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Volumn 5, Issue 5, 2014, Pages 569-589

Delivering the promise of small ncRNA therapeutics

Author keywords

[No Author keywords available]

Indexed keywords

SMALL UNTRANSLATED RNA; SOLID LIPID NANOPARTICLE; UNTRANSLATED RNA;

EID: 84904004156     PISSN: 20415990     EISSN: 20416008     Source Type: Journal    
DOI: 10.4155/tde.14.23     Document Type: Review
Times cited : (10)

References (144)
  • 1
    • 33748201603 scopus 로고    scopus 로고
    • Running interference: Prospects and obstacles to using small interfering RNAs as small molecule drugs
    • Dykxhoorn DM, Lieberman J. Running interference: prospects and obstacles to using small interfering RNAs as small molecule drugs. Annu. Rev. Biomed. Eng. 8, 377-402 (2006).
    • (2006) Annu. Rev. Biomed. Eng. , vol.8 , pp. 377-402
    • Dykxhoorn, D.M.1    Lieberman, J.2
  • 2
    • 33645148088 scopus 로고    scopus 로고
    • The silent treatment: SiRNAs as small molecule drugs
    • Dykxhoorn DM, Palliser D, Lieberman J. The silent treatment: siRNAs as small molecule drugs. Gene Ther. 13(6), 541-552 (2006).
    • (2006) Gene Ther. , vol.13 , Issue.6 , pp. 541-552
    • Dykxhoorn, D.M.1    Palliser, D.2    Lieberman, J.3
  • 4
    • 70349316690 scopus 로고    scopus 로고
    • Therapeutic applications of RNAi for silencing virus replication
    • Tripp RA, Tompkins SM. Therapeutic applications of RNAi for silencing virus replication. Methods Mol. Biol. 555, 43-61 (2009).
    • (2009) Methods Mol. Biol. , vol.555 , pp. 43-61
    • Tripp, R.A.1    Tompkins, S.M.2
  • 5
    • 82255175105 scopus 로고    scopus 로고
    • Biology and applications of small nucleolar RNAs
    • Bratkovic T, Rogelj B. Biology and applications of small nucleolar RNAs. Cell. Mol. Life Sci. 68(23), 3843-3851 (2011).
    • (2011) Cell. Mol. Life Sci. , vol.68 , Issue.23 , pp. 3843-3851
    • Bratkovic, T.1    Rogelj, B.2
  • 6
    • 84866295474 scopus 로고    scopus 로고
    • Characterizing ncRNAs in human pathogenic protists using high-throughput sequencing technology
    • Collins LJ. Characterizing ncRNAs in human pathogenic protists using high-throughput sequencing technology. Front. Genet. 2, 96 (2011).
    • (2011) Front. Genet. , vol.2 , pp. 96
    • Collins, L.J.1
  • 7
    • 79958274671 scopus 로고    scopus 로고
    • An introduction to small non-coding RNAs: MiRNA and snoRNA
    • Holley CL, Topkara VK. An introduction to small non-coding RNAs: miRNA and snoRNA. Cardiovasc. Drugs Ther. 25(2), 151-159 (2011).
    • (2011) Cardiovasc. Drugs Ther. , vol.25 , Issue.2 , pp. 151-159
    • Holley, C.L.1    Topkara, V.K.2
  • 8
    • 80155208165 scopus 로고    scopus 로고
    • SNOntology: Myriads of novel SnoRNAs or just a mirage?
    • Makarova JA, Kramerov DA. SNOntology: myriads of novel SnoRNAs or just a mirage? BMC Genomics 12(1), 543 (2011).
    • (2011) BMC Genomics , vol.12 , Issue.1 , pp. 543
    • Makarova, J.A.1    Kramerov, D.A.2
  • 9
    • 80053574876 scopus 로고    scopus 로고
    • From snoRNA to miRNA: Dual function regulatory non-coding RNAs
    • Scott MS, Ono M. From snoRNA to miRNA: dual function regulatory non-coding RNAs. Biochimie 93(11), 1987-1992 (2011).
    • (2011) Biochimie , vol.93 , Issue.11 , pp. 1987-1992
    • Scott, M.S.1    Ono, M.2
  • 10
    • 81355142141 scopus 로고    scopus 로고
    • Non-coding RNAs in human disease
    • Esteller M. Non-coding RNAs in human disease. Nat. Rev. Genet. 12(12), 861-874 (2011).
    • (2011) Nat. Rev. Genet. , vol.12 , Issue.12 , pp. 861-874
    • Esteller, M.1
  • 11
    • 84904082355 scopus 로고    scopus 로고
    • RNA interference therapeutics for tumour therapy: Promising work in progress
    • Lattime EC, Gerson SL (Eds). Academic Press
    • Tsujiuchi T, Wakabayashi T, Miller AD, Natsume A. RNA interference therapeutics for tumour therapy: promising work in progress. In:Gene Therapy of Cancer (3rd Edition) . Lattime EC, Gerson SL (Eds). Academic Press, 393-408 (2014).
    • (2014) Gene Therapy of Cancer (3rd Edition) , pp. 393-408
    • Tsujiuchi, T.1    Wakabayashi, T.2    Miller, A.D.3    Natsume, A.4
  • 12
    • 84876440888 scopus 로고    scopus 로고
    • CRISPR-Cas systems and RNA-guided interference Wiley Interdiscip
    • Barrangou R. CRISPR-Cas systems and RNA-guided interference. Wiley Interdiscip. Rev. RNA 4(3), 267-278 (2013).
    • (2013) Rev. RNA , vol.4 , Issue.3 , pp. 267-278
    • Barrangou, R.1
  • 13
    • 84879264708 scopus 로고    scopus 로고
    • ZFN TALEN and CRISPR/Cas-based methods for genome engineering
    • Gaj T, Gersbach CA, Barbas CF 3rd. ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. Trends Biotechnol. 31(7), 397-405 (2013).
    • (2013) Trends Biotechnol. , vol.31 , Issue.7 , pp. 397-405
    • Gaj, T.1    Gersbach, C.A.2    Barbas III, C.F.3
  • 14
    • 84879584456 scopus 로고    scopus 로고
    • CRISPR interference: A structural perspective
    • Reeks J, Naismith JH, White MF. CRISPR interference: a structural perspective. Biochem. J. 453(2), 155-166 (2013).
    • (2013) Biochem. J. , vol.453 , Issue.2 , pp. 155-166
    • Reeks, J.1    Naismith, J.H.2    White, M.F.3
  • 15
    • 84880121815 scopus 로고    scopus 로고
    • Exploiting CRISPR/Cas: Interference mechanisms and applications
    • Richter H, Randau L, Plagens A. Exploiting CRISPR/Cas: interference mechanisms and applications. Int. J. Mol. Sci. 14(7), 14518-14531 (2013).
    • (2013) Int. J. Mol. Sci. , vol.14 , Issue.7 , pp. 14518-14531
    • Richter, H.1    Randau, L.2    Plagens, A.3
  • 16
    • 36849044732 scopus 로고    scopus 로고
    • Nonviral delivery of synthetic siRNAs in vivo
    • Akhtar S, Benter IF. Nonviral delivery of synthetic siRNAs in vivo. J. Clin. Invest. 117(12), 3623-3632 (2007).
    • (2007) J. Clin. Invest. , vol.117 , Issue.12 , pp. 3623-3632
    • Akhtar, S.1    Benter, I.F.2
  • 17
    • 34047190259 scopus 로고    scopus 로고
    • Hydrophobization and bioconjugation for enhanced siRNA delivery and targeting
    • De Paula D, Bentley MV, Mahato RI. Hydrophobization and bioconjugation for enhanced siRNA delivery and targeting. RNA 13(4), 431-456 (2007).
    • (2007) RNA , vol.13 , Issue.4 , pp. 431-456
    • De Paula, D.1    Bentley, M.V.2    Mahato, R.I.3
  • 18
    • 67249096677 scopus 로고    scopus 로고
    • Nonviral methods for siRNA delivery
    • Gao K, Huang L. Nonviral methods for siRNA delivery. Mol. Pharm. 6(3), 651-658 (2009).
    • (2009) Mol. Pharm. , vol.6 , Issue.3 , pp. 651-658
    • Gao, K.1    Huang, L.2
  • 20
    • 57349114898 scopus 로고    scopus 로고
    • Stability study of unmodified siRNA and relevance to clinical use
    • Hickerson RP, Vlassov AV, Wang Q et al. Stability study of unmodified siRNA and relevance to clinical use. Oligonucleotides 18(4), 345-354 (2008).
