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Volumn 13, Issue 4, 2017, Pages

Bioenergetic status modulates motor neuron vulnerability and pathogenesis in a zebrafish model of spinal muscular atrophy

Author keywords

[No Author keywords available]

Indexed keywords

PHOSPHOGLYCERATE KINASE; PHOSPHOGLYCERATE KINASE 1; TERAZOSIN; UNCLASSIFIED DRUG; ADENOSINE TRIPHOSPHATE; PGK1 PROTEIN, MOUSE; PRAZOSIN; SMN1 PROTEIN, ZEBRAFISH; SURVIVAL MOTOR NEURON PROTEIN 1;

EID: 85018371495     PISSN: 15537390     EISSN: 15537404     Source Type: Journal    
DOI: 10.1371/journal.pgen.1006744     Document Type: Article
Times cited : (75)

References (82)
  • 1
    • 84859816485 scopus 로고    scopus 로고
    • Childhood spinal muscular atrophy: controversies and challenges
    • 22516079
    • Mercuri E, Bertini E, Iannaccone ST, Childhood spinal muscular atrophy: controversies and challenges. Lancet Neurol. 2012;11(5):443–52. doi: 10.1016/S1474-4422(12)70061-3 22516079
    • (2012) Lancet Neurol , vol.11 , Issue.5 , pp. 443-452
    • Mercuri, E.1    Bertini, E.2    Iannaccone, S.T.3
  • 2
    • 0028797783 scopus 로고
    • Identification and characterization of a spinal muscular atrophy-determining gene
    • 7813012
    • Lefebvre S, Burglen L, Reboullet S, Clermont O, Burlet P, Viollet L, et al. Identification and characterization of a spinal muscular atrophy-determining gene. Cell. 1995;80(1):155–65. 7813012
    • (1995) Cell , vol.80 , Issue.1 , pp. 155-165
    • Lefebvre, S.1    Burglen, L.2    Reboullet, S.3    Clermont, O.4    Burlet, P.5    Viollet, L.6
  • 3
    • 0030931720 scopus 로고    scopus 로고
    • Inactivation of the survival motor neuron gene, a candidate gene for human spinal muscular atrophy, leads to massive cell death in early mouse embryos
    • 9275227
    • Schrank B, Gotz R, Gunnersen JM, Ure JM, Toyka KV, Smith AG, et al. Inactivation of the survival motor neuron gene, a candidate gene for human spinal muscular atrophy, leads to massive cell death in early mouse embryos. Proc Natl Acad Sci U S A. 1997;94(18):9920–5. 9275227
    • (1997) Proc Natl Acad Sci U S A , vol.94 , Issue.18 , pp. 9920-9925
    • Schrank, B.1    Gotz, R.2    Gunnersen, J.M.3    Ure, J.M.4    Toyka, K.V.5    Smith, A.G.6
  • 4
    • 0033033434 scopus 로고    scopus 로고
    • A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy
    • 10339583
    • Lorson CL, Hahnen E, Androphy EJ, Wirth B, A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy. Proc Natl Acad Sci U S A. 1999;96(11):6307–11. 10339583
    • (1999) Proc Natl Acad Sci U S A , vol.96 , Issue.11 , pp. 6307-6311
    • Lorson, C.L.1    Hahnen, E.2    Androphy, E.J.3    Wirth, B.4
  • 5
    • 0032799998 scopus 로고    scopus 로고
    • A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2
    • 10369862
    • Monani UR, Lorson CL, Parsons DW, Prior TW, Androphy EJ, Burghes AH, et al. A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2. Hum Mol Genet. 1999;8(7):1177–83. 10369862
    • (1999) Hum Mol Genet , vol.8 , Issue.7 , pp. 1177-1183
    • Monani, U.R.1    Lorson, C.L.2    Parsons, D.W.3    Prior, T.W.4    Androphy, E.J.5    Burghes, A.H.6
  • 6
    • 0031734722 scopus 로고    scopus 로고
    • Correlation of SMNt and SMNc gene copy number with age of onset and survival in spinal muscular atrophy
    • 9801871
    • Taylor JE, Thomas NH, Lewis CM, Abbs SJ, Rodrigues NR, Davies KE, et al. Correlation of SMNt and SMNc gene copy number with age of onset and survival in spinal muscular atrophy. Eur J Hum Genet. 1998;6(5):467–74. doi: 10.1038/sj.ejhg.5200210 9801871
    • (1998) Eur J Hum Genet , vol.6 , Issue.5 , pp. 467-474
    • Taylor, J.E.1    Thomas, N.H.2    Lewis, C.M.3    Abbs, S.J.4    Rodrigues, N.R.5    Davies, K.E.6
  • 7
    • 0034639645 scopus 로고    scopus 로고
    • The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn(-/-) mice and results in a mouse with spinal muscular atrophy
    • 10655541
    • Monani UR, Sendtner M, Coovert DD, Parsons DW, Andreassi C, Le TT, et al. The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn(-/-) mice and results in a mouse with spinal muscular atrophy. Hum Mol Genet. 2000;9(3):333–9. 10655541
    • (2000) Hum Mol Genet , vol.9 , Issue.3 , pp. 333-339
    • Monani, U.R.1    Sendtner, M.2    Coovert, D.D.3    Parsons, D.W.4    Andreassi, C.5    Le, T.T.6
  • 8
    • 0030928716 scopus 로고    scopus 로고
    • The SMN-SIP1 complex has an essential role in spliceosomal snRNP biogenesis
    • 9323130
    • Fischer U, Liu Q, Dreyfuss G, The SMN-SIP1 complex has an essential role in spliceosomal snRNP biogenesis. Cell. 1997;90(6):1023–9. 9323130
    • (1997) Cell , vol.90 , Issue.6 , pp. 1023-1029
