Screening of mRNA chemical modification to maximize protein expression with reduced immunogenicity

Pharmaceutics

Volumn 7, Issue 3, 2015, Pages 137-151

  Uchida, Satoshi ^a   Kataoka, Kazunori ^a,b   Itaka, Keiji ^a  

^a UNIVERSITY OF TOKYO   (Japan)

^b UNIVERSITY OF TOKYO   (Japan)

Author keywords

Immunogenicity; Modification; mRNA; Nucleasemediated degradation; Translation efficiency

Indexed keywords

BETA INTERFERON; INTERLEUKIN 1BETA; INTERLEUKIN 6; LUCIFERASE; MESSENGER RNA; NUCLEASE; TUMOR NECROSIS FACTOR ALPHA;

ADULT; ANIMAL CELL; ANIMAL TISSUE; ARTICLE; CELL LINE; CELL VIABILITY; CHEMICAL MODIFICATION; CONTROLLED STUDY; CYTOKINE PRODUCTION; DOWN REGULATION; EMBRYO; GENETIC TRANSFECTION; HEPATOCELLULAR CARCINOMA CELL LINE; HUMAN; HUMAN CELL; IMMUNOGENICITY; IN VITRO STUDY; MACROPHAGE; MEF CELL LINE; MESSENGER RNA MODIFICATION; MOUSE; NONHUMAN; PROTEIN ANALYSIS; PROTEIN EXPRESSION; RAW 264.7 CELL LINE; RNA ANALYSIS; RNA DEGRADATION; RNA STABILITY; RNA TRANSLATION; UPREGULATION;

EID: 84938361667     PISSN: None     EISSN: 19994923     Source Type: Journal    
DOI: 10.3390/pharmaceutics7030137     Document Type: Article

References (24)

