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Volumn 3, Issue 9, 2012, Pages 1061-1076

RNAi-based therapies for Huntingtons disease: Delivery challenges and opportunities

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; ANTIDEPRESSANT AGENT; ANTIOXIDANT; APOPTOSIS INHIBITOR; BACULOVIRUS VECTOR; BREAST CANCER RESISTANCE PROTEIN; CELL PENETRATING PEPTIDE; CHITOSAN; CHOLESTEROL; DOPAMINE; DOPAMINE RECEPTOR BLOCKING AGENT; DOUBLE STRANDED RNA; GLUTAMATE RECEPTOR ANTAGONIST; HUNTINGTIN; LENTIVIRUS VECTOR; LIPOSOME; MACROGOL; MONOAMINE OXIDASE INHIBITOR; NEUROLEPTIC AGENT; PARVOVIRUS VECTOR; POLYAMIDOAMINE; POLYETHYLENEIMINE; POLYGLUTAMINE; RETROVIRUS VECTOR; RNA INDUCED SILENCING COMPLEX; SHORT HAIRPIN RNA; SMALL INTERFERING RNA; TETRABENAZINE; UNINDEXED DRUG; VESICULAR MONOAMINE TRANSPORTER 2;

EID: 84865766602     PISSN: 20415990     EISSN: 20416008     Source Type: Journal    
DOI: 10.4155/tde.12.80     Document Type: Review
Times cited : (5)

References (103)
  • 3
    • 77950857170 scopus 로고    scopus 로고
    • Huntington's disease: Pathogenesis to animal models
    • Kumar P, Kalonia H, Kumar A. Huntington's disease: pathogenesis to animal models. Pharmacol. Rep. 62(1), 1-14 (2010).
    • (2010) Pharmacol. Rep. , vol.62 , Issue.1 , pp. 1-14
    • Kumar, P.1    Kalonia, H.2    Kumar, A.3
  • 4
    • 44949123944 scopus 로고    scopus 로고
    • Molecular genetics of Huntington's Disease: When size does matter
    • Ersoy N. Molecular genetics of Huntington's Disease: when size does matter. J. Cell Mol. Biol. 6(1), 1-8 (2007). (Pubitemid 351802493)
    • (2007) Journal of Cell and Molecular Biology , vol.6 , Issue.1 , pp. 1-8
    • Ersoy, N.1
  • 5
    • 0035504919 scopus 로고    scopus 로고
    • Polyglutamine expansion neurodegenerative disease
    • DOI 10.1016/S0361-9230(01)00577-9, PII S0361923001005779
    • Fischbeck KH. Polyglutamine expansion neurodegenerative disease. Brain Res. Bull. 56(3-4), 161-163 (2001). (Pubitemid 33062337)
    • (2001) Brain Research Bulletin , vol.56 , Issue.3-4 , pp. 161-163
    • Fischbeck, K.H.1
  • 6
    • 0034640011 scopus 로고    scopus 로고
    • Fourteen and counting: Unraveling trinucleotide repeat diseases
    • Cummings CJ, Zoghbi HY. Fourteen and counting: unraveling trinucleotide repeat diseases. Human Mol. Genet. 9(6), 909-916 (2000). (Pubitemid 30216102)
    • (2000) Human Molecular Genetics , vol.9 , Issue.6 , pp. 909-916
    • Cummings, C.J.1    Zoghbi, H.Y.2
  • 7
    • 78650228452 scopus 로고    scopus 로고
    • Huntington's disease: From molecular basis to therapeutic advances
    • Overview of Huntington's disease
    • Krobitsch S, Kazantsev AG. Huntington's disease: from molecular basis to therapeutic advances. Int. J. Biochem. Cell Biol. 43(1), 20-24 (2011). Overview of Huntington's disease.
    • (2011) Int. J. Biochem. Cell Biol. , vol.43 , Issue.1 , pp. 20-24
    • Krobitsch, S.1    Kazantsev, A.G.2
  • 8
    • 1242338856 scopus 로고    scopus 로고
    • Huntingtin-protein interactions and the pathogenesis of Huntington's disease
    • Li SH, Li XJ. Huntingtin-protein interactions and the pathogenesis of Huntington's disease. Trends Genet. 20(3), 146-154 (2004).
    • (2004) Trends Genet. , vol.20 , Issue.3 , pp. 146-154
    • Li, S.H.1    Li, X.J.2
  • 9
    • 35348980793 scopus 로고    scopus 로고
    • Nucleocytoplasmic trafficking and transcription effects of huntingtin in Huntington's disease
    • DOI 10.1016/j.pneurobio.2006.11.004, PII S0301008206001547, Chromatin Dysfunction in Huntington's Disease
    • Truant R, Atwal RS, Burtnik A. Nucleocytoplasmic trafficking and transcription effects of huntingtin in Huntington's disease. Prog. Neurobiol. 83(4), 211-227 (2007). (Pubitemid 47595404)
    • (2007) Progress in Neurobiology , vol.83 , Issue.4 , pp. 211-227
    • Truant, R.1    Atwal, R.S.2    Burtnik, A.3
  • 10
    • 0034283877 scopus 로고    scopus 로고
    • Transcriptional dysregulation in Huntington's disease
    • Cha JH. Transcriptional dysregulation in Huntington's disease. Trends Neurosci. 23(9), 387-392 (2000).