    • Fischer, U.1    Liu, Q.2    Dreyfuss, G.3
  • 9
    • 0030931727 scopus 로고    scopus 로고
    • The spinal muscular atrophy disease gene product, SMN, and its associated protein SIP1 are in a complex with spliceosomal snRNP proteins
    • 9323129
    • Liu Q, Fischer U, Wang F, Dreyfuss G, The spinal muscular atrophy disease gene product, SMN, and its associated protein SIP1 are in a complex with spliceosomal snRNP proteins. Cell. 1997;90(6):1013–21. 9323129
    • (1997) Cell , vol.90 , Issue.6 , pp. 1013-1021
    • Liu, Q.1    Fischer, U.2    Wang, F.3    Dreyfuss, G.4
  • 10
    • 0036724532 scopus 로고    scopus 로고
    • The SMN complex is associated with snRNPs throughout their cytoplasmic assembly pathway
    • 12192051
    • Massenet S, Pellizzoni L, Paushkin S, Mattaj IW, Dreyfuss G, The SMN complex is associated with snRNPs throughout their cytoplasmic assembly pathway. Mol Cell Biol. 2002;22(18):6533–41. doi: 10.1128/MCB.22.18.6533-6541.2002 12192051
    • (2002) Mol Cell Biol , vol.22 , Issue.18 , pp. 6533-6541
    • Massenet, S.1    Pellizzoni, L.2    Paushkin, S.3    Mattaj, I.W.4    Dreyfuss, G.5
  • 11
    • 0345599021 scopus 로고    scopus 로고
    • Smn, the spinal muscular atrophy-determining gene product, modulates axon growth and localization of beta-actin mRNA in growth cones of motoneurons
    • 14623865
    • Rossoll W, Jablonka S, Andreassi C, Kroning AK, Karle K, Monani UR, et al. Smn, the spinal muscular atrophy-determining gene product, modulates axon growth and localization of beta-actin mRNA in growth cones of motoneurons. J Cell Biol. 2003;163(4):801–12. doi: 10.1083/jcb.200304128 14623865
    • (2003) J Cell Biol , vol.163 , Issue.4 , pp. 801-812
    • Rossoll, W.1    Jablonka, S.2    Andreassi, C.3    Kroning, A.K.4    Karle, K.5    Monani, U.R.6
  • 12
    • 84862145991 scopus 로고    scopus 로고
    • Spinal muscular atrophy: the role of SMN in axonal mRNA regulation
    • 22330725
    • Fallini C, Bassell GJ, Rossoll W, Spinal muscular atrophy: the role of SMN in axonal mRNA regulation. Brain Res. 2012;1462:81–92. doi: 10.1016/j.brainres.2012.01.044 22330725
    • (2012) Brain Res , vol.1462 , pp. 81-92
    • Fallini, C.1    Bassell, G.J.2    Rossoll, W.3
  • 13
    • 84897548490 scopus 로고    scopus 로고
    • Dysregulation of ubiquitin homeostasis and beta-catenin signaling promote spinal muscular atrophy
    • 24590288
    • Wishart TM, Mutsaers CA, Riessland M, Reimer MM, Hunter G, Hannam ML, et al. Dysregulation of ubiquitin homeostasis and beta-catenin signaling promote spinal muscular atrophy. J Clin Invest. 2014;124(4):1821–34. doi: 10.1172/JCI71318 24590288
    • (2014) J Clin Invest , vol.124 , Issue.4 , pp. 1821-1834
    • Wishart, T.M.1    Mutsaers, C.A.2    Riessland, M.3    Reimer, M.M.4    Hunter, G.5    Hannam, M.L.6
  • 14
    • 84942511422 scopus 로고    scopus 로고
    • UBA1: At the Crossroads of Ubiquitin Homeostasis and Neurodegeneration
    • 26432019
    • Groen EJ, Gillingwater TH, UBA1: At the Crossroads of Ubiquitin Homeostasis and Neurodegeneration. Trends Mol Med. 2015;21(10):622–32. doi: 10.1016/j.molmed.2015.08.003 26432019
    • (2015) Trends Mol Med , vol.21 , Issue.10 , pp. 622-632
    • Groen, E.J.1    Gillingwater, T.H.2
  • 15
    • 85055597996 scopus 로고    scopus 로고
    • Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy
    • Powis RA, Karyka E, Boyd P, Come J, Jones RA, Zheng Y, et al. Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy. JCI Insight. 2016;1(11).
    • (2016) JCI Insight , vol.1 , Issue.11
    • Powis, R.A.1    Karyka, E.2    Boyd, P.3    Come, J.4    Jones, R.A.5    Zheng, Y.6
  • 16
    • 84979556428 scopus 로고    scopus 로고
    • Decreased function of survival motor neuron protein impairs endocytic pathways
    • 27402754
    • Dimitriadi M, Derdowski A, Kalloo G, Maginnis MS, O'Hern P, Bliska B, et al. Decreased function of survival motor neuron protein impairs endocytic pathways. Proc Natl Acad Sci U S A. 2016;113(30):E4377–86. doi: 10.1073/pnas.1600015113 27402754
    • (2016) Proc Natl Acad Sci U S A , vol.113 , Issue.30 , pp. 4377-4386
    • Dimitriadi, M.1    Derdowski, A.2    Kalloo, G.3    Maginnis, M.S.4    O'Hern, P.5    Bliska, B.6
  • 17
    • 84999711589 scopus 로고    scopus 로고
    • The Power of Human Protective Modifiers: PLS3 and CORO1C Unravel Impaired Endocytosis in Spinal Muscular Atrophy and Rescue SMA Phenotype
    • Hosseinibarkooie S, Peters M, Torres-Benito L, Rastetter RH, Hupperich K, Hoffmann A, et al. The Power of Human Protective Modifiers: PLS3 and CORO1C Unravel Impaired Endocytosis in Spinal Muscular Atrophy and Rescue SMA Phenotype. Am J Hum Genet. 2016.
