메뉴 건너뛰기




Volumn 4, Issue 3, 2013, Pages 457-484

Antisense gene silencing: Therapy for neurodegenerative disorders?

Author keywords

CNS; Neurodegenerative disorders; RNA interference

Indexed keywords

HERPESVIRUS VECTOR; LENTIVIRUS VECTOR; MICRORNA; PARVOVIRUS VECTOR; RETROVIRUS VECTOR; SHORT HAIRPIN RNA; SMALL INTERFERING RNA; 4 AMINOBUTYRIC ACID; 4 AMINOBUTYRIC ACID RECEPTOR; ADENOVIRUS VECTOR; ALPHA SYNUCLEIN; AMYLOID PRECURSOR PROTEIN; COPPER ZINC SUPEROXIDE DISMUTASE; DICER; DOPAMINE 1 RECEPTOR; DOPAMINE 2 RECEPTOR; DOUBLE STRANDED RNA; ENVELOPE PROTEIN; GLUTAMATE DECARBOXYLASE 67; LEUCINE RICH REPEAT KINASE 2; LIPOSOME; LOCKED NUCLEIC ACID; MACROGOL; MICROTUBULE ASSOCIATED PROTEIN; NANOPARTICLE; PEPTIDE NUCLEIC ACID; POLYGLUTAMINE; PRION PROTEIN; PROGRANULIN; VIRUS VECTOR;

EID: 84884158801     PISSN: None     EISSN: 20734425     Source Type: Journal    
DOI: 10.3390/genes4030457     Document Type: Review
Times cited : (15)

References (154)
  • 1
    • 0032545933 scopus 로고    scopus 로고
    • Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
    • Fire, X.S.; Montgomery, M.K.; Kostas, S.A.; Driver, S.E.; Mello, C.C. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998, 19, 806-811.
    • (1998) Nature , vol.19 , pp. 806-811
    • Fire, X.S.1    Montgomery, M.K.2    Kostas, S.A.3    Driver, S.E.4    Mello, C.C.5
  • 2
    • 0035863097 scopus 로고    scopus 로고
    • RNA interference is mediated by 21-and 22-nucleotide RNAs
    • Elbashir, S.M.; Lendeckel, W.; Tuschl, T. RNA interference is mediated by 21-and 22-nucleotide RNAs. Genes Dev. 2001, 15, 188-200.
    • (2001) Genes Dev. , vol.15 , pp. 188-200
    • Elbashir, S.M.1    Lendeckel, W.2    Tuschl, T.3
  • 3
    • 0035942736 scopus 로고    scopus 로고
    • Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
    • Elbashir, S.M.; Harborth, J.; Lendeckel, W.; Yalcin, A.; Weber, K.; Tuschl, T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001, 411, 494-498.
    • (2001) Nature , vol.411 , pp. 494-498
    • Elbashir, S.M.1    Harborth, J.2    Lendeckel, W.3    Yalcin, A.4    Weber, K.5    Tuschl, T.6
  • 4
    • 84884147900 scopus 로고    scopus 로고
    • U.S. National Institues of Health. Available online: (accessed on 2 July 2013)
    • U.S. National Institues of Health. Available online: http://www.ClinicalTrials.gov/ (accessed on 2 July 2013).
  • 5
    • 0036787671 scopus 로고    scopus 로고
    • siRNA-mediated gene silencing in vitro and in vivo
    • Xia, H.; Mao, Q.; Paulson, H.L.; Davidson, B.L. 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
  • 7
    • 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, J.L.; Pitzer, M.R.; Boudreau, R.L.; Dufour, B.; Hobbs, T.; Ojeda, S.R.; Davidson, B.L. 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    Dufour, B.4    Hobbs, T.5    Ojeda, S.R.6    Davidson, B.L.7
  • 8
    • 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, J.B.; Warby, S.C.; Southwell, A.L.; Doty, C.N.; Greenlee, S.; Skotte, N.; Hung, G.; Bennett, C.F.; Freier, S.M.; Hayden, M.R. 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    Doty, C.N.4    Greenlee, S.5    Skotte, N.6    Hung, G.7    Bennett, C.F.8    Freier, S.M.9    Hayden, M.R.10
  • 9
    • 0034673638 scopus 로고    scopus 로고
    • An RNA-directed nuclease mediates post-transcriptional gene silencing in Drosophila cells
    • Hammond, S.M.; Bernstein, E.; Beach, D.; Hannon, G.J. An RNA-directed nuclease mediates post-transcriptional gene silencing in Drosophila cells. Nature 2000, 404, 293-296.
    • (2000) Nature , vol.404 , pp. 293-296
    • Hammond, S.M.1    Bernstein, E.2    Beach, D.3    Hannon, G.J.4
  • 10
    • 0035905766 scopus 로고    scopus 로고
    • Role for a bidentate ribonuclease in the initiation step of RNA interference
    • Bernstein, E.; Caudy, A.A.; Hammond, S.M.; Hannon, G.J. Role for a bidentate ribonuclease in the initiation step of RNA interference. Nature 2001, 409, 363-366.
    • (2001) Nature , vol.409 , pp. 363-366
    • Bernstein, E.1    Caudy, A.A.2    Hammond, S.M.3    Hannon, G.J.4
  • 11
    • 0034737298 scopus 로고    scopus 로고
    • RNAi: Double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals
    • Zamore, P.D.; Tuschl, T.; Sharp, P.A.; Bartel, D.P. RNAi: Double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals. Cell 2000, 101, 25-33.
    • (2000) Cell , vol.101 , pp. 25-33
    • Zamore, P.D.1    Tuschl, T.2    Sharp, P.A.3    Bartel, D.P.4
  • 12
    • 0027751663 scopus 로고
    • The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14
    • Lee, R.C.; Feinbaum, R.L.; Ambros, V. The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14. Cell 1993, 75, 843-854.
    • (1993) Cell , vol.75 , pp. 843-854
    • Lee, R.C.1    Feinbaum, R.L.2    Ambros, V.3
  • 13
    • 33846283385 scopus 로고    scopus 로고
    • The evolution of gene regulation by transcription factors and microRNAs
    • Chen, K.; Rajewsky, N. The evolution of gene regulation by transcription factors and microRNAs. Nat. Rev. Genet. 2007, 8, 93-103.
    • (2007) Nat. Rev. Genet. , vol.8 , pp. 93-103
    • Chen, K.1    Rajewsky, N.2
  • 14
    • 84884132532 scopus 로고    scopus 로고
    • Manchester University. Available online: (accessed on 2 July 2013)
    • Manchester University. The miRBase. Available online: http://www.mirbase.org/cgi-bin/mirna _summary.pl?org=has/ (accessed on 2 July 2013).
    • The miRBase
  • 15
    • 0037009364 scopus 로고    scopus 로고
    • A maturation: Stepwise processing and subcellular localization
    • Lee, Y.; Jeon, K.; Lee, J.T.; Kim, S.; Kim, V.N.; Micro, R.N. A maturation: Stepwise processing and subcellular localization. EMBO J. 2002, 21, 4663-4670.
