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Volumn 33, Issue 7, 2018, Pages 1033-1041

Huntington's disease: Current and future therapeutic prospects

Author keywords

Huntington's Disease; Therapy

Indexed keywords

COMPLEMENTARY DNA; DIMEBON; TETRABENAZINE;

EID: 85052083963     PISSN: 08853185     EISSN: 15318257     Source Type: Journal    
DOI: 10.1002/mds.27363     Document Type: Review
Times cited : (33)

References (93)
  • 1
    • 0027480960 scopus 로고
    • A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes
    • The Huntington's Disease Collaborative Research Group. A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. Cell 1993;72:971-983.
    • (1993) Cell , vol.72 , pp. 971-983
  • 2
    • 0018303366 scopus 로고
    • Huntington disease: clinical care and evaluation
    • Shoulson I, Fahn S. Huntington disease: clinical care and evaluation. Neurology 1979;29:1-3.
    • (1979) Neurology , vol.29 , pp. 1-3
    • Shoulson, I.1    Fahn, S.2
  • 3
    • 85052489438 scopus 로고    scopus 로고
    • The CREST-E study of creatine for Huntington disease. A randomized controlled trial
    • Hersch SM, Schiffito G, Oakes D, et al. The CREST-E study of creatine for Huntington disease. A randomized controlled trial. Neurology 2017;89:1-8.
    • (2017) Neurology , vol.89 , pp. 1-8
    • Hersch, S.M.1    Schiffito, G.2    Oakes, D.3
  • 4
    • 85009061038 scopus 로고    scopus 로고
    • A randomized, double-blind, placebo-controlled trial of coenzyme Q10 in Huntington disease
    • McGarry A, McDermott M, Kieburtz K, et al. A randomized, double-blind, placebo-controlled trial of coenzyme Q10 in Huntington disease. Neurology 2017;88:152-159.
    • (2017) Neurology , vol.88 , pp. 152-159
    • McGarry, A.1    McDermott, M.2    Kieburtz, K.3
  • 5
    • 78649369122 scopus 로고    scopus 로고
    • A futility study of minocycline in Huntington's disease
    • Huntington Study Group DOMINO Investigators. A futility study of minocycline in Huntington's disease. Mov Disord 2010:25(12):2219-2224.
    • (2010) Mov Disord , vol.25 , Issue.12 , pp. 2219-2224
  • 6
    • 84861456266 scopus 로고    scopus 로고
    • Dimebon slows progression of proteinopathy in γ-synuclein transgenic mice
    • Bachurin SO, Shelkovnikova TA, Ustyugov AA, et al. Dimebon slows progression of proteinopathy in γ-synuclein transgenic mice. Neurotox Res 2012;22:33-42.
    • (2012) Neurotox Res , vol.22 , pp. 33-42
    • Bachurin, S.O.1    Shelkovnikova, T.A.2    Ustyugov, A.A.3
  • 7
    • 76149084493 scopus 로고    scopus 로고
    • A randomized, placebo-controlled trial of latrepirdine in Huntington disease
    • Kieburtz K, McDermott MP, Voss TS, et al. A randomized, placebo-controlled trial of latrepirdine in Huntington disease. Arch Neurol 2010;67:154-160.
    • (2010) Arch Neurol , vol.67 , pp. 154-160
    • Kieburtz, K.1    McDermott, M.P.2    Voss, T.S.3
  • 8
    • 84875552322 scopus 로고    scopus 로고
    • A randomized, double-blind, placebo-controlled study of latrepirdine in patients with mild to moderate Huntington disease
    • HORIZON Investigators of the Huntington Study Group and European Huntington's Disease Network. A randomized, double-blind, placebo-controlled study of latrepirdine in patients with mild to moderate Huntington disease. JAMA Neurol 2013;70:25-33.
    • (2013) JAMA Neurol , vol.70 , pp. 25-33
  • 9
    • 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
  • 10
    • 84977592334 scopus 로고    scopus 로고
    • Effect of deutetrabenazine on chorea among patients with Huntington disease: a randomized clinical trial
    • Huntington Study Group; Frank S, Testa CM, Stamler D, et al. Effect of deutetrabenazine on chorea among patients with Huntington disease: a randomized clinical trial. JAMA 2016;316:40-50.
    • (2016) JAMA , vol.316 , pp. 40-50
    • Frank, S.1    Testa, C.M.2    Stamler, D.3
  • 13
    • 85011573727 scopus 로고    scopus 로고
    • Deutetrabenazine—not a revolution but welcome evolution for treating chorea in Huntington disease
    • Reilmann R. Deutetrabenazine—not a revolution but welcome evolution for treating chorea in Huntington disease. JAMA Neurol. 2016;73:1404-1406.
    • (2016) JAMA Neurol. , vol.73 , pp. 1404-1406
    • Reilmann, R.1
  • 14
    • 85018390919 scopus 로고    scopus 로고
    • KINECT 3: a phase 3 randomized, double-blind, placebo-controlled trial of valbenazine for tardive dyskinesia
    • Hauser RA, Factor SA, Marder SR, et al. KINECT 3: a phase 3 randomized, double-blind, placebo-controlled trial of valbenazine for tardive dyskinesia. Am J Psychiatry 2017;174:476-484
    • (2017) Am J Psychiatry , vol.174 , pp. 476-484
    • Hauser, R.A.1    Factor, S.A.2    Marder, S.R.3
  • 15
    • 84944514959 scopus 로고    scopus 로고
    • NBI-98854, a selective monoamine transport inhibitor for the treatment of tardive dyskinesia: A randomized, double-blind, placebo-controlled study
    • O'Brien CF, Jimenez R, Hauser RA, et al. NBI-98854, a selective monoamine transport inhibitor for the treatment of tardive dyskinesia: A randomized, double-blind, placebo-controlled study. Mov Disord 2015;30:1681-1687.