    • (2016) Am J Hum Genet
    • Hosseinibarkooie, S.1    Peters, M.2    Torres-Benito, L.3    Rastetter, R.H.4    Hupperich, K.5    Hoffmann, A.6
  • 18
    • 84947967882 scopus 로고    scopus 로고
    • Selective loss of alpha motor neurons with sparing of gamma motor neurons and spinal cord cholinergic neurons in a mouse model of spinal muscular atrophy
    • 26576026
    • Powis RA, Gillingwater TH, Selective loss of alpha motor neurons with sparing of gamma motor neurons and spinal cord cholinergic neurons in a mouse model of spinal muscular atrophy. J Anat. 2016;228(3):443–51. doi: 10.1111/joa.12419 26576026
    • (2016) J Anat , vol.228 , Issue.3 , pp. 443-451
    • Powis, R.A.1    Gillingwater, T.H.2
  • 19
    • 71749091926 scopus 로고    scopus 로고
    • Neurofilament accumulation at the motor endplate and lack of axonal sprouting in a spinal muscular atrophy mouse model
    • 12023986
    • Cifuentes-Diaz C, Nicole S, Velasco ME, Borra-Cebrian C, Panozzo C, Frugier T, et al. Neurofilament accumulation at the motor endplate and lack of axonal sprouting in a spinal muscular atrophy mouse model. Hum Mol Genet. 2002;11(12):1439–47. 12023986
    • (2002) Hum Mol Genet , vol.11 , Issue.12 , pp. 1439-1447
    • Cifuentes-Diaz, C.1    Nicole, S.2    Velasco, M.E.3    Borra-Cebrian, C.4    Panozzo, C.5    Frugier, T.6
  • 20
    • 48249145306 scopus 로고    scopus 로고
    • Reduced SMN protein impairs maturation of the neuromuscular junctions in mouse models of spinal muscular atrophy
    • 18492800
    • Kariya S, Park GH, Maeno-Hikichi Y, Leykekhman O, Lutz C, Arkovitz MS, et al. Reduced SMN protein impairs maturation of the neuromuscular junctions in mouse models of spinal muscular atrophy. Hum Mol Genet. 2008;17(16):2552–69. doi: 10.1093/hmg/ddn156 18492800
    • (2008) Hum Mol Genet , vol.17 , Issue.16 , pp. 2552-2569
    • Kariya, S.1    Park, G.H.2    Maeno-Hikichi, Y.3    Leykekhman, O.4    Lutz, C.5    Arkovitz, M.S.6
  • 21
    • 41149113045 scopus 로고    scopus 로고
    • Selective vulnerability of motor neurons and dissociation of pre- and post-synaptic pathology at the neuromuscular junction in mouse models of spinal muscular atrophy
    • 18065780
    • Murray LM, Comley LH, Thomson D, Parkinson N, Talbot K, Gillingwater TH, Selective vulnerability of motor neurons and dissociation of pre- and post-synaptic pathology at the neuromuscular junction in mouse models of spinal muscular atrophy. Hum Mol Genet. 2008;17(7):949–62. doi: 10.1093/hmg/ddm367 18065780
    • (2008) Hum Mol Genet , vol.17 , Issue.7 , pp. 949-962
    • Murray, L.M.1    Comley, L.H.2    Thomson, D.3    Parkinson, N.4    Talbot, K.5    Gillingwater, T.H.6
  • 22
    • 58849103600 scopus 로고    scopus 로고
    • Impaired synaptic vesicle release and immaturity of neuromuscular junctions in spinal muscular atrophy mice
    • 19158308
    • Kong L, Wang X, Choe DW, Polley M, Burnett BG, Bosch-Marce M, et al. Impaired synaptic vesicle release and immaturity of neuromuscular junctions in spinal muscular atrophy mice. J Neurosci. 2009;29(3):842–51. doi: 10.1523/JNEUROSCI.4434-08.2009 19158308
    • (2009) J Neurosci , vol.29 , Issue.3 , pp. 842-851
    • Kong, L.1    Wang, X.2    Choe, D.W.3    Polley, M.4    Burnett, B.G.5    Bosch-Marce, M.6
  • 23
    • 78649723966 scopus 로고    scopus 로고
    • Synaptic defects in the spinal and neuromuscular circuitry in a mouse model of spinal muscular atrophy
    • 21085654
    • Ling KK, Lin MY, Zingg B, Feng Z, Ko CP, Synaptic defects in the spinal and neuromuscular circuitry in a mouse model of spinal muscular atrophy. PLoS One. 2010;5(11):e15457. doi: 10.1371/journal.pone.0015457 21085654
    • (2010) PLoS One , vol.5 , Issue.11 , pp. 15457
    • Ling, K.K.1    Lin, M.Y.2    Zingg, B.3    Feng, Z.4    Ko, C.P.5
  • 24
    • 84872862067 scopus 로고    scopus 로고
    • Synaptic defects in type I spinal muscular atrophy in human development
    • 22847626
    • Martinez-Hernandez R, Bernal S, Also-Rallo E, Alias L, Barcelo MJ, Hereu M, et al. Synaptic defects in type I spinal muscular atrophy in human development. J Pathol. 2013;229(1):49–61. doi: 10.1002/path.4080 22847626
    • (2013) J Pathol , vol.229 , Issue.1 , pp. 49-61
    • Martinez-Hernandez, R.1    Bernal, S.2    Also-Rallo, E.3    Alias, L.4    Barcelo, M.J.5    Hereu, M.6
  • 25
    • 84871718500 scopus 로고    scopus 로고
    • Spinal muscular atrophy: going beyond the motor neuron
    • 23228902
    • Hamilton G, Gillingwater TH, Spinal muscular atrophy: going beyond the motor neuron. Trends Mol Med. 2013;19(1):40–50. doi: 10.1016/j.molmed.2012.11.002 23228902
    • (2013) Trends Mol Med , vol.19 , Issue.1 , pp. 40-50
    • Hamilton, G.1    Gillingwater, T.H.2
  • 26
    • 83455213467 scopus 로고    scopus 로고
    • Severe neuromuscular denervation of clinically relevant muscles in a mouse model of spinal muscular atrophy
    • 21968514
    • Ling KK, Gibbs RM, Feng Z, Ko CP, Severe neuromuscular denervation of clinically relevant muscles in a mouse model of spinal muscular atrophy. Hum Mol Genet. 2012;21(1):185–95. doi: 10.1093/hmg/ddr453 21968514
    • (2012) Hum Mol Genet , vol.21 , Issue.1 , pp. 185-195
    • Ling, K.K.1    Gibbs, R.M.2    Feng, Z.3    Ko, C.P.4
  • 27
    • 85014071926 scopus 로고    scopus 로고
    • Transcriptional profiling of differentially vulnerable motor neurons at pre-symptomatic stage in the Smn (2b/-) mouse model of spinal muscular atrophy
    • 26374403
    • Murray LM, Beauvais A, Gibeault S, Courtney NL, Kothary R, Transcriptional profiling of differentially vulnerable motor neurons at pre-symptomatic stage in the Smn (2b/-) mouse model of spinal muscular atrophy. Acta Neuropathol Commun. 2015;3:55. doi: 10.1186/s40478-015-0231-1 26374403
    • (2015) Acta Neuropathol Commun , vol.3 , pp. 55
    • Murray, L.M.1    Beauvais, A.2    Gibeault, S.3    Courtney, N.L.4    Kothary, R.5
  • 28
    • 84871451459 scopus 로고    scopus 로고
