A 1998 status report on orthopedic gene therapy

Current Opinion in Orthopaedics

Volumn 9, Issue 6, 1998, Pages 75-80

  Evans, C H ^a   Ghivizzani, Steven C ^a,b   Robbins, Paul D ^a  

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

^b Megabios Corporation   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ARTHRITIS; ARTICLE; BONE DISEASE; GENE THERAPY; GENETIC DISORDER; HUMAN; INTERVERTEBRAL DISK DISEASE; ORTHOPEDICS; PRIORITY JOURNAL; PROTEIN SYNTHESIS INHIBITION; RNA SYNTHESIS; SPORTS MEDICINE; TISSUE REPAIR;

EID: 0032465712     PISSN: 10419918     EISSN: None     Source Type: Journal    
DOI: 10.1097/00001433-199812000-00013     Document Type: Article

References (67)

1. 1. Evans CH, Robbins PD: Possible orthopaedic applications of gene therapy, J Bone Joint Surg Am 1995, 77:1103-1114.
2. 2. Bandara G, Robbins PD, Mueller GM, Galea-Lauri J, Georgescu HI, et al.: Gene transfer to synovium. Trans Orthop Res Soc 1993, 18:242.
3. 3. Bandara G, Robbins PD, Georgescu HI, Mueller GM, Glorioso JC, Evans CH: Gene transfer to synoviocytes: prospects for gene treatment for arthritis. DNA Cell Biol 1992, 11:227-231.
4. 4. Evans CH, Ghivizzani SC, Whalen JD, Robins PD: Gene therapy in autoimmune diseases. Ann Rheum Dis 1998, 57:125-127.
5. 5. Evans CH, Ghivizzani SC, Robbins PD: Blocking cytokines with genes. J Leukoc Biol 1998, 64:55-61.
6. 6. Evans CH, Ghivizzani SC, Kang R, Muzzonigro TA, Wasko MC, Herndon JH, Robbins PD: Gene therapy for rheumatic diseases. Arthritis Rheum 1999, 42:1-16. This is a detailed, exhaustive review of the entire field.
7. 7. Roessler BJ, Alolen ED, Wilson JM, Hartman JW, Davidson BL: Adenoviral-mediated gene transfer to rabbit synovium in vivo. J Clin Invest 1993, 92:1085-1092.
8. 8. Bandara G, Mueller GM, Galea-Lauri J, Tindal MH, Georgescu HI, Suchanek MK, et al.: Intra-articular expression of biologically active interleukin-1 receptor antagonist protein by ex vivo gene transfer. Proc Natl Acad Sci U S A 1993, 90:10764-10768.
9. 9. Nita I, Ghivizzani SC, Galea-Lauri J, Bandara G, Georgescu HI, Robbins PD, Evans CH: Direct gene delivery to synovium: an evaluation of potential vectors in vitro and in vivo. Arthritis Rheum 1996, 39:820-828.
10. 10. Tomita T, Hashimoto H, Tomita N, Morishita R, Lee SB, Hayashida K, et al.: In vivo direct gene transfer into articular cartilage by intraarticular injection mediated by HVJ (sendai virus) and liposomes. Arthritis Rheum 1997, 40:901-906. This paper reports remarkably efficient gene transfer to synovium and cartilage by nonviral means.
11. 11. Ghivizanni SC, Lechman ER, Tio C, MulÇ KM, Chada S, McCormack JE, et al.: Direct retroviral mediated gene transfer to the synovium of the rabbit knee: implications for arthritis gene therapy. Gene Ther 1997, 4:977-982.
12. 12. Jorgensen C, Demoly P, Noel D, Mathieu M, Piechaczyc M, Gougat C, et al.: Gene transfer to human rheumatoid tissue engrafted in SCID mice. J Rheumatol 1997, 24:2076-2079.
13. 13. Evans CH, Robbins PD: Getting genes into human synovium. J Rheumatol 1997, 24:2061-2063.
14. 14. Yovandich J, O'Malley B, Sikes M, Ledley FD: Gene transfer to synovial cells by intra-articular administration of plasmid DNA. Hum Gene Ther 1995, 6:603-610.
15. 15. Makarov SS, Olsen JC, Johnston WN, Anderle SK, Brown RR, Baldwin AS, et al: Suppression of experimental arthritis by gene transfer of interleukin-1 receptor antagonist cDNA. Proc Natl Acad Sci U S A 1996, 93:402-406.
16. 16. Otani K, Nita I, Macaulay W, Georgescu HI, Robbins PD, Evans CH: Suppression of antigen-induced arthritis by gene therapy. J Immunol 1996, 156:3558-3562.
