-
1
-
-
7244236320
-
Inclusion body formation reduces levels of mutant huntingtin and the risk of neuronal death
-
DOI 10.1038/nature02998
-
Arrasate M, Mitra S, Schweitzer ES, Segal MR, Finkbeiner S (2004) Inclusion body formation reduces levels of mutant huntingtin and the risk of neuronal death. Nature 431:805-810. (Pubitemid 39434070)
-
(2004)
Nature
, vol.431
, Issue.7010
, pp. 805-810
-
-
Arrasate, M.1
Mitra, S.2
Schweitzer, E.S.3
Segal, M.R.4
Finkbeiner, S.5
-
2
-
-
0035894739
-
Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: The retina as a model
-
Auricchio A, Kobinger G, Anand V, Hildinger M, O'Connor E, Maguire AM, Wilson JM, Bennett J (2001) Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model. Hum Mol Genet 10:3075-3081. (Pubitemid 34083493)
-
(2001)
Human Molecular Genetics
, vol.10
, Issue.26
, pp. 3075-3081
-
-
Auricchio, A.1
Kobinger, G.2
Anand, V.3
Hildinger, M.4
O'Connor, E.5
Maguire, A.M.6
Wilson, J.M.7
Bennett, J.8
-
3
-
-
33748083041
-
Long-Term Clinical Improvement in MPTP-Lesioned Primates after Gene Therapy with AAV-hAADC
-
DOI 10.1016/j.ymthe.2006.05.005, PII S1525001606001778
-
Bankiewicz KS, Forsayeth J, Eberling JL, Sanchez-Pernaute R, Pivirotto P, Bringas J, Herscovitch P, Carson RE, Eckelman W, Reutter B, Cunningham J (2006) Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC. Mol Ther 14:564-570. (Pubitemid 44296090)
-
(2006)
Molecular Therapy
, vol.14
, Issue.4
, pp. 564-570
-
-
Bankiewicz, K.S.1
Forsayeth, J.2
Eberling, J.L.3
Sanchez-Pernaute, R.4
Pivirotto, P.5
Bringas, J.6
Herscovitch, P.7
Carson, R.E.8
Eckelman, W.9
Reutter, B.10
Cunningham, J.11
-
4
-
-
0032550793
-
Selective and rapid uptake of adeno-associated virus type 2 in brain
-
Bartlett JS, Samulski RJ, McCown TJ (1998) Selective and rapid uptake of adeno-associated virus type 2 in brain. Hum Gene Ther 9:1181-1186.
-
(1998)
Hum Gene Ther
, vol.9
, pp. 1181-1186
-
-
Bartlett, J.S.1
Samulski, R.J.2
McCown, T.J.3
-
5
-
-
33748561495
-
Chaperonin TRiC Promotes the Assembly of polyQ Expansion Proteins into Nontoxic Oligomers
-
DOI 10.1016/j.molcel.2006.08.017, PII S1097276506005983
-
Behrends C, Langer CA, Boteva R, Böttcher UM, Stemp MJ, Schaffar G, Rao BV, Giese A, Kretzschmar H, Siegers K, Hartl FU (2006) Chaperonin TRiC promotes the assembly of polyQ expansion proteins into nontoxic oligomers. Mol Cell 23:887-897. (Pubitemid 44363048)
-
(2006)
Molecular Cell
, vol.23
, Issue.6
, pp. 887-897
-
-
Behrends, C.1
Langer, C.A.2
Boteva, R.3
Bottcher, U.M.4
Stemp, M.J.5
Schaffar, G.6
Rao, B.V.7
Giese, A.8
Kretzschmar, H.9
Siegers, K.10
Hartl, F.U.11
-
6
-
-
67349100160
-
Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice
-
Boudreau RL, McBride JL, Martins I, Shen S, Xing Y, Carter BJ, Davidson BL (2009) Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. Mol Ther 17:1053-1063.
-
(2009)
Mol Ther
, vol.17
, pp. 1053-1063
-
-
Boudreau, R.L.1
McBride, J.L.2
Martins, I.3
Shen, S.4
Xing, Y.5
Carter, B.J.6
Davidson, B.L.7
-
7
-
-
0037041420
-
Inherent toxicity of aggregates implies a common mechanism for protein misfolding diseases
-
DOI 10.1038/416507a
-
Bucciantini M, Giannoni E, Chiti F, Baroni F, Formigli L, Zurdo J, Taddei N, Ramponi G, Dobson CM, Stefani M (2002) Inherent toxicity of aggregates implies a common mechanism for protein misfolding diseases. Nature 416:507-511. (Pubitemid 34288846)
-
(2002)
Nature
, vol.416
, Issue.6880
, pp. 507-511
-
-
Bucciantini, M.1
Giannoni, E.2
Chiti, F.3
Baroni, F.4
Formigli, L.5
Zurdo, J.6
Taddei, N.7
Ramponi, G.8
Dobson, C.M.9
Stefani, M.10
-
8
-
-
0032853699
-
Immune responses to adenovirus and adeno-associated virus in humans
-
DOI 10.1038/sj.gt.3300994
-
Chirmule N, Propert K, Magosin S, Qian Y, Qian R, Wilson J (1999) Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther 6:1574-1583. (Pubitemid 29465516)
-
(1999)
Gene Therapy
, vol.6
, Issue.9
, pp. 1574-1583
-
-
Chirmule, N.1
Propert, K.J.2
Magosin, S.A.3
Qian, Y.4
Qian, R.5
Wilson, J.M.6
-
9
-
-
19944395483
-
Potent inhibition of huntingtin aggregation and cytotoxicity by a disulfide bond-free single-domain intracellular antibody
-
DOI 10.1073/pnas.0408134101
-
Colby DW, Chu Y, Cassady JP, Duennwald M, Zazulak H, Webster JM, Messer A, Lindquist S, Ingram VM, Wittrup KD (2004) Potent inhibition of huntingtin aggregation and cytotoxicity by a disulfide bond-free single-domain intracellular antibody. Proc Natl Acad Sci U S A 101:17616-17621. (Pubitemid 40051935)
-
(2004)
Proceedings of the National Academy of Sciences of the United States of America
