메뉴 건너뛰기




Volumn 28, Issue 3, 2010, Pages 256-263

Harnessing chaperone-mediated autophagy for the selective degradation of mutant huntingtin protein

Author keywords

[No Author keywords available]

Indexed keywords

ADAPTOR MOLECULE; BINDING MOTIF; BINDING PEPTIDE; CHAPERONE-MEDIATED AUTOPHAGY; CULTURED CELL; HEAT-SHOCK; HUNTINGTON'S DISEASE; MISFOLDED PROTEINS; MOUSE MODELS; MUTANT HUNTINGTIN; POLYGLUTAMINE; POLYGLUTAMINE (POLYQ); SELECTIVE DEGRADATION; SPECIFIC BINDING; THERAPEUTIC POTENTIALS;

EID: 77749319356     PISSN: 10870156     EISSN: 15461696     Source Type: Journal    
DOI: 10.1038/nbt.1608     Document Type: Article
Times cited : (209)

References (61)
  • 1
    • 0034094873 scopus 로고    scopus 로고
    • Glutamine repeats and neurodegeneration
    • Zoghbi, H.Y. & Orr, H.T. Glutamine repeats and neurodegeneration. Annu. Rev. Neurosci. 23, 217-247 (2000).
    • (2000) Annu. Rev. Neurosci. , vol.23 , pp. 217-247
    • Zoghbi, H.Y.1    Orr, H.T.2
  • 2
    • 0030752709 scopus 로고    scopus 로고
    • Aggregation of huntingtin in neuronal intranuclear inclusions and dystrophic neurites in brain
    • DiFiglia, M. et al. Aggregation of huntingtin in neuronal intranuclear inclusions and dystrophic neurites in brain. Science 277, 1990-1993 (1997).
    • (1997) Science , vol.277 , pp. 1990-1993
    • Difiglia, M.1
  • 3
    • 18544410106 scopus 로고    scopus 로고
    • Formation of neuronal intranuclear inclusions underlies the neurological dysfunction in mice transgenic for the HD mutation
    • Davies, S.W. et al. Formation of neuronal intranuclear inclusions underlies the neurological dysfunction in mice transgenic for the HD mutation. Cell 90, 537-548 (1997).
    • (1997) Cell , vol.90 , pp. 537-548
    • Davies, S.W.1
  • 4
    • 0037117499 scopus 로고    scopus 로고
    • Aggregation of proteins with expanded glutamine and alanine repeats of the glutamine-rich and asparagine-rich domains of Sup35 and of the amyloid beta-peptide of amyloid plaques
    • Perutz, M.F., Pope, B.J., Owen, D., Wanker, E.E. & Scherzinger, E. Aggregation of proteins with expanded glutamine and alanine repeats of the glutamine-rich and asparagine-rich domains of Sup35 and of the amyloid beta-peptide of amyloid plaques. Proc. Natl. Acad. Sci. USA 99, 5596-5600 (2002).
    • (2002) Proc. Natl. Acad. Sci. USA , vol.99 , pp. 5596-5600
    • Perutz, M.F.1    Pope, B.J.2    Owen, D.3    Wanker, E.E.4    Scherzinger, E.5
  • 5
    • 0141668882 scopus 로고    scopus 로고
    • Expansion of polyglutamine induces the formation of quasi-aggregate in the early stage of protein fbrillization
    • Tanaka, M. et al. Expansion of polyglutamine induces the formation of quasi-aggregate in the early stage of protein fbrillization. J. Biol. Chem. 278, 34717-34724 (2003).
    • (2003) J. Biol. Chem. , vol.278 , pp. 34717-34724
    • Tanaka, M.1
  • 6
    • 0037150687 scopus 로고    scopus 로고
    • Sp1 and TAFII130 transcriptional activity disrupted in early Huntington's disease
    • Dunah, A.W. et al. Sp1 and TAFII130 transcriptional activity disrupted in early Huntington's disease. Science 296, 2238-2243 (2002).
    • (2002) Science , vol.296 , pp. 2238-2243
    • Dunah, A.W.1
  • 7
    • 0035937523 scopus 로고    scopus 로고
    • Interference by huntingtin and atrophin-1 with cbp-mediated transcription leading to cellular toxicity
    • Nucifora, F.C. Jr. et al. Interference by huntingtin and atrophin-1 with cbp-mediated transcription leading to cellular toxicity. Science 291, 2423-2428 (2001).
