Spine fusion by gene therapy

Gene Therapy

Volumn 11, Issue 4, 2004, Pages 360-367

  Yoon, S T ^a,b,c   Boden, S D ^a,b,c  

^a EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c ATLANTA VA MEDICAL CENTER   (United States)

Author keywords

BMP 2; BMP 7; Bone morphogenetic proteins; Bone regeneration; Growth factors; LMP 1; NeOsteo; Osteoporosis; Spine fusion

Indexed keywords

BONE MORPHOGENETIC PROTEIN; BONE MORPHOGENETIC PROTEIN 2; BONE MORPHOGENETIC PROTEIN 9; COMPLEMENTARY DNA; DRUG CARRIER; EFFECTENE; LIM PROTEIN; NEOSTEO; OP 1; OSTEOGENIC PROTEIN 1; PARATHYROID HORMONE[1-34]; REAGENT; RECOMBINANT BONE MORPHOGENETIC PROTEIN 2; RECOMBINANT CYTOKINE; RECOMBINANT OSTEOGENIC PROTEIN 1; SUPERFECT; TITANIUM; UNCLASSIFIED DRUG;

ARTHRODESIS; BONE GRAFT; DRUG APPROVAL; DRUG COST; DRUG EFFICACY; DRUG POTENCY; FOOD AND DRUG ADMINISTRATION; FRACTURE HEALING; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; HUMAN; IN VITRO STUDY; IN VIVO STUDY; LUMBAR SPINE; NONHUMAN; OSSIFICATION; PILOT STUDY; PRIORITY JOURNAL; RETROSPECTIVE STUDY; REVIEW; SPINE FUSION; UNITED STATES; VERTEBRA BODY;

ANIMALS; BONE MORPHOGENETIC PROTEINS; CARRIER PROTEINS; GENE THERAPY; GENETIC VECTORS; HUMANS; INTRACELLULAR SIGNALING PEPTIDES AND PROTEINS; OSTEOGENESIS; SPINAL FUSION;

EID: 1342327562     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3302203     Document Type: Review

References (75)