    • Morphological characteristics of motor neurons do not determine their relative susceptibility to degeneration in a mouse model of severe spinal muscular atrophy
    • 23285108
    • Thomson SR, Nahon JE, Mutsaers CA, Thomson D, Hamilton G, Parson SH, et al. Morphological characteristics of motor neurons do not determine their relative susceptibility to degeneration in a mouse model of severe spinal muscular atrophy. PLoS One. 2012;7(12):e52605. doi: 10.1371/journal.pone.0052605 23285108
    • (2012) PLoS One , vol.7 , Issue.12 , pp. 52605
    • Thomson, S.R.1    Nahon, J.E.2    Mutsaers, C.A.3    Thomson, D.4    Hamilton, G.5    Parson, S.H.6
  • 29
    • 0034175513 scopus 로고    scopus 로고
    • Early and selective loss of neuromuscular synapse subtypes with low sprouting competence in motoneuron diseases
    • 10729333
    • Frey D, Schneider C, Xu L, Borg J, Spooren W, Caroni P, Early and selective loss of neuromuscular synapse subtypes with low sprouting competence in motoneuron diseases. J Neurosci. 2000;20(7):2534–42. 10729333
    • (2000) J Neurosci , vol.20 , Issue.7 , pp. 2534-2542
    • Frey, D.1    Schneider, C.2    Xu, L.3    Borg, J.4    Spooren, W.5    Caroni, P.6
  • 30
    • 35349025311 scopus 로고    scopus 로고
    • Time course of preferential motor unit loss in the SOD1 G93A mouse model of amyotrophic lateral sclerosis
    • 17766128
    • Hegedus J, Putman CT, Gordon T, Time course of preferential motor unit loss in the SOD1 G93A mouse model of amyotrophic lateral sclerosis. Neurobiol Dis. 2007;28(2):154–64. doi: 10.1016/j.nbd.2007.07.003 17766128
    • (2007) Neurobiol Dis , vol.28 , Issue.2 , pp. 154-164
    • Hegedus, J.1    Putman, C.T.2    Gordon, T.3
  • 31
    • 77954103868 scopus 로고    scopus 로고
    • Motor neuron diversity in development and disease
    • 20367447
    • Kanning KC, Kaplan A, Henderson CE, Motor neuron diversity in development and disease. Annu Rev Neurosci. 2010;33:409–40. doi: 10.1146/annurev.neuro.051508.135722 20367447
    • (2010) Annu Rev Neurosci , vol.33 , pp. 409-440
    • Kanning, K.C.1    Kaplan, A.2    Henderson, C.E.3
  • 32
    • 79960167259 scopus 로고    scopus 로고
    • Selective neuronal vulnerability in neurodegenerative diseases: from stressor thresholds to degeneration
    • 21745636
    • Saxena S, Caroni P, Selective neuronal vulnerability in neurodegenerative diseases: from stressor thresholds to degeneration. Neuron. 2011;71(1):35–48. doi: 10.1016/j.neuron.2011.06.031 21745636
    • (2011) Neuron , vol.71 , Issue.1 , pp. 35-48
    • Saxena, S.1    Caroni, P.2
  • 33
    • 84978760325 scopus 로고    scopus 로고
    • Selective Motor Neuron Resistance and Recovery in a New Inducible Mouse Model of TDP-43 Proteinopathy
    • 27445147
    • Spiller KJ, Cheung CJ, Restrepo CR, Kwong LK, Stieber AM, Trojanowski JQ, et al. Selective Motor Neuron Resistance and Recovery in a New Inducible Mouse Model of TDP-43 Proteinopathy. J Neurosci. 2016;36(29):7707–17. doi: 10.1523/JNEUROSCI.1457-16.2016 27445147
    • (2016) J Neurosci , vol.36 , Issue.29 , pp. 7707-7717
    • Spiller, K.J.1    Cheung, C.J.2    Restrepo, C.R.3    Kwong, L.K.4    Stieber, A.M.5    Trojanowski, J.Q.6
  • 34
    • 84865961091 scopus 로고    scopus 로고
    • Synaptic energy use and supply
    • 22958818
    • Harris JJ, Jolivet R, Attwell D, Synaptic energy use and supply. Neuron. 2012;75(5):762–77. doi: 10.1016/j.neuron.2012.08.019 22958818
    • (2012) Neuron , vol.75 , Issue.5 , pp. 762-777
    • Harris, J.J.1    Jolivet, R.2    Attwell, D.3
  • 35
    • 84907371314 scopus 로고    scopus 로고
    • A computational model of motor neuron degeneration
    • 25088365
    • Le Masson G, Przedborski S, Abbott LF, A computational model of motor neuron degeneration. Neuron. 2014;83(4):975–88. doi: 10.1016/j.neuron.2014.07.001 25088365
    • (2014) Neuron , vol.83 , Issue.4 , pp. 975-988
    • Le Masson, G.1    Przedborski, S.2    Abbott, L.F.3
  • 36
    • 67349164383 scopus 로고    scopus 로고
    • A role for motoneuron subtype-selective ER stress in disease manifestations of FALS mice
    • 19330001
    • Saxena S, Cabuy E, Caroni P, A role for motoneuron subtype-selective ER stress in disease manifestations of FALS mice. Nat Neurosci. 2009;12(5):627–36. doi: 10.1038/nn.2297 19330001
    • (2009) Nat Neurosci , vol.12 , Issue.5 , pp. 627-636
    • Saxena, S.1    Cabuy, E.2    Caroni, P.3
  • 37
    • 0037320528 scopus 로고    scopus 로고
    • Accurate and reproducible gene expression profiles from laser capture microdissection, transcript amplification, and high density oligonucleotide microarray analysis
    • 12552074
    • Luzzi V, Mahadevappa M, Raja R, Warrington JA, Watson MA, Accurate and reproducible gene expression profiles from laser capture microdissection, transcript amplification, and high density oligonucleotide microarray analysis. J Mol Diagn. 2003;5(1):9–14. doi: 10.1016/S1525-1578(10)60445-X 12552074
    • (2003) J Mol Diagn , vol.5 , Issue.1 , pp. 9-14
    • Luzzi, V.1    Mahadevappa, M.2    Raja, R.3    Warrington, J.A.4    Watson, M.A.5
  • 38
    • 0033971898 scopus 로고    scopus 로고
    • Mitochondria and neuronal survival
    • 10617771
    • Nicholls DG, Budd SL, Mitochondria and neuronal survival. Physiol Rev. 2000;80(1):315–60. 10617771
    • (2000) Physiol Rev , vol.80 , Issue.1 , pp. 315-360
    • Nicholls, D.G.1    Budd, S.L.2
  • 39
    • 23044506102 scopus 로고    scopus 로고
    • Synaptic mitochondria are critical for mobilization of reserve pool vesicles at Drosophila neuromuscular junctions
    • 16055061
    • Verstreken P, Ly CV, Venken KJ, Koh TW, Zhou Y, Bellen HJ, Synaptic mitochondria are critical for mobilization of reserve pool vesicles at Drosophila neuromuscular junctions. Neuron. 2005;47(3):365–78. doi: 10.1016/j.neuron.2005.06.018 16055061
    • (2005) Neuron , vol.47 , Issue.3 , pp. 365-378