, vol.101
, Issue.51
, pp. 17616-17621
-
-
Colby, D.W.1
Chu, Y.2
Cassady, J.P.3
Duennwald, M.4
Zazulak, H.5
Webster, J.M.6
Messer, A.7
Lindquist, S.8
Ingram, V.M.9
Wittrup, K.D.10
-
10
-
-
0036580677
-
Lentiviral-Mediated Delivery of Mutant Huntingtin in the Striatum of Rats Induces a Selective Neuropathology Modulated by Polyglutamine Repeat Size, Huntingtin Expression Levels, and Protein Length
-
de Almeida LP, Ross CA, Zala D, Aebischer P, Déglon N (2002) Lentiviral-mediated delivery of mutant huntingtin in the striatum of rats induces a selective neuropathology modulated by polyglutamine repeat size, huntingtin expression levels, and protein length. J Neurosci 22:3473-3483. (Pubitemid 37465753)
-
(2002)
Journal of Neuroscience
, vol.22
, Issue.9
, pp. 3473-3483
-
-
De Almeida, L.P.1
Ross, C.A.2
Zala, D.3
Aebischer, P.4
Deglon, N.5
-
11
-
-
33746635502
-
Flanking sequences profoundly after polyglutamine toxicity in yeast
-
DOI 10.1073/pnas.0604547103
-
Duennwald ML, Jagadish S, Muchowski PJ, Lindquist S (2006) Flanking sequences profoundly alter polyglutamine toxicity in yeast. Proc Natl Acad Sci U S A 103:11045-11050. (Pubitemid 44148415)
-
(2006)
Proceedings of the National Academy of Sciences of the United States of America
, vol.103
, Issue.29
, pp. 11045-11050
-
-
Duennwald, M.L.1
Jagadish, S.2
Muchowski, P.J.3
Lindquist, S.4
-
13
-
-
34147130637
-
Isolation of a Human Single Chain Antibody Fragment Against Oligomeric alpha-Synuclein that Inhibits Aggregation and Prevents alpha-Synuclein-induced Toxicity
-
DOI 10.1016/j.jmb.2007.02.089, PII S0022283607003075
-
Emadi S, Barkhordarian H, Wang MS, Schulz P, Sierks MR (2007) Isolation of a human single chain antibody fragment against oligomeric alpha-synuclein that inhibits aggregation and prevents alpha-synuclein-induced toxicity. J Mol Biol 368:1132-1144. (Pubitemid 46574670)
-
(2007)
Journal of Molecular Biology
, vol.368
, Issue.4
, pp. 1132-1144
-
-
Emadi, S.1
Barkhordarian, H.2
Wang, M.S.3
Schulz, P.4
Sierks, M.R.5
-
14
-
-
33745003424
-
Cleavage at the Caspase-6 Site Is Required for Neuronal Dysfunction and Degeneration Due to Mutant Huntingtin
-
DOI 10.1016/j.cell.2006.04.026, PII S0092867406005587
-
Graham RK, Deng Y, Slow EJ, Haigh B, Bissada N, Lu G, Pearson J, Shehadeh J, Bertram L, Murphy Z, Warby SC, Doty CN, Roy S, Wellington CL, Leavitt BR, Raymond LA, Nicholson DW, Hayden MR (2006) Cleavage at the caspase-6 site Is required for neuronal dysfunction and degeneration due to mutant huntingtin. Cell 125:1179-1191. (Pubitemid 43866195)
-
(2006)
Cell
, vol.125
, Issue.6
, pp. 1179-1191
-
-
Graham, R.K.1
Deng, Y.2
Slow, E.J.3
Haigh, B.4
Bissada, N.5
Lu, G.6
Pearson, J.7
Shehadeh, J.8
Bertram, L.9
Murphy, Z.10
Warby, S.C.11
Doty, C.N.12
Roy, S.13
Wellington, C.L.14
Leavitt, B.R.15
Raymond, L.A.16
Nicholson, D.W.17
Hayden, M.R.18
-
15
-
-
46749157501
-
Fulllength human mutant huntingtin with a stable polyglutamine repeat can elicit progressive and selective neuropathogenesis in BACHD mice
-
Gray M, Shirasaki DI, Cepeda C, André VM, Wilburn B, Lu XH, Tao J, Yamazaki I, Li SH, Sun YE, Li XJ, Levine MS, Yang XW (2008) Fulllength human mutant huntingtin with a stable polyglutamine repeat can elicit progressive and selective neuropathogenesis in BACHD mice. J Neurosci 28:6182-6195.
-
(2008)
J Neurosci
, vol.28
, pp. 6182-6195
-
-
Gray, M.1
Shirasaki, D.I.2
Cepeda, C.3
André, V.M.4
Wilburn, B.5
Lu, X.H.6
Tao, J.7
Yamazaki, I.8
Li, S.H.9
Sun, Y.E.10
Li, X.J.11
Levine, M.S.12
Yang, X.W.13
-
16
-
-
0033614773
-
Evidence for both the nucleus and cytoplasm as subcellular sites of pathogenesis in huntington's disease in cell culture and in transgenic mice expressing mutant huntingtin
-
Hackam AS, Hodgson JG, Singaraja R, Zhang T, Gan L, Gutekunst CA, Hersch SM, Hayden MR (1999) Evidence for both the nucleus and cytoplasm as subcellular sites of pathogenesis in Huntington's disease in cell culture and in transgenic mice expressing mutant huntingtin. Philos Trans R Soc Lond B Biol Sci 354:1047-1055. (Pubitemid 29477966)
-
(1999)
Philosophical Transactions of the Royal Society B: Biological Sciences
, vol.354
, Issue.1386
, pp. 1047-1055
-
-
Hackam, A.S.1
Hodgson, J.G.2
Singaraja, R.3
Zhang, T.4
Gan, L.5
Gutekunst, C.-A.6
Hersch, S.M.7
Hayden, M.R.8
-
17
-
-
66149181896
-
Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs
-
Hu J, Matsul M, Gagnon KT, Schwartz JC, Gabillet S, Arar K, Bezprozvanny I, Corey DR (2009) Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs. Nat Biotechnol 27:478-484.
