메뉴 건너뛰기




Volumn 23, Issue 9, 2015, Pages 1456-1464

Sequence-engineered mRNA Without Chemical Nucleoside Modifications Enables an Effective Protein Therapy in Large Animals

Author keywords

[No Author keywords available]

Indexed keywords

ERYTHROPOIETIN; LIPID NANOPARTICLE; MESSENGER RNA; NANOPARTICLE; NUCLEOSIDE; RECOMBINANT ERYTHROPOIETIN; UNCLASSIFIED DRUG; LIPID;

EID: 84940720241     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.103     Document Type: Article
Times cited : (393)

References (45)
  • 1
    • 0015230553 scopus 로고
    • Use of frog eggs and oocytes for the study of messenger RNA and its translation in living cells
    • Gurdon, JB, Lane, CD, Woodland, HR and Marbaix, G (1971). Use of frog eggs and oocytes for the study of messenger RNA and its translation in living cells. Nature 233: 177-182.
    • (1971) Nature , vol.233 , pp. 177-182
    • Gurdon, J.B.1    Lane, C.D.2    Woodland, H.R.3    Marbaix, G.4
  • 2
    • 0015450033 scopus 로고
    • Translation of encephalomyocarditis viral RNA in oocytes of Xenopus laevis
    • Laskey, RA, Gurdon, JB and Crawford, LV (1972). Translation of encephalomyocarditis viral RNA in oocytes of Xenopus laevis. Proc Natl Acad Sci USA 69: 3665-3669.
    • (1972) Proc Natl Acad Sci USA , vol.69 , pp. 3665-3669
    • Laskey, R.A.1    Gurdon, J.B.2    Crawford, L.V.3
  • 3
    • 0029839058 scopus 로고    scopus 로고
    • Dendritic cells pulsed with RNA are potent antigen-presenting cells in vitro and in vivo
    • Boczkowski, D, Nair, SK, Snyder, D and Gilboa, E (1996). Dendritic cells pulsed with RNA are potent antigen-presenting cells in vitro and in vivo. J Exp Med 184: 465-472.
    • (1996) J Exp Med , vol.184 , pp. 465-472
    • Boczkowski, D.1    Nair, S.K.2    Snyder, D.3    Gilboa, E.4
  • 6
    • 0026522713 scopus 로고
    • Reversal of diabetes insipidus in Brattleboro rats: Intrahypothalamic injection of vasopressin mRNA
    • Jirikowski, GF, Sanna, PP, Maciejewski-Lenoir, D and Bloom, FE (1992). Reversal of diabetes insipidus in Brattleboro rats: intrahypothalamic injection of vasopressin mRNA. Science 255: 996-998.
    • (1992) Science , vol.255 , pp. 996-998
    • Jirikowski, G.F.1    Sanna, P.P.2    Maciejewski-Lenoir, D.3    Bloom, F.E.4
  • 7
    • 0035909372 scopus 로고    scopus 로고
    • Recognition of double-stranded RNA and activation of NF-kappaB by Toll-like receptor 3
    • Alexopoulou, L, Holt, AC, Medzhitov, R and Flavell, RA (2001). Recognition of double-stranded RNA and activation of NF-kappaB by Toll-like receptor 3. Nature 413: 732-738.
    • (2001) Nature , vol.413 , pp. 732-738
    • Alexopoulou, L.1    Holt, A.C.2    Medzhitov, R.3    Flavell, R.A.4
  • 8
    • 1542317550 scopus 로고    scopus 로고
    • Innate antiviral responses by means of TLR7-mediated recognition of single-stranded RNA
    • Diebold, SS, Kaisho, T, Hemmi, H, Akira, S and Reis e Sousa, C (2004). Innate antiviral responses by means of TLR7-mediated recognition of single-stranded RNA. Science 303: 1529-1531.
    • (2004) Science , vol.303 , pp. 1529-1531
    • Diebold, S.S.1    Kaisho, T.2    Hemmi, H.3    Akira, S.4    Reis Sousa, E.C.5
  • 9
    • 1542317578 scopus 로고    scopus 로고
    • Species-specific recognition of single-stranded RNA via toll-like receptor 7 and 8
    • Heil, F, Hemmi, H, Hochrein, H, Ampenberger, F, Kirschning, C, Akira, S et al. (2004). Species-specific recognition of single-stranded RNA via toll-like receptor 7 and 8. Science 303: 1526-1529.
    • (2004) Science , vol.303 , pp. 1526-1529
