메뉴 건너뛰기




Volumn 195, Issue , 2014, Pages 49-54

Current issues of RNAi therapeutics delivery and development

(1)  Haussecker, D a  

a NONE   (Germany)

Author keywords

Delivery; RNAi therapeutics; Synthetic RNAi triggers

Indexed keywords

DELIVERY; RNAI THERAPEUTICS; SYNTHETIC RNAI TRIGGERS;

EID: 84908564426     PISSN: 01683659     EISSN: 18734995     Source Type: Journal    
DOI: 10.1016/j.jconrel.2014.07.056     Document Type: Article
Times cited : (170)

References (50)
  • 1
    • 44349192034 scopus 로고    scopus 로고
    • The business of RNAi Therapeutics
    • D. Haussecker The business of RNAi Therapeutics Hum. Gene Ther. 19 2008 451
    • (2008) Hum. Gene Ther. , vol.19 , pp. 451
    • Haussecker, D.1
  • 2
    • 76649098887 scopus 로고    scopus 로고
    • Lipid-like materials for low-dose, in vivo gene silencing
    • K.T. Love Lipid-like materials for low-dose, in vivo gene silencing Proc. Natl. Acad. Sci. 107 2010 1864
    • (2010) Proc. Natl. Acad. Sci. , vol.107 , pp. 1864
    • Love, K.T.1
  • 3
    • 58849112575 scopus 로고    scopus 로고
    • Biogenesis of small RNAs in animals
    • V.N. Kim Biogenesis of small RNAs in animals Nat. Rev. Mol. Cell Biol. 10 2009 126
    • (2009) Nat. Rev. Mol. Cell Biol. , vol.10 , pp. 126
    • Kim, V.N.1
  • 4
    • 79953144826 scopus 로고    scopus 로고
    • Improved specificity of gene silencing by siRNAs containing unlocked nucleobase analogs
    • N. Vaish Improved specificity of gene silencing by siRNAs containing unlocked nucleobase analogs Nucleic Acids Res. 39 2011 1823
    • (2011) Nucleic Acids Res. , vol.39 , pp. 1823
    • Vaish, N.1
  • 5
    • 84865682650 scopus 로고    scopus 로고
    • Single-stranded siRNAs activate RNAi in animals
    • W.F. Lima Single-stranded siRNAs activate RNAi in animals Cell 150 2012 883
    • (2012) Cell , vol.150 , pp. 883
    • Lima, W.F.1
  • 6
    • 0038606999 scopus 로고    scopus 로고
    • Structural variations and stabilizing modifications of synthetic siRNAs in mammalian cells
    • F. Czauderna Structural variations and stabilizing modifications of synthetic siRNAs in mammalian cells Nucleic Acids Res. 31 2003 2705
    • (2003) Nucleic Acids Res. , vol.31 , pp. 2705
    • Czauderna, F.1
  • 7
    • 23444445660 scopus 로고    scopus 로고
    • Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs
    • D.V. Morrissey Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs Nat. Biotechnol. 23 2005 1002
    • (2005) Nat. Biotechnol. , vol.23 , pp. 1002
    • Morrissey, D.V.1
  • 8
    • 38649099969 scopus 로고    scopus 로고
    • Strand-specific 5′-O-methylation of siRNA duplexes controls guide strand selection and targeting specificity
    • P.Y. Chen Strand-specific 5′-O-methylation of siRNA duplexes controls guide strand selection and targeting specificity RNA 14 2008 263
    • (2008) RNA , vol.14 , pp. 263
    • Chen, P.Y.1
  • 9
    • 84890082930 scopus 로고    scopus 로고
    • Novel hydrophobically modified asymmetric RNAi compounds (sd-rxRNA) demonstrate robust efficacy in the eye
    • M. Byrne Novel hydrophobically modified asymmetric RNAi compounds (sd-rxRNA) demonstrate robust efficacy in the eye J. Ocul. Pharmacol. Ther. 29 2013 855
    • (2013) J. Ocul. Pharmacol. Ther. , vol.29 , pp. 855
    • Byrne, M.1
  • 10
    • 2642556483 scopus 로고    scopus 로고