    • (2015) Mov Disord , vol.30 , pp. 1681-1687
    • O'Brien, C.F.1    Jimenez, R.2    Hauser, R.A.3
  • 16
    • 84918785958 scopus 로고    scopus 로고
    • Safety, tolerability, and efficacy of PBT2 in Huntington's disease: a phase 2, randomised, double-blind, placebo-controlled trial
    • Huntington Study Group. Safety, tolerability, and efficacy of PBT2 in Huntington's disease: a phase 2, randomised, double-blind, placebo-controlled trial. Lancet Neurol 2015;14:39-47.
    • (2015) Lancet Neurol , vol.14 , pp. 39-47
  • 17
    • 73649139180 scopus 로고    scopus 로고
    • The dopaminergic stabilizers pridopidine (ACR16) and (-)-OSU6162 display dopamine D(2) receptor antagonism and fast receptor dissociation properties
    • Dyhring T, Nielsen EO, Sonesson C, et al. The dopaminergic stabilizers pridopidine (ACR16) and (-)-OSU6162 display dopamine D(2) receptor antagonism and fast receptor dissociation properties. Eur J Pharmacol 2010;628:19-26.
    • (2010) Eur J Pharmacol , vol.628 , pp. 19-26
    • Dyhring, T.1    Nielsen, E.O.2    Sonesson, C.3
  • 18
    • 84995734018 scopus 로고    scopus 로고
    • Pridopidine activates neuroprotective pathways impaired in Huntington Disease
    • Geva M, Kusko R, Soares H, et al. Pridopidine activates neuroprotective pathways impaired in Huntington Disease. Hum Mol Genet 2016;25:3975-3987.
    • (2016) Hum Mol Genet , vol.25 , pp. 3975-3987
    • Geva, M.1    Kusko, R.2    Soares, H.3
  • 19
    • 84871295100 scopus 로고    scopus 로고
    • The dopamine stabilizers ACR16 and (-)-OSU6162 display nanomolar affinities at the sigma-1 receptor
    • Sahlholm K, Arhem P, Fuxe K, Marcellino D. The dopamine stabilizers ACR16 and (-)-OSU6162 display nanomolar affinities at the sigma-1 receptor. Mol Psychiatry 2013;18:12-14.
    • (2013) Mol Psychiatry , vol.18 , pp. 12-14
    • Sahlholm, K.1    Arhem, P.2    Fuxe, K.3    Marcellino, D.4
  • 20
    • 81255195330 scopus 로고    scopus 로고
    • Pridopidine for the treatment of motor function in patients with Huntington's disease (MermaiHD): a phase 3, randomised, double-blind, placebo-controlled trial
    • de Yebenes JG, Landwehrmeyer B, Squitieri F, et al. Pridopidine for the treatment of motor function in patients with Huntington's disease (MermaiHD): a phase 3, randomised, double-blind, placebo-controlled trial. Lancet Neurol 2011;10:1049-1057.
    • (2011) Lancet Neurol , vol.10 , pp. 1049-1057
    • de Yebenes, J.G.1    Landwehrmeyer, B.2    Squitieri, F.3
  • 21
    • 84884724757 scopus 로고    scopus 로고
    • A randomized, double-blind, placebo-controlled trial of pridopidine in Huntington's disease
    • Huntington Study Group. A randomized, double-blind, placebo-controlled trial of pridopidine in Huntington's disease. Mov Disord 2013;28:1407-1415.
    • (2013) Mov Disord , vol.28 , pp. 1407-1415
  • 22
    • 85052531793 scopus 로고    scopus 로고
    • Efficacy, safety, and tolerability of pridopidine in Huntington's disease (HD): results from the phase ii dose-ranging study, Pride-HD
    • Reilmann R, McGarry A, Landwehrmeyer GB, et al. Efficacy, safety, and tolerability of pridopidine in Huntington's disease (HD): results from the phase ii dose-ranging study, Pride-HD. Mov Disord 2017;32(Suppl S2):323-324.
    • (2017) Mov Disord , vol.32 , pp. 323-324
    • Reilmann, R.1    McGarry, A.2    Landwehrmeyer, G.B.3
  • 23
    • 84982102818 scopus 로고    scopus 로고
    • Laquinimod rescues striatal, cortical and white matter pathology and results in modest behavioural improvements in the YAC128 model of Huntington disease
    • Garcia-Miralles M, Hong X, Tan LJ, et al. Laquinimod rescues striatal, cortical and white matter pathology and results in modest behavioural improvements in the YAC128 model of Huntington disease. Sci Rep 2016;6:31652.
    • (2016) Sci Rep , vol.6 , pp. 31652
    • Garcia-Miralles, M.1    Hong, X.2    Tan, L.J.3
  • 24
    • 85037047644 scopus 로고    scopus 로고
    • Design of the LEGATO-HD study: A multinational, randomized, double-blind, placebo-controlled, parallelgroup study to evaluate the efficacy and safety of laquinimod (0.5, 1.0 and 1.5 mg/day) as treatment in patients with Huntington's disease
    • Reilmann R, Tabrizi SJ, Leavitt BR, et al. Design of the LEGATO-HD study: A multinational, randomized, double-blind, placebo-controlled, parallelgroup study to evaluate the efficacy and safety of laquinimod (0.5, 1.0 and 1.5 mg/day) as treatment in patients with Huntington's disease. Mov Disord 2015;30 (Suppl 1):537.