    • (2001) Science , vol.291 , pp. 2423-2428
    • Nucifora Jr., F.C.1
  • 8
    • 40949135766 scopus 로고    scopus 로고
    • Mutant Huntingtin reduces HSP70 expression through the sequestration of NF-Y transcription factor
    • Yamanaka, T. et al. Mutant Huntingtin reduces HSP70 expression through the sequestration of NF-Y transcription factor. EMBO J. 27, 827-839 (2008).
    • (2008) EMBO J. , vol.27 , pp. 827-839
    • Yamanaka, T.1
  • 9
    • 69949170793 scopus 로고    scopus 로고
    • The pathogenic mechanisms of polyglutamine diseases and current therapeutic strategies
    • Bauer, P.O. & Nukina, N. The pathogenic mechanisms of polyglutamine diseases and current therapeutic strategies. J. Neurochem. 110, 1737-1765 (2009).
    • (2009) J. Neurochem. , vol.110 , pp. 1737-1765
    • Bauer, P.O.1    Nukina, N.2
  • 10
    • 0034737299 scopus 로고    scopus 로고
    • Reversal of neuropathology and motor dysfunction in a conditional model of Huntington's disease
    • Yamamoto, A., Lucas, J.J. & Hen, R. Reversal of neuropathology and motor dysfunction in a conditional model of Huntington's disease. Cell 101, 57-66 (2000).
    • (2000) Cell , vol.101 , pp. 57-66
    • Yamamoto, A.1    Lucas, J.J.2    Hen, R.3
  • 11
    • 26844494370 scopus 로고    scopus 로고
    • Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA
    • Wang, Y.L. et al. Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA. Neurosci. Res. 53, 241-249 (2005).
    • (2005) Neurosci. Res. , vol.53 , pp. 241-249
    • Wang, Y.L.1
  • 12
    • 36749033738 scopus 로고    scopus 로고
    • Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral defcits
    • DiFiglia, M. et al. Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral defcits. Proc. Natl. Acad. Sci. USA. 104, 17204-17209 (2007).
    • (2007) Proc. Natl. Acad. Sci. USA. , vol.104 , pp. 17204-17209
    • Difiglia, M.1
  • 13
    • 20244378556 scopus 로고    scopus 로고
    • RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model
    • Harper, S.Q. et al. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc. Natl. Acad. Sci. USA. 102, 5820-5825 (2005).
    • (2005) Proc. Natl. Acad. Sci. USA. , vol.102 , pp. 5820-5825
    • Harper, S.Q.1
  • 14
    • 25144464388 scopus 로고    scopus 로고
    • Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice
    • Rodriguez-Lebron, E., Denovan-Wright, E.M., Nash, K., Lewin, A.S. & Mandel, R.J. Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice. Mol. Ther. 12, 618-633 (2005).
    • (2005) Mol. Ther. , vol.12 , pp. 618-633
    • Rodriguez-Lebron, E.1    Denovan-Wright, E.M.2    Nash, K.3    Lewin, A.S.4    Mandel, R.J.5
  • 15
    • 2642586352 scopus 로고    scopus 로고
    • Inhibition of mTOR induces autophagy and reduces toxicity of polyglutamine expansions in fy and mouse models of Huntington disease
    • Ravikumar, B. et al. Inhibition of mTOR induces autophagy and reduces toxicity of polyglutamine expansions in fy and mouse models of Huntington disease. Nat. Genet. 36, 585-595 (2004).
    • (2004) Nat. Genet. , vol.36 , pp. 585-595
    • Ravikumar, B.1
  • 16
    • 34248994604 scopus 로고    scopus 로고
    • Small molecules enhance autophagy and reduce toxicity in Huntington's disease models
    • Sarkar, S. et al. Small molecules enhance autophagy and reduce toxicity in Huntington's disease models. Nat. Chem. Biol. 3, 331-338 (2007).
    • (2007) Nat. Chem. Biol. , vol.3 , pp. 331-338
    • Sarkar, S.1
  • 17
    • 53349127249 scopus 로고    scopus 로고
    • Blocking acid-sensing ion channel 1 alleviates Huntington's disease pathology via an ubiquitin-proteasome system-dependent mechanism
    • Wong, H.K. et al. Blocking acid-sensing ion channel 1 alleviates Huntington's disease pathology via an ubiquitin-proteasome system-dependent mechanism. Hum. Mol. Genet. 17, 3223-3235 (2008).