-
(2009)
Nat Biotechnol
, vol.27
, pp. 478-484
-
-
Hu, J.1
Matsul, M.2
Gagnon, K.T.3
Schwartz, J.C.4
Gabillet, S.5
Arar, K.6
Bezprozvanny, I.7
Corey, D.R.8
-
18
-
-
65649121163
-
Intranuclear degradation of polyglutamine aggregates by the ubiquitin-proteasome system
-
Iwata A, Nagashima Y, Matsumoto L, Suzuki T, Yamanaka T, Date H, Deoka K, Nukina N, Tsuji S (2009) Intranuclear degradation of polyglutamine aggregates by the ubiquitin-proteasome system. J Biol Chem 284:9796-9803.
-
(2009)
J Biol Chem
, vol.284
, pp. 9796-9803
-
-
Iwata, A.1
Nagashima, Y.2
Matsumoto, L.3
Suzuki, T.4
Yamanaka, T.5
Date, H.6
Deoka, K.7
Nukina, N.8
Tsuji, S.9
-
19
-
-
34250683023
-
Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: An open label, phase I trial
-
DOI 10.1016/S0140-6736(07)60982-9, PII S0140673607609829
-
Kaplitt MG, Feigin A, Tang C, Fitzsimons HL, Mattis P, Lawlor PA, Bland RJ, Young D, Strybing K, Eidelberg D, During MJ (2007) Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 369:2097-2105. (Pubitemid 46935946)
-
(2007)
Lancet
, vol.369
, Issue.9579
, pp. 2097-2105
-
-
Kaplitt, M.G.1
Feigin, A.2
Tang, C.3
Fitzsimons, H.L.4
Mattis, P.5
Lawlor, P.A.6
Bland, R.J.7
Young, D.8
Strybing, K.9
Eidelberg, D.10
During, M.J.11
-
20
-
-
0242668337
-
Common structure of soluble amyloid oligomers implies common mechanism of pathogenesis
-
DOI 10.1126/science.1079469
-
Kayed R, Head E, Thompson JL, McIntire TM, Milton SC, Cotman CW, Glabe CG (2003) Common structure of soluble amyloid oligomers implies common mechanism of pathogenesis. Science 300:486-489. (Pubitemid 36444329)
-
(2003)
Science
, vol.300
, Issue.5618
, pp. 486-489
-
-
Kayed, R.1
Head, E.2
Thompson, J.L.3
McIntire, T.M.4
Milton, S.C.5
Cotman, C.W.6
Glabel, C.G.7
-
22
-
-
33749176269
-
Cytosolic chaperonin prevents polyglutamine toxicity with altering the aggregation state
-
DOI 10.1038/ncb1478, PII NCB1478
-
Kitamura A, Kubota H, Pack CG, Matsumoto G, Hirayama S, Takahashi Y, Kimura H, Kinjo M, Morimoto RI, Nagata K (2006) Cytosolic chaperonin prevents polyglutamine toxicity with altering the aggregation state. Nat Cell Biol 8:1163-1170. (Pubitemid 44473613)
-
(2006)
Nature Cell Biology
, vol.8
, Issue.10
, pp. 1163-1170
-
-
Kitamura, A.1
Kubota, H.2
Pack, C.-G.3
Matsumoto, G.4
Hirayama, S.5
Takahashi, Y.6
Kimura, H.7
Kinjo, M.8
Morimoto, R.I.9
Nagata, K.10
-
23
-
-
0032051603
-
Neuron-specific transduction in the rat septohippocampal or nigrostriatal pathway by recombinant adeno-associated virus vectors
-
DOI 10.1006/exnr.1997.6736
-
Klein RL, Meyer EM, Peel AL, Zolotukhin S, Meyers C, Muzyczka N, King MA (1998) Neuron-specific transduction in the rat septohippocampal or nigrostriatal pathway by recombinant adeno-associated virus vectors. Exp Neurol 150:183-194. (Pubitemid 28231949)
-
(1998)
Experimental Neurology
, vol.150
, Issue.2
, pp. 183-194
-
-
Klein, R.L.1
Meyer, E.M.2
Peel, A.L.3
Zolotukhin, S.4
Meyers, C.5
Muzyczka, N.6
King, M.A.7
-
24
-
-
0036315645
-
Dose and promoter effects of adeno-associated viral vector for green fluorescent protein expression in the rat brain
-
DOI 10.1006/exnr.2002.7942
-
Klein RL, Hamby ME, Gong Y, Hirko AC, Wang S, Hughes JA, King MA, Meyer EM (2002) Dose and promoter effects of adeno-associated viral vector for green fluorescent protein expression in the rat brain. Exp Neurol 176:66-74. (Pubitemid 34762106)
-
(2002)
Experimental Neurology
, vol.176
, Issue.1
, pp. 66-74
-
-
Klein, R.L.1
Hamby, M.E.2
Gong, Y.3
Hirko, A.C.4
Wang, S.5
Hughes, J.A.6
King, M.A.7
Meyer, E.M.8
-
25
-
-
0035502934
-
New anti-huntingtin monoclonal antibodies: Implications for huntingtin conformation and its binding proteins
-
DOI 10.1016/S0361-9230(01)00599-8, PII S0361923001005998
-
Ko J, Ou S, Patterson PH (2001) New anti-huntingtin monoclonal antibodies: implications for huntingtin conformation and its binding proteins. Brain Res Bull 56:319-329. (Pubitemid 33062356)
-
(2001)
Brain Research Bulletin
, vol.56
, Issue.3-4
, pp. 319-329
-
-
Ko, J.1
Ou, S.2
Patterson, P.H.3
-
26
-
-
0035836675
-
Human single-chain Fv intrabodies counteract in situ huntingtin aggregation in cellular models of Huntington's disease
-
DOI 10.1073/pnas.071058398
-
Lecerf JM, Shirley TL, Zhu Q, Kazantsev A, Amersdorfer P, Housman DE, Messer A, Huston JS (2001) Human single-chain Fv intrabodies counteract in situ huntingtin aggregation in cellular models of Huntington's disease. Proc Natl Acad Sci U S A 98:4764-4769. (Pubitemid 32295050)
-
(2001)
Proceedings of the National Academy of Sciences of the United States of America
, vol.98
, Issue.8
, pp. 4764-4769
-
-
Lecerf, J.-M.1
Shirley, T.L.2
Zhu, Q.3
Kazantsev, A.4
Amersdorfer, P.5
Housman, D.E.6
Messer, A.7
Huston, J.S.8
-
27
-
-
69249139853
-
Monoclonal antibodies recognize distinct conformational epitopes formed by polyglutamine in a mutant huntingtin fragment
-
Legleiter J, Lotz GP, Miller J, Ko J, Ng C, Williams GL, Finkbeiner S, Patterson PH, Muchowski PJ (2009) Monoclonal antibodies recognize distinct conformational epitopes formed by polyglutamine in a mutant huntingtin fragment. J Biol Chem 284:21647-21658.
