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Volumn 14, Issue 8, 2012, Pages 521-529

Reversal of pathology in CHMP 2B-mediated frontotemporal dementia patient cells using RNA interference

Author keywords

CHMP2B; Frontotemporal dementia; FTD 3; Lentiviral vectors; RNAi

Indexed keywords

LENTIVIRUS VECTOR; SHORT HAIRPIN RNA; CD63 ANTIGEN; CD63 PROTEIN, HUMAN; CHMP2B PROTEIN, HUMAN; ESCRT PROTEIN; SMALL INTERFERING RNA;

EID: 84865432155     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.2649     Document Type: Article
Times cited : (13)

References (40)
  • 3
    • 0031672540 scopus 로고    scopus 로고
    • Frontotemporal lobar degeneration: a consensus on clinical diagnostic criteria
    • Neary D, Snowden JS, Gustafson L, et al. Frontotemporal lobar degeneration: a consensus on clinical diagnostic criteria. Neurology 1998; 51: 1546-1554.
    • (1998) Neurology , vol.51 , pp. 1546-1554
    • Neary, D.1    Snowden, J.S.2    Gustafson, L.3
  • 4
    • 0034764622 scopus 로고    scopus 로고
    • Clinical and pathological diagnosis of frontotemporal dementia: report of the Work Group on Frontotemporal Dementia and Pick's Disease
    • McKhann GM, Albert MS, Grossman M, et al. Clinical and pathological diagnosis of frontotemporal dementia: report of the Work Group on Frontotemporal Dementia and Pick's Disease. Arch Neurol 2001; 58: 1803-1809.
    • (2001) Arch Neurol , vol.58 , pp. 1803-1809
    • McKhann, G.M.1    Albert, M.S.2    Grossman, M.3
  • 6
    • 80054832080 scopus 로고    scopus 로고
    • Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS
    • DeJesus-Hernandez M, Mackenzie IR, Boeve BF, et al. Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS. Neuron 2011; 72: 245-256.
    • (2011) Neuron , vol.72 , pp. 245-256
    • DeJesus-Hernandez, M.1    Mackenzie, I.R.2    Boeve, B.F.3
  • 7
    • 80054837386 scopus 로고    scopus 로고
    • A hexanucleotide repeat expansion in C9ORF72 is the cause of chromosome 9p21-linked ALS-FTD
    • Renton AE, Majounie E, Waite A, et al. A hexanucleotide repeat expansion in C9ORF72 is the cause of chromosome 9p21-linked ALS-FTD. Neuron 2011; 72: 257-268.
    • (2011) Neuron , vol.72 , pp. 257-268
    • Renton, A.E.1    Majounie, E.2    Waite, A.3
  • 8
    • 23044471011 scopus 로고    scopus 로고
    • Mutations in the endosomal ESCRTIII-complex subunit CHMP2B in frontotemporal dementia
    • Skibinski G, Parkinson NJ, Brown JM, et al. Mutations in the endosomal ESCRTIII-complex subunit CHMP2B in frontotemporal dementia. Nat Genet 2005; 37: 806-808.
    • (2005) Nat Genet , vol.37 , pp. 806-808
    • Skibinski, G.1    Parkinson, N.J.2    Brown, J.M.3
  • 9
    • 37849023471 scopus 로고    scopus 로고
    • CHMP2B C-truncating mutations in frontotemporal lobar degeneration are associated with an aberrant endosomal phenotype in vitro
    • van der Zee J, Urwin H, Engelborghs S, et al. CHMP2B C-truncating mutations in frontotemporal lobar degeneration are associated with an aberrant endosomal phenotype in vitro. Hum Mol Genet 2008; 17: 313-322.
    • (2008) Hum Mol Genet , vol.17 , pp. 313-322
    • van der Zee, J.1    Urwin, H.2    Engelborghs, S.3
  • 11
    • 0036902646 scopus 로고    scopus 로고
    • Receptor downregulation and multivesicular-body sorting
    • Katzmann DJ, Odorizzi G, Emr SD. Receptor downregulation and multivesicular-body sorting. Nat Rev Mol Cell Biol 2002; 3: 893-905.
    • (2002) Nat Rev Mol Cell Biol , vol.3 , pp. 893-905
    • Katzmann, D.J.1    Odorizzi, G.2    Emr, S.D.3
