Posibilidades de la terapia génica en el sistema musculoesquelético

Revista de Ortopedia y Traumatología - Tập 49 - Trang 202-213 - 2005
G. Mazzolini1, J. Ruiz1, J. Prieto1
1Unidad de Hepatología y Terapia Génica. Departamento de Medicina Interna. Clínica Universitaria. Facultad de Medicina. Universidad de Navarra

Tài liệu tham khảo

Miller, 1992, Human gene therapy comes of age, Nature, 357, 455, 10.1038/357455a0 Anderson, 2002, The current status of clinical gene therapy, Hum Gene Ther, 13, 1261, 10.1089/104303402760128496 Mulligan, 1993, The basic science of gene therapy, Science, 260, 926, 10.1126/science.8493530 Culver, 1991, Correction of ADA deficiency in human T lymphocytes using retroviral-mediated gene transfer, Transplant Proc, 23, 170 Muul, 2003, Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial, Blood, 101, 2563, 10.1182/blood-2002-09-2800 www.wiley.co.uk/genetherapy/clinical/. Somia, 2000, Gene therapy: trials and tribulations, Nat Rev Genet, 1, 91, 10.1038/35038533 Cavazzana-Calvo, 2000, Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease, Science, 288, 669, 10.1126/science.288.5466.669 Hacein-Bey-Abina, 2003, A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency, N Engl J Med, 348, 255, 10.1056/NEJM200301163480314 Schmidt-Wolf, 2003, Non-viral and hybrid vectors in human gene therapy: an update, Trends Mol Med, 9, 67, 10.1016/S1471-4914(03)00005-4 Yang, 1996, Direct gene transfer to mouse melanoma by intratumor injection of free DNA, Gene Ther, 3, 542 Liu, 2001, Hydrodynamics-based gene delivery, Curr Opin Mol Ther, 3, 192 Pedroso de Lima, 2003, Cationic liposomes for gene delivery: from biophysics to biological applications, Curr Med Chem, 10, 1221, 10.2174/0929867033457430 Wu, 1991, Receptor-mediated gene delivery in vivo Partial correction of genetic analbuminemia in Nagase rats, J Biol Chem, 266, 14338, 10.1016/S0021-9258(18)98689-8 Yang, 1990, In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment, Proc Natl Acad Sci USA, 87, 9568, 10.1073/pnas.87.24.9568 Rakhmilevich, 1997, Cytokine gene therapy of cancer using gene gun technology: superior antitumor activity of interleukin-12, Hum Gene Ther, 8, 1303, 10.1089/hum.1997.8.11-1303 Kay, 2001, Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics, Nat Med, 7, 33, 10.1038/83324 Heise, 1997, ONYX-015, an E1B gene-attenuated adenovirus, causes tumor-specific cytolysis and antitumoral efficacy that can be augmented by standard chemotherapeutic agents, Nat Med, 3, 639, 10.1038/nm0697-639 Shenk T. Adenoviridae: the viruses and their replication. In: BN F, editor. Fundamental virology. 1996; p. 979-1016. Friedmann, 1995, Pseudotyped retroviral vectors for studies of human gene therapy, Nat Med, 1, 275, 10.1038/nm0395-275 Vigna, 2000, Lentiviral vectors: excellent tools for experimental gene transfer and promising candidates for gene therapy, J Gene Med, 2, 308, 10.1002/1521-2254(200009/10)2:5<308::AID-JGM131>3.0.CO;2-3 Fisher, 1997, Recombinant adeno-associated virus for muscle directed gene therapy, Nat Med, 3, 306, 10.1038/nm0397-306 Peng, 2000, Transduction of hepatocellular carcinoma (HCC) using recombinant adenoassociated virus (rAAV): in vitro and in vivo effects of genotoxic agents, J Hepatol, 32, 975, 10.1016/S0168-8278(00)80102-6 Burton, 2002, Gene delivery using herpes simplex virus vectors, DNA Cell Biol, 21, 915, 