-
(2009)
J Biol Chem
, vol.284
, pp. 21647-21658
-
-
Legleiter, J.1
Lotz, G.P.2
Miller, J.3
Ko, J.4
Ng, C.5
Williams, G.L.6
Finkbeiner, S.7
Patterson, P.H.8
Muchowski, P.J.9
-
28
-
-
16044373842
-
Exon I of the HD gene with an expanded CAG repeat is sufficient to cause a progressive neurological phenotype in transgenic mice
-
DOI 10.1016/S0092-8674(00)81369-0
-
Mangiarini L, Sathasivam K, Seller M, Cozens B, Harper A, Hetherington C, Lawton M, Trottier Y, Lehrach H, Davies SW, Bates GP (1996) Exon 1 of the HD gene with an expanded CAG repeat is sufficient to cause a progressive neurological phenotype in transgenic mice. Cell 87:493-506. (Pubitemid 26374323)
-
(1996)
Cell
, vol.87
, Issue.3
, pp. 493-506
-
-
Mangiarini, L.1
Sathasivam, K.2
Seller, M.3
Cozens, B.4
Harper, A.5
Hetherington, C.6
Lawton, M.7
Trottier, Y.8
Lehrach, H.9
Davies, S.W.10
Bates, G.P.11
-
29
-
-
41949110690
-
Safety and tolerability of intraputaminal delivery of CERE-120 (adenoassociated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: An open-label, phase I trial
-
Marks WJ Jr, Ostrem JL, Verhagen L, Starr PA, Larson PS, Bakay RA, Taylor R, Cahn-Weiner DA, Stoessl AJ, Olanow CW, Bartus RT (2008) Safety and tolerability of intraputaminal delivery of CERE-120 (adenoassociated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol 7:400-408.
-
(2008)
Lancet Neurol
, vol.7
, pp. 400-408
-
-
Marks Jr., W.J.1
Ostrem, J.L.2
Verhagen, L.3
Starr, P.A.4
Larson, P.S.5
Bakay, R.A.6
Taylor, R.7
Cahn-Weiner, D.A.8
Stoessl, A.J.9
Olanow, C.W.10
Bartus, R.T.11
-
30
-
-
0030003480
-
Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector
-
DOI 10.1016/0006-8993(95)01488-8
-
McCown TJ, Xiao X, Li J, Breese GR, Samulski RJ (1996) Differential and persistent expression patterns of CNS gene transfer by an adenoassociated virus (AAV) vector. Brain Res 713:99-107. (Pubitemid 26114833)
-
(1996)
Brain Research
, vol.713
, Issue.1-2
, pp. 99-107
-
-
McCown, T.J.1
Xiao, X.2
Li, J.3
Breese, G.R.4
Samulski, R.J.5
-
31
-
-
44949240513
-
Combinational approach of intrabody with enhanced Hsp70 expression addresses multiple pathologies in a fly model of Huntington's disease
-
DOI 10.1096/fj.07-099689
-
McLear JA, Lebrecht D, Messer A, Wolfgang WJ (2008) Combinational approach of intrabody with enhanced Hsp70 expression addresses multiple pathologies in a fly model of Huntington's disease. FASEB J 22:2003-2011. (Pubitemid 351811476)
-
(2008)
FASEB Journal
, vol.22
, Issue.6
, pp. 2003-2011
-
-
McLear, J.A.1
Lebrecht, D.2
Messer, A.3
Wolfgang, W.J.4
-
32
-
-
33744825168
-
Immune responses to AAV in a phase I study for Canavan disease
-
DOI 10.1002/jgm.885
-
McPhee SW, Janson CG, Li C, Samulski RJ, Camp AS, Francis J, Shera D, Lioutermann L, Feely M, Freese A, Leone P (2006) Immune responses to AAV in a phase I study for Canavan disease. J Gene Med 8:577-588. (Pubitemid 43827251)
-
(2006)
Journal of Gene Medicine
, vol.8
, Issue.5
, pp. 577-588
-
-
McPhee, S.W.J.1
Janson, C.G.2
Li, C.3
Samulski, R.J.4
Camp, A.S.5
Francis, J.6
Shera, D.7
Lioutermann, L.8
Feely, M.9
Freese, A.10
Leone, P.11
-
33
-
-
17544377783
-
A human single-chain Fv intrabody preferentially targets amino-terminal huntingtin fragments in striatal models of Huntington's disease
-
DOI 10.1016/j.nbd.2004.11.003
-
Miller TW, Zhou C, Gines S, MacDonald ME, Mazarakis ND, Bates GP, Huston JS, Messer A (2005) A human single-chain Fv intrabody preferentially targets amino-terminal Huntingtin's fragments in striatal models of Huntington's disease. Neurobiol Dis 19:47-56. (Pubitemid 40552820)
-
(2005)
Neurobiology of Disease
, vol.19
, Issue.1-2
, pp. 47-56
-
-
Miller, T.W.1
Zhou, C.2
Gines, S.3
MacDonald, M.E.4
Mazarakis, N.D.5
Bates, G.P.6
Huston, J.S.7
Messer, A.8
-
34
-
-
44049094771
-
Clinical gene therapy using recombinant adeno-associated virus vectors
-
DOI 10.1038/gt.2008.68, PII GT200868, Special Issue: AAV Vectors for Clinical GeneTherapy
-
Mueller C, Flotte TR (2008) Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther 15:858-863. (Pubitemid 351712619)
-
(2008)
Gene Therapy