17. 17. Bakker AC, Joosten LAB, Arntz OJ, Helsen MMA, Benderle AM, Van De Loo FAJ, Van den Berg WB: Prevention of murine collagen-induced arthritis in the knee and ipsilateral paw by local expression of human interleukin-1 receptor antagonist protein in the knee. Arthritis Rheum 1997, 40:893-900.
18. 18. Ghivizzani SC, Lechman ER, Kang R, Tio C, Kolls J, Evans CH, Robbins PD: Direct adenovirus-mediated gene transfer of interleukin-1 and tumor necrosis factor β soluble receptors to rabbit knees with experimental arthritis has local and distal anti-arthritic effects. Proc Natl Acad Sci U S A 1998, 95:4613-4618. This article demonstrates for the first time the disease suppression in multiple arthritic joints after gene treatment of only one.
19. 19. Whalen JD, Lechman ER, Carlos CA, Weiss K, Kovesdi I, Robbins PD, Evans CH: Adenoviral transfer of the viral IL-10 gene periarticularly to mouse paws suppresses development of collagen-induced arthritis in both injected an uninjected paws. J Immunol, in press.
20. 20. Zhang H, Yang Y, Horton JL, Samoilova EB, Judge TA, Turka LA, et al.: Amelioration of collagen-induced arthritis by CD95 (Apo-1 /Fas)-ligand gene transfer. J Clin Invest 1997, 100:1951 -1957.
21. 21. Makarov S, Baldwin AS: Local suppression of NF-kB inhibits experimental arthritis in rats [abstract 563]. Arthritis Rheum 1996, 39(suppl):121.
22. 22. Pelletier JP, Caron JP, Evans CH, Robbins PD, Georgescu HI, Jovanovic D, et al.: In vivo suppression of early experimental osteoarthritis by IL-1 Ra using gene therapy. Arthritis Rheum 1997, 40:1012-1019.
23. 23. Le CH, Nicolson AG, Morales AN, Sewell KL: Suppression of collagen-induced arthritis through adenovirus-mediated transfer of a modified tumor necrosis factor β receptor gene. Arthritis Rheum 1997, 40:1662-1669.
24. 24. Chernajovsky Y, Adams G, Podhajcer O, Mueller G, Robbins PD, Feldman M: Inhibition of transfer of collagen induced arthritis into SCID mice by gene transfer of spleen cells with retrovirus expressing soluble human tumor necrosis factor receptor. Gene Ther 1995, 2:731-735.
25. 25. Chernajovsky Y, Adams G, Triantaphyllopoulos K, Ledda MF, Podhajcer OL: Pathogenic lymphoid cells engineered to express TGF-β1 ameliorate disease in a collagen-induced arthritis model. Gene Ther 1997, 4:553-559.
26. 26. Song XY, Gu ML, Jin WW, Klinman DM, Wahl SM: Plasmid DNA encoding transforming growth factor-1 suppresses chronic disease in a streptococcal cell wall-induced arthritis model. J Clin Invest 1998, 101:2615-2621. A convincing demonstration of how naked DNA can be used to produce a strong antiarthritic effect.
27. 27. Apparailly F, Verwaerde C, Jacquet C, Auriault C, Snay J, Jorgensen C: Adenovirus-mediated transfer of viral IL-10 gene inhibits murine collagen-induced arthritis. J Immunol 1998, 160:5213-5220.
28. 28. Bessis N, Boissier MC, Ferrara P, Blankenstein T, Fradelizi D, Fournier C: Attenuation of collagen-induced arthritis in mice by treatment with vector cells engineered to secrete interleukin-13. Eur J Immunol 1996, 26:2399-2403.
29. 29. Shiozawa S, Shimizu K, Tanaka K, Hino K: Studies on the contribution of c-fos/AP-1 to arthritic joint destruction. J Clin Invest 1997, 99:1210-1216.
30. 30. Evans CH, Robbins PD, Ghivizzani SC, Herndon JH, Kang R, Bahnson AB, et al.: Clinical trial to assess the safety, feasibility, and efficacy of transferring a potentially anti-arthritic cytokine gene to human joints with rheumatoid arthritis. Hum Gene Ther 1996, 7:1261-1280.
31. 31. Ghivizzani SC, Kang R, Muzzonigro T, Whalen J, Watkins SC, Herndon JH, et al.: Gene therapy for arthritis-treatment of the first three patients [abstract 1155]. Arthritis Rheum 1997, 40(suppl):223.
32. 32. Gerich T, Fu FH, Robbins PD, Evans CH: Prospects for gene therapy in sports medicine. Knee Surg Sports Traumatol Arthrosc 1996, 4:180-187.