    • (2000) Trends Neurosci. , vol.23 , Issue.9 , pp. 387-392
    • Cha, J.H.1
  • 11
    • 0037101835 scopus 로고    scopus 로고
    • Dysregulation of gene expression in the R6/2 model of polyglutamine disease: Parallel changes in muscle and brain
    • Demonstrated functional disruption of genes upon mutant HTT expression
    • Luthi-Carter R, Hanson SA, Strand AD et al. Dysregulation of gene expression in the R6/2 model of polyglutamine disease: parallel changes in muscle and brain. Hum. Mol. Genet. 11(17), 1911-1926 (2002). Demonstrated functional disruption of genes upon mutant HTT expression.
    • (2002) Hum. Mol. Genet. , vol.11 , Issue.17 , pp. 1911-1926
    • Luthi-Carter, R.1    Hanson, S.A.2    Strand, A.D.3
  • 12
    • 0037408279 scopus 로고    scopus 로고
    • Transcriptional abnormalities in Huntington disease
    • DOI 10.1016/S0168-9525(03)00074-X
    • Sugars KL, Rubinsztein DC. Transcriptional abnormalities in Huntington disease. Trends Genet. 19(5), 233-238 (2003). (Pubitemid 36511427)
    • (2003) Trends in Genetics , vol.19 , Issue.5 , pp. 233-238
    • Sugars, K.L.1    Rubinsztein, D.C.2
  • 16
    • 84856304670 scopus 로고    scopus 로고
    • A stringent yeast two-hybrid matrix screening approach for protein-protein interaction discovery
    • Worseck JM, Grossmann A, Weimann M, Hegele A, Stelzl U. A stringent yeast two-hybrid matrix screening approach for protein-protein interaction discovery. Methods Mol. Biol. 812, 63-87 (2012).
    • (2012) Methods Mol. Biol. , vol.812 , pp. 63-87
    • Worseck, J.M.1    Grossmann, A.2    Weimann, M.3    Hegele, A.4    Stelzl, U.5
  • 17
    • 84861402086 scopus 로고    scopus 로고
    • Huntingtin protein interactions altered by polyglutamine expansion as determined by quantitative proteomic analysis
    • Ratovitski T, Chighladze E, Arbez N et al. Huntingtin protein interactions altered by polyglutamine expansion as determined by quantitative proteomic analysis. Cell Cycle. 11(10), 2006-2021 (2012).
    • (2012) Cell Cycle. , vol.11 , Issue.10 , pp. 2006-2021
    • Ratovitski, T.1    Chighladze, E.2    Arbez, N.3
  • 18
    • 0033010987 scopus 로고    scopus 로고
    • Recent advances in understanding the pathogenesis of Huntington's disease
    • DOI 10.1016/S0166-2236(99)01415-0, PII S0166223699014150
    • Reddy PH, Williams M, Tagle DA. Recent advances in understanding the pathogenesis of Huntington's disease. Trends NeuroSci. 22(6), 248-255 (1999). (Pubitemid 29259990)
    • (1999) Trends in Neurosciences , vol.22 , Issue.6 , pp. 248-255
    • Reddy, P.H.1    Williams, M.2    Tagle, D.A.3
  • 19
    • 0041963057 scopus 로고    scopus 로고
    • Huntingtin and huntingtin-associated protein 1 influence neuronal calcium signaling mediated by inositol-(1, 4, 5) triphosphate receptor type 1
    • Tang TS, Tu H, Chan EY et al. Huntingtin and huntingtin-associated protein 1 influence neuronal calcium signaling mediated by inositol-(1, 4, 5) triphosphate receptor type 1. Neuron. 39(2), 293-297 (2003).
    • (2003) Neuron. , vol.39 , Issue.2 , pp. 293-297
    • Tang, T.S.1    Tu, H.2    Chan, E.Y.3
  • 20
    • 49349101933 scopus 로고    scopus 로고
    • Huntington's disease: Roles of huntingtin-interacting protein 1 (HIP-1) and its molecular partner HIPPI in the regulation of apoptosis and transcription
    • Bhattacharyya NP, Banerjee M, Majumder P. Huntington's disease: roles of huntingtin-interacting protein 1 (HIP-1) and its molecular partner HIPPI in the regulation of apoptosis and transcription. FEBS J. 275(17), 4271-4279 (2008).
    • (2008) FEBS J. , vol.275 , Issue.17 , pp. 4271-4279
    • Bhattacharyya, N.P.1    Banerjee, M.2    Majumder, P.3
  • 21
    • 34248166420 scopus 로고    scopus 로고
    • Multiple pathways contribute to the pathogenesis of Huntington disease
    • Li S, Li XJ. Multiple pathways contribute to the pathogenesis of Huntington disease. Mol Neurodegener. 1(16), 19 (2006).
    • (2006) Mol Neurodegener. , vol.1 , Issue.16 , pp. 19
    • Li, S.1    Li, X.J.2
  • 24
    • 52049085768 scopus 로고    scopus 로고
    • Pathophysiology of Huntington's disease: From huntingtin functions to potential treatments
    • Roze E, Saudou F, Caboche J. Pathophysiology of Huntington's disease: from huntingtin functions to potential treatments. Curr. Opin. Neurol. 21(4), 497-503 (2008).
    • (2008) Curr. Opin. Neurol. , vol.21 , Issue.4 , pp. 497-503
    • Roze, E.1    Saudou, F.2    Caboche, J.3
  • 25
    • 77957859207 scopus 로고    scopus 로고
    • Symptomatic treatment and management of Huntington's disease: An overview
    • Tyagi SN, Tyagi LK, Shekhar R, Singh M, Kori ML. Symptomatic treatment and management of Huntington's disease: an overview. Global J. Pharmacol. 4(1), 6-12 (2010).
