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Volumn 29, Issue 11, 2014, Pages 1391-1396

Preclinical models: Needed in translation? A Pro/Con debate

Author keywords

Animal models; HTT; Huntington's; Transgenic; Translation

Indexed keywords

ANIMAL; DISEASE MODEL; GENETICS; HUMAN; HUNTINGTON CHOREA; PRECLINICAL STUDY; PROCEDURES; TRANSLATIONAL RESEARCH;

EID: 84907926731     PISSN: 08853185     EISSN: 15318257     Source Type: Journal    
DOI: 10.1002/mds.26010     Document Type: Article
Times cited : (11)

References (41)
  • 1
    • 84884537922 scopus 로고    scopus 로고
    • Choosing an animal model for the study of Huntington's disease
    • Pouladi MA, Morton AJ, Hayden MR. Choosing an animal model for the study of Huntington's disease. Nat Rev Neurosci 2013;14:708-721.
    • (2013) Nat Rev Neurosci , vol.14 , pp. 708-721
    • Pouladi, M.A.1    Morton, A.J.2    Hayden, M.R.3
  • 2
    • 0033524413 scopus 로고    scopus 로고
    • Polyglutamine-mediated dysfunction and apoptotic death of a Caenorhabditis elegans sensory neuron
    • Faber PW, Alter JR, MacDonald ME, Hart AC. Polyglutamine-mediated dysfunction and apoptotic death of a Caenorhabditis elegans sensory neuron. Proc Natl Acad Sci USA 1999;96:179-84.
    • (1999) Proc Natl Acad Sci USA , vol.96 , pp. 179-184
    • Faber, P.W.1    Alter, J.R.2    MacDonald, M.E.3    Hart, A.C.4
  • 4
    • 16044373842 scopus 로고    scopus 로고
    • Exon 1 of the HD gene with an expanded CAG repeat is sufficient to cause a progressive neurological phenotype in transgenic mice
    • Mangiarini L, Sathasivam K, Seller M, et al. Exon 1 of the HD gene with an expanded CAG repeat is sufficient to cause a progressive neurological phenotype in transgenic mice. Cell 1996;87:493-506.
    • (1996) Cell , vol.87 , pp. 493-506
    • Mangiarini, L.1    Sathasivam, K.2    Seller, M.3
  • 5
    • 0032168160 scopus 로고    scopus 로고
    • Polyglutamine-expanded human huntingtin transgenes induce degeneration of Drosophila photoreceptor neurons
    • Jackson GR, Salecker I, Dong X, et al. Polyglutamine-expanded human huntingtin transgenes induce degeneration of Drosophila photoreceptor neurons. Neuron 1998;21:633-642.
    • (1998) Neuron , vol.21 , pp. 633-642
    • Jackson, G.R.1    Salecker, I.2    Dong, X.3
  • 6
    • 37549004790 scopus 로고    scopus 로고
    • Suppression of neurodegeneration and increased neurotransmission caused by expanded full-length huntingtin accumulating in the cytoplasm
    • Romero E, Cha G-H, Verstreken P, et al. Suppression of neurodegeneration and increased neurotransmission caused by expanded full-length huntingtin accumulating in the cytoplasm. Neuron 2008;57:27-40.
    • (2008) Neuron , vol.57 , pp. 27-40
    • Romero, E.1    Cha, G.-H.2    Verstreken, P.3
  • 7
    • 77649258646 scopus 로고    scopus 로고
    • A Drosophila model for TDP-43 proteinopathy
    • Li Y, Ray P, Rao EJ, et al. A Drosophila model for TDP-43 proteinopathy. Proc Natl Acad Sci U S A 2010;107:3169-3174.
    • (2010) Proc Natl Acad Sci U S A , vol.107 , pp. 3169-3174
    • Li, Y.1    Ray, P.2    Rao, E.J.3
  • 8
    • 84884907135 scopus 로고    scopus 로고
    • A guide to neurotoxic animal models of Parkinson's disease
    • Tieu K. A guide to neurotoxic animal models of Parkinson's disease. Cold Spring Harb Perspect Med 2011;1:a009316.
    • (2011) Cold Spring Harb Perspect Med , vol.1 , pp. a009316
    • Tieu, K.1
  • 9
    • 84868110606 scopus 로고    scopus 로고
    • A novel BACHD transgenic rat exhibits characteristic neuropathological features of Huntington disease
    • Yu-Taeger L, Petrasch-Parwez E, Osmand AP, et al. A novel BACHD transgenic rat exhibits characteristic neuropathological features of Huntington disease. J Neurosci 2012;32:15426-15438.
    • (2012) J Neurosci , vol.32 , pp. 15426-15438
