Bone regeneration by regulated in vivo gene transfer using biocompatible polyplex nanomicelles

Molecular Therapy

Volumn 15, Issue 9, 2007, Pages 1655-1662

  Itaka, Keiji ^a   Ohba, Shinsuke ^a   Miyata, Kanjiro ^a   Kawaguchi, Hiroshi ^b   Nakamura, Kozo ^b   Takato, Tsuyoshi ^b   Chung, Ung Il ^a   Kataoka, Kazunori ^a  

^a UNIVERSITY OF TOKYO   (Japan)

^b UNIVERSITY OF TOKYO   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

ACTIVIN RECEPTOR LIKE KINASE 6; AZIRIDINE DERIVATIVE; BIOMATERIAL; CALCIUM PHOSPHATE; CEMENT; FUGENE6; MACROGOL; OSTEOGENIC PROTEIN 1; PLASMID DNA; POLY(ETHYLENIMINE); PROTEIN TYROSINE KINASE; TRANSCRIPTION FACTOR RUNX2; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BONE REGENERATION; CELL BASED GENE THERAPY; DRUG SAFETY; GENE DELIVERY SYSTEM; GENE EXPRESSION PROFILING; GENE TRANSFER; GENETIC TRANSFECTION; IMMUNE RESPONSE; IN VIVO STUDY; MATRIX ATTACHMENT REGION; MOUSE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; OSSIFICATION; SKULL DEFECT;

ANIMALS; BIOCOMPATIBLE MATERIALS; BONE MORPHOGENETIC PROTEIN RECEPTORS, TYPE I; BONE REGENERATION; CELL LINE; CORE BINDING FACTOR ALPHA 1 SUBUNIT; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; MICE; MICELLES; NANOTECHNOLOGY; POLYETHYLENE GLYCOLS; POLYETHYLENEIMINE;

EID: 34548065400     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/sj.mt.6300218     Document Type: Article

References (49)

