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SPECIAL FOCUS: RNA Vaccines - Review

mRNA transcript therapy

References

  • Wolff JA, Malone RW, Williams P, et al. Direct gene transfer into mouse muscle in vivo. Science 1990;247(4949 Pt 1):1465-8
  • Jirikowski GF, Sanna PP, Maciejewski-Lenoir D, Bloom FE. Reversal of diabetes insipidus in Brattleboro rats: intrahypothalamic injection of vasopressin mRNA. Science 1992;255(5047):996-8
  • Bernal JA. RNA-based tools for nuclear reprogramming and lineage-conversion: towards clinical applications. J Cardiovasc Transl Res 2013;6(6):956-68
  • Cannon G, Weissman D. RNA based vaccines. DNA Cell Biol 2002;21(12):953-61
  • Kreiter S, Diken M, Selmi A, et al. Tumor vaccination using messenger RNA: prospects of a future therapy. Curr Opin Immunol 2011;23(3):399-406
  • Kuhn AN, Beibetaert T, Simon P, et al. mRNA as a versatile tool for exogenous protein expression. Curr Gene Ther 2012;12(5):347-61
  • Pascolo S. Vaccination with messenger RNA (mRNA). Handbook of experimental pharmacology 2008(183):221-35
  • Hacein-Bey-Abina S, von Kalle C, Schmidt M, et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003;348(3):255-6
  • Hacein-Bey-Abina S, Von Kalle C, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003;302(5644):415-19
  • Kapeli K, Yeo GW. Genome-wide approaches to dissect the roles of RNA binding proteins in translational control: implications for neurological diseases. Frontiers in neuroscience 2012;6:144
  • Lodish HF. Translational control of protein synthesis: the early years. J Biol Chem 2012;287(43):36528-35
  • Morgens DW. The protein invasion: a broad review on the origin of the translational system. J Mol Evol 2013;77(4):185-96
  • Roy B, Jacobson A. The intimate relationships of mRNA decay and translation. Trends Genet 2013;29(12):691-9
  • Anderson BR, Muramatsu H, Jha BK, et al. Nucleoside modifications in RNA limit activation of 2’-5’-oligoadenylate synthetase and increase resistance to cleavage by RNase L. Nucleic Acids Res 2011;39(21):9329-38
  • Kariko K, Muramatsu H, Welsh FA, et al. Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability. Mol Ther 2008;16(11):1833-40
  • Kormann MS, Hasenpusch G, Aneja MK, et al. Expression of therapeutic proteins after delivery of chemically modified mRNA in mice. Nat Biotechnol 2011;29(2):154-7
  • Wang Y, Su HH, Yang Y, et al. Systemic delivery of modified mRNA encoding herpes simplex virus 1 thymidine kinase for targeted cancer gene therapy. Mol Ther 2013;21(2):358-67
  • Hornung V, Ellegast J, Kim S, et al. 5’-Triphosphate RNA is the ligand for RIG-I. Science 2006;314(5801):994-7
  • Pichlmair A, Schulz O, Tan CP, et al. RIG-I-mediated antiviral responses to single-stranded RNA bearing 5’-phosphates. Science 2006;314(5801):997-1001
  • Anderson BR, Muramatsu H, Nallagatla SR, et al. Incorporation of pseudouridine into mRNA enhances translation by diminishing PKR activation. Nucleic Acids Res 2010;38(17):5884-92
  • Nallagatla SR, Bevilacqua PC. Nucleoside modifications modulate activation of the protein kinase PKR in an RNA structure-specific manner. RNA 2008;14(6):1201-13
  • Kariko K, Muramatsu H, Ludwig J, Weissman D. Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA. Nucleic Acids Res 2011;39(21):e142
  • Holtkamp S, Kreiter S, Selmi A, et al. Modification of antigen-encoding RNA increases stability, translational efficacy, and T-cell stimulatory capacity of dendritic cells. Blood 2006;108(13):4009-17
  • Kuhn AN, Diken M, Kreiter S, et al. Phosphorothioate cap analogs increase stability and translational efficiency of RNA vaccines in immature dendritic cells and induce superior immune responses in vivo. Gene Ther 2010;17(8):961-71
  • Dimitrov DS. Therapeutic proteins. Methods in molecular biology 2012;899:1-26
  • Leader B, Baca QJ, Golan DE. Protein therapeutics: a summary and pharmacological classification. Nat Rev Drug discovery 2008;7(1):21-39
