Constitutive and inducible co-expression systems for non-viral osteoinductive gene therapy

European Cells and Materials

Volumn 27, Issue , 2014, Pages 166-184

  Feichtinger, Georg A ^a   Hofmann, A T ^a   Wassermann, K ^a   Zimmermann, A ^a   van Griensven, M ^a,b   Redl, H ^a  

^a LUDWIG BOLTZMANN INSTITUTE FOR EXPERIMENTAL AND CLINICAL TRAUMATOLOGY   (Austria)

^b TECHNICAL UNIVERSITY OF MUNICH   (Germany)

Author keywords

BMP2 7; Bone morphogenetic protein; Co expression; Heterodimer; Inducible; Intramuscular; Non viral gene therapy

Indexed keywords

BIOMATERIAL; BONE MORPHOGENETIC PROTEIN 2; DOXYCYCLINE; NAKED DNA; OSTEOCALCIN; OSTEOGENIC PROTEIN 1; RECOMBINANT GROWTH FACTOR;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; COMPUTER ASSISTED TOMOGRAPHY; CONTROLLED STUDY; GENE CASSETTE; GENE EXPRESSION; GENE TRANSFER; HISTOPATHOLOGY; IN VITRO STUDY; IN VIVO STUDY; LEG MUSCLE; MOUSE; MUSCLE TISSUE; NONHUMAN; NONVIRAL GENE THERAPY; OSSIFICATION; OSTEOCLASTOGENESIS; REPORTER GENE; TISSUE REGENERATION; TRANSGENE;

ANIMALS; BONE MORPHOGENETIC PROTEIN 2; BONE MORPHOGENETIC PROTEIN 7; CELL LINE; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MESENCHYMAL STROMAL CELLS; MICE; OSTEOBLASTS; OSTEOCALCIN; OSTEOGENESIS; TRANSCRIPTIONAL ACTIVATION;

EID: 84896716293     PISSN: 14732262     EISSN: None     Source Type: Journal    
DOI: 10.22203/eCM.v027a13     Document Type: Article

References (78)