    • (2015) Mov Disord , vol.30 , pp. 537
    • Reilmann, R.1    Tabrizi, S.J.2    Leavitt, B.R.3
  • 25
    • 85025168735 scopus 로고    scopus 로고
    • Safety/tolerability of the anti-semaphorin 4D Antibody VX15/2503 in a randomized phase 1 trial
    • LaGanke C, Samkoff L, Edwards K, et al. Safety/tolerability of the anti-semaphorin 4D Antibody VX15/2503 in a randomized phase 1 trial. Neurol Neuroimmunol Neuroinflamm 2017; 4:e367.
    • (2017) Neurol Neuroimmunol Neuroinflamm , vol.4
    • LaGanke, C.1    Samkoff, L.2    Edwards, K.3
  • 26
    • 84922773047 scopus 로고    scopus 로고
    • Anti-semaphorin 4D immunotherapy ameliorates neuropathology and some cognitive impairment in the YAC128 mouse model of Huntington disease
    • Southwell AL, Franciosi S, Villanueva EB, et al. Anti-semaphorin 4D immunotherapy ameliorates neuropathology and some cognitive impairment in the YAC128 mouse model of Huntington disease. Neurobiol Dis 2015;76:46-56.
    • (2015) Neurobiol Dis , vol.76 , pp. 46-56
    • Southwell, A.L.1    Franciosi, S.2    Villanueva, E.B.3
  • 27
    • 0037335074 scopus 로고    scopus 로고
    • Specific progressive cAMP reduction implicates energy deficit in presymptomatic Huntington's disease knock-in mice
    • Gines S, Seong IS, Fossale E, et al. Specific progressive cAMP reduction implicates energy deficit in presymptomatic Huntington's disease knock-in mice. Hum Mol Genet 2003;12:497-508.
    • (2003) Hum Mol Genet , vol.12 , pp. 497-508
    • Gines, S.1    Seong, I.S.2    Fossale, E.3
  • 28
    • 1042289730 scopus 로고    scopus 로고
    • Decreased cAMP response element-mediated transcription: an early event in exon 1 and full-length cell models of Huntington's disease that contributes to polyglutamine pathogenesis
    • Sugars KL, Brown R, Cook LJ, Swartz J, Rubinsztein DC. Decreased cAMP response element-mediated transcription: an early event in exon 1 and full-length cell models of Huntington's disease that contributes to polyglutamine pathogenesis. J Biol Chem 2004;279:4988-4999.
    • (2004) J Biol Chem , vol.279 , pp. 4988-4999
    • Sugars, K.L.1    Brown, R.2    Cook, L.J.3    Swartz, J.4    Rubinsztein, D.C.5
  • 29
    • 84929340487 scopus 로고    scopus 로고
    • Facilitation of corticostriatal transmission following pharmacological inhibition of striatal phosphodiesterase 10A: role of nitric oxide-soluble guanylyl cyclase-cGMP signaling pathways
    • Padovan-Neto FE, Sammut S, Chakroborty S, et al. Facilitation of corticostriatal transmission following pharmacological inhibition of striatal phosphodiesterase 10A: role of nitric oxide-soluble guanylyl cyclase-cGMP signaling pathways. J Neurosci 2015;35:5781-5791.
    • (2015) J Neurosci , vol.35 , pp. 5781-5791
    • Padovan-Neto, F.E.1    Sammut, S.2    Chakroborty, S.3
  • 30
    • 62449195496 scopus 로고    scopus 로고
    • Inhibition of Phosphodiesterase 10A Increases the Responsiveness of Striatal Projection Neurons to Cortical Stimulation
    • Threlfell S, Sammut S, Menniti FS, Schmidt CJ, West AR. Inhibition of Phosphodiesterase 10A Increases the Responsiveness of Striatal Projection Neurons to Cortical Stimulation. J Pharmacol Exp Ther 2009;328:785-795.
    • (2009) J Pharmacol Exp Ther , vol.328 , pp. 785-795
    • Threlfell, S.1    Sammut, S.2    Menniti, F.S.3    Schmidt, C.J.4    West, A.R.5
  • 31
    • 84888876330 scopus 로고    scopus 로고
    • Review: modulation of striatal neuron activity by cyclic nucleotide signaling and phosphodiesterase inhibition
    • Threlfell S, West AR. Review: modulation of striatal neuron activity by cyclic nucleotide signaling and phosphodiesterase inhibition. Basal Ganglia 2013;3:137-146.
    • (2013) Basal Ganglia , vol.3 , pp. 137-146
    • Threlfell, S.1    West, A.R.2
  • 32
    • 78149452347 scopus 로고    scopus 로고
    • Inhibition of the striatal specific phosphodiesterase PDE10A ameliorates striatal and cortical pathology in R6/2 mouse model of Huntington's disease
    • Giampa C, Laurenti D, Anzilotti S, Bernardi G, Menniti FS, Fusco FR. Inhibition of the striatal specific phosphodiesterase PDE10A ameliorates striatal and cortical pathology in R6/2 mouse model of Huntington's disease. PLoS One 2010;5:e13417.
    • (2010) PLoS One , vol.5
    • Giampa, C.1    Laurenti, D.2    Anzilotti, S.3    Bernardi, G.4    Menniti, F.S.5    Fusco, F.R.6
  • 33
    • 34748846288 scopus 로고    scopus 로고
    • Pharmacological properties of BN82451: a novel multitargeting neuroprotective agent
    • Chabrier PE, Auguet M. Pharmacological properties of BN82451: a novel multitargeting neuroprotective agent. CNS Drug Rev 2007;13:317-332.
