Abstract
Gene therapy represents a novel approach for treating joint and bone disorders (Evans et al. 1997; Evans, Ghivizzani, and Robbins 1998; Lattermann et al. 1998). As discussed in other chapters, gene transfer can be used for delivering therapeutic agents to synovium, cartilage, ligaments, tendons, meniscus, intervertebral disc, and bone to block disease progression or to promote repair. In addition, the recent completion of the first gene therapy trial for rheumatoid arthritis (RA) has demonstrated the feasibility of using gene transfer for the treatment of orthopaedic and rheumatologic disorders. The use of gene transfer to deliver a therapeutic agent offers certain advantages over the use of recombinant protein. In particular, the use of genes as therapeutic agents can results in persistent expression locally at the site of disease, bypassing the need for multiple injections and preventing possible side effects associated with systemic administration.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
Preview
Unable to display preview. Download preview PDF.
References
Apparailly, F., Verwaerde, C., Jacquest, C., Auriault, C., Sany, J., and Jorgensen, C. 1998. Adenovirus-mediated transfer of viral IL-10 gene inhibits murine collagen-induced arthritis. J Rheumatol 160:5213–20.
Baltzer, A.W.A., Lattermann, C., Weiss, K., Wooley, P., Grimm, M., Ghivizzani, S.C., Robbins, P.D., and Evans, C.H. In press. Genetic enhancement of fracture repair: Evaluation of Ad-BMP-2 in a lapine femoral defect model. Gene Ther.
Bandara, G., Mueller, G.M., Galea-Lauri, J., Tyndall, M.H., Georgescu, H.I., Suchanek, M.K., Hung, G.L., Glorioso, J.C., Robbins, P.D., and Evans, C.H. 1993. Intraarticular expression of the interleukin-1 receptor antagonist protein by ex vivo gene transfer. Proc Natl Acad Sci USA 90:10764–8.
Byun, J., Kim, S.-H., Kim, J.M., Robbins, P.D., Yim, J., and Kim, S. 1996. Analysis of the relative level of gene expression from different retroviral vectors used for gene therapy. Gene Ther 3:780–8.
Dranoff, G., Jaffee, E., Lazenby, A., Golumbek, P., Levitsky, H., Brose, K., Jackson, V., Hamada, H., Pardoll, D., and Mulligan, R.C. 1993. Vaccination with irradiated tumor cells engineered to secrete murine granulocyte-macrophage colony-stimulating factor stimulates potent, specific, and long-lasting anti-tumor immunity. Proc Natl Acad Sci USA 90:3539–43.
Evans, C.H., Ghivizzani, S.C., Kang, R., Muzzonigro, T., Wasko, M.C., Herndon, J.H., and Robbins, P.D. 1997. Gene Therapy in Rheumatic Diseases. Arthritis Rheum 42:1–16.
Evans, C.H., Ghivizzani S.C., and Robbins, P.D. 1998. Blocking cytokines with genes. J Leuk Biol 64:55–61.
Evans, C.H. and Robbins, P.D. 1997. Getting genes into human synovium. J Rheumatol 24:2061–3.
Evans, C.H., Robbins, P.D., Ghivizzani, S.C., Herndon, J.H., Kang, R., Bahnson, A.B., Barranger, J.A., Elders, E.M., Gay, S., Tomaino, M.M., Wasko, C., Watkins, S.C., Whiteside, T.L., Glorioso, J.C., Lotze, M.T., and Wright, T.M. 1996. Clinical trial to assess the safety, feasibility, and efficacy of transferring a potentially antiarthritic cytokine gene to human joints with rheumatoid arthritis. Hum Gene Ther 7:1261–80.
Ferrari, F.K., Samulski, T., Shenk, T., and Samulski, R.J. 1996. Second strand synthesis is a rate limiting step for efficient transduction by recombinant adeno-associated virus vectors. J Virol 70:3227–34.
Fink, D.J. and Glorioso, J.C. 1997a. Engineering herpes simplex virus vectors for gene transfer to neurons. Nat Med 3:357–9.
