메뉴 건너뛰기




Volumn 246, Issue , 2017, Pages 60-70

Self-aggregating 1.8 kDa polyethylenimines with dissolution switch at endosomal acidic pH are delivery carriers for plasmid DNA, mRNA, siRNA and exon-skipping oligonucleotides

Author keywords

Delivery system; Exon skipping; Gene delivery; mdx mice; mRNA delivery; Muscular dystrophy; Phosphorothioate RNA; Polyethylenimine (PEI); siRNA delivery; Transfection

Indexed keywords

AMIDES; AMINES; AROMATIC COMPOUNDS; CELL PROLIFERATION; CHEMICAL MODIFICATION; DNA; DRUG PRODUCTS; DRUG THERAPY; EFFICIENCY; GENE TRANSFER; GENES; MAMMALS; MOLECULAR WEIGHT; POLYETHYLENE GLYCOLS; POLYETHYLENE OXIDES; RNA;

EID: 85006931080     PISSN: 01683659     EISSN: 18734995     Source Type: Journal    
DOI: 10.1016/j.jconrel.2016.12.005     Document Type: Article
Times cited : (62)

References (62)
  • 2
    • 0034019945 scopus 로고    scopus 로고
    • Cationic polymer based gene delivery systems
    • [2] De Smedt, S.C., Demeester, J., Hennink, W.E., Cationic polymer based gene delivery systems. Pharm. Res., 17, 2000, 113-26.
    • (2000) Pharm. Res. , vol.17 , pp. 113-26
    • De Smedt, S.C.1    Demeester, J.2    Hennink, W.E.3
  • 3
    • 84942046481 scopus 로고    scopus 로고
    • Nucleic acid therapeutics using polyplexes: a journey of 50 years (and beyond)
    • [3] Lachelt, U., Wagner, E., Nucleic acid therapeutics using polyplexes: a journey of 50 years (and beyond). Chem. Rev. 115 (2015), 11043–11078.
    • (2015) Chem. Rev. , vol.115 , pp. 11043-11078
    • Lachelt, U.1    Wagner, E.2
  • 4
    • 84858642577 scopus 로고    scopus 로고
    • Rational design of smart supramolecular assemblies for gene delivery: chemical challenges in the creation of artificial viruses
    • [4] Miyata, K., Nishiyama, N., Kataoka, K., Rational design of smart supramolecular assemblies for gene delivery: chemical challenges in the creation of artificial viruses. Chem. Soc. Rev. 41 (2012), 2562–2574.
    • (2012) Chem. Soc. Rev. , vol.41 , pp. 2562-2574
    • Miyata, K.1    Nishiyama, N.2    Kataoka, K.3
  • 5
    • 84857693358 scopus 로고    scopus 로고
    • Molecular dynamics simulations for complexation of DNA with 2 kDa PEI reveal profound effect of PEI architecture on complexation
    • [5] Sun, C., Tang, T., Uludag, H., Molecular dynamics simulations for complexation of DNA with 2 kDa PEI reveal profound effect of PEI architecture on complexation. J. Phys. Chem. B 116 (2012), 2405–2413.
    • (2012) J. Phys. Chem. B , vol.116 , pp. 2405-2413
    • Sun, C.1    Tang, T.2    Uludag, H.3
  • 6
    • 84959553586 scopus 로고    scopus 로고
    • Viral vectors for gene therapy and gene modification approaches
    • [6] Merten, O.-W., Gaillet, B., Viral vectors for gene therapy and gene modification approaches. Biochem. Eng. J. 108 (2016), 98–115.
    • (2016) Biochem. Eng. J. , vol.108 , pp. 98-115
    • Merten, O.-W.1    Gaillet, B.2
  • 7
    • 84866450859 scopus 로고    scopus 로고
    • Oncolytic adenoviruses in anticancer therapy: current status and prospects
    • [7] Svyatchenko, V.A., Tarasova, M.V., Netesov, S.V., Chumakov, M., Oncolytic adenoviruses in anticancer therapy: current status and prospects. Mol. Biol. 46 (2012), 496–507.