    • Oral bioavailability and multiple dose tolerability of an antisense oligonucleotide tablet formulated with sodium caprate
    • A.A. Raoof Oral bioavailability and multiple dose tolerability of an antisense oligonucleotide tablet formulated with sodium caprate J. Pharm. Sci. 93 2004 1431
    • (2004) J. Pharm. Sci. , vol.93 , pp. 1431
    • Raoof, A.A.1
  • 11
    • 84878887964 scopus 로고    scopus 로고
    • Protection and systemic translocation of siRNA following oral administration of chitosan/siRNA nanoparticles
    • B. Ballarin-Gonzalez Protection and systemic translocation of siRNA following oral administration of chitosan/siRNA nanoparticles Mol. Ther. Nucleic Acids 2 2013 e76
    • (2013) Mol. Ther. Nucleic Acids , vol.2 , pp. 76
    • Ballarin-Gonzalez, B.1
  • 12
    • 65949106130 scopus 로고    scopus 로고
    • Orally delivered siRNA targeting macrophage Map4k4 suppresses systemic inflammation
    • M. Aouadi Orally delivered siRNA targeting macrophage Map4k4 suppresses systemic inflammation Nature 458 2009 1180
    • (2009) Nature , vol.458 , pp. 1180
    • Aouadi, M.1
  • 13
    • 84908589428 scopus 로고    scopus 로고
    • http://www.alnylam.com/web/wp-content/uploads/2013/10/ALNY-ConjugateUpdate-OTS2013.pdf
  • 14
    • 9444279605 scopus 로고    scopus 로고
    • Ligand binding and kinetics of folate receptor recycling in vivo: Impact on receptor-mediated drug delivery
    • C.M. Paulos Ligand binding and kinetics of folate receptor recycling in vivo: impact on receptor-mediated drug delivery Mol. Pharmacol. 66 2004 1406
    • (2004) Mol. Pharmacol. , vol.66 , pp. 1406
    • Paulos, C.M.1
  • 15
    • 84908589427 scopus 로고    scopus 로고
    • http://www.alnylam.com/web/wp-content/uploads/2013/09/ALN-TTRsc-PhI-HFSA-Poster-Sep2013.pdf
  • 16
    • 84877026189 scopus 로고    scopus 로고
    • Hepatocyte-targeted RNAi therapeutics for the treatment of chronic hepatitis B virus infection
    • C.I. Wooddell Hepatocyte-targeted RNAi therapeutics for the treatment of chronic hepatitis B virus infection Mol. Ther. 21 2013 973
    • (2013) Mol. Ther. , vol.21 , pp. 973
    • Wooddell, C.I.1
  • 17
    • 84870570270 scopus 로고    scopus 로고
    • Co-injection of a targeted, reversibly masked endosomolytic polymer dramatically improves the efficacy of cholesterol-conjugated small interfering RNAs in vivo
    • S.C. Wong Co-injection of a targeted, reversibly masked endosomolytic polymer dramatically improves the efficacy of cholesterol-conjugated small interfering RNAs in vivo Nucleic Acid Ther. 22 2012 380
    • (2012) Nucleic Acid Ther. , vol.22 , pp. 380
    • Wong, S.C.1
  • 18
    • 84908589426 scopus 로고    scopus 로고
    • http://files.shareholder.com/downloads/ABEA-50QJTB/2628241206x0x695629/1af9ba39-fa32-4d8c-b45a-971ed117f1b8/Webinar-Slides4Web.pdf
  • 19
    • 84883118140 scopus 로고    scopus 로고
    • Safety and efficacy of RNAi therapy for transthyretin amyloidosis
    • T. Coelho Safety and efficacy of RNAi therapy for transthyretin amyloidosis N. Engl. J. Med. 369 2013 819
    • (2013) N. Engl. J. Med. , vol.369 , pp. 819
    • Coelho, T.1
  • 20
    • 84908589425 scopus 로고    scopus 로고
    • http://www.alnylam.com/web/wp-content/uploads/2013/11/ALNY-ISFAP-ALN-TTRprogram-Nov2013.pdf
  • 21
    • 84877120847 scopus 로고    scopus 로고