    • Yu-Taeger, L.1    Petrasch-Parwez, E.2    Osmand, A.P.3
  • 10
    • 0022446150 scopus 로고
    • Replication of the neurochemical characteristics of Huntington's disease by quinolinic acid
    • Beal MF, Kowall NW, Ellison DW, Mazurek MF, Swartz KJ, Martin JB. Replication of the neurochemical characteristics of Huntington's disease by quinolinic acid. Nature 1986;321:168-171.
    • (1986) Nature , vol.321 , pp. 168-171
    • Beal, M.F.1    Kowall, N.W.2    Ellison, D.W.3    Mazurek, M.F.4    Swartz, K.J.5    Martin, J.B.6
  • 12
    • 77952526316 scopus 로고    scopus 로고
    • An ovine transgenic Huntington's disease model
    • Jacobsen JC, Bawden CS, Rudiger SR, et al. An ovine transgenic Huntington's disease model. Hum Mol Genet 2010;19:1873-1882.
    • (2010) Hum Mol Genet , vol.19 , pp. 1873-1882
    • Jacobsen, J.C.1    Bawden, C.S.2    Rudiger, S.R.3
  • 13
    • 77957732707 scopus 로고    scopus 로고
    • Expression of Huntington's disease protein results in apoptotic neurons in the brains of cloned transgenic pigs
    • Yang D, Wang C-E, Zhao B, et al. Expression of Huntington's disease protein results in apoptotic neurons in the brains of cloned transgenic pigs. Hum Mol Genet 2010;19:3983-3994.
    • (2010) Hum Mol Genet , vol.19 , pp. 3983-3994
    • Yang, D.1    Wang, C.-E.2    Zhao, B.3
  • 14
    • 84881610091 scopus 로고    scopus 로고
    • Human pluripotent stem cell differentiation into authentic striatal projection neurons
    • Delli Carri A, Onorati M, Castiglioni V, et al. Human pluripotent stem cell differentiation into authentic striatal projection neurons. Stem Cell Rev 2013;9:461-474.
    • (2013) Stem Cell Rev , vol.9 , pp. 461-474
    • Delli Carri, A.1    Onorati, M.2    Castiglioni, V.3
  • 15
    • 74049164709 scopus 로고    scopus 로고
    • Non-cell autonomous toxicity in neurodegenerative disorders: ALS and beyond
    • Ilieva H, Polymenidou M, Cleveland DW. Non-cell autonomous toxicity in neurodegenerative disorders: ALS and beyond. J Cell Biol 2009;187:761-772.
    • (2009) J Cell Biol , vol.187 , pp. 761-772
    • Ilieva, H.1    Polymenidou, M.2    Cleveland, D.W.3
  • 16
    • 0036345741 scopus 로고    scopus 로고
    • Impaired glutamate transport and glutamate-glutamine cycling: downstream effects of the Huntington mutation
    • Behrens PF, Franz P, Woodman B, Lindenberg KS, Landwehrmeyer GB. Impaired glutamate transport and glutamate-glutamine cycling: downstream effects of the Huntington mutation. Brain 2002;125:1908-1922.
    • (2002) Brain , vol.125 , pp. 1908-1922
    • Behrens, P.F.1    Franz, P.2    Woodman, B.3    Lindenberg, K.S.4    Landwehrmeyer, G.B.5
  • 17
    • 84862663712 scopus 로고    scopus 로고
    • Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis
    • Kordasiewicz HB, Stanek LM, Wancewicz EV, et al. Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Neuron 2012;74:1031-1044.
    • (2012) Neuron , vol.74 , pp. 1031-1044
    • Kordasiewicz, H.B.1    Stanek, L.M.2    Wancewicz, E.V.3
  • 18
    • 84879051700 scopus 로고    scopus 로고
    • Huntington's disease: the past, present, and future search for disease modifiers
    • Clabough EBD. Huntington's disease: the past, present, and future search for disease modifiers. Yale J Biol Med 2013;86:217.
    • (2013) Yale J Biol Med , vol.86 , pp. 217
    • Clabough, E.B.D.1
  • 19
    • 78650031174 scopus 로고    scopus 로고
    • Huntington's disease: from molecular pathogenesis to clinical treatment
    • Ross CA, Tabrizi SJ. Huntington's disease: from molecular pathogenesis to clinical treatment. Lancet Neurol 2011;10:83-98.
    • (2011) Lancet Neurol , vol.10 , pp. 83-98
    • Ross, C.A.1    Tabrizi, S.J.2
  • 20
    • 84856287728 scopus 로고    scopus 로고
    • Screening of therapeutic strategies for Huntington's disease in YAC128 transgenic mice