    • (2012) Mol. Biol. , vol.46 , pp. 496-507
    • Svyatchenko, V.A.1    Tarasova, M.V.2    Netesov, S.V.3    Chumakov, M.4
  • 9
    • 84918578407 scopus 로고    scopus 로고
    • Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing
    • [9] Nair, J.K., Willoughby, J.L., Chan, A., Charisse, K., Alam, M.R., Wang, Q., et al. Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing. J. Am. Chem. Soc. 136 (2014), 16958–16961.
    • (2014) J. Am. Chem. Soc. , vol.136 , pp. 16958-16961
    • Nair, J.K.1    Willoughby, J.L.2    Chan, A.3    Charisse, K.4    Alam, M.R.5    Wang, Q.6
  • 10
    • 61649104319 scopus 로고    scopus 로고
    • Combinatorial evaluation of cations, pH-sensitive and hydrophobic moieties for polymeric vector design
    • [10] Wong, S.Y., Sood, N., Putnam, D., Combinatorial evaluation of cations, pH-sensitive and hydrophobic moieties for polymeric vector design. Mol. Ther. 17 (2009), 480–490.
    • (2009) Mol. Ther. , vol.17 , pp. 480-490
    • Wong, S.Y.1    Sood, N.2    Putnam, D.3
  • 12
    • 80053073179 scopus 로고    scopus 로고
    • Nucleic acid carriers based on precise polymer conjugates
    • [12] Troiber, C., Wagner, E., Nucleic acid carriers based on precise polymer conjugates. Bioconjug. Chem. 22 (2011), 1737–1752.
    • (2011) Bioconjug. Chem. , vol.22 , pp. 1737-1752
    • Troiber, C.1    Wagner, E.2
  • 14
    • 31544468071 scopus 로고    scopus 로고
    • Physiochemical properties of low and high molecular weight poly(ethylene glycol)-grafted poly(ethylene imine) copolymers and their complexes with oligonucleotides
    • [14] Glodde, M., Sirsi, S.R., Lutz, G.J., Physiochemical properties of low and high molecular weight poly(ethylene glycol)-grafted poly(ethylene imine) copolymers and their complexes with oligonucleotides. Biomacromolecules 7 (2006), 347–356.
    • (2006) Biomacromolecules , vol.7 , pp. 347-356
    • Glodde, M.1    Sirsi, S.R.2    Lutz, G.J.3
  • 15
    • 72149101228 scopus 로고    scopus 로고
    • The induction of tumor apoptosis in B16 melanoma following STAT3 siRNA delivery with a lipid-substituted polyethylenimine
    • [15] Alshamsan, A., Hamdy, S., Samuel, J., El-Kadi, A.O., Lavasanifar, A., Uludag, H., The induction of tumor apoptosis in B16 melanoma following STAT3 siRNA delivery with a lipid-substituted polyethylenimine. Biomaterials 31 (2010), 1420–1428.
    • (2010) Biomaterials , vol.31 , pp. 1420-1428
    • Alshamsan, A.1    Hamdy, S.2    Samuel, J.3    El-Kadi, A.O.4    Lavasanifar, A.5    Uludag, H.6
  • 16
    • 84879136327 scopus 로고    scopus 로고
    • Arginine-rich hydrophobic polyethylenimine: potent agent with simple components for nucleic acid delivery
    • [16] Parhiz, H., Hashemi, M., Hatefi, A., Shier, W.T., Amel Farzad, S., Ramezani, M., Arginine-rich hydrophobic polyethylenimine: potent agent with simple components for nucleic acid delivery. Int. J. Biol. Macromol. 60 (2013), 18–27.
    • (2013) Int. J. Biol. Macromol. , vol.60 , pp. 18-27
    • Parhiz, H.1    Hashemi, M.2    Hatefi, A.3    Shier, W.T.4    Amel Farzad, S.5    Ramezani, M.6
  • 17
    • 84885096635 scopus 로고    scopus 로고
    • Stabilization of cancer-specific gene carrier via hydrophobic interaction for a clear-cut response to cancer signaling
    • [17] Kim, C.W., Toita, R., Kang, J.H., Li, K., Lee, E.K., Zhao, G.X., et al. Stabilization of cancer-specific gene carrier via hydrophobic interaction for a clear-cut response to cancer signaling. J. Control. Release 170 (2013), 469–476.