    • First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement
    • J. Tabernero First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement Cancer Discov. 3 2013 406
    • (2013) Cancer Discov. , vol.3 , pp. 406
    • Tabernero, J.1
  • 22
    • 84881101924 scopus 로고    scopus 로고
    • A human in vitro whole blood assay to predict the systemic cytokine response to therapeutic oligonucleotide siRNA
    • C. Coch A human in vitro whole blood assay to predict the systemic cytokine response to therapeutic oligonucleotide siRNA PLoS One 8 2013 e71057
    • (2013) PLoS One , vol.8 , pp. 71057
    • Coch, C.1
  • 23
    • 33645235714 scopus 로고    scopus 로고
    • Diffusible-PEG-lipid stabilized plasmid lipid particles
    • I. MacLachlan, and P. Cullis Diffusible-PEG-lipid stabilized plasmid lipid particles Adv. Genet. 53 2009 157
    • (2009) Adv. Genet. , vol.53 , pp. 157
    • Maclachlan, I.1    Cullis, P.2
  • 24
    • 68049136123 scopus 로고    scopus 로고
    • SiRNA targeted to p53 attenuates ischemic and cisplatin-induced acute kidney injury
    • B.A. Molitoris siRNA targeted to p53 attenuates ischemic and cisplatin-induced acute kidney injury J. Am. Soc. Nephrol. 20 2008 1754
    • (2008) J. Am. Soc. Nephrol. , vol.20 , pp. 1754
    • Molitoris, B.A.1
  • 25
    • 75649119274 scopus 로고    scopus 로고
    • Solid polymeric microparticles enhance the delivery of siRNA to macrophages in vivo
    • S. Lee Solid polymeric microparticles enhance the delivery of siRNA to macrophages in vivo Nucleic Acids Res. 37 2009 e145
    • (2009) Nucleic Acids Res. , vol.37 , pp. 145
    • Lee, S.1
  • 26
    • 84864995694 scopus 로고    scopus 로고
    • Systemic RNAi-mediated gene silencing in nonhuman primate and rodent myeloid cells
    • T.I. Novobrantseva Systemic RNAi-mediated gene silencing in nonhuman primate and rodent myeloid cells Mol. Ther. Nucleic Acids 1 2012 e4
    • (2012) Mol. Ther. Nucleic Acids , vol.1 , pp. 4
    • Novobrantseva, T.I.1
  • 27
    • 84875801524 scopus 로고    scopus 로고
    • A daunting task: Manipulating leukocyte function with RNAi
    • D. Peer A daunting task: manipulating leukocyte function with RNAi Immunol. Rev. 253 2013 185
    • (2013) Immunol. Rev. , vol.253 , pp. 185
    • Peer, D.1
  • 28
    • 57149086661 scopus 로고    scopus 로고
    • Atu027, a liposomal small interfering RNA formulation targeting protein kinase N3, inhibits cancer progression
    • M. Aleku Atu027, a liposomal small interfering RNA formulation targeting protein kinase N3, inhibits cancer progression Cancer Res. 68 2008 9788
    • (2008) Cancer Res. , vol.68 , pp. 9788
    • Aleku, M.1
  • 29
    • 84897570422 scopus 로고    scopus 로고
    • Delivery of therapeutic siRNA to the lung endothelium via novel lipoplex formulation DACC
    • V. Fehring Delivery of therapeutic siRNA to the lung endothelium via novel lipoplex formulation DACC Mol. Ther. 22 2014 811
    • (2014) Mol. Ther. , vol.22 , pp. 811
    • Fehring, V.1
  • 30
    • 76249087751 scopus 로고    scopus 로고
    • Pharmacokinetics and brain uptake of a genetically engineered bifunctional fusion antibody targeting the mouse transferrin receptor
    • R.J. Boado Pharmacokinetics and brain uptake of a genetically engineered bifunctional fusion antibody targeting the mouse transferrin receptor Mol. Pharm. 7 2010 237