1. Bucholz, RW, Heckman, JD (2006). Rockwood and Green's Fractures in Adults. Lippincott Williams & Wilkins: Philadelphia, PA. 587pp.
2. Banwart, JC, Asher, MA and Hassanein, RS (1995). Iliac crest bone graft harvest donor site morbidity. A statistical evaluation. Spine 20: 1055-1060.
3. Arrington, ED, Smith, WJ, Chambers, HG, Bucknell, AL and Davino, NA (1996). Complications of iliac crest bone graft harvesting. Clin Orthop Relat Res 329: 300-309.
4. Langer, R and Vacanti, JP (1993). Tissue engineering. Science 260: 920-926.
5. Bruder, SP and Caplan, AI (2000). Principles of Tissue Engineering Academic Press: San Diego, CA. 683pp.
6. Winn, SR, Hu, Y, Sfeir, C and Hollinger, JO (2000). Gene therapy approaches for modulating bone regeneration. Adv Drug Deliv Rev 42: 121-138.
7. Baltzer, AW and Lieberman, JR (2004). Regional gene therapy to enhance bone repair. Gene Ther 11: 344-350.
8. Katagiri, T and Takahashi, N (2002). Regulatory mechanisms of osteoblast and osteoclast differentiation. Oral Dis 8: 147-159.
9. Long, F, Chung, UI, Ohba, S, McMahon, J, Kronenberg, HM and McMahon, AP (2004). Ihh signaling is directly required for the osteoblast lineage in the endochondral skeleton. Development 131: 1309-1318.
10. Komori, T (2003). Requisite roles of Runx2 and Cbfb in skeletal development. J Bone Miner Metab 21: 193-197.
11. Patel, MS and Karsenty, G (2002). Regulation of bone formation and vision by LRPS. N Engl J Med 346: 1572-1574.
12. Ogata, N, Chikazu, D, Kubota, N, Terauchi, Y, Tobe, K, Azuma, Y et al. (2000). Insulin receptor substrate-1 in osteoblast is indispensable for maintaining bone turnover. J Clin Invest 105 935-943.
13. Govender, S, Csimma, C, Genant, HK, Valentin-Opran, A, Amit, Y, Arbel, R et al. (2002). Recombinant human bone morphogenetic protein-2 for treatment of open tibial fractures: A prospective, controlled, randomized study of four hundred and fifty patients. J Bone Joint Surg Am 84-A: 2123-2134.
14. Valentin-Opran, A, Wozney. J, Csimma, C, Lilly, L and Riedel, GE (2002). Clinical evaluation of recombinant human bone morphogenetic protein-2. Clin Orthop Relat Res 395: 110-120.
15. Gerstenfeld, LC, Cullinane, DM, Barnes, GL, Graves, DT and Einhorn, TA (2003). Fracture healing as a post-natal developmental process: molecular, spatial, and temporal aspects of its regulation. J Cell Biochem 88: 873-884.
16. Howell, TH, Fiorellini, J, Jones, A, Alder, M, Nummikoski, P, Lazaro, M et al. (1997). A feasibility study evaluating rhBMP-2/absorbable collagen sponge device for local alveolar ridge preservation or augmentation. Int J Periodontics Restorative Dent 17: 124-139.
17. Zhao, M, Zhao, Z, Koh, JT, Jin, T and Franceschi, RT (2005). Combinatorial gene therapy for bone regeneration: Cooperative interactions between adenovirus vectors expressing bone morphogenetic proteins 2, 4, and 7. J Cell Biochem 95: 1-16.
18. Riew, KD, Wright, NM, Cheng, S, Avioli, LV and Lou, J (1998). Induction of bone formation using a recombinant adenoviral vector carrying the human BMP-2 gene in a rabbit spinal fusion model. Calcif Tissue Int 63: 357-360.
19. Rutherford, RB, Moalli, M, Franceschi, RT, Wang, D, Gu, K and Krebsbach, PH (2002). Bone morphogenetic protein-transduced human fibroblasts convert to osteoblasts and form bone in vivo. Tissue Eng 8: 441-452.
20. Schek, RM, Hollister, SJ and Krebsbach, PH (2004). Delivery and protection of adenoviruses using biocompatible hydrogels for localized gene therapy. Mol Ther 9, 130-138.
21. Gafni, Y, Pelled, G, Zilberman, Y, Turgeman, G, Apparailly. F, Yotvat, H et al. (2004). Gene therapy platform for bone regeneration using an exogenously regulated, AAV-2-based gene expression system. Mol Ther 9: 587-595.
22. Lundstrom, K (2003). Latest development in viral vectors for gene therapy. Trends Biotechnol 21: 117-122.
23. Egermann, M, Lill, CA, Griesbeck, K, Evans, CH, Robbins, PD, Schneider, E et al. (2006). Effect of BMP-2 gene transfer on bone healing in sheep. Gene Ther 13: 1290-1299.
24. Kanayama, N, Fukushima, S, Nishiyama, N, Itaka, K, Jang, WD, Miyata, K 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.
25. Harada-Shiba, M, Yamauchi, K, Harada, A, Takamisawa, I, Shimokado, K and Kataoka, K (2002). Polyion complex micelles as vectors in gene therapy - pharmacokinetics and in vivo gene transfer. Gene Ther 9 407-414.
26. Itaka, K, Harada, A, Nakamura, K, Kawaguchi, H and Kataoka, K (2002). Evaluation by fluorescence resonance energy transfer of the stability of nonviral gene delivery vectors under physiological conditions. Biomacromolecules 3: 841-845.
27. Itaka, K, Yamauchi, K, Harada, A, Nakamura, K, Kawaguchi, H and Kataoka, K (2003). Polyion complex micelles from plasmid DNA and poly(ethylene glycol)-poly(L-lysine) block copolymer as serum-tolerable polyplex system: Physicochemical properties of micelles relevant to gene transfection efficiency. Biomaterials 24: 4495-4506.