Fink, D.J. and Glorioso, J.C. 1997b. Herpes simplex virus based vectors: problems and some solutions. Adv Neurol 72:149–56.
Fink, D.J., Ramakrishnan, R., Marconi, P., Goins, W.F., Holland, T.C., and Glorioso, J.C. 1995. Advances in the development of herpes simplex virus based gene transfer vectors for the nervous system. Clin Neurosci 3:284–91.
Fisher, K.J., Jooss, K., Alston, J., Yang, Y., Haecker, S.E., High, K., Pathak, R., Raper, S.E., and Wilson, J.M. 1997. Recombinant adeno-associated virus for muscle directed gene therapy. Nat Med 3:306–12.
Gao, X. and Huang, L. 1995. Cationic liposome mediated gene transfer. Gene Ther 2:710–22.
Geller, A.I. 1997. Herpes simplex virus-1 plasmid vectors for gene transfer into neurons. Adv Neurol 72:143–8.
Gerich, T.G., Kang, R., Fu, F.H., Robbins, P.D., and Evans, C.H. 1997. Gene transfer to the patellar tendon. Knee Surg Sports Traumatol Arthrosc 5:118–23.
Ghivizzani, S.C., Lechman, E.R., Kang, R., Tio, C., Kolls, J., Evans, C.H., and Robbins, P.D. 1998. Direct adenoviral mediated gene transfer of IL-1 and TNF-alpha soluble receptors to rabbit knees with experimental arthritis has local and distal antiarthritic effects. Proc Natl Acad Sci USA 95:4613–8.
Ghivizzani, S.C., Lechman, E.R., Tio, C., McCormick, J., Chada, S., Kang, R., Mule, K.M., Evans, C.H., and Robbins, P.D. 1997. Direct retroviral mediated gene transfer to the synovium of the rabbit knee. Gene Ther 4:977–82.
Glorioso, J.C., Robbins, P.D., Krisky, D., Marconi, P., Goins, W.F, Schmidt, M.C., and Evans, C.H. 1997. Progress in the development of herpes simplex viurs gene vectors for treatment of rheumatoid arthritis. In B. Davidson (ed.), Advanced Drug Delivery Reviews Vol. 27:41–57.
Graham, F.L. and Prevec, L. 1995. Methods for construction of adenovirus vectors. Mol Biotechnol 3:207–20.
Guild, B.C., Finer, M.H, Housman, D.E., and Mulligan, R.C. 1988. Development of retrovirus vectors useful for expressing genes in cultured murine embryonal cells and hematopoietic cells in vivo. J Virol 62:3795–801.
Hildegrand, K.A., Dele, M., Allen, C.R., Smith, D.W., Georgescu, H.I., Robbins, P.D., Evans, C.H., and Woo, S.L.-Y. In Press. The expression of marker genes in the medial collateral and anterior cruciate ligaments: the use of different viral vectors and the effects of injury. J Orth Res.
Hung, G.L., Bandara, G., Galea-Lauri, J., Mueller, G.M., Georgescu, H.I., Larkin, L.A., Tyndall, M.H., Robbins, P.D., and Evans, C.H. 1994. Suppression of intraarticular response to interleukin-1 by gene transfer to synovium. Gene Ther 1:64–9.
Jorgensen, C., Demoly, P., Noel, D., Mathieu, M., Piecharczyc, M., Gougat, C., Bousquet, J., and Sany, J. 1997. Gene transfer to human rheumatoid synovial tissue engrafted in SCID mice. J Rheumatol 24:2076–9.
Kim, S.H., Yu, S.S., Park, J.S., Robbins, P.D., An, C.S., and Kim, S. 1997. Construction of retroviral vectors with improved safety, gene expression, and versatility. J Virol 72:994–1004.
Kochanek, S., Clemens, P.R., Mitani, K., Chen, H.H., Chan, S., and Caskey, C.T. 1996. A new adenoviral vector: replacement of all viral coding sequences with 28 kb of DNA independently expressing both full length dystrophin and β-galactosidase. Proc Natl Acad Sci USA 93:5731–6.