    • (2002) EMBO J. , vol.21 , pp. 4663-4670
    • Lee, Y.1    Jeon, K.2    Lee, J.T.3    Kim, S.4    Kim, V.N.5    Micro, R.N.6
  • 16
    • 10944254152 scopus 로고    scopus 로고
    • Transcription and processing of human microRNA precursors
    • Cullen, B.R. Transcription and processing of human microRNA precursors. Mol. Cell. 2004, 16, 861-865.
    • (2004) Mol. Cell. , vol.16 , pp. 861-865
    • Cullen, B.R.1
  • 17
    • 0035929322 scopus 로고    scopus 로고
    • A role for the RNase III enzyme DCR-1 in RNA interference and germ line development in Caenorhabditis elegans
    • Knight, S.W.; Bass, B.L. A role for the RNase III enzyme DCR-1 in RNA interference and germ line development in Caenorhabditis elegans. Science 2001, 293, 2269-2271.
    • (2001) Science , vol.293 , pp. 2269-2271
    • Knight, S.W.1    Bass, B.L.2
  • 18
    • 0035887005 scopus 로고    scopus 로고
    • Dicer functions in RNA interference and in synthesis of small RNA involved in developmental timing in C. elegans
    • Ketting, R.F.; Fischer, S.E.; Bernstein, E.; Sijen, T.; Hannon, G.J.; Plasterk, R.H. Dicer functions in RNA interference and in synthesis of small RNA involved in developmental timing in C. elegans. Genes Dev. 2001, 15, 2654-2659.
    • (2001) Genes Dev. , vol.15 , pp. 2654-2659
    • Ketting, R.F.1    Fischer, S.E.2    Bernstein, E.3    Sijen, T.4    Hannon, G.J.5    Plasterk, R.H.6
  • 19
    • 13244278197 scopus 로고    scopus 로고
    • siRNA and miRNA: An insight into RISCs
    • Tang, G. siRNA and miRNA: an insight into RISCs. Trends Biochem. Sci. 2005, 30, 106-114.
    • (2005) Trends Biochem. Sci. , vol.30 , pp. 106-114
    • Tang, G.1
  • 21
    • 77749334621 scopus 로고    scopus 로고
    • CCR4-NOT deadenylates mRNA associated with RNA-induced silencing complexes in human cells
    • Piao, X.; Zhang, X.; Wu, L.; Belasco, J.G. CCR4-NOT deadenylates mRNA associated with RNA-induced silencing complexes in human cells. Mol. Cell. Biol. 2010, 30, 1486-1494.
    • (2010) Mol. Cell. Biol. , vol.30 , pp. 1486-1494
    • Piao, X.1    Zhang, X.2    Wu, L.3    Belasco, J.G.4
  • 22
    • 77955644289 scopus 로고    scopus 로고
    • Mammalian microRNAs predominantly act to decrease target mRNA levels
    • Guo, H.; Ingolia, N.T.; Weissman, J.S.; Bartel, D.P. Mammalian microRNAs predominantly act to decrease target mRNA levels. Nature 2010, 466, 835-840.
    • (2010) Nature , vol.466 , pp. 835-840
    • Guo, H.1    Ingolia, N.T.2    Weissman, J.S.3    Bartel, D.P.4
  • 25
    • 77955475953 scopus 로고    scopus 로고
    • Expanding the microRNA targeting code: Functional sites with centered pairing
    • Shin, C.; Nam, J.W.; Farh, K.K.; Chiang, H.R.; Shkumatava, A.; Bartel, D.P. Expanding the microRNA targeting code: Functional sites with centered pairing. Mol. Cell. 2010, 38, 789-802.
    • (2010) Mol. Cell. , vol.38 , pp. 789-802
    • Shin, C.1    Nam, J.W.2    Farh, K.K.3    Chiang, H.R.4    Shkumatava, A.5    Bartel, D.P.6
  • 26
    • 82955233704 scopus 로고    scopus 로고
    • Rational design of therapeutic siRNAs: Minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease
    • Boudreau, R.L.; Spengler, R.M.; Davidson, B.L. Rational design of therapeutic siRNAs: Minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease. Mol. Ther. 2011, 19, 2169-2177.
    • (2011) Mol. Ther. , vol.19 , pp. 2169-2177
    • Boudreau, R.L.1    Spengler, R.M.2    Davidson, B.L.3
  • 27
    • 1642491747 scopus 로고    scopus 로고
    • Enhancement of RNAi activity by improved siRNA duplexes
    • Hohjoh, H. Enhancement of RNAi activity by improved siRNA duplexes. FEBS Lett. 2004, 557, 193-198.
    • (2004) FEBS Lett. , vol.557 , pp. 193-198
    • Hohjoh, H.1
  • 28
    • 0037134020 scopus 로고    scopus 로고
    • A system for stable expression of short interfering RNAs in mammalian cells
    • Brummelkamp, T.R.; Bernards, R.; Agami, R. A system for stable expression of short interfering RNAs in mammalian cells. Science 2002, 19, 550-553.
    • (2002) Science , vol.19 , pp. 550-553
    • Brummelkamp, T.R.1    Bernards, R.2    Agami, R.3
  • 29
    • 0036726313 scopus 로고    scopus 로고
    • Stable suppression of tumorigenicity by virus-mediated RNA interference
    • Brummelkamp, T.R.; Bernards, R.; Agami, R. Stable suppression of tumorigenicity by virus-mediated RNA interference. Cancer Cell 2002, 2, 243-247.
    • (2002) Cancer Cell , vol.2 , pp. 243-247
    • Brummelkamp, T.R.1    Bernards, R.2    Agami, R.3
  • 30
    • 0042267194 scopus 로고    scopus 로고
    • Efficient lentiviral vectors for short hairpin RNA delivery into human cells
    • An, D.S.; Xie, Y.; Mao, S.H.; Morizono, K.; Kung, S.K.; Chen, I.S. Efficient lentiviral vectors for short hairpin RNA delivery into human cells. Hum. Gene Ther. 2003, 14, 1207-1212.
    • (2003) Hum. Gene Ther. , vol.14 , pp. 1207-1212
    • An, D.S.1    Xie, Y.2    Mao, S.H.3    Morizono, K.4    Kung, S.K.5    Chen, I.S.6
  • 31
    • 0037197914 scopus 로고    scopus 로고
    • RNA interference by expression of short-interfering RNAs and hairpin RNAs in mammalian cells
    • Yu, J.Y.; DeRuiter, S.L.; Turner, D.L. RNA interference by expression of short-interfering RNAs and hairpin RNAs in mammalian cells. Proc. Natl. Acad. Sci. USA 2002, 99, 6047-6052.
    • (2002) Proc. Natl. Acad. Sci. USA , vol.99 , pp. 6047-6052
    • Yu, J.Y.1    DeRuiter, S.L.2    Turner, D.L.3
  • 33
    • 77956355233 scopus 로고    scopus 로고
    • Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver
    • Grimm, D.; Wang, L.; Lee, J.S.; Schurmann, N.; Gu, S.; Borner, K.; Storm, T.A.; Kay, M.A. Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver. J. Clin. Invest. 2010, 120, 3106-3119.