    • (2008) Hum. Mol. Genet. , vol.17 , pp. 3223-3235
    • Wong, H.K.1
  • 18
    • 67649690944 scopus 로고    scopus 로고
    • Inhibition of rho kinases enhances the degradation of mutant huntingtin
    • Bauer, P.O. et al. Inhibition of rho kinases enhances the degradation of mutant huntingtin. J. Biol. Chem. 284, 13153-13164 (2009).
    • (2009) J. Biol. Chem. , vol.284 , pp. 13153-13164
    • Bauer, P.O.1
  • 20
    • 0029837453 scopus 로고    scopus 로고
    • A receptor for the selective uptake and degradation of proteins by lysosomes
    • Cuervo, A.M. & Dice, J.F. A receptor for the selective uptake and degradation of proteins by lysosomes. Science 273, 501-503 (1996).
    • (1996) Science , vol.273 , pp. 501-503
    • Cuervo, A.M.1    Dice, J.F.2
  • 21
    • 0034914206 scopus 로고    scopus 로고
    • A molecular chaperone complex at the lysosomal membrane is required for protein translocation
    • Agarraberes, F.A. & Dice, J.F. A molecular chaperone complex at the lysosomal membrane is required for protein translocation. J. Cell Sci. 114, 2491-2499 (2001).
    • (2001) J. Cell Sci. , vol.114 , pp. 2491-2499
    • Agarraberes, F.A.1    Dice, J.F.2
  • 22
    • 0034615932 scopus 로고    scopus 로고
    • Inhibition of polyglutamine protein aggregation and cell death by novel peptides identifed by phage display screening
    • Nagai, Y. et al. Inhibition of polyglutamine protein aggregation and cell death by novel peptides identifed by phage display screening. J. Biol. Chem. 275, 10437-10442 (2000).
    • (2000) J. Biol. Chem. , vol.275 , pp. 10437-10442
    • Nagai, Y.1
  • 23
    • 4344659685 scopus 로고    scopus 로고
    • Impaired degradation of mutant alpha-synuclein by chaperone-mediated autophagy
    • Cuervo, A.M., Stefanis, L., Fredenburg, R., Lansbury, P.T. & Sulzer, D. Impaired degradation of mutant alpha-synuclein by chaperone-mediated autophagy. Science 305, 1292-1295 (2004).
    • (2004) Science , vol.305 , pp. 1292-1295
    • Cuervo, A.M.1    Stefanis, L.2    Fredenburg, R.3    Lansbury, P.T.4    Sulzer, D.5
  • 24
    • 0037413688 scopus 로고    scopus 로고
    • Cathepsin A regulates chaperone-mediated autophagy through cleavage of the lysosomal receptor
    • Cuervo, A.M., Mann, L., Bonten, E.J., d'Azzo, A. & Dice, J.F Cathepsin A regulates chaperone-mediated autophagy through cleavage of the lysosomal receptor. EMBO J. 22, 47-59 (2003).
    • (2003) EMBO J. , vol.22 , pp. 47-59
    • Cuervo, A.M.1    Mann, L.2    Bonten, E.J.3    D'Azzo, A.4    Dice, J.F.5
  • 25
    • 12444292683 scopus 로고    scopus 로고
    • Prevention of polyglutamine oligomerization and neurodegeneration by the peptide inhibitor QBP1 in Drosophila
    • Nagai, Y. et al. Prevention of polyglutamine oligomerization and neurodegeneration by the peptide inhibitor QBP1 in Drosophila. Hum. Mol. Genet. 12, 1253-1259 (2003).
    • (2003) Hum. Mol. Genet. , vol.12 , pp. 1253-1259
    • Nagai, Y.1
  • 26
    • 34247247115 scopus 로고    scopus 로고
    • A toxic monomeric conformer of the polyglutamine protein
    • Nagai, Y. et al. A toxic monomeric conformer of the polyglutamine protein. Nat. Struct. Mol. Biol. 14, 332-340 (2007).
    • (2007) Nat. Struct. Mol. Biol. , vol.14 , pp. 332-340
    • Nagai, Y.1
  • 27
    • 16844384763 scopus 로고    scopus 로고
    • Semi-rational engineering of a coral fuorescent protein into an effcient highlighter
    • Tsutsui, H., Karasawa, S., Shimizu, H., Nukina, N. & Miyawaki, A. Semi-rational engineering of a coral fuorescent protein into an effcient highlighter. EMBO Rep. 6, 233-238 (2005).