Krougliak, V. and Graham, F.L. 1995. Development of cell lines capable of complementing E1, E4, and protein IX defective adenovirus type 5 mutants. Hum Gene Ther 6:1575–86.
Lattermann, C., Baltzer, A.W.A., Whalen, J.D., Evans, C.H., Evans, C.H., Robbins, P.D., and Fu, F.H. 1998. Gene therapy in sports medicine. Sports Med Arthrosc Rev 6:83–8.
Lechman, E.R., Jaffurs, D., Ghivizzani, S.C., Gambotto, A., Kovesdi, I., Mi, Z., Evans, C.H., and Robbins, P.D. 1999. Direct adenoviral mediated gene transfer of vIL-10 to rabbit knees with experimental arthritis halts disease progression both locally and systemically. J Immunol 163:2202–8.
Liu, F., Yang, J., Huang, L., and Liu, D. 1996. New cationic lipid formulations for gene transfer. Pharm Res 13:1856–60.
Ma, Y., Thornton, S., Duwel, L.E., Boivin, G.P., Giannini, E.H., Leiden, J.M., Blue-stone, J.A., and Hirsch, R. 1998. Inhibition of collagen-induced arthritis in mice by viral IL-10 gene transfer. J Immunol 161:1516–24.
Marconi, P., Krisky, D., Origino, T., Poliani, P.L., Ramakrishnan, R., Goins, W.F., Fink, D.J., and Glorioso, J.C. 1996. Replication defective herpes simplex virus vectors for gene transfer in vivo. Proc Natl Acad Sci USA 93:11319–20.
Miagkov, A.V., Kovelenko, D.V., Brown, C.E., Didsbury, J.R., Cogswell, J.P, Stimpson, S.A., Bladwin, A.S., and Makarov, S.S. 1998. NF-kB activation provides the potential link between inflammation and hyperplasia in the arthritic joint. Proc Natl Acad Sci USA 95:13859–64.
Miller, A.D. 1992. Retroviral vectors. Curr Topics Micro Immunol 158:1–24.
Miller, A.D., Miller, D.G, Garcia, J.V., and Lynch, C.M. 1993. Use of retroviral vectors for gene transfer and expression. Meth Enzym 217:581–99.
Naldini, L., Blomer, U., Gage, F.H., Trono, D., and Verma, I.M. 1996. Efficient transfer, integration, and sustained long term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc Natl Acad Sci USA 93:11382–8.
Naldini, L., Blomer, U., Gallay, P., Ory, D., Mulligan, R., Gage, F.H., Verma, I.M., and Trono, D. 1996. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272:263–7.
Nguyen, K.H., Boyle, D.L., McCormack, J.E., Chada, S., Jolly, D.J., and Firestein, G.S. 1998. Direct synovial gene transfer with retroviral vectors in rat adjuvant arthritis. J Rheumatol 25:1118–25.
Nita, I., Ghivizzani, S., Galea-Lauri, J., Bandara, G., Georgescu, H.I., Robbins, P.D., and Evans, C.H. 1996. Direct gene delivery to synovium: an evaluation of potential vectors in vitro and in vivo. Arthritis Rheum 39:820–8.
Oligino, T., Ghivizzani, S.C., Wolfe, D., Lechman, E.R., Krisky, D., Mi, Z., Evans, C.H., Robbins, P.D., and Glorioso, J.C. 1999. Herpes virus viral vector mediated delivery of the IL-1Ra gene to rabbit joints reduces the pathophysiology of IL-1 induced arthritis. Gene Therapy 6:1713–20.
Oligino, T., Yao, O., Xiao, X., Glorioso, J.C., Evans, C.H., Robbins, P.D., and Ghivizzani, S.C. Under revision. Intra-articular expression of the IL-1Ra gene folllowing direct delivery with an adeno-associated virus vector. Gene Ther.
Otani, K., Nita, I., Macaulay, W., Georgescu, H.I., Mueller, G.M., Robbins, P.D., and Evans, C.H. 1996. Suppression of antigen induced arthritis in rabbits by ex vivo gene therapy. J Immunol 156:3558–62.