    • Heil, F.1    Hemmi, H.2    Hochrein, H.3    Ampenberger, F.4    Kirschning, C.5    Akira, S.6
  • 11
    • 33646342149 scopus 로고    scopus 로고
    • Differential roles of MDA5 and RIG-I helicases in the recognition of RNA viruses
    • Kato, H, Takeuchi, O, Sato, S, Yoneyama, M, Yamamoto, M, Matsui, K et al. (2006). Differential roles of MDA5 and RIG-I helicases in the recognition of RNA viruses. Nature 441: 101-105.
    • (2006) Nature , vol.441 , pp. 101-105
    • Kato, H.1    Takeuchi, O.2    Sato, S.3    Yoneyama, M.4    Yamamoto, M.5    Matsui, K.6
  • 12
    • 33750984771 scopus 로고    scopus 로고
    • RIG-I-mediated antiviral responses to single-stranded RNA bearing 5'-phosphates
    • Pichlmair, A, Schulz, O, Tan, CP, Naslund, TI, Liljestrom, P, Weber, F et al. (2006). RIG-I-mediated antiviral responses to single-stranded RNA bearing 5'-phosphates. Science 314: 997-1001.
    • (2006) Science , vol.314 , pp. 997-1001
    • Pichlmair, A.1    Schulz, O.2    Tan, C.P.3    Naslund, T.I.4    Liljestrom, P.5    Weber, F.6
  • 13
    • 75749140581 scopus 로고    scopus 로고
    • RIG-I detects viral genomic RNA during negative-strand RNA virus infection
    • Rehwinkel, J, Tan, CP, Goubau, D, Schulz, O, Pichlmair, A, Bier, K et al. (2010). RIG-I detects viral genomic RNA during negative-strand RNA virus infection. Cell 140: 397-408.
    • (2010) Cell , vol.140 , pp. 397-408
    • Rehwinkel, J.1    Tan, C.P.2    Goubau, D.3    Schulz, O.4    Pichlmair, A.5    Bier, K.6
  • 15
    • 79955630363 scopus 로고    scopus 로고
    • Intradermal vaccinations with RNA coding for TAA generate CD8+ and CD4+ immune responses and induce clinical benefit in vaccinated patients
    • Rittig, SM, Haentschel, M, Weimer, KJ, Heine, A, Muller, MR, Brugger, W et al. (2011). Intradermal vaccinations with RNA coding for TAA generate CD8+ and CD4+ immune responses and induce clinical benefit in vaccinated patients. Mol Ther 19: 990-999.
    • (2011) Mol Ther , vol.19 , pp. 990-999
    • Rittig, S.M.1    Haentschel, M.2    Weimer, K.J.3    Heine, A.4    Muller, M.R.5    Brugger, W.6
  • 16
    • 33750248345 scopus 로고    scopus 로고
    • Therapeutic anti-tumor immunity triggered by injections of immunostimulating single-stranded RNA
    • Scheel, B, Aulwurm, S, Probst, J, Stitz, L, Hoerr, I, Rammensee, HG et al. (2006). Therapeutic anti-tumor immunity triggered by injections of immunostimulating single-stranded RNA. Eur J Immunol 36: 2807-2816.
    • (2006) Eur J Immunol , vol.36 , pp. 2807-2816
    • Scheel, B.1    Aulwurm, S.2    Probst, J.3    Stitz, L.4    Hoerr, I.5    Rammensee, H.G.6
  • 17
    • 44949195901 scopus 로고    scopus 로고
    • Results of the first phase I/II clinical vaccination trial with direct injection of mRNA
    • Weide, B, Carralot, JP, Reese, A, Scheel, B, Eigentler, TK, Hoerr, I et al. (2008). Results of the first phase I/II clinical vaccination trial with direct injection of mRNA. J Immunother 31: 180-188.
    • (2008) J Immunother , vol.31 , pp. 180-188
    • Weide, B.1    Carralot, J.P.2    Reese, A.3    Scheel, B.4    Eigentler, T.K.5    Hoerr, I.6
  • 18
    • 67651152679 scopus 로고    scopus 로고
    • Direct injection of protamine-protected mRNA: Results of a phase. Vaccination trial in metastatic melanoma patients
    • Weide, B, Pascolo, S, Scheel, B, Derhovanessian, E, Pflugfelder, A, Eigentler, TK et al. (2009). Direct injection of protamine-protected mRNA: results of a phase . vaccination trial in metastatic melanoma patients. J Immunother 32: 498-507.
    • (2009) J Immunother , vol.32 , pp. 498-507