    • (2008) Oligonucleotides , vol.18 , Issue.4 , pp. 345-354
    • Hickerson, R.P.1    Vlassov, A.V.2    Wang, Q.3
  • 21
    • 74049153383 scopus 로고    scopus 로고
    • Systemic delivery and quantification of unformulated interfering RNAs in vivo
    • Morin A, Gallou-Kabani C, Mathieu JR, Cabon F. Systemic delivery and quantification of unformulated interfering RNAs in vivo. Curr. Top. Med. Chem. 9(12), 1117-1129 (2009).
    • (2009) Curr. Top. Med. Chem. , vol.9 , Issue.12 , pp. 1117-1129
    • Morin, A.1    Gallou-Kabani, C.2    Mathieu, J.R.3    Cabon, F.4
  • 23
    • 67249142681 scopus 로고    scopus 로고
    • SiRNA delivery and targeting
    • Cheng K, Mahato RI. siRNA delivery and targeting. Mol. Pharm. 6(3), 649-650 (2009).
    • (2009) Mol. Pharm. , vol.6 , Issue.3 , pp. 649-650
    • Cheng, K.1    Mahato, R.I.2
  • 24
    • 55649089421 scopus 로고    scopus 로고
    • State of the art and perspectives for the delivery of antisense oligonucleotides and siRNA by polymeric nanocarriers
    • Fattal E, Bochot A. State of the art and perspectives for the delivery of antisense oligonucleotides and siRNA by polymeric nanocarriers. Int. J. Pharm. 364(2), 237-248 (2008).
    • (2008) Int. J. Pharm. , vol.364 , Issue.2 , pp. 237-248
    • Fattal, E.1    Bochot, A.2
  • 25
    • 41949117146 scopus 로고    scopus 로고
    • Liposomal nanomedicines: An emerging field
    • Fenske DB, Chonn A, Cullis PR. Liposomal nanomedicines: an emerging field. Toxicol. Pathol. 36(1), 21-29 (2008).
    • (2008) Toxicol. Pathol. , vol.36 , Issue.1 , pp. 21-29
    • Fenske, D.B.1    Chonn, A.2    Cullis, P.R.3
  • 27
    • 57349152290 scopus 로고    scopus 로고
    • Targeted delivery of siRNA by nonviral vectors: Lessons learned from recent advances
    • Li SD, Huang L. Targeted delivery of siRNA by nonviral vectors: lessons learned from recent advances. Curr. Opin. Investig. Drugs 9(12), 1317-1323 (2008).
    • (2008) Curr. Opin. Investig. Drugs , vol.9 , Issue.12 , pp. 1317-1323
    • Li, S.D.1    Huang, L.2
  • 28
    • 33847099637 scopus 로고    scopus 로고
    • Lipid-based nanoparticles for nucleic acid delivery
    • Li W, Szoka FC Jr. Lipid-based nanoparticles for nucleic acid delivery. Pharm. Res. 24(3), 438-449 (2007).
    • (2007) Pharm. Res. , vol.24 , Issue.3 , pp. 438-449
    • Li, W.1    Szoka Jr., F.C.2
  • 29
    • 59249099019 scopus 로고    scopus 로고
    • Extracellular targeting of synthetic therapeutic nucleic acid formulations
    • Philipp A, Meyer M, Wagner E. Extracellular targeting of synthetic therapeutic nucleic acid formulations. Curr. Gene Ther. 8(5), 324-334 (2008).
    • (2008) Curr. Gene Ther. , vol.8 , Issue.5 , pp. 324-334
    • Philipp, A.1    Meyer, M.2    Wagner, E.3
  • 30
    • 72949119713 scopus 로고    scopus 로고
    • Lipidic systems for in vivo siRNA delivery
    • Wu SY, McMillan NA. Lipidic systems for in vivo siRNA delivery. AAPS J. 11(4), 639-652 (2009).
    • (2009) AAPS J. , vol.11 , Issue.4 , pp. 639-652
    • Wu, S.Y.1    McMillan, N.A.2
  • 33
    • 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
  • 34
    • 27744466964 scopus 로고    scopus 로고
    • Synthetic, self-assembly ABCD nanoparticles; A structural paradigm for viable synthetic non-viral vectors
    • Kostarelos K, Miller AD. Synthetic, self-assembly ABCD nanoparticles; a structural paradigm for viable synthetic non-viral vectors. Chem. Soc. Rev. 34(11), 970-994 (2005).
    • (2005) Chem. Soc. Rev. , vol.34 , Issue.11 , pp. 970-994
    • Kostarelos, K.1    Miller, A.D.2
  • 35
    • 60149094505 scopus 로고    scopus 로고
    • RNA interference: From basic research to therapeutic applications
    • Kurreck J. RNA interference: from basic research to therapeutic applications. Angew. Chem. Int. Ed. Engl. 48(8), 1378-1398 (2009).
    • (2009) Angew. Chem. Int. Ed. Engl. , vol.48 , Issue.8 , pp. 1378-1398
    • Kurreck, J.1
  • 36
    • 45249103527 scopus 로고    scopus 로고
    • Towards safe nanoparticle technologies for nucleic acid therapeutics
    • Miller AD. Towards safe nanoparticle technologies for nucleic acid therapeutics. Tumori 94(2), 234-245 (2008).
    • (2008) Tumori , vol.94 , Issue.2 , pp. 234-245
    • Miller, A.D.1
  • 37
    • 80053513757 scopus 로고    scopus 로고
    • Synthetic nucleic acid delivery systems in gene therapy
    • doi:10.1002/9780470015902.a0005745.pub2, Wiley & Sons, Chichester, UK
    • Miller AD. Synthetic nucleic acid delivery systems in gene therapy. In: Encyclopedia of Life Sciences. doi:10.1002/9780470015902.a0005745.pub2 Wiley & Sons, Chichester, UK (2008).
    • (2008) Encyclopedia of Life Sciences
    • Miller, A.D.1
  • 39
    • 84887762938 scopus 로고    scopus 로고
    • Delivery of RNAi therapeutics: Work in progress
    • Miller AD. Delivery of RNAi therapeutics: work in progress. Expert Rev. Med. Devices 10(6), 781-811 (2013).
    • (2013) Expert Rev. Med. Devices , vol.10 , Issue.6 , pp. 781-811
    • Miller, A.D.1
  • 41
    • 0038482054 scopus 로고    scopus 로고
    • The problem with cationic liposome/micelle-based non-viral vector systems for gene therapy
    • Miller AD. The problem with cationic liposome/micelle-based non-viral vector systems for gene therapy. Curr. Med. Chem. 10(14), 1195-1211 (2003).
    • (2003) Curr. Med. Chem. , vol.10 , Issue.14 , pp. 1195-1211
    • Miller, A.D.1
  • 42
    • 0032858150 scopus 로고    scopus 로고
    • Stabilized plasmid-lipid particles for regional gene therapy: Formulation and transfection properties
    • Zhang YP, Sekirov L, Saravolac EG et al. Stabilized plasmid-lipid particles for regional gene therapy: formulation and transfection properties. Gene Ther. 6(8), 1438-1447 (1999).
    • (1999) Gene Ther. , vol.6 , Issue.8 , pp. 1438-1447
    • Zhang, Y.P.1    Sekirov, L.2    Saravolac, E.G.3
  • 43
    • 0032991616 scopus 로고    scopus 로고
    • Stabilized plasmid-lipid particles: Construction and characterization
    • Wheeler JJ, Palmer L, Ossanlou M et al. Stabilized plasmid-lipid particles: construction and characterization. Gene Ther. 6(2), 271-281 (1999).
    • (1999) Gene Ther. , vol.6 , Issue.2 , pp. 271-281
    • Wheeler, J.J.1    Palmer, L.2    Ossanlou, M.3
  • 44
    • 0033036668 scopus 로고    scopus 로고
    • Stabilized plasmid-lipid particles: Factors influencing plasmid entrapment and transfection properties
    • Mok KW, Lam AM, Cullis PR. Stabilized plasmid-lipid particles: factors influencing plasmid entrapment and transfection properties. Biochim. Biophys. Acta 1419(2), 137-150 (1999).
    • (1999) Biochim. Biophys. Acta , vol.1419 , Issue.2 , pp. 137-150
    • Mok, K.W.1    Lam, A.M.2    Cullis, P.R.3
  • 45
    • 23444445660 scopus 로고    scopus 로고
    • Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs
    • Morrissey DV, Lockridge JA, Shaw L et al. Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs. Nat. Biotechnol. 23(8), 1002-1007 (2005).