    • Verstreken, P.1    Ly, C.V.2    Venken, K.J.3    Koh, T.W.4    Zhou, Y.5    Bellen, H.J.6
  • 40
    • 79959305691 scopus 로고    scopus 로고
    • Mitochondria: the next (neurode)generation
    • 21689593
    • Schon EA, Przedborski S, Mitochondria: the next (neurode)generation. Neuron. 2011;70(6):1033–53. doi: 10.1016/j.neuron.2011.06.003 21689593
    • (2011) Neuron , vol.70 , Issue.6 , pp. 1033-1053
    • Schon, E.A.1    Przedborski, S.2
  • 41
    • 67651140516 scopus 로고    scopus 로고
    • Mitochondrial dysfunction in a neural cell model of spinal muscular atrophy
    • 19437551
    • Acsadi G, Lee I, Li X, Khaidakov M, Pecinova A, Parker GC, et al. Mitochondrial dysfunction in a neural cell model of spinal muscular atrophy. J Neurosci Res. 2009;87(12):2748–56. doi: 10.1002/jnr.22106 19437551
    • (2009) J Neurosci Res , vol.87 , Issue.12 , pp. 2748-2756
    • Acsadi, G.1    Lee, I.2    Li, X.3    Khaidakov, M.4    Pecinova, A.5    Parker, G.C.6
  • 42
    • 84931026162 scopus 로고    scopus 로고
    • Impaired Muscle Mitochondrial Biogenesis and Myogenesis in Spinal Muscular Atrophy
    • 25844556
    • Ripolone M, Ronchi D, Violano R, Vallejo D, Fagiolari G, Barca E, et al. Impaired Muscle Mitochondrial Biogenesis and Myogenesis in Spinal Muscular Atrophy. JAMA Neurol. 2015;72(6):666–75. doi: 10.1001/jamaneurol.2015.0178 25844556
    • (2015) JAMA Neurol , vol.72 , Issue.6 , pp. 666-675
    • Ripolone, M.1    Ronchi, D.2    Violano, R.3    Vallejo, D.4    Fagiolari, G.5    Barca, E.6
  • 43
    • 84949294054 scopus 로고    scopus 로고
    • Central and peripheral defects in motor units of the diaphragm of spinal muscular atrophy mice
    • 26621405
    • Neve A, Trub J, Saxena S, Schumperli D, Central and peripheral defects in motor units of the diaphragm of spinal muscular atrophy mice. Mol Cell Neurosci. 2016;70:30–41. doi: 10.1016/j.mcn.2015.11.007 26621405
    • (2016) Mol Cell Neurosci , vol.70 , pp. 30-41
    • Neve, A.1    Trub, J.2    Saxena, S.3    Schumperli, D.4
  • 44
    • 77957322783 scopus 로고    scopus 로고
    • Ultrastructural changes in diaphragm neuromuscular junctions in a severe mouse model for Spinal Muscular Atrophy and their prevention by bifunctional U7 snRNA correcting SMN2 splicing
    • 20832308
    • Voigt T, Meyer K, Baum O, Schumperli D, Ultrastructural changes in diaphragm neuromuscular junctions in a severe mouse model for Spinal Muscular Atrophy and their prevention by bifunctional U7 snRNA correcting SMN2 splicing. Neuromuscul Disord. 2010;20(11):744–52. doi: 10.1016/j.nmd.2010.06.010 20832308
    • (2010) Neuromuscul Disord , vol.20 , Issue.11 , pp. 744-752
    • Voigt, T.1    Meyer, K.2    Baum, O.3    Schumperli, D.4
  • 45
  • 46
    • 84954470130 scopus 로고    scopus 로고
    • Abnormal mitochondrial transport and morphology as early pathological changes in human models of spinal muscular atrophy
    • 26586529
    • Xu CC, Denton KR, Wang ZB, Zhang X, Li XJ, Abnormal mitochondrial transport and morphology as early pathological changes in human models of spinal muscular atrophy. Dis Model Mech. 2016;9(1):39–49. doi: 10.1242/dmm.021766 26586529
    • (2016) Dis Model Mech , vol.9 , Issue.1 , pp. 39-49
    • Xu, C.C.1    Denton, K.R.2    Wang, Z.B.3    Zhang, X.4    Li, X.J.5
  • 47
    • 0042887389 scopus 로고    scopus 로고
    • Knockdown of the survival motor neuron (Smn) protein in zebrafish causes defects in motor axon outgrowth and pathfinding
    • 12952942
    • McWhorter ML, Monani UR, Burghes AH, Beattie CE, Knockdown of the survival motor neuron (Smn) protein in zebrafish causes defects in motor axon outgrowth and pathfinding. J Cell Biol. 2003;162(5):919–31. doi: 10.1083/jcb.200303168 12952942
    • (2003) J Cell Biol , vol.162 , Issue.5 , pp. 919-931
    • McWhorter, M.L.1    Monani, U.R.2    Burghes, A.H.3    Beattie, C.E.4
  • 48
    • 84901822168 scopus 로고    scopus 로고
    • SMN deficiency alters Nrxn2 expression and splicing in zebrafish and mouse models of spinal muscular atrophy
    • 24218366
    • See K, Yadav P, Giegerich M, Cheong PS, Graf M, Vyas H, et al. SMN deficiency alters Nrxn2 expression and splicing in zebrafish and mouse models of spinal muscular atrophy. Hum Mol Genet. 2014;23(7):1754–70. doi: 10.1093/hmg/ddt567 24218366
    • (2014) Hum Mol Genet , vol.23 , Issue.7 , pp. 1754-1770
    • See, K.1    Yadav, P.2    Giegerich, M.3    Cheong, P.S.4    Graf, M.5    Vyas, H.6
  • 49
    • 84892981582 scopus 로고    scopus 로고
    • Chondrolectin affects cell survival and neuronal outgrowth in in vitro and in vivo models of spinal muscular atrophy
    • 24067532
    • Sleigh JN, Barreiro-Iglesias A, Oliver PL, Biba A, Becker T, Davies KE, et al. Chondrolectin affects cell survival and neuronal outgrowth in in vitro and in vivo models of spinal muscular atrophy. Hum Mol Genet. 2014;23(4):855–69. doi: 10.1093/hmg/ddt477 24067532
    • (2014) Hum Mol Genet , vol.23 , Issue.4 , pp. 855-869
    • Sleigh, J.N.1    Barreiro-Iglesias, A.2    Oliver, P.L.3    Biba, A.4    Becker, T.5    Davies, K.E.6
  • 50
    • 84878941262 scopus 로고    scopus 로고
    • Temporal requirement for SMN in motoneuron development
    • 23459934
    • Hao le T, Duy PQ, Jontes JD, Wolman M, Granato M, Beattie CE, Temporal requirement for SMN in motoneuron development. Hum Mol Genet. 2013;22(13):2612–25. doi: 10.1093/hmg/ddt110 23459934
    • (2013) Hum Mol Genet , vol.22 , Issue.13 , pp. 2612-2625
    • Hao le, T.1    Duy, P.Q.2    Jontes, J.D.3    Wolman, M.4    Granato, M.5    Beattie, C.E.6
  • 51
    • 84930965780 scopus 로고    scopus 로고
    • Effective heritable gene knockdown in zebrafish using synthetic microRNAs
    • 26051838
    • Giacomotto J, Rinkwitz S, Becker TS, Effective heritable gene knockdown in zebrafish using synthetic microRNAs. Nat Commun. 2015;6:7378. doi: 10.1038/ncomms8378 26051838
    • (2015) Nat Commun , vol.6 , pp. 7378
    • Giacomotto, J.1    Rinkwitz, S.2    Becker, T.S.3
  • 52
