Safety assessment of intradiscal gene therapy II effect of dosing and vector choice

Spine

Volumn 33, Issue 14, 2008, Pages 1509-1516

  Levicoff, Eric A ^a   Kim, Joseph S ^a   Sobajima, Satoshi ^a   Wallach, Corey J ^a   Larson, James W ^a   Robbins, Paul D ^a   Xiao, Xiao ^a   Juan, Li ^a   Vadala, Gianluca ^b   Gilbertson, Lars G ^a   Kang, James D ^a  

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

^b UNIVERSITY CAMPUS BIO MEDICO   (Italy)

Author keywords

Adeno associated virus; Adenovirus; Gene therapy; Intervertebral disc; Safety

Indexed keywords

ADENOVIRUS VECTOR; BETA GALACTOSIDASE; BONE MORPHOGENETIC PROTEIN 2; COMPLEMENTARY DNA; GREEN FLUORESCENT PROTEIN; TRANSFORMING GROWTH FACTOR; BONE MORPHOGENETIC PROTEIN; TRANSFORMING GROWTH FACTOR BETA; TRANSFORMING GROWTH FACTOR BETA1;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CHEMICAL ANALYSIS; CONTROLLED STUDY; EPIDURAL SPACE; FEMALE; GENE DELIVERY SYSTEM; GENE THERAPY; HISTOLOGY; INTERVERTEBRAL DISK; MORBIDITY; NONHUMAN; PRIORITY JOURNAL; RISK ASSESSMENT; SUBARACHNOID SPACE; TRANSGENE; ADENO ASSOCIATED VIRUS; ADENOVIRUS; ANIMAL; DISEASE MODEL; GENE VECTOR; GENETICS; INTRASPINAL DRUG ADMINISTRATION; LACTOSE OPERON; LUMBAR VERTEBRA; METHODOLOGY; RABBIT; SPINE DISEASE;

ADENOVIRIDAE; ANIMALS; BONE MORPHOGENETIC PROTEINS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DNA, COMPLEMENTARY; EPIDURAL SPACE; FEMALE; GENE THERAPY; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; INJECTIONS, SPINAL; LAC OPERON; LUMBAR VERTEBRAE; RABBITS; SPINAL DISEASES; TRANSFORMING GROWTH FACTOR BETA; TRANSFORMING GROWTH FACTOR BETA1;

EID: 50649112211     PISSN: 03622436     EISSN: 15281159     Source Type: Journal    
DOI: 10.1097/BRS.0b013e318178866c     Document Type: Article

References (32)