33. 33. Lamsam C, Fu FH, Robbins PD, Evans CH: Gene therapy in sports in medicine. Sports Med 1998, 25:73-77.
34. 34. Latterman C, Robbins PD, Evans CH, Fu FH: Gene therapy in sports medicine. In Sports and Children. Edited by Chen KM, Micheli LJ. Hong Kong: Williams & Wilkins, 1998:210-216.
35. 35. Kang R, Marui T, Ghivizzani SC, Nita IM, Georgescu HI, Suh JK, et al.: Ex vivo gene transfer to chondrocytes in full-thickness articular cartilage defects: a feasibility study. Osteoarthritis Cartilage 1997, 5:139-143.
36. 36. Baragi VM, Renkiewicz RR, Qiu L, Brammer D, Riley JM, Sigler RE, Frenkel SR, Amin A, Abramson SB, Roessler BJ: Transplantation of adenovirally transduced allogeneic chondrocytes into articular cartilage defects in vivo. Osteoarthritis Cartilage 1997; 5:275-282
37. 37. Mason JM, Grande DA, Barcia M, Grant R, Pergolizzi RG, Breitbart AS: Expression of human bone morphogenetic protein 7 in primary rabbit periosteal cells: potential utility in gene therapy for osteochondral repair. Gene Ther 1998, 5:1098-1104.
38. 38. Baragi VM, Renkiewicz RR, Jordan H, Bonadio J, Hartman JW, Roessler BJ: Transplantation of transduced chondrocytes protects articular cartilage from interleukin-1 induced extracellular matrix degradation. J Clin Invest 1995, 96:2454-2460.
39. 39. Doherty PJ, Zhang H, Tremblay L, Manolopoulos V, Marshall KW: Resurfacing of articular cartilage explants with genetically-modified human chondrocytes in vitro. Osteoarthritis Cartilage 1998, 6:153-169.
40. 40. Arai Y, Kubo T, Kobayashi K, Takeshita K, Takahashi K, Ikeda T, et al.: Adenovirus vector-mediated gene transduction to chondrocytes: in vitro evaluation of therapeutic efficacy of transforming growth factor-1 and heat shock protein 70 gene transduction. J Rheumatol 1997, 24:1787-1795.
41. 41. Shuler FD, Georgescu HI, Niyibizi C, Studer R, Mi Z, Robbins PD, Evans CH: Up-regulation of matrix synthesis following adenoviral mediated transfer of transforming growth factor β-1 (TGFβ-1) cDNA into chondrocytes. Submitted.
42. 42. Goto H, Shuler FD, Lamsam C, Möller HD, Robbins PD, Niyibizi C, et al.: In vivo and ex vivo gene transfer to an avascular meniscal lesion and up-regulation of matrix synthesis by meniscal fibrochondrocytes following virally mediated transfer of a transforming growth factor-1 cDNA. Submitted.
43. 43. Evans CH, Bandara G, Robbins PD, Mueller GM, Georgescu HI, Glorioso JC: Gene therapy for ligament healing. In: The Anterior Cruciate Ligament: Current and Future Concepts. Edited by Jackson DW. New York: Raven Press. 1993:419-422.
44. 44. Gerich TG, Kang R, Fu FH, Robbins PD, Evans CH: Gene transfer to the rabbit patellar tendon: potential for genetic enhancement of tendon and ligament healing. Gene Ther 1996, 3:1089-1093.
45. 45. Lou J, Manske PR, Aoki M, Joyce ME: Adenovirus-mediated gene transfer into tendon and tendon sheath. J Orthop Res 1996, 14:513-517.
46. 46. Nakamura N, Horibe S, Matsumoto N, Tomita T, Natsuume T, Kaneda Y, et al.: Transient introduction of a foreign gene into healing rat patellar ligament. J Clin Invest 1996, 97:226-231.
47. 47. Nakamura N, Horibe S, Natsuume T, Tomita T, Matsumoto N, Hashimoto H, et al.: Early effect of in vivo introduction of platelet-derived growth factor [PDGF] gene into healing patellar ligament [abstract]. Trans Orthop Res Soc 1996, 21:193.
48. 48. Kasemkijwattana C, Menetrey J, Day CS, Booth CS, Fu FH, Moreland MS, et al.: Biologic intervention in muscle healing and regeneration. Sports Med Arthrosc Rev 1998, 6:95-102.
49. 49. Monahan PE, Tazelaar J, Xiao X, Nicols TC, Bellinger DA, Read MS, et al.: Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia. Gene Ther 1998, 5:40-49.