    • (2010) J. Clin. Invest. , vol.120 , pp. 3106-3119
    • Grimm, D.1    Wang, L.2    Lee, J.S.3    Schurmann, N.4    Gu, S.5    Borner, K.6    Storm, T.A.7    Kay, M.A.8
  • 35
    • 19444386359 scopus 로고    scopus 로고
    • Gene silencing in alveolar type II cells using cell-specific promoter in vitro and in vivo
    • Gou, D.; Narasaraju, T.; Chintagari, N.R.; Jin, N.; Wang, P.; Liu, L. Gene silencing in alveolar type II cells using cell-specific promoter in vitro and in vivo. Nucleic Acids Res. 2004, 32, e134.
    • (2004) Nucleic Acids Res. , vol.32
    • Gou, D.1    Narasaraju, T.2    Chintagari, N.R.3    Jin, N.4    Wang, P.5    Liu, L.6
  • 36
    • 50549092566 scopus 로고    scopus 로고
    • Expression of shRNA From a Tissue-specific pol II Promoter Is an Effective and Safe RNAi Therapeutic
    • Giering, J.C.; Grimm, D.; Storm, T.A.; Kay, M.A. Expression of shRNA From a Tissue-specific pol II Promoter Is an Effective and Safe RNAi Therapeutic. Mol. Ther. 2008, 16, 1630-1636.
    • (2008) Mol. Ther. , vol.16 , pp. 1630-1636
    • Giering, J.C.1    Grimm, D.2    Storm, T.A.3    Kay, M.A.4
  • 37
    • 28644450043 scopus 로고    scopus 로고
    • An RNA polymerase II construct synthesizes short-hairpin RNA with a quantitative indicator and mediates highly efficient RNAi
    • Zhou, H.; Xia, X.G.; Xu, Z. An RNA polymerase II construct synthesizes short-hairpin RNA with a quantitative indicator and mediates highly efficient RNAi. Nucleic Acids Res. 2005, 33, e62.
    • (2005) Nucleic Acids Res. , vol.33
    • Zhou, H.1    Xia, X.G.2    Xu, Z.3
  • 38
    • 24944560377 scopus 로고    scopus 로고
    • A lentiviral microRNA-based system for single-copy polymerase II-regulated RNA interference in mammalian cells
    • Stegmeier, F.; Hu, G.; Rickles, R.J.; Hannon, G.J.; Elledge, S.J. A lentiviral microRNA-based system for single-copy polymerase II-regulated RNA interference in mammalian cells. Proc. Natl. Acad. Sci. USA 2005, 102, 13212-13217.
    • (2005) Proc. Natl. Acad. Sci. USA , vol.102 , pp. 13212-13217
    • Stegmeier, F.1    Hu, G.2    Rickles, R.J.3    Hannon, G.J.4    Elledge, S.J.5
  • 40
    • 68949213635 scopus 로고    scopus 로고
    • Neuron-specific RNA interference using lentiviral vectors
    • Nielsen, T.T.; 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
  • 41
    • 14144250911 scopus 로고    scopus 로고
    • Recent advances with liposomes as pharmaceutical carriers
    • Torchilin, V.P. Recent advances with liposomes as pharmaceutical carriers. Nat. Rev. Drug Discov. 2005, 4, 145-160.
    • (2005) Nat. Rev. Drug Discov. , vol.4 , pp. 145-160
    • Torchilin, V.P.1
  • 44
    • 25444482607 scopus 로고    scopus 로고
    • Sequence-specific knockdown of EWS-FLI1 by targeted, nonviral delivery of small interfering RNA inhibits tumor growth in a murine model of metastatic Ewing's sarcoma
    • Hu-Lieskovan, S.; Heidel, J.D.; Bartlett, D.W.; Davis, M.E.; Triche, T.J. Sequence-specific knockdown of EWS-FLI1 by targeted, nonviral delivery of small interfering RNA inhibits tumor growth in a murine model of metastatic Ewing's sarcoma. Cancer Res. 2005, 65, 8984-8992.
    • (2005) Cancer Res. , vol.65 , pp. 8984-8992
    • Hu-Lieskovan, S.1    Heidel, J.D.2    Bartlett, D.W.3    Davis, M.E.4    Triche, T.J.5
  • 47
    • 0035525965 scopus 로고    scopus 로고
    • Introduction to retroviruses and retroviral vectors
    • Buchschacher, G.L., Jr. Introduction to retroviruses and retroviral vectors. Somat. Cell Mol. Genet. 2001, 26, 1-11.
    • (2001) Somat. Cell Mol. Genet. , vol.26 , pp. 1-11
    • Buchschacher Jr., G.L.1
  • 48
    • 0020582124 scopus 로고
    • Construction of a retrovirus packaging mutant and its use to produce helper-free defective retrovirus
    • Mann, R.; Mulligan, R.C.; Baltimore, D. Construction of a retrovirus packaging mutant and its use to produce helper-free defective retrovirus. Cell 1983, 33, 153-159.
    • (1983) Cell , vol.33 , pp. 153-159
    • Mann, R.1    Mulligan, R.C.2    Baltimore, D.3
  • 49
    • 0036145392 scopus 로고    scopus 로고
    • Oncoretrovirus and lentivirus vectors pseudotyped with lymphocytic choriomeningitis virus glycoprotein: Generation, concentration, and broad host range
    • Beyer, W.R.; Westphal, M.; Ostertag, W.; von Laer, D. Oncoretrovirus and lentivirus vectors pseudotyped with lymphocytic choriomeningitis virus glycoprotein: Generation, concentration, and broad host range. J. Virol. 2002, 76, 1488-1495.
    • (2002) J. Virol. , vol.76 , pp. 1488-1495
    • Beyer, W.R.1    Westphal, M.2    Ostertag, W.3    von Laer, D.4
  • 52
    • 22944458202 scopus 로고    scopus 로고
    • Altering the tropism of lentiviral vectors through pseudotyping
    • Cronin, J.; Zhang, X.Y.; Reiser, J. Altering the tropism of lentiviral vectors through pseudotyping. Curr. Gene Ther. 2005, 5, 387-398.
    • (2005) Curr. Gene Ther. , vol.5 , pp. 387-398
    • Cronin, J.1    Zhang, X.Y.2    Reiser, J.3
  • 53
    • 0031214241 scopus 로고    scopus 로고
    • Production of high-titer human immunodeficiency virus type 1 pseudotyped with vesicular stomatitis virus glycoprotein
    • Bartz, S.R.; Vodicka, M.A. Production of high-titer human immunodeficiency virus type 1 pseudotyped with vesicular stomatitis virus glycoprotein. Methods 1997, 12, 337-342.
    • (1997) Methods , vol.12 , pp. 337-342
    • Bartz, S.R.1    Vodicka, M.A.2
  • 54
    • 0029996147 scopus 로고    scopus 로고
    • In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
    • Naldini, L.; Blomer, U.; Gallay, P.; Ory, D.; Mulligan, R.; Gage, F.H.; Verma, I.M.; Trono, D. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996, 272, 263-267.