, vol.15
, Issue.11
, pp. 858-863
-
-
Mueller, C.1
Flotte, T.R.2
-
35
-
-
0036244335
-
Hippocampal damage and exploratory preferences in rats: Memory for objects, places, and contexts
-
DOI 10.1101/lm.41302
-
Mumby DG, Gaskin S, Glenn MJ, Schramek TE, Lehmann H (2002) Hippocampal damage and exploratory preferences in rats: memory for objects, places, and contexts. Learn Mem 9:49-57. (Pubitemid 34507491)
-
(2002)
Learning and Memory
, vol.9
, Issue.2
, pp. 49-57
-
-
Mumby, D.G.1
Gaskin, S.2
Glenn, M.J.3
Schramek, T.E.4
Lehmann, H.5
-
36
-
-
35548984920
-
Perirhinal cortex damage and anterograde object-recognition in rats after long retention intervals
-
DOI 10.1016/j.bbr.2007.07.026, PII S016643280700366X
-
Mumby DG, Piterkin P, Lecluse V, Lehmann H (2007) Perirhinal cortex damage and anterograde object-recognition in rats after long retention intervals. Behav Brain Res 185:82-87. (Pubitemid 350011107)
-
(2007)
Behavioural Brain Research
, vol.185
, Issue.2
, pp. 82-87
-
-
Mumby, D.G.1
Piterkin, P.2
Lecluse, V.3
Lehmann, H.4
-
37
-
-
1242351323
-
A single-chain Fv intrabody provides functional protection against the effects of mutant protein in an organotypic slice culture model of Huntington's disease
-
Murphy RC, Messer A (2004) A single-chain Fv intrabody provides functional protection against the effects of mutant protein in an organotypic slice culture model of Huntington's disease. Mol Brain Res 121:141-145.
-
(2004)
Mol Brain Res
, vol.121
, pp. 141-145
-
-
Murphy, R.C.1
Messer, A.2
-
38
-
-
0029996147
-
In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
-
Naldini L, Blömer U, Gallay P, Ory D, Mulligan R, Gage FH, Verma IM, Trono D (1996) In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272:263-267.
-
(1996)
Science
, vol.272
, pp. 263-267
-
-
Naldini, L.1
Blömer, U.2
Gallay, P.3
Ory, D.4
Mulligan, R.5
Gage, F.H.6
Verma, I.M.7
Trono, D.8
-
39
-
-
0029862497
-
A stable human-derived packaging cell line for production of high titer retrovirus/vesicular stomatitis virus G pseudotypes
-
Ory DS, Neugeboren BA, Mulligan RC (1996) A stable human-derived packaging cell line for production of high titer retrovirus/vesicular stomatitis virus G pseudotypes. Proc Natl Acad Sci U S A 93:11400-11406.
-
(1996)
Proc Natl Acad Sci U S A
, vol.93
, pp. 11400-11406
-
-
Ory, D.S.1
Neugeboren, B.A.2
Mulligan, R.C.3
-
40
-
-
15444374558
-
Beta-site specific intrabodies to decrease and prevent generation of Alzheimer's Abeta peptide
-
DOI 10.1083/jcb.200410047
-
Paganetti P, Calanca V, Galli C, Stefani M, Molinari M (2005) beta-site specific intrabodies to decrease and prevent generation of Alzheimer's Abeta peptide. J Cell Biol 168:863-868. (Pubitemid 40397001)
-
(2005)
Journal of Cell Biology
, vol.168
, Issue.6
, pp. 863-868
-
-
Paganetti, P.1
Calanca, V.2
Galli, C.3
Stefani, M.4
Molinari, M.5
-
41
-
-
0034212887
-
Adeno-associated virus vectors: Activity and applications in the CNS
-
DOI 10.1016/S0165-0270(00)00183-7, PII S0165027000001837
-
Peel AL, Klein RL (2000) Adeno-associated virus vectors: activity and applications in the CNS. J Neurosci Methods 98:95-104. (Pubitemid 30398575)
-
(2000)
Journal of Neuroscience Methods
, vol.98
, Issue.2
, pp. 95-104
-
-
Peel, A.L.1
Klein, R.L.2
-
42
-
-
0032847708
-
Nuclear targeting of mutant huntingtin increases toxicity
-
DOI 10.1006/mcne.1999.0773
-
Peters MF, Nucifora FC Jr, Kushi J, Seaman HC, Cooper JK, Herring WJ, Dawson VL, Dawson TM, Ross CA (1999) Nuclear targeting of mutant Huntingtin increases toxicity. Mol Cell Neurosci 14:121-128. (Pubitemid 29444882)
-
(1999)
Molecular and Cellular Neurosciences
, vol.14
, Issue.2
, pp. 121-128
-
-
Peters, M.F.1
Nucifora Jr., F.C.2
Kushi, J.3
Seaman, H.C.4
Cooper, J.K.5
Herring, W.J.6
Dawson, V.L.7
Dawson, T.M.8
Ross, C.A.9
-
43
-
-
67349159137
-
Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients
-
Pfister EL, Kennington L, Straubhaar J, Wagh S, Liu W, DiFiglia M, Landwehrmeyer B, Vonsattel JP, Zamore PD, Aronin N (2009) Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients. Curr Biol 19:774-778.