    • (2010) Mol. Pharm. , vol.7 , pp. 237
    • Boado, R.J.1
  • 31
    • 84891804917 scopus 로고    scopus 로고
    • Increased brain penetration and potency of a therapeutic antibody using a monovalent molecular shuttle
    • J. Niewoehner Increased brain penetration and potency of a therapeutic antibody using a monovalent molecular shuttle Neuron 81 2014 49
    • (2014) Neuron , vol.81 , pp. 49
    • Niewoehner, J.1
  • 32
    • 84863721493 scopus 로고    scopus 로고
    • Tumor vasculature is a key determinant for the efficacy of nanoparticle-mediated siRNA delivery
    • L. Li Tumor vasculature is a key determinant for the efficacy of nanoparticle-mediated siRNA delivery Gene Ther. 19 2012 775
    • (2012) Gene Ther. , vol.19 , pp. 775
    • Li, L.1
  • 33
    • 70349482671 scopus 로고    scopus 로고
    • C-end rule peptides mediate neuropilin-1-dependent cell, vascular, and tissue penetration
    • T. Teesalu C-end rule peptides mediate neuropilin-1-dependent cell, vascular, and tissue penetration Proc. Natl. Acad. Sci. U. S. A. 106 2009 16157
    • (2009) Proc. Natl. Acad. Sci. U. S. A. , vol.106 , pp. 16157
    • Teesalu, T.1
  • 34
    • 84864670257 scopus 로고    scopus 로고
    • Size-dependent localization and penetration of ultrasmall gold nanoparticles in cancer cells, multicellular spheroids, and tumors in vivo
    • K. Huang Size-dependent localization and penetration of ultrasmall gold nanoparticles in cancer cells, multicellular spheroids, and tumors in vivo ACS Nano 6 2012 4483
    • (2012) ACS Nano , vol.6 , pp. 4483
    • Huang, K.1
  • 35
    • 34248680739 scopus 로고    scopus 로고
    • Exogenous siRNA delivery using peptide transduction domains/cell penetrating peptides
    • B.R. Meade Exogenous siRNA delivery using peptide transduction domains/cell penetrating peptides Adv. Drug Deliv. Rev. 59 2007 134
    • (2007) Adv. Drug Deliv. Rev. , vol.59 , pp. 134
    • Meade, B.R.1
  • 36
    • 34548735210 scopus 로고    scopus 로고
    • Dynamic PolyConjugates for targeted in vivo delivery of siRNA to hepatocytes
    • D.B. Rozema Dynamic PolyConjugates for targeted in vivo delivery of siRNA to hepatocytes Proc. Natl. Acad. Sci. U. S. A. 104 2007 12982
    • (2007) Proc. Natl. Acad. Sci. U. S. A. , vol.104 , pp. 12982
    • Rozema, D.B.1
  • 37
    • 77954243341 scopus 로고    scopus 로고
    • Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanism
    • A. Akinc Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanism Mol. Ther. 18 2010 1357
    • (2010) Mol. Ther. , vol.18 , pp. 1357
    • Akinc, A.1
  • 38
    • 84880274434 scopus 로고    scopus 로고
    • Efficiency of siRNA delivery by lipid nanoparticles is limited by endocytic recycling
    • G. Sahav Efficiency of siRNA delivery by lipid nanoparticles is limited by endocytic recycling Nat. Biotechnol. 31 2013 653
    • (2013) Nat. Biotechnol. , vol.31 , pp. 653
    • Sahav, G.1
  • 39
    • 0033984862 scopus 로고    scopus 로고
    • Phosphorothioate oligodeoxynucleotides distribute similarly in class A scavenger receptor knockout and wild-type mice
    • M. Butler Phosphorothioate oligodeoxynucleotides distribute similarly in class A scavenger receptor knockout and wild-type mice J. Pharmacol. Exp. Ther. 292 2000 489
    • (2000) J. Pharmacol. Exp. Ther. , vol.292 , pp. 489
    • Butler, M.1
  • 40
