Progress and Prospects: Genetic treatments for disorders of bones and joints

Gene Therapy

Volumn 16, Issue 8, 2009, Pages 944-952

  Evans, C H ^a   Ghivizzani, S C ^b   Robbins, P D ^c  

^a HARVARD MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^c UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; BONE MORPHOGENETIC PROTEIN 2; COMPLEMENTARY DNA; ETANERCEPT; IMMUNOGLOBULIN ENHANCER BINDING PROTEIN; INTERLEUKIN 1; INTERLEUKIN 1 RECEPTOR BLOCKING AGENT; INTERLEUKIN 10; INTERLEUKIN 4; LENTIVIRUS VECTOR; LIPOSOME; NITRATE REDUCTASE; RETROVIRUS VECTOR; SMALL INTERFERING RNA; SOMATOMEDIN C RECEPTOR; TRANSFORMING GROWTH FACTOR BETA1; TUMOR NECROSIS FACTOR;

ANKYLOSING SPONDYLITIS; ARTHRITIS; CD4+ T LYMPHOCYTE; CELL BASED GENE THERAPY; CELLULAR IMMUNITY; CLINICAL TRIAL; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG FATALITY; DRUG MECHANISM; DRUG SAFETY; DRUG STABILITY; FRACTURE HEALING; GENE EXPRESSION; GENE TARGETING; GENE THERAPY; GENETIC TRANSDUCTION; HUMAN; IMMUNE RESPONSE; LIPOSOMAL GENE DELIVERY SYSTEM; MESENCHYMAL STEM CELL; NONHUMAN; OSTEOARTHRITIS; PRIORITY JOURNAL; PROMOTER REGION; PROSTHESIS LOOSENING; REPEATED DRUG DOSE; REVIEW; RHEUMATOID ARTHRITIS; RNA INTERFERENCE; TISSUE REGENERATION; TISSUE REPAIR; TRANSGENE; VIRAL GENE DELIVERY SYSTEM;

ARTHRITIS; BONE DISEASES; CLINICAL TRIALS AS TOPIC; GENE THERAPY; GENE TRANSFER TECHNIQUES; JOINT DISEASES;

EID: 69249156419     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2009.73     Document Type: Review

References (57)

