Transcript-activated collagen matrix as sustained mRNA delivery system for bone regeneration

Journal of Controlled Release

Volumn 239, Issue , 2016, Pages 137-148

  Badieyan, Zohreh Sadat ^a   Berezhanskyy, Taras ^a,b   Utzinger, Maximilian ^b,c   Aneja, Manish Kumar ^b   Emrich, Daniela ^b   Erben, Reinhold ^d   Schüler, Christiane ^d   Altpeter, Philipp ^c   Ferizi, Mehrije ^b   Hasenpusch, Günther ^b   Rudolph, Carsten ^b   Plank, Christian ^a,b  

^a TECHNICAL UNIVERSITY OF MUNICH   (Germany)

^b ETHRIS GMBH   (Germany)

^c UNIVERSITY OF MUNICH   (Germany)

^d UNIVERSITY OF VETERINARY MEDICINE   (Austria)

Author keywords

Bone regeneration; Chemically modified mRNA (cmRNA); Human bone morphogenetic protein 2 (hBMP 2); Sustained delivery; Transcript therapy; Transcript activated matrix (TAM)

Indexed keywords

BONE; COLLAGEN; GENE THERAPY; NUCLEIC ACIDS; PROTEINS; RECOMBINANT PROTEINS; RNA; TISSUE ENGINEERING;

BONE REGENERATION; CHEMICALLY MODIFIED; HUMAN BONE MORPHOGENETIC PROTEIN; SUSTAINED DELIVERY; TRANSCRIPT THERAPY; TRANSCRIPT-ACTIVATED MATRIX (TAM);

TISSUE REGENERATION;

BONE MORPHOGENETIC PROTEIN 2; COLLAGEN; COLLAGEN SPONGE; INTERLEUKIN 6; LIPOPLEX; MESSENGER RNA; TRANSCRIPT ACTIVATED MATRIX; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BONE DEFECT; BONE REGENERATION; CELL DIFFERENTIATION; CELL VIABILITY; CONTROLLED STUDY; CYTOKINE RELEASE; FEMUR; FLUORESCENCE MICROSCOPY; GENE DELIVERY SYSTEM; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; IMMUNOGENICITY; IN VITRO STUDY; IN VIVO STUDY; NONHUMAN; PARTICLE SIZE; PERIPHERAL BLOOD MONONUCLEAR CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; RAT; ROOM TEMPERATURE; SCANNING ELECTRON MICROSCOPY; TISSUE ENGINEERING; TRANSCRIPTION REGULATION; ZETA POTENTIAL; A-549 CELL LINE; ADMINISTRATION AND DOSAGE; ANIMAL; CELL CULTURE; CELL SURVIVAL; CHEMISTRY; DELAYED RELEASE FORMULATION; DIAGNOSTIC IMAGING; DRUG EFFECTS; GENE THERAPY; GENE TRANSFER; GENETICS; HEP-G2 CELL LINE; MALE; METABOLISM; MOUSE; NIH 3T3 CELL LINE; PHYSIOLOGY; PROCEDURES; SPRAGUE DAWLEY RAT;

A549 CELLS; ANIMALS; BONE REGENERATION; CELL SURVIVAL; CELLS, CULTURED; COLLAGEN; DELAYED-ACTION PREPARATIONS; FEMUR; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; HEP G2 CELLS; HUMANS; MALE; MICE; NIH 3T3 CELLS; RATS; RATS, SPRAGUE-DAWLEY; RNA, MESSENGER;

EID: 84984788004     PISSN: 01683659     EISSN: 18734995     Source Type: Journal    
DOI: 10.1016/j.jconrel.2016.08.037     Document Type: Article

References (41)