    • (2010) Global J. Pharmacol. , vol.4 , Issue.1 , pp. 6-12
    • Tyagi, S.N.1    Tyagi, L.K.2    Shekhar, R.3    Singh, M.4    Kori, M.L.5
  • 26
    • 79959333019 scopus 로고    scopus 로고
    • Potential drugs and methods for preventing or delaying the progression of Huntington's disease
    • Sari Y. Potential drugs and methods for preventing or delaying the progression of Huntington's disease. Recent Pat. CNS Drug Discov. 6(2), 80-90 (2011).
    • (2011) Recent Pat. CNS Drug Discov. , vol.6 , Issue.2 , pp. 80-90
    • Sari, Y.1
  • 27
    • 30344476118 scopus 로고    scopus 로고
    • Pharmaceutical, cellular and genetic therapies for Huntington's disease
    • DOI 10.1042/CS20050148
    • Handley OJ, Naji JJ, Dunnett SB, Rosser AE. Pharmaceutical, cellular and genetic therapies for Huntington's disease. Clin. Sci. (Lond.). 110(1), 73-88 (2006). (Pubitemid 43059046)
    • (2006) Clinical Science , vol.110 , Issue.1 , pp. 73-88
    • Handley, O.J.1    Naji, J.J.2    Dunnett, S.B.3    Rosser, A.E.4
  • 29
    • 0345490948 scopus 로고    scopus 로고
    • RNA interference in biology and medicine
    • DOI 10.1124/pr.55.4.1
    • Milhavet O, Gary DS, Mattson MP. RNA interference in biology and medicine. Pharmacol. Rev. 55(4), 629-648 (2003). (Pubitemid 37500523)
    • (2003) Pharmacological Reviews , vol.55 , Issue.4 , pp. 629-648
    • Milhavet, O.1    Gary, D.S.2    Mattson, M.P.3
  • 30
    • 4644350944 scopus 로고    scopus 로고
    • Unlocking the potential of the human genome with RNA interference
    • DOI 10.1038/nature02870
    • Hannon GJ, Rossi JJ. Unlocking the potential of the human genome with RNA interference. Nature 431(7006), 371-378 (2004). (Pubitemid 39265678)
    • (2004) Nature , vol.431 , Issue.7006 , pp. 371-378
    • Hannon, G.J.1    Rossi, J.J.2
  • 32
    • 0035942736 scopus 로고    scopus 로고
    • Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
    • Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K, Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 411(6836), 494-497 (2001).
    • (2001) Nature , vol.411 , Issue.6836 , pp. 494-497
    • Elbashir, S.M.1    Harborth, J.2    Lendeckel, W.3    Yalcin, A.4    Weber, K.5    Tuschl, T.6
  • 34
    • 33847036990 scopus 로고    scopus 로고
    • Strategies for silencing human disease using RNA interference
    • Kim DH, Rossi JJ. Strategies for silencing human disease using RNA interference. Nat. Rev. Genet. 8(3), 173-184 (2007).
    • (2007) Nat. Rev. Genet. , vol.8 , Issue.3 , pp. 173-184
    • Kim, D.H.1    Rossi, J.J.2
  • 35
    • 10944254152 scopus 로고    scopus 로고
    • Transcription and processing of human microRNA precursors
    • DOI 10.1016/j.molcel.2004.12.002, PII S1097276504007567
    • Cullen BR. Transcription and processing of human microRNA precursors. Molecular Cell 16(6), 861-865 (2004). (Pubitemid 40018397)
    • (2004) Molecular Cell , vol.16 , Issue.6 , pp. 861-865
    • Cullen, B.R.1
  • 36
    • 0347361541 scopus 로고    scopus 로고
    • Exportin-5 mediates the nuclear export of pre-microRNAs and short hairpin RNAs
    • DOI 10.1101/gad.1158803
    • Yi R, Qin Y, Macara IG, Cullen BR. Exportin-5 mediates the nuclear export of pre-microRNAs and short hairpin RNAs. Genes Dev. 17(24), 3011-3016 (2003). (Pubitemid 38040764)
    • (2003) Genes and Development , vol.17 , Issue.24 , pp. 3011-3016
    • Yi, R.1    Qin, Y.2    Macara, I.G.3    Cullen, B.R.4
  • 37
    • 65749088084 scopus 로고    scopus 로고
    • Sirna vs. shRNA: Similarities and differences
    • Overview of the pros and cons of using siRNA versus shRNA
    • Rao DD, Vorhies JS, Senzer N, Nemunaitis J. siRNA vs. shRNA: similarities and differences. Adv. Drug Deliv. Rev. 61(9), 746-759 (2009). Overview of the pros and cons of using siRNA versus shRNA.
    • (2009) Adv. Drug Deliv. Rev. , vol.61 , Issue.9 , pp. 746-759
    • Rao, D.D.1    Vorhies, J.S.2    Senzer, N.3    Nemunaitis, J.4
  • 38
    • 70350130799 scopus 로고    scopus 로고
    • Comparative assessment of siRNA and shRNA off target effects: What is slowing clinical development
    • Rao DD, Senzer N, Cleary MA, Nemunaitis J. Comparative assessment of siRNA and shRNA off target effects: what is slowing clinical development. Cancer Gene. Ther. 16(11), 807-809 (2009).
    • (2009) Cancer Gene. Ther. , vol.16 , Issue.11 , pp. 807-809
    • Rao, D.D.1    Senzer, N.2    Cleary, M.A.3    Nemunaitis, J.4
  • 40
    • 22344454846 scopus 로고    scopus 로고
    • Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA
    • DOI 10.1038/nbt1081
    • Judge AD, Sood V, Shaw JR, Fang D, McClintock K, MacLachlan I. Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA. Nat. Biotechnol. 23(4), 457-462 (2005). (Pubitemid 41724921)
    • (2005) Nature Biotechnology , vol.23 , Issue.4 , pp. 457-462
    • Judge, A.D.1    Sood, V.2    Shaw, J.R.3    Fang, D.4    McClintock, K.5    MacLachlan, I.6
  • 41
    • 36849087968 scopus 로고    scopus 로고
    • Therapeutic application of RNAi: Is mRNA targeting finally ready for prime time?