1. Tavernier, G.; Andries, O.; Demeester, J.; Sanders, N. N.; De Smedt, S. C.; Rejman, J. mRNA as gene therapeutic: How to control protein expression. J. Control. Release 2011, 150, 238-247.
2. Sahin, U.; Kariko, K.; Tureci, O. mRNA-based therapeutics-Developing a new class of drugs. Nat. Rev. Drug Discov. 2014, 13, 759-780.
3. Warren, L.; Manos, P. D.; Ahfeldt, T.; Loh, Y. H.; Li, H.; Lau, F.; Ebina, W.; Mandal, P. K.; Smith, Z. D.; Meissner, A.; et al. Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA. Cell Stem Cell 2010, 7, 618-630.
4. Mandal, P. K.; Rossi, D. J. Reprogramming human fibroblasts to pluripotency using modified mRNA. Nature Protocols 2013, 8, 568-582.
5. Doyon, Y.; McCammon, J. M.; Miller, J. C.; Faraji, F.; Ngo, C.; Katibah, G. E.; Amora, R.; Hocking, T. D.; Zhang, L.; Rebar, E. J.; et al. Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases. Nat. Biotechnol. 2008, 26, 702-708.
6. Wood, A. J.; Lo, T. W.; Zeitler, B.; Pickle, C. S.; Ralston, E. J.; Lee, A. H.; Amora, R.; Miller, J. C.; Leung, E.; Meng, X.; et al. Targeted genome editing across species using ZFNs and TALENs. Science 2011, 333, 307.
7. Hwang, W. Y.; Fu, Y.; Reyon, D.; Maeder, M. L.; Tsai, S. Q.; Sander, J. D.; Peterson, R. T.; Yeh, J. R.; Joung, J. K. Efficient genome editing in zebrafish using a CRISPR-Cas system. Nat. Biotechnol. 2013, 31, 227-229.
8. Kormann, M. S.; Hasenpusch, G.; Aneja, M. K.; Nica, G.; Flemmer, A. W.; Herber-Jonat, S.; Huppmann, M.; Mays, L. E.; Illenyi, M.; Schams, A.; et al. Expression of therapeutic proteins after delivery of chemically modified mRNA in mice. Nat. Biotechnol. 2011, 29, 154-157.
9. Wang, Y.; Su, H. H.; Yang, Y.; Hu, Y.; Zhang, L.; Blancafort, P.; Huang, L. Systemic delivery of modified mRNA encoding herpes simplex virus 1 thymidine kinase for targeted cancer gene therapy. Mol. Ther. 2013, 21, 358-367.
10. Zangi, L.; Lui, K. O.; von Gise, A.; Ma, Q.; Ebina, W.; Ptaszek, L. M.; Spater, D.; Xu, H.; Tabebordbar, M.; Gorbatov, R.; et al. Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction. Nat. Biotechnol. 2013, 31, 898-907.
11. Baba, M.; Itaka, K.; Kondo, K.; Yamasoba, T.; Kataoka, K. Treatment of neurological disorders by introducing mRNA in vivo using polyplex nanomicelles. J. Control. Release 2015, 201, 41-48.
12. Kariko, K.; Ni, H.; Capodici, J.; Lamphier, M.; Weissman, D. mRNA is an endogenous ligand for Toll-like receptor 3. J. Biol. Chem. 2004, 279, 12542-12550.
13. Kawai, T.; Akira, S. The role of pattern-recognition receptors in innate immunity: Update on Tolllike receptors. Nat. Immunol. 2010, 11, 373-384.
14. Weide, B.; Carralot, J. P.; Reese, A.; Scheel, B.; Eigentler, T. K.; Hoerr, I.; Rammensee, H. G.; Garbe, C.; Pascolo, S. Results of the first phase I/II clinical vaccination trial with direct injection of mRNA. J. Immunother. 2008, 31, 180-188.
15. Weide, B.; Pascolo, S.; Scheel, B.; Derhovanessian, E.; Pflugfelder, A.; Eigentler, T. K.; Pawelec, G.; Hoerr, I.; Rammensee, H. G.; Garbe, C. Direct injection of protamine-protected mRNA: Results of a phase 1/2 vaccination trial in metastatic melanoma patients. J. Immunother. 2009, 32, 498-507.
16. Deering, R. P.; Kommareddy, S.; Ulmer, J. B.; Brito, L. A.; Geall, A. J. Nucleic acid vaccines: Prospects for non-viral delivery of mRNA vaccines. Expert Opin. Drug Deliv. 2014, 11, 885-899.
17. 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, 165-175.
18. 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, doi: 10. 1093/nar/gkr695.
19. Mays, L. E.; Ammon-Treiber, S.; Mothes, B.; Alkhaled, M.; Rottenberger, J.; Muller-Hermelink, E. S.; Grimm, M.; Mezger, M.; Beer-Hammer, S.; von Stebut, E.; et al. Modified Foxp3 mRNA protects against asthma through an IL-10-dependent mechanism. J. Clin. Investig. 2013, 123, 1216-1228.
20. Uchida, S.; Itaka, K.; Uchida, H.; Hayakawa, K.; Ogata, T.; Ishii, T.; Fukushima, S.; Osada, K.; Kataoka, K. In vivo messenger RNA introduction into the central nervous system using polyplex nanomicelle. PLoS ONE 2013, 8, e56220.
21. Wurdinger, T.; Badr, C.; Pike, L.; de Kleine, R.; Weissleder, R.; Breakefield, X. O.; Tannous, B. A. A secreted luciferase for ex vivo monitoring of in vivo processes. Nat. Methods 2008, 5, 171-173.
22. Andries, O.; De Filette, M.; De Smedt, S. C.; Demeester, J.; Van Poucke, M.; Peelman, L.; Sanders, N. N. Innate immune response and programmed cell death following carrier-mediated delivery of unmodified mRNA to respiratory cells. J. Control. Release 2013, 167, 157-166.
23. Pollard, C.; Rejman, J.; De Haes, W.; Verrier, B.; Van Gulck, E.; Naessens, T.; De Smedt, S.; Bogaert, P.; Grooten, J.; Vanham, G.; et al. Type I IFN counteracts the induction of antigen-specific immune responses by lipid-based delivery of mRNA vaccines. Mol. Ther. 2013, 21, 251-259.
24. Vila, M.; Portoles, M. T.; Marques, P. A.; Feito, M. J.; Matesanz, M. C.; Ramirez-Santillan, C.; Goncalves, G.; Cruz, S. M.; Nieto, A.; Vallet-Regi, M. Cell uptake survey of pegylated nanographene oxide. Nanotechnology 2012, 23, 465103.