    • (2007) CNS Drug Rev , vol.13 , pp. 317-332
    • Chabrier, P.E.1    Auguet, M.2
  • 34
    • 0037971143 scopus 로고    scopus 로고
    • Increased survival and neuroprotective effects of BN82451 in a transgenic mouse model of Huntington's disease
    • Klivenyi P, Ferrante RJ, Gardian G, Browne S, Chabrier PE, Beal MF. Increased survival and neuroprotective effects of BN82451 in a transgenic mouse model of Huntington's disease. J Neurochem 2003;86:267-272.
    • (2003) J Neurochem , vol.86 , pp. 267-272
    • Klivenyi, P.1    Ferrante, R.J.2    Gardian, G.3    Browne, S.4    Chabrier, P.E.5    Beal, M.F.6
  • 35
    • 0036172346 scopus 로고    scopus 로고
    • Prolonged survival and decreased abnormal movements in transgenic model of Huntington disease, with administration of the transglutaminase inhibitor cystamine
    • Karpuj MV, Becher MW, Springer JE, et al. Prolonged survival and decreased abnormal movements in transgenic model of Huntington disease, with administration of the transglutaminase inhibitor cystamine. Nat Med 2002;8:143-149.
    • (2002) Nat Med , vol.8 , pp. 143-149
    • Karpuj, M.V.1    Becher, M.W.2    Springer, J.E.3
  • 36
    • 25644445315 scopus 로고    scopus 로고
    • Cystamine treatment is neuroprotective in the YAC128 mouse model of Huntington disease
    • Van Raamsdonk JM, Pearson J, Bailey CD, et al. Cystamine treatment is neuroprotective in the YAC128 mouse model of Huntington disease. J Neurochem 2005;95:210-220.
    • (2005) J Neurochem , vol.95 , pp. 210-220
    • Van Raamsdonk, J.M.1    Pearson, J.2    Bailey, C.D.3
  • 37
    • 33746275522 scopus 로고    scopus 로고
    • The protective effects of cystamine in the R6/2 Huntington's disease mouse involve mechanisms other than the inhibition of tissue transglutaminase
    • Bailey CD, Johnson GV. The protective effects of cystamine in the R6/2 Huntington's disease mouse involve mechanisms other than the inhibition of tissue transglutaminase. Neurobiol Aging 2006;27:871-879.
    • (2006) Neurobiol Aging , vol.27 , pp. 871-879
    • Bailey, C.D.1    Johnson, G.V.2
  • 38
    • 84922262761 scopus 로고    scopus 로고
    • A phase II, open-label evaluation of cysteamine tolerability in patients with Huntington's disease
    • Prundean A, Youssov K, Humbert S, Bonneau D, Verny C. A phase II, open-label evaluation of cysteamine tolerability in patients with Huntington's disease. Mov Disord 2015;30:288-289.
    • (2015) Mov Disord , vol.30 , pp. 288-289
    • Prundean, A.1    Youssov, K.2    Humbert, S.3    Bonneau, D.4    Verny, C.5
  • 39
    • 53249114029 scopus 로고    scopus 로고
    • Inhibition of specific HDACs and sirtuins suppresses pathogenesis in a Drosophila model of Huntington's disease
    • Pallos J, Bodai L, Lukacsovich T, et al. Inhibition of specific HDACs and sirtuins suppresses pathogenesis in a Drosophila model of Huntington's disease. Hum Mol Genet 2008;17:3767-3775.
    • (2008) Hum Mol Genet , vol.17 , pp. 3767-3775
    • Pallos, J.1    Bodai, L.2    Lukacsovich, T.3
  • 40
    • 84899965665 scopus 로고    scopus 로고
    • A potent and selective Sirtuin 1 inhibitor alleviates pathology in multiple animal and cell models of Huntington's disease
    • Smith MR, Syed A, Lukacsovich T, et al. A potent and selective Sirtuin 1 inhibitor alleviates pathology in multiple animal and cell models of Huntington's disease. Hum Mol Genet 2014;23:2995-3007.
    • (2014) Hum Mol Genet , vol.23 , pp. 2995-3007
    • Smith, M.R.1    Syed, A.2    Lukacsovich, T.3
  • 41
    • 84855563516 scopus 로고    scopus 로고
    • Sirt1 mediates neuroprotection from mutant huntingtin by activation of the TORC1 and CREB transcriptional pathway
    • Jeong H, Cohen DE, Cui L, et al. Sirt1 mediates neuroprotection from mutant huntingtin by activation of the TORC1 and CREB transcriptional pathway. Nat Med 2011;18:159-65.
    • (2011) Nat Med , vol.18 , pp. 159-165
    • Jeong, H.1    Cohen, D.E.2    Cui, L.3
  • 42
    • 84855544817 scopus 로고    scopus 로고
    • Neuroprotective role of Sirt1 in mammalian models of Huntington's disease through activation of multiple Sirt1 targets
    • Jiang M, Wang J, Fu J, et al. Neuroprotective role of Sirt1 in mammalian models of Huntington's disease through activation of multiple Sirt1 targets. Nat Med 2011;18:153-158.
    • (2011) Nat Med , vol.18 , pp. 153-158
    • Jiang, M.1    Wang, J.2    Fu, J.3
  • 43
    • 84855519786 scopus 로고    scopus 로고
    • Finding a sirtuin truth in Huntington's disease
    • La Spada AR. Finding a sirtuin truth in Huntington's disease. Nat Med 2012;18:24-26.