    • DOI 10.1172/JCI34129
    • Grimm D, Kay MA. Therapeutic application of RNAi: is mRNA targeting finally ready for prime time? J. Clin. Invest. 117(12), 3633-3641 (2007). (Pubitemid 350224069)
    • (2007) Journal of Clinical Investigation , vol.117 , Issue.12 , pp. 3633-3641
    • Grimm, D.1    Kay, M.A.2
  • 43
    • 77957925036 scopus 로고    scopus 로고
    • A status report on RNAi therapeutics
    • Vaishnaw AK, Gollob J, Gamba-Vitalo C et al. A status report on RNAi therapeutics. Silence 1(1), 14 (2010).
    • (2010) Silence , vol.1 , Issue.1 , pp. 14
    • Vaishnaw, A.K.1    Gollob, J.2    Gamba-Vitalo, C.3
  • 44
    • 34248682403 scopus 로고    scopus 로고
    • RNAi therapeutics: Principles, prospects and challenges
    • DOI 10.1016/j.addr.2007.03.005, PII S0169409X07000166, Opportunities and Challenges for Therapeutic Gene Silencing using RNAi and microRNA Technologies
    • Aagaard L, Rossi JJ. RNAi therapeutics: principles, prospects and challenges. Adv. Drug Del. Rev. 59(2-3), 75-86 (2007). (Pubitemid 46770900)
    • (2007) Advanced Drug Delivery Reviews , vol.59 , Issue.2-3 , pp. 75-86
    • Aagaard, L.1    Rossi, J.J.2
  • 45
    • 77952584927 scopus 로고    scopus 로고
    • RNAi therapeutics for CNS disorders
    • Boudreau RL, Davidson BL. RNAi therapeutics for CNS disorders. Brain Res. 1338, 112-121 (2010).
    • (2010) Brain Res. , vol.1338 , pp. 112-121
    • Boudreau, R.L.1    Davidson, B.L.2
  • 47
    • 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
    • DOI 10.1016/j.ymthe.2005.05.006, PII S1525001605002005
    • Rodriguez-Lebron E, Denovan-Wright EM, Nash K, Lewin AS, Mandel RJ. 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(4), 618-633 (2005). (Pubitemid 41350143)
    • (2005) Molecular Therapy , vol.12 , Issue.4 , pp. 618-633
    • Rodriguez-Lebron, E.1    Denovan-Wright, E.M.2    Nash, K.3    Lewin, A.S.4    Mandel, R.J.5
  • 48
    • 26844494370 scopus 로고    scopus 로고
    • Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA
    • DOI 10.1016/j.neures.2005.06.021, PII S0168010205001926
    • Wang YL, 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. 53(3), 241-249 (2005). (Pubitemid 41463777)
    • (2005) Neuroscience Research , vol.53 , Issue.3 , pp. 241-249
    • Wang, Y.-L.1    Liu, W.2    Wada, E.3    Murata, M.4    Wada, K.5    Kanazawa, I.6
  • 51
    • 34248146697 scopus 로고    scopus 로고
    • High-capacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo
    • DOI 10.1089/hum.2006.160
    • Huang B, Schiefer J, Sass C, Landwehrmeyer GB, Kosinski CM, Kochanek S. High-capacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo. Hum. Gene Ther. 18(4), 303-311 (2007). (Pubitemid 46709342)
    • (2007) Human Gene Therapy , vol.18 , Issue.4 , pp. 303-311
    • Huang, B.1    Schiefer, J.2    Sass, C.3    Landwehrmeyer, G.B.4    Kosinski, C.M.5    Kochanek, S.6
  • 52
    • 42549134402 scopus 로고    scopus 로고
    • AAV vector-mediated RNAi of mutant Huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease
    • DOI 10.1038/mt.2008.50, PII MT200850
    • Franich NR, Fitzsimons HL, Fong DM, Klugmann M, During MJ, Young D. AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease. Mol Ther. 16(5), 947-956 (2008). (Pubitemid 351587106)
    • (2008) Molecular Therapy , vol.16 , Issue.5 , pp. 947-956
    • Franich, N.R.1    Fitzsimons, H.L.2    Fong, D.M.3    Klugmann, M.4    During, M.J.5    Young, D.6
  • 53
    • 1342324908 scopus 로고    scopus 로고
    • Specific Inhibition of Huntington's disease gene expression by siRNA's in cultured cells
    • Liu W, Goto J, Wang YL, Murata M, Wada K, Kanazawa I. Specific Inhibition of Huntington's disease gene expression by siRNA's in cultured cells. Proc. Japan Acad. B 79B (10), 293-298 (2003).
    • (2003) Proc. Japan Acad. B , vol.79 B , Issue.10 , pp. 293-298
    • Liu, W.1    Goto, J.2    Wang, Y.L.3    Murata, M.4    Wada, K.5    Kanazawa, I.6
  • 54
    • 19644375195 scopus 로고    scopus 로고
    • Adenovirus-mediated silencing of huntingtin expression by shRNA
    • DOI 10.1089/hum.2005.16.618
    • Huang B, Kochanek S. Adenovirus-mediated silencing of huntingtin expression by shRNA. Hum. Gen. Ther. 16(5), 618-626 (2005). (Pubitemid 40740929)
    • (2005) Human Gene Therapy , vol.16 , Issue.5 , pp. 618-626
    • Huang, B.1    Kochanek, S.2
  • 55
    • 84857721326 scopus 로고    scopus 로고
    • An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases
    • Fiszer A, Olejniczak M, Switonski PM et al. An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases. BMC Mol. Biol. 13 (6) (2012).