1. Frymoyer JW, Durett CL. The economics of spinal disorders. In: Frymoyer JW, Ducker TB, Hadler NM, et al, eds. The Adult Spine: Principles and Practice. Philadelphia, PA: Lippincott-Raven; 1997;143-50.
2. Lively MW. Sports medicine approach to low back pain. South Med J 2002; 95:642-6.
3. Buckwalter JA. Aging and degeneration of the human intervertebral disc. Spine 1995;20:1307-14.
4. Moore RJ, Crotti TN, Osti OL, et al. Osteoarthrosis of the facet joints resulting from anular rim lesions in sheep lumbar discs. Spine 1999;24: 519-25.
5. Vernon-Roberts B. Disc pathology and disease states. In: Ghosh, P, ed. The Biology of the Intervertebral Disc Vol II. Boca Raton, FL: CRC Press; 1988: 73-119.
6. Kang JD, Georgescu HI, McIntyre-Larkin L, et al. Herniated lumber intervertebral discs spontaneously produce matrix metalloproteinases, nitric oxide, interleukin-6, and prostaglandin E2. Spine 1996;21:271-7.
7. Kanemoto M, Hukuda S, Komiya Y, et al. Immunohistochemical study of matrix metalloproteinase-3 and tissue inhibitor of metalloproteinase-1 human intervertebral discs. Spine 1996;21:1-8.
8. Yoon ST, Park JS, Kim KS, et al. ISSLS prize winner: LMP-1 upregulates intervertebral disc cell production of proteoglycans and BMPs in vitro and in vivo. Spine 2004;29:2603-11.
9. Moon S, Nishida K, Gilbertson LG, et al. Biologic Response of Human Intervertebral Disc Cell to Gene Therapy Cocktail. Orthopaedic Research Society, San Francisco, CA; 2001.
10. Paul R, Haydon RC, Cheng H, et al. Potential use of Sox9 gene therapy for intervertebral degenerative disc disease. Spine 2003;28:755-63.
11. Wallach CJ, Sobajima S, Watanabe Y, et al. Gene transfer of the catabolic inhibitor TIMP-1 increases measured proteoglycans in cells from degenerated human intervertebral discs. Spine 2003;28:2331-7.
12. Marshall E. Gene therapy death prompts review of adenovirus vector. Science1999;286:2244-5.
13. Blacklow NR. Adeno-associated virus of humans. In: Pattison JR, ed. Parvovirus and Human Disease. Boca Raton, FL: CRC Press; 1988;165-74.
14. Carter PJ, Samulski RJ. Adeno-associated viral vectors as gene delivery vehicles. Int J Mol Med 2000;6:17-27.
15. Tal J. Adeno-associated virus-based vectors in gene therapy. J Biomed Sci2000;7:279-91.
16. Favre D, Provost N, Blouin V, et al. Immediate and long-term safety of recombinant adeno-associated virus injection into the nonhuman primate muscle. Mol Ther 2001;4:559-66.
17. Lattermann C, Oxner WM, Xiao X, et al. The adeno-associated viral vector as a strategy for intradiscal gene transfer in immune competent and preexposed rabbits. Spine 2005;30:497-504.
18. Wallach CJ, Kim JS, Sobajima S, et al. Safety assessment of intradiscal gene transfer: a pilot study. Spine J 2006;6:107-12.
19. Nishida K, Kang JD, Suh JK, et al. Adenovirus-mediated gene transfer to nucleus pulposus cells. Implications for the treatment of intervertebral disc degeneration. Spine 1998;23:2437-42.
20. Graham FL, van der Eb AJ. A new technique for the assay of infectivity of human adenovirus 5 DNA. Virology 1973;52:456-67.
21. Xiao X, Li J, Samulski RJ. Production of high-titer recombinant adenoassociated virus vectors in the absence of helper adenovirus. J Virol 1998; 72:2224-32.
22. Moon SH, Gilbertson LG, Nishida K, et al. Human intervertebral disc cells are genetically modifiable by adenovirus-mediated gene transfer: implications for the clinical management of intervertebral disc disorders. Spine2000;25:2573-9.
23. Brodsky A, Binder W. Lumbar discography: its value in diagnosis and treatment of lumbar disc lesions. Spine 1979;4:110-20.
24. Zaiss AK, Liu Q, Bowen GP, et al. Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors. J Virol 2002; 76:4580-90.
25. Boulis NM, Willmarth NE, Song DK, et al. Intraneural colchicine inhibition of adenoviral and adeno-associated viral vector remote spinal cord gene delivery. Neurosurgery 2003;52:381-7.
26. Nishida K, Kang JD, Gilbertson LG, et al. Modulation of the biologic activity of the rabbit intervertebral disc by gene therapy: an in vivo study of adenovirus-mediated transfer of the human transforming growth factor beta 1 encoding gene. Spine 1999;24:2419-25.
27. Eslami MH, Gangadharan SP, Sui X, et al. Gene delivery to in situ veins: differential effects of adenovirus and adeno-associated viral vectors. J Vasc Surg 2000;31:1149-59.
28. Christ M. Preclinical evaluation of gene transfer products: safety and immunological aspects. Toxicology 2002;174:13-9.
29. Xiao X, Li J, Samulski RJ. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol 1996; 70:8098-108.
30. McPhee SW, Janson CG, Li C, et al. Immune responses to AAV in a phase I study for Canavan disease. J Gen Med 2006;8:577-88.
31. Mizuguchi H, Xu ZL, Sakurai F, et al. Tight positive regulation of transgene expression by a single adenovirus vector containing the rtTA and tTS expression cassettes in separate genome regions. Hum Gene Ther 2003;14: 1265-77.
32. Ueblacker P, Wagner B, Kruger A, et al. Inducible nonviral gene expression in the treatment of osteochondral defects. Osteoarthritis Cartilage 2004;12: 711-9.