    • (2012) Nat Med , vol.18 , pp. 24-26
    • La Spada, A.R.1
  • 44
    • 84930012088 scopus 로고    scopus 로고
    • An exploratory double blind, randomized clinical trial with selisistat, a SirT1 inhibitor, in patients with Huntington's disease
    • Sussmuth SD, Haider S, Landwehrmeyer GB, et al. An exploratory double blind, randomized clinical trial with selisistat, a SirT1 inhibitor, in patients with Huntington's disease. Br J Clin Pharmacol 2015;79(3):465-476.
    • (2015) Br J Clin Pharmacol , vol.79 , Issue.3 , pp. 465-476
    • Sussmuth, S.D.1    Haider, S.2    Landwehrmeyer, G.B.3
  • 45
    • 84907928725 scopus 로고    scopus 로고
    • Safety and tolerability of selisistat for the treatment of Huntington's disease: results from a randomized, double-blind, placebo-controlled phase II trial
    • Reilmann R, Squitieri F, Priller J, et al. Safety and tolerability of selisistat for the treatment of Huntington's disease: results from a randomized, double-blind, placebo-controlled phase II trial. Neurology 2014,82(Suppl 10):S47.004.
    • (2014) Neurology , vol.82 , pp. S47.004
    • Reilmann, R.1    Squitieri, F.2    Priller, J.3
  • 46
    • 84924599725 scopus 로고    scopus 로고
    • A randomized, placebo-controlled trial of AFQ056 for the treatment of chorea in Huntington's disease
    • Reilmann R, Rouzade-Dominguez ML, Saft C, et al. A randomized, placebo-controlled trial of AFQ056 for the treatment of chorea in Huntington's disease. Mov Disord 2015;30:427-431.
    • (2015) Mov Disord , vol.30 , pp. 427-431
    • Reilmann, R.1    Rouzade-Dominguez, M.L.2    Saft, C.3
  • 47
    • 84907932420 scopus 로고    scopus 로고
    • Stem cells for cell replacement therapy: A therapeutic strategy for HD?
    • Rosser A, Svendsen CN. Stem cells for cell replacement therapy: A therapeutic strategy for HD? Mov Disord 2014;29:1446-1454.
    • (2014) Mov Disord , vol.29 , pp. 1446-1454
    • Rosser, A.1    Svendsen, C.N.2
  • 49
    • 84904787076 scopus 로고    scopus 로고
    • Mutant huntingtin is present in neuronal grafts in Huntington disease patients
    • Cicchetti F, Lacroix S, Cisbani G, et al. Mutant huntingtin is present in neuronal grafts in Huntington disease patients. Ann Neurol. 2014;76:31-42.
    • (2014) Ann Neurol. , vol.76 , pp. 31-42
    • Cicchetti, F.1    Lacroix, S.2    Cisbani, G.3
  • 50
    • 43249114934 scopus 로고    scopus 로고
    • Lewy body-like pathology in long-term embryonic nigral transplants in Parkinson's disease
    • Kordower JH, Chu Y, Hauser RA, Freeman TB, Olanow CW. Lewy body-like pathology in long-term embryonic nigral transplants in Parkinson's disease. Nat Med 2008;14:504-506.
    • (2008) Nat Med , vol.14 , pp. 504-506
    • Kordower, J.H.1    Chu, Y.2    Hauser, R.A.3    Freeman, T.B.4    Olanow, C.W.5
  • 51
    • 84906306620 scopus 로고    scopus 로고
    • Slowing of neurodegeneration in Parkinson's disease and Huntington's disease: future therapeutic perspectives
    • Schapira AH, Olanow CW, Greenamyre JT, Bezard E. Slowing of neurodegeneration in Parkinson's disease and Huntington's disease: future therapeutic perspectives. Lancet 2014;384:545-555.
    • (2014) Lancet , vol.384 , pp. 545-555
    • Schapira, A.H.1    Olanow, C.W.2    Greenamyre, J.T.3    Bezard, E.4
  • 52
    • 84896123184 scopus 로고    scopus 로고
    • Antisense oligonucleotide-mediated correction of transcriptional dysregulation is correlated with behavioral benefits in the YAC128 mouse model of Huntington's disease
    • Stanek LM, Yang W, Angus S, et al. Antisense oligonucleotide-mediated correction of transcriptional dysregulation is correlated with behavioral benefits in the YAC128 mouse model of Huntington's disease. J Huntingtons Dis 2013; 2:217-228.
    • (2013) J Huntingtons Dis , vol.2 , pp. 217-228
    • Stanek, L.M.1    Yang, W.2    Angus, S.3
  • 53
    • 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
  • 54
    • 85015189278 scopus 로고    scopus 로고
    • Design, characterization, and lead selection of therapeutic miRNAs targeting huntingtin for development of gene therapy for Huntington's disease
    • Miniarikova J, Zanella I, Huseinovic A, et al. Design, characterization, and lead selection of therapeutic miRNAs targeting huntingtin for development of gene therapy for Huntington's disease. Mol Ther Nucleic Acids 2016;7:e297.
    • (2016) Mol Ther Nucleic Acids , vol.7
    • Miniarikova, J.1    Zanella, I.2    Huseinovic, A.3
  • 55
    • 67349100160 scopus 로고    scopus 로고
    • Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice
    • Boudreau RL, McBride JL, Martins I, et al. Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. Mol Ther 2009;17:1053-1063.
    • (2009) Mol Ther , vol.17 , pp. 1053-1063
    • Boudreau, R.L.1    McBride, J.L.2    Martins, I.3
  • 56
    • 82955199935 scopus 로고    scopus 로고
    • Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease
    • McBride JL, Pitzer MR, Boudreau RL, et al. Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease. Mol Ther 2011;19:2152-2162.