    • BMC Mol. Biol. , vol.13 , Issue.6 , pp. 2012
    • Fiszer, A.1    Olejniczak, M.2    Switonski, P.M.3
  • 56
    • 78649379362 scopus 로고    scopus 로고
    • Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat
    • Gagnon KT, Pendergraff HM, Deleavey GF et al. Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat. Biochemistry 49(47), 10166-10178 (2010).
    • (2010) Biochemistry , vol.49 , Issue.47 , pp. 10166-10178
    • Gagnon, K.T.1    Pendergraff, H.M.2    Deleavey, G.F.3
  • 57
    • 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. sms 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. sms in the Huntington disease gene/allele-specific silencing of mutant huntingtin. Mol. Ther. 19(12), 2178-2185 (2011).
    • (2011) Mol. Ther. , vol.19 , Issue.12 , pp. 2178-2185
    • Carroll, J.B.1    Warby, S.C.2    Southwell, A.L.3
  • 58
    • 36849094017 scopus 로고    scopus 로고
    • Chemical modification: The key to clinical application of RNA interference?
    • DOI 10.1172/JCI33483
    • Corey DR. Chemical modification: the key to clinical application of RNA interference? J. Clin. Invest. 117(12), 3615-3622 (2007). Principles for chemical modification of siRNA. (Pubitemid 350224067)
    • (2007) Journal of Clinical Investigation , vol.117 , Issue.12 , pp. 3615-3622
    • Corey, D.R.1
  • 59
    • 82955199935 scopus 로고    scopus 로고
    • Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease
    • McBride JL, Pitzer MR, Boudreau RL et al. Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease. Mol. Ther. 19(12), 2152-2162 (2011).
    • (2011) Mol. Ther. , vol.19 , Issue.12 , pp. 2152-2162
    • McBride, J.L.1    Pitzer, M.R.2    Boudreau, R.L.3
  • 60
    • 84860192454 scopus 로고    scopus 로고
    • Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum
    • Grondin R, Kaytor MD, Ai Y et al. Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum. Brain 135(Pt 4), 1197-1209 (2012).
    • (2012) Brain , vol.135 , Issue.4 PART , pp. 1197-1209
    • Grondin, R.1    Kaytor, M.D.2    Ai, Y.3
  • 61
    • 23044463005 scopus 로고    scopus 로고
    • RNA interference in biology and disease
    • Sledz CA, Williams BR. RNA interference in biology and disease. Gene Ther. 106(3), 787-784 (2005).
    • (2005) Gene Ther. , vol.106 , Issue.3 , pp. 787-784
    • Sledz, C.A.1    Williams, B.R.2
  • 62
    • 11844302173 scopus 로고    scopus 로고
    • SiRNA therapeutics: Big potential from small RNAs
    • DOI 10.1038/sj.gt.3302356
    • Ryther RC, Flynt AS, Phillips JA 3rd, Patton JG. siRNA therapeutics: big potential from small RNAs. Gene Ther. 12(1), 5-11 (2005). (Pubitemid 40090039)
    • (2005) Gene Therapy , vol.12 , Issue.1 , pp. 5-11
    • Ryther, R.C.C.1    Flynt, A.S.2    Phillips III, J.A.3    Patton, J.G.4
  • 63
    • 33748069824 scopus 로고    scopus 로고
    • SiRNA and isRNA: two edges of one sword
    • DOI 10.1016/j.ymthe.2006.06.001, PII S1525001606002176
    • Schlee M, Hornung V, Hartmann G. siRNA and isRNA: two edges of one sword. Mol. Ther. 14(4), 463-470 (2006). (Pubitemid 44296098)
    • (2006) Molecular Therapy , vol.14 , Issue.4 , pp. 463-470
    • Schlee, M.1    Hornung, V.2    Hartmann, G.3
  • 64
    • 58749104364 scopus 로고    scopus 로고
    • The promises and pitfalls of RNA-interference-based therapeutics
    • Castanotto D, Rossi JJ. The promises and pitfalls of RNA-interference- based therapeutics. Nature 457(7228), 426-433 (2009).
    • (2009) Nature , vol.457 , Issue.7228 , pp. 426-433
    • Castanotto, D.1    Rossi, J.J.2
  • 66
    • 33646188259 scopus 로고    scopus 로고
    • 3' UTR seed matches, but not overall identity, are associated with RNAi off-targets
    • Birmingham A, Anderson EM, Reynolds A et al. 3' UTR seed matches, but not overall identity, are associated with RNAi off-targets. Nat. Methods 3(3), 199-204 (2006).
    • (2006) Nat. Methods , vol.3 , Issue.3 , pp. 199-204
    • Birmingham, A.1    Anderson, E.M.2    Reynolds, A.3
  • 67
    • 15044358407 scopus 로고    scopus 로고
    • A systematic analysis of the silencing effects of an active siRNA at all single-nucleotide mismatched target sites
    • DOI 10.1093/nar/gki312
    • Du Q, Thonberg H, Wang J, Wahlestedt C, Liang Z. A systematic analysis of the silencing effects of an active siRNA at all single-nucleotide mismatched target sites. Nucleic Acids Res. 33(5), 1671-1677 (2007). (Pubitemid 41418395)
    • (2005) Nucleic Acids Research , vol.33 , Issue.5 , pp. 1671-1677
    • Du, Q.1    Thonberg, H.2    Wang, J.3    Wahlestedt, C.4    Liang, Z.5
  • 68
    • 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
    • Principles for designing siRNA for minimal off-target effects - includes microarray analyses
    • Boudreau RL, Spengler RM, Davidson BL. Rational design of therapeutic siRNAs: minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease. Mol. Ther. 19(12), 2169-2177 (2011). Principles for designing siRNA for minimal off-target effects - includes microarray analyses.