  • Lorenz C, Fotin-Mleczek M, Roth G, et al. Protein expression from exogenous mRNA: uptake by receptor-mediated endocytosis and trafficking via the lysosomal pathway. RNA Biol 2011;8(4):627-36
  • Van Tendeloo VF, Ponsaerts P, Lardon F, et al. Highly efficient gene delivery by mRNA electroporation in human hematopoietic cells: superiority to lipofection and passive pulsing of mRNA and to electroporation of plasmid cDNA for tumor antigen loading of dendritic cells. Blood 2001;98(1):49-56
  • Chen AK, Behlke MA, Tsourkas A. Efficient cytosolic delivery of molecular beacon conjugates and flow cytometric analysis of target RNA. Nucleic Acids Res 2008;36(12):e69
  • Besse F, Ephrussi A. Translational control of localized mRNAs: restricting protein synthesis in space and time. Nat Rev Molecular cell biology 2008;9(12):971-80
  • Zubiaga AM, Belasco JG, Greenberg ME. The nonamer UUAUUUAUU is the key AU-rich sequence motif that mediates mRNA degradation. Mol Cell Biol 1995;15(4):2219-30
  • Meyer S, Temme C, Wahle E. Messenger RNA turnover in eukaryotes: pathways and enzymes. Crit Rev Biochem Mol Biol 2004;39(4):197-216
  • Rittig SM, Haentschel M, Weimer KJ, et al. Intradermal vaccinations with RNA coding for TAA generate CD8+ and CD4+ immune responses and induce clinical benefit in vaccinated patients. Mol Ther 2011;19(5):990-9
  • Weide B, Carralot JP, Reese A, et al. Results of the first phase I/II clinical vaccination trial with direct injection of mRNA. J Immunother 2008;31(2):180-8
  • Weide B, Pascolo S, Scheel B, et al. Direct injection of protamine-protected mRNA: results of a phase 1/2 vaccination trial in metastatic melanoma patients. J Immunother 2009;32(5):498-507
  • Trial of RNActive®-Derived Cancer Vaccine and Local Radiation in in Stage IV Non Small Cell Lung Cancer (NSCLC). Available from: https://clinicaltrials.gov/ct2/results?term=NCT01915524&Search=Search
  • Trial of RNActive®-Derived Prostate Cancer Vaccine in Metastatic Castrate-refractory Prostate Cancer. Available from: https://clinicaltrials.gov/ct2/results?term=NCT01817738&Search=Search
  • Kreiter S, Selmi A, Diken M, et al. Intranodal vaccination with naked antigen-encoding RNA elicits potent prophylactic and therapeutic antitumoral immunity. Cancer Res 2010;70(22):9031-40
  • Van Lint S, Goyvaerts C, Maenhout S, et al. Preclinical evaluation of TriMix and antigen mRNA-based antitumor therapy. Cancer Res 2012;72(7):1661-71
  • RBL001/RBL002 Phase I Clinical Trial. Available from: https://clinicaltrials.gov/ct2/results?term=NCT01684241&Search=Search
  • Kariko K, Muramatsu H, Keller JM, Weissman D. Increased erythropoiesis in mice injected with submicrogram quantities of pseudouridine-containing mRNA encoding erythropoietin. Mol Ther 2012;20(5):948-53
  • Morrissey DV, Lockridge JA, Shaw L, et al. Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs. Nat Biotechnol 2005;23(8):1002-7
  • Zimmermann TS, Lee AC, Akinc A, et al. RNAi-mediated gene silencing in non-human primates. Nature 2006;441(7089):111-14
  • Brower V. RNA interference advances to early-stage clinical trials. J Natl Cancer Inst 2010;102(19):1459-61
  • Davis ME, Zuckerman JE, Choi CH, et al. Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature 2010;464(7291):1067-70
  • Shi B, Keough E, Matter A, et al. Biodistribution of small interfering RNA at the organ and cellular levels after lipid nanoparticle-mediated delivery. The journal of histochemistry and cytochemistry : official journal of the Histochemistry Society 2011;59(8):727-40
  • Ziemniak M, Strenkowska M, Kowalska J, Jemielity J. Potential therapeutic applications of RNA cap analogs. Future medicinal chemistry 2013;5(10):1141-72
  • Li Y, Kiledjian M. Regulation of mRNA decapping. Wiley interdisciplinary reviews. RNA 2010;1(2):253-65
  • Ensinger MJ, Martin SA, Paoletti E, Moss B. Modification of the 5’-terminus of mRNA by soluble guanylyl and methyl transferases from vaccinia virus. Proc Natl Acad Sci USA 1975;72(7):2525-9