    • (2011) Mol Ther , vol.19 , pp. 2152-2162
    • McBride, J.L.1    Pitzer, M.R.2    Boudreau, R.L.3
  • 57
    • 84907920445 scopus 로고    scopus 로고
    • Huntingtin-lowering strategies in Huntington's disease: antisense oligonucleotides, small RNAs, and gene editing
    • Aronin N, Difiglia M. Huntingtin-lowering strategies in Huntington's disease: antisense oligonucleotides, small RNAs, and gene editing. Mov Disord 2014;29:1455-1461.
    • (2014) Mov Disord , vol.29 , pp. 1455-1461
    • Aronin, N.1    Difiglia, M.2
  • 58
    • 84868538087 scopus 로고    scopus 로고
    • Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice
    • Garriga-Canut M, Agustín-Pavón C, Herrmann F, et al. Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice. Proc Natl Acad Sci USA 2012;109:E3136-F3145.
    • (2012) Proc Natl Acad Sci USA , vol.109 , pp. E3136-F3145
    • Garriga-Canut, M.1    Agustín-Pavón, C.2    Herrmann, F.3
  • 59
    • 84937734362 scopus 로고    scopus 로고
    • Gene delivery of AAV2-neurturin to putamen and substantia nigra in Parkinson's disease: a double-blind, randomized, controlled trial
    • Olanow CW, Bartus RT, Baumann T, et al. Gene delivery of AAV2-neurturin to putamen and substantia nigra in Parkinson's disease: a double-blind, randomized, controlled trial. Ann Neurol 2015;78:248-257.
    • (2015) Ann Neurol , vol.78 , pp. 248-257
    • Olanow, C.W.1    Bartus, R.T.2    Baumann, T.3
  • 60
    • 0042837887 scopus 로고    scopus 로고
    • A double blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease
    • Olanow CW, Goetz CG, Kordower JH, et al. A double blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease. Ann Neurol 2003;54:403-414.
    • (2003) Ann Neurol , vol.54 , pp. 403-414
    • Olanow, C.W.1    Goetz, C.G.2    Kordower, J.H.3
  • 61
    • 0025290717 scopus 로고
    • Huntington's disease in Venezuela: 7 years of follow-up on symptomatic and asymptomatic individuals
    • Penney JB Jr, Young AB, Shoulson I, et al. Huntington's disease in Venezuela: 7 years of follow-up on symptomatic and asymptomatic individuals. Mov Disord 1990;5:93-99.
    • (1990) Mov Disord , vol.5 , pp. 93-99
    • Penney, J.B.1    Young, A.B.2    Shoulson, I.3
  • 62
    • 85009332113 scopus 로고    scopus 로고
    • Unified Huntington's Disease Rating Scale: reliability and consistency
    • Huntington Study Group. Unified Huntington's Disease Rating Scale: reliability and consistency. Mov Disord 1996;11:136-142.
    • (1996) Mov Disord , vol.11 , pp. 136-142
  • 63
    • 85039840959 scopus 로고    scopus 로고
    • Motor, cognitive, and functional decline form a single progressive factor in Huntington disease
    • Schobel SA, Palermo G, Auinger P, et al; for the TRACK-HD, COHORT, CARE-HD, and 2CARE HSG investigators. Motor, cognitive, and functional decline form a single progressive factor in Huntington disease. Neurology 2017;89(24):2495-2502.
    • (2017) Neurology , vol.89 , Issue.24 , pp. 2495-2502
    • Schobel, S.A.1    Palermo, G.2    Auinger, P.3
  • 64
    • 84867693278 scopus 로고    scopus 로고
    • Design innovations and baseline findings in a long-term Parkinson's trial: the National Institute of Neurological Disorders and Stroke Exploratory Trials in Parkinson's Disease Long-Term Study-1
    • Elm JJ; NINDS NET-PD Investigators. Design innovations and baseline findings in a long-term Parkinson's trial: the National Institute of Neurological Disorders and Stroke Exploratory Trials in Parkinson's Disease Long-Term Study-1. Mov Disord 2012;27:1513-1521.
    • (2012) Mov Disord , vol.27 , pp. 1513-1521
    • Elm, J.J.1
  • 65
    • 84880932230 scopus 로고    scopus 로고
    • Disease duration and the integrity of the nigrostriatal system in Parkinson's disease
    • Kordower JH, Olanow CW, Dodiya HB, et al. Disease duration and the integrity of the nigrostriatal system in Parkinson's disease. Brain 2013;136:2419-31
    • (2013) Brain , vol.136 , pp. 2419-2431
    • Kordower, J.H.1    Olanow, C.W.2    Dodiya, H.B.3
  • 66
    • 84898017417 scopus 로고    scopus 로고
    • Huntington disease: natural history, biomarkers and prospects for therapeutics
    • Ross CA, Aylward EH, Wild EJ, et al. Huntington disease: natural history, biomarkers and prospects for therapeutics. Nat Rev Neurol 2014;10:204-216.
    • (2014) Nat Rev Neurol , vol.10 , pp. 204-216
    • Ross, C.A.1    Aylward, E.H.2    Wild, E.J.3
  • 67
    • 78650850960 scopus 로고    scopus 로고
    • Tapping linked to function and structure in premanifest and symptomatic Huntington disease
    • Bechtel N, Scahill RI, Rosas HD, et al. Tapping linked to function and structure in premanifest and symptomatic Huntington disease. Neurology 2010;75:2150-2160.