-
(2009)
Curr Biol
, vol.19
, pp. 774-778
-
-
Pfister, E.L.1
Kennington, L.2
Straubhaar, J.3
Wagh, S.4
Liu, W.5
Difiglia, M.6
Landwehrmeyer, B.7
Vonsattel, J.P.8
Zamore, P.D.9
Aronin, N.10
-
44
-
-
0242609127
-
Early and reversible neuropathology induced by tetracycline-regulated lentiviral overexpression of mutant huntingtin in rat striatum
-
Régulier E, Trottier Y, Perrin V, Aebischer P, Déglon N (2003) Early and reversible neuropathology induced by tetracycline-regulated lentiviral overexpression of mutant huntingtin in rat striatum. Hum Mol Genet 12:2827-2836.
-
(2003)
Hum Mol Genet
, vol.12
, pp. 2827-2836
-
-
Régulier, E.1
Trottier, Y.2
Perrin, V.3
Aebischer, P.4
Déglon, N.5
-
45
-
-
0036533795
-
Lessons from animal models of Huntington's disease
-
DOI 10.1016/S0168-9525(01)02625-7, PII S0168952501026257
-
Rubinsztein DC (2002) Lessons from animal models of Huntington's disease. Trends Genet 18:202-209. (Pubitemid 34273741)
-
(2002)
Trends in Genetics
, vol.18
, Issue.4
, pp. 202-209
-
-
Rubinsztein, D.C.1
-
46
-
-
0037461730
-
Pivotal role of oligomerization in expanded polyglutamine neurodegenerative disorders
-
DOI 10.1038/nature01301
-
Sánchez I, Mahlke C, Yuan J (2003) Pivotal role of oligomerization in expanded polyglutamine neurodegenerative disorders. Nature 421:373-379. (Pubitemid 36157935)
-
(2003)
Nature
, vol.421
, Issue.6921
, pp. 373-379
-
-
Sanchez, I.1
Mahlke, C.2
Yuan, J.3
-
47
-
-
0032475931
-
Huntingtin acts in the nucleus to induce apoptosis but death does not correlate with the formation of intranuclear inclusions
-
DOI 10.1016/S0092-8674(00)81782-1
-
Saudou F, Finkbeiner S, Devys D, Greenberg ME (1998) Huntingtin acts in the nucleus to induce apoptosis but death does not correlate with the formation of intranuclear inclusion. Cell 95:55-66. (Pubitemid 28458024)
-
(1998)
Cell
, vol.95
, Issue.1
, pp. 55-56
-
-
Saudou, F.1
Finkbeiner, S.2
Devys, D.3
Greenberg, M.E.4
-
48
-
-
4344636957
-
Nuclear-targeting of mutant huntingtin fragments produces Huntington's disease-like phenotypes in transgenic mice
-
DOI 10.1093/hmg/ddh175
-
Schilling G, Savonenko AV, Klevytska A, Morton JL, Tucker SM, Poirier M, Gale A, Chan N, Gonzales V, Slunt HH, Coonfield ML, Jenkins NA, Copeland NG, Ross CA, Borchelt DR (2004) Nuclear-targeting of mutant huntingtin fragments produces Huntington's disease-like phenotypes in transgenic mice. Hum Mol Genet 13:1599-1610. (Pubitemid 39136716)
-
(2004)
Human Molecular Genetics
, vol.13
, Issue.15
, pp. 1599-1610
-
-
Schilling, G.1
Savonenko, A.V.2
Klevytska, A.3
Morton, J.L.4
Tucker, S.M.5
Poirier, M.6
Gale, A.7
Chan, N.8
Gonzales, V.9
Slunt, H.H.10
Coonfield, M.L.11
Jenkins, N.A.12
Copeland, N.G.13
Ross, C.A.14
Borchelt, D.R.15
-
49
-
-
0037333815
-
Genetic fate of recombinant adeno-associated virus vector genomes in muscle
-
Schnepp BC, Clark KR, Klemanski DL, Pacak CA, Johnson PR (2003) Genetic fate of recombinant adeno-associated virus vector genomes in muscle. J Virol 77:3495-3504.
-
(2003)
J Virol
, vol.77
, pp. 3495-3504
-
-
Schnepp, B.C.1
Clark, K.R.2
Klemanski, D.L.3
Pacak, C.A.4
Johnson, P.R.5
-
50
-
-
10744227174
-
Selective striatal neuronal loss in a YAC128 mouse model of Huntington disease
-
DOI 10.1093/hmg/ddg169
-
Slow EJ, van Raamsdonk J, Rogers D, Coleman SH, Graham RK, Deng Y, Oh R, Bissada N, Hossain SM, Yang YZ, Li XJ, Simpson EM, Gutekunst CA, Leavitt BR, Hayden MR (2003) Selective striatal neuronal loss in a YAC128 mouse model of Huntington disease. Hum Mol Genet 12:1555-1567. (Pubitemid 36857303)
-
(2003)
Human Molecular Genetics
, vol.12
, Issue.13
, pp. 1555-1567
-
-
Slow, E.J.1
Van Raamsdonk, J.2
Rogers, D.3
Coleman, S.H.4
Graham, R.K.5
Deng, Y.6
Oh, R.7
Bissada, N.8
Hossain, S.M.9
Yang, Y.-Z.10
Li, X.-J.11
Simpson, E.M.12
Gutekunst, C.-A.13
Leavitt, B.R.14
Hayden, M.R.15
-
51
-
-
23844472610
-
Absence of behavioral abnormalities and neurodegeneration in vivo despite widespread neuronal huntingtin inclusions
-
DOI 10.1073/pnas.0503634102
-
Slow EJ, Graham RK, Osmand AP, Devon RS, Lu G, Deng Y, Pearson J, Vaid K, Bissada N, Wetzel R, Leavitt BR, Hayden MR (2005) Absence of behavioral abnormalities and neurodegeneration in vivo despite widespread neuronal huntingtin inclusions. Proc Natl Acad Sci U S A 102:11402-11407. (Pubitemid 41153832)
-
(2005)
Proceedings of the National Academy of Sciences of the United States of America
, vol.102
, Issue.32
, pp. 11402-11407
-
-
Slow, E.J.1
Graham, R.K.2
Osmand, A.P.3
Devon, R.S.4
Lu, G.5
Deng, Y.6
Pearson, J.7
Vaid, K.8
Bissada, N.9
Wetzel, R.10
Leavitt, B.R.11
Hayden, M.R.12
-
52
-
-
54049111928
-
Intrabodies binding the proline-rich domains of mutant huntingtin increase its turnover and reduce neurotoxicity
-
Southwell AL, Khoshnan A, Dunn DE, Bugg CW, Lo DC, Patterson PH (2008) Intrabodies binding the proline-rich domains of mutant huntingtin increase its turnover and reduce neurotoxicity. J Neurosci 28:9013-9020.
