for ALS/MND: report on the 142nd ENMC international workshop
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This consortium of ALS researchers set up guidelines in establishing rigorous preclinical trials in mSOD1 mice.
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Ludolph A.C., Bendotti C., Blaugrund E., Hengerer B., Loffler J.P., Martin J., Meininger V., Meyer T., Moussaoui S., Robberecht W., et al. Guidelines for the preclinical in vivo evaluation of pharmacological active drugs for ALS/MND: report on the 142nd ENMC international workshop. Amyotroph Lateral Scler 8 (2007) 217-223. This consortium of ALS researchers set up guidelines in establishing rigorous preclinical trials in mSOD1 mice.
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Amyotroph Lateral Scler
, vol.8
, pp. 217-223
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Ludolph, A.C.1
Bendotti, C.2
Blaugrund, E.3
Hengerer, B.4
Loffler, J.P.5
Martin, J.6
Meininger, V.7
Meyer, T.8
Moussaoui, S.9
Robberecht, W.10
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