    • (2005) EMBO Rep. , vol.6 , pp. 233-238
    • Tsutsui, H.1    Karasawa, S.2    Shimizu, H.3    Nukina, N.4    Miyawaki, A.5
  • 28
    • 33644606491 scopus 로고    scopus 로고
    • Tracker dyes to probe mitochondrial autophagy (mitophagy) in rat hepatocytes
    • Rodriguez-Enriquez, S., Kim, I., Currin, R.T. & Lemasters, J.J. Tracker dyes to probe mitochondrial autophagy (mitophagy) in rat hepatocytes. Autophagy 2, 39-46 (2006).
    • (2006) Autophagy , vol.2 , pp. 39-46
    • Rodriguez-Enriquez, S.1    Kim, I.2    Currin, R.T.3    Lemasters, J.J.4
  • 29
    • 38049139425 scopus 로고    scopus 로고
    • Sensitive biochemical aggregate detection reveals aggregation onset before symptom development in cellular and murine models of Huntington's disease
    • Weiss, A. et al. Sensitive biochemical aggregate detection reveals aggregation onset before symptom development in cellular and murine models of Huntington's disease. J. Neurochem. 104, 846-858 (2008).
    • (2008) J. Neurochem. , vol.104 , pp. 846-858
    • Weiss, A.1
  • 30
    • 20944451721 scopus 로고    scopus 로고
    • Decreased expression of hypothalamic neuropeptides in Huntington disease transgenic mice with expanded polyglutamine-EGFP fuorescent aggregates
    • Kotliarova, S. et al. Decreased expression of hypothalamic neuropeptides in Huntington disease transgenic mice with expanded polyglutamine-EGFP fuorescent aggregates. J. Neurochem. 93, 641-653 (2005).
    • (2005) J. Neurochem. , vol.93 , pp. 641-653
    • Kotliarova, S.1
  • 31
    • 2342598416 scopus 로고    scopus 로고
    • Experimental therapeutics in transgenic mouse models of Huntington's disease
    • Beal, M.F. & Ferrante, R.J. Experimental therapeutics in transgenic mouse models of Huntington's disease. Nat. Rev. Neurosci. 5, 373-384 (2004).
    • (2004) Nat. Rev. Neurosci. , vol.5 , pp. 373-384
    • Beal, M.F.1    Ferrante, R.J.2
  • 32
    • 26844498655 scopus 로고    scopus 로고
    • The use of the R6 transgenic mouse models of Huntington's Disease in attempts to develop novel therapeutic strategies
    • Li, J.Y., Popovic, N. & Brundin, P. The use of the R6 transgenic mouse models of Huntington's Disease in attempts to develop novel therapeutic strategies. NeuroRx 2, 447-464 (2005).
    • (2005) NeuroRx , vol.2 , pp. 447-464
    • Li, J.Y.1    Popovic, N.2    Brundin, P.3
  • 33
    • 1642633757 scopus 로고    scopus 로고
    • Trehalose alleviates polyglutamine-mediated pathology in a mouse model of Huntington disease
    • Tanaka, M. et al. Trehalose alleviates polyglutamine-mediated pathology in a mouse model of Huntington disease. Nat. Med. 10, 148-154 (2004).
    • (2004) Nat. Med. , vol.10 , pp. 148-154
    • Tanaka, M.1
  • 34
    • 0037461730 scopus 로고    scopus 로고
    • Pivotal role of oligomerization in expanded polyglutamine neurodegenerative disorders
    • Sanchez, I., Mahlke, C. & Yuan, J. Pivotal role of oligomerization in expanded polyglutamine neurodegenerative disorders. Nature 421, 373-379 (2003).
    • (2003) Nature , vol.421 , pp. 373-379
    • Sanchez, I.1    Mahlke, C.2    Yuan, J.3
  • 35
    • 0033594894 scopus 로고    scopus 로고
    • Transglutaminase aggregates huntingtin into nonamyloidogenic polymers, and its enzymatic activity increases in Huntington's disease brain nuclei
    • Karpuj, M.V. et al. Transglutaminase aggregates huntingtin into nonamyloidogenic polymers, and its enzymatic activity increases in Huntington's disease brain nuclei. Proc. Natl. Acad. Sci. USA 96, 7388-7393 (1999).