    • (2013) J. Control. Release , vol.170 , pp. 469-476
    • Kim, C.W.1    Toita, R.2    Kang, J.H.3    Li, K.4    Lee, E.K.5    Zhao, G.X.6
  • 18
    • 84907937451 scopus 로고    scopus 로고
    • Hydrophobic polyalanine modified hyperbranched polyethylenimine as high efficient pDNA and siRNA carrier
    • [18] Guo, Z., Tian, H., Lin, L., Chen, J., He, C., Tang, Z., et al. Hydrophobic polyalanine modified hyperbranched polyethylenimine as high efficient pDNA and siRNA carrier. Macromol. Biosci. 14 (2014), 1406–1414.
    • (2014) Macromol. Biosci. , vol.14 , pp. 1406-1414
    • Guo, Z.1    Tian, H.2    Lin, L.3    Chen, J.4    He, C.5    Tang, Z.6
  • 19
    • 0029160083 scopus 로고
    • A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine
    • [19] Boussif, O., Lezoualc'h, F., Zanta, M.A., Mergny, M.D., Scherman, D., Demeneix, B., et al. 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), 7297–7301.
    • (1995) Proc. Natl. Acad. Sci. U. S. A. , vol.92 , pp. 7297-7301
    • Boussif, O.1    Lezoualc'h, F.2    Zanta, M.A.3    Mergny, M.D.4    Scherman, D.5    Demeneix, B.6
  • 20
    • 0037437033 scopus 로고    scopus 로고
    • Low-molecular-weight polyethylenimine as a non-viral vector for DNA delivery: comparison of physicochemical properties, transfection efficiency and in vivo distribution with high-molecular-weight polyethylenimine
    • [20] Kunath, K., von Harpe, A., Fischer, D., Peterson, H., Bickel, U., Voigt, K., et al. Low-molecular-weight polyethylenimine as a non-viral vector for DNA delivery: comparison of physicochemical properties, transfection efficiency and in vivo distribution with high-molecular-weight polyethylenimine. J. Control. Release 89 (2003), 113–125.
    • (2003) J. Control. Release , vol.89 , pp. 113-125
    • Kunath, K.1    von Harpe, A.2    Fischer, D.3    Peterson, H.4    Bickel, U.5    Voigt, K.6
  • 22
    • 68249122805 scopus 로고    scopus 로고
    • Nonviral siRNA delivery to the lung: investigation of PEG-PEI polyplexes and their in vivo performance
    • [22] Merkel, O.M., Beyerle, A., Librizzi, D., Pfestroff, A., Behr, T.M., Sproat, B., et al. Nonviral siRNA delivery to the lung: investigation of PEG-PEI polyplexes and their in vivo performance. Mol. Pharm. 6 (2009), 1246–1260.
    • (2009) Mol. Pharm. , vol.6 , pp. 1246-1260
    • Merkel, O.M.1    Beyerle, A.2    Librizzi, D.3    Pfestroff, A.4    Behr, T.M.5    Sproat, B.6
  • 23
    • 77953303124 scopus 로고    scopus 로고
    • Toxicity pathway focused gene expression profiling of PEI-based polymers for pulmonary applications
    • [23] Beyerle, A., Irmler, M., Beckers, J., Kissel, T., Stoeger, T., Toxicity pathway focused gene expression profiling of PEI-based polymers for pulmonary applications. Mol. Pharm. 7 (2010), 727–737.
    • (2010) Mol. Pharm. , vol.7 , pp. 727-737
    • Beyerle, A.1    Irmler, M.2    Beckers, J.3    Kissel, T.4    Stoeger, T.5
  • 24
    • 65349175903 scopus 로고    scopus 로고
    • Comparison of toxicity and transfection efficiency of amphiphilic block copolymers and polycationic polymers in striated muscles
    • [24] Roques, C., Fattal, E., Fromes, Y., Comparison of toxicity and transfection efficiency of amphiphilic block copolymers and polycationic polymers in striated muscles. J. Gene Med. 11 (2009), 240–249.
    • (2009) J. Gene Med. , vol.11 , pp. 240-249
    • Roques, C.1    Fattal, E.2    Fromes, Y.3
  • 25
    • 61849103493 scopus 로고    scopus 로고
    • In vivo pharmacokinetics, tissue distribution and underlying mechanisms of various PEI(-PEG)/siRNA complexes
    • [25] Malek, A., Merkel, O., Fink, L., Czubayko, F., Kissel, T., Aigner, A., In vivo pharmacokinetics, tissue distribution and underlying mechanisms of various PEI(-PEG)/siRNA complexes. Toxicol. Appl. Pharmacol. 236 (2009), 97–108.