    • (2005) Nat. Biotechnol. , vol.23 , Issue.8 , pp. 1002-1007
    • Morrissey, D.V.1    Lockridge, J.A.2    Shaw, L.3
  • 46
    • 33646185371 scopus 로고    scopus 로고
    • RNAi-mediated gene silencing in non-human primates
    • Zimmermann TS, Lee AC, Akinc A et al. RNAi-mediated gene silencing in non-human primates. Nature 441(7089), 111-114 (2006).
    • (2006) Nature , vol.441 , Issue.7089 , pp. 111-114
    • Zimmermann, T.S.1    Lee, A.C.2    Akinc, A.3
  • 47
    • 76349111051 scopus 로고    scopus 로고
    • Rational design of cationic lipids for siRNA delivery
    • Semple SC, Akinc A, Chen J et al. Rational design of cationic lipids for siRNA delivery. Nat. Biotechnol. 28(2), 172-176 (2010).
    • (2010) Nat. Biotechnol. , vol.28 , Issue.2 , pp. 172-176
    • Semple, S.C.1    Akinc, A.2    Chen, J.3
  • 48
    • 82955233888 scopus 로고    scopus 로고
    • Influence of cationic lipid composition on gene silencing properties of lipid nanoparticle formulations of siRNA in antigen-presenting cells
    • Basha G, Novobrantseva TI, Rosin N et al. Influence of cationic lipid composition on gene silencing properties of lipid nanoparticle formulations of siRNA in antigen-presenting cells. Mol. Ther. 19(12), 2186-2200 (2011).
    • (2011) Mol. Ther. , vol.19 , Issue.12 , pp. 2186-2200
    • Basha, G.1    Novobrantseva, T.I.2    Rosin, N.3
  • 49
    • 84863201108 scopus 로고    scopus 로고
    • Lipid nanoparticle siRNA systems for silencing the androgen receptor in human prostate cancer in vivo
    • Lee JB, Zhang K, Tam YY et al. Lipid nanoparticle siRNA systems for silencing the androgen receptor in human prostate cancer in vivo. Int. J. Cancer 131(5), E781-E790 (2012).
    • (2012) Int. J. Cancer , vol.131 , Issue.5
    • Lee, J.B.1    Zhang, K.2    Tam, Y.Y.3
  • 50
    • 84865079107 scopus 로고    scopus 로고
    • Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo
    • Jayaraman M, Ansell SM, Mui BL et al. Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo. Angew. Chem. Int. Ed. Engl. 51(34), 8529-8533 (2012).
    • (2012) Angew. Chem. Int. Ed. Engl. , vol.51 , Issue.34 , pp. 8529-8533
    • Jayaraman, M.1    Ansell, S.M.2    Mui, B.L.3
  • 51
    • 0024557781 scopus 로고
    • Effects of apolipoproteins A-IV and A-I on the uptake of phospholipid liposomes by hepatocytes
    • Bisgaier CL, Siebenkas MV, Williams KJ. Effects of apolipoproteins A-IV and A-I on the uptake of phospholipid liposomes by hepatocytes. J. Biol. Chem. 264(2), 862-866 (1989).
    • (1989) J. Biol. Chem. , vol.264 , Issue.2 , pp. 862-866
    • Bisgaier, C.L.1    Siebenkas, M.V.2    Williams, K.J.3
  • 52
    • 74149092120 scopus 로고    scopus 로고
    • Evaluation of efficacy, biodistribution, and inflammation for a potent siRNA nanoparticle: Effect of dexamethasone co-treatment
    • Abrams MT, Koser ML, Seitzer J et al. Evaluation of efficacy, biodistribution, and inflammation for a potent siRNA nanoparticle: effect of dexamethasone co-treatment. Mol. Ther. 18(1), 171-180 (2010).
    • (2010) Mol. Ther. , vol.18 , Issue.1 , pp. 171-180
    • Abrams, M.T.1    Koser, M.L.2    Seitzer, J.3
  • 53
    • 78650180150 scopus 로고    scopus 로고
    • Analysis of lipid nanoparticles by Cryo-EM for characterizing siRNA delivery vehicles
    • Crawford R, Dogdas B, Keough E et al. Analysis of lipid nanoparticles by Cryo-EM for characterizing siRNA delivery vehicles. Int. J. Pharm. 403(1-2), 237-244 (2011).
    • (2011) Int. J. Pharm. , vol.403 , Issue.1-2 , pp. 237-244
    • Crawford, R.1    Dogdas, B.2    Keough, E.3
  • 54
    • 78649683035 scopus 로고    scopus 로고
    • Quantitative evaluation of siRNA delivery in vivo
    • Pei Y, Hancock PJ, Zhang H et al. Quantitative evaluation of siRNA delivery in vivo. RNA 16(12), 2553-2563 (2010).
    • (2010) RNA , vol.16 , Issue.12 , pp. 2553-2563
    • Pei, Y.1    Hancock, P.J.2    Zhang, H.3
  • 55
    • 79952187464 scopus 로고    scopus 로고
    • Mechanistically probing lipid-siRNA nanoparticle-associated toxicities identifies Jak inhibitors effective in mitigating multifaceted toxic responses
    • Tao W, Mao X, Davide JP et al. Mechanistically probing lipid-siRNA nanoparticle-associated toxicities identifies Jak inhibitors effective in mitigating multifaceted toxic responses. Mol. Ther. 19(3), 567-575 (2011).
    • (2011) Mol. Ther. , vol.19 , Issue.3 , pp. 567-575
    • Tao, W.1    Mao, X.2    Davide, J.P.3
  • 56
    • 33748331337 scopus 로고    scopus 로고
    • RNA interference in the mouse vascular endothelium by systemic administration of siRNA-lipoplexes for cancer therapy
    • Santel A, Aleku M, Keil O et al. RNA interference in the mouse vascular endothelium by systemic administration of siRNA-lipoplexes for cancer therapy. Gene Ther. 13(18), 1360-1370 (2006).
    • (2006) Gene Ther. , vol.13 , Issue.18 , pp. 1360-1370
    • Santel, A.1    Aleku, M.2    Keil, O.3
  • 57
    • 33746883220 scopus 로고    scopus 로고
    • A novel siRNA-lipoplex technology for RNA interference in the mouse vascular endothelium
    • Santel A, Aleku M, Keil O et al. A novel siRNA-lipoplex technology for RNA interference in the mouse vascular endothelium. Gene Ther. 13(16), 1222-1234 (2006).
    • (2006) Gene Ther. , vol.13 , Issue.16 , pp. 1222-1234
    • Santel, A.1    Aleku, M.2    Keil, O.3
  • 58
    • 57149086661 scopus 로고    scopus 로고
    • Atu027, a liposomal small interfering RNA formulation targeting protein kinase N3, inhibits cancer progression
    • Aleku M, Schulz P, Keil O et al. Atu027, a liposomal small interfering RNA formulation targeting protein kinase N3, inhibits cancer progression. Cancer Res. 68(23), 9788-9798 (2008).
    • (2008) Cancer Res. , vol.68 , Issue.23 , pp. 9788-9798
    • Aleku, M.1    Schulz, P.2    Keil, O.3
  • 59
    • 85027927478 scopus 로고    scopus 로고
    • An amino acid-based amphoteric liposomal delivery system for systemic administration of siRNA
    • Adami RC, Seth S, Harvie P et al. An amino acid-based amphoteric liposomal delivery system for systemic administration of siRNA. Mol. Ther. 19(6), 1141-1151 (2011).
    • (2011) Mol. Ther. , vol.19 , Issue.6 , pp. 1141-1151
    • Adami, R.C.1    Seth, S.2    Harvie, P.3
  • 60
    • 55349125661 scopus 로고    scopus 로고
    • Tumor regression in mice by delivery of Bcl-2 small interfering RNA with pegylated cationic liposomes
    • Sonoke S, Ueda T, Fujiwara K et al. Tumor regression in mice by delivery of Bcl-2 small interfering RNA with pegylated cationic liposomes. Cancer Res. 68(21), 8843-8851 (2008).
    • (2008) Cancer Res. , vol.68 , Issue.21 , pp. 8843-8851
    • Sonoke, S.1    Ueda, T.2    Fujiwara, K.3
  • 61
    • 84891831905 scopus 로고    scopus 로고
    • Knockdown of beta-catenin with dicer-substrate siRNAs reduces liver tumor burden in vivo
    • Dudek H, Wong DH, Arvan R et al. Knockdown of beta-catenin with dicer-substrate siRNAs reduces liver tumor burden in vivo. Mol. Ther. 22(1), 92-101 (2014).