    • (2011) Mol. Ther. , vol.19 , Issue.12 , pp. 2169-2177
    • Boudreau, R.L.1    Spengler, R.M.2    Davidson, B.L.3
  • 70
    • 67249122712 scopus 로고    scopus 로고
    • Biological barriers to therapy with antisense and siRNA oligonucleotides
    • Juliano R, Bauman J, Kang H, Ming X. Biological barriers to therapy with antisense and siRNA oligonucleotides. Mol. Pharm. 6(3), 686-695 (2009).
    • (2009) Mol. Pharm. , vol.6 , Issue.3 , pp. 686-695
    • Juliano, R.1    Bauman, J.2    Kang, H.3    Ming, X.4
  • 72
    • 24344500241 scopus 로고    scopus 로고
    • Sequence, chemical, and structural variation of small interfering RNAs and short hairpin RNAs and the effect on mammalian gene silencing
    • Harborth J, Elbashir SM, Vandenburgh K et al. Sequence, chemical, and structural variation of small interfering RNAs and short hairpin RNAs and the effect on mammalian gene silencing. Antisense Nucleic Acid Drug Dev. 13(2), 83-105 (2003).
    • (2003) Antisense Nucleic Acid Drug Dev. , vol.13 , Issue.2 , pp. 83-105
    • Harborth, J.1    Elbashir, S.M.2    Vandenburgh, K.3
  • 73
    • 77956667224 scopus 로고    scopus 로고
    • Selective gene silencing by viral delivery of short hairpin RNA
    • Sliva K, Schnierle BS. Selective gene silencing by viral delivery of short hairpin RNA. Virol. J. 7, 248-259 (2010).
    • (2010) Virol. J. , vol.7 , pp. 248-259
    • Sliva, K.1    Schnierle, B.S.2
  • 74
    • 33645155231 scopus 로고    scopus 로고
    • Reconstituted influenza virus envelopes as an efficient carrier system for cellular delivery of small-interfering RNAs
    • de Jonge J, Holtrop M W J, Huckriede A. Reconstituted influenza virus envelopes as an efficient carrier system for cellular delivery of small-interfering RNAs. Gene Ther. 13(5), 400-411 (2006).
    • (2006) Gene Ther. , vol.13 , Issue.5 , pp. 400-411
    • De Jonge, J.1    Holtrop, M.W.J.2    Huckriede, A.3
  • 75
    • 0038745599 scopus 로고    scopus 로고
    • Progress and problems with the use of viral vectors for gene therapy
    • DOI 10.1038/nrg1066
    • Thomas CE, Ehrhardt A, Kay MA. Progress and problems with the use of viral vectors for gene therapy. Nat. Rev. Genet. 4(5), 346-358 (2003). (Pubitemid 36538359)
    • (2003) Nature Reviews Genetics , vol.4 , Issue.5 , pp. 346-358
    • Thomas, C.E.1    Ehrhardt, A.2    Kay, M.A.3
  • 76
    • 68549087056 scopus 로고    scopus 로고
    • Viral vectors: From virology to transgene expression
    • Bouard D, Alazard-Dany D, Cosset FL. Viral vectors: from virology to transgene expression. Br. J. Pharmacol. 157(2), 153-165 (2009).
    • (2009) Br. J. Pharmacol. , vol.157 , Issue.2 , pp. 153-165
    • Bouard, D.1    Alazard-Dany, D.2    Cosset, F.L.3
  • 77
    • 78649396316 scopus 로고    scopus 로고
    • Baculovirus vector-mediated transfer of NIS gene into colon tumor cells for radionuclide therapy
    • Yin HY, Zhou X, Wu HF, Li B, Zhang YF. Baculovirus vector-mediated transfer of NIS gene into colon tumor cells for radionuclide therapy. World J. Gastroenterol. 16(42), 5367-5374 (2010).
    • (2010) World J. Gastroenterol. , vol.16 , Issue.42 , pp. 5367-5374
    • Yin, H.Y.1    Zhou, X.2    Wu, H.F.3    Li, B.4    Zhang, Y.F.5
  • 78
    • 34548818764 scopus 로고    scopus 로고
    • Cationic lipids and polymers mediated vectors for delivery of siRNA
    • DOI 10.1016/j.jconrel.2007.07.016, PII S0168365907003914
    • Zhang S, Zhao B, Jiang H, Wang B, Ma B. Cationic lipids and polymers mediated vectors for delivery of siRNA. J. Control. Release 123(1), 1-10 (2007). (Pubitemid 47444388)
    • (2007) Journal of Controlled Release , vol.123 , Issue.1 , pp. 1-10
    • Zhang, S.1    Zhao, B.2    Jiang, H.3    Wang, B.4    Ma, B.5
  • 79
    • 36849044732 scopus 로고    scopus 로고
    • Nonviral delivery of synthetic siRNAs in vivo
    • DOI 10.1172/JCI33494
    • Akhtar S, Benter IF. Nonviral delivery of synthetic siRNAs in vivo. J. Clin. Invest. 117(12), 3623-3632 (2007). (Pubitemid 350224068)
    • (2007) Journal of Clinical Investigation , vol.117 , Issue.12 , pp. 3623-3632
    • Akhtar, S.1    Benter, I.F.2
  • 81
    • 80155147118 scopus 로고    scopus 로고
    • Research progress on siRNA delivery with non-viral carriers
    • Gao Y, Liu XL, Li XR. Research progress on siRNA delivery with non-viral carriers. Int. J. Nanomed. 6, 1017-1025 (2011).