    • (1996) Science , vol.272 , pp. 263-267
    • Naldini, L.1    Blomer, U.2    Gallay, P.3    Ory, D.4    Mulligan, R.5    Gage, F.H.6    Verma, I.M.7    Trono, D.8
  • 55
    • 0347634408 scopus 로고    scopus 로고
    • Human immunodeficiency virus type 1-derived lentivirus vectors pseudotyped with envelope glycoproteins derived from Ross River virus and Semliki Forest virus
    • Kahl, C.A.; Marsh, J.; Fyffe, J.; Sanders, D.A.; Cornetta, K. Human immunodeficiency virus type 1-derived lentivirus vectors pseudotyped with envelope glycoproteins derived from Ross River virus and Semliki Forest virus. J. Virol. 2004, 78, 1421-1430.
    • (2004) J. Virol. , vol.78 , pp. 1421-1430
    • Kahl, C.A.1    Marsh, J.2    Fyffe, J.3    Sanders, D.A.4    Cornetta, K.5
  • 56
    • 33745752261 scopus 로고    scopus 로고
    • Herpes simplex virus RNAi and neprilysin gene transfer vectors reduce accumulation of Alzheimer's disease-related amyloid-beta peptide in vivo
    • Hong, C.S.; Goins, W.F.; Goss, J.R.; Burton, E.A.; Glorioso, J.C. Herpes simplex virus RNAi and neprilysin gene transfer vectors reduce accumulation of Alzheimer's disease-related amyloid-beta peptide in vivo. Gene Ther. 2006, 13, 1068-1079.
    • (2006) Gene Ther. , vol.13 , pp. 1068-1079
    • Hong, C.S.1    Goins, W.F.2    Goss, J.R.3    Burton, E.A.4    Glorioso, J.C.5
  • 57
    • 0029849257 scopus 로고    scopus 로고
    • Quantitative analysis of the packaging capacity of recombinant adeno-associated virus
    • Dong, J.Y.; Fan, P.D.; Frizzell, R.A. Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum. Gene Ther. 1996, 7, 2101-2112.
    • (1996) Hum. Gene Ther. , vol.7 , pp. 2101-2112
    • Dong, J.Y.1    Fan, P.D.2    Frizzell, R.A.3
  • 59
    • 0027092001 scopus 로고
    • Characterization of a preferred site on human chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination
    • Kotin, R.M.; Linden, R.M.; Berns, K.I. Characterization of a preferred site on human chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination. EMBO J. 1992, 11, 5071-5078.
    • (1992) EMBO J. , vol.11 , pp. 5071-5078
    • Kotin, R.M.1    Linden, R.M.2    Berns, K.I.3
  • 60
    • 0028169741 scopus 로고
    • Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain
    • Kaplitt, M.G.; Leone, P.; Samulski, R.J.; Xiao, X.; Pfaff, D.W.; O'Malley, K.L.; During, M.J. Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat. Genet. 1994, 8, 148-154.
    • (1994) Nat. Genet. , vol.8 , pp. 148-154
    • Kaplitt, M.G.1    Leone, P.2    Samulski, R.J.3    Xiao, X.4    Pfaff, D.W.5    O'Malley, K.L.6    During, M.J.7
  • 61
    • 0030003480 scopus 로고    scopus 로고
    • Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector
    • McCown, T.J.; Xiao, X.; Li, J.; Breese, G.R.; Samulski, R.J. Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain Res. 1996, 713, 99-107.
    • (1996) Brain Res. , vol.713 , pp. 99-107
    • McCown, T.J.1    Xiao, X.2    Li, J.3    Breese, G.R.4    Samulski, R.J.5
  • 62
    • 0010390169 scopus 로고
    • Cloning of adeno-associated virus into pBR322: Rescue of intact virus from the recombinant plasmid in human cells
    • Samulski, R.J.; Berns, K.I.; Tan, M.; Muzyczka, N. Cloning of adeno-associated virus into pBR322: Rescue of intact virus from the recombinant plasmid in human cells. Proc. Natl. Acad. Sci. USA 1982, 79, 2077-2081.
    • (1982) Proc. Natl. Acad. Sci. USA , vol.79 , pp. 2077-2081
    • Samulski, R.J.1    Berns, K.I.2    Tan, M.3    Muzyczka, N.4
  • 63
    • 84862025459 scopus 로고    scopus 로고
    • Directed evolution of novel adeno-associated viruses for therapeutic gene delivery
    • Bartel, M.A.; Weinstein, J.R.; Schaffer, D.V. Directed evolution of novel adeno-associated viruses for therapeutic gene delivery. Gene Ther. 2012, 19, 694-700.
    • (2012) Gene Ther. , vol.19 , pp. 694-700
    • Bartel, M.A.1    Weinstein, J.R.2    Schaffer, D.V.3
  • 65
    • 84875240244 scopus 로고    scopus 로고
    • Adeno-associated virus (AAV) gene therapy for neurological disease
    • Weinberg, M.S.; Samulski, R.J.; McCown, T.J. Adeno-associated virus (AAV) gene therapy for neurological disease. Neuropharmacology 2013, 69, 82-88.
    • (2013) Neuropharmacology , vol.69 , pp. 82-88
    • Weinberg, M.S.1    Samulski, R.J.2    McCown, T.J.3
  • 66
    • 0034212434 scopus 로고    scopus 로고
    • US gene therapy in crisis
    • Teichler, Z.D. US gene therapy in crisis. Trends Genet. 2000, 16, 272-275.
    • (2000) Trends Genet. , vol.16 , pp. 272-275
    • Teichler, Z.D.1
  • 69
    • 0025963855 scopus 로고
    • Pseudotype formation of murine leukemia virus with the G protein of vesicular stomatitis virus
    • Emi, N.; Friedmann, T.; Yee, J.K. Pseudotype formation of murine leukemia virus with the G protein of vesicular stomatitis virus. J. Virol. 1991, 65, 1202-1207.
    • (1991) J. Virol. , vol.65 , pp. 1202-1207
    • Emi, N.1    Friedmann, T.2    Yee, J.K.3
  • 71
    • 3242762057 scopus 로고    scopus 로고
    • Overexpression of glial cell line-derived neurotrophic factor using a lentiviral vector induces time-and dose-dependent downregulation of tyrosine hydroxylase in the intact nigrostriatal dopamine system
    • Georgievska, B.; Kirik, D.; Bjorklund, A. Overexpression of glial cell line-derived neurotrophic factor using a lentiviral vector induces time-and dose-dependent downregulation of tyrosine hydroxylase in the intact nigrostriatal dopamine system. J. Neuro. Sci. 2004, 24, 6437-6445.
    • (2004) J. Neuro. Sci. , vol.24 , pp. 6437-6445
    • Georgievska, B.1    Kirik, D.2    Bjorklund, A.3
  • 73
    • 33744820829 scopus 로고    scopus 로고
    • Efficient transduction of neurons using Ross River glycoprotein-pseudotyped lentiviral vectors
    • Jakobsson, J.; Nielsen, T.T.; Staflin, K.; Georgievska, B.; Lundberg, C. Efficient transduction of neurons using Ross River glycoprotein-pseudotyped lentiviral vectors. Gene Ther. 2006, 13, 966-973.