    • Gil-Mohapel JM. Screening of therapeutic strategies for Huntington's disease in YAC128 transgenic mice. CNS Neurosci Ther 2012;18:77-86.
    • (2012) CNS Neurosci Ther , vol.18 , pp. 77-86
    • Gil-Mohapel, J.M.1
  • 21
    • 60549103857 scopus 로고    scopus 로고
    • The R6 lines of transgenic mice: a model for screening new therapies for Huntington's disease
    • Gil JM, Rego AC. The R6 lines of transgenic mice: a model for screening new therapies for Huntington's disease. Brain Res Rev 2009;59:410-431.
    • (2009) Brain Res Rev , vol.59 , pp. 410-431
    • Gil, J.M.1    Rego, A.C.2
  • 22
    • 84856815851 scopus 로고    scopus 로고
    • Perspectives on molecular targeted therapies and clinical trials for neurodegenerative diseases
    • Katsuno M, Tanaka F, Sobue G. Perspectives on molecular targeted therapies and clinical trials for neurodegenerative diseases. J Neurol Neurosurg Psychiatry 2012;83:329-335.
    • (2012) J Neurol Neurosurg Psychiatry , vol.83 , pp. 329-335
    • Katsuno, M.1    Tanaka, F.2    Sobue, G.3
  • 23
    • 23844525922 scopus 로고    scopus 로고
    • Huntington's disease genetics
    • Myers RH. Huntington's disease genetics. NeuroRx 2004;1:255-262.
    • (2004) NeuroRx , vol.1 , pp. 255-262
    • Myers, R.H.1
  • 24
    • 33645798913 scopus 로고    scopus 로고
    • Tetrabenazine as antichorea therapy in Huntington disease: a randomized controlled trial
    • Huntington Study Group. Tetrabenazine as antichorea therapy in Huntington disease: a randomized controlled trial. Neurology 2006;66:366-372.
    • (2006) Neurology , vol.66 , pp. 366-372
  • 27
    • 34548860125 scopus 로고    scopus 로고
    • The origins of oncomice: a history of the first transgenic mice genetically engineered to develop cancer
    • Hanahan D, Wagner EF, Palmiter RD. The origins of oncomice: a history of the first transgenic mice genetically engineered to develop cancer. Genes Dev 2007;21:2258-2270.
    • (2007) Genes Dev , vol.21 , pp. 2258-2270
    • Hanahan, D.1    Wagner, E.F.2    Palmiter, R.D.3
  • 28
    • 0034059140 scopus 로고    scopus 로고
    • Cre recombinase: the universal reagent for genome tailoring
    • Nagy A. Cre recombinase: the universal reagent for genome tailoring. Genesis 2000;26:99-109.
    • (2000) Genesis , vol.26 , pp. 99-109
    • Nagy, A.1
  • 29
    • 84902996303 scopus 로고    scopus 로고
    • Neuronal targets for reducing mutant huntingtin expression to ameliorate disease in a mouse model of Huntington's disease
    • Wang N, Gray M, Lu X-H, et al. Neuronal targets for reducing mutant huntingtin expression to ameliorate disease in a mouse model of Huntington's disease. Nat Med 2014;20:536-541.
    • (2014) Nat Med , vol.20 , pp. 536-541
    • Wang, N.1    Gray, M.2    Lu, X.-H.3
  • 30
    • 79953756679 scopus 로고    scopus 로고
    • Mutant huntingtin causes metabolic imbalance by disruption of hypothalamic neurocircuits
    • Hult S, Soylu R, Björklund T, et al. Mutant huntingtin causes metabolic imbalance by disruption of hypothalamic neurocircuits. Cell Metab 2011;13:428-439.
    • (2011) Cell Metab , vol.13 , pp. 428-439
    • Hult, S.1    Soylu, R.2    Björklund, T.3
  • 31
    • 84897405847 scopus 로고    scopus 로고
    • Mutant Huntingtin promotes autonomous microglia activation via myeloid lineage-determining factors
    • Crotti A, Benner C, Kerman BE, et al. Mutant Huntingtin promotes autonomous microglia activation via myeloid lineage-determining factors. Nat Neurosci 2014;17:513-521.
    • (2014) Nat Neurosci , vol.17 , pp. 513-521
    • Crotti, A.1    Benner, C.2    Kerman, B.E.3
  • 32
    • 33747195353 scopus 로고    scopus 로고
    • Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors
    • Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 2006;126:663-676.
    • (2006) Cell , vol.126 , pp. 663-676
    • Takahashi, K.1    Yamanaka, S.2