    • (2010) Neurology , vol.75 , pp. 2150-2160
    • Bechtel, N.1    Scahill, R.I.2    Rosas, H.D.3
  • 68
    • 84879032355 scopus 로고    scopus 로고
    • Predictors of phenotypic progression and disease onset in premanifest and early-stage Huntington's disease in the TRACK-HD study: analysis of 36-month observational data
    • Tabrizi SJ, Scahill RI, Owen G, et al. Predictors of phenotypic progression and disease onset in premanifest and early-stage Huntington's disease in the TRACK-HD study: analysis of 36-month observational data. Lancet Neurol 2013;12:637-649.
    • (2013) Lancet Neurol , vol.12 , pp. 637-649
    • Tabrizi, S.J.1    Scahill, R.I.2    Owen, G.3
  • 69
    • 84907940340 scopus 로고    scopus 로고
    • Diagnostic criteria for Huntington's disease based on natural history
    • Reilmann R, Leavitt BR, Ross CA. Diagnostic criteria for Huntington's disease based on natural history. Mov Disord 2014;29:1335-1341.
    • (2014) Mov Disord , vol.29 , pp. 1335-1341
    • Reilmann, R.1    Leavitt, B.R.2    Ross, C.A.3
  • 70
    • 84873455635 scopus 로고    scopus 로고
    • Clinical impairment in premanifest and early Huntington's disease is associated with regionally specific atrophy
    • Scahill RI, Hobbs NZ, Say MJ, et al. Clinical impairment in premanifest and early Huntington's disease is associated with regionally specific atrophy. Hum Brain Mapp 2013;34:519-529.
    • (2013) Hum Brain Mapp , vol.34 , pp. 519-529
    • Scahill, R.I.1    Hobbs, N.Z.2    Say, M.J.3
  • 71
    • 84882667713 scopus 로고    scopus 로고
    • The structural correlates of functional deficits in early Huntington's disease
    • Delmaire C, Dumas EM, Sharman MA, et al. The structural correlates of functional deficits in early Huntington's disease. Hum Brain Mapp 2013;34:2141-2153.
    • (2013) Hum Brain Mapp , vol.34 , pp. 2141-2153
    • Delmaire, C.1    Dumas, E.M.2    Sharman, M.A.3
  • 72
    • 84924599725 scopus 로고    scopus 로고
    • A randomized, placebo-controlled trial of AFQ056 for the treatment of chorea in Huntington's disease
    • Reilmann R, Rouzade-Dominguez ML, Saft C, et al. A randomized, placebo-controlled trial of AFQ056 for the treatment of chorea in Huntington's disease. Mov Disord 2015;30:427-431.
    • (2015) Mov Disord , vol.30 , pp. 427-431
    • Reilmann, R.1    Rouzade-Dominguez, M.L.2    Saft, C.3
  • 73
    • 84929012551 scopus 로고    scopus 로고
    • Quantification of mutant huntingtin protein in cerebrospinal fluid from Huntington's disease patients
    • Wild EJ, Boggio R, Langbehn D, et al. Quantification of mutant huntingtin protein in cerebrospinal fluid from Huntington's disease patients. J Clin Invest 2015;125:1979-1986.
    • (2015) J Clin Invest , vol.125 , pp. 1979-1986
    • Wild, E.J.1    Boggio, R.2    Langbehn, D.3
  • 74
    • 84948830873 scopus 로고    scopus 로고
    • Ultrasensitive measurement of huntingtin protein in cerebrospinal fluid demonstrates increase with Huntington disease stage and decrease following brain huntingtin suppression
    • Southwell AL, Smith SE, Davis TR, et al. Ultrasensitive measurement of huntingtin protein in cerebrospinal fluid demonstrates increase with Huntington disease stage and decrease following brain huntingtin suppression. Sci Rep 2015; 5:12166.
    • (2015) Sci Rep , vol.5 , pp. 12166
    • Southwell, A.L.1    Smith, S.E.2    Davis, T.R.3
  • 75
    • 68249113963 scopus 로고    scopus 로고
    • Biological and clinical manifestations of Huntington's disease in the longitudinal TRACK-HD study: cross-sectional analysis of baseline data
    • Tabrizi SJ, Langbehn DR, Leavitt BR, et al. Biological and clinical manifestations of Huntington's disease in the longitudinal TRACK-HD study: cross-sectional analysis of baseline data. Lancet Neurol 2009;8:791-801.
    • (2009) Lancet Neurol , vol.8 , pp. 791-801
    • Tabrizi, S.J.1    Langbehn, D.R.2    Leavitt, B.R.3
  • 76
    • 84901341459 scopus 로고    scopus 로고
    • Clinical and biomarker changes in premanifest Huntington disease show trial feasibility: a decade of the PREDICT-HD study
    • Paulsen JS, Long JD, Johnson HJ, et al. Clinical and biomarker changes in premanifest Huntington disease show trial feasibility: a decade of the PREDICT-HD study. Front Aging Neurosci 2014 6:78.
    • (2014) Front Aging Neurosci , vol.6 , pp. 78
    • Paulsen, J.S.1    Long, J.D.2    Johnson, H.J.3
  • 77
    • 84919418279 scopus 로고    scopus 로고
    • Prediction of manifest Huntington's disease with clinical and imaging measures: a prospective observational study
    • Paulsen JS, Long JD, Ross CA, et al. Prediction of manifest Huntington's disease with clinical and imaging measures: a prospective observational study. Lancet Neurol 2014;13:1193-1201.
    • (2014) Lancet Neurol , vol.13 , pp. 1193-1201
    • Paulsen, J.S.1    Long, J.D.2    Ross, C.A.3
  • 79
    • 84875514732 scopus 로고    scopus 로고
    • Regulatory innovation and drug development for early-stage Alzheimer's disease
    • Kozauer N, Katz R. Regulatory innovation and drug development for early-stage Alzheimer's disease. N Engl J Med 2013;368:1169-1171.