    • Weide, B.1    Pascolo, S.2    Scheel, B.3    Derhovanessian, E.4    Pflugfelder, A.5    Eigentler, T.K.6
  • 19
    • 78650640612 scopus 로고    scopus 로고
    • Messenger RNA-based vaccines with dual activity induce balanced TLR-7 dependent adaptive immune responses and provide antitumor activity
    • Fotin-Mleczek, M, Duchardt, KM, Lorenz, C, Pfeiffer, R, Ojki?-Zrna, S, Probst, J et al. (2011). Messenger RNA-based vaccines with dual activity induce balanced TLR-7 dependent adaptive immune responses and provide antitumor activity. J Immunother 34: 1-15.
    • (2011) J Immunother , vol.34 , pp. 1-15
    • Fotin-Mleczek, M.1    Duchardt, K.M.2    Lorenz, C.3    Pfeiffer, R.4    Ojki-Zrna, S.5    Probst, J.6
  • 20
    • 23844464444 scopus 로고    scopus 로고
    • Suppression of RNA recognition by Toll-like receptors: The impact of nucleoside modification and the evolutionary origin of RNA
    • Kariko, K, Buckstein, M, Ni, H and Weissman, D (2005). Suppression of RNA recognition by Toll-like receptors: The impact of nucleoside modification and the evolutionary origin of RNA. Immunity 23: 165-175.
    • (2005) Immunity , vol.23 , pp. 165-175
    • Kariko, K.1    Buckstein, M.2    Ni, H.3    Weissman, D.4
  • 21
    • 82255164455 scopus 로고    scopus 로고
    • Nucleoside modifications in RNA limit activation of 2'-5'-oligoadenylate synthetase and increase resistance to cleavage by RNase L
    • Anderson, BR, Muramatsu, H, Jha, BK, Silverman, RH, Weissman, D and Kariko, K (2011). Nucleoside modifications in RNA limit activation of 2'-5'-oligoadenylate synthetase and increase resistance to cleavage by RNase L. Nucleic Acids Res 39: 9329-9338.
    • (2011) Nucleic Acids Res , vol.39 , pp. 9329-9338
    • Anderson, B.R.1    Muramatsu, H.2    Jha, B.K.3    Silverman, R.H.4    Weissman, D.5    Kariko, K.6
  • 23
    • 54949112814 scopus 로고    scopus 로고
    • Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability
    • Kariko, K, Muramatsu, H, Welsh, FA, Ludwig, J, Kato, H, Akira, S et al. (2008). Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability. Mol Ther 16: 1833-1840.
    • (2008) Mol Ther , vol.16 , pp. 1833-1840
    • Kariko, K.1    Muramatsu, H.2    Welsh, F.A.3    Ludwig, J.4    Kato, H.5    Akira, S.6
  • 24
    • 77958536106 scopus 로고    scopus 로고
    • Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA
    • Warren, L, Manos, PD, Ahfeldt, T, Loh, YH, Li, H, Lau, F et al. (2010). Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA. Cell Stem Cell 7: 618-630.
    • (2010) Cell Stem Cell , vol.7 , pp. 618-630
    • Warren, L.1    Manos, P.D.2    Ahfeldt, T.3    Loh, Y.H.4    Li, H.5    Lau, F.6
  • 25
    • 84885676364 scopus 로고    scopus 로고
    • Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction
    • Zangi, L, Lui, KO, von Gise, A, Ma, Q, Ebina, W, Ptaszek, LM et al. (2013). Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction. Nat Biotechnol 31: 898-907.
    • (2013) Nat Biotechnol , vol.31 , pp. 898-907
    • Zangi, L.1    Lui, K.O.2    Von Gise, A.3    Ma, Q.4    Ebina, W.5    Ptaszek, L.M.6
  • 27
    • 84860539841 scopus 로고    scopus 로고
    • Increased erythropoiesis in mice injected with submicrogram quantities of pseudouridine-containing mRNA encoding erythropoietin
    • Kariko, K, Muramatsu, H, Keller, JM and Weissman, D (2012). Increased erythropoiesis in mice injected with submicrogram quantities of pseudouridine-containing mRNA encoding erythropoietin. Mol Ther 20: 948-953.
    • (2012) Mol Ther , vol.20 , pp. 948-953
    • Kariko, K.1    Muramatsu, H.2    Keller, J.M.3    Weissman, D.4