1. Evans CH, Ghivizzani SC, Robbins PD. Orthopedic gene therapy in 2008. Mol Ther 2009; 17: 231-244.
2. Evans CH, Ghivizzani SC, Robbins PD. Gene therapy for the rheumatic diseases 1998-2008. Arthritis Res Ther 2009; 11: 209-221.
3. Gouze E, Gouze JN, Palmer GD, Pilapil C, Evans CH, Ghivizzani SC. Transgene persistence and cell turnover in the diarthrodial joint: implications for gene therapy of chronic joint diseases. Mol Ther 2007; 15: 1114-1120.
4. Sandalon Z, Bruckheimer EM, Lustig KH, Burstein H. Longterm suppression of experimental arthritis following intramuscular administration of a pseudotyped AAV2/1-TNFR:FC Vector. Mol Ther 2007; 15: 264-269.
5. Adriaansen J, Fallaux FJ, de Cortie CJ, Vervoordeldonk MJ, Tak PP. Local delivery of beta interferon using an adeno-associated virus type 5 effectively inhibits adjuvant arthritis in rats. J Gen Virol 2007; 88: 1717-1721.
6. Adriaansen J, Khoury M, de Cortie CJ, Fallaux FJ, Bigey P, Scherman D et al. Reduction of arthritis following intra-articular administration of an adeno-associated virus serotype 5 expressing a disease-inducible TNF-blocking agent. Ann Rheum Dis 2007; 66 1143-1150.
7. Khoury M, Adriaansen J, Vervoordeldonk MJ, Gould D, Chernajovsky Y, Bigey P et al. Inflammation-inducible antiTNF gene expression mediated by intra-articular injection of serotype 5 adeno-associated virus reduces arthritis. J Gene Med 2007; 9: 596-604.
8. Boissier MC, Lemeiter D, Clavel C, Valvason C, Laroche L, Begue T et al. Synoviocyte infection with adeno-associated virus (AAV) is neutralized by human synovial fluid from arthritis patients and depends on AAV serotype. Hum Gene Ther 2007; 18: 525-535.
9. Kay JD, Gouze E, Oligino TJ, Gouze JN, Watson RS, Levings PP et al Intra-articular gene delivery and expression of IL-1Ra mediated by self-complementary adeno-associated virus. J Gene Med 2009; 11 605-614.
10. Ghivizzani SC, Gouze E, Gouze JN, Kay JD, Bush ML, Watson RS et al Perspectives on the use of gene therapy for chronic joint diseases. Curr Gene Ther 2008; 8: 273-286.
11. Rachakonda PS, Rai MF, Schmidt MF. Application of inflammation- responsive promoter for an in vitro arthritis model. Arthritis Rheum 2008; 58: 2088-2097.
12. Geurts J, Arntz OJ, Bennink MB, Joosten LA, van den Berg WB, van de Loo FA. Application of a disease-regulated promoter is a safer mode of local IL-4 gene therapy for arthritis. Gene Therapy 2007; 14: 1632-1638.
13. Gould D, Yousaf N, Fatah R, Subang MC, Chernajovsky Y. Gene therapy with an improved doxycycline-regulated plasmid encoding a tumour necrosis factor-alpha inhibitor in experimental arthritis. Arthritis Res Ther 2007; 9: R7.
14. Bloquel C, Denys A, Boissier MC, Apparailly F, Bigey P, Scherman D et al. Intra-articular electrotransfer of plasmid encoding soluble TNF receptor variants in normal and arthritic mice. J Gene Med 2007; 9: 986-993.
15. Haas CS, Amin MA, Ruth JH, Allen BL, Ahmed S, Pakozdi A et al. In vivo inhibition of angiogenesis by interleukin-13 gene therapy in a rat model of rheumatoid arthritis. Arthritis Rheum 2007; 56: 2535-2548.
16. Shiau AL, Chen SY, Chang MY, Su CH, Chung SY, Yo YT et al. Prothymosin alpha lacking the nuclear localization signal as an effective gene therapeutic strategy in collagen-induced arthritis. J Immunol 2007; 178: 4688-4694.
17. Leng J, Yao H, Shen J, Wang K, Zhuo G, Wang Z. Co-expression of IL-18 binding protein and IL-4 regulates Th1/Th2 cytokine response in murine collagen-induced arthritis. Acta Biochim Biophys Sinica 2008; 40: 116-124.
18. Izal I, Acosta CA, Ripalda P, Zaratiegui M, Ruiz J, Forriol F. IGF-1 gene therapy to protect articular cartilage in a rat model of joint damage. Arch Orthop Trauma Surg 2008; 128: 239-247.
19. Khoury M, Escriou V, Courties G, Galy A, Yao R, Largeau C et al. Efficient suppression of murine arthritis by combined anticytokine small interfering RNA lipoplexes. Arthritis Rheum 2008; 58: 2356-2367.