    • (2014) Mol. Ther. , vol.22 , Issue.1 , pp. 92-101
    • Dudek, H.1    Wong, D.H.2    Arvan, R.3
  • 62
    • 54949125913 scopus 로고    scopus 로고
    • Folate-linked lipid-based nanoparticles for synthetic siRNA delivery in KB tumor xenografts
    • Yoshizawa T, Hattori Y, Hakoshima M, Koga K, Maitani Y. Folate-linked lipid-based nanoparticles for synthetic siRNA delivery in KB tumor xenografts. Eur. J. Pharm. Biopharm. 70(3), 718-725 (2008).
    • (2008) Eur. J. Pharm. Biopharm. , vol.70 , Issue.3 , pp. 718-725
    • Yoshizawa, T.1    Hattori, Y.2    Hakoshima, M.3    Koga, K.4    Maitani, Y.5
  • 63
    • 33845679807 scopus 로고    scopus 로고
    • Surface-modified LPD nanoparticles for tumor targeting
    • Li SD, Huang L. Surface-modified LPD nanoparticles for tumor targeting. Ann. N. Y. Acad. Sci. 1082, 1-8 (2006).
    • (2006) Ann. N. Y. Acad. Sci. , vol.1082 , pp. 1-8
    • Li, S.D.1    Huang, L.2
  • 64
    • 33750417183 scopus 로고    scopus 로고
    • Targeted delivery of antisense oligodeoxynucleotide and small interference RNA into lung cancer cells
    • Li SD, Huang L. Targeted delivery of antisense oligodeoxynucleotide and small interference RNA into lung cancer cells. Mol. Pharm. 3(5), 579-588 (2006).
    • (2006) Mol. Pharm. , vol.3 , Issue.5 , pp. 579-588
    • Li, S.D.1    Huang, L.2
  • 65
    • 52049121804 scopus 로고    scopus 로고
    • An efficient and low immunostimulatory nanoparticle formulation for systemic siRNA delivery to the tumor
    • Chono S, Li SD, Conwell CC, Huang L. An efficient and low immunostimulatory nanoparticle formulation for systemic siRNA delivery to the tumor. J. Control. Release 131(1), 64-69 (2008).
    • (2008) J. Control. Release , vol.131 , Issue.1 , pp. 64-69
    • Chono, S.1    Li, S.D.2    Conwell, C.C.3    Huang, L.4
  • 66
    • 67249131198 scopus 로고    scopus 로고
    • Novel cationic lipid that delivers siRNA and enhances therapeutic effect in lung cancer cells
    • Chen Y, Sen J, Bathula SR, Yang Q, Fittipaldi R, Huang L. Novel cationic lipid that delivers siRNA and enhances therapeutic effect in lung cancer cells. Mol. Pharm. 6(3), 696-705 (2009).
    • (2009) Mol. Pharm. , vol.6 , Issue.3 , pp. 696-705
    • Chen, Y.1    Sen, J.2    Bathula, S.R.3    Yang, Q.4    Fittipaldi, R.5    Huang, L.6
  • 67
    • 70349542520 scopus 로고    scopus 로고
    • Nanoparticles evading the reticuloendothelial system: Role of the supported bilayer
    • Li SD, Huang L. Nanoparticles evading the reticuloendothelial system: role of the supported bilayer. Biochim. Biophys Acta 1788(10), 2259-2266 (2009).
    • (2009) Biochim. Biophys Acta , vol.1788 , Issue.10 , pp. 2259-2266
    • Li, S.D.1    Huang, L.2
  • 68
    • 38849166053 scopus 로고    scopus 로고
    • Systemic leukocyte-directed siRNA delivery revealing cyclin D1 as an anti-inflammatory target
    • Peer D, Park EJ, Morishita Y, Carman CV, Shimaoka M. Systemic leukocyte-directed siRNA delivery revealing cyclin D1 as an anti-inflammatory target. Science 319(5863), 627-630 (2008).
    • (2008) Science , vol.319 , Issue.5863 , pp. 627-630
    • Peer, D.1    Park, E.J.2    Morishita, Y.3    Carman, C.V.4    Shimaoka, M.5
  • 69
    • 65349118108 scopus 로고    scopus 로고
    • Systemic siRNA delivery to leukocyte-implicated diseases
    • Peer D, Shimaoka M. Systemic siRNA delivery to leukocyte-implicated diseases. Cell Cycle 8(6), 853-859 (2009).
    • (2009) Cell Cycle , vol.8 , Issue.6 , pp. 853-859
    • Peer, D.1    Shimaoka, M.2
  • 70
    • 84555195893 scopus 로고    scopus 로고
    • Integrin-targeted stabilized nanoparticles for an efficient delivery of siRNAs in vitro and in vivo
    • Srinivasan C, Peer D, Shimaoka M. Integrin-targeted stabilized nanoparticles for an efficient delivery of siRNAs in vitro and in vivo. Methods Mol. Biol. 820, 105-116 (2012).
    • (2012) Methods Mol. Biol. , vol.820 , pp. 105-116
    • Srinivasan, C.1    Peer, D.2    Shimaoka, M.3
  • 71
    • 67249148563 scopus 로고    scopus 로고
    • Controlling HBV replication in vivo by intravenous administration of triggered PEGylated siRNA-nanoparticles
    • Carmona S, Jorgensen MR, Kolli S et al. Controlling HBV replication in vivo by intravenous administration of triggered PEGylated siRNA-nanoparticles. Mol. Pharm. 6(3), 706-717 (2009).
    • (2009) Mol. Pharm. , vol.6 , Issue.3 , pp. 706-717
    • Carmona, S.1    Jorgensen, M.R.2    Kolli, S.3
  • 72
    • 84875467836 scopus 로고    scopus 로고
    • pH-Triggered nanoparticle mediated delivery of siRNA to liver cells in vitro and in vivo
    • Kolli S, Wong SP, Harbottle R, Johnston B, Thanou M, Miller AD. pH-Triggered nanoparticle mediated delivery of siRNA to liver cells in vitro and in vivo. Bioconjug. Chem. 24(3), 314-332 (2013).
    • (2013) Bioconjug. Chem. , vol.24 , Issue.3 , pp. 314-332
    • Kolli, S.1    Wong, S.P.2    Harbottle, R.3    Johnston, B.4    Thanou, M.5    Miller, A.D.6
  • 73
    • 78651352062 scopus 로고    scopus 로고
    • Novel multifunctional nanoparticle mediates siRNA tumour delivery, visualisation and therapeutic tumour reduction in vivo
    • Kenny GD, Kamaly N, Kalber TL et al. Novel multifunctional nanoparticle mediates siRNA tumour delivery, visualisation and therapeutic tumour reduction in vivo. J. Control. Release 149(2), 111-116 (2011).
    • (2011) J. Control. Release , vol.149 , Issue.2 , pp. 111-116
    • Kenny, G.D.1    Kamaly, N.2    Kalber, T.L.3
  • 74
    • 80052552280 scopus 로고    scopus 로고
    • Efficient topical delivery of plasmid DNA to lung in vivo mediated by putative triggered, PEGylated pDNA nanoparticles
    • Aissaoui A, Chami M, Hussein M, Miller AD. Efficient topical delivery of plasmid DNA to lung in vivo mediated by putative triggered, PEGylated pDNA nanoparticles. J. Control. Release 154(3), 275-284 (2011).
    • (2011) J. Control. Release , vol.154 , Issue.3 , pp. 275-284
    • Aissaoui, A.1    Chami, M.2    Hussein, M.3    Miller, A.D.4
  • 75
    • 38949123316 scopus 로고    scopus 로고
    • Bimodal paramagnetic and fluorescent liposomes for cellular and tumor magnetic resonance imaging
    • Kamaly N, Kalber T, Ahmad A et al. Bimodal paramagnetic and fluorescent liposomes for cellular and tumor magnetic resonance imaging. Bioconjug. Chem. 19(1), 118-129 (2008).
    • (2008) Bioconjug. Chem. , vol.19 , Issue.1 , pp. 118-129
    • Kamaly, N.1    Kalber, T.2    Ahmad, A.3
  • 76
    • 65249161653 scopus 로고    scopus 로고
    • Folate receptor targeted bimodal liposomes for tumor magnetic resonance imaging
    • Kamaly N, Kalber T, Thanou M, Bell JD, Miller AD. Folate receptor targeted bimodal liposomes for tumor magnetic resonance imaging. Bioconjug. Chem. 20(4), 648-655 (2009).