    • 0030063033 scopus 로고    scopus 로고
    • Structure and expression of the mouse necdin gene. Identification of a postmitotic neuron-restrictive core promoter
    • 8557705
    • Uetsuki T, Takagi K, Sugiura H, Yoshikawa K, Structure and expression of the mouse necdin gene. Identification of a postmitotic neuron-restrictive core promoter. J Biol Chem. 1996;271(2):918–24. 8557705
    • (1996) J Biol Chem , vol.271 , Issue.2 , pp. 918-924
    • Uetsuki, T.1    Takagi, K.2    Sugiura, H.3    Yoshikawa, K.4
  • 53
    • 84961839432 scopus 로고    scopus 로고
    • Promotion of mitochondrial biogenesis by necdin protects neurons against mitochondrial insults
    • 26971449
    • Hasegawa K, Yasuda T, Shiraishi C, Fujiwara K, Przedborski S, Mochizuki H, et al. Promotion of mitochondrial biogenesis by necdin protects neurons against mitochondrial insults. Nat Commun. 2016;7:10943. doi: 10.1038/ncomms10943 26971449
    • (2016) Nat Commun , vol.7 , pp. 10943
    • Hasegawa, K.1    Yasuda, T.2    Shiraishi, C.3    Fujiwara, K.4    Przedborski, S.5    Mochizuki, H.6
  • 54
    • 79951977334 scopus 로고    scopus 로고
    • Regulation of mitochondrial biogenesis
    • 20533901
    • Jornayvaz FR, Shulman GI, Regulation of mitochondrial biogenesis. Essays Biochem. 2010;47:69–84. doi: 10.1042/bse0470069 20533901
    • (2010) Essays Biochem , vol.47 , pp. 69-84
    • Jornayvaz, F.R.1    Shulman, G.I.2
  • 55
    • 84958122602 scopus 로고    scopus 로고
    • Vascular Defects and Spinal Cord Hypoxia in Spinal Muscular Atrophy
    • 26506088
    • Somers E, Lees RD, Hoban K, Sleigh JN, Zhou H, Muntoni F, et al. Vascular Defects and Spinal Cord Hypoxia in Spinal Muscular Atrophy. Ann Neurol. 2016;79(2):217–30. doi: 10.1002/ana.24549 26506088
    • (2016) Ann Neurol , vol.79 , Issue.2 , pp. 217-230
    • Somers, E.1    Lees, R.D.2    Hoban, K.3    Sleigh, J.N.4    Zhou, H.5    Muntoni, F.6
  • 56
    • 33745171758 scopus 로고    scopus 로고
    • The identification of a recurrent phosphoglycerate kinase mutation associated with chronic haemolytic anaemia and neurological dysfunction in a family from USA
    • 16740138
    • Flanagan JM, Rhodes M, Wilson M, Beutler E, The identification of a recurrent phosphoglycerate kinase mutation associated with chronic haemolytic anaemia and neurological dysfunction in a family from USA. Br J Haematol. 2006;134(2):233–7. doi: 10.1111/j.1365-2141.2006.06143.x 16740138
    • (2006) Br J Haematol , vol.134 , Issue.2 , pp. 233-237
    • Flanagan, J.M.1    Rhodes, M.2    Wilson, M.3    Beutler, E.4
  • 57
    • 33644869707 scopus 로고    scopus 로고
    • Two new phosphoglycerate kinase mutations associated with chronic haemolytic anaemia and neurological dysfunction in two patients from Spain
    • 16412025
    • Noel N, Flanagan JM, Ramirez Bajo MJ, Kalko SG, Manu Mdel M, Garcia Fuster JL, et al. Two new phosphoglycerate kinase mutations associated with chronic haemolytic anaemia and neurological dysfunction in two patients from Spain. Br J Haematol. 2006;132(4):523–9. doi: 10.1111/j.1365-2141.2005.05882.x 16412025
    • (2006) Br J Haematol , vol.132 , Issue.4 , pp. 523-529
    • Noel, N.1    Flanagan, J.M.2    Ramirez Bajo, M.J.3    Kalko, S.G.4    Manu Mdel, M.5    Garcia Fuster, J.L.6
  • 58
    • 84962634585 scopus 로고    scopus 로고
    • Glycolytic Enzymes Localize to Synapses under Energy Stress to Support Synaptic Function
    • 27068791
    • Jang S, Nelson JC, Bend EG, Rodriguez-Laureano L, Tueros FG, Cartagenova L, et al. Glycolytic Enzymes Localize to Synapses under Energy Stress to Support Synaptic Function. Neuron. 2016;90(2):278–91. doi: 10.1016/j.neuron.2016.03.011 27068791
    • (2016) Neuron , vol.90 , Issue.2 , pp. 278-291
    • Jang, S.1    Nelson, J.C.2    Bend, E.G.3    Rodriguez-Laureano, L.4    Tueros, F.G.5    Cartagenova, L.6
  • 59
    • 84924923805 scopus 로고    scopus 로고
    • Terazosin activates Pgk1 and Hsp90 to promote stress resistance
    • 25383758
    • Chen X, Zhao C, Li X, Wang T, Li Y, Cao C, et al. Terazosin activates Pgk1 and Hsp90 to promote stress resistance. Nat Chem Biol. 2015;11(1):19–25. doi: 10.1038/nchembio.1657 25383758
    • (2015) Nat Chem Biol , vol.11 , Issue.1 , pp. 19-25
    • Chen, X.1    Zhao, C.2    Li, X.3    Wang, T.4    Li, Y.5    Cao, C.6
  • 60
    • 84923329869 scopus 로고    scopus 로고
    • Motor neurons with differential vulnerability to degeneration show distinct protein signatures in health and ALS
    • 25697826
    • Comley L, Allodi I, Nichterwitz S, Nizzardo M, Simone C, Corti S, et al. Motor neurons with differential vulnerability to degeneration show distinct protein signatures in health and ALS. Neuroscience. 2015;291:216–29. doi: 10.1016/j.neuroscience.2015.02.013 25697826
    • (2015) Neuroscience , vol.291 , pp. 216-229
    • Comley, L.1    Allodi, I.2    Nichterwitz, S.3    Nizzardo, M.4    Simone, C.5    Corti, S.6
  • 61
    • 84974784742 scopus 로고    scopus 로고
    • Selective Expression of Osteopontin in ALS-resistant Motor Neurons is a Critical Determinant of Late Phase Neurodegeneration Mediated by Matrix Metalloproteinase-9
    • 27264390
    • Morisaki Y, Niikura M, Watanabe M, Onishi K, Tanabe S, Moriwaki Y, et al. Selective Expression of Osteopontin in ALS-resistant Motor Neurons is a Critical Determinant of Late Phase Neurodegeneration Mediated by Matrix Metalloproteinase-9. Sci Rep. 2016;6:27354. doi: 10.1038/srep27354 27264390
    • (2016) Sci Rep , vol.6 , pp. 27354
    • Morisaki, Y.1    Niikura, M.2    Watanabe, M.3    Onishi, K.4    Tanabe, S.5    Moriwaki, Y.6
  • 62
    • 85014810407 scopus 로고    scopus 로고
    • Synaptotagmin-2, and -1, linked to neurotransmission impairment and vulnerability in Spinal Muscular Atrophy
    • Tejero R, Lopez-Manzaneda M, Arumugam S, Tabares L, Synaptotagmin-2, and -1, linked to neurotransmission impairment and vulnerability in Spinal Muscular Atrophy. Hum Mol Genet. 2016.