20. Chen LX, Lin L, Wang HJ, Wei XL, Fu X, Zhang JY et al. Suppression of early experimental osteoarthritis by in vivo delivery of the adenoviral vector-mediated NF-kappaBp65-specific siRNA. Osteoarthr Cartil 2008; 16: 174-184.
21. Choi JJ, Yoo SA, Park SJ, Kang YJ, Kim WU, Oh IH et al. Mesenchymal stem cells overexpressing interleukin-10 attenuate collagen-induced arthritis in mice. Clin Exp Immunol 2008; 153 269-276.
22. Guichelaar T, ten Brink CB, van Kooten PJ, Berlo SE, Lafeber FP, Broeren CP et al. Cartilage proteoglycan-specific T cells as vectors of immunomodulatory biologicals in chronic proteoglycan-induced arthritis. Mol Immunol 2008; 45: 3526-3535.
23. Kim SH, Bianco NR, Shufesky WJ, Morelli AE, Robbins PD. Effective treatment of inflammatory disease models with exosomes derived from dendritic cells genetically modified to express IL-4. J Immunol 2007; 179: 2242-2249.
24. Mease PJ, Hobbs K, Chalmers A, El-Gabalawy H, Bookman A, Keystone E et al. Local delivery of a recombinant adeno-associated vector containing a tumor necrosis factor-{alpha} antagonist gene in inflammatory arthritis: A phase 1 dose-escalation safety and tolerability study. Ann Rheum Dis 2008. 4 August e-pub ahead of print.
25. Evans CH, Ghivizzani SC, Robbins PD. Arthritis gene therapy's first death. Arthritis Res Ther 2008; 10: 110.
26. Mease P, Wei N, Fudman E, Kivitz A, Anklesaria P, Heald A. Local treatment for inflammatory arthritis: A phase 1/2 clinical study of intra-articular administration of a recombinant adenoassociated vector containing a TNF-a antagonist gene. Arthritis Rheum 2007; 56 suppl 9): S793.
27. Wehling P, Reinecke J, Baltzer AWA, Granrath M, Schulitz KP, Schultz C et al. Clinical responses to gene therapy in joints of two subjects with rheumatoid arthritis. Hum Gene Ther 2009; 20: 97-101.
28. de Poorter JJ, Hoeben RC, Hogendoorn S, Mautner V, Ellis J, Obermann WR et al. Gene therapy and cement injection for restabilization of loosened hip prostheses. Hum Gene Ther 2008; 19: 83-95.
29. Evans CH, Palmer GD, Pascher A, Porter R, Kwong FN, Gouze E et al. Facilitated endogenous repair: Making tissue engineering simple, practical, and economical. Tissue Eng 2007; 13: 1987-1993.
30. Ishihara A, Shields KM, Litsky AS, Mattoon JS, Weisbrode SE, Bartlett JS et al. Osteogenic gene regulation and relative acceleration of healing by adenoviral-mediated transfer of human BMP-2 or -6 in equine osteotomy and ostectomy models. J Orthop Res 2008; 26: 764-771.
31. Lu SS, Zhang X, Soo C, Hsu T, Napoli A, Aghaloo T et al. The osteoinductive properties of Nell-1 in a rat spinal fusion model. Spine J 2007; 7: 50-60.
32. Betz OB, Betz VM, Nazarian A, Egermann M, Gerstenfeld LC, Einhorn TA et al. Delayed administration of adenoviral BMP-2 vector improves the formation of bone in osseous defects. Gene Therapy 2007; 14: 1039-1044.
33. Betz VM, Betz OB, Glatt V, Gerstenfeld LC, Einhorn TA, Bouxsein ML et al. Healing of segmental bone defects by direct percutaneous gene delivery: Effect of vector dose. Hum Gene Ther 2007; 18: 907-915.
34. Rundle CH, Strong DD, Chen ST, Linkhart TA, Sheng MH, Wergedal JE et al. Retroviral-based gene therapy with cyclooxygenase-2 promotes the union of bony callus tissues and accelerates fracture healing in the rat. J Gene Med 2008; 10: 229-241.
35. Tu Q, Valverde P, Li S, Zhang J, Yang P, Chen J. Osterix overexpression in mesenchymal stem cells stimulates healing of critical-sized defects in murine calvarial bone. Tissue Eng 2007; 13: 2431-2440.
36. Lattanzi W, Parrilla C, Fetoni A, Logroscino G, Straface G, Pecorini G et al. Ex vivo-transduced autologous skin fibroblasts expressing human Lim mineralization protein-3 efficiently form new bone in animal models. Gene Therapy 2008; 15: 1330-1343.
37. Hsu WK, Wang JC, Liu NQ, Krenek L, Zuk PA, Hedrick MH et al. Stem cells from human fat as cellular delivery vehicles in an athymic rat posterolateral spine fusion model. J Bone Joint Surg 2008; 90 1043-1052.
38. Virk MS, Conduah A, Park SH, Liu N, Sugiyama O, Cuomo A et al. Influence of short-term adenoviral vector and prolonged lentiviral vector mediated bone morphogenetic protein-2 expression on the quality of bone repair in a rat femoral defect model. Bone 2008; 42: 921-931.