    • (2011) Int. J. Nanomed. , vol.6 , pp. 1017-1025
    • Gao, Y.1    Liu, X.L.2    Li, X.R.3
  • 82
    • 84861595819 scopus 로고    scopus 로고
    • Efficient and safe delivery of siRNA using anionic lipids: Formulation optimization studies
    • Kapoor M, Burgess DJ. Efficient and safe delivery of siRNA using anionic lipids: formulation optimization studies. Int. J. Pharm. 432(1-2), 80-90 (2012).
    • (2012) Int. J. Pharm. , vol.432 , Issue.1-2 , pp. 80-90
    • Kapoor, M.1    Burgess, D.J.2
  • 83
    • 70449089802 scopus 로고    scopus 로고
    • Strategies for targeted nonviral delivery of siRNAs in vivo
    • Kim SS, Garg H, Joshi A, Manjunath N. Strategies for targeted nonviral delivery of siRNAs in vivo. Trends Mol. Med. 15(11), 491-500 (2009).
    • (2009) Trends Mol. Med. , vol.15 , Issue.11 , pp. 491-500
    • Kim, S.S.1    Garg, H.2    Joshi, A.3    Manjunath, N.4
  • 84
    • 33646144003 scopus 로고    scopus 로고
    • Sirna-containing liposomes modified with polyarginine effectively silence the targeted gene
    • Zhang C, Tang N, Liu X, Liang W, Xu W, Torchilin VP. siRNA-containing liposomes modified with polyarginine effectively silence the targeted gene. J. Cont. Rel. 112(2), 229-239 (2006).
    • (2006) J. Cont. Rel. , vol.112 , Issue.2 , pp. 229-239
    • Zhang, C.1    Tang, N.2    Liu, X.3    Liang, W.4    Xu, W.5    Torchilin, V.P.6
  • 85
    • 14544298301 scopus 로고    scopus 로고
    • Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle
    • Schiffelers RM, Ansari A, Xu J et al. Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle. Nucleic Acid Res. 32(19), e149 (2004).
    • (2004) Nucleic Acid Res. , vol.32 , Issue.19
    • Schiffelers, R.M.1    Ansari, A.2    Xu, J.3
  • 86
    • 79952939225 scopus 로고    scopus 로고
    • Recent advances of siRNA delivery by nanoparticles
    • Yuan X, Naguib S, Wu Z. Recent advances of siRNA delivery by nanoparticles. Expert Opin. Drug Deliv. 8(4), 521-536 (2011).
    • (2011) Expert Opin. Drug Deliv. , vol.8 , Issue.4 , pp. 521-536
    • Yuan, X.1    Naguib, S.2    Wu, Z.3
  • 87
    • 84864631153 scopus 로고    scopus 로고
    • Cell-penetrating peptides: Classes, origin, and current landscape
    • Milletti F. Cell-penetrating peptides: classes, origin, and current landscape. Drug Disc. Today. 17(15-16), 850-860 (2012).
    • (2012) Drug Disc. Today. , vol.17 , Issue.15-16 , pp. 850-860
    • Milletti, F.1
  • 88
    • 79959365312 scopus 로고    scopus 로고
    • Mechanisms of cellular uptake of cell-penetrating peptides
    • doi:10.1155/2011/414729 2011 Epub ahead of print
    • Madani F, Lindberg S, Langel U, Futaki S, Gräslund A. Mechanisms of cellular uptake of cell-penetrating peptides. J. Biophys. doi:10.1155/2011/ 414729 (2011) (Epub ahead of print).
    • J. Biophys.
    • Madani, F.1    Lindberg, S.2    Langel, U.3    Futaki, S.4    Gräslund, A.5
  • 89
    • 58149091387 scopus 로고    scopus 로고
    • Distinct uptake routes of cell-penetrating peptide conjugates
    • Lundin P, Johansson H, Guterstam P et al. Distinct uptake routes of cell-penetrating peptide conjugates. Bioconj. Chem. 19(12), 2535-2542 (2008).
    • (2008) Bioconj. Chem. , vol.19 , Issue.12 , pp. 2535-2542
    • Lundin, P.1    Johansson, H.2    Guterstam, P.3
  • 90
    • 84862734920 scopus 로고    scopus 로고
    • Cell-penetrating peptides for the delivery of nucleic acids
    • Lehto T, Kurrikoff K, Langel U. Cell-penetrating peptides for the delivery of nucleic acids. Expert Opin. Drug Deliv. 9(7), 823-836 (2012).
    • (2012) Expert Opin. Drug Deliv. , vol.9 , Issue.7 , pp. 823-836
    • Lehto, T.1    Kurrikoff, K.2    Langel, U.3
  • 91
    • 84856917252 scopus 로고    scopus 로고
    • Comparison of cationic and amphipathic cell penetrating peptides for siRNA delivery and efficacy
    • Mo RH, Zaro JL, Shen WC. Comparison of cationic and amphipathic cell penetrating peptides for siRNA delivery and efficacy. Mol. Pharm. 9(2), 299-309 (2012).
