In Vivo Messenger RNA Introduction into the Central Nervous System Using Polyplex Nanomicelle

PLoS ONE

Volumn 8, Issue 2, 2013, Pages

  Uchida, Satoshi ^a   Itaka, Keiji ^a   Uchida, Hirokuni ^b   Hayakawa, Kentaro ^c   Ogata, Toru ^c   Ishii, Takehiko ^b   Fukushima, Shigeto ^b   Osada, Kensuke ^b   Kataoka, Kazunori ^a,b  

^a UNIVERSITY OF TOKYO   (Japan)

^b UNIVERSITY OF TOKYO   (Japan)

^c NATIONAL REHABILITATION CENTER FOR PERSONS WITH DISABILITIES   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

COPOLYMER; MACROGOL; MESSENGER RNA; POLYPLEX NANOMICELLE; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BRAIN; CEREBROSPINAL FLUID; CYTOPLASM; DRUG MECHANISM; EMBRYO; ENDOSOME; FEMALE; HUMAN; HUMAN CELL; IMMUNOGENICITY; IN VITRO STUDY; IN VIVO GENE TRANSFER; MOUSE; NONHUMAN; PARTICLE SIZE; PROTEIN EXPRESSION; RAT; SPINAL CORD;

AMINO ACIDS; ANIMALS; CELL LINE, TUMOR; CENTRAL NERVOUS SYSTEM; FEMALE; GENE EXPRESSION; GREEN FLUORESCENT PROTEINS; HEK293 CELLS; HUMANS; INJECTIONS, SPINAL; LUCIFERASES; MICE; MICE, INBRED BALB C; MICELLES; MICROSCOPY, FLUORESCENCE; NANOSTRUCTURES; POLYETHYLENE GLYCOLS; POLYMERS; RATS; RATS, SPRAGUE-DAWLEY; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA, MESSENGER; TOLL-LIKE RECEPTOR 7; TOLL-LIKE RECEPTOR 9;

EID: 84873906718     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0056220     Document Type: Article

References (45)