Pan, R.Y., Xiao, X., Chen, S.L., Li, J., Lin, L.C., Wang, H.J., and Tsao, Y.P. 1999. Disease-inducible transgene expression from a recombinant adeno-associated virus vector in a rat arthritis model. J Virol 73(4):3410–7.
Roessler, B.J., Allen, E.D., Wilson, J.M., Hartman, J.W., and Davidson, B.L. 1993. Adenoviral gene transfer to rabbit synovium in vivo. J Clin Invest 92:1085–92.
Rolling, F. and Samulski, R.J. 1995. AAV as a viral vector for human gene therapy. Generation of recombinant virus. Mol Biotechnol 3:9–15.
Russell, D.W., Alexander, I.E., and Miller, A.D. 1995. DNA synthesis and topoisomerase inhibitors increase transduction by adeno-associated virus vectors. Proc Natl Acad Sci USA 92: 5719–23.
Samulski, R.J. 1993. Adeno-associated virus: integration at a specific chromosomal locus. Curr Opin Genet Dev 3:74–80.
Sant, S.M., Suarez, T.M., Moalli, M.R., Wu, B.Y., Blaivas, M., Laing, T.J., and Roessler, B.J. 1998. Molecular lysis of synovial lining cells by in vivo herpes simplex virus-thymidine kinase gene transfer. Hum Gene Ther 9:2735–43.
Snyder, R.O., Miao, C., Meuse, L., Tubb, J., Donahue, B.A., Lin, H.F., Stafford, D.W., Patel, S., Thompson, A.R., Nichols, T., Read, M.S., Bellinger, D.A., Brinkhous, K.M., and Kay, M.A. 1999. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat Med 5:64–70.
Tomita, T., Hashimoto, H., Tomitaq, N., Morishita, R., Lee, S.B., Hayashida, K., Nakamura, N., Yonenobu, K., Kaneda, Y., and Ochi, T. 1997. In vivo direct gene transfer into articular cartilage by intraarticular injection mediated by HVJ (Sevdai Virus) and liposomes. Arthritis Rheum 40:901–6.
Wang, Q. and Finer, M.H. 1996. Second generation adenovirus vectors. Nat Med 2:714–6.
Whalen, J.D., Lechman, E.R., Carlos, C.A., Weiss, K., Kovesdi, I., Robbins, P.D., and Evans, C.H. 1999. Adenoviral transfer of the viral interleukin 10 gene periarticularly to mouse paws suppresses development of collagen induced arthritis in both injected and uninjected paws. J Immunol 162:3625–32.
Wolfe, D., Goins, W.F., Kaplan, T.J., Capuano, S., Murphey-Corb, M., Cohen, J.B., Robbins, P.D., and Glorioso, J.C. Submitted for publication. Systemic accumulation of biologically active nerve growth factor following intra-articular herpesvirus gene transfer.
Wu, N., Watkins, S.C., Schaffer, P.A., and DeLuca, N.A. 1996. Prolonged gene expression and cell survival after infection by a herpes simplex virus mutant defective in the immediate-early genes encoding ICP4, ICP27, and ICP22. J Virol 70:6358–69.
Yovandich, J., O’Malley, B., Sikes, M., and Ledley, F.D. 1995. Gene transfer to synovial cells by intraarticular adminstration of plasmid DNA. Hum Gene Ther 6:603–10.
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2000 Birkhäuser Boston
About this chapter
Cite this chapter
Robbins, P.D., Ghivizzani, S.C., Glorioso, J.C., Evans, C.H. (2000). Vectors for Gene Transfer to Joints. In: Huard, J., Fu, F.H. (eds) Gene Therapy and Tissue Engineering in Orthopaedic and Sports Medicine. Methods in Bioengineering. Birkhäuser Boston. https://doi.org/10.1007/978-1-4612-2126-5_14
Download citation
DOI: https://doi.org/10.1007/978-1-4612-2126-5_14
Publisher Name: Birkhäuser Boston
Print ISBN: 978-1-4612-7424-7
Online ISBN: 978-1-4612-2126-5
eBook Packages: Springer Book Archive