    • (2012) Mol. Pharm. , vol.9 , Issue.2 , pp. 299-309
    • Mo, R.H.1    Zaro, J.L.2    Shen, W.C.3
  • 92
    • 33646450620 scopus 로고    scopus 로고
    • Physiology of the blood-brain barrier and its consequences for drug transport to the brain
    • DOI 10.1016/j.ics.2005.02.008, PII S0531513105000798, Drug Transport(ers) and the Diseased Brain
    • Abbott NJ. Physiology of the blood-brain barrier and its consequences for drug transport to the brain. Int. Congress Series 1277, 3-18 (2005). (Pubitemid 43693341)
    • (2005) International Congress Series , vol.1277 , pp. 3-18
    • Abbott, N.J.1
  • 93
    • 80053496670 scopus 로고    scopus 로고
    • Blood-brain barrier P450 enzymes and multidrug transporters in drug resistance: A synergistic role in neurological diseases
    • Ghosh C, Puvenna V, Gonzalez-Martinez J, Janigro D, Marchi N. Blood-brain barrier P450 enzymes and multidrug transporters in drug resistance: a synergistic role in neurological diseases. Curr. Drug Metab. 12(8), 742-749 (2011).
    • (2011) Curr. Drug Metab. , vol.12 , Issue.8 , pp. 742-749
    • Ghosh, C.1    Puvenna, V.2    Gonzalez-Martinez, J.3    Janigro, D.4    Marchi, N.5
  • 94
    • 33646825160 scopus 로고    scopus 로고
    • Drug delivery across the blodd-brain barrier: Why is it difficult? How to measure and improve it?
    • DOI 10.1517/17425247.3.3.419
    • Su Y, Sinko PJ. Drug delivery across the blood-brain barrier: why is it difficult? How to measure and improve it? Expert Opin. Drug Deliv. 3(3), 419-435 (2006). (Pubitemid 43772716)
    • (2006) Expert Opinion on Drug Delivery , vol.3 , Issue.3 , pp. 419-435
    • Su, Y.1    Sinko, P.J.2
  • 95
    • 34248146697 scopus 로고    scopus 로고
    • High-capacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo
    • DOI 10.1089/hum.2006.160
    • Huang B, Schiefer J, Sass C, Landwehrmeyer GB, Kosinski CM, Kochanek S. High-capacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo. Hum. Gene Ther. Apr. 18(4), 303-311 (2007). (Pubitemid 46709342)
    • (2007) Human Gene Therapy , vol.18 , Issue.4 , pp. 303-311
    • Huang, B.1    Schiefer, J.2    Sass, C.3    Landwehrmeyer, G.B.4    Kosinski, C.M.5    Kochanek, S.6
  • 97
    • 77956205950 scopus 로고    scopus 로고
    • Liposome-siRNA-peptide complexes cross the blood-brain barrier and significantly decrease PrPC on neuronal cells and PrPRES ininfected cell cultures
    • Pulford B, Reim N, Bell A et al. Liposome-siRNA-peptide complexes cross the blood-brain barrier and significantly decrease PrPC on neuronal cells and PrPRES ininfected cell cultures. PLoS ONE 5(6), e11085 (2010).
    • (2010) PLoS ONE , vol.5 , Issue.6
    • Pulford, B.1    Reim, N.2    Bell, A.3
  • 98
    • 84862201678 scopus 로고    scopus 로고
    • Brain-targeting gene delivery using a rabies virus glycoprotein peptide modulated hollow liposome: Bio-behavioral study
    • Tao Y, Han J, Huanyu D. Brain-targeting gene delivery using a rabies virus glycoprotein peptide modulated hollow liposome: bio-behavioral study. J. Mat. Chem. 22, 11808-11815 (2012).
    • (2012) J. Mat. Chem. , vol.22 , pp. 11808-11815
    • Tao, Y.1    Han, J.2    Huanyu, D.3
  • 100
    • 79953858598 scopus 로고    scopus 로고
    • Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes
    • Alvarez-Erviti L, Seow Y, Yin H, Betts C, Lakhal S, Wood MJ. Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes. Nat. Biotech. 29(4), 341-345 (2011).
    • (2011) Nat. Biotech. , vol.29 , Issue.4 , pp. 341-345
    • Alvarez-Erviti, L.1    Seow, Y.2    Yin, H.3    Betts, C.4    Lakhal, S.5    Wood, M.J.6
  • 101
    • 67649317011 scopus 로고    scopus 로고
    • Delivery of small-interfering RNA (siRNA) to the brain
    • Mathupala SP. Delivery of small-interfering RNA (siRNA) to the brain. Expert Opin. Ther. Pat. 19(2), 137-140 (2009).
    • (2009) Expert Opin. Ther. Pat. , vol.19 , Issue.2 , pp. 137-140
    • Mathupala, S.P.1
  • 102
    • 77949833508 scopus 로고    scopus 로고
    • Intranasal delivery to the central nervous system: Mechanisms and experimental considerations
    • Dhuria SV, Hanson LR, Frey WH 2nd. Intranasal delivery to the central nervous system: mechanisms and experimental considerations. J. Pharm. Sci. 99(4), 1654-1673 (2010).
    • (2010) J. Pharm. Sci. , vol.99 , Issue.4 , pp. 1654-1673
    • Dhuria, S.V.1    Hanson, L.R.2    Frey II, W.H.3
  • 103
    • 68549104413 scopus 로고    scopus 로고
    • Progress and challenges in RNA interference therapy for Huntington disease
    • Harper SQ. Progress and challenges in RNA interference therapy for Huntington disease. Neurol. Rev. 66(8), 933-938 (2009).
    • (2009) Neurol. Rev. , vol.66 , Issue.8 , pp. 933-938
    • Harper, S.Q.1


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