  • 33
    • 84898778301 scopus 로고    scopus 로고
    • A guide to genome engineering with programmable nucleases
    • Kim H, Kim J-S. A guide to genome engineering with programmable nucleases. Nat Rev Genet 2014;15:321-334.
    • (2014) Nat Rev Genet , vol.15 , pp. 321-334
    • Kim, H.1    Kim, J.-S.2
  • 34
    • 84864628471 scopus 로고    scopus 로고
    • Induced pluripotent stem cells from patients with Huntington's disease show CAG-repeat-expansion-associated phenotypes
    • HD iPSC Consortium. Induced pluripotent stem cells from patients with Huntington's disease show CAG-repeat-expansion-associated phenotypes. Cell Stem Cell 2012;11:264-278.
    • (2012) Cell Stem Cell , vol.11 , pp. 264-278
  • 35
    • 84864631163 scopus 로고    scopus 로고
    • Genetic correction of Huntington's disease phenotypes in induced pluripotent stem cells
    • An MC, Zhang N, Scott G, Montoro D, Wittkop T, Mooney S, et al. Genetic correction of Huntington's disease phenotypes in induced pluripotent stem cells. Cell Stem Cell 2012;11:253-263.
    • (2012) Cell Stem Cell , vol.11 , pp. 253-263
    • An, M.C.1    Zhang, N.2    Scott, G.3    Montoro, D.4    Wittkop, T.5    Mooney, S.6
  • 36
    • 84876313188 scopus 로고    scopus 로고
    • Modeling Huntington's disease with induced pluripotent stem cells
    • Kaye JA, Finkbeiner S. Modeling Huntington's disease with induced pluripotent stem cells. Mol Cell Neurosci 2013;56:50-64.
    • (2013) Mol Cell Neurosci , vol.56 , pp. 50-64
    • Kaye, J.A.1    Finkbeiner, S.2
  • 37
    • 84906829737 scopus 로고    scopus 로고
    • Human-induced pluripotent stem cells: potential for neurodegenerative diseases
    • May 13.
    • Ross CA, Akimov SS. Human-induced pluripotent stem cells: potential for neurodegenerative diseases. Hum Mol Genet 2014;May 13.
    • (2014) Hum Mol Genet
    • Ross, C.A.1    Akimov, S.S.2
  • 38
    • 84871586691 scopus 로고    scopus 로고
    • Developmentally coordinated extrinsic signals drive human pluripotent stem cell differentiation toward authentic DARPP-32+ medium-sized spiny neurons
    • Carri AD, Onorati M, Lelos MJ, et al. Developmentally coordinated extrinsic signals drive human pluripotent stem cell differentiation toward authentic DARPP-32+ medium-sized spiny neurons. Development 2013;140:301-312.
    • (2013) Development , vol.140 , pp. 301-312
    • Carri, A.D.1    Onorati, M.2    Lelos, M.J.3
  • 39
    • 20944431926 scopus 로고    scopus 로고
    • Pathological cell-cell interactions elicited by a neuropathogenic form of mutant Huntingtin contribute to cortical pathogenesis in HD mice
    • Gu X, Li C, Wei W, et al. Pathological cell-cell interactions elicited by a neuropathogenic form of mutant Huntingtin contribute to cortical pathogenesis in HD mice. Neuron 2005;46:433-444.
    • (2005) Neuron , vol.46 , pp. 433-444
    • Gu, X.1    Li, C.2    Wei, W.3
  • 40
    • 34249864115 scopus 로고    scopus 로고
    • Pathological cell-cell interactions are necessary for striatal pathogenesis in a conditional mouse model of Huntington's disease
    • Gu X, André VM, Cepeda C, et al. Pathological cell-cell interactions are necessary for striatal pathogenesis in a conditional mouse model of Huntington's disease. Mol Neurodegen 2007;2:8.
    • (2007) Mol Neurodegen , vol.2 , pp. 8
    • Gu, X.1    André, V.M.2    Cepeda, C.3
  • 41
    • 84899525577 scopus 로고    scopus 로고
    • Astrocyte Kir4.1 ion channel deficits contribute to neuronal dysfunction in Huntington's disease model mice
    • Tong X, Ao Y, Faas GC, et al. Astrocyte Kir4.1 ion channel deficits contribute to neuronal dysfunction in Huntington's disease model mice. Nat Neurosci 2014;17:694-703.
    • (2014) Nat Neurosci , vol.17 , pp. 694-703
    • Tong, X.1    Ao, Y.2    Faas, G.C.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.