1. [1] Yamamoto, A., Kormann, M., Rosenecker, J., Rudolph, C., Current prospects for mRNA gene delivery. Eur. J. Pharm. Biopharm. 71 (2009), 484–489.
2. [2] 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 150 (2011), 238–247.
3. [3] 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., Expression of therapeutic proteins after delivery of chemically modified mRNA in mice. Nat. Biotechnol. 29 (2011), 154–157.
4. [4] Esteller, M., Non-coding RNAs in human disease. Nat. Rev. Genet. 12 (2011), 861–874.
5. [5] Karikó, 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 23 (2005), 165–175.
6. [6] Fenton, O.S., Kauffman, K.J., McClellan, R.L., Appel, E.A., Dorkin, J.R., Tibbitt, M.W., Heartlein, M.W., DeRosa, F., Langer, R., Anderson, D.G., Bioinspired alkenyl amino alcohol ionizable lipid materials for highly potent in vivo mRNA delivery. Adv. Mater. 28:15 (2016), 2939–2943.
7. [7] Kauffman, K.J., Dorkin, J.R., Yang, J.H., Heartlein, M.W., DeRosa, F., Mir, F.F., Fenton, O.S., Anderson, D.G., Optimization of lipid nanoparticle formulations for mRNA delivery in vivo with fractional factorial and definitive screening designs. Nano Lett. 15 (2015), 7300–7306.
8. [8] Kauffman, K.J., Webber, M.J., Anderson, D.G., Materials for non-viral intracellular delivery of messenger RNA therapeutics. J. Control. Release, 2015.
9. [9] De Laporte, L., Shea, L.D., Matrices and scaffolds for DNA delivery in tissue engineering. Adv. Drug Deliv. Rev. 59 (2007), 292–307.
10. [10] Chevallay, B., Herbage, D., Collagen-based biomaterials as 3D scaffold for cell cultures: applications for tissue engineering and gene therapy. Med. Biol. Eng. Comput. 38 (2000), 211–218.
11. [11] Reckhenrich, A.K., Hopfner, U., Krötz, F., Zhang, Z., Koch, C., Kremer, M., Machens, H.-G., Plank, C., Egaña, J.T., Bioactivation of dermal scaffolds with a non-viral copolymer-protected gene vector. Biomaterials 32 (2011), 1996–2003.
12. [12] Scherer, F., Schillinger, U., Putz, U., Stemberger, A., Plank, C., Nonviral vector loaded collagen sponges for sustained gene delivery in vitro and in vivo. J. Gene Med. 4 (2002), 634–643.
13. [13] Geiger, F., Bertram, H., Berger, I., Lorenz, H., Wall, O., Eckhardt, C., Simank, H.G., Richter, W., Vascular endothelial growth factor gene-activated matrix (VEGF165-GAM) enhances osteogenesis and angiogenesis in large segmental bone defects. J. Bone Miner. Res. 20 (2005), 2028–2035.
14. [14] Franceschi, R., Biological approaches to bone regeneration by gene therapy. J. Dent. Res. 84 (2005), 1093–1103.
15. [15] Tierney, E.G., Duffy, G.P., Cryan, S.-A., Curtin, C.M., O'Brien, F.J., Non-viral gene-activated matrices: next generation constructs for bone repair. Organogenesis 9 (2013), 22–28.
16. [16] Balmayor, E.R., Geiger, J.P., Aneja, M.K., Berezhanskyy, T., Utzinger, M., Mykhaylyk, O., Rudolph, C., Plank, C., Chemically modified RNA induces osteogenesis of stem cells and human tissue explants as well as accelerates bone healing in rats. Biomaterials 87 (2016), 131–146.
17. [17] Elangovan, S., Khorsand, B., Do, A.-V., Hong, L., Dewerth, A., Kormann, M., Ross, R.D., Sumner, D.R., Allamargot, C., Salem, A.K., Chemically modified RNA activated matrices enhance bone regeneration. J. Control. Release 218 (2015), 22–28.
18. [18] Jesorka, A., Orwar, O., Liposomes: technologies and analytical applications. Annu. Rev. Anal. Chem. 1 (2008), 801–832.
19. [19] Balmayor, E.R., Flicker, M., Käser, T., Saalmüller, A., Erben, R.G., Human placental alkaline phosphatase as a tracking marker for bone marrow mesenchymal stem cells. BioResearch open access 2 (2013), 346–355.
20. [20] Wolbank, S., Peterbauer, A., Fahrner, M., Hennerbichler, S., Van Griensven, M., Stadler, G., Redl, H., Gabriel, C., Dose-dependent immunomodulatory effect of human stem cells from amniotic membrane: a comparison with human mesenchymal stem cells from adipose tissue. Tissue Eng. 13 (2007), 1173–1183.