1. DePalma AF, Rothman RH. The nature of pseudarthrosis. Clin Orthop 1968; 59: 113-118.
2. Steinmann JC, Herkowitz HN. Pseudarthrosis of the spine. Clin Orthop 1992; 284: 80-90.
3. Zoma A et al. Surgical stabilisation of the rheumatoid cervical spine: a review of indications and results. J Bone Joint Surg 1987; 49-B: 8-12.
4. Ferlic DC et al. Surgical treatment of the symptomatic unstable cervical spine in rheumatoid arthritis. J Bone Joint Surg 1975; 57-A: 349-354.
5. Conaty JP, Mongan ES. Cervical fusion in rheumatoid arthritis. J Bone Joint Surg 1981; 63-A: 1218-1227.
6. West III JL, Bradford DS, Ogilvie JW. Results of spinal arthrodesis with pedicle screw plate fixation. J Bone Joint Surg 1991; 73-A: 1179-1184.
7. Bridwell KH et al. The role of fusion and instrumentation in the treatment of degenerative spondylolisthesis with spinal stenosis. J Spinal Disord 1993; 6: 461-472.
8. McGuire RA, Amundson GM. The use of primary internal fixation in spondylolisthesis. Spine 1993; 18: 1662-1672.
9. Zdeblick TA. A prospective, randomized study of lumbar fusion: Preliminary results. Spine 1993; 18: 983-991.
10. Brodsky AE, Kovalsky ES, Khalil MA. Correlation of radiographic assessment of lumbar spine fusions with surgical exploration. Spine 1991; 16: S261-S265.
11. Boden SD. Bone repair and enhancement clinical trial design. Spine applications. Clin Orthop 1998: S336-S346.
12. Schimandle JH, Boden SD. The use of animal models to study spinal fusion. Spine 1994; 19: 1998-2006.
13. Boden SD, Schimandle JH, Hutton WC. An experimental lumbar intertransverse process spinal fusion model: radiographic, histologic, and biomechanical healing characteristics. Spine 1995; 20: 412-420.
14. Urist MR. Bone: formation by autoinduction. Science 1965; 150: 893-899.
15. Urist MR, Strates BS. Bone morphogenetic protein. J Dent Res Suppl 1971; 50: 1392-1406.
16. Teixeira JOC, Urist MR. Bone morphogenetic protein induced repair of compartmentalized segmental diaphyseal defects. Arch Orthop Trauma Surg 1998; 117: 27-34.
17. Johnson EE, Urist MR, Finerman GAM. Resistant nonunions and partial or complete segmental defects of long bones: treatment with implants of a composite of human bone morphogenetic protein (BMP) and autolyzed, antigen-extracted, allogeneic (AAA) bone. Clin Orthop 1992; 277: 229-237.
18. Johnson EE, Urist MR, Finerman GAM. Bone morphogenetic protein augmentation grafting of resistant femoral nonunions: a preliminary report. Clin Orthop 1988; 230: 257-265.
19. Wozney JM et al. Novel regulators of bone formation: molecular clones and activities. Science 1988; 242: 1528-1534.
20. Ozkaynak E et al. OP-1 cDNA encodes an osteogenic protein in the TGF-b family. Eur Mol Biol Organ J 1990; 9: 2085-2093.
21. Sampath TK, Muthukumaran N, Reddi AH. Isolation of osteogenin, an extracellular matrix-associated, bone-inductive protein, by heparin affinity chromatography. Proc Natl Acad Sci USA 1987; 84: 7109-7113.
22. Boden SD, Schimandle JH, Hutton WC. 1995 Volvo Award in Basic Sciences. The use of an osteoinductive growth factor for lumbar spinal fusion. Part II: study of dose, carrier, and species. Spine 1995; 20: 2633-2644.
23. Martin GJ et al. Posterolateral intertransverse process spinal fusion arthrodesis with rhBMP-2 in a non-human primate-Important lessons learned regarding dose, carrier, and safety. J Spinal Disord 1999; 12: 179-186.
24. Boden SD et al. Laparoscopic anterior spinal arthrodesis with rhBMP-2 in a titanium interbody threaded cage. J Spinal Disord 1998; 11: 95-101.
25. Boden SD, Schimandle JH, Hutton WC. Evaluation of a bovine-derived osteoinductive bone protein in a non-human primate model of lumbar spinal fusion. Trans Orthop Res Soc 1996; 21: 118.
26. Boden SD et al. The use of rhBMP-2 in interbody fusion cages. Definitive evidence of osteoinduction in humans: a preliminary report. Spine 2000; 25: 376-381.
27. McKay B, Sandhu HS. Use of recombinant human bone morphogenetic protein-2 in spinal fusion applications. Spine 2002; 27: S66-S85.