1. Andersen CR, Nielsen LS, Baer A, Tolstrup AB, Weilguny D (2011) Efficient expression from one CMV enhancer controlling two core promoters. Mol Biotechnol 48: 128-137.
2. Andre FM, Cournil-Henrionnet C, Vernerey D, Opolon P, Mir LM (2006) Variability of naked DNA expression after direct local injection: the influence of the injection speed. Gene Ther 13: 1619-1627.
3. Banfi A, von Degenfeld G, Gianni-Barrera R, Reginato S, Merchant MJ, McDonald DM, Blau HM (2012) Therapeutic angiogenesis due to balanced single-vector delivery of VEGF and PDGF-BB. FASEB J 26: 2486-2497.
4. Baron U Freundlieb S, Gossen M, Bujard H (1995) Co-regulation of two gene activities by tetracycline via a bidirectional promoter. Nucleic Acids Res 23: 3605-3606.
5. Bleiziffer O, Eriksson E, Yao F, Horch F, Kneser U (2007) Gene transfer strategies in tissue engineering. J Cell Mol Med 11: 206-223.
6. Bonadio J, Smiley E, Patil P, Goldstein S (1999) Localized, direct plasmid gene delivery in vivo: prolonged therapy results in reproducible tissue regeneration. Nat Med 5: 753-759.
7. Callen BP, Shearwin KE, Egan JB (2004) Transcriptional interference between convergent promoters caused by elongation over the promoter. Mol Cell 14: 647-656.
8. Cawthorne C, Swindell R, Stratford IJ, Dive C, Welman A (2007) Comparison of doxycycline delivery methods for Tet-inducible gene expression in a subcutaneous xenograft model. J Biomol Tech 18: 120-123.
9. Chakrabarti S, Joffe S, Seidman MM (1985) Recombination and deletion of sequences in shuttle vector plasmids in mammalian cells. Mol Cell Biol 5: 2265-2271.
10. Chakrabarti S, Seidman MM (1986) Intramolecular recombination between transfected repeated sequences in mammalian cells is nonconservative. Mol Cell Biol 6: 2520-2526.
11. Chen ZY, He CY, Ehrhardt A, Kay MA (2003) Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol Ther 8: 495-500.
12. Coelho-Castelo AA, Trombone AP, Rosada RS, Santos RR, Jr., Bonato VL, Sartori A, Silva CL (2006) Tissue distribution of a plasmid DNA encoding Hsp65 gene is dependent on the dose administered through intramuscular delivery. Genetic Vaccines Ther 4: 1.
13. Curtin JA, Dane AP, Swanson A, Alexander IE, Ginn SL (2008) Bidirectional promoter interference between two widely used internal heterologous promoters in a late-generation lentiviral construct. Gene Ther 15: 384-390.
14. Degnin C, Jean F, Thomas G, Christian JL (2004) Cleavages within the prodomain direct intracellular trafficking and degradation of mature bone morphogenetic protein-4. Mol Biol Cell 15: 5012-5020.
15. Einhorn T (2003) Clinical applications of recombinant human BMPs: Early experience and future development. J Bone Joint Surg 85-A: 82-88.
16. Feichtinger GA, Hofmann AT, Slezak P, Schuetzenberger S, Kaipel M, Schwartz E, Neef AB, Nomikou N, Nau T, van Griensven M, McHale AP, Redl H (2014) Sonoporation increases therapeutic efficacy of inducible and constitutive BMP2/7 in vivo gene delivery. Hum Gene Ther Meth 25: 57-71.
17. Feichtinger GA, Morton TJ, Zimmermann A, Dopler D, Banerjee A, Redl H, van Griensven M (2010) Enhanced reporter gene assay for the detection of osteogenic differentiation. Tissue Eng Part C Methods 17: 401-410.
18. Franceschi RT (2005) Biological approaches to bone regeneration by gene therapy. J Dent Res 84: 1093-1103.
19. Gehl J (2003) Electroporation: theory and methods, perspectives for drug delivery, gene therapy and research. Acta Physiol Scand 177: 437-447.
20. Gelehrter T, Collins F, Ginsburg D (1998) Gene therapy. In: Principles of Medical Genetics (Kelly P, ed), Williams & Wilkins, Baltimore, MD, pp 311-328.