    • (2006) Gene Ther. , vol.13 , pp. 966-973
    • Jakobsson, J.1    Nielsen, T.T.2    Staflin, K.3    Georgievska, B.4    Lundberg, C.5
  • 74
    • 0029993858 scopus 로고    scopus 로고
    • Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector
    • Naldini, L.; Blomer, U.; Gage, F.H.; Trono, D.; Verma, I.M. Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc. Natl. Acad. Sci. USA 1996, 93, 11382-11388.
    • (1996) Proc. Natl. Acad. Sci. USA , vol.93 , pp. 11382-11388
    • Naldini, L.1    Blomer, U.2    Gage, F.H.3    Trono, D.4    Verma, I.M.5
  • 78
    • 11144229400 scopus 로고    scopus 로고
    • Directed evolution of retrovirus envelope protein cytoplasmic tails guided by functional incorporation into lentivirus particles
    • Merten, C.A.; Stitz, J.; Braun, G.; Poeschla, E.M.; Cichutek, K.; Buchholz, C.J. Directed evolution of retrovirus envelope protein cytoplasmic tails guided by functional incorporation into lentivirus particles. J. Virol. 2005, 79, 834-840.
    • (2005) J. Virol. , vol.79 , pp. 834-840
    • Merten, C.A.1    Stitz, J.2    Braun, G.3    Poeschla, E.M.4    Cichutek, K.5    Buchholz, C.J.6
  • 79
    • 84881480856 scopus 로고    scopus 로고
    • Engineering a serum-resistant and thermostable vesicular stomatitis virus G glycoprotein for pseudotyping retroviral and lentiviral vectors
    • doi:10.1038/gt.2013.1
    • Hwang, B.Y.; Schaffer, D.V. Engineering a serum-resistant and thermostable vesicular stomatitis virus G glycoprotein for pseudotyping retroviral and lentiviral vectors. Gene Ther. 2013, doi:10.1038/gt.2013.1.
    • (2013) Gene Ther.
    • Hwang, B.Y.1    Schaffer, D.V.2
  • 81
    • 68649111166 scopus 로고    scopus 로고
    • Adeno-associated virus vector integration
    • Deyle, D.R.; Russell, D.W. Adeno-associated virus vector integration. Curr. Opin. Mol. Ther. 2009, 11, 442-447.
    • (2009) Curr. Opin. Mol. Ther. , vol.11 , pp. 442-447
    • Deyle, D.R.1    Russell, D.W.2
  • 83
    • 84884153747 scopus 로고    scopus 로고
    • UCL Institute of Child Health and Great Ormond Street Hospital for Children NHS Trust. Available online: (accessed on 2 July 2013)
    • UCL Institute of Child Health and Great Ormond Street Hospital for Children NHS Trust. GOSH Announces Leukaemia Case Following Gene Therapy for X-SCID. Available online: http://www.ich.ucl.ac.uk/pressoffice/pressrelease_00591/ (accessed on 2 July 2013).
    • GOSH Announces Leukaemia Case Following Gene Therapy for X-SCID.
  • 84
    • 80054069178 scopus 로고    scopus 로고
    • The influence of epileptic neuropathology and prior peripheral immunity on CNS transduction by rAAV2 and rAAV5
    • Weinberg, M.S.; Blake, B.L.; Samulski, R.J.; McCown, T.J. The influence of epileptic neuropathology and prior peripheral immunity on CNS transduction by rAAV2 and rAAV5. Gene Ther. 2011, 18, 961-968.
    • (2011) Gene Ther. , vol.18 , pp. 961-968
    • Weinberg, M.S.1    Blake, B.L.2    Samulski, R.J.3    McCown, T.J.4
  • 85
    • 77649270699 scopus 로고    scopus 로고
    • Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates
    • Dodiya, H.B.; Bjorklund, T.; Stansell, J., III; Mandel, R.J.; Kirik, D.; Kordower, J.H. Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates. Mol. Ther. 2010, 18, 579-587.
    • (2010) Mol. Ther. , vol.18 , pp. 579-587
    • Dodiya, H.B.1    Bjorklund, T.2    Stansell III, J.3    Mandel, R.J.4    Kirik, D.5    Kordower, J.H.6
  • 86
    • 33645452310 scopus 로고    scopus 로고
    • Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain
    • Hadaczek, P.; Kohutnicka, M.; Krauze, M.T.; Bringas, J.; Pivirotto, P.; Cunningham, J.; Bankiewicz, K. Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain. Hum. Gene Ther. 2006, 17, 291-302.
    • (2006) Hum. Gene Ther. , vol.17 , pp. 291-302
    • Hadaczek, P.1    Kohutnicka, M.2    Krauze, M.T.3    Bringas, J.4    Pivirotto, P.5    Cunningham, J.6    Bankiewicz, K.7
  • 88
    • 79957686690 scopus 로고    scopus 로고
    • Efficient gene transfer into neurons in monkey brain by adeno-associated virus 8
    • Masamizu, Y.; Okada, T.; Ishibashi, H.; Takeda, S.; Yuasa, S.; Nakahara, K. Efficient gene transfer into neurons in monkey brain by adeno-associated virus 8. Neuroreport 2010, 21, 447-451.
    • (2010) Neuroreport , vol.21 , pp. 447-451
    • Masamizu, Y.1    Okada, T.2    Ishibashi, H.3    Takeda, S.4    Yuasa, S.5    Nakahara, K.6
  • 90
    • 37549061385 scopus 로고    scopus 로고
    • AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: Effects of serotype, promoter and purification method
    • Klein, R.L.; Dayton, R.D.; Tatom, J.B.; Henderson, K.M.; Henning, P.P. AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method. Mol. Ther. 2008, 16, 89-96.
    • (2008) Mol. Ther. , vol.16 , pp. 89-96
    • Klein, R.L.1    Dayton, R.D.2    Tatom, J.B.3    Henderson, K.M.4    Henning, P.P.5
  • 91
    • 33750600649 scopus 로고    scopus 로고
    • Enhanced gene transfer efficiency in the murine striatum and an orthotopic glioblastoma tumor model, using AAV-7-and AAV-8-pseudotyped vectors
    • Harding, T.C.; Dickinson, P.J.; Roberts, B.N.; Yendluri, S.; Gonzalez-Edick, M.; Lecouteur, R.A.; Jooss, K.U. Enhanced gene transfer efficiency in the murine striatum and an orthotopic glioblastoma tumor model, using AAV-7-and AAV-8-pseudotyped vectors. Hum. Gene Ther. 2006, 17, 807-820.
    • (2006) Hum. Gene Ther. , vol.17 , pp. 807-820
    • Harding, T.C.1    Dickinson, P.J.2    Roberts, B.N.3    Yendluri, S.4    Gonzalez-Edick, M.5    Lecouteur, R.A.6    Jooss, K.U.7
  • 92
    • 80053923197 scopus 로고    scopus 로고
    • Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system
    • Rahim, A.A.; Wong, A.M.; Hoefer, K.; Buckley, S.M.; Mattar, C.N.; Cheng, S.H.; Chan, J.K.; Cooper, J.D.; Waddington, S.N. Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system. FASEB J. 2011, 25, 3505-3518.