10.1089/104454902762053864 Chen, 2001, Orthopedic applications of gene therapy, J Orthop Sci, 6, 199, 10.1007/s007760100072 Lieberman, 2002, Gene transfer approaches to the healing of bone and cartilage, Mol Ther, 6, 141, 10.1006/mthe.2000.0663 Wu, 2003, Gene therapy and tissue engineering in repair of the musculoskeletal system, J Cell Biochem, 88, 467, 10.1002/jcb.10332 Sporn, 1990, TGF-beta: problems and prospects, Cell Regul, 1, 875, 10.1091/mbc.1.12.875 Shi, 2003, Mechanisms of TGF-beta signaling from cell membrane to the nucleus, Cell, 113, 685, 10.1016/S0092-8674(03)00432-X Urist, 1971, Bone morphogenetic protein, J Dent Res, 50, 1392, 10.1177/00220345710500060601 Sampath, 1981, Dissociative extraction and reconstitution of extracellular matrix components involved in local bone differentiation, Proc Natl Acad Sci USA, 78, 7599, 10.1073/pnas.78.12.7599 Hogan, 1996, Bone morphogenetic proteins: multifunctional regulators of vertebrate development, Genes Dev, 10, 1580, 10.1101/gad.10.13.1580 Cheng, 2003, Osteogenic activity of the fourteen types of human bone morphogenetic proteins (BMPs), J Bone Joint Surg Am, 85A, 1544, 10.2106/00004623-200308000-00017 Govender, 2002, Recombinant human bone morphogenetic protein-2 for treatment of open tibial fractures: a prospective, controlled, randomized study of four hundred and fifty patients, J Bone Joint Surg Am, 84A, 2123, 10.2106/00004623-200212000-00001 Johnsson, 2002, Randomized radiostereometric study comparing osteogenic protein-1 (BMP-7) and autograft bone in human noninstrumented posterolateral lumbar fusion: 2002 Volvo Award in clinical studies, Spine, 27, 2654, 10.1097/00007632-200212010-00004 Peng, 2002, Synergistic enhancement of bone formation and healing by stem cell-expressed VEGF and bone morphogenetic protein-4, J Clin Invest, 110, 751, 10.1172/JCI15153 Cook, 1995, Effect of recombinant human osteogenic protein-1 on healing of segmental defects in non-human primates, J Bone Joint Surg Am, 77A, 734, 10.2106/00004623-199505000-00010 Gerhart, 1993, Healing segmental femoral defects in sheep using recombinant human bone morphogenetic protein, Clin Orthop, 293, 317 Lieberman, 1999, The effect of regional gene therapy with bone morphogenetic protein-2-producing bone-marrow cells on the repair of segmental femoral defects in rats, J Bone Joint Surg Am, 81A, 905, 10.2106/00004623-199907000-00002 Breitbart, 1999, Gene-enhanced tissue engineering: applications for bone healing using cultured periosteal cells transduced retrovirally with the BMP-7 gene, Ann Plast Surg, 42, 488, 10.1097/00000637-199905000-00005 Lee, 2001, Effect of bone morphogenetic protein-2-expressing muscle-derived cells on healing of critical-sized bone defects in mice, J Bone Joint Surg Am, 83A, 1032, 10.2106/00004623-200107000-00008 Baltzer, 2000, Genetic enhancement of fracture repair: healing of an experimental segmental defect by adenoviral transfer of the BMP-2 gene, Gene Ther, 7, 734, 10.1038/sj.gt.3301166 Fang, 1996, Stimulation of new bone formation by direct transfer of osteogenic plasmid genes, Proc Natl Acad Sci USA, 93, 5753, 10.1073/pnas.93.12.5753 Hunziker, 1996, Repair of partial-thickness defects in articular cartilage: cell recruitment from the synovial membrane, J Bone Joint Surg Am, 78A, 721, 10.2106/00004623-199605000-00012 Johnstone, 1999, Autologous mesenchymal progenitor cells in