  • Martin SA, Paoletti E, Moss B. Purification of mRNA guanylyltransferase and mRNA (guanine-7-) methyltransferase from vaccinia virions. J Biol Chem 1975;250(24):9322-9
  • Malone RW, Felgner PL, Verma IM. Cationic liposome-mediated RNA transfection. Proc Natl Acad Sci USA 1989;86(16):6077-81
  • Pasquinelli AE, Dahlberg JE, Lund E. Reverse 5’ caps in RNAs made in vitro by phage RNA polymerases. RNA 1995;1(9):957-67
  • Jemielity J, Fowler T, Zuberek J, et al. Novel “anti-reverse” cap analogs with superior translational properties. RNA 2003;9(9):1108-22
  • Stepinski J, Waddell C, Stolarski R, et al. Synthesis and properties of mRNAs containing the novel “anti-reverse” cap analogs 7-methyl(3’-O-methyl)GpppG and 7-methyl (3’-deoxy)GpppG. RNA 2001;7(10):1486-95
  • Bontkes HJ, Kramer D, Ruizendaal JJ, et al. Dendritic cells transfected with interleukin-12 and tumor-associated antigen messenger RNA induce high avidity cytotoxic T cells. Gene Ther 2007;14(4):366-75
  • Mockey M, Goncalves C, Dupuy FP, et al. mRNA transfection of dendritic cells: synergistic effect of ARCA mRNA capping with Poly(A) chains in cis and in trans for a high protein expression level. Biochem Biophys Res Commun 2006;340(4):1062-8
  • Rabinovich PM, Komarovskaya ME, Ye ZJ, et al. Synthetic messenger RNA as a tool for gene therapy. Hum Gene Ther 2006;17(10):1027-35
  • Grudzien-Nogalska E, Jemielity J, Kowalska J, et al. Phosphorothioate cap analogs stabilize mRNA and increase translational efficiency in mammalian cells. RNA 2007;13(10):1745-55
  • Kowalska J, Lewdorowicz M, Zuberek J, et al. Synthesis and characterization of mRNA cap analogs containing phosphorothioate substitutions that bind tightly to eIF4E and are resistant to the decapping pyrophosphatase DcpS. RNA 2008;14(6):1119-31
  • Jia J, Yao P, Arif A, Fox PL. Regulation and dysregulation of 3’UTR-mediated translational control. Curr Opin Genet Dev 2013;23(1):29-34
  • Perez-Ortin JE, Alepuz P, Chavez S, Choder M. Eukaryotic mRNA decay: methodologies, pathways, and links to other stages of gene expression. J Mol Biol 2013;425(20):3750-75
  • Somers J, Poyry T, Willis AE. A perspective on mammalian upstream open reading frame function. Int J Biochem Cell Biol 2013;45(8):1690-700
  • Szostak E, Gebauer F. Translational control by 3’-UTR-binding proteins. Briefings in functional genomics 2013;12(1):58-65
  • Ross J, Sullivan TD. Half-lives of beta and gamma globin messenger RNAs and of protein synthetic capacity in cultured human reticulocytes. Blood 1985;66(5):1149-54
  • Yu J, Russell JE. Structural and functional analysis of an mRNP complex that mediates the high stability of human beta-globin mRNA. Mol Cell Biol 2001;21(17):5879-88
  • Benteyn D, Anguille S, Van Lint S, et al. Design of an Optimized Wilms’ Tumor 1 (WT1) mRNA Construct for Enhanced WT1 Expression and Improved Immunogenicity In Vitro and In Vivo. Mol Ther Nucleic acids 2013;2:e134
  • Chen CY, Shyu AB. AU-rich elements: characterization and importance in mRNA degradation. Trends Biochem Sci 1995;20(11):465-70
  • Belmont BJ, Niles JC. Engineering a direct and inducible protein-RNA interaction to regulate RNA biology. ACS Chem Biol 2010;5(9):851-61
  • Clery A, Bourguignon-Igel V, Allmang C, et al. An improved definition of the RNA-binding specificity of SECIS-binding protein 2, an essential component of the selenocysteine incorporation machinery. Nucleic Acids Res 2007;35(6):1868-84
  • Bakke O, Nordeng TW. Intracellular traffic to compartments for MHC class II peptide loading: signals for endosomal and polarized sorting. Immunol Rev 1999;172:171-87
  • Marks MS. Protein sorting within the MHC class II antigen-processing pathway. Immunol Res 1998;17(1-2):141-54
  • Bonehill A, Heirman C, Tuyaerts S, et al. Messenger RNA-electroporated dendritic cells presenting MAGE-A3 simultaneously in HLA class I and class II molecules. Journal of immunology 2004;172(11):6649-57