-
(2008)
J Neurosci
, vol.28
, pp. 9013-9020
-
-
Southwell, A.L.1
Khoshnan, A.2
Dunn, D.E.3
Bugg, C.W.4
Lo, D.C.5
Patterson, P.H.6
-
53
-
-
24144475005
-
Chronology of behavioral symptoms and neuropathological sequela in R6/2 Huntington's disease transgenic mice
-
DOI 10.1002/cne.20680
-
Stack EC, Kubilus JK, Smith K, Cormier K, Del Signore SJ, Guelin E, Ryu H, Hersch SM, Ferrante RJ (2005) Chronology of behavioral symptoms and neuropathological sequela in R6/2 Huntington's disease transgenic mice. J Comp Neurol 490:354-370. (Pubitemid 41232558)
-
(2005)
Journal of Comparative Neurology
, vol.490
, Issue.4
, pp. 354-370
-
-
Stack, E.C.1
Kubilus, J.K.2
Smith, K.3
Cormier, K.4
Del Signore, S.J.5
Guelin, E.6
Ryu, H.7
Hersch, S.M.8
Ferrante, R.J.9
-
54
-
-
38349158062
-
Soluble polyglutamine oligomers formed prior to inclusion body formation are cytotoxic
-
Takahashi T, Kikuchi S, Katada S, Nagai Y, Nishizawa M, Onodera O (2008) Soluble polyglutamine oligomers formed prior to inclusion body formation are cytotoxic. Hum Mol Genet 17:345-356.
-
(2008)
Hum Mol Genet
, vol.17
, pp. 345-356
-
-
Takahashi, T.1
Kikuchi, S.2
Katada, S.3
Nagai, Y.4
Nishizawa, M.5
Onodera, O.6
-
55
-
-
0025959353
-
FVB/N: An inbred mouse strain preferable for transgenic analyses
-
Taketo M, Schroeder AC, Mobraaten LE, Gunning KB, Hanten G, Fox RR, Roderick TH, Stewart CL, Lilly F, Hansen CT (1991) FVB/N: an inbred mouse strain preferable for transgenic analyses. Proc Natl Acad Sci U S A 88:2065-2069. (Pubitemid 21916354)
-
(1991)
Proceedings of the National Academy of Sciences of the United States of America
, vol.88
, Issue.6
, pp. 2065-2069
-
-
Taketo, M.1
Schroeder, A.C.2
Mobraaten, L.E.3
Gunning, K.B.4
Hanten, G.5
Fox, R.R.6
Roderick, T.H.7
Stewart, C.L.8
Lilly, F.9
Hansen, C.T.10
Overbeek, P.A.11
-
56
-
-
0034647801
-
Tropism of AAV-2 vectors for neurons of the globus pallidus
-
Tenenbaum L, Jurysta F, Stathopoulos A, Puschban Z, Melas C, Hermens WT, Verhaagen J, Pichon B, Velu T, Levivier M (2000) Tropism of AAV-2 vectors for neurons of the globus pallidus. Neuroreport 11:2277-2283. (Pubitemid 30485576)
-
(2000)
NeuroReport
, vol.11
, Issue.10
, pp. 2277-2283
-
-
Tenenbaum, L.1
Jurysta, F.2
Stathopoulos, A.3
Puschban, Z.4
Melas, C.5
Hermens, W.T.J.M.C.6
Verhaagen, J.7
Pichon, B.8
Velu, T.9
Levivier, M.10
-
57
-
-
4344702714
-
Recombinant AAV-mediated gene delivery to the central nervous system
-
DOI 10.1002/jgm.506
-
Tenenbaum L, Chtarto A, Lehtonen E, Velu T, Brotchi J, Levivier M (2004) Recombinant AAV-mediated gene delivery to the central nervous system. J Gene Med 6:S212-S222. (Pubitemid 40228030)
-
(2004)
Journal of Gene Medicine
, vol.6
, Issue.SUPPL. 1
-
-
Tenenbaum, L.1
Chtarto, A.2
Lehtonen, E.3
Velu, T.4
Brotchi, J.5
Levivier, M.6
-
58
-
-
64049119303
-
Polyglutamine disruption of the huntingtin exon 1Nterminus triggers a complex aggregation mechanism
-
Thakur AK, Jayaraman M, Mishra R, Thakur M, Chellgren VM, Byeon IJ, Anjum DH, Kodali R, Creamer TP, Conway JF, Gronenborn AM, Wetzel R (2009) Polyglutamine disruption of the huntingtin exon 1Nterminus triggers a complex aggregation mechanism. Nat Struct Mol Biol 16:380-389.