    • (2009) Bioconjug. Chem. , vol.20 , Issue.4 , pp. 648-655
    • Kamaly, N.1    Kalber, T.2    Thanou, M.3    Bell, J.D.4    Miller, A.D.5
  • 77
    • 76849089676 scopus 로고    scopus 로고
    • A novel bimodal lipidic contrast agent for cellular labelling and tumour MRI
    • Kamaly N, Kalber T, Kenny G, Bell J, Jorgensen M, Miller A. A novel bimodal lipidic contrast agent for cellular labelling and tumour MRI. Org. Biomol. Chem. 8(1), 201-211 (2010).
    • (2010) Org. Biomol. Chem. , vol.8 , Issue.1 , pp. 201-211
    • Kamaly, N.1    Kalber, T.2    Kenny, G.3    Bell, J.4    Jorgensen, M.5    Miller, A.6
  • 78
    • 84885178465 scopus 로고    scopus 로고
    • Examination of the effect of increasing the number of intra-disulfide amino functional groups on the performance of small molecule cyclic polyamine disulfide vectors
    • Drake CR, Aissaoui A, Argyros O, Thanou M, Steinke JH, Miller AD. Examination of the effect of increasing the number of intra-disulfide amino functional groups on the performance of small molecule cyclic polyamine disulfide vectors. J. Control. Release 171(1), 81-90 (2013).
    • (2013) J. Control. Release , vol.171 , Issue.1 , pp. 81-90
    • Drake, C.R.1    Aissaoui, A.2    Argyros, O.3    Thanou, M.4    Steinke, J.H.5    Miller, A.D.6
  • 79
    • 0034743833 scopus 로고    scopus 로고
    • Physico-chemical analysis of cationic liposome-DNA complexes (lipoplexes) with respect to in vitro and in vivo gene delivery efficiency
    • Stewart L, Manvell M, Hillery E et al. Physico-chemical analysis of cationic liposome-DNA complexes (lipoplexes) with respect to in vitro and in vivo gene delivery efficiency. J. Chem. Soc., Perkin Trans. 2, 624-632 (2001).
    • (2001) J. Chem. Soc., Perkin Trans. , vol.2 , pp. 624-632
    • Stewart, L.1    Manvell, M.2    Hillery, E.3
  • 81
    • 61849132133 scopus 로고    scopus 로고
    • Targeting the urokinase plasminogen activator receptor with synthetic self-assembly nanoparticles
    • Wang M, Lowik DW, Miller AD, Thanou M. Targeting the urokinase plasminogen activator receptor with synthetic self-assembly nanoparticles. Bioconjug. Chem. 20(1), 32-40 (2009).
    • (2009) Bioconjug. Chem. , vol.20 , Issue.1 , pp. 32-40
    • Wang, M.1    Lowik, D.W.2    Miller, A.D.3    Thanou, M.4
  • 82
    • 84880534127 scopus 로고    scopus 로고
    • Effect of surface charge and ligand organization on the specific cell-uptake of uPAR-targeted nanoparticles
    • Wang M, Miller AD, Thanou M. Effect of surface charge and ligand organization on the specific cell-uptake of uPAR-targeted nanoparticles. J. Drug Target. 21(7), 684-692 (2013).
    • (2013) J. Drug Target. , vol.21 , Issue.7 , pp. 684-692
    • Wang, M.1    Miller, A.D.2    Thanou, M.3
  • 83
    • 12444339877 scopus 로고    scopus 로고
    • Nuclear localisation sequence templated nonviral gene delivery vectors. Investigation of intracellular trafficking events of LMD and LD vector systems
    • Keller M, Harbottle RP, Perouzel E et al. Nuclear localisation sequence templated nonviral gene delivery vectors. investigation of intracellular trafficking events of LMD and LD vector systems. Chembiochem 4(4), 286-298 (2003).
    • (2003) Chembiochem , vol.4 , Issue.4 , pp. 286-298
    • Keller, M.1    Harbottle, R.P.2    Perouzel, E.3
  • 84
    • 0035078410 scopus 로고    scopus 로고
    • Steric stabilization of fusogenic liposomes by a low-pH sensitive PEG - Diortho ester - Lipid conjugate
    • Guo X, Szoka FC Jr. Steric stabilization of fusogenic liposomes by a low-pH sensitive PEG - diortho ester - lipid conjugate. Bioconjug. Chem. 12(2), 291-300 (2001).
    • (2001) Bioconjug. Chem. , vol.12 , Issue.2 , pp. 291-300
    • Guo, X.1    Szoka Jr., F.C.2
  • 85
    • 14044252938 scopus 로고    scopus 로고
    • Toward synthetic viruses: Endosomal pH-triggered deshielding of targeted polyplexes greatly enhances gene transfer in vitro and in vivo
    • Walker GF, Fella C, Pelisek J et al. Toward synthetic viruses: endosomal pH-triggered deshielding of targeted polyplexes greatly enhances gene transfer in vitro and in vivo. Mol. Ther. 11(3), 418-425 (2005).
    • (2005) Mol. Ther. , vol.11 , Issue.3 , pp. 418-425
    • Walker, G.F.1    Fella, C.2    Pelisek, J.3
  • 86
    • 37349044610 scopus 로고    scopus 로고
    • A novel IRQ ligand-modified nano-carrier targeted to a unique pathway of caveolar endocytic pathway
    • Mudhakir D, Akita H, Tan E, Harashima H. A novel IRQ ligand-modified nano-carrier targeted to a unique pathway of caveolar endocytic pathway. J. Control. Release 125(2), 164-173 (2008).
    • (2008) J. Control. Release , vol.125 , Issue.2 , pp. 164-173
    • Mudhakir, D.1    Akita, H.2    Tan, E.3    Harashima, H.4
  • 87
    • 69949155656 scopus 로고    scopus 로고
    • A pH-sensitive fusogenic peptide facilitates endosomal escape and greatly enhances the gene silencing of siRNA-containing nanoparticles in vitro and in vivo
    • Hatakeyama H, Ito E, Akita H et al. A pH-sensitive fusogenic peptide facilitates endosomal escape and greatly enhances the gene silencing of siRNA-containing nanoparticles in vitro and in vivo. J. Control. Release 139(2), 127-132 (2009).
    • (2009) J. Control. Release , vol.139 , Issue.2 , pp. 127-132
    • Hatakeyama, H.1    Ito, E.2    Akita, H.3
  • 88
    • 33845625261 scopus 로고    scopus 로고
    • Development of a novel systemic gene delivery system for cancer therapy with a tumor-specific cleavable PEG-lipid
    • Hatakeyama H, Akita H, Kogure K et al. Development of a novel systemic gene delivery system for cancer therapy with a tumor-specific cleavable PEG-lipid. Gene Ther. 14(1), 68-77 (2007).
    • (2007) Gene Ther. , vol.14 , Issue.1 , pp. 68-77
    • Hatakeyama, H.1    Akita, H.2    Kogure, K.3
  • 89
    • 0344873301 scopus 로고    scopus 로고
    • Pharmaco attributes of dioleoylphosphatidylethanolamine/ cholesterylhemisuccinate liposomes containing different types of cleavable lipopolymers
    • Zhang JX, Zalipsky S, Mullah N, Pechar M, Allen TM. Pharmaco attributes of dioleoylphosphatidylethanolamine/ cholesterylhemisuccinate liposomes containing different types of cleavable lipopolymers. Pharmacol. Res. 49(2), 185-198 (2004).
    • (2004) Pharmacol. Res. , vol.49 , Issue.2 , pp. 185-198
    • Zhang, J.X.1    Zalipsky, S.2    Mullah, N.3    Pechar, M.4    Allen, T.M.5
  • 90
    • 84875464303 scopus 로고    scopus 로고
    • Enzyme-triggered PEGylated pDNA-nanoparticles for controlled release of pDNA in tumors
    • Yingyuad P, Mevel M, Prata C et al. Enzyme-triggered PEGylated pDNA-nanoparticles for controlled release of pDNA in tumors. Bioconjug. Chem. 24(3), 343-362 (2013).