28. Vaccaro AR, Anderson DG, Toth CA. Recombinant human osteogenic protein-1 (bone morphogenetic protein-7) as an osteoinductive agent in spinal fusion. Spine 2002; 27: S59-S65.
29. Damien CJ et al. Purified bovine BMP extract and collagen for spine arthrodesis: preclinical safety and efficacy. Spine 2002; 27: S50-S58.
30. Louis-Ugbo J et al. Retention of 125I-labeled recombinant human bone morphogenetic protein- 2 by biphasic calcium phosphate or a composite sponge in a rabbit posterolateral spine arthrodesis model. J Orthop Res 2002; 20: 1050-1059.
31. Muschler GF et al. Treatment of established fibial nonunions using recombinant osteogenic protein-1. Trans Am Acad Orthop Surg 1998; 65: 172-173.
32. Jeppsson C et al. OP-1 for cervical spine fusion: bridging bone in only 1 of 4 rheumatoid patients but prednisolone did not inhibit bone induction in rats. Acta Orthop Scand 1999; 70: 559-563.
33. Laursen M et al. Recombinant bone morphogenetic protein-7 as an intracorporal bone growth stimulator in unstable thoracolumbar burst fractures in humans: preliminary results. Eur Spine J 1999; 8: 485-490.
34. Fang J et al. Stimulation of new bone formation by direct transfer of osteogenic plasmid genes. Proc Natl Acad Sci USA 1996; 93: 5753-5758.
35. Lieberman JR et al. Regional gene therapy with a BMP-2-producing murine stromal cell line induces heterotopic and orthotopic bone formation in rodents. Journal of Orthopaedic Research 1998; 16: 330-339.
36. Okubo Y et al. In vitro and in vivo studies of a bone morphogenetic protein-2 expressing adenoviral vector. J Bone Joint Surg Am 2001; 83-A (Suppl 1): S99-104.
37. Bosch P et al. The efficiency of muscle-derived cell-mediated bone formation. Cell Transplant 2000; 9: 463-470.
38. Musgrave DS et al. Human skeletal muscle cells in ex vivo gene therapy to deliver bone morphogenetic protein-2. J Bone Joint Surg Br 2002; 84: 120-127.
39. Lou J et al. Gene therapy: adenovirus-mediated human bone morphogenetic protein-2 gene transfer induces mesenchymal progenitor cell proliferation and differentiation in vitro and bone formation in vivo. J Orthop Res 1999; 17: 43-50.
40. Partridge K et al. Adenoviral BMP-2 gene transfer in mesenchymal stem cells: in vitro and in vivo bone formation on biodegradable polymer scaffolds. Biochem Biophys Res Commun 2002; 292: 144-152.
41. Lee JY et al. Effect of bone morphogenetic protein-2-expressing muscle-derived cells on healing of critical-sized bone defects in mice. J Bone Joint Surg Am 2001; 83-A: 1032-1039.
42. Lee JY et al. Enhancement of bone healing based on ex vivo gene therapy using human muscle-derived cells expressing bone morphogenetic protein 2. Hum Gene Ther 2002; 13: 1201-1211.
43. Moutsatsos IK et al. Exogenously regulated stem cell-mediated gene therapy for bone regeneration. Mol Ther 2001; 3: 449-461.
44. Gazit D et al. Engineered pluripotent mesenchymal cells integrate and differentiate in regenerating bone: a novel cell-mediated gene therapy. J Gene Med 1999; 1: 121-133.
45. Lieberman JR et al. The effect of regional gene therapy with bone morphogenetic protein-2-producing bone-marrow cells on repair of segmental femoral defects in rats. J Bone Joint Surg 1999; 81-A: 905-917.
46. Riew KD et al. Induction of bone formation using a recombinant adenoviral vector carrying the human BMP-2 gene in a rabbit spinal fusion model. Calcif Tissue Int 1998; 63: 357-360.
47. Franceschi RT et al. Gene therapy for bone formation: in vitro and in vivo osteogenic activity of an adenovirus expressing BMP7. J Cell Biochem 2000; 78: 476-486.
48. Rutherford RB et al. Bone morphogenetic protein-transduced human fibroblasts convert to osteoblasts and form bone in vivo. Tissue Eng 2002; 8: 441-452.
49. Breitbart AS et al. Gene-enhanced tissue engineering: applications for bone healing using cultured periosteal cells transduced retrovirally with the BMP-7 gene. Ann Plast Surg 1999; 42: 488-495.