-
(2009)
Nat Struct Mol Biol
, vol.16
, pp. 380-389
-
-
Thakur, A.K.1
Jayaraman, M.2
Mishra, R.3
Thakur, M.4
Chellgren, V.M.5
Byeon, I.J.6
Anjum, D.H.7
Kodali, R.8
Creamer, T.P.9
Conway, J.F.10
Gronenborn, A.M.11
Wetzel, R.12
-
59
-
-
0027480960
-
A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes
-
DOI 10.1016/0092-8674(93)90585-E
-
The Huntington's Disease Collaborative Research Group (1993) A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. Cell 72:971-983. (Pubitemid 23110238)
-
(1993)
Cell
, vol.72
, Issue.6
, pp. 971-983
-
-
MacDonald, M.E.1
Ambrose, C.M.2
Duyao, M.P.3
Myers, R.H.4
Lin, C.5
Srinidhi, L.6
Barnes, G.7
Taylor, S.A.8
James, M.9
Groot, N.10
MacFarlane, H.11
Jenkins, B.12
Anderson, M.A.13
Wexler, N.S.14
Gusella, J.F.15
Bates, G.P.16
Baxendale, S.17
Hummerich, H.18
Kirby, S.19
-
60
-
-
34250666701
-
Production and purification of lentiviral vectors
-
Tiscornia G, Singer O, Verma IM (2006) Production and purification of lentiviral vectors. Nat Protoc 1:241-245.
-
(2006)
Nat Protoc
, vol.1
, pp. 241-245
-
-
Tiscornia, G.1
Singer, O.2
Verma, I.M.3
-
61
-
-
17644368893
-
Cognitive dysfunction precedes neuropathology and motor abnormalities in the YAC128 mouse model of Huntington's disease
-
DOI 10.1523/JNEUROSCI.0590-05.2005
-
Van Raamsdonk JM, Pearson J, Slow EJ, Hossain SM, Leavitt BR, Hayden MR (2005) Cognitive dysfunction precedes neuropathology and motor abnormalities in the YAC128 mouse model of Huntington's disease. J Neurosci 25:4169-4180. (Pubitemid 40563393)
-
(2005)
Journal of Neuroscience
, vol.25
, Issue.16
, pp. 4169-4180
-
-
Van Raamsdonk, J.M.1
Pearson, J.2
Slow, E.J.3
Hossain, S.M.4
Leavitt, B.R.5
Hayden, M.R.6
-
62
-
-
0042525989
-
Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain
-
DOI 10.1038/sj.gt.3302011
-
Wang C, Wang CM, Clark KR, Sferra TJ (2003) Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain. Gene Ther 10:1528-1534. (Pubitemid 36987147)
-
(2003)
Gene Therapy
, vol.10
, Issue.17
, pp. 1528-1534
-
-
Wang, C.1
Wang, C.-M.2
Clark, K.R.3
Sferra, T.J.4
-
63
-
-
44649113841
-
Suppression of neuropil aggregates and neurological symptoms by an intracellular antibody implicates the cytoplasmic toxicity of mutant huntingtin
-
DOI 10.1083/jcb.200710158
-
Wang CE, Zhou H, McGuire JR, Cerullo V, Lee B, Li SH, Li XJ (2008) Suppression of neuropil aggregates and neurological symptoms by an intracellular antibody implicates the cytoplasmic toxicity of mutant huntingtin. J Cell Biol 181:803-816. (Pubitemid 351786985)
-
(2008)
Journal of Cell Biology
, vol.181
, Issue.5
, pp. 803-816
-
-
Wang, C.-E.1
Zhou, H.2
McGuire, J.R.3
Cerullo, V.4
Lee, B.5
Li, S.-H.6
Li, X.-J.7
-
64
-
-
61549104503
-
CAG expansion in the Huntington's disease gene is associated with a specific and targetable predisposing haplogroup
-
Warby SC, Montpetit A, Hayden AR, Carroll JB, Butland SL, Visscher H, Collins JA, Semaka A, Hudson TJ, Hayden MR (2009) CAG expansion in the Huntington's disease gene is associated with a specific and targetable predisposing haplogroup. Am J Hum Genet 84:351-366.
-
(2009)
Am J Hum Genet
, vol.84
, pp. 351-366
-
-
Warby, S.C.1
Montpetit, A.2
Hayden, A.R.3
Carroll, J.B.4
Butland, S.L.5
Visscher, H.6
Collins, J.A.7
Semaka, A.8
Hudson, T.J.9
Hayden, M.R.10
-
65
-
-
23844543718
-
Suppression of Huntington's disease pathology in Drosophila by human single-chain Fv antibodies
-
Wolfgang WJ, Miller TW, Webster JM, Huston JS, Thompson LM, Marsh JL, Messer A (2005) Suppression of Huntington's disease pathology in Drosophila by human single-chain Fv antibodies. Proc Natl Acad Sci U S A 102:11563-11568.
-
(2005)
Proc Natl Acad Sci U S A
, vol.102
, pp. 11563-11568
-
-
Wolfgang, W.J.1
Miller, T.W.2
Webster, J.M.3
Huston, J.S.4
Thompson, L.M.5
Marsh, J.L.6
Messer, A.7
-
66
-
-
10344257196
-
A human single-chain Fv intrabody blocks aberrant cellular effects of overexpressed alpha-synuclein
-
DOI 10.1016/j.ymthe.2004.08.019, PII S1525001604014157
-
Zhou C, Emadi S, Sierks MR, Messer A (2004) A human single-chain Fv intrabody blocks aberrant cellular effects of overexpressed alpha-synuclein. Mol Ther 10:1023-1031. (Pubitemid 39625246)
-
(2004)
Molecular Therapy
, vol.10
, Issue.6
, pp. 1023-1031
-
-
Zhou, C.1
Emadi, S.2
Sierks, M.R.3
Messer, A.4
|