    • (2013) Bioconjug. Chem. , vol.24 , Issue.3 , pp. 343-362
    • Yingyuad, P.1    Mevel, M.2    Prata, C.3
  • 92
    • 78649944492 scopus 로고    scopus 로고
    • Bioresponsive small molecule polyamines as non-cytotoxic alternative to polyethylenimine
    • Drake CR, Aissaoui A, Argyros O et al. Bioresponsive small molecule polyamines as non-cytotoxic alternative to polyethylenimine. Mol. Pharm. 7(6), 2040-2055 (2010).
    • (2010) Mol. Pharm. , vol.7 , Issue.6 , pp. 2040-2055
    • Drake, C.R.1    Aissaoui, A.2    Argyros, O.3
  • 93
    • 0033199557 scopus 로고    scopus 로고
    • New detachable poly(ethylene glycol) conjugates: Cysteine-cleavable lipopolymers regenerating natural phospholipid, diacyl phosphatidylethanolamine
    • Zalipsky S, Qazen M, Walker JA 2nd, Mullah N, Quinn YP, Huang SK. New detachable poly(ethylene glycol) conjugates: cysteine-cleavable lipopolymers regenerating natural phospholipid, diacyl phosphatidylethanolamine. Bioconjug. Chem. 10(5), 703-707 (1999).
    • (1999) Bioconjug. Chem. , vol.10 , Issue.5 , pp. 703-707
    • Zalipsky, S.1    Qazen, M.2    Walker II, J.A.3    Mullah, N.4    Quinn, Y.P.5    Huang, S.K.6
  • 94
    • 69949164681 scopus 로고    scopus 로고
    • Novel peptide ligand directs liposomes toward EGF-R high-expressing cancer cells in vitro and in vivo
    • Song S, Liu D, Peng J et al. Novel peptide ligand directs liposomes toward EGF-R high-expressing cancer cells in vitro and in vivo. FASEB J. 23(5), 1396-1404 (2009).
    • (2009) FASEB J. , vol.23 , Issue.5 , pp. 1396-1404
    • Song, S.1    Liu, D.2    Peng, J.3
  • 95
    • 77954243341 scopus 로고    scopus 로고
    • Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms
    • Akinc A, Querbes W, De S et al. Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms. Mol. Ther. 18(7), 1357-1364 (2010).
    • (2010) Mol. Ther. , vol.18 , Issue.7 , pp. 1357-1364
    • Akinc, A.1    Querbes, W.2    De, S.3
  • 96
    • 38849095313 scopus 로고    scopus 로고
    • Clostridium neurotoxin fragments as potential targeting moieties for liposomal gene delivery to the CNS
    • Andreu A, Fairweather N, Miller AD. Clostridium neurotoxin fragments as potential targeting moieties for liposomal gene delivery to the CNS. Chembiochem 9(2), 219-231 (2008).
    • (2008) Chembiochem , vol.9 , Issue.2 , pp. 219-231
    • Andreu, A.1    Fairweather, N.2    Miller, A.D.3
  • 97
    • 22144437300 scopus 로고    scopus 로고
    • Synthesis and Application of integrin targeting lipopeptides in targeted gene delivery
    • Waterhouse JE, Harbottle RP, Keller M et al. Synthesis and Application of integrin targeting lipopeptides in targeted gene delivery. Chembiochem 6(7), 1212-1223 (2005).
    • (2005) Chembiochem , vol.6 , Issue.7 , pp. 1212-1223
    • Waterhouse, J.E.1    Harbottle, R.P.2    Keller, M.3
  • 98
    • 56349102126 scopus 로고    scopus 로고
    • Tf-lipoplexes for neuronal siRNA delivery: A promising system to mediate gene silencing in the CNS
    • Cardoso AL, Simoes S, De Almeida LP et al. Tf-lipoplexes for neuronal siRNA delivery: a promising system to mediate gene silencing in the CNS. J. Control. Release 132(2), 113-123 (2008).
    • (2008) J. Control. Release , vol.132 , Issue.2 , pp. 113-123
    • Cardoso, A.L.1    Simoes, S.2    De Almeida, L.P.3
  • 99
    • 47649089265 scopus 로고    scopus 로고
    • Induction of apoptosis in murine neuroblastoma by systemic delivery of transferrin-shielded siRNA polyplexes for downregulation of RNA
    • Tietze N, Pelisek J, Philipp A et al. Induction of apoptosis in murine neuroblastoma by systemic delivery of transferrin-shielded siRNA polyplexes for downregulation of RNA. Oligonucleotides 18(2), 161-174 (2008).
    • (2008) Oligonucleotides , vol.18 , Issue.2 , pp. 161-174
    • Tietze, N.1    Pelisek, J.2    Philipp, A.3
  • 100
    • 30744457637 scopus 로고    scopus 로고
    • Tumor-targeting nanoimmunoliposome complex for short interfering RNA delivery
    • Pirollo KF, Zon G, Rait A et al. Tumor-targeting nanoimmunoliposome complex for short interfering RNA delivery. Hum. Gene Ther. 17(1), 117-124 (2006).
    • (2006) Hum. Gene Ther. , vol.17 , Issue.1 , pp. 117-124
    • Pirollo, K.F.1    Zon, G.2    Rait, A.3
  • 101
    • 0032077216 scopus 로고    scopus 로고
    • An RGD-oligolysine peptide: A prototype construct for integrin-mediated gene delivery
    • Harbottle RP, Cooper RG, Hart SL et al. An RGD-oligolysine peptide: a prototype construct for integrin-mediated gene delivery. Hum. Gene Ther. 9(7), 1037-1047 (1998).
    • (1998) Hum. Gene Ther. , vol.9 , Issue.7 , pp. 1037-1047
    • Harbottle, R.P.1    Cooper, R.G.2    Hart, S.L.3
  • 102
    • 33644827912 scopus 로고    scopus 로고
    • RGD-based strategies for selective delivery of therapeutics and imaging agents to the tumour vasculature
    • Temming K, Schiffelers RM, Molema G, Kok RJ. RGD-based strategies for selective delivery of therapeutics and imaging agents to the tumour vasculature. Drug Resist. Updat. 8(6), 381-402 (2005).
    • (2005) Drug Resist. Updat. , vol.8 , Issue.6 , pp. 381-402
    • Temming, K.1    Schiffelers, R.M.2    Molema, G.3    Kok, R.J.4
  • 103
    • 33845654382 scopus 로고    scopus 로고
    • Small interfering RNA delivery to the liver by intravenous administration of galactosylated cationic liposomes in mice
    • Sato A, Takagi M, Shimamoto A, Kawakami S, Hashida M. Small interfering RNA delivery to the liver by intravenous administration of galactosylated cationic liposomes in mice. Biomaterials 28(7), 1434-1442 (2007).
    • (2007) Biomaterials , vol.28 , Issue.7 , pp. 1434-1442
    • Sato, A.1    Takagi, M.2    Shimamoto, A.3    Kawakami, S.4    Hashida, M.5
  • 106
    • 28544444484 scopus 로고    scopus 로고
    • Phosphorothioate-stimulated uptake of short interfering RNA by human cells
    • Overhoff M, Sczakiel G. Phosphorothioate-stimulated uptake of short interfering RNA by human cells. EMBO Rep. 6(12), 1176-1181 (2005).
    • (2005) EMBO Rep. , vol.6 , Issue.12 , pp. 1176-1181
    • Overhoff, M.1    Sczakiel, G.2
  • 107
    • 64549083734 scopus 로고    scopus 로고
    • The therapeutic potential of LNA-modified siRNAs: Reduction of off-target effects by chemical modification of the siRNA sequence
    • Fluiter K, Mook OR, Baas F. The therapeutic potential of LNA-modified siRNAs: reduction of off-target effects by chemical modification of the siRNA sequence. Methods Mol. Biol. 487, 189-203 (2009).
    • (2009) Methods Mol. Biol. , vol.487 , pp. 189-203
    • Fluiter, K.1    Mook, O.R.2    Baas, F.3
  • 108
    • 67649865399 scopus 로고    scopus 로고
    • The effect of chemical modification and nanoparticle formulation on stability and biodistribution of siRNA in mice
    • Gao S, Dagnaes-Hansen F, Nielsen EJ et al. The effect of chemical modification and nanoparticle formulation on stability and biodistribution of siRNA in mice. Mol. Ther. 17(7), 1225-1233 (2009).
    • (2009) Mol. Ther. , vol.17 , Issue.7 , pp. 1225-1233
    • Gao, S.1    Dagnaes-Hansen, F.2    Nielsen, E.J.3
  • 109
    • 66249133061 scopus 로고    scopus 로고
    • Naked siLNA-mediated gene silencing of lung bronchoepithelium EGFP expression after intravenous administration
    • Glud SZ, Bramsen JB, Dagnaes-Hansen F et al. Naked siLNA-mediated gene silencing of lung bronchoepithelium EGFP expression after intravenous administration. Oligonucleotides 19(2), 163-168 (2009).