50. 50. Chen HH, Mack LM, Kelly R, Ontell M, Kochanek S, Clemens PR: Persistence in muscle of an adenoviral vector that lacks all viral genes. Proc Natl Acad Sci U S A 1997, 94:1645-1650.
51. 51. Huard J, Akkaraju GR, Watkins SC, Pike-Cavalcoli M, Glorioso JC: Lac Z. gene transfer to skeletal muscle using a replication defective herpes simplex virus type I mutant vector. Hum Gene Ther 1997, 8:439-452.
52. 52. Booth DK, Floyd SS, Day CS, Glorioso JC, Kovesdi I, Huard J: Myoblast mediated ex vivo gene transfer to mature muscle. Tissue Eng 1997, 3:125-133.
53. 53. Niyibizi C, Baltzer A, Lattermann C, Oyama M, Whalen JD, Robbins PD, Evans CH: Potential role for gene therapy in the augmentation of fracture healing. Clin Orthop, 1998, 335(suppl): S148-S153.
54. 54. Fang J, Zhu YY, Smiley E, Bonadino J, Rouleau JP, Goldstein SA, et al.: Stimulation of new bone formation by direct transfer of osteogenic plasmid genes. Proc Natl Acad Sci U S A 1996, 93:5753-5758.
55. 55. Baltzer AWA, Lattermann C, Whalen JD, Robbins PD, Evans CH: A gene therapy approach to accelerating bone healing in athletes. Knee Surg Sports Traumatol Arthrosc, in press.
56. 56. Allay JA, Dennis JE, Haynesworth SE, Majumdar MK, Clapp DW, Shultz LD, et al: Lac Z and interleukin-3 expression in vivo after retroviral transduction of marrow-derived human osteogenic mesenchymal progenitors. Hum Gene Ther 1997, 8:1417-1427.
57. 57. Oyama M, Tatlock A, Fukuta S, Kavalkovich K, Nishimura K, Johnstone B, et al.: Retrovirally transduced bone marrow cells isolated from a mouse model of osteogenesis imperfecta (oim) persist in bone and retain the ability to form bone and cartilage after extended passaging. Gene Ther, in press.
58. 58. Lieberman JR, Le LQ, Wu L, Finerman GAM, Berk A, Witte ON, Stevenson S: Regional gene therapy with a BMP-2 producing murine stromal cell line induces heterotopic and orthotopic bone formation in rodents. J Orthop Res 1998, 16:330-339.
59. 59. Marini JC, Gerber NL: Osteogenesis imperfecta: rehabilitation and prospects for gene therapy. JAMA 1997, 277:746-750.
60. 60. Wang Q, Marini JC: Abtisense oligodeoxynucleotides selectively suppress expression of a mutant α2(I) collagen allele in type IV osteogenesis imperfecta fibroblasts: A molecular approach to therapeutics of dominant negative disorders. J Clin Invest 1996, 97:448-454.
61. 61. Baltzer AWA, Whalen JD, Lattermann C, Wooley PH, Robbins PD, Evans CH: Gene therapy with human IL-1 Ra prevents bone loss in a murine ovariectomy model of osteoporosis [abstract]. Trans Orthop Res Soc 1998, 23:320.
62. 62. Wooley PH, Sud S, Robbins PD, Whalen JD, Evans CH, Nasser S: Gene therapy using a retroviral vector encoding IRAP in the murine air pouch model of aseptic loosening [abstract]. Trans Orthop Res Soc 1997, 22:741.
63. 63. Wehling P, Schulitz KP, Robbins PD, Evans CH, Reinecke JA: Transfer of genes to chondrocytic cells of the lumbar spine. Spine 1997, 22:1092-1097.
64. 64. Nishida K, Kang JD, Suh JK, Robbins PD, Evans CH, Gilbertson LG: Adenovirus-mediated gene transfer to nucleus pulposus cells: implications for treatment of intervertebral disc degeneration. Spine, in press.
65. 65. Nishida K, Kang JD, Suh JK, Robbins PD, Evans CH, Gilbertson LG: Adenovirus-mediated transfer of human TGF-β1 encoding gene to the rabbit intervertebral disc in vivo upregulates proteoglycan synthesis. Submitted.
66. 66. Barranger JA, Rice E, Sansieri C, Bahnson A, Mohney T, Swancy N, et al.: Transfer of the glucocerebrosidase gene to CD34 cells and their autologous transplantation in patients with Gaucher disease [abstract]. Blood 1998, 405.
67. 67. Clemens PR, Caskey CT: Gene therapy prospects for Duchenne muscular dystrophy. Eur Neurol 1994, 34:181-185.