articular cartilage repair, Clin Orthop, 367, 156, 10.1097/00003086-199910001-00017 Brittberg, 1994, Treatment of deep cartilage defects in the knee with autologous chondrocyte transplantation, N Engl J Med, 331, 889, 10.1056/NEJM199410063311401 Mason, 2000, Cartilage and bone regeneration using gene-enhanced tissue engineering, Clin Orthop, 379, 171, 10.1097/00003086-200010001-00023 Mi, 2000, Adenovirus-mediated gene transfer of insulin-like growth factor 1 stimulates proteoglycan synthesis in rabbit joints, Arthritis Rheum, 43, 2563, 10.1002/1529-0131(200011)43:11<2563::AID-ANR25>3.0.CO;2-8 Mi, 2003, Adverse effects of adenovirus-mediated gene transfer of human transforming growth factor beta 1 into rabbit knees, Arthritis Res Ther, 5, R132, 10.1186/ar745 Tomita, 1997, In vivo direct gene transfer into articular cartilage by intraarticular injection mediated by HVJ (Sendai virus) and liposomes, Arthritis Rheum, 40, 901, 10.1002/art.1780400518 Johnstone, 1998, In vitro chondrogenesis of bone marrow-derived mesenchymal progenitor cells, Exp Cell Res, 238, 265, 10.1006/excr.1997.3858 Wakitani, 1994, Mesenchymal cell-based repair of large, fullthickness defects of articular cartilage, J Bone Joint Surg Am, 76A, 579, 10.2106/00004623-199404000-00013 Baragi, 1997, Transplantation of adenovirally transduced allogeneic chondrocytes into articular cartilage defects in vivo, Osteoarthritis Cartilage, 5, 275, 10.1016/S1063-4584(97)80023-4 Alden, 1999, Percutaneous spinal fusion using bone morphogenetic protein-2 gene therapy, J Neurosurg, 90 Suppl, 109 Alden, 1999, In vivo endochondral bone formation using a bone morphogenetic protein 2 adenoviral vector, Hum Gene Ther, 10, 2245, 10.1089/10430349950017220 Wang, 2003, Effect of regional gene therapy with bone morphogenetic protein-2-producing bone marrow cells on spinal fusion in rats, J Bone Joint Surg Am, 85A, 905, 10.2106/00004623-200305000-00020 Boden, 1998, Lumbar spine fusion by local gene therapy with a cDNA encoding a novel osteoinductive protein (LMP-1), Spine, 23, 2486, 10.1097/00007632-199812010-00003 Amiel, 1995, Intrinsic properties of ACL and MCL cells and their responses to growth factors, Med Sci Sports Exerc, 27, 844, 10.1249/00005768-199506000-00008 Hildebrand, 1999, Early expression of marker genes in the rabbit medial collateral and anterior cruciate ligaments: the use of different viral vectors and the effects of injury, J Orthop Res, 17, 37, 10.1002/jor.1100170107 Nakamura, 1998, Early biological effect of in vivo gene transfer of platelet-derived growth factor (PDGF)-B into healing patellar ligament, Gene Ther, 5, 1165, 10.1038/sj.gt.3300712 Helm, 2001, A light and electron microscopic study of ectopic tendon and ligament formation induced by bone morphogenetic protein-13 adenoviral gene therapy, J Neurosurg, 95, 298, 10.3171/jns.2001.95.2.0298 Young, 1998, Use of mesenchymal stem cells in a collagen matrix for Achilles tendon repair, J Orthop Res, 16, 406, 10.1002/jor.1100160403 Charissoux, 1999, Treatment of experimental osteosarcoma tumors in rat by herpes simplex thymidine kinase gene transfer and ganciclovir, Anticancer Res, 19, 77 Mazzolini, 2003, Gene therapy of cancer with interleukin-12, Curr Pharm Des, 9, 1981, 10.2174/1381612033454261 Jia, 2003, Aerosol gene therapy with PEI: IL-12 eradicates osteosarcoma lung metastases, Clin Cancer Res, 9, 3462