  • Zhong F, Cao W, Chan E, et al. Deviation from major codons in the Toll-like receptor genes is associated with low Toll-like receptor expression. Immunology 2005;114(1):83-93
  • Ngumbela KC, Ryan KP, Sivamurthy R, et al. Quantitative effect of suboptimal codon usage on translational efficiency of mRNA encoding HIV-1 gag in intact T cells. PLoS ONE 2008;3(6):e2356
  • Van Gulck ER, Ponsaerts P, Heyndrickx L, et al. Efficient stimulation of HIV-1-specific T cells using dendritic cells electroporated with mRNA encoding autologous HIV-1 Gag and Env proteins. Blood 2006;107(5):1818-27
  • Kimchi-Sarfaty C, Oh JM, Kim IW, et al. A “silent” polymorphism in the MDR1 gene changes substrate specificity. Science 2007;315(5811):525-8
  • Ho O, Green WR. Alternative translational products and cryptic T cell epitopes: expecting the unexpected. J Immunol 2006;177(12):8283-9
  • Ho O, Green WR. Cytolytic CD8+ T cells directed against a cryptic epitope derived from a retroviral alternative reading frame confer disease protection. Journal of immunology 2006;176(4):2470-5
  • Malarkannan S, Horng T, Shih PP, et al. Presentation of out-of-frame peptide/MHC class I complexes by a novel translation initiation mechanism. Immunity 1999;10(6):681-90
  • Saulquin X, Scotet E, Trautmann L, et al. +1 Frameshifting as a novel mechanism to generate a cryptic cytotoxic T lymphocyte epitope derived from human interleukin 10. J Exp Med 2002;195(3):353-8
  • Schirmbeck R, Riedl P, Fissolo N, et al. Translation from cryptic reading frames of DNA vaccines generates an extended repertoire of immunogenic, MHC class I-restricted epitopes. J Immunol 2005;174(8):4647-56
  • Schwab SR, Li KC, Kang C, Shastri N. Constitutive display of cryptic translation products by MHC class I molecules. Science 2003;301(5638):1367-71
  • Gallie DR. The cap and poly(A) tail function synergistically to regulate mRNA translational efficiency. Genes Dev 1991;5(11):2108-16
  • Tcherepanova IY, Adams MD, Feng X, et al. Ectopic expression of a truncated CD40L protein from synthetic post-transcriptionally capped RNA in dendritic cells induces high levels of IL-12 secretion. BMC Mol Biol 2008;9:90
  • Brencicova E, Diebold SS. Nucleic acids and endosomal pattern recognition: how to tell friend from foe? Frontiers in cellular and infection microbiology 2013;3:37
  • Cerboni S, Gentili M, Manel N. Diversity of pathogen sensors in dendritic cells. Adv Immunol 2013;120:211-37
  • Crampton SP, Bolland S. Spontaneous activation of RNA-sensing pathways in autoimmune disease. Curr Opin Immunol 2013;25(6):712-19
  • Gantier MP. Processing of double-stranded RNA in Mammalian cells: a direct antiviral role? J Interferon Cytokine Res 2014;34(6):469-77
  • Kindler E, Thiel V. To sense or not to sense viral RNA-essentials of coronavirus innate immune evasion. Curr Opin Microbiol 2014;20C:69-75
  • Nellimarla S, Mossman KL. Extracellular dsRNA: its function and mechanism of cellular uptake. J Interferon Cytokine Res 2014;34(6):419-26
  • Rawling DC, Pyle AM. Parts, assembly and operation of the RIG-I family of motors. Curr Opin Struct Biol 2014;25:25-33
  • Vabret N, Blander JM. Sensing microbial RNA in the cytosol. Frontiers in immunology 2013;4:468
  • Wu J, Chen ZJ. Innate immune sensing and signaling of cytosolic nucleic acids. Annu Rev Immunol 2014;32:461-88
  • Goubau D, Deddouche S, Reis e Sousa C. Cytosolic sensing of viruses. Immunity 2013;38(5):855-69
  • Pollard C, Rejman J, De Haes W, et al. Type I IFN counteracts the induction of antigen-specific immune responses by lipid-based delivery of mRNA vaccines. Mol Ther 2013;21(1):251-9
  • Bourquin C, Schmidt L, Hornung V, et al. Immunostimulatory RNA oligonucleotides trigger an antigen-specific cytotoxic T-cell and IgG2a response. Blood 2007;109(7):2953-60
  • Bourquin C, Wurzenberger C, Heidegger S, et al. Delivery of immunostimulatory RNA oligonucleotides by gelatin nanoparticles triggers an efficient antitumoral response. J Immunother 2010;33(9):935-44