    • (1999) Proc. Natl. Acad. Sci. USA , vol.96 , pp. 7388-7393
    • Karpuj, M.V.1
  • 36
    • 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, M.V. et al. Prolonged survival and decreased abnormal movements in transgenic model of Huntington disease, with administration of the transglutaminase inhibitor cystamine. Nat. Med. 8, 143-149 (2002).
    • (2002) Nat. Med. , vol.8 , pp. 143-149
    • Karpuj, M.V.1
  • 37
    • 0037109665 scopus 로고    scopus 로고
    • Therapeutic effects of cystamine in a murine model of Huntington's disease
    • Dedeoglu, A. et al. Therapeutic effects of cystamine in a murine model of Huntington's disease. J. Neurosci. 22, 8942-8950 (2002).
    • (2002) J. Neurosci. , vol.22 , pp. 8942-8950
    • Dedeoglu, A.1
  • 38
    • 0142157600 scopus 로고    scopus 로고
    • Histone deacetylase inhibition by sodium butyrate chemotherapy ameliorates the neurodegenerative phenotype in Huntington's disease mice
    • Ferrante, R.J. et al. Histone deacetylase inhibition by sodium butyrate chemotherapy ameliorates the neurodegenerative phenotype in Huntington's disease mice. J. Neurosci. 23, 9418-9427 (2003).
    • (2003) J. Neurosci. , vol.23 , pp. 9418-9427
    • Ferrante, R.J.1
  • 39
    • 33745120560 scopus 로고    scopus 로고
    • Dose ranging and effcacy study of high-dose coenzyme Q10 formulations in Huntington's disease mice
    • Smith, K.M. et al. Dose ranging and effcacy study of high-dose coenzyme Q10 formulations in Huntington's disease mice. Biochim. Biophys. Acta 1762, 616-626 (2006).
    • (2006) Biochim. Biophys. Acta , vol.1762 , pp. 616-626
    • Smith, K.M.1
  • 40
    • 23844482456 scopus 로고    scopus 로고
    • Clioquinol down-regulates mutant huntingtin expression in vitro and mitigates pathology in a Huntington's disease mouse model
    • Nguyen, T., Hamby, A. & Massa, S.M. Clioquinol down-regulates mutant huntingtin expression in vitro and mitigates pathology in a Huntington's disease mouse model. Proc. Natl. Acad. Sci. USA 102, 11840-11845 (2005).
    • (2005) Proc. Natl. Acad. Sci. USA , vol.102 , pp. 11840-11845
    • Nguyen, T.1    Hamby, A.2    Massa, S.M.3
  • 41
    • 0033587128 scopus 로고    scopus 로고
    • Inhibition of caspase-1 slows disease progression in a mouse model of Huntington's disease
    • Ona, V.O. et al. Inhibition of caspase-1 slows disease progression in a mouse model of Huntington's disease. Nature 399, 263-267 (1999).
    • (1999) Nature , vol.399 , pp. 263-267
    • Ona, V.O.1
  • 42
    • 29144517660 scopus 로고    scopus 로고
    • FGF-2 promotes neurogenesis and neuroprotection and prolongs survival in a transgenic mouse model of Huntington's disease
    • Jin, K. et al. FGF-2 promotes neurogenesis and neuroprotection and prolongs survival in a transgenic mouse model of Huntington's disease. Proc. Natl. Acad. Sci. USA 102, 18189-18194 (2005).
    • (2005) Proc. Natl. Acad. Sci. USA , vol.102 , pp. 18189-18194
    • Jin, K.1
  • 43
    • 39249084415 scopus 로고    scopus 로고
    • The antidepressant sertraline improves the phenotype, promotes neurogenesis and increases BDNF levels in the R6/2 Huntington's disease mouse model
    • Peng, Q. et al. The antidepressant sertraline improves the phenotype, promotes neurogenesis and increases BDNF levels in the R6/2 Huntington's disease mouse model. Exp. Neurol. 210, 154-163 (2008).
    • (2008) Exp. Neurol. , vol.210 , pp. 154-163
    • Peng, Q.1
  • 44
    • 20844455450 scopus 로고    scopus 로고
    • Chemotherapy for the brain: The antitumor antibiotic mithramycin prolongs survival in a mouse model of Huntington's disease
    • Ferrante, R.J. et al. Chemotherapy for the brain: the antitumor antibiotic mithramycin prolongs survival in a mouse model of Huntington's disease. J. Neurosci. 24, 10335-10342 (2004).