21. [21] Caron, G., Duluc, D., Frémaux, I., Jeannin, P., David, C., Gascan, H., Delneste, Y., Direct stimulation of human T cells via TLR5 and TLR7/8: flagellin and R-848 up-regulate proliferation and IFN-γ production by memory CD4 + T cells. J. Immunol. 175 (2005), 1551–1557.
22. [22] Ceuppens, J., Baroja, M., Lorre, K., Van Damme, J., Billiau, A., Human T cell activation with phytohemagglutinin. The function of IL-6 as an accessory signal. J. Immunol. 141 (1988), 3868–3874.
23. [23] Erben, R.G., Embedding of bone samples in methylmethacrylate: an improved method suitable for bone histomorphometry, histochemistry, and immunohistochemistry. J. Histochem. Cytochem. 45 (1997), 307–313.
24. [24] Erben, R.G., Glosmann, M., Histomorphometry in rodents. Methods Mol. Biol. 816 (2012), 279–303.
25. [25] Kannan, V., Balabathula, P., Thoma, L.A., Wood, G.C., Effect of sucrose as a lyoprotectant on the integrity of paclitaxel-loaded liposomes during lyophilization. J. Lipid Res., 2014, 1–9.
26. [26] Dohmen, C., Plank, C., Rudolph, C., Koch, C., Compositions for Introducing RNA Into Cells, in, Google Patents. 2014.
27. [27] Anderson, M., Omri, A., The effect of different lipid components on the in vitro stability and release kinetics of liposome formulations. Drug Deliv. 11 (2004), 33–39.
28. [28] Liang, X., Mao, G., Ng, K.S., Mechanical properties and stability measurement of cholesterol-containing liposome on mica by atomic force microscopy. J. Colloid Interface Sci. 278 (2004), 53–62.
29. [29] Meinel, L., Hofmann, S., Betz, O., Fajardo, R., Merkle, H.P., Langer, R., Evans, C.H., Vunjak-Novakovic, G., Kaplan, D.L., Osteogenesis by human mesenchymal stem cells cultured on silk biomaterials: comparison of adenovirus mediated gene transfer and protein delivery of BMP-2. Biomaterials 27 (2006), 4993–5002.
30. [30] Kim, J., Kim, I.S., Cho, T.H., Lee, K.B., Hwang, S.J., Tae, G., Noh, I., Lee, S.H., Park, Y., Sun, K., Bone regeneration using hyaluronic acid-based hydrogel with bone morphogenic protein-2 and human mesenchymal stem cells. Biomaterials 28 (2007), 1830–1837.
31. [31] Lee, J.W., Kang, K.S., Lee, S.H., Kim, J.-Y., Lee, B.-K., Cho, D.-W., Bone regeneration using a microstereolithography-produced customized poly (propylene fumarate)/diethyl fumarate photopolymer 3D scaffold incorporating BMP-2 loaded PLGA microspheres. Biomaterials 32 (2011), 744–752.
32. [32] Dimitriou, R., Tsiridis, E., Giannoudis, P.V., Current concepts of molecular aspects of bone healing. Injury 36 (2005), 1392–1404.
33. [33] Damoiseaux, J., Döpp, E., Calame, W., Chao, D., MacPherson, G., Dijkstra, C., Rat macrophage lysosomal membrane antigen recognized by monoclonal antibody ED1. Immunology, 83, 1994, 140.
34. [34] Lüllmann-Rauch, R., Histologie. De Boeck Supérieur, 2008.
35. [35] Domb, A.J., Kumar, N., Biodegradable Polymers in Clinical Use and Clinical Development. 2011, John Wiley & Sons.
36. [36] Mueller-Klieser, W., Three-dimensional cell cultures: from molecular mechanisms to clinical applications. Am. J. Phys. Cell Phys. 273 (1997), C1109–C1123.
37. [37] Klug, W.S., Cummings, M.R., Spencer, C.A., Palladino, M.A., Concepts of Genetics. Benjamin Cummings, 2014.
38. [38] Mountziaris, P.M., Mikos, A.G., Modulation of the inflammatory response for enhanced bone tissue regeneration. Tissue Eng. B Rev. 14 (2008), 179–186.
39. [39] Xu, F., Shi, J., Yu, B., Ni, W., Wu, X., Gu, Z., Chemokines mediate mesenchymal stem cell migration toward gliomas in vitro. Oncol. Rep. 23 (2010), 1561–1567.
40. [40] Gonzalez-Fernandez, B.S.T., McCarthy, H.O., Dunne, N.J., O′Brien, F., Kelly, D.J., MSC fate following non-viral transfection with therapeutic genes strongly depends on the choice of gene delivery vector. Eur. Cell. Mater. 31 (2016), 110–111.
41. [41] Keibl, C., Fügl, A., Zanoni, G., Tangl, S., Wolbank, S., Redl, H., Van Griensven, M., Human adipose derived stem cells reduce callus volume upon BMP-2 administration in bone regeneration. Injury 42 (2011), 814–820.