  • 28
    • 84865079107 scopus 로고    scopus 로고
    • Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo
    • Jayaraman, M, Ansell, SM, Mui, BL, Tam, YK, Chen, J, Du, X et al. (2012). Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo. Angew Chem Int Ed Engl 51: 8529-8533.
    • (2012) Angew Chem Int Ed Engl , vol.51 , pp. 8529-8533
    • Jayaraman, M.1    Ansell, S.M.2    Mui, B.L.3    Tam, Y.K.4    Chen, J.5    Du, X.6
  • 29
    • 0027170483 scopus 로고
    • Induction of virus-specific cytotoxic T lymphocytes in vivo by liposome-entrapped mRNA
    • Martinon, F, Krishnan, S, Lenzen, G, Magne, R, Gomard, E, Guillet, JG et al. (1993). Induction of virus-specific cytotoxic T lymphocytes in vivo by liposome-entrapped mRNA. Eur J Immunol 23: 1719-1722.
    • (1993) Eur J Immunol , vol.23 , pp. 1719-1722
    • Martinon, F.1    Krishnan, S.2    Lenzen, G.3    Magne, R.4    Gomard, E.5    Guillet, J.G.6
  • 30
    • 0033989908 scopus 로고    scopus 로고
    • In vivo application of RNA leads to induction of specific cytotoxic T lymphocytes and antibodies
    • Hoerr, I, Obst, R, Rammensee, HG and Jung, G (2000). In vivo application of RNA leads to induction of specific cytotoxic T lymphocytes and antibodies. Eur J Immunol 30: 1-7.
    • (2000) Eur J Immunol , vol.30 , pp. 1-7
    • Hoerr, I.1    Obst, R.2    Rammensee, H.G.3    Jung, G.4
  • 31
    • 84870869040 scopus 로고    scopus 로고
    • Protective efficacy of in vitro synthesized, specific mRNA vaccines against influenza A virus infection
    • Petsch, B, Schnee, M, Vogel, AB, Lange, E, Hoffmann, B, Voss, D et al. (2012). Protective efficacy of in vitro synthesized, specific mRNA vaccines against influenza A virus infection. Nat Biotechnol 30: 1210-1216.
    • (2012) Nat Biotechnol , vol.30 , pp. 1210-1216
    • Petsch, B.1    Schnee, M.2    Vogel, A.B.3    Lange, E.4    Hoffmann, B.5    Voss, D.6
  • 32
    • 84919872967 scopus 로고    scopus 로고
    • MTOR inhibition improves antitumor effects of vaccination with antigen-encoding RNA
    • Diken, M, Kreiter, S, Vascotto, F, Selmi, A, Attig, S, Diekmann, J et al. (2013). mTOR inhibition improves antitumor effects of vaccination with antigen-encoding RNA. Cancer Immunol Res 1: 386-392.
    • (2013) Cancer Immunol Res , vol.1 , pp. 386-392
    • Diken, M.1    Kreiter, S.2    Vascotto, F.3    Selmi, A.4    Attig, S.5    Diekmann, J.6
  • 33
    • 80053347071 scopus 로고    scopus 로고
    • FLT3 ligand enhances the cancer therapeutic potency of naked RNA vaccines
    • Kreiter, S, Diken, M, Selmi, A, Diekmann, J, Attig, S, Husemann, Y et al. (2011). FLT3 ligand enhances the cancer therapeutic potency of naked RNA vaccines. Cancer Res 71: 6132-6142.
    • (2011) Cancer Res , vol.71 , pp. 6132-6142
    • Kreiter, S.1    Diken, M.2    Selmi, A.3    Diekmann, J.4    Attig, S.5    Husemann, Y.6
  • 34
    • 78549283854 scopus 로고    scopus 로고
    • Intranodal vaccination with naked antigen-encoding RNA elicits potent prophylactic and therapeutic antitumoral immunity
    • Kreiter, S, Selmi, A, Diken, M, Koslowski, M, Britten, CM, Huber, C et al. (2010). Intranodal vaccination with naked antigen-encoding RNA elicits potent prophylactic and therapeutic antitumoral immunity. Cancer Res 70: 9031-9040.
    • (2010) Cancer Res , vol.70 , pp. 9031-9040
    • Kreiter, S.1    Selmi, A.2    Diken, M.3    Koslowski, M.4    Britten, C.M.5    Huber, C.6
  • 36
    • 3442896311 scopus 로고    scopus 로고
    • Messenger RNA-based vaccines
    • Pascolo, S (2004). Messenger RNA-based vaccines. Expert Opin Biol Ther 4: 1285-1294.