39. Kwong FN, Richardson SM, Evans CH. Chordin knockdown enhances the osteogenic differentiation of human mesenchymal stem cells. Arthritis Res Ther 2008; 10: R65.
40. Guo CA, Liu XG, Huo JZ, Jiang C, Wen XJ, Chen ZR. Novel gene-modified-tissue engineering of cartilage using stable transforming growth factor-beta1-transfected mesenchymal stem cells grown on chitosan scaffolds. J Biosci Bioeng 2007; 103: 547-556.
41. Pagnotto MR, Wang Z, Karpie JC, Ferretti M, Xiao X, Chu CR. Adeno-associated viral gene transfer of transforming growth factor-beta1 to human mesenchymal stem cells improves cartilage repair. Gene Therapy 2007; 14: 804-813.
42. Steinert AF, Palmer GD, Capito R, Hofstaetter JG, Pilapil C, Ghivizzani SC et al. Genetically enhanced engineering of meniscus tissue using ex vivo delivery of transforming growth factor-beta 1 complementary deoxyribonucleic acid. Tissue Eng 2007; 13: 2227-2237.
43. Gelse K, Muhle C, Franke O, Park J, Jehle M, Durst K et al. Cell-based resurfacing of large cartilage defects: Long-term evaluation of grafts from autologous transgene-activated periosteal cells in a porcine model of osteoarthritis. Arthritis Rheum 2008; 58: 475-488.
44. Goodrich LR, Hidaka C, Robbins PD, Evans CH, Nixon AJ. Genetic modification of chondrocytes with insulin-like growth factor-1 enhances cartilage healing in an equine model. J Bone Joint Surg Br 2007; 89: 672-685.
45. Steinert AF, Palmer GD, Pilapil C, Noth U, Evans CH, Ghivizzani SC. Enhanced in vitro chondrogenesis by primary mesenchymal stem cells by combined gene transfer. Tissue Eng 2009; 115: 1127-1139.
46. Morisset S, Frisbie DD, Robbins PD, Nixon AJ, McIlwraith CW. IL-1ra/ IGF-1 gene therapy modulates repair of microfractured chondral defects. Clin Orthop Relat Res 2007; 462: 221-228.
47. Evans C. Point of view. Spine 2008; 33: 1517.
48. Zhang Y, Anderson DG, Phillips FM, Thonar EJ, He TC, Pietryla D et al Comparative effects of bone morphogenetic proteins and Sox9 overexpression on matrix accumulation by bovine anulus fibrosus cells: implications for anular repair. Spine 2007; 32: 2515-2520.
49. Zhang Y, Phillips FM, Thonar EJ, Oegema T, An HS, Roman-Blas JA et al Cell therapy using articular chondrocytes overexpressing BMP-7 or BMP-10 in a rabbit disc organ culture model. Spine 2008; 33: 831-838.
50. Moon SH, Nishida K, Gilbertson LG, Lee HM, Kim H, Hall RA et al. Biologic response of human intervertebral disc cells to gene therapy cocktail. Spine 2008; 33: 1850-1855.
51. Le Maitre CL, Hoyland JA, Freemont AJ. Interleukin-1 receptor antagonist delivered directly and by gene therapy inhibits matrix degradation in the intact degenerate human intervertebral disc: An in situ zymographic and gene therapy study. Arthritis Res Ther 2007; 9: R83.
52. Bolt P, Clerk AN, Luu HH, Kang Q, Kummer JL, Deng ZL et al. BMP-14 gene therapy increases tendon tensile strength in a rat model of Achilles tendon injury. J Bone Joint Surg 2007; 89: 1315-1320.
53. Majewski M, Betz O, Ochsner PE, Liu F, Porter RM, Evans CH. Ex vivo adenoviral transfer of bone morphogenetic protein 12 (BMP-12) cDNA improves Achilles tendon healing in a rat model. Gene Therapy 2008; 15: 1139-1146.
54. Basile P, Dadali T, Jacobson J, Hasslund S, Ulrich-Vinther M, Soballe K et al. Freeze-dried tendon allografts as tissueengineering scaffolds for Gdf5 gene delivery. Mol Ther 2008; 16: 466-473.
55. Li F, Jia H, Yu C. ACL reconstruction in a rabbit model using irradiated Achilles autograft seeded with mesenchymal stem cells or PDGF-B gene-transfected mesenchymal stem cells. Knee Surg SPorts Traumatol Arthrosc 2007; 15: 1219-1227.
56. Steinert AF, Weber M, Kunz M, Palmer GD, Noth U, Evans CH et al. In situ IGF-1 gene delivery to cells emerging from the injured anterior cruciate ligament. Biomaterials 2008; 29: 904-916.
57. Evans CH. Arthritis gene therapy at an inflection point. Future Rheumatology 2008; 3: 207-210.