    • (2011) FASEB J. , vol.25 , pp. 3505-3518
    • Rahim, A.A.1    Wong, A.M.2    Hoefer, K.3    Buckley, S.M.4    Mattar, C.N.5    Cheng, S.H.6    Chan, J.K.7    Cooper, J.D.8    Waddington, S.N.9
  • 93
    • 79957959362 scopus 로고    scopus 로고
    • RNAi momentum fizzles as pharma shifts priorities
    • Schmidt, C. RNAi momentum fizzles as pharma shifts priorities. Nat. Biotechnol. 2011, 29, 93-94.
    • (2011) Nat. Biotechnol. , vol.29 , pp. 93-94
    • Schmidt, C.1
  • 94
    • 26844494370 scopus 로고    scopus 로고
    • Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA
    • Wang, Y.L.; Liu, W.; Wada, E.; Murata, M.; Wada, K.; Kanazawa, I. Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA. Neurosci. Res. 2005, 53, 241-249.
    • (2005) Neurosci. Res. , vol.53 , pp. 241-249
    • Wang, Y.L.1    Liu, W.2    Wada, E.3    Murata, M.4    Wada, K.5    Kanazawa, I.6
  • 97
    • 80052250736 scopus 로고    scopus 로고
    • Inhibition of mutant huntingtin expression by RNA duplex targeting expanded CAG repeats
    • Fiszer, A.; Mykowska, A.; Krzyzosiak, W.J. Inhibition of mutant huntingtin expression by RNA duplex targeting expanded CAG repeats. Nucleic. Acid. Res. 2011, 39, 5578-5585.
    • (2011) Nucleic. Acid. Res. , vol.39 , pp. 5578-5585
    • Fiszer, A.1    Mykowska, A.2    Krzyzosiak, W.J.3
  • 98
    • 78649359013 scopus 로고    scopus 로고
    • Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism
    • Hu, J.; Liu, J.; Corey, D.R. Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism. Chem. Biol. 2010, 17, 1183-1188.
    • (2010) Chem. Biol. , vol.17 , pp. 1183-1188
    • Hu, J.1    Liu, J.2    Corey, D.R.3
  • 99
    • 47549105506 scopus 로고    scopus 로고
    • Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts
    • Van Bilsen, P.H.; Jaspers, L.; Lombardi, M.S.; Odekerken, J.C.; Burright, E.N.; Kaemmerer, W.F. Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts. Hum. Gene Ther. 2008, 19, 710-719.
    • (2008) Hum. Gene Ther. , vol.19 , pp. 710-719
    • van Bilsen, P.H.1    Jaspers, L.2    Lombardi, M.S.3    Odekerken, J.C.4    Burright, E.N.5    Kaemmerer, W.F.6
  • 105
    • 79960269997 scopus 로고    scopus 로고
    • Splice isoform-specific suppression of the Cav2.1 variant underlying spinocerebellar ataxia type 6
    • Tsou, W.L.; Soong, B.W.; Paulson, H.L.; Rodriguez-Lebron, E. Splice isoform-specific suppression of the Cav2.1 variant underlying spinocerebellar ataxia type 6. Neurobiol. Dis. 2011, 43, 533-542.
    • (2011) Neurobiol. Dis. , vol.43 , pp. 533-542
    • Tsou, W.L.1    Soong, B.W.2    Paulson, H.L.3    Rodriguez-Lebron, E.4
  • 106
    • 33846856920 scopus 로고    scopus 로고
    • RNA interference-mediated knockdown of alpha-synuclein protects human dopaminergic neuroblastoma cells from MPP(+) toxicity and reduces dopamine transport
    • Fountaine, T.M.; Wade-Martins, R. RNA interference-mediated knockdown of alpha-synuclein protects human dopaminergic neuroblastoma cells from MPP(+) toxicity and reduces dopamine transport. J. Neurosci. Res. 2007, 85, 351-363.
    • (2007) J. Neurosci. Res. , vol.85 , pp. 351-363
    • Fountaine, T.M.1    Wade-Martins, R.2
  • 107
    • 33645854350 scopus 로고    scopus 로고
    • Silencing of human alpha-synuclein in vitro and in rat brain using lentiviral-mediated RNAi
    • Sapru, M.K.; Yates, J.W.; Hogan, S.; Jiang, L.; Halter, J.; Bohn, M.C. Silencing of human alpha-synuclein in vitro and in rat brain using lentiviral-mediated RNAi. Exp. Neurol. 2006, 198, 382-390.
    • (2006) Exp. Neurol. , vol.198 , pp. 382-390
    • Sapru, M.K.1    Yates, J.W.2    Hogan, S.3    Jiang, L.4    Halter, J.5    Bohn, M.C.6
  • 108
    • 79959330427 scopus 로고    scopus 로고
    • Efficient allele-specific targeting of LRRK2 R1441 mutations mediated by RNAi
    • De Ynigo-Mojado, L.; Martin-Ruiz, I.; Sutherland, J.D. Efficient allele-specific targeting of LRRK2 R1441 mutations mediated by RNAi. PLoS One 2011, 6, e21352.
    • (2011) PLoS One , vol.6
    • De Ynigo-Mojado, L.1    Martin-Ruiz, I.2    Sutherland, J.D.3
  • 109
    • 84868132813 scopus 로고    scopus 로고
    • Silencing of Parkinson's disease-associated genes with artificial mirtron mimics of miR-1224
    • Sibley, C.R.; Seow, Y.; Curtis, H.; Weinberg, M.S.; Wood, M.J. Silencing of Parkinson's disease-associated genes with artificial mirtron mimics of miR-1224. Nucleic. Acid. Res. 2012, 40, 9863-9875.
    • (2012) Nucleic. Acid. Res. , vol.40 , pp. 9863-9875
    • Sibley, C.R.1    Seow, Y.2    Curtis, H.3    Weinberg, M.S.4    Wood, M.J.5
  • 111
    • 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, T.T.; 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
  • 112
    • 0242379146 scopus 로고    scopus 로고
    • Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis
    • Ding, H.; Schwarz, D.S.; Keene, A.; Affar, E.; Fenton, L.; Xia, X.; Shi, Y.; Zamore, P.D.; Xu, Z.; et al. Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis. Aging Cell 2003, 2, 209-217.
    • (2003) Aging Cell , vol.2 , pp. 209-217
    • Ding, H.1    Schwarz, D.S.2    Keene, A.3    Affar, E.4    Fenton, L.5    Xia, X.6    Shi, Y.7    Zamore, P.D.8    Xu, Z.9
  • 114
    • 33747201641 scopus 로고    scopus 로고
    • Allele-specific RNAi selectively silences mutant SOD1 and achieves significant therapeutic benefit in vivo
    • Xia, X.; Zhou, H.; Huang, Y.; Xu, Z. Allele-specific RNAi selectively silences mutant SOD1 and achieves significant therapeutic benefit in vivo. Neurobiol. Dis. 2006, 23, 578-586.