50. Dumont RJ et al. Ex vivo bone morphogenetic protein-9 gene therapy using human mesenchymal stem cells induces spinal fusion in rodents. Neurosurgery 2002; 51: 1239-1245.
51. Musgrave DS et al. Adenovirus-mediated direct gene therapy with bone morophogenetic protein-2 produces bone. Bone 1999; 24: 541-547.
52. Alden TD et al. In vivo endochondral bone formation using a bone morphogenetic protein 2 adenoviral vector. Hum Gene Ther 1999; 10: 2245-2253.
53. Alden TD et al. The use of bone morphogenetic protein gene therapy in craniofacial bone repair. J Craniofac Surg 2000; 11: 24-30.
54. Alden TD et al. Percutaneous spinal fusion using bone morphogenetic protein-2 gene therapy. J Neurosurg 1999; 90 (Spine 1): 109-114.
55. Boden SD et al. 1995 Volvo Award in Basic Sciences. The use of an osteoinductive growth factor for lumbar spinal fusion. Part I: the biology of spinal fusion. Spine 1995; 20: 2626-2632.
56. Boden SD, Sumner DR. Biologic factors affecting spinal fusion and bone regeneration. Spine 1995; 20: 102S-112S.
57. Liu Y et al. BMP-6 induces a novel LIM protein involved in bone mineralization and osteocalcin secretion. J Bone Min Res 1997; 12: S115.
58. Dawid IB, Breen JJ, Toyama R. LIM domains: multiple roles as adapters and functional modifiers in protein interactions. Trends Genet 1998; 14: 156-162.
59. Smith AE. Viral vectors in gene therapy. Annu Rev Microbiol 1995; 49: 807-838.
60. Wagner E et al. Transferrin-polycation conjugates as carriers for DNA uptake into cells. Proc Natl Acad Sci USA 1990; 87: 3410-3414.
61. Baltzer AWA et al. A gene therapy approach to accelerating bone healing. Evaluation of gene expression in a New Zealand white rabbit model. Knee Surg Sports Traumatol Arthrosc 1999; 7: 197-202.
62. Guzman RJ et al. In vivo suppression of injury-induced vascular smooth muscle cell accumulation using adenovirus-mediated transfer of the herpes simplex virus thymidine kinase gene. Proc Natl Acad Sci USA 1994; 91: 10732-10736.
63. Watanabe T et al. Enhancement of adenovirus-mediated gene transfer to human bone marrow cells. Leukemia Lymphoma 1998; 29: 439-451.
64. Xiao W et al. Adeno-associated virus as a vector for liver-directed gene therapy. J Virol 1998; 72: 10222-10226.
65. Blomer U et al. Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector. J Virol 1997; 71: 6641-6649.
66. Goldman MJ et al. Lentiviral vectors for gene therapy of cystic fibrosis. Hum Gene Ther 1997; 10: 2261-2268.
67. Boden SD et al. LMP-1, A LIM-domain protein, mediates BMP-6 effects on bone formation. Endocrinology 1998; 139: 5125-5134.
68. Boden SD et al. 1998 Volvo Award in Basic Sciences: lumbar spine fusion by local gene therapy with a cDNA encoding a novel osteoinductive protein (LMP-1). Spine 1998; 23: 2486-2492.
69. Viggeswarapu M et al. Adenoviral delivery of LIM mineralization protein-1 induces new-bone formation in vitro and in vivo. J Bone Joint Surg Am 2001; 83-A: 364-376.
70. Kozak M. Possible role of flanking nucleotides in recognition of the AUG initiator codon by eukaryotic ribosomes. Nucleic Acids Res 1981; 9: 5233-5262.
71. Kozak M. Compilation and analysis of sequences upstream from the translational start site in eukaryotic mRNAs. Nucleic Acids Res 1984; 12: 857-872.
72. Kozak M. At least six nucleotides preceding the AUG initiator codon enhance translation in mammalian cells. J Mol Biol 1987; 196: 947-950.
73. Kozak M. Downstream secondary structure facilitates recognition of initiator codons by eukaryotic ribosomes. Proc Natl Acad Sci USA 1987; 87: 8301-8305.
74. Kozak M. Recognition of AUG and alternative initiator codons is augmented by G in position +4 but is not generally affected by the nucleotides in positions +5 and +6. EMBO J 1997; 16: 2482-2492.
75. Suh DY et al. Delivery of recombinant human bone morphogenetic protein-2 (rhBMP-2) using a compression resistant matrix in posterolateral spine fusion in the rabbit and non-human primate. Spine 2002; 27: 353-360.