    • (2009) Oligonucleotides , vol.19 , Issue.2 , pp. 163-168
    • Glud, S.Z.1    Bramsen, J.B.2    Dagnaes-Hansen, F.3
  • 110
    • 42249093319 scopus 로고    scopus 로고
    • LNA-mediated microRNA silencing in non-human primates
    • Elmen J, Lindow M, Schutz S et al. LNA-mediated microRNA silencing in non-human primates. Nature 452(7189), 896-899 (2008).
    • (2008) Nature , vol.452 , Issue.7189 , pp. 896-899
    • Elmen, J.1    Lindow, M.2    Schutz, S.3
  • 111
    • 79953202455 scopus 로고    scopus 로고
    • Silencing of microRNA families by seed-targeting tiny LNAs
    • Obad S, Dos Santos CO, Petri A et al. Silencing of microRNA families by seed-targeting tiny LNAs. Nature Genet. 43(4), 371-378 (2011).
    • (2011) Nature Genet. , vol.43 , Issue.4 , pp. 371-378
    • Obad, S.1    Dos Santos, C.O.2    Petri, A.3
  • 112
    • 78649877710 scopus 로고    scopus 로고
    • Short locked nucleic acid antisense oligonucleotides potently reduce apolipoprotein B mRNA and serum cholesterol in mice and non-human primates
    • Straarup EM, Fisker N, Hedtjarn M et al. Short locked nucleic acid antisense oligonucleotides potently reduce apolipoprotein B mRNA and serum cholesterol in mice and non-human primates. Nucleic Acids Res. 38(20), 7100-7111 (2010).
    • (2010) Nucleic Acids Res. , vol.38 , Issue.20 , pp. 7100-7111
    • Straarup, E.M.1    Fisker, N.2    Hedtjarn, M.3
  • 113
    • 84883118140 scopus 로고    scopus 로고
    • Safety and efficacy of RNAi therapy for transthyretin amyloidosis
    • Coelho T, Adams D, Silva A et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N. Engl. J. Med. 369(9), 819-829 (2013).
    • (2013) N. Engl. J. Med. , vol.369 , Issue.9 , pp. 819-829
    • Coelho, T.1    Adams, D.2    Silva, A.3
  • 114
    • 84892741671 scopus 로고    scopus 로고
    • Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: A randomised, single-blind, placebo-controlled, Phase 1 trial
    • Fitzgerald K, Frank-Kamenetsky M, Shulga-Morskaya S et al. Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, Phase 1 trial. Lancet 383(9911), 60-68 (2014).
    • (2014) Lancet , vol.383 , Issue.9911 , pp. 60-68
    • Fitzgerald, K.1    Frank-Kamenetsky, M.2    Shulga-Morskaya, S.3
  • 115
    • 33748254700 scopus 로고    scopus 로고
    • MAGfect: A novel liposome formulation for MRI labelling and visualization of cells
    • Oliver M, Ahmad A, Kamaly N et al. MAGfect: a novel liposome formulation for MRI labelling and visualization of cells. Org. Biomol. Chem. 4(18), 3489-3497 (2006).
    • (2006) Org. Biomol. Chem. , vol.4 , Issue.18 , pp. 3489-3497
    • Oliver, M.1    Ahmad, A.2    Kamaly, N.3
  • 116
    • 84887632056 scopus 로고    scopus 로고
    • Lipid-based nanoparticles in cancer diagnosis and therapy
    • Miller AD. Lipid-based nanoparticles in cancer diagnosis and therapy. J. Drug Deliv. 2013, 165981 (2013).
    • (2013) J. Drug Deliv. , vol.2013 , pp. 165981
    • Miller, A.D.1
  • 117
    • 36649027868 scopus 로고    scopus 로고
    • Pulmonary delivery of nucleic acids
    • Birchall J. Pulmonary delivery of nucleic acids. Expert Opin. Drug Deliv. 4(6), 575-578 (2007).
    • (2007) Expert Opin. Drug Deliv. , vol.4 , Issue.6 , pp. 575-578
    • Birchall, J.1
  • 119
    • 34547645206 scopus 로고    scopus 로고
    • Blood-brain barrier transport of non-viral gene and RNAi therapeutics
    • Boado RJ. Blood-brain barrier transport of non-viral gene and RNAi therapeutics. Pharm. Res. 24(9), 1772-1787 (2007).
    • (2007) Pharm. Res. , vol.24 , Issue.9 , pp. 1772-1787
    • Boado, R.J.1
  • 120
    • 56749131859 scopus 로고    scopus 로고
    • Assessing siRNA pharmacodynamics in a luciferase-expressing mouse
    • Svensson RU, Shey MR, Ballas ZK et al. Assessing siRNA pharmacodynamics in a luciferase-expressing mouse. Mol. Ther. 16(12), 1995-2001 (2008).
    • (2008) Mol. Ther. , vol.16 , Issue.12 , pp. 1995-2001
    • Svensson, R.U.1    Shey, M.R.2    Ballas, Z.K.3
  • 122
    • 67649948599 scopus 로고    scopus 로고
    • MMP-9 gene silencing by a quantum dot-siRNA nanoplex delivery to maintain the integrity of the blood brain barrier
    • Bonoiu A, Mahajan SD, Ye L et al. MMP-9 gene silencing by a quantum dot-siRNA nanoplex delivery to maintain the integrity of the blood brain barrier. Brain Res. 1282, 142-155 (2009).
    • (2009) Brain Res. , vol.1282 , pp. 142-155
    • Bonoiu, A.1    Mahajan, S.D.2    Ye, L.3
  • 123
    • 58149088420 scopus 로고    scopus 로고
    • Quantum dot-carrier peptide conjugates suitable for imaging and delivery applications
    • Walther C, Meyer K, Rennert R, Neundorf I. Quantum dot-carrier peptide conjugates suitable for imaging and delivery applications. Bioconjug. Chem. 19(12), 2346-2356 (2008).
    • (2008) Bioconjug. Chem. , vol.19 , Issue.12 , pp. 2346-2356
    • Walther, C.1    Meyer, K.2    Rennert, R.3    Neundorf, I.4
  • 124
    • 37549064689 scopus 로고    scopus 로고
    • Cell-penetrating peptide for enhanced delivery of nucleic acids and drugs to ocular tissues including retina and cornea
    • Johnson LN, Cashman SM, Kumar-Singh R. Cell-penetrating peptide for enhanced delivery of nucleic acids and drugs to ocular tissues including retina and cornea. Mol. Ther. 16(1), 107-114 (2008).
    • (2008) Mol. Ther. , vol.16 , Issue.1 , pp. 107-114
    • Johnson, L.N.1    Cashman, S.M.2    Kumar-Singh, R.3
  • 125
    • 34848918210 scopus 로고    scopus 로고
    • Impact of tumor-specific targeting on the biodistribution and efficacy of siRNA nanoparticles measured by multimodality in vivo imaging
    • Bartlett DW, Su H, Hildebrandt IJ, Weber WA, Davis ME. Impact of tumor-specific targeting on the biodistribution and efficacy of siRNA nanoparticles measured by multimodality in vivo imaging. Proc. Natl Acad. Sci. USA 104(39), 15549-15554 (2007).
    • (2007) Proc. Natl Acad. Sci. USA , vol.104 , Issue.39 , pp. 15549-15554
    • Bartlett, D.W.1    Su, H.2    Hildebrandt, I.J.3    Weber, W.A.4    Davis, M.E.5
  • 126
    • 33847720312 scopus 로고    scopus 로고
    • In vivo imaging of siRNA delivery and silencing in tumors
    • Medarova Z, Pham W, Farrar C, Petkova V, Moore A. In vivo imaging of siRNA delivery and silencing in tumors. Nat. Med. 13(3), 372-377 (2007).
    • (2007) Nat. Med. , vol.13 , Issue.3 , pp. 372-377
    • Medarova, Z.1    Pham, W.2    Farrar, C.3    Petkova, V.4    Moore, A.5
  • 128
    • 47349091366 scopus 로고    scopus 로고
    • Proton-sponge coated quantum dots for siRNA delivery and intracellular imaging
    • Yezhelyev MV, Qi L, O'Regan RM, Nie S, Gao X. Proton-sponge coated quantum dots for siRNA delivery and intracellular imaging. J. Am. Chem. Soc. 130(28), 9006-9012 (2008).