21. Gossen M, Bujard H (1992) Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc Natl Acad Sci USA 89: 5547-5551.
22. Gossen M, Freundlieb S, Bender G, Muller G, Hillen W, Bujard H (1995) Transcriptional activation by tetracyclines in mammalian cells. Science 268: 1766-1769.
23. Goverdhana S, Puntel M, Xiong W, Zirger JM, Barcia C, Curtin JF, Soffer EB, Mondkar S, King GD, Hu J, Sciascia SA, Candolfi M, Greengold DS, Lowenstein PR, Castro MG (2005) Regulatable gene expression systems for gene therapy applications: progress and future challenges. Mol Ther 12: 189-211.
24. Grossin L, Cournil-Henrionnet C, Mir LM, Liagre B, Dumas D, Etienne S, Guingamp C, Netter P, Gillet P (2003) Direct gene transfer into rat articular cartilage by in vivo electroporation. FASEB J 17: 829-835.
25. Guo-ping W, Xiao-chuan H, Zhi-hui Y, Li G (2010) Influence on the osteogenic activity of the human bone marrow mesenchymal stem cells transfected by liposome-mediated recombinant plasmid pIRES-hBMP2-hVEGF165 in vitro. Ann Plastic Surg 65: 80-84.
26. Gurtu V, Yan G, Zhang G (1996) IRES bicistronic expression vectors for efficient creation of stable mammalian cell lines. Biochem Biophys Res Commun 229: 295-298.
27. Hengge UR, Dexling B, Mirmohammadsadegh A (2001) Safety and pharmacokinetics of naked plasmid DNA in the skin: studies on dissemination and ectopic expression. J Invest Dermatol116: 979-982.
28. Herweijer H, Wolff JA (2003) Progress and prospects: naked DNA gene transfer and therapy. Gene Ther 10: 453-458.
29. Hohlweg U, Doerfler W (2001) On the fate of plant or other foreign genes upon the uptake in food or after intramuscular injection in mice. Mol Genet Genomics 265: 225-233.
30. Israel DI, Nove J, Kerns KM, Kaufman RJ, Rosen V, Cox KA, Wozney JM (1996) Heterodimeric bone morphogenetic proteins show enhanced activity in vitro and in vivo. Growth Factors 13: 291-300.
31. Johnson E, Urist M (2000) Human bone morphogenetic protein allografting for reconstruction of femoral nonunion. Clin Orthopaed 371: 61-74.
32. Kaito T, Johnson J, Ellerman J, Tian H, Aydogan M, Chatsrinopkun M, Ngo S, Choi C, Wang JC (2013) Synergistic effect of bone morphogenetic proteins 2 and 7 by ex vivo gene therapy in a rat spinal fusion model. J Bone Joint Surg Am 95: 1612-1619.
33. Katagiri T, Akiyama S, Namiki M, Komaki M, Yamaguchi A, Rosen V, Wozney JM, Fujisawa-Sehara A, Suda T (1997) Bone morphogenetic protein-2 inhibits terminal differentiation of myogenic cells by suppressing the transcriptional activity of MyoD and myogenin. Exp Cell Res 230: 342-351.
34. Katagiri T Yamaguchi A, Komaki M, Abe E, Takahashi N, Ikeda T, Rosen V, Wozney JM, Fujisawa-Sehara A, Suda T (1994) Bone morphogenetic protein-2 converts the differentiation pathway of C2C12 myoblasts into the osteoblast lineage. J Cell Biol 127: 1755-1766.
35. Kawai M, Bessho K, Maruyama H, Miyazaki J, Yamamoto T (2006) Simultaneous gene transfer of bone morphogenetic protein (BMP)-2 and BMP-7 by in vivo electroporation induces rapid bone formation and BMP-4 expression. BMC Musculoskelet Disord 7: 62.
36. Kawai M, Maruyama H, Bessho K, Yamamoto H, Miyazaki J, Yamamoto T (2009) Simple strategy for bone regeneration with a BMP-2/7 gene expression cassette vector. Biochem Biophys Res Commun 390: 1012-1017.
37. Kerrigan JJ, Xie Q, Ames RS, Lu Q (2011) Production of protein complexes via co-expression. Protein Expr Purif 75: 1-14.
38. Kusumanto YH, Mulder NH, Dam WA, Losen M, De Baets MH, Meijer C, Hospers GA (2007) Improvement of in vivo transfer of plasmid DNA in muscle: comparison of electroporation versus ultrasound. Drug Deliv 14: 273-277.