  • Sander LE, Davis MJ, Boekschoten MV, et al. Detection of prokaryotic mRNA signifies microbial viability and promotes immunity. Nature 2011;474(7351):385-9
  • Hoerr I, Obst R, Rammensee HG, Jung G. In vivo application of RNA leads to induction of specific cytotoxic T lymphocytes and antibodies. Eur J Immunol 2000;30(1):1-7
  • Weissman D, Ni H, Scales D, et al. HIV gag mRNA transfection of dendritic cells (DC) delivers encoded antigen to MHC class I and II molecules, causes DC maturation, and induces a potent human in vitro primary immune response. Journal of immunology 2000;165(8):4710-17
  • Fotin-Mleczek M, Duchardt KM, Lorenz C, et al. Messenger RNA-based vaccines with dual activity induce balanced TLR-7 dependent adaptive immune responses and provide antitumor activity. J Immunother 2011;34(1):1-15
  • Rettig L, Haen SP, Bittermann AG, et al. Particle size and activation threshold: a new dimension of danger signaling. Blood 2010;115(22):4533-41
  • Kariko K, Buckstein M, Ni H, Weissman D. Suppression of RNA recognition by Toll-like receptors: the impact of nucleoside modification and the evolutionary origin of RNA. Immunity 2005;23(2):165-75
  • Arnaud-Barbe N, Cheynet-Sauvion V, Oriol G, et al. Transcription of RNA templates by T7 RNA polymerase. Nucleic Acids Res 1998;26(15):3550-4
  • Milligan JF, Groebe DR, Witherell GW, Uhlenbeck OC. Oligoribonucleotide synthesis using T7 RNA polymerase and synthetic DNA templates. Nucleic Acids Res 1987;15(21):8783-98
  • Nacheva GA, Berzal-Herranz A. Preventing nondesired RNA-primed RNA extension catalyzed by T7 RNA polymerase. European journal of biochemistry/ FEBS 2003;270(7):1458-65
  • Triana-Alonso FJ, Dabrowski M, Wadzack J, Nierhaus KH. Self-coded 3’-extension of run-off transcripts produces aberrant products during in vitro transcription with T7 RNA polymerase. J Biol Chem 1995;270(11):6298-307
  • Conry RM, LoBuglio AF, Wright M, et al. Characterization of a messenger RNA polynucleotide vaccine vector. Cancer Res 1995;55(7):1397-400
  • Heiser A, Coleman D, Dannull J, et al. Autologous dendritic cells transfected with prostate-specific antigen RNA stimulate CTL responses against metastatic prostate tumors. J Clin Invest 2002;109(3):409-17
  • Nair SK, Boczkowski D, Morse M, et al. Induction of primary carcinoembryonic antigen (CEA)-specific cytotoxic T lymphocytes in vitro using human dendritic cells transfected with RNA. Nat Biotechnol 1998;16(4):364-9
  • Nair SK, Heiser A, Boczkowski D, et al. Induction of cytotoxic T cell responses and tumor immunity against unrelated tumors using telomerase reverse transcriptase RNA transfected dendritic cells. Nat Med 2000;6(9):1011-17
  • Rains N, Cannan RJ, Chen W, Stubbs RS. Development of a dendritic cell (DC)-based vaccine for patients with advanced colorectal cancer. Hepatogastroenterology 2001;48(38):347-51
  • Van Lint S, Heirman C, Thielemans K. Breckpot K. mRNA: from a chemical blueprint for protein production to an off-the-shelf therapeutic. Hum Vaccin Immunother 2013;9(2):265-74
  • Bonehill A, Tuyaerts S, Van Nuffel AM, et al. Enhancing the T-cell stimulatory capacity of human dendritic cells by co-electroporation with CD40L, CD70 and constitutively active TLR4 encoding mRNA. Mol Ther 2008;16(6):1170-80
  • Calderhead DM, DeBenedette MA, Ketteringham H, et al. Cytokine maturation followed by CD40L mRNA electroporation results in a clinically relevant dendritic cell product capable of inducing a potent proinflammatory CTL response. J Immunother 2008;31(8):731-41
  • Cisco RM, Abdel-Wahab Z, Dannull J, et al. Induction of human dendritic cell maturation using transfection with RNA encoding a dominant positive toll-like receptor 4. Journal of immunology 2004;172(11):7162-8
  • Routy JP, Boulassel MR, Yassine-Diab B, et al. Immunologic activity and safety of autologous HIV RNA-electroporated dendritic cells in HIV-1 infected patients receiving antiretroviral therapy. Clinical immunology 2010;134(2):140-7