    • (2008) J. Am. Chem. Soc. , vol.130 , Issue.28 , pp. 9006-9012
    • Yezhelyev, M.V.1    Qi, L.2    O'regan, R.M.3    Nie, S.4    Gao, X.5
  • 129
    • 59849091278 scopus 로고    scopus 로고
    • Peptide-mediated cellular delivery of oligonucleotide-based therapeutics in vitro: Quantitative evaluation of overall efficacy employing easy to handle reporter systems
    • Laufer SD, Restle T. Peptide-mediated cellular delivery of oligonucleotide-based therapeutics in vitro: quantitative evaluation of overall efficacy employing easy to handle reporter systems. Curr. Pharm. Des. 14(34), 3637-3655 (2008).
    • (2008) Curr. Pharm. Des. , vol.14 , Issue.34 , pp. 3637-3655
    • Laufer, S.D.1    Restle, T.2
  • 130
    • 35348861314 scopus 로고    scopus 로고
    • Cellular uptake and intracellular release are major obstacles to the therapeutic application of siRNA: Novel options by phosphorothioate-stimulated delivery
    • Mescalchin A, Detzer A, Wecke M, Overhoff M, Wunsche W, Sczakiel G. Cellular uptake and intracellular release are major obstacles to the therapeutic application of siRNA: novel options by phosphorothioate-stimulated delivery. Expert Opin. Biol. Ther. 7(10), 1531-1538 (2007).
    • (2007) Expert Opin. Biol. Ther. , vol.7 , Issue.10 , pp. 1531-1538
    • Mescalchin, A.1    Detzer, A.2    Wecke, M.3    Overhoff, M.4    Wunsche, W.5    Sczakiel, G.6
  • 131
    • 59849125915 scopus 로고    scopus 로고
    • Phosphorothioate-stimulated cellular uptake of siRNA: A cell culture model for mechanistic studies
    • Detzer A, Overhoff M, Mescalchin A, Rompf M, Sczakiel G. Phosphorothioate-stimulated cellular uptake of siRNA: a cell culture model for mechanistic studies. Curr. Pharm. Des. 14(34), 3666-3673 (2008).
    • (2008) Curr. Pharm. Des. , vol.14 , Issue.34 , pp. 3666-3673
    • Detzer, A.1    Overhoff, M.2    Mescalchin, A.3    Rompf, M.4    Sczakiel, G.5
  • 132
    • 62549161328 scopus 로고    scopus 로고
    • Increased RNAi is related to intracellular release of siRNA via a covalently attached signal peptide
    • Detzer A, Overhoff M, Wunsche W et al. Increased RNAi is related to intracellular release of siRNA via a covalently attached signal peptide. RNA 15(4), 627-636 (2009).
    • (2009) RNA , vol.15 , Issue.4 , pp. 627-636
    • Detzer, A.1    Overhoff, M.2    Wunsche, W.3
  • 133
    • 74049105646 scopus 로고    scopus 로고
    • Phosphorothioate-stimulated uptake of siRNA by mammalian cells: A novel route for delivery
    • Detzer A, Sczakiel G. Phosphorothioate-stimulated uptake of siRNA by mammalian cells: a novel route for delivery. Curr. Top. Med. Chem. 9(12), 1109-1116 (2009).
    • (2009) Curr. Top. Med. Chem. , vol.9 , Issue.12 , pp. 1109-1116
    • Detzer, A.1    Sczakiel, G.2
  • 134
    • 54249143980 scopus 로고    scopus 로고
    • Barriers to successful delivery of short interfering RNA after systemic administration
    • White PJ. Barriers to successful delivery of short interfering RNA after systemic administration. Clin. Exp. Pharmacol. Physiol. 35(11), 1371-1376 (2008).
    • (2008) Clin. Exp. Pharmacol. Physiol. , vol.35 , Issue.11 , pp. 1371-1376
    • White, P.J.1
  • 135
    • 37549032712 scopus 로고    scopus 로고
    • Breaking the bonds: Non-viral vectors become chemically dynamic
    • Wolff JA, Rozema DB. Breaking the bonds: non-viral vectors become chemically dynamic. Mol. Ther. 16(1), 8-15 (2008).
    • (2008) Mol. Ther. , vol.16 , Issue.1 , pp. 8-15
    • Wolff, J.A.1    Rozema, D.B.2
  • 136
    • 34548484580 scopus 로고    scopus 로고
    • Delivery of short interfering RNA using endosomolytic cell-penetrating peptides
    • Lundberg P, El-Andaloussi S, Sutlu T, Johansson H, Langel U. Delivery of short interfering RNA using endosomolytic cell-penetrating peptides. FASEB J. 21(11), 2664-2671 (2007).
    • (2007) FASEB J. , vol.21 , Issue.11 , pp. 2664-2671
    • Lundberg, P.1    El-Andaloussi, S.2    Sutlu, T.3    Johansson, H.4    Langel, U.5
  • 137
    • 24944577514 scopus 로고    scopus 로고
    • Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids
    • Heyes J, Palmer L, Bremner K, Maclachlan I. Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids. J. Control. Release 107(2), 276-287 (2005).
    • (2005) J. Control. Release , vol.107 , Issue.2 , pp. 276-287
    • Heyes, J.1    Palmer, L.2    Bremner, K.3    Maclachlan, I.4
  • 138
    • 34848901058 scopus 로고    scopus 로고
    • Specific subcellular localization of siRNAs delivered by lipoplex in MCF-7 breast cancer cells
    • Lavigne C, Thierry AR. Specific subcellular localization of siRNAs delivered by lipoplex in MCF-7 breast cancer cells. Biochimie 89(10), 1245-1251 (2007).
    • (2007) Biochimie , vol.89 , Issue.10 , pp. 1245-1251
    • Lavigne, C.1    Thierry, A.R.2
  • 139
    • 70849097385 scopus 로고    scopus 로고
    • Monitoring the disassembly of siRNA polyplexes in serum is crucial for predicting their biological efficacy
    • Buyens K, Meyer M, Wagner E, Demeester J, De Smedt SC, Sanders NN. Monitoring the disassembly of siRNA polyplexes in serum is crucial for predicting their biological efficacy. J. Control. Release 141(1), 38-41 (2010).
    • (2010) J. Control. Release , vol.141 , Issue.1 , pp. 38-41
    • Buyens, K.1    Meyer, M.2    Wagner, E.3    Demeester, J.4    De Smedt, S.C.5    Sanders, N.N.6
  • 140
    • 30944456368 scopus 로고    scopus 로고
    • Navigare necessere est. Improved navigation would help to solve two crucial problems in modern drug therapy: Toxicity and precise delivery
    • Kraljevic S, Pavelic K. Navigare necessere est. Improved navigation would help to solve two crucial problems in modern drug therapy: toxicity and precise delivery. EMBO Rep. 6(8), 695-700 (2005).
    • (2005) EMBO Rep. , vol.6 , Issue.8 , pp. 695-700
    • Kraljevic, S.1    Pavelic, K.2
  • 141
    • 24644504130 scopus 로고    scopus 로고
    • Toxicogenomics of cationic lipid-based vectors for gene therapy: Impact of microarray technology
    • Omidi Y, Barar J, Akhtar S. Toxicogenomics of cationic lipid-based vectors for gene therapy: impact of microarray technology. Curr. Drug Deliv. 2(4), 429-441 (2005).
    • (2005) Curr. Drug Deliv. , vol.2 , Issue.4 , pp. 429-441
    • Omidi, Y.1    Barar, J.2    Akhtar, S.3
  • 142
    • 57149137351 scopus 로고    scopus 로고
    • Systemic delivery of DNA or siRNA mediated by linear polyethylenimine (L-PEI) does not induce an inflammatory response
    • Bonnet ME, Erbacher P, Bolcato-Bellemin AL. Systemic delivery of DNA or siRNA mediated by linear polyethylenimine (L-PEI) does not induce an inflammatory response. Pharm. Res. 25(12), 2972-2982 (2008).
    • (2008) Pharm. Res. , vol.25 , Issue.12 , pp. 2972-2982
    • Bonnet, M.E.1    Erbacher, P.2    Bolcato-Bellemin, A.L.3
  • 144
    • 85157183564 scopus 로고    scopus 로고
    • PCT/US2009/036223
    • Bumcrot D: PCT/US2009/036223 (2009).
    • (2009)
    • Bumcrot, D.1


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