    • (2009) Toxicol. Appl. Pharmacol. , vol.236 , pp. 97-108
    • Malek, A.1    Merkel, O.2    Fink, L.3    Czubayko, F.4    Kissel, T.5    Aigner, A.6
  • 26
    • 84873954134 scopus 로고    scopus 로고
    • Specific effects of PEGylation on gene delivery efficacy of polyethylenimine: interplay between PEG substitution and N/P ratio
    • [26] Fitzsimmons, R.E., Uludag, H., Specific effects of PEGylation on gene delivery efficacy of polyethylenimine: interplay between PEG substitution and N/P ratio. Acta Biomater. 8 (2012), 3941–3955.
    • (2012) Acta Biomater. , vol.8 , pp. 3941-3955
    • Fitzsimmons, R.E.1    Uludag, H.2
  • 27
    • 84953439973 scopus 로고    scopus 로고
    • Efficient and tumor targeted siRNA delivery by polyethylenimine-graft-polycaprolactone-block-poly(ethylene glycol)-folate (PEI-PCL-PEG-Fol)
    • [27] Liu, L., Zheng, M., Librizzi, D., Renette, T., Merkel, O.M., Kissel, T., Efficient and tumor targeted siRNA delivery by polyethylenimine-graft-polycaprolactone-block-poly(ethylene glycol)-folate (PEI-PCL-PEG-Fol). Mol. Pharm. 13 (2016), 134–143.
    • (2016) Mol. Pharm. , vol.13 , pp. 134-143
    • Liu, L.1    Zheng, M.2    Librizzi, D.3    Renette, T.4    Merkel, O.M.5    Kissel, T.6
  • 28
    • 0035131596 scopus 로고    scopus 로고
    • Polyethylenimine/DNA complexes shielded by transferrin target gene expression to tumors after systemic application
    • [28] Kircheis, R., Wightman, L., Schreiber, A., Robitza, B., Rossler, V., Kursa, M., et al. Polyethylenimine/DNA complexes shielded by transferrin target gene expression to tumors after systemic application. Gene Ther. 8 (2001), 28–40.
    • (2001) Gene Ther. , vol.8 , pp. 28-40
    • Kircheis, R.1    Wightman, L.2    Schreiber, A.3    Robitza, B.4    Rossler, V.5    Kursa, M.6
  • 29
    • 75449101757 scopus 로고    scopus 로고
    • DNA/polyethyleneimine/hyaluronic acid small complex particles and tumor suppression in mice
    • [29] Ito, T., Yoshihara, C., Hamada, K., Koyama, Y., DNA/polyethyleneimine/hyaluronic acid small complex particles and tumor suppression in mice. Biomaterials 31 (2010), 2912–2918.
    • (2010) Biomaterials , vol.31 , pp. 2912-2918
    • Ito, T.1    Yoshihara, C.2    Hamada, K.3    Koyama, Y.4
  • 30
    • 84870309549 scopus 로고    scopus 로고
    • Polyethyleneimine/DNA polyplexes with reduction-sensitive hyaluronic acid derivatives shielding for targeted gene delivery
    • [30] He, Y., Cheng, G., Xie, L., Nie, Y., He, B., Gu, Z., Polyethyleneimine/DNA polyplexes with reduction-sensitive hyaluronic acid derivatives shielding for targeted gene delivery. Biomaterials 34 (2013), 1235–1245.
    • (2013) Biomaterials , vol.34 , pp. 1235-1245
    • He, Y.1    Cheng, G.2    Xie, L.3    Nie, Y.4    He, B.5    Gu, Z.6
  • 31
    • 4143087473 scopus 로고    scopus 로고
    • Effect of poly(ethylene imine) molecular weight and pegylation on organ distribution and pharmacokinetics of polyplexes with oligodeoxynucleotides in mice
    • [31] Fischer, D., Osburg, B., Petersen, H., Kissel, T., Bickel, U., Effect of poly(ethylene imine) molecular weight and pegylation on organ distribution and pharmacokinetics of polyplexes with oligodeoxynucleotides in mice. Drug Metab. Dispos. 32 (2004), 983–992.