  • Van Lint S, Wilgenhof S, Heirman C, et al. Optimized dendritic cell-based immunotherapy for melanoma: the TriMix-formula. Cancer Immunol Immunother 2014;63(9):959-67
  • Wilgenhof S, Van Nuffel AM, Benteyn D, et al. A phase IB study on intravenous synthetic mRNA electroporated dendritic cell immunotherapy in pretreated advanced melanoma patients. Ann Oncol 2013;24(10):2686-93
  • Phase 3 Trial of Autologous Dendritic Cell Immunotherapy (AGS-003) Plus Standard Treatment of Advanced Renal Cell Carcinoma (RCC). Available from: https://clinicaltrials.gov/ct2/results?term=NCT01582672&Search=Search
  • Fotin-Mleczek M, Zanzinger K, Heidenreich R, et al. Highly potent mRNA based cancer vaccines represent an attractive platform for combination therapies supporting an improved therapeutic effect. J Gene Med 2012;14(6):428-39
  • Diken M, Kreiter S, Vascotto F, et al. mTOR inhibition improves antitumor effects of vaccination with antigen-encoding RNA. Cancer Immunol Res 2013;1(6):386-92
  • Kreiter S, Diken M, Selmi A, et al. FLT3 ligand enhances the cancer therapeutic potency of naked RNA vaccines. Cancer Res 2011;71(19):6132-42
  • Van Driessche A, Van de Velde AL, Nijs G, et al. Clinical-grade manufacturing of autologous mature mRNA-electroporated dendritic cells and safety testing in acute myeloid leukemia patients in a phase I dose-escalation clinical trial. Cytotherapy 2009;11(5):653-68
  • Castle JC, Kreiter S, Diekmann J, et al. Exploiting the mutanome for tumor vaccination. Cancer Res 2012;72(5):1081-91
  • Kreiter S, Castle JC, Tureci O, Sahin U. Targeting the tumor mutanome for personalized vaccination therapy. Oncoimmunology 2012;1(5):768-9
  • IVAC MUTANOME Phase I Clinical Trial. Available from: https://clinicaltrials.gov/ct2/results?term=NCT02035956&Search=Search
  • Petsch B, Schnee M, Vogel AB, et al. Protective efficacy of in vitro synthesized, specific mRNA vaccines against influenza A virus infection. Nat Biotechnol 2012;30(12):1210-16
  • Mandl CW, Aberle JH, Aberle SW, et al. In vitro-synthesized infectious RNA as an attenuated live vaccine in a flavivirus model. Nat Med 1998;4(12):1438-40
  • Anraku I, Harvey TJ, Linedale R, et al. Kunjin virus replicon vaccine vectors induce protective CD8+ T-cell immunity. J Virol 2002;76(8):3791-9
  • Fleeton MN, Chen M, Berglund P, et al. Self-replicative RNA vaccines elicit protection against influenza A virus, respiratory syncytial virus, and a tick borne encephalitis virus. J Infect Dis 2001;183(9):1395-8
  • Greer CE, Zhou F, Legg HS, et al. A chimeric alphavirus RNA replicon gene-based vaccine for human parainfluenza virus type 3 induces protective immunity against intranasal virus challenge. Vaccine 2007;25(3):481-9
  • Geall AJ, Verma A, Otten GR, et al. Nonviral delivery of self-amplifying RNA vaccines. Proc Natl Acad Sci USA 2012;109(36):14604-9
  • Brito LA, Chan M, Shaw CA, et al. A cationic nanoemulsion for the delivery of next generation RNA vaccines. Mol Ther 2014;10.1038/mt.2014.133
  • Allard SD, De Keersmaecker B, de Goede AL, et al. A phase I/IIa immunotherapy trial of HIV-1-infected patients with Tat, Rev and Nef expressing dendritic cells followed by treatment interruption. Clinical Immunol 2012;142(3):252-68
  • Van Gulck E, Vlieghe E, Vekemans M, et al. mRNA-based dendritic cell vaccination induces potent antiviral T-cell responses in HIV-1-infected patients. Aids 2012;26(4):F1-12
  • Baena-Cagnani CE, Gomez RM. Current status of therapy with omalizumab in children. Curr Opin Allergy Clin Immunol 2014;14(2):149-54
  • Keet CA, Wood RA. Emerging therapies for food allergy. J Clin Invest 2014;124(5):1880-6
  • Normansell R, Walker S, Milan SJ, et al. Omalizumab for asthma in adults and children. Cochrane Database Syst Rev 2014;1:Cd003559
  • Vashisht P, Casale T. Omalizumab for treatment of allergic rhinitis. Expert Opin Biol Ther 2013;13(6):933-45