    • (2004) Expert Opin Biol Ther , vol.4 , pp. 1285-1294
    • Pascolo, S.1
  • 37
    • 34447633941 scopus 로고    scopus 로고
    • Spontaneous cellular uptake of exogenous messenger RNA in vivo is nucleic acidspecific, saturable and ion dependent
    • Probst, J, Weide, B, Scheel, B, Pichler, BJ, Hoerr, I, Rammensee, HG et al. (2007). Spontaneous cellular uptake of exogenous messenger RNA in vivo is nucleic acidspecific, saturable and ion dependent. Gene Ther 14: 1175-1180.
    • (2007) Gene Ther , vol.14 , pp. 1175-1180
    • Probst, J.1    Weide, B.2    Scheel, B.3    Pichler, B.J.4    Hoerr, I.5    Rammensee, H.G.6
  • 38
    • 82255194361 scopus 로고    scopus 로고
    • Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA
    • Kariko, K, Muramatsu, H, Ludwig, J and Weissman, D (2011). Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA. Nucleic Acids Res 39: e142.
    • (2011) Nucleic Acids Res , vol.39 , pp. e142
    • Kariko, K.1    Muramatsu, H.2    Ludwig, J.3    Weissman, D.4
  • 39
    • 0031684212 scopus 로고    scopus 로고
    • Pharmacokinetics and pharmacodynamics of recombinant human erythropoietin after single and multiple subcutaneous doses to healthy subjects
    • Cheung, WK, Goon, BL, Guilfoyle, MC and Wacholtz, MC (1998). Pharmacokinetics and pharmacodynamics of recombinant human erythropoietin after single and multiple subcutaneous doses to healthy subjects. Clin Pharmacol Ther 64: 412-423.
    • (1998) Clin Pharmacol Ther , vol.64 , pp. 412-423
    • Cheung, W.K.1    Goon, B.L.2    Guilfoyle, M.C.3    Wacholtz, M.C.4
  • 40
    • 49149120764 scopus 로고    scopus 로고
    • Pharmacokinetic and pharmacodynamic profiles of extended dosing of epoetin alfa in anemic patients who have chronic kidney disease and are not on dialysis
    • McGowan, T, Vaccaro, NM, Beaver, JS, Massarella, J and Wolfson, M (2008). Pharmacokinetic and pharmacodynamic profiles of extended dosing of epoetin alfa in anemic patients who have chronic kidney disease and are not on dialysis. Clin J Am Soc Nephrol 3: 1006-1014.
    • (2008) Clin J Am Soc Nephrol , vol.3 , pp. 1006-1014
    • McGowan, T.1    Vaccaro, N.M.2    Beaver, J.S.3    Massarella, J.4    Wolfson, M.5
  • 41
    • 84905506792 scopus 로고    scopus 로고
    • Enhancing siRNA delivery by employing lipid nanoparticles
    • Hope, MJ (2014). Enhancing siRNA delivery by employing lipid nanoparticles. Therapeutic Delivery 5: 663-673.
    • (2014) Therapeutic Delivery , vol.5 , pp. 663-673
    • Hope, M.J.1
  • 43
    • 84881292568 scopus 로고    scopus 로고
    • Biodegradable lipids enabling rapidly eliminated lipid nanoparticles for systemic delivery of RNAi therapeutics
    • Maier, MA, Jayaraman, M, Matsuda, S, Liu, J, Barros, S, Querbes, W et al. (2013). Biodegradable lipids enabling rapidly eliminated lipid nanoparticles for systemic delivery of RNAi therapeutics. Mol Ther 21: 1570-1578.
    • (2013) Mol Ther , vol.21 , pp. 1570-1578
    • Maier, M.A.1    Jayaraman, M.2    Matsuda, S.3    Liu, J.4    Barros, S.5    Querbes, W.6
  • 44
  • 45
    • 77954243341 scopus 로고    scopus 로고
    • Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms
    • Akinc, A, Querbes, W, De, S, Qin, J, Frank-Kamenetsky, M, Jayaprakash, KN et al. (2010). Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms. Mol Ther 18: 1357-1364.
    • (2010) Mol Ther , vol.18 , pp. 1357-1364
    • Akinc, A.1    Querbes, W.2    De, S.3    Qin, J.4    Frank-Kamenetsky, M.5    Jayaprakash, K.N.6


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.