    • (2004) Drug Metab. Dispos. , vol.32 , pp. 983-992
    • Fischer, D.1    Osburg, B.2    Petersen, H.3    Kissel, T.4    Bickel, U.5
  • 32
    • 17644401347 scopus 로고    scopus 로고
    • Cross-linked small polyethylenimines: while still nontoxic, deliver DNA efficiently to mammalian cells in vitro and in vivo
    • [32] Thomas, M., Ge, Q., JJ, L., Chen, J.Z., Klibanov, A.M., Cross-linked small polyethylenimines: while still nontoxic, deliver DNA efficiently to mammalian cells in vitro and in vivo. Pharm. Res. 22 (2005), 373–380.
    • (2005) Pharm. Res. , vol.22 , pp. 373-380
    • Thomas, M.1    Ge, Q.2    JJ, L.3    Chen, J.Z.4    Klibanov, A.M.5
  • 33
    • 0032892476 scopus 로고    scopus 로고
    • Size matters: molecular weight affects the efficiency of poly(ethylenimine) as a gene delivery vehicle
    • [33] Godbey, W.T., KK, W., Mikos, A.G., Size matters: molecular weight affects the efficiency of poly(ethylenimine) as a gene delivery vehicle. J. Biomed. Mater. Res. 45 (1999), 268–275.
    • (1999) J. Biomed. Mater. Res. , vol.45 , pp. 268-275
    • Godbey, W.T.1    KK, W.2    Mikos, A.G.3
  • 34
    • 58549114410 scopus 로고    scopus 로고
    • Controlled cytoplasmic and nuclear localization of plasmid DNA and siRNA by differentially tailored polyethylenimine
    • [34] Shim, M.S., Kwon, Y.J., Controlled cytoplasmic and nuclear localization of plasmid DNA and siRNA by differentially tailored polyethylenimine. J. Control. Release 133 (2009), 206–213.
    • (2009) J. Control. Release , vol.133 , pp. 206-213
    • Shim, M.S.1    Kwon, Y.J.2
  • 35
    • 84881026547 scopus 로고    scopus 로고
    • Hydrophobic modification of low molecular weight polyethylenimine for improved gene transfection
    • [35] Teo, P.Y., Yang, C., Hedrick, J.L., Engler, A.C., Coady, D.J., Ghaem-Maghami, S., et al. Hydrophobic modification of low molecular weight polyethylenimine for improved gene transfection. Biomaterials 34 (2013), 7971–7979.
    • (2013) Biomaterials , vol.34 , pp. 7971-7979
    • Teo, P.Y.1    Yang, C.2    Hedrick, J.L.3    Engler, A.C.4    Coady, D.J.5    Ghaem-Maghami, S.6
  • 36
    • 78651085655 scopus 로고    scopus 로고
    • A comparative evaluation of disulfide-linked and hydrophobically-modified PEI for plasmid delivery
    • [36] Remant Bahadur, K.C., Uludag, H., A comparative evaluation of disulfide-linked and hydrophobically-modified PEI for plasmid delivery. J. Biomater. Sci. Polym. Ed. 22 (2011), 873–892.
    • (2011) J. Biomater. Sci. Polym. Ed. , vol.22 , pp. 873-892
    • Remant Bahadur, K.C.1    Uludag, H.2
  • 37
    • 84861981048 scopus 로고    scopus 로고
    • Structure-activity relationships of siRNA carriers based on sequence-defined oligo (ethane amino) amides
    • [37] Frohlich, T., Edinger, D., Klager, R., Troiber, C., Salcher, E., Badgujar, N., et al. Structure-activity relationships of siRNA carriers based on sequence-defined oligo (ethane amino) amides. J. Control. Release 160 (2012), 532–541.
    • (2012) J. Control. Release , vol.160 , pp. 532-541
    • Frohlich, T.1    Edinger, D.2    Klager, R.3    Troiber, C.4    Salcher, E.5    Badgujar, N.6
  • 38
    • 58149400636 scopus 로고    scopus 로고
    • Self-assembling polyethylenimine derivatives mediate efficient siRNA delivery in mammalian cells
    • [38] Creusat, G., Zuber, G., Self-assembling polyethylenimine derivatives mediate efficient siRNA delivery in mammalian cells. Chembiochem 9 (2008), 2787–2789.