  • Roesler E, Weiss R, Weinberger EE, et al. Immunize and disappear-safety-optimized mRNA vaccination with a panel of 29 allergens. J Allergy Clin Immunol 2009;124(5):1070-7. e1071-1011
  • Weinberger EE, Isakovic A, Scheiblhofer S, et al. The influence of antigen targeting to sub-cellular compartments on the anti-allergic potential of a DNA vaccine. Vaccine 2013;31(51):6113-21
  • Weiss R, Scheiblhofer S, Roesler E, et al. Prophylactic mRNA vaccination against allergy. Curr Opin Allergy Clin Immunol 2010;10(6):567-74
  • Weiss R, Scheiblhofer S, Roesler E, et al. mRNA vaccination as a safe approach for specific protection from type I allergy. Expert Rev Vaccines 2012;11(1):55-67
  • Weiss R, Scheiblhofer S, Thalhamer J. Allergens are not pathogens: Why immunization against allergy differs from vaccination against infectious diseases. Hum Vaccin Immunother 2013;10:3
  • Maus MV, Grupp SA, Porter DL, June CH. Antibody-modified T cells: CARs take the front seat for hematologic malignancies. Blood 2014;123(17):2625-35
  • Almasbak H, Rian E, Hoel HJ, et al. Transiently redirected T cells for adoptive transfer. Cytotherapy 2011;13(5):629-40
  • Barrett DM, Liu X, Jiang S, et al. Regimen-specific effects of RNA-modified chimeric antigen receptor T cells in mice with advanced leukemia. Hum Gene Ther 2013;24(8):717-27
  • Barrett DM, Zhao Y, Liu X, et al. Treatment of advanced leukemia in mice with mRNA engineered T cells. Hum Gene Ther 2011;22(12):1575-86
  • Zhao Y, Moon E, Carpenito C, et al. Multiple injections of electroporated autologous T cells expressing a chimeric antigen receptor mediate regression of human disseminated tumor. Cancer Res 2010;70(22):9053-61
  • Autologous Redirected RNA Meso CAR T Cells for Pancreatic Cancer. Available from: https://clinicaltrials.gov/ct2/results?term=NCT01897415&Search=Search
  • Hockemeyer D, Wang H, Kiani S, et al. Genetic engineering of human pluripotent cells using TALE nucleases. Nat Biotechnol 2011;29(8):731-4
  • Miller JC, Holmes MC, Wang J, et al. An improved zinc-finger nuclease architecture for highly specific genome editing. Nat Biotechnol 2007;25(7):778-85
  • Mali P, Aach J, Stranges PB, et al. CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering. Nat Biotechnol 2013;31(9):833-8
  • Mali P, Yang L, Esvelt KM, et al. RNA-guided human genome engineering via Cas9. Science 2013;339(6121):823-6
  • Fu Y, Foden JA, Khayter C, et al. High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nature biotechnology 2013;31(9):822-6
  • Auer TO, Duroure K, De Cian A, et al. Highly efficient CRISPR/Cas9-mediated knock-in in zebrafish by homology-independent DNA repair. Genome Res 2014;24(1):142-53
  • Bedell VM, Wang Y, Campbell JM, et al. In vivo genome editing using a high-efficiency TALEN system. Nature 2012;491(7422):114-18
  • Geurts AM, Cost GJ, Freyvert Y, et al. Knockout rats via embryo microinjection of zinc-finger nucleases. Science 2009;325(5939):433
  • Hwang WY, Fu Y, Reyon D, et al. Efficient genome editing in zebrafish using a CRISPR-Cas system. Nat Biotechnol 2013;31(3):227-9
  • Tesson L, Usal C, Menoret S, et al. Knockout rats generated by embryo microinjection of TALENs. Nat Biotechnol 2011;29(8):695-6
  • Wang H, Yang H, Shivalila CS, et al. One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering. Cell 2013;153(4):910-18
  • Wefers B, Panda SK, Ortiz O, et al. Generation of targeted mouse mutants by embryo microinjection of TALEN mRNA. Nat Protoc 2013;8(12):2355-79
  • Wood AJ, Lo TW, Zeitler B, et al. Targeted genome editing across species using ZFNs and TALENs. Science 2011;333(6040):307
  • Yamanaka S, Takahashi K. [Induction of pluripotent stem cells from mouse fibroblast cultures]. Tanpakushitsu kakusan koso 2006;51(15):2346-51
  • Warren L, Ni Y, Wang J, Guo X. Feeder-free derivation of human induced pluripotent stem cells with messenger RNA. Sci Rep 2012;2:657