    • (2004) J. Neurosci. , vol.24 , pp. 10335-10342
    • Ferrante, R.J.1
  • 45
    • 34447312020 scopus 로고    scopus 로고
    • ESET/SETDB1 gene expression and histone H3 (K9) trimethylation in Huntington's disease
    • Ryu, H. et al. ESET/SETDB1 gene expression and histone H3 (K9) trimethylation in Huntington's disease. Proc. Natl. Acad. Sci. USA 103, 19176-19181 (2006).
    • (2006) Proc. Natl. Acad. Sci. USA , vol.103 , pp. 19176-19181
    • Ryu, H.1
  • 46
    • 56949098809 scopus 로고    scopus 로고
    • Delivery of the aggregate inhibitor peptide QBP1 into the mouse brain using PTDs and its therapeutic effect on polyglutamine disease mice
    • Popiel, H.A., Nagai, Y., Fujikake, N. & Toda, T. Delivery of the aggregate inhibitor peptide QBP1 into the mouse brain using PTDs and its therapeutic effect on polyglutamine disease mice. Neurosci. Lett. 449, 87-92 (2009).
    • (2009) Neurosci. Lett. , vol.449 , pp. 87-92
    • Popiel, H.A.1    Nagai, Y.2    Fujikake, N.3    Toda, T.4
  • 47
    • 70350543879 scopus 로고    scopus 로고
    • Intrabody gene therapy ameliorates motor, cognitive, and neuropathological symptoms in multiple mouse models of Huntington's disease
    • Southwell, A.L., Ko, J. & Patterson, P.H. Intrabody gene therapy ameliorates motor, cognitive, and neuropathological symptoms in multiple mouse models of Huntington's disease. J. Neurosci. 29, 13589-13602 (2009).
    • (2009) J. Neurosci. , vol.29 , pp. 13589-13602
    • Southwell, A.L.1    Ko, J.2    Patterson, P.H.3
  • 48
    • 20244378556 scopus 로고    scopus 로고
    • RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model
    • Harper, S.Q. et al. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc. Natl. Acad. Sci. USA 102, 5820-5825 (2005).
    • (2005) Proc. Natl. Acad. Sci. USA , vol.102 , pp. 5820-5825
    • Harper, S.Q.1
  • 49
    • 25144464388 scopus 로고    scopus 로고
    • Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice
    • Rodriguez-Lebron, E., Denovan-Wright, E.M., Nash, K., Lewin, A.S. & Mandel, R.J. Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice. Mol. Ther. 12, 618-633 (2005).
    • (2005) Mol. Ther. , vol.12 , pp. 618-633
    • Rodriguez-Lebron, E.1    Denovan-Wright, E.M.2    Nash, K.3    Lewin, A.S.4    Mandel, R.J.5
  • 50
    • 36749033738 scopus 로고    scopus 로고
    • Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral defcits
    • DiFiglia, M. et al. Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral defcits. Proc. Natl. Acad. Sci. USA 104, 17204-17209 (2007).
    • (2007) Proc. Natl. Acad. Sci. USA , vol.104 , pp. 17204-17209
    • Difiglia, M.1
  • 51
    • 34248146697 scopus 로고    scopus 로고
    • High-capacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo
    • Huang, B. et al. High-capacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo. Hum. Gene Ther. 18, 303-311 (2007).
    • (2007) Hum. Gene Ther. , vol.18 , pp. 303-311
    • Huang, B.1
  • 52
    • 57249086448 scopus 로고    scopus 로고
    • Allele-specifc silencing of mutant Huntington's disease gene
    • Zhang, Y., Engelman, J. & Friedlander, R.M. Allele-specifc silencing of mutant Huntington's disease gene. J. Neurochem. 108, 82-90 (2009).
    • (2009) J. Neurochem. , vol.108 , pp. 82-90
    • Zhang, Y.1    Engelman, J.2    Friedlander, R.M.3
  • 53
    • 34948855498 scopus 로고    scopus 로고
    • Induction of neostriatal neurogenesis slows disease progression in a transgenic murine model of Huntington disease
    • Cho, S.R. et al. Induction of neostriatal neurogenesis slows disease progression in a transgenic murine model of Huntington disease. J. Clin. Invest. 117, 2889-2902 (2007).