    • 76349111051 scopus 로고    scopus 로고
    • Rational design of cationic lipids for siRNA delivery
    • S.C. Semple Rational design of cationic lipids for siRNA delivery Nat. Biotechnol. 28 2010 172
    • (2010) Nat. Biotechnol. , vol.28 , pp. 172
    • Semple, S.C.1
  • 41
    • 0029160083 scopus 로고
    • A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: Polyethylenimine
    • O. Boussif A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine Proc. Natl. Acad. Sci. U. S. A. 92 1995 7297
    • (1995) Proc. Natl. Acad. Sci. U. S. A. , vol.92 , pp. 7297
    • Boussif, O.1
  • 42
    • 66249129385 scopus 로고    scopus 로고
    • Selection, characterization and application of new RNA HIV gp120 aptamers for facile delivery of Dicer substrate siRNAs into HIV infected cells
    • J. Zhou Selection, characterization and application of new RNA HIV gp120 aptamers for facile delivery of Dicer substrate siRNAs into HIV infected cells Nucleic Acids Res. 37 2009 3094
    • (2009) Nucleic Acids Res. , vol.37 , pp. 3094
    • Zhou, J.1
  • 43
    • 84860348641 scopus 로고    scopus 로고
    • The promise, pitfalls and progress of RNA-interference-based antiviral therapy for respiratory viruses
    • J.P. DeVincenzo The promise, pitfalls and progress of RNA-interference-based antiviral therapy for respiratory viruses Antivir. Ther. 17 2012 213
    • (2012) Antivir. Ther. , vol.17 , pp. 213
    • Devincenzo, J.P.1
  • 44
    • 84903837437 scopus 로고    scopus 로고
    • Gene silencing in skin after deposition of self-delivery siRNA with a motorized microneedle array device
    • R.P. Hickerson Gene silencing in skin after deposition of self-delivery siRNA with a motorized microneedle array device Mol. Ther. Nucleic Acids 2 2013 e129
    • (2013) Mol. Ther. Nucleic Acids , vol.2 , pp. 129
    • Hickerson, R.P.1
  • 45
    • 0035942736 scopus 로고    scopus 로고
    • Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
    • S.M. Elbashir Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells Nature 411 2001 494
    • (2001) Nature , vol.411 , pp. 494
    • Elbashir, S.M.1
  • 46
    • 57149086661 scopus 로고    scopus 로고
    • Atu027, a liposomal small interfering RNA formulation targeting protein kinase N3, inhibits cancer progression
    • M. Aleku Atu027, a liposomal small interfering RNA formulation targeting protein kinase N3, inhibits cancer progression Cancer Res. 68 2008 9788
    • (2008) Cancer Res. , vol.68 , pp. 9788
    • Aleku, M.1
  • 47
    • 84867652280 scopus 로고    scopus 로고
    • The business of RNAi Therapeutics in 2012
    • D. Haussecker The business of RNAi Therapeutics in 2012 Mol. Ther. Nucleic Acids 1 2012 e8
    • (2012) Mol. Ther. Nucleic Acids , vol.1 , pp. 8
    • Haussecker, D.1
  • 48
    • 22344454846 scopus 로고    scopus 로고
    • Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA
    • A.D. Judge Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA Nat. Biotechnol. 23 2005 457
    • (2005) Nat. Biotechnol. , vol.23 , pp. 457
    • Judge, A.D.1
  • 49
    • 84908589424 scopus 로고    scopus 로고
    • http://investor.tekmirapharm.com/releasedetail.cfm?releaseid=534353
  • 50
    • 84908589423 scopus 로고    scopus 로고
    • http://www.alnylam.com/web/assets/Roundtable-ESC-GalNAc-Conjugates-072214.pdf


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.