  • 13
    • 35148831808 scopus 로고    scopus 로고
    • Structural basis for selective recognition of ESCRT-III by the AAA ATPase Vps4
    • Obita T, Saksena S, Ghazi-Tabatabai S, et al. Structural basis for selective recognition of ESCRT-III by the AAA ATPase Vps4. Nature 2007; 449: 735-739.
    • (2007) Nature , vol.449 , pp. 735-739
    • Obita, T.1    Saksena, S.2    Ghazi-Tabatabai, S.3
  • 14
    • 77953583994 scopus 로고    scopus 로고
    • Disruption of endocytic trafficking in frontotemporal dementia with CHMP2B mutations
    • Urwin H, Authier A, Nielsen JE, et al. Disruption of endocytic trafficking in frontotemporal dementia with CHMP2B mutations. Hum Mol Genet 2010; 19: 2228-2238.
    • (2010) Hum Mol Genet , vol.19 , pp. 2228-2238
    • Urwin, H.1    Authier, A.2    Nielsen, J.E.3
  • 15
    • 78649751019 scopus 로고    scopus 로고
    • The role of ESCRT proteins in fusion events involving lysosomes, endosomes and autophagosomes
    • Metcalf D, Isaacs AM. The role of ESCRT proteins in fusion events involving lysosomes, endosomes and autophagosomes. Biochem Soc Trans 2010; 38: 1469-1473.
    • (2010) Biochem Soc Trans , vol.38 , pp. 1469-1473
    • Metcalf, D.1    Isaacs, A.M.2
  • 16
    • 84857568926 scopus 로고    scopus 로고
    • Progressive neuronal inclusion formation and axonal degeneration in CHMP2B mutant transgenic mice
    • Ghazi-Noori S, Froud KE, Mizielinska S, et al. Progressive neuronal inclusion formation and axonal degeneration in CHMP2B mutant transgenic mice. Brain 2012; 135: 819-832.
    • (2012) Brain , vol.135 , pp. 819-832
    • Ghazi-Noori, S.1    Froud, K.E.2    Mizielinska, S.3
  • 17
    • 84865410504 scopus 로고    scopus 로고
    • Open Biosystems. Vector product information. [17 July 2012].
    • Open Biosystems. Vector product information. https://www.openbiosystems.com/ProductLiterature.aspx?AliasPath=/RNAi/shRNAmirLibraries/GIPZLentiviralshRNAmir&CatalogNumber=RHS4346 [17 July 2012].
  • 18
    • 0030819379 scopus 로고    scopus 로고
    • Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo
    • Zufferey R, Nagy D, Mandel RJ, Naldini L, Trono D. Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat Biotechnol 1997; 15: 871-875.
    • (1997) Nat Biotechnol , vol.15 , pp. 871-875
    • Zufferey, R.1    Nagy, D.2    Mandel, R.J.3    Naldini, L.4    Trono, D.5
  • 19
    • 61949341072 scopus 로고    scopus 로고
    • Incorporating double copies of a chromatin insulator into lentiviral vectors results in less viral integrants
    • Nielsen TT, Jakobsson J, Rosenqvist N, Lundberg C. Incorporating double copies of a chromatin insulator into lentiviral vectors results in less viral integrants. BMC Biotechnol 2009; 9: 13.
    • (2009) BMC Biotechnol , vol.9 , pp. 13
    • Nielsen, T.T.1    Jakobsson, J.2    Rosenqvist, N.3    Lundberg, C.4
  • 20
    • 68949213635 scopus 로고    scopus 로고
    • Neuron-specific RNA interference using lentiviral vectors
    • Nielsen TT, Marion I, Hasholt L, Lundberg C. Neuron-specific RNA interference using lentiviral vectors. J Gene Med 2009; 11: 559-569.
    • (2009) J Gene Med , vol.11 , pp. 559-569
    • Nielsen, T.T.1    Marion, I.2    Hasholt, L.3    Lundberg, C.4
  • 21
    • 79955129749 scopus 로고    scopus 로고
    • Knockdown of GAD67 protein levels normalizes neuronal activity in a rat model of Parkinson's disease
    • Horvath L, van Marion I, Tai K, Nielsen TT, Lundberg C. Knockdown of GAD67 protein levels normalizes neuronal activity in a rat model of Parkinson's disease. J Gene Med 2011; 13: 188-197.