    • (2008) Chembiochem , vol.9 , pp. 2787-2789
    • Creusat, G.1    Zuber, G.2
  • 39
    • 77952555362 scopus 로고    scopus 로고
    • Proton sponge trick for pH-sensitive disassembly of polyethylenimine-based siRNA delivery systems
    • [39] Creusat, G., Rinaldi, A.S., Weiss, E., Elbaghdadi, R., Remy, J.S., Mulherkar, R., et al. Proton sponge trick for pH-sensitive disassembly of polyethylenimine-based siRNA delivery systems. Bioconjug. Chem. 21 (2010), 994–1002.
    • (2010) Bioconjug. Chem. , vol.21 , pp. 994-1002
    • Creusat, G.1    Rinaldi, A.S.2    Weiss, E.3    Elbaghdadi, R.4    Remy, J.S.5    Mulherkar, R.6
  • 40
    • 84897858887 scopus 로고    scopus 로고
    • Quantitative measurement of delivery and gene silencing activities of siRNA polyplexes containing pyridylthiourea-grafted polyethylenimines
    • [40] Pinel, S., Aman, E., Erblang, F., Dietrich, J., Frisch, B., Sirman, J., et al. Quantitative measurement of delivery and gene silencing activities of siRNA polyplexes containing pyridylthiourea-grafted polyethylenimines. J. Control. Release 182 (2014), 1–12.
    • (2014) J. Control. Release , vol.182 , pp. 1-12
    • Pinel, S.1    Aman, E.2    Erblang, F.3    Dietrich, J.4    Frisch, B.5    Sirman, J.6
  • 41
    • 84877608256 scopus 로고    scopus 로고
    • MiR-20a regulates ASK1 expression and TLR4-dependent cytokine release in rheumatoid fibroblast-like synoviocytes
    • [41] Philippe, L., Alsaleh, G., Pichot, A., Ostermann, E., Zuber, G., Frisch, B., et al. MiR-20a regulates ASK1 expression and TLR4-dependent cytokine release in rheumatoid fibroblast-like synoviocytes. Ann. Rheum. Dis. 72 (2013), 1071–1079.
    • (2013) Ann. Rheum. Dis. , vol.72 , pp. 1071-1079
    • Philippe, L.1    Alsaleh, G.2    Pichot, A.3    Ostermann, E.4    Zuber, G.5    Frisch, B.6
  • 42
    • 84857369666 scopus 로고    scopus 로고
    • Pyridylthiourea-grafted polyethylenimine offers an effective assistance to siRNA-mediated gene silencing in vitro and in vivo
    • [42] Creusat, G., Thomann, J.S., Maglott, A., Pons, B., Dontenwill, M., Guerin, E., et al. Pyridylthiourea-grafted polyethylenimine offers an effective assistance to siRNA-mediated gene silencing in vitro and in vivo. J. Control. Release 157 (2012), 418–426.
    • (2012) J. Control. Release , vol.157 , pp. 418-426
    • Creusat, G.1    Thomann, J.S.2    Maglott, A.3    Pons, B.4    Dontenwill, M.5    Guerin, E.6
  • 43
    • 65649085603 scopus 로고    scopus 로고
    • Confirming the RNAi-mediated mechanism of action of siRNA-based cancer therapeutics in mice
    • [43] Judge, A.D., Robbins, M., Tavakoli, I., Levi, J., Hu, L., Fronda, A., et al. Confirming the RNAi-mediated mechanism of action of siRNA-based cancer therapeutics in mice. J. Clin. Invest. 119 (2009), 661–673.
    • (2009) J. Clin. Invest. , vol.119 , pp. 661-673
    • Judge, A.D.1    Robbins, M.2    Tavakoli, I.3    Levi, J.4    Hu, L.5    Fronda, A.6
  • 44
    • 0032485884 scopus 로고    scopus 로고
    • Up-regulation of luciferase gene expression with antisense oligonucleotides: implications and applications in functional assay development
    • [44] Kang, S.H., Cho, M.J., Kole, R., Up-regulation of luciferase gene expression with antisense oligonucleotides: implications and applications in functional assay development. Biochemistry 37 (1998), 6235–6239.