1. Callis J, Fromm M, Walbot V, (1987) Expression of mRNA electroporated into plant and animal cells. Nucleic Acids Res 15: 5823-5831.
2. Malone RW, Felgner PL, Verma IM, (1989) Cationic liposome-mediated RNA transfection. Proc Natl Acad Sci U S A 86: 6077-6081.
3. Tavernier G, Andries O, Demeester J, Sanders NN, De Smedt SC, et al. (2011) mRNA as gene therapeutic: how to control protein expression. J Control Release 150: 238-247.
4. Heil F, Hemmi H, Hochrein H, Ampenberger F, Kirschning C, et al. (2004) Species-specific recognition of single-stranded RNA via toll-like receptor 7 and 8. Science 303: 1526-1529.
5. Kariko K, Ni H, Capodici J, Lamphier M, Weissman D, (2004) mRNA is an endogenous ligand for Toll-like receptor 3. J Biol Chem 279: 12542-12550.
6. Mitchell DA, Nair SK, (2000) RNA-transfected dendritic cells in cancer immunotherapy. J Clin Invest 106: 1065-1069.
7. Van Tendeloo VF, Ponsaerts P, Berneman ZN, (2007) mRNA-based gene transfer as a tool for gene and cell therapy. Curr Opin Mol Ther 9: 423-431.
8. Weide B, Carralot JP, Reese A, Scheel B, Eigentler TK, et al. (2008) Results of the first phase I/II clinical vaccination trial with direct injection of mRNA. J Immunother 31: 180-188.
9. Weide B, Pascolo S, Scheel B, Derhovanessian E, Pflugfelder A, et al. (2009) Direct injection of protamine-protected mRNA: results of a phase 1/2 vaccination trial in metastatic melanoma patients. J Immunother 32: 498-507.
10. Kormann MS, Hasenpusch G, Aneja MK, Nica G, Flemmer AW, et al. (2011) Expression of therapeutic proteins after delivery of chemically modified mRNA in mice. Nat Biotechnol 29: 154-157.
11. Kariko K, Muramatsu H, Keller JM, Weissman D, (2012) Increased Erythropoiesis in Mice Injected With Submicrogram Quantities of Pseudouridine-containing mRNA Encoding Erythropoietin. Mol Ther 20: 948-953.
12. Brennan CM, Steitz JA, (2001) HuR and mRNA stability. Cell Mol Life Sci 58: 266-277.
13. Rejman J, Tavernier G, Bavarsad N, Demeester J, De Smedt SC, (2010) mRNA transfection of cervical carcinoma and mesenchymal stem cells mediated by cationic carriers. J Control Release 147: 385-391.
14. Plews JR, Li J, Jones M, Moore HD, Mason C, et al. (2010) Activation of pluripotency genes in human fibroblast cells by a novel mRNA based approach. PLoS One 5: e14397.
15. Warren L, Manos PD, Ahfeldt T, Loh YH, Li H, et al. (2010) Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA. Cell Stem Cell 7: 618-630.
16. Tavernier G, Wolfrum K, Demeester J, De Smedt SC, Adjaye J, et al. (2011) Activation of pluripotency-associated genes in mouse embryonic fibroblasts by non-viral transfection with in vitro-derived mRNAs encoding Oct4, Sox2, Klf4 and cMyc. Biomaterials.
17. Wolff JA, Malone RW, Williams P, Chong W, Acsadi G, et al. (1990) Direct gene transfer into mouse muscle in vivo. Science 247: 1465-1468.
18. Probst J, Weide B, Scheel B, Pichler BJ, Hoerr I, et al. (2007) Spontaneous cellular uptake of exogenous messenger RNA in vivo is nucleic acid-specific, saturable and ion dependent. Gene Ther 14: 1175-1180.
19. Qiu P, Ziegelhoffer P, Sun J, Yang NS, (1996) Gene gun delivery of mRNA in situ results in efficient transgene expression and genetic immunization. Gene Ther 3: 262-268.
20. Scheel B, Teufel R, Probst J, Carralot JP, Geginat J, et al. (2005) Toll-like receptor-dependent activation of several human blood cell types by protamine-condensed mRNA. Eur J Immunol 35: 1557-1566.
21. Zou S, Scarfo K, Nantz MH, Hecker JG, (2010) Lipid-mediated delivery of RNA is more efficient than delivery of DNA in non-dividing cells. Int J Pharm 389: 232-243.
22. Su X, Fricke J, Kavanagh DG, Irvine DJ, (2011) In Vitro and in Vivo mRNA Delivery Using Lipid-Enveloped pH-Responsive Polymer Nanoparticles. Mol Pharm 8: 774-787.
23. Kariko K, Buckstein M, Ni H, 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.
24. Anderson BR, Muramatsu H, Nallagatla SR, Bevilacqua PC, Sansing LH, et al. (2010) Incorporation of pseudouridine into mRNA enhances translation by diminishing PKR activation. Nucleic Acids Res 38: 5884-5892.
25. Kariko K, Muramatsu H, Welsh FA, Ludwig J, Kato H, et al. (2008) Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability. Mol Ther 16: 1833-1840.
26. Charette M, Gray MW, (2000) Pseudouridine in RNA: what, where, how, and why. IUBMB Life 49: 341-351.
27. Itaka K, Kataoka K, (2009) Recent development of nonviral gene delivery systems with virus-like structures and mechanisms. Eur J Pharm Biopharm 71: 475-483.
28. Abbott NJ, (2004) Evidence for bulk flow of brain interstitial fluid: significance for physiology and pathology. Neurochem Int 45: 545-552.
29. Kataoka K, Harada A, Nagasaki Y, (2001) Block copolymer micelles for drug delivery: design, characterization and biological significance. Adv Drug Deliv Rev 47: 113-131.
30. Kanayama N, Fukushima S, Nishiyama N, Itaka K, Jang WD, et al. (2006) A PEG-based biocompatible block catiomer with high buffering capacity for the construction of polyplex micelles showing efficient gene transfer toward primary cells. ChemMedChem 1: 439-444.
31. Itaka K, Kataoka K, (2011) Progress and prospects of polyplex nanomicelles for plasmid DNA delivery. Curr Gene Ther 11: 457-465.
32. Miyata K, Oba M, Nakanishi M, Fukushima S, Yamasaki Y, et al. (2008) Polyplexes from poly(aspartamide) bearing 1,2-diaminoethane side chains induce pH-selective, endosomal membrane destabilization with amplified transfection and negligible cytotoxicity. J Am Chem Soc 130: 16287-16294.
33. Masago K, Itaka K, Nishiyama N, Chung UI, Kataoka K, (2007) Gene delivery with biocompatible cationic polymer: pharmacogenomic analysis on cell bioactivity. Biomaterials 28: 5169-5175.
34. Itaka K, Ishii T, Hasegawa Y, Kataoka K, (2010) Biodegradable polyamino acid-based polycations as safe and effective gene carrier minimizing cumulative toxicity. Biomaterials 31: 3707-3714.
35. Giulietti A, Overbergh L, Valckx D, Decallonne B, Bouillon R, et al. (2001) An overview of real-time quantitative PCR: applications to quantify cytokine gene expression. Methods 25: 386-401.
36. Listvanova S, Temmerman S, Stordeur P, Verscheure V, Place S, et al. (2003) Optimal kinetics for quantification of antigen-induced cytokines in human peripheral blood mononuclear cells by real-time PCR and by ELISA. J Immunol Methods 281: 27-35.
37. Krieg AM, Vollmer J, (2007) Toll-like receptors 7, 8, and 9: linking innate immunity to autoimmunity. Immunol Rev 220: 251-269.
38. Akira S, Takeda K, Kaisho T, (2001) Toll-like receptors: critical proteins linking innate and acquired immunity. Nat Immunol 2: 675-680.
39. Tannous BA, (2009) Gaussia luciferase reporter assay for monitoring biological processes in culture and in vivo. Nat Protoc 4: 582-591.
40. Miyata K, Nishiyama N, Kataoka K, (2012) Rational design of smart supramolecular assemblies for gene delivery: chemical challenges in the creation of artificial viruses. Chem Soc Rev 41: 2562-2574.
41. Lv H, Zhang S, Wang B, Cui S, Yan J, (2006) Toxicity of cationic lipids and cationic polymers in gene delivery. J Control Release 114: 100-109.
42. Bettinger T, Carlisle RC, Read ML, Ogris M, Seymour LW, (2001) Peptide-mediated RNA delivery: a novel approach for enhanced transfection of primary and post-mitotic cells. Nucleic Acids Res 29: 3882-3891.
43. Reijneveld JC, Taphoorn MJ, Voest EE, (1999) A simple mouse model for leptomeningeal metastases and repeated intrathecal therapy. J Neurooncol 42: 137-142.
44. Veening JG, Barendregt HP, (2010) The regulation of brain states by neuroactive substances distributed via the cerebrospinal fluid; a review. Cerebrospinal Fluid Res 7: 1.
45. Kawamoto T, (2003) Use of a new adhesive film for the preparation of multi-purpose fresh-frozen sections from hard tissues, whole-animals, insects and plants. Arch Histol Cytol 66: 123-143.