    • (2011) J Gene Med , vol.13 , pp. 188-197
    • Horvath, L.1    van Marion, I.2    Tai, K.3    Nielsen, T.T.4    Lundberg, C.5
  • 22
    • 0035925642 scopus 로고    scopus 로고
    • The human ITGB4BP gene is constitutively expressed in vitro, but highly modulated in vivo
    • Donadini A, Giodini A, Sanvito F, Marchisio PC, Biffo S. The human ITGB4BP gene is constitutively expressed in vitro, but highly modulated in vivo. Gene 2001; 266: 35-43.
    • (2001) Gene , vol.266 , pp. 35-43
    • Donadini, A.1    Giodini, A.2    Sanvito, F.3    Marchisio, P.C.4    Biffo, S.5
  • 23
    • 67349133131 scopus 로고    scopus 로고
    • Preferential labelling of inhibitory and excitatory cortical neurons by endogenous tropism of adeno-associated virus and lentivirus vectors
    • Nathanson JL, Yanagawa Y, Obata K, Callaway EM. Preferential labelling of inhibitory and excitatory cortical neurons by endogenous tropism of adeno-associated virus and lentivirus vectors. Neuroscience 2009; 161: 441-450.
    • (2009) Neuroscience , vol.161 , pp. 441-450
    • Nathanson, J.L.1    Yanagawa, Y.2    Obata, K.3    Callaway, E.M.4
  • 24
    • 63049114730 scopus 로고    scopus 로고
    • Targeted knock-down of neuronal nitric oxide synthase expression in basal forebrain with RNA interference
    • Mahairaki V, Xu L, Farah MH, et al. Targeted knock-down of neuronal nitric oxide synthase expression in basal forebrain with RNA interference. J Neurosci Methods 2009; 179: 292-299.
    • (2009) J Neurosci Methods , vol.179 , pp. 292-299
    • Mahairaki, V.1    Xu, L.2    Farah, M.H.3
  • 25
    • 79952279226 scopus 로고    scopus 로고
    • Effect of lentiviral shRNA of Nogo receptor on rat cortex neuron axon outgrowth
    • Xu S, Liu M, Zhang T, et al. Effect of lentiviral shRNA of Nogo receptor on rat cortex neuron axon outgrowth. Can J Neurol Sci 2011; 38: 133-138.
    • (2011) Can J Neurol Sci , vol.38 , pp. 133-138
    • Xu, S.1    Liu, M.2    Zhang, T.3
  • 26
    • 0036787671 scopus 로고    scopus 로고
    • siRNA-mediated gene silencing in vitro and in vivo
    • Xia H, Mao Q, Paulson HL, Davidson BL. siRNA-mediated gene silencing in vitro and in vivo. Nat Biotechnol 2002; 20: 1006-1010.
    • (2002) Nat Biotechnol , vol.20 , pp. 1006-1010
    • Xia, H.1    Mao, Q.2    Paulson, H.L.3    Davidson, B.L.4
  • 27
    • 44449121785 scopus 로고    scopus 로고
    • Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi
    • McBride JL, Boudreau RL, Harper SQ, et al. Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci USA 2008; 105: 5868-5873.
    • (2008) Proc Natl Acad Sci USA , vol.105 , pp. 5868-5873
    • McBride, J.L.1    Boudreau, R.L.2    Harper, S.Q.3
  • 28
    • 59449090759 scopus 로고    scopus 로고
    • Presymptomatic generalized brain atrophy in frontotemporal dementia caused by CHMP2B mutation
    • Rohrer JD, Ahsan RL, Isaacs AM, et al. Presymptomatic generalized brain atrophy in frontotemporal dementia caused by CHMP2B mutation. Dement Geriatr Cogn Disord 2009; 27: 182-186.
    • (2009) Dement Geriatr Cogn Disord , vol.27 , pp. 182-186
    • Rohrer, J.D.1    Ahsan, R.L.2    Isaacs, A.M.3
  • 31
    • 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
  • 32
    • 67349159137 scopus 로고    scopus 로고
    • Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients
    • Pfister EL, Kennington L, Straubhaar J, et al. Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients. Curr Biol 2009; 19: 774-778.