    • (2006) Neurobiol. Dis. , vol.23 , pp. 578-586
    • Xia, X.1    Zhou, H.2    Huang, Y.3    Xu, Z.4
  • 115
    • 44349169012 scopus 로고    scopus 로고
    • Systemic AAV6 delivery mediating RNA interference against SOD1: Neuromuscular transduction does not alter disease progression in fALS mice
    • Towne, C.; Raoul, C.; Schneider, B.L.; Aebischer, P. Systemic AAV6 delivery mediating RNA interference against SOD1: Neuromuscular transduction does not alter disease progression in fALS mice. Mol. Ther. 2008, 16, 1018-1025.
    • (2008) Mol. Ther. , vol.16 , pp. 1018-1025
    • Towne, C.1    Raoul, C.2    Schneider, B.L.3    Aebischer, P.4
  • 116
    • 79551641133 scopus 로고    scopus 로고
    • Neuroprotection by gene therapy targeting mutant SOD1 in individual pools of motor neurons does not translate into therapeutic benefit in fALS mice
    • Towne, C.; Setola, V.; Schneider, B.L.; Aebischer, P. Neuroprotection by gene therapy targeting mutant SOD1 in individual pools of motor neurons does not translate into therapeutic benefit in fALS mice. Mol. Ther. 2011, 19, 274-283.
    • (2011) Mol. Ther. , vol.19 , pp. 274-283
    • Towne, C.1    Setola, V.2    Schneider, B.L.3    Aebischer, P.4
  • 117
    • 4544225643 scopus 로고    scopus 로고
    • Effects of RNA interference-mediated silencing of gamma-secretase complex components on cell sensitivity to caspase-3 activation
    • Xie, Z.; Romano, D.M.; Kovacs, D.M.; Tanzi, R.E. Effects of RNA interference-mediated silencing of gamma-secretase complex components on cell sensitivity to caspase-3 activation. J. Biol. Chem. 2004, 279, 34130-34137.
    • (2004) J. Biol. Chem. , vol.279 , pp. 34130-34137
    • Xie, Z.1    Romano, D.M.2    Kovacs, D.M.3    Tanzi, R.E.4
  • 118
    • 84855213321 scopus 로고    scopus 로고
    • siRNA against presenilin 1 (PS1) down regulates amyloid beta42 production in IMR-32 cells
    • Kandimalla, R.J.; Wani, W.Y.; Binukumar, B.K.; Gill, K.D. siRNA against presenilin 1 (PS1) down regulates amyloid beta42 production in IMR-32 cells. J. Biomed. Sci. 2012, 19, 2.
    • (2012) J. Biomed. Sci. , vol.19 , pp. 2
    • Kandimalla, R.J.1    Wani, W.Y.2    Binukumar, B.K.3    Gill, K.D.4
  • 119
    • 80053632331 scopus 로고    scopus 로고
    • Lentivirus-mediated APP695-RNAi reduces apoptosis in APP transgenic mouse neurons
    • Zhao, X.; Wen, L.; Li, G.; Ba, Q.; Cui, Y.; Han, Z.; Jia, Y.; Xu, Y. Lentivirus-mediated APP695-RNAi reduces apoptosis in APP transgenic mouse neurons. Neuroreport 2011, 22, 804-808.
    • (2011) Neuroreport , vol.22 , pp. 804-808
    • Zhao, X.1    Wen, L.2    Li, G.3    Ba, Q.4    Cui, Y.5    Han, Z.6    Jia, Y.7    Xu, Y.8
  • 123
    • 72749101173 scopus 로고    scopus 로고
    • Divalent metal transporter 1 is involved in amyloid precursor protein processing and Abeta generation
    • Zheng, W.; Xin, N.; Chi, Z.H.; Zhao, B.L.; Zhang, J.; Li, J.Y.; Wang, Z.Y. Divalent metal transporter 1 is involved in amyloid precursor protein processing and Abeta generation. FASEB J. 2009, 23, 4207-4217.
    • (2009) FASEB J. , vol.23 , pp. 4207-4217
    • Zheng, W.1    Xin, N.2    Chi, Z.H.3    Zhao, B.L.4    Zhang, J.5    Li, J.Y.6    Wang, Z.Y.7
  • 125
    • 84857052288 scopus 로고    scopus 로고
    • Current concepts in multiple sclerosis: Autoimmunity versus oligodendrogliopathy
    • Nakahara, J.; Maeda, M.; Aiso, S.; Suzuki, N. Current concepts in multiple sclerosis: Autoimmunity versus oligodendrogliopathy. Clin. Rev. Allergy Immunol. 2012, 42, 26-34.
    • (2012) Clin. Rev. Allergy Immunol , vol.42 , pp. 26-34
    • Nakahara, J.1    Maeda, M.2    Aiso, S.3    Suzuki, N.4
  • 132
    • 34547136437 scopus 로고    scopus 로고
    • Expression of mutated huntingtin fragment in the putamen is sufficient to produce abnormal movement in non-human primates
    • Palfi, S.; Brouillet, E.; Jarraya, B.; Bloch, J.; Jan, C.; Shin, M.; Conde, F.; Li, X.J.; Aebischer, P.; Hantraye, P.; et al. Expression of mutated huntingtin fragment in the putamen is sufficient to produce abnormal movement in non-human primates. Mol. Ther. 2007, 15, 1444-1451.
    • (2007) Mol. Ther. , vol.15 , pp. 1444-1451
    • Palfi, S.1    Brouillet, E.2    Jarraya, B.3    Bloch, J.4    Jan, C.5    Shin, M.6    Conde, F.7    Li, X.J.8    Aebischer, P.9    Hantraye, P.10
  • 134
    • 33745003424 scopus 로고    scopus 로고
    • Cleavage at the caspase-6 site is required for neuronal dysfunction and degeneration due to mutant huntingtin
    • Graham, R.K.; Deng, Y.; Slow, E.J.; Haigh, B.; Bissada, N.; Lu, G.; Pearson, J.; Shehadeh, J.; Bertram, L.; Murphy, Z.; et al. Cleavage at the caspase-6 site is required for neuronal dysfunction and degeneration due to mutant huntingtin. Cell 2006, 125, 1179-1191.
    • (2006) Cell , vol.125 , 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
  • 135
    • 0011093279 scopus 로고    scopus 로고
    • Bird, T.D., Pagon, R.A., Adam, M.P., Dolan, C.R., Fong, C.T., Stephens, K., Eds.; University of Washington: Seattle, WA, USA
    • Bird, T.D. Hereditary Ataxia Overview; Bird, T.D., Pagon, R.A., Adam, M.P., Dolan, C.R., Fong, C.T., Stephens, K., Eds.; University of Washington: Seattle, WA, USA, 2012.
    • (2012) Hereditary Ataxia Overview
    • Bird, T.D.1
  • 136
    • 41149163183 scopus 로고    scopus 로고
    • Parkinson's disease: Clinical features and diagnosis
    • Jankovic, J. Parkinson's disease: Clinical features and diagnosis. J. Neurol. Neurosurg. Psychiatry 2008, 79, 368-376.
    • (2008) J. Neurol. Neurosurg. Psychiatry , vol.79 , pp. 368-376
    • Jankovic, J.1
  • 137
    • 44949138796 scopus 로고    scopus 로고
    • A review of Parkinson's disease
    • Davie, C.A. A review of Parkinson's disease. Br. Med. Bull. 2008, 86, 109-127.