  • Yakubov E, Rechavi G, Rozenblatt S, Givol D. Reprogramming of human fibroblasts to pluripotent stem cells using mRNA of four transcription factors. Biochem Biophys Res Commun 2010;394(1):189-93
  • Miller JD, Ganat YM, Kishinevsky S, et al. Human iPSC-based modeling of late-onset disease via progerin-induced aging. Cell Stem Cell 2013;13(6):691-705
  • Okano H, Nakamura M, Yoshida K, et al. Steps toward safe cell therapy using induced pluripotent stem cells. Circ Res 2013;112(3):523-33
  • Kariko K, Weissman D. Naturally occurring nucleoside modifications suppress the immunostimulatory activity of RNA: implication for therapeutic RNA development. Curr Opin Drug Discov Dev 2007;10(5):523-32
  • Mays LE, Ammon-Treiber S, Mothes B, et al. Modified Foxp3 mRNA protects against asthma through an IL-10-dependent mechanism. J Clin Invest 2013;123(3):1216-28
  • Zangi L, Lui KO, von Gise A, et al. Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction. Nat Biotechnol 2013;31(10):898-907
  • Levy O, Zhao W, Mortensen LJ, et al. mRNA-engineered mesenchymal stem cells for targeted delivery of interleukin-10 to sites of inflammation. Blood 2013;122(14):e23-32
  • Weissman D, Pardi N, Muramatsu H, Kariko K. HPLC purification of in vitro transcribed long RNA. Methods Mol Biol 2013;969:43-54
  • Fattori E, Cappelletti M, Zampaglione I, et al. Gene electro-transfer of an improved erythropoietin plasmid in mice and non-human primates. J Gene Med 2005;7(2):228-36
  • Lee S, Lee Y, Hong K, et al. Effect of recombinant alpha1-antitrypsin Fc-fused (AAT-Fc)protein on the inhibition of inflammatory cytokine production and streptozotocin-induced diabetes. Mol Med 2013;19:65-71
  • Barash S, Wang W, Shi Y. Human secretory signal peptide description by hidden Markov model and generation of a strong artificial signal peptide for secreted protein expression. Biochem Biophys Res Commun 2002;294(4):835-42
  • Kolarich D, Weber A, Turecek PL, et al. Comprehensive glyco-proteomic analysis of human alpha1-antitrypsin and its charge isoforms. Proteomics 2006;6(11):3369-80
  • Sontag EM, Vonk WI, Frydman J. Sorting out the trash: the spatial nature of eukaryotic protein quality control. Curr Opin Cell Biol 2014;26:139-46
  • Seidah NG, Chretien M. Proprotein and prohormone convertases: a family of subtilases generating diverse bioactive polypeptides. Brain Res 1999;848(1-2):45-62
  • Nakayama K. Furin: a mammalian subtilisin/Kex2p-like endoprotease involved in processing of a wide variety of precursor proteins. Biochem J 1997;327(Pt 3):625-35
  • Koren E, Smith HW, Shores E, et al. Recommendations on risk-based strategies for detection and characterization of antibodies against biotechnology products. J Immunol Methods 2008;333(1-2):1-9
  • Murphy K. The Humoral Immune Response. In: Janeway C, editor. Immunobiology. Garland Science Publishing; New York: 2011. p. 367-408
  • Gao G, Lebherz C, Weiner DJ, et al. Erythropoietin gene therapy leads to autoimmune anemia in macaques. Blood 2004;103(9):3300-2
  • Casadevall N, Nataf J, Viron B, et al. Pure red-cell aplasia and antierythropoietin antibodies in patients treated with recombinant erythropoietin. N Engl J Med 2002;346(7):469-75
  • Cournoyer D, Toffelmire EB, Wells GA, et al. Anti-erythropoietin antibody-mediated pure red cell aplasia after treatment with recombinant erythropoietin products: recommendations for minimization of risk. J Am Soc Nephrol 2004;15(10):2728-34
  • Kromminga A, Schellekens H. Antibodies against erythropoietin and other protein-based therapeutics: an overview. Ann N Y Acad Sci 2005;1050:257-65
  • Hwang SH, Lee H, Yamamoto M, et al. B cell TLR7 expression drives anti-RNA autoantibody production and exacerbates disease in systemic lupus erythematosus-prone mice. Journal of immunology 2012;189(12):5786-96
  • Lipes BD, Keene JD. Autoimmune epitopes in messenger RNA. RNA 2002;8(6):762-71
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