    • (2016) Hum Mol Genet
    • Tejero, R.1    Lopez-Manzaneda, M.2    Arumugam, S.3    Tabares, L.4
  • 63
    • 84892919280 scopus 로고    scopus 로고
    • Neuronal matrix metalloproteinase-9 is a determinant of selective neurodegeneration
    • 24462097
    • Kaplan A, Spiller KJ, Towne C, Kanning KC, Choe GT, Geber A, et al. Neuronal matrix metalloproteinase-9 is a determinant of selective neurodegeneration. Neuron. 2014;81(2):333–48. doi: 10.1016/j.neuron.2013.12.009 24462097
    • (2014) Neuron , vol.81 , Issue.2 , pp. 333-348
    • Kaplan, A.1    Spiller, K.J.2    Towne, C.3    Kanning, K.C.4    Choe, G.T.5    Geber, A.6
  • 64
    • 84872289257 scopus 로고    scopus 로고
    • Unravelling the enigma of selective vulnerability in neurodegeneration: motor neurons resistant to degeneration in ALS show distinct gene expression characteristics and decreased susceptibility to excitotoxicity
    • 23143228
    • Brockington A, Ning K, Heath PR, Wood E, Kirby J, Fusi N, et al. Unravelling the enigma of selective vulnerability in neurodegeneration: motor neurons resistant to degeneration in ALS show distinct gene expression characteristics and decreased susceptibility to excitotoxicity. Acta Neuropathol. 2013;125(1):95–109. doi: 10.1007/s00401-012-1058-5 23143228
    • (2013) Acta Neuropathol , vol.125 , Issue.1 , pp. 95-109
    • Brockington, A.1    Ning, K.2    Heath, P.R.3    Wood, E.4    Kirby, J.5    Fusi, N.6
  • 65
    • 84940828987 scopus 로고    scopus 로고
    • Oxidative stress, mitochondrial dysfunction and neurodegenerative diseases; a mechanistic insight
    • 26349970
    • Bhat AH, Dar KB, Anees S, Zargar MA, Masood A, Sofi MA, et al. Oxidative stress, mitochondrial dysfunction and neurodegenerative diseases; a mechanistic insight. Biomed Pharmacother. 2015;74:101–10. doi: 10.1016/j.biopha.2015.07.025 26349970
    • (2015) Biomed Pharmacother , vol.74 , pp. 101-110
    • Bhat, A.H.1    Dar, K.B.2    Anees, S.3    Zargar, M.A.4    Masood, A.5    Sofi, M.A.6
  • 66
    • 79955479720 scopus 로고    scopus 로고
    • Regulation of axonal mitochondrial transport and its impact on synaptic transmission
    • 21352858
    • Cai Q, Davis ML, Sheng ZH, Regulation of axonal mitochondrial transport and its impact on synaptic transmission. Neurosci Res. 2011;70(1):9–15. doi: 10.1016/j.neures.2011.02.005 21352858
    • (2011) Neurosci Res , vol.70 , Issue.1 , pp. 9-15
    • Cai, Q.1    Davis, M.L.2    Sheng, Z.H.3
  • 67
    • 84991238742 scopus 로고    scopus 로고
    • Triheptanoin Protects Motor Neurons and Delays the Onset of Motor Symptoms in a Mouse Model of Amyotrophic Lateral Sclerosis
    • 27564703
    • Tefera TW, Wong Y, Barkl-Luke ME, Ngo ST, Thomas NK, McDonald TS, et al. Triheptanoin Protects Motor Neurons and Delays the Onset of Motor Symptoms in a Mouse Model of Amyotrophic Lateral Sclerosis. PLoS One. 2016;11(8):e0161816. doi: 10.1371/journal.pone.0161816 27564703
    • (2016) PLoS One , vol.11 , Issue.8 , pp. 161816
    • Tefera, T.W.1    Wong, Y.2    Barkl-Luke, M.E.3    Ngo, S.T.4    Thomas, N.K.5    McDonald, T.S.6
  • 68
    • 84946510057 scopus 로고    scopus 로고
    • Central presynaptic terminals are enriched in ATP but the majority lack mitochondria
    • 25928229
    • Chavan V, Willis J, Walker SK, Clark HR, Liu X, Fox MA, et al. Central presynaptic terminals are enriched in ATP but the majority lack mitochondria. PLoS One. 2015;10(4):e0125185. doi: 10.1371/journal.pone.0125185 25928229
    • (2015) PLoS One , vol.10 , Issue.4 , pp. 125185
    • Chavan, V.1    Willis, J.2    Walker, S.K.3    Clark, H.R.4    Liu, X.5    Fox, M.A.6
  • 69
    • 84859651967 scopus 로고    scopus 로고
    • Density, calibre and ramification of muscle capillaries are altered in a mouse model of severe spinal muscular atrophy
    • 22153987
    • Somers E, Stencel Z, Wishart TM, Gillingwater TH, Parson SH, Density, calibre and ramification of muscle capillaries are altered in a mouse model of severe spinal muscular atrophy. Neuromuscul Disord. 2012;22(5):435–42. doi: 10.1016/j.nmd.2011.10.021 22153987
    • (2012) Neuromuscul Disord , vol.22 , Issue.5 , pp. 435-442
    • Somers, E.1    Stencel, Z.2    Wishart, T.M.3    Gillingwater, T.H.4    Parson, S.H.5
  • 70
    • 84873323723 scopus 로고    scopus 로고
    • Vesicular glycolysis provides on-board energy for fast axonal transport
    • 23374344
    • Zala D, Hinckelmann MV, Yu H, Lyra da Cunha MM, Liot G, Cordelieres FP, et al. Vesicular glycolysis provides on-board energy for fast axonal transport. Cell. 2013;152(3):479–91. doi: 10.1016/j.cell.2012.12.029 23374344
    • (2013) Cell , vol.152 , Issue.3 , pp. 479-491
    • Zala, D.1    Hinckelmann, M.V.2    Yu, H.3    Lyra da Cunha, M.M.4    Liot, G.5    Cordelieres, F.P.6
  • 71
    • 80054712173 scopus 로고    scopus 로고
    • The spinal muscular atrophy mouse model, SMADelta7, displays altered axonal transport without global neurofilament alterations
    • 21681521
    • Dale JM, Shen H, Barry DM, Garcia VB, Rose FF, Jr.Lorson CL, et al. The spinal muscular atrophy mouse model, SMADelta7, displays altered axonal transport without global neurofilament alterations. Acta Neuropathol. 2011;122(3):331–41. doi: 10.1007/s00401-011-0848-5 21681521