    • (2013) N Engl J Med , vol.368 , pp. 1169-1171
    • Kozauer, N.1    Katz, R.2
  • 80
    • 79551554590 scopus 로고    scopus 로고
    • The importance of integrating basic and clinical research toward the development of new therapies for Huntington disease
    • Munoz-Sanjuan I, Bates GP. The importance of integrating basic and clinical research toward the development of new therapies for Huntington disease. J Clin Invest 2011;121:476-483.
    • (2011) J Clin Invest , vol.121 , pp. 476-483
    • Munoz-Sanjuan, I.1    Bates, G.P.2
  • 81
    • 84855922033 scopus 로고    scopus 로고
    • Pharmacologic approaches to the treatment of Huntington's disease
    • Venuto CS, McGarry A, Ma Q, Kieburtz K. Pharmacologic approaches to the treatment of Huntington's disease. Mov Disord 2012;27:31-41.
    • (2012) Mov Disord , vol.27 , pp. 31-41
    • Venuto, C.S.1    McGarry, A.2    Ma, Q.3    Kieburtz, K.4
  • 82
    • 84907923806 scopus 로고    scopus 로고
    • Clinical trials in Huntington's disease: Interventions in early clinical development and newer methodological approaches
    • Sampaio C, Borowsky B, Reilmann R. Clinical trials in Huntington's disease: Interventions in early clinical development and newer methodological approaches. Mov Disord 2014;29:1419-1428.
    • (2014) Mov Disord , vol.29 , pp. 1419-1428
    • Sampaio, C.1    Borowsky, B.2    Reilmann, R.3
  • 83
    • 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
  • 84
    • 31644449783 scopus 로고    scopus 로고
    • Mouse models of triplet repeat diseases
    • Bates GP, Gonitel R. Mouse models of triplet repeat diseases. Mol Biotechnol 2006;32:147-58.
    • (2006) Mol Biotechnol , vol.32 , pp. 147-158
    • Bates, G.P.1    Gonitel, R.2
  • 85
    • 0037444426 scopus 로고    scopus 로고
    • Transgenic rat model of Huntington's disease
    • von Horsten S, Schmitt I, Nguyen HP, et al. Transgenic rat model of Huntington's disease. Hum Mol Genet 2003;12:617-624.
    • (2003) Hum Mol Genet , vol.12 , pp. 617-624
    • von Horsten, S.1    Schmitt, I.2    Nguyen, H.P.3
  • 86
    • 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
  • 87
    • 84902330133 scopus 로고    scopus 로고
    • Large genetic animal models of Huntington's disease
    • Morton AJ, Howland DS. Large genetic animal models of Huntington's disease. J Huntingtons Dis 2013;2:3-19.
    • (2013) J Huntingtons Dis , vol.2 , pp. 3-19
    • Morton, A.J.1    Howland, D.S.2
  • 88
    • 77952526316 scopus 로고    scopus 로고
    • An ovine transgenic Huntington's disease model
    • acobsen 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
    • acobsen, J.C.1    Bawden, C.S.2    Rudiger, S.R.3
  • 89
    • 84885468845 scopus 로고    scopus 로고
    • A transgenic minipig model of Huntington's disease
    • Baxa M, Hruska-Plochan M, Juhas S, et al. A transgenic minipig model of Huntington's disease. J Huntingtons Dis 2013;2:47-68.
    • (2013) J Huntingtons Dis , vol.2 , pp. 47-68
    • Baxa, M.1    Hruska-Plochan, M.2    Juhas, S.3
  • 90
    • 84954271904 scopus 로고    scopus 로고
    • Behavioral phenotyping of minipigs transgenic for the Huntington gene
    • Schramke S, Schuldenzucker V, Schubert R, et al. Behavioral phenotyping of minipigs transgenic for the Huntington gene. J Neurosci Methods 2016;265:34-45.
    • (2016) J Neurosci Methods , vol.265 , pp. 34-45
    • Schramke, S.1    Schuldenzucker, V.2    Schubert, R.3
  • 91
    • 84960154359 scopus 로고    scopus 로고
    • Neuroimaging of a minipig model of Huntington's disease: feasibility of volumetric, diffusion-weighted and spectroscopic assessments
    • Schubert R, Frank F, Nagelmann N, et al. Neuroimaging of a minipig model of Huntington's disease: feasibility of volumetric, diffusion-weighted and spectroscopic assessments. J Neurosci Methods 2016;265:46-55.
    • (2016) J Neurosci Methods , vol.265 , pp. 46-55
    • Schubert, R.1    Frank, F.2    Nagelmann, N.3
  • 92
    • 84907939186 scopus 로고    scopus 로고
    • Mind the gap: models in multiple species needed for therapeutic development in Huntington's disease
    • Howland DS, Munoz-Sanjuan I. Mind the gap: models in multiple species needed for therapeutic development in Huntington's disease. Mov Disord 2014;29:1397-403.
    • (2014) Mov Disord , vol.29 , pp. 1397-1403
    • Howland, D.S.1    Munoz-Sanjuan, I.2
  • 93
    • 84873473495 scopus 로고    scopus 로고
    • Obstacles to the development of a neuroprotective therapy for Parkinson's disease
    • Stocchi F, Olanow CW. Obstacles to the development of a neuroprotective therapy for Parkinson's disease. Mov Disord 2013;28:3-7.
    • (2013) Mov Disord , vol.28 , pp. 3-7
    • Stocchi, F.1    Olanow, C.W.2


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