    • (1998) Biochemistry , vol.37 , pp. 6235-6239
    • Kang, S.H.1    Cho, M.J.2    Kole, R.3
  • 45
    • 33845986379 scopus 로고    scopus 로고
    • The influence of antisense oligonucleotide length on dystrophin exon skipping
    • [45] Harding, P.L., Fall, A.M., Honeyman, K., Fletcher, S., Wilton, S.D., The influence of antisense oligonucleotide length on dystrophin exon skipping. Mol. Ther. 15 (2007), 157–166.
    • (2007) Mol. Ther. , vol.15 , pp. 157-166
    • Harding, P.L.1    Fall, A.M.2    Honeyman, K.3    Fletcher, S.4    Wilton, S.D.5
  • 46
    • 84872083766 scopus 로고    scopus 로고
    • Oligobenzylethylenimine enriches linear polyethylenimine with a pH-sensitive membrane-disruptive property and leads to enhanced gene delivery activity
    • [46] Chandrashekhar, C., Pons, B., Muller, C.D., Tounsi, N., Mulherkar, R., Zuber, G., Oligobenzylethylenimine enriches linear polyethylenimine with a pH-sensitive membrane-disruptive property and leads to enhanced gene delivery activity. Acta Biomater. 9 (2013), 4985–4993.
    • (2013) Acta Biomater. , vol.9 , pp. 4985-4993
    • Chandrashekhar, C.1    Pons, B.2    Muller, C.D.3    Tounsi, N.4    Mulherkar, R.5    Zuber, G.6
  • 47
    • 70350077962 scopus 로고    scopus 로고
    • Synthetic Viruslike particles for targeted gene delivery to alpha(v)beta(3) integrin-presenting endothelial cells
    • [47] Zuber, G., Dontenwill, M., Behr, J.P., Synthetic Viruslike particles for targeted gene delivery to alpha(v)beta(3) integrin-presenting endothelial cells. Mol. Pharm. 6 (2009), 1544–1552.
    • (2009) Mol. Pharm. , vol.6 , pp. 1544-1552
    • Zuber, G.1    Dontenwill, M.2    Behr, J.P.3
  • 48
    • 0034602124 scopus 로고    scopus 로고
    • Characterization of commercially available and synthesized polyethylenimines for gene delivery
    • [48] von Harpe, A., Petersen, H., Li, Y., Kissel, T., Characterization of commercially available and synthesized polyethylenimines for gene delivery. J. Control. Release 69 (2000), 309–322.
    • (2000) J. Control. Release , vol.69 , pp. 309-322
    • von Harpe, A.1    Petersen, H.2    Li, Y.3    Kissel, T.4
  • 49
    • 0242414440 scopus 로고    scopus 로고
    • Chloride accumulation and swelling in endosomes enhances DNA transfer by polyamine-DNA polyplexes
    • [49] Sonawane, N.D., Szoka, F.C. Jr., Verkman, A.S., Chloride accumulation and swelling in endosomes enhances DNA transfer by polyamine-DNA polyplexes. J. Biol. Chem. 278 (2003), 44826–44831.
    • (2003) J. Biol. Chem. , vol.278 , pp. 44826-44831
    • Sonawane, N.D.1    Szoka, F.C.2    Verkman, A.S.3
  • 51
    • 78049475611 scopus 로고    scopus 로고
    • mRNA transfection of cervical carcinoma and mesenchymal stem cells mediated by cationic carriers
    • [51] Rejman, J., Tavernier, G., Bavarsad, N., Demeester, J., De Smedt, S.C., mRNA transfection of cervical carcinoma and mesenchymal stem cells mediated by cationic carriers. J. Control. Release 147 (2010), 385–391.
    • (2010) J. Control. Release , vol.147 , pp. 385-391
    • Rejman, J.1    Tavernier, G.2    Bavarsad, N.3    Demeester, J.4    De Smedt, S.C.5
  • 52
    • 0141497030 scopus 로고    scopus 로고
    • Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing
    • [52] Sazani, P., Kole, R., Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing. J. Clin. Invest. 112 (2003), 481–486.
    • (2003) J. Clin. Invest. , vol.112 , pp. 481-486
    • Sazani, P.1    Kole, R.2
  • 53
    • 84937251741 scopus 로고    scopus 로고
    • Exon skipping therapy for Duchenne muscular dystrophy
    • [53] Kole, R., Krieg, A.M., Exon skipping therapy for Duchenne muscular dystrophy. Adv. Drug Deliv. Rev. 87 (2015), 104–107.