39. Le Guiner C, Stieger K, Snyder RO, Rolling F, Moullier P (2007) Immune responses to gene product of inducible promoters. Curr Gene Ther 7: 334-346.
40. Ledwith BJ, Manam S, Troilo PJ, Barnum AB, Pauley CJ, Griffiths TG 2nd, Harper LB, Schock HB, Zhang H, Faris JE, Way PA, Beare CM, Bagdon WJ, Nichols WW (2000) Plasmid DNA vaccines: assay for integration into host genomic DNA. Dev Biol 104: 33-43.
41. Martin T, Parker SE, Hedstrom R, Le T, Hoffman SL, Norman J, Hobart P, Lew D (1999) Plasmid DNA malaria vaccine: the potential for genomic integration after intramuscular injection. Human Gene Ther 10: 759-768.
42. McMahon JM, Wells DJ (2004) Electroporation for gene transfer to skeletal muscles: current status. BioDrugs 18: 155-165.
43. Michlits W, Mittermayr R, Schafer R, Redl H, Aharinejad S (2007) Fibrin-embedded administration of VEGF plasmid enhances skin flap survival. Wound Repair Regen 15: 360-367.
44. Mirabella R, Franken C, van der Krogt GN, Bisseling T, Geurts R (2004) Use of the fluorescent timer DsRED-E5 as reporter to monitor dynamics of gene activity in plants. Plant Physiol 135: 1879-1887.
45. Mittermayr R, Morton T, Hofmann M, Helgerson S, van Griensven M, Redl H (2008) Sustained (rh)VEGF(165) release from a sprayed fibrin biomatrix induces angiogenesis, up-regulation of endogenous VEGF-R2, and reduces ischemic flap necrosis. Wound Repair Regen 16: 542-550.
46. Miyazaki J, Takaki S, Araki K, Tashiro F, Tominaga A, Takatsu K, Yamamura K (1989) Expression vector system based on the chicken beta-actin promoter directs efficient production of interleukin-5. Gene 79: 269-277.
47. Ngo V, Laverriere JN, Gourdji D (1993) Binding capacity and cis-acting efficiency of DNA regulatory sequences can be distinguished in an in vivo competition assay. Nucleic Acids Res 21: 5795-5796.
48. Niwa H, Yamamura K, Miyazaki J (1991) Efficient selection for high-expression transfectants with a novel eukaryotic vector. Gene 108: 193-199.
49. Osawa K, Okubo Y, Nakao K, Koyama N, Bessho K (2009) Osteoinduction by microbubble-enhanced transcutaneous sonoporation of human bone morphogenetic protein-2. J Gene Med 11: 633-641.
50. Osawa K, Okubo Y, Nakao K, Koyama N, Bessho K (2010) Osteoinduction by repeat plasmid injection of human bone morphogenetic protein-2. J Gene Med 12: 937-944.
51. Perez N, Plence P, Millet V, Greuet D, Minot C, Noel D, Danos O, Jorgensen C, Apparailly F (2002) Tetracycline transcriptional silencer tightly controls transgene expression after in vivo intramuscular electrotransfer: application to interleukin 10 therapy in experimental arthritis. Hum Gene Ther 13: 2161-2172.
52. Puttini S, Beggah AT, Ouvrard-Pascaud A, Legris C, Blot-Chabaud M, Farman N, Jaisser F (2001) Tetracycline-inducible gene expression in cultured rat renal CD cells and in intact CD from transgenic mice. Am J Physiol 281: F1164-1172.
53. Rauh G, Pieczek A, Irwin W, Schainfeld R, Isner JM (2001) In vivo analysis of intramuscular gene transfer in human subjects studied by on-line ultrasound imaging. Hum Gene Ther 12: 1543-1549.
54. Robert MA, Lin Y, Bendjelloul M, Zeng Y, Dessolin S, Broussau S, Larochelle N, Nalbantoglu J, Massie B, Gilbert R (2012) Strength and muscle specificity of a compact promoter derived from the slow troponin I gene in the context of episomal (gutless adenovirus) and integrating (lentiviral) vectors. J Gene Med 14: 746-760.
55. Rubnitz J, Subramani S (1985) Rapid assay for extrachromosomal homologous recombination in monkey cells. Mol Cell Biol 5: 529-537.
56. Satkauskas S, Bureau MF, Mahfoudi A, Mir LM (2001) Slow accumulation of plasmid in muscle cells: supporting evidence for a mechanism of DNA uptake by receptor-mediated endocytosis. Mol Ther 4: 317-323.