    • (2007) J. Clin. Invest. , vol.117 , pp. 2889-2902
    • Cho, S.R.1
  • 54
    • 54049111928 scopus 로고    scopus 로고
    • Intrabodies binding the proline-rich domains of mutant huntingtin increase its turnover and reduce neurotoxicity
    • Southwell, A.L. et al. Intrabodies binding the proline-rich domains of mutant huntingtin increase its turnover and reduce neurotoxicity. J. Neurosci. 28, 9013-9020 (2008).
    • (2008) J. Neurosci. , vol.28 , pp. 9013-9020
    • Southwell, A.L.1
  • 55
    • 34147130637 scopus 로고    scopus 로고
    • Isolation of a human single chain antibody fragment against oligomeric alpha-synuclein that inhibits aggregation and prevents alpha-synuclein-induced toxicity
    • Emadi, S., Barkhordarian, H., Wang, M.S., Schulz, P. & Sierks, M.R. 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 (2007).-
    • (2007) J. Mol. Biol. , vol.368 , pp. 1132-1144
    • Emadi, S.1    Barkhordarian, H.2    Wang, M.S.3    Schulz, P.4    Sierks, M.R.5
  • 56
    • 39649109675 scopus 로고    scopus 로고
    • An scFv intrabody against the nonamyloid component of alpha-synuclein reduces intracellular aggregation and toxicity
    • Lynch, S.M., Zhou, C. & Messer, A. An scFv intrabody against the nonamyloid component of alpha-synuclein reduces intracellular aggregation and toxicity. J. Mol. Biol. 377, 136-147 (2008).
    • (2008) J. Mol. Biol. , vol.377 , pp. 136-147
    • Lynch, S.M.1    Zhou, C.2    Messer, A.3
  • 57
    • 0033588476 scopus 로고    scopus 로고
    • Caspase activation during apoptotic cell death induced by expanded polyglutamine in N2a cells
    • Wang, G.H. et al. Caspase activation during apoptotic cell death induced by expanded polyglutamine in N2a cells. Neuroreport 10, 2435-2438 (1999).
    • (1999) Neuroreport , vol.10 , pp. 2435-2438
    • Wang, G.H.1
  • 58
    • 0033867992 scopus 로고    scopus 로고
    • The MJD1 gene product, interacts with the two human homologs of yeast DNA repair protein RAD23, HHR23A and HHR23B
    • Wang, G.H., Sawai, N., Kotliarova, S., Kanazawa, I. & Nukina, N. Ataxin-3, the MJD1 gene product, interacts with the two human homologs of yeast DNA repair protein RAD23, HHR23A and HHR23B. Hum. Mol. Genet. 9, 1795-1803 (2000).
    • (2000) Hum. Mol. Genet. , vol.9 , pp. 1795-1803
    • Wang, G.H.1    Sawai, N.2    Kotliarova, S.3    Kanazawa, I.4
  • 59
    • 0032692386 scopus 로고    scopus 로고
    • Preparation of human cDNas encoding expanded polyglutamine repeats
    • Peters, M.F. & Ross, C.A. Preparation of human cDNas encoding expanded polyglutamine repeats. Neurosci. Lett. 275, 129-132 (1999).
    • (1999) Neurosci. Lett. , vol.275 , pp. 129-132
    • Peters, M.F.1    Ross, C.A.2
  • 60
    • 0141918859 scopus 로고    scopus 로고
    • Pro-apoptotic protein kinase C delta is associated with intranuclear inclusions in a transgenic model of Huntington's disease
    • Zemskov, E.A. et al. Pro-apoptotic protein kinase C delta is associated with intranuclear inclusions in a transgenic model of Huntington's disease. J. Neurochem. 87, 395-406 (2003).
    • (2003) J. Neurochem. , vol.87 , pp. 395-406
    • Zemskov, E.A.1
  • 61
    • 0023655791 scopus 로고
    • Purifcation and characterization of lysosomes from Chinese hamster ovary cells
    • Madden, E.A., Wirt, J.B. & Storrie, B. Purifcation and characterization of lysosomes from Chinese hamster ovary cells. Arch. Biochem. Biophys. 257, 27-38 (1987).
    • (1987) Arch. Biochem. Biophys. , vol.257 , pp. 27-38
    • Madden, E.A.1    Wirt, J.B.2    Storrie, B.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.