    • (2015) Adv. Drug Deliv. Rev. , vol.87 , pp. 104-107
    • Kole, R.1    Krieg, A.M.2
  • 54
    • 77952729474 scopus 로고    scopus 로고
    • Formulation and delivery of splice-correction antisense oligonucleotides by amino acid modified polyethylenimine
    • [54] Zaghloul, E.M., Viola, J.R., Zuber, G., Smith, C.I.E., Lundin, K.E., Formulation and delivery of splice-correction antisense oligonucleotides by amino acid modified polyethylenimine. Mol. Pharm. 7 (2010), 652–663.
    • (2010) Mol. Pharm. , vol.7 , pp. 652-663
    • Zaghloul, E.M.1    Viola, J.R.2    Zuber, G.3    Smith, C.I.E.4    Lundin, K.E.5
  • 55
    • 85006842632 scopus 로고    scopus 로고
    • FDA issues complete response letter for KyndrisaTM for Duchenne muscular dystrophy amenable to exon 51 skipping
    • [55] FDA issues complete response letter for KyndrisaTM for Duchenne muscular dystrophy amenable to exon 51 skipping. http://investors.bmrn.com/releasedetail.cfm?releaseid=9503092016.
  • 56
    • 85006957392 scopus 로고    scopus 로고
    • FDA grants accelerated approval to first drug for Duchenne muscular dystrophy
    • [56] FDA grants accelerated approval to first drug for Duchenne muscular dystrophy. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm521263.htm2016.
  • 57
    • 84891739039 scopus 로고    scopus 로고
    • Pluronic-PEI copolymers enhance exon-skipping of 2′-O-methyl phosphorothioate oligonucleotide in cell culture and dystrophic mdx mice
    • [57] Wang, M., Wu, B., Lu, P., Tucker, J.D., Milazi, S., Shah, S.N., et al. Pluronic-PEI copolymers enhance exon-skipping of 2′-O-methyl phosphorothioate oligonucleotide in cell culture and dystrophic mdx mice. Gene Ther. 21 (2014), 52–59.
    • (2014) Gene Ther. , vol.21 , pp. 52-59
    • Wang, M.1    Wu, B.2    Lu, P.3    Tucker, J.D.4    Milazi, S.5    Shah, S.N.6
  • 58
    • 4344693568 scopus 로고    scopus 로고
    • Targeted exon skipping in transgenic hDMD mice: a model for direct preclinical screening of human-specific antisense oligonucleotides
    • [58] Bremmer-Bout, M., Aartsma-Rus, A., de Meijer, E.J., Kaman, W.E., Janson, A.A., Vossen, R.H., et al. Targeted exon skipping in transgenic hDMD mice: a model for direct preclinical screening of human-specific antisense oligonucleotides. Mol. Ther. 10 (2004), 232–240.
    • (2004) Mol. Ther. , vol.10 , pp. 232-240
    • Bremmer-Bout, M.1    Aartsma-Rus, A.2    de Meijer, E.J.3    Kaman, W.E.4    Janson, A.A.5    Vossen, R.H.6
  • 59
    • 0037344590 scopus 로고    scopus 로고
    • Microbubble ultrasound improves the efficiency of gene transduction in skeletal muscle in vivo with reduced tissue damage
    • [59] QL, L., Liang, H.D., Partridge, T., Blomley, M.J., Microbubble ultrasound improves the efficiency of gene transduction in skeletal muscle in vivo with reduced tissue damage. Gene Ther. 10 (2003), 396–405.
    • (2003) Gene Ther. , vol.10 , pp. 396-405
    • QL, L.1    Liang, H.D.2    Partridge, T.3    Blomley, M.J.4
  • 60
    • 33745289016 scopus 로고    scopus 로고
    • Induction of dystrophin expression by exon skipping in mdx mice following intramuscular injection of antisense oligonucleotides complexed with PEG-PEI copolymers
    • [60] Williams, J.H., Sirsi, S.R., Latta, D.R., Lutz, G.J., Induction of dystrophin expression by exon skipping in mdx mice following intramuscular injection of antisense oligonucleotides complexed with PEG-PEI copolymers. Mol. Ther. 14 (2006), 88–96.
    • (2006) Mol. Ther. , vol.14 , pp. 88-96
    • Williams, J.H.1    Sirsi, S.R.2    Latta, D.R.3    Lutz, G.J.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.