    • (2009) Curr Biol , vol.19 , pp. 774-778
    • Pfister, E.L.1    Kennington, L.2    Straubhaar, J.3
  • 33
    • 61549104503 scopus 로고    scopus 로고
    • CAG expansion in the Huntington disease gene is associated with a specific and targetable predisposing haplogroup
    • Warby SC, Montpetit A, Hayden AR, et al. CAG expansion in the Huntington disease gene is associated with a specific and targetable predisposing haplogroup. Am J Hum Genet 2009; 84: 351-366.
    • (2009) Am J Hum Genet , vol.84 , pp. 351-366
    • Warby, S.C.1    Montpetit, A.2    Hayden, A.R.3
  • 34
    • 65249131740 scopus 로고    scopus 로고
    • Sustained effects of nonallele-specific Huntingtin silencing
    • Drouet V, Perrin V, Hassig R, et al. Sustained effects of nonallele-specific Huntingtin silencing. Ann Neurol 2009; 65: 276-285.
    • (2009) Ann Neurol , vol.65 , pp. 276-285
    • Drouet, V.1    Perrin, V.2    Hassig, R.3
  • 35
    • 54449095083 scopus 로고    scopus 로고
    • Allele-specific RNA silencing of mutant ataxin-3 mediates neuroprotection in a rat model of Machado-Joseph disease
    • Alves S, Nascimento-Ferreira I, Auregan G, et al. Allele-specific RNA silencing of mutant ataxin-3 mediates neuroprotection in a rat model of Machado-Joseph disease. PLoS One 2008; 3: e3341.
    • (2008) PLoS One , vol.3
    • Alves, S.1    Nascimento-Ferreira, I.2    Auregan, G.3
  • 36
    • 82955237522 scopus 로고    scopus 로고
    • Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the huntington disease gene/allele-specific silencing of mutant huntingtin
    • Carroll JB, Warby SC, Southwell AL, et al. Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the huntington disease gene/allele-specific silencing of mutant huntingtin. Mol Ther 2011; 19: 2178-2185.
    • (2011) Mol Ther , vol.19 , pp. 2178-2185
    • Carroll, J.B.1    Warby, S.C.2    Southwell, A.L.3
  • 37
    • 0038132996 scopus 로고    scopus 로고
    • Allele-specific silencing of dominant disease genes
    • Miller VM, Xia H, Marrs GL, et al. Allele-specific silencing of dominant disease genes. Proc Natl Acad Sci USA 2003; 100: 7195-7200.
    • (2003) Proc Natl Acad Sci USA , vol.100 , pp. 7195-7200
    • Miller, V.M.1    Xia, H.2    Marrs, G.L.3
  • 38
    • 77956902003 scopus 로고    scopus 로고
    • CHMP2B mutants linked to frontotemporal dementia impair maturation of dendritic spines
    • Belly A, Bodon G, Blot B, et al. CHMP2B mutants linked to frontotemporal dementia impair maturation of dendritic spines. J Cell Sci 2010; 123: 2943-2954.
    • (2010) J Cell Sci , vol.123 , pp. 2943-2954
    • Belly, A.1    Bodon, G.2    Blot, B.3
  • 39
    • 34548492271 scopus 로고    scopus 로고
    • ESCRT-III dysfunction causes autophagosome accumulation and neurodegeneration
    • Lee JA, Beigneux A, Ahmad ST, Young SG, Gao FB. ESCRT-III dysfunction causes autophagosome accumulation and neurodegeneration. Curr Biol 2007; 17: 1561-1567.
    • (2007) Curr Biol , vol.17 , pp. 1561-1567
    • Lee, J.A.1    Beigneux, A.2    Ahmad, S.T.3    Young, S.G.4    Gao, F.B.5
  • 40
    • 79952640255 scopus 로고    scopus 로고
    • ESCRT-III protein requirements for HIV-1 budding
    • Morita E, Sandrin V, McCullough J, et al. ESCRT-III protein requirements for HIV-1 budding. Cell Host Microbe 2011; 9: 235-242.
    • (2011) Cell Host Microbe , vol.9 , pp. 235-242
    • Morita, E.1    Sandrin, V.2    McCullough, J.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.