    • (2011) Acta Neuropathol , vol.122 , Issue.3 , pp. 331-341
    • Dale, J.M.1    Shen, H.2    Barry, D.M.3    Garcia, V.B.4    Rose, F.F.5    Lorson, C.L.6
  • 72
    • 79952390939 scopus 로고    scopus 로고
    • The survival of motor neuron (SMN) protein interacts with the mRNA-binding protein HuD and regulates localization of poly(A) mRNA in primary motor neuron axons
    • 21389246
    • Fallini C, Zhang H, Su Y, Silani V, Singer RH, Rossoll W, et al. The survival of motor neuron (SMN) protein interacts with the mRNA-binding protein HuD and regulates localization of poly(A) mRNA in primary motor neuron axons. J Neurosci. 2011;31(10):3914–25. doi: 10.1523/JNEUROSCI.3631-10.2011 21389246
    • (2011) J Neurosci , vol.31 , Issue.10 , pp. 3914-3925
    • Fallini, C.1    Zhang, H.2    Su, Y.3    Silani, V.4    Singer, R.H.5    Rossoll, W.6
  • 73
    • 84961992254 scopus 로고    scopus 로고
    • Deficiency of the Survival of Motor Neuron Protein Impairs mRNA Localization and Local Translation in the Growth Cone of Motor Neurons
    • 27030765
    • Fallini C, Donlin-Asp PG, Rouanet JP, Bassell GJ, Rossoll W, Deficiency of the Survival of Motor Neuron Protein Impairs mRNA Localization and Local Translation in the Growth Cone of Motor Neurons. J Neurosci. 2016;36(13):3811–20. doi: 10.1523/JNEUROSCI.2396-15.2016 27030765
    • (2016) J Neurosci , vol.36 , Issue.13 , pp. 3811-3820
    • Fallini, C.1    Donlin-Asp, P.G.2    Rouanet, J.P.3    Bassell, G.J.4    Rossoll, W.5
  • 74
    • 84940388753 scopus 로고    scopus 로고
    • Moving towards treatments for spinal muscular atrophy: hopes and limits
    • 25920617
    • Wirth B, Barkats M, Martinat C, Sendtner M, Gillingwater TH, Moving towards treatments for spinal muscular atrophy: hopes and limits. Expert Opin Emerg Drugs. 2015;20(3):353–6. doi: 10.1517/14728214.2015.1041375 25920617
    • (2015) Expert Opin Emerg Drugs , vol.20 , Issue.3 , pp. 353-356
    • Wirth, B.1    Barkats, M.2    Martinat, C.3    Sendtner, M.4    Gillingwater, T.H.5
  • 76
    • 77952295831 scopus 로고    scopus 로고
    • SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy
    • 20097677
    • Riessland M, Ackermann B, Forster A, Jakubik M, Hauke J, Garbes L, et al. SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy. Hum Mol Genet. 2010;19(8):1492–506. doi: 10.1093/hmg/ddq023 20097677
    • (2010) Hum Mol Genet , vol.19 , Issue.8 , pp. 1492-1506
    • Riessland, M.1    Ackermann, B.2    Forster, A.3    Jakubik, M.4    Hauke, J.5    Garbes, L.6
  • 78
    • 27744602432 scopus 로고    scopus 로고
    • Neuromuscular synapses can form in vivo by incorporation of initially aneural postsynaptic specializations
    • 16162647
    • Flanagan-Steet H, Fox MA, Meyer D, Sanes JR, Neuromuscular synapses can form in vivo by incorporation of initially aneural postsynaptic specializations. Development. 2005;132(20):4471–81. doi: 10.1242/dev.02044 16162647
    • (2005) Development , vol.132 , Issue.20 , pp. 4471-4481
    • Flanagan-Steet, H.1    Fox, M.A.2    Meyer, D.3    Sanes, J.R.4
  • 79
    • 32544440733 scopus 로고    scopus 로고
    • Proteomics of early zebrafish embryos
    • 16412219
    • Link V, Shevchenko A, Heisenberg CP, Proteomics of early zebrafish embryos. BMC Dev Biol. 2006;6:1. doi: 10.1186/1471-213X-6-1 16412219
    • (2006) BMC Dev Biol , vol.6 , pp. 1
    • Link, V.1    Shevchenko, A.2    Heisenberg, C.P.3
  • 80
    • 84883345978 scopus 로고    scopus 로고
    • Total protein analysis as a reliable loading control for quantitative fluorescent Western blotting
    • 24023619
    • Eaton SL, Roche SL, Llavero Hurtado M, Oldknow KJ, Farquharson C, Gillingwater TH, et al. Total protein analysis as a reliable loading control for quantitative fluorescent Western blotting. PLoS One. 2013;8(8):e72457. doi: 10.1371/journal.pone.0072457 24023619
    • (2013) PLoS One , vol.8 , Issue.8 , pp. 72457
    • Eaton, S.L.1    Roche, S.L.2    Llavero Hurtado, M.3    Oldknow, K.J.4    Farquharson, C.5    Gillingwater, T.H.6
  • 81
    • 84881520627 scopus 로고    scopus 로고
    • ALS-associated mutations in FUS disrupt the axonal distribution and function of SMN
    • 23681068
    • Groen EJ, Fumoto K, Blokhuis AM, Engelen-Lee J, Zhou Y, van den Heuvel DM, et al. ALS-associated mutations in FUS disrupt the axonal distribution and function of SMN. Hum Mol Genet. 2013;22(18):3690–704. doi: 10.1093/hmg/ddt222 23681068
    • (2013) Hum Mol Genet , vol.22 , Issue.18 , pp. 3690-3704
    • Groen, E.J.1    Fumoto, K.2    Blokhuis, A.M.3    Engelen-Lee, J.4    Zhou, Y.5    van den Heuvel, D.M.6
  • 82
    • 84966549121 scopus 로고    scopus 로고
    • Comparative interactomics analysis of different ALS-associated proteins identifies converging molecular pathways
    • 27164932
    • Blokhuis AM, Koppers M, Groen EJ, van den Heuvel DM, Dini Modigliani S, Anink JJ, et al. Comparative interactomics analysis of different ALS-associated proteins identifies converging molecular pathways. Acta Neuropathol. 2016;132(2):175–96. doi: 10.1007/s00401-016-1575-8 27164932
    • (2016) Acta Neuropathol , vol.132 , Issue.2 , pp. 175-196
    • Blokhuis, A.M.1    Koppers, M.2    Groen, E.J.3    van den Heuvel, D.M.4    Dini Modigliani, S.5    Anink, J.J.6


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