28. Boussif, O, Lezoualch, F, Zanta, MA, Mergny, MD, Scherman, D, Demeneix, B et al. (1995). A versatile vector for gene and oligonucleotice transfer into cells in culture and in vivo: Polyethylenimine. Proc Natl Acad Sci USA 92: 7297-7301.
29. Hellgren, I, Drvota, V, Pieper, R, Enoksson, S, Blomberg, P. Islam, KB et al. (2000). Highly efficient cell-mediated gene transfer using non-viral vectors and FuGene6: in vitro and in vivo studies. Cell Mol Life Sci 57: 1326-1333.
30. Weiskirchen, R, Kneifel, J, Weiskirchen, S, van de Leur, E, Kunz, D and Gressner, AM (2000). Comparative evaluation of gene delivery devices in primary cultures of rat hepatic stellate cells and rat myofibroblasts. BMC Cell Biol 1: 4.
31. Lee, MJ, Cho, SS, You, JR, Lee, Y, Kang, BD, Choi, JS et al. (2002). Intraperitoneal gene delivery mediated by a novel cationic liposome in a peritoneal disseminated ovarian cancer model. Gene Ther 9: 859-866.
32. Elmadbouh, I, Rossignol, F, Meilhac, O, Vranckx, R, Pichon, C, Pouzet, B et al. (2004). Optimization of in vitro vascular cell transfection with non-viral vectors for in vivo applications. J Gene Med 6: 1112-1124.
33. Tinsley, RB, Faijerson, J and Eriksson, PS (2006). Efficient non-viral transfection of adult neural stem/progenitor cells, without affecting viability, proliferation or differentiation. J Gene Med 8: 72-81.
34. Ohba, S, Ikeda, T, Kugimiya, F, Yano, F, Lichtler, AC, Nakamura, K et al. (2007). Identification of a potent combination of osteogenic genes for bone regeneration using embryonic stem (ES) cell-based sensor. FASEB J (epub ahead of print).
35. Hunter, AC (2006). Molecular hurdles in polyfectin design and mechanistic background to polycation induced cytotoxicity. Adv Drug Deliv Rev 58: 1523-1531.
36. Bonadio, J, Smiley, E, Patil, P and Goldstein, S (1999). Localized, direct plasmid gene delivery in vivo: Prolonged therapy results in reproducible tissue regeneration. Nat Med 5: 753-759.
37. Verma, IM and Somia, N (1997). Gene therapy - promises, problems and prospects. Nature 389: 239-242.
38. Bright, C, Park, YS, Sieber, AN, Kostuik, JP and Leong, KW (2006). In vivo evaluation of plasmid DNA encoding OP-1 protein for spine fusion. Spine 31: 2163-2172.
39. Huang, YC, Simmons, C, Kaigler, D, Rice, KG and Mooney, DJ (2005). Bone regeneration in a rat cranial defect with delivery of PEI-condensed plasmid DNA encoding for bone morphogenetic protein-4 (BMP-4). Gene Ther 12: 418-426.
40. Moghimi, SM, Symonds, F, Murray, JC, Hunter, AC, Debska, G and Szewczyk, A (2005). A two-stage poly(ethylenimine)-mediated cytotoxicity: implications for gene transfer/therapy. Mol Ther 11: 990-995.
41. Geiger, F, Bertram, H, Berger, I, Lorenz, H, Wall, O, Eckhardt, C et al. (2005). Vascular endothelial growth factor gene-activated matrix (VEGF165-GAM) enhances osteogenesis and angiogenesis in large segmental bone defects. J Bone Miner Res 20: 2028-2035.
42. Omidi, Y, Hollins, AJ, Benboubetra, M, Drayton, R, Benter, IF and Akhtar, S (2003). Toxicogenomics of non-viral vectors for gene therapy: a microarray study of lipofectin- and oligofectamine-induced gene expression changes in human epithelial cells. J Drug Target 11 311-323.
43. Kabanov, AV, Batrakova, EV, Sriadibhatla, S, Yang, Z, Kelly, DL and Alakov, VY (2005). Polymer genomics: Shifting the gene and drug delivery paradigms. J Control Release 101: 259-271.
44. Buttery, LD, Bourne, S, Xynos, JD, Wood, H, Hughes, FJ, Hughes, SP et al. (2001). Differentiation of osteoblasts and in vitro bone formation from murine embryonic stem cells. Tissue Eng 7: 89-99.
45. Jiang, Y, Vaessen, B, Lenvik, T, Blackstad, M, Reyes, M and Verfaillie, CM (2002). Multipotent progenitor cells can be isolated from postnatal murine bone marrow, muscle, and brain. Exp Hematol 30: 896-904.
46. Seto, H, Kamekura, S, Miura, T, Yamamoto, A, Chikuda, H, Ogata, T et al. (2004). Distinct roles of Smad pathways and p38 pathways in cartilage-specific gene expression in synovial fibroblasts. J Clin Invest 113: 718-726.
47. Hirata, K, Tsukazaki, T, Kadowaki, A, Furukawa, K, Shibata, Y, Moriishi, T et al. (2003). Transplantation of skin fibroblasts expressing BMP-2 promotes bone repair more effectively than those expressing Runx2. Bone 32: 502-512.
48. Cowan, CM, Shi, YY, Aalami, OO, Chou, YF, Mari, C, Thomas, R et al. (2004). Adipose-derived adult stromal cells heal critical-size mouse calvarial defects. Nat Biotechnol 22: 560-567.
49. Kugimiya, F, Kawaguchi, H, Kamekura, S, Chikuda, H, Ohba, S, Yano, F et al. (2005). Involvement of endogenous bone morphogenetic protein (BMP) 2 and BMP6 in bone formation. J Biol Chem 280: 35704-35712.