    • (2008) Br. Med. Bull. , vol.86 , pp. 109-127
    • Davie, C.A.1
  • 138
    • 37649004547 scopus 로고    scopus 로고
    • Cell systems and the toxic mechanism(s) of alpha-synuclein
    • Cookson, M.R.; van der Brug, M. Cell systems and the toxic mechanism(s) of alpha-synuclein. Exp. Neurol. 2008, 209, 5-11.
    • (2008) Exp. Neurol. , vol.209 , pp. 5-11
    • Cookson, M.R.1    van der Brug, M.2
  • 141
    • 84874374889 scopus 로고    scopus 로고
    • Advances with RNA interference in Alzheimer's disease research
    • Chen, S.; Ge, X.; Chen, Y.; Lv, N.; Liu, Z.; Yuan, W. Advances with RNA interference in Alzheimer's disease research. Drug Des. Dev. Ther. 2013, 7, 117-125.
    • (2013) Drug Des. Dev. Ther. , vol.7 , pp. 117-125
    • Chen, S.1    Ge, X.2    Chen, Y.3    Lv, N.4    Liu, Z.5    Yuan, W.6
  • 143
    • 0344442731 scopus 로고    scopus 로고
    • Selective modifications in GAD67 mRNA levels in striatonigral and striatopallidal pathways correlate to dopamine agonist priming in 6-hydroxydopamine-lesioned rats
    • Carta, A.R.; Fenu, S.; Pala, P.; Tronci, E.; Morelli, M. Selective modifications in GAD67 mRNA levels in striatonigral and striatopallidal pathways correlate to dopamine agonist priming in 6-hydroxydopamine-lesioned rats. Eur. J. Neurosci. 2003, 18, 2563-2572.
    • (2003) Eur. J. Neurosci. , vol.18 , pp. 2563-2572
    • Carta, A.R.1    Fenu, S.2    Pala, P.3    Tronci, E.4    Morelli, M.5
  • 144
    • 0036929017 scopus 로고    scopus 로고
    • Pathophysiology of Parkinson's disease: From clinical neurology to basic neuroscience and back
    • Bergman, H.; Deuschl, G. Pathophysiology of Parkinson's disease: From clinical neurology to basic neuroscience and back. Mov. Disord. 2002, 17, S28-S40.
    • (2002) Mov. Disord. , vol.17
    • Bergman, H.1    Deuschl, G.2
  • 145
    • 0037728902 scopus 로고    scopus 로고
    • Regulation of neuropeptide mRNA expression in the basal ganglia by intrastriatal and intranigral transplants in the rat Parkinson model
    • Winkler, C.; Bentlage, C.; Cenci, M.A.; Nikkhah, G.; Bjorklund, A. Regulation of neuropeptide mRNA expression in the basal ganglia by intrastriatal and intranigral transplants in the rat Parkinson model. Neuroscience 2003, 118, 1063-1077.
    • (2003) Neuroscience , vol.118 , pp. 1063-1077
    • Winkler, C.1    Bentlage, C.2    Cenci, M.A.3    Nikkhah, G.4    Bjorklund, A.5
  • 146
    • 0026516393 scopus 로고
    • Messenger RNAs encoding glutamatedecarboxylases are differentially affected by nigrostriatal lesions in subpopulations of striatal neurons
    • Soghomonian, J.J.; Gonzales, C.; Chesselet, M.F. Messenger RNAs encoding glutamatedecarboxylases are differentially affected by nigrostriatal lesions in subpopulations of striatal neurons. Brain Res. 1992, 576, 68-79.
    • (1992) Brain Res. , vol.576 , pp. 68-79
    • Soghomonian, J.J.1    Gonzales, C.2    Chesselet, M.F.3
  • 147
    • 0038389600 scopus 로고    scopus 로고
    • Pathophysiology of Parkinson's disease: The MPTP primate model of the human disorder
    • Wichmann, T.; DeLong, M.R. Pathophysiology of Parkinson's disease: The MPTP primate model of the human disorder. Ann. N. Y. Acad. Sci. 2003, 991, 199-213.
    • (2003) Ann. N. Y. Acad. Sci. , vol.991 , pp. 199-213
    • Wichmann, T.1    DeLong, M.R.2
  • 148
    • 0031031536 scopus 로고    scopus 로고
    • Consequences of nigrostriatal denervation on the functioning of the basal ganglia in human and nonhuman primates: An in situ hybridization study of cytochrome oxidase subunit I mRNA
    • Vila, M.; Levy, R.; Herrero, M.T.; Ruberg, M.; Faucheux, B.; Obeso, J.A.; Agid, Y.; Hirsch, E.C. Consequences of nigrostriatal denervation on the functioning of the basal ganglia in human and nonhuman primates: An in situ hybridization study of cytochrome oxidase subunit I mRNA. J. Neurosci. 1997, 17, 765-773.
    • (1997) J. Neurosci. , vol.17 , pp. 765-773
    • Vila, M.1    Levy, R.2    Herrero, M.T.3    Ruberg, M.4    Faucheux, B.5    Obeso, J.A.6    Agid, Y.7    Hirsch, E.C.8
  • 149
    • 33745785622 scopus 로고    scopus 로고
    • Evidence for disease-regulated transgene expression in the brain with use of lentiviral vectors
    • Jakobsson, J.; Rosenqvist, N.; Marild, K.; Agoston, V.; Lundberg, C. Evidence for disease-regulated transgene expression in the brain with use of lentiviral vectors. J. Neurosci. Res. 2006, 84, 58-67.
    • (2006) J. Neurosci. Res. , vol.84 , pp. 58-67
    • Jakobsson, J.1    Rosenqvist, N.2    Marild, K.3    Agoston, V.4    Lundberg, C.5
  • 150
    • 0023214660 scopus 로고
    • Increase of enkephalin and decrease of substance P immunoreactivity in the dorsal and ventral striatum of the rat after midbrain 6-hydroxydopamine lesions
    • Voorn, P.; Roest, G.; Groenewegen, H.J. Increase of enkephalin and decrease of substance P immunoreactivity in the dorsal and ventral striatum of the rat after midbrain 6-hydroxydopamine lesions. Brain Res. 1987, 412, 391-396.
    • (1987) Brain Res. , vol.412 , pp. 391-396
    • Voorn, P.1    Roest, G.2    Groenewegen, H.J.3
  • 152
    • 84863463743 scopus 로고    scopus 로고
    • CNS-specific therapy for ongoing EAE by silencing IL-17 pathway in astrocytes
    • Yan, Y.; Ding, X.; Li, K.; Ciric, B.; Wu, S.; Xu, H.; Gran, B.; Rostami, A.; Zhang, G.X. CNS-specific therapy for ongoing EAE by silencing IL-17 pathway in astrocytes. Mol. Ther. 2012, 20, 1338-1348.
    • (2012) Mol. Ther. , vol.20 , pp. 1338-1348
    • Yan, Y.1    Ding, X.2    Li, K.3    Ciric, B.4    Wu, S.5    Xu, H.6    Gran, B.7    Rostami, A.8    Zhang, G.X.9


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.