57. Schertzer JD, Plant DR, Lynch GS (2006) Optimizing plasmid-based gene transfer for investigating skeletal muscle structure and function. Mol Ther 13: 795-803.
58. Schmittgen TD, Livak KJ (2008) Analyzing real-time PCR data by the comparative C(T) method. Nature Protoc 3: 1101-1108.
59. Schwake G, Youssef S, Kuhr JT, Gude S, David MP, Mendoza E, Frey E, Radler JO (2010) Predictive modeling of non-viral gene transfer. Biotechnol Bioeng 105: 805-813.
60. Scott MA, Levi B, Askarinam A, Nguyen A, Rackohn T, Ting K, Soo C, James AW (2012) Brief review of models of ectopic bone formation. Stem Cells Dev 21: 655-667.
61. Shearwin KE, Callen BP, Egan JB (2005) Transcriptional interference--a crash course. Trends Genet 21: 339-345.
62. Steinert AF, Palmer GD, Pilapil C, Noth U, Evans CH, Ghivizzani SC (2009) Enhanced in vitro chondrogenesis of primary mesenchymal stem cells by combined gene transfer. Tissue Eng Part A 15: 1127-1139.
63. Sundararaman S, Miller TJ, Pastore JM, Kiedrowski M, Aras R, Penn MS (2011) Plasmid-based transient human stromal cell-derived factor-1 gene transfer improves cardiac function in chronic heart failure. Gene Ther 18: 867-873.
64. Tepper OM, Mehrara BJ (2002) Gene therapy in plastic surgery. Plast Reconstruct Surg 109: 716-734.
65. Timme TL, Wood CM, Moses RE (1989) Intermolecular plasmid recombination in fibroblasts from humans with DNA damage-processing defects. Plasmid 22: 1-9.
66. Tolmachov O (2009) Designing plasmid vectors. Meth Mol Biol 542: 117-129.
67. Tolmachov OE (2011) Building mosaics of therapeutic plasmid gene vectors. Curr Gene Ther 11: 466-478.
68. Verkhusha VV, Kuznetsova IM, Stepanenko OV, Zaraisky AG, Shavlovsky MM, Turoverov KK, Uversky VN (2003) High stability of Discosoma DsRed as compared to Aequorea EGFP. Biochemistry 42: 7879-7884.
69. Voon DC, Subrata LS, Baltic S, Leu MP, Whiteway JM, Wong A, Knight SA, Christiansen FT, Daly JM (2005) Use of mRNA-and protein-destabilizing elements to develop a highly responsive reporter system. Nucleic Acids Res 33: e27.
70. Wan R, Hu J, Zhou Q, Wang J, Liu P, Wei Y (2012) Application of co-expressed genes to articular cartilage: new hope for the treatment of osteoarthritis (review). Mol Med Rep 6: 16-18.
71. Warnke PH, Springer IN, Wiltfang J, Acil Y, Eufinger H, Wehmoller M, Russo PA, Bolte H, Sherry E, Behrens E, Terheyden H (2004) Growth and transplantation of a custom vascularised bone graft in a man. Lancet 364: 766-770.
72. Wolff JA, Budker V (2005) The mechanism of naked DNA uptake and expression. Adv Genet 54: 3-20.
73. Yew NS (2005) Controlling the kinetics of transgene expression by plasmid design. Adv Drug Deliv Rev 57: 769-780.
74. Zhang X, Yang M, Lin L, Chen P, Ma KT, Zhou CY, Ao YF. (2006) Runx2 overexpression enhances osteoblastic differentiation and mineralization in adipose--derived stem cells in vitro and in vivo. Calcif Tissue Int 79: 169-178.
75. Zhao M, Zhao Z, Koh JT, Jin T, 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.
76. Zheng Y, Wu G, Zhao J, Wang L, Sun P, Gu Z (2010) rhBMP2/7 heterodimer: an osteoblastogenesis inducer of not higher potency but lower effective concentration compared with rhBMP2 and rhBMP7 homodimers. Tissue Eng Part A 16: 879-887.
77. Zhu W, Rawlins BA, Boachie-Adjeu O, Myers ER, Arimizu J, Choi E, Liebermann JR, Crystal RG, Hidaka C (2004) Combined bone morphogenetic protein-2 and-7 gene transfer enhances osteoblastic differentiation and spine fusion in a rodent model. J Bone Miner Res 19: 2021-2032.
78. Kang JH, Chung JK (2008) Molecular-genetic imaging based on reporter gene expression. J Nucl Med 49 Suppl 2: 164S-179S.