Improvement of skeletal lesions in mice with mucopolysaccharidosis type VII by neonatal adenoviral gene transfer

Molecular Therapy

Volumn 8, Issue 5, 2003, Pages 718-725

  Kanaji, Arihiko ^a,b,c   Kosuga, Motomichi ^a,b,c   Li, Xiao Kang ^a   Fukuhara, Yasuyuki ^a,b,c   Tanabe, Akiko ^a   Kamata, Yuko ^a,c   Azuma, Noriyuki ^a,c   Yamada, Masao ^a   Sakamaki, Toyonori ^b,c   Toyama, Yoshiaki ^b   Okuyama, Torayuki ^a,b,c  

^a National Center for Child Health and Development   (Japan)

^b KEIO UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^c NATIONAL CENTER FOR CHILD HEALTH AND DEVELOPMENT   (Japan)

Author keywords

Adenovirus; Gene therapy; Mucopolysaccharidosis; Skeletal lesions

Indexed keywords

ADENOVIRUS VECTOR; BETA GLUCURONIDASE; SOMATOMEDIN B RECEPTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; ARTICULAR CARTILAGE; BONE LESION; CELL VACUOLE; CLINICAL FEATURE; DISEASE MODEL; DRUG MECHANISM; ENZYME ACTIVITY; GENE TRANSFER; GROWTH PLATE; GROWTH RETARDATION; HISTOPATHOLOGY; KNEE; MOUSE; MUCOPOLYSACCHARIDOSIS TYPE 7; NEWBORN; NEWBORN PERIOD; NONHUMAN; PHENOTYPE; SKELETON MALFORMATION; VIRAL GENE DELIVERY SYSTEM;

ADENOVIRIDAE; ANIMALIA; MURINAE; TRIXIS;

EID: 10744225556     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2003.07.004     Document Type: Article

References (44)

1. Sly, W. S., Quinton, B. A., McAlister, W. H., and Rimoin, D. L. (1973). β-Glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis. J. Pediatr. 82: 249-257.
2. Birkenmeier, E. H., et al, (1989). Murine mucopolysaccharidosis type VII: characterization of a mouse with beta-glucuronidase deficiency. J. Clin. Invest. 83: 1258-1266.
3. Sands, M. S., and Birkenmeier, E. H. (1993). A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII: a single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII. Proc. Natl. Acad Sci. USA 90: 6567-6571.
4. Vogler, C., et al. (1990). A murine model of mucopolysaccharidosis VII: gross and microscopic findings in beta-glucuronidase-deficient mice. Am. J. Pathol. 136: 207-217.
5. Sands, M. S., et al. (1997). Gene therapy for murine mucopolysaccharidosis type VII. Neuromuscul. Disord. 7: 352-360.
6. Birkenmeier, E. H., et al. (1991). Increased life span and correction of metabolic defects in murine mucapolysaccharidosis type VII after syngeneic bone marrow transplantation. Blood 78: 3081-3092.
7. Sands, M. S., et al. (1994). Enzyme replacement therapy for murine mucopolysaccharidosis type VII. J. Clin. Invest. 93: 2324-2331.
8. Vogler, C., et al. (1997). Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation. J. Clin. Invest. 99: 1596-1605.
9. Vogler, C., et al. (1999). Enzyme replacement in murine mucapolysaccharidosis type VII: neuronal and glial response to β-glucuronidase requires early initiation of enzyme replacement therapy. Pediatr. Res. 45: 838-844.
10. O'Conner, L. H., et al. (1998). Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvement in behavior and auditory function. J. Clin. Invest. 101: 1394-1400.
11. Sands, M. S., et al. (1993). Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates. Lab. Invest. 68: 676-686.
12. Vogler, C., Sands, M. S., Levy, B., Galvin, N. J., Birkenmeier, E. H.,and Sly, W. S. (1999). Enzyme replacement with recombinant β-glucuronidase in murine mucopolysaccharidosis type VII: impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth, and survival. Pediatr. Res. 45: 838-844.
13. Gao, C., Sands, M. S., Harkins, M. E., and Ponder, K. P. (2000). Delivery of a retroviral vector expressing human β-glucuronidase to liver and spleen decreases lysosomal storage in mucopolysaccharidosis type VII mice. Mol. Ther. 2: 233-244, doi:10.1006/mthe.2000.0121..
14. Taylor, R. M., and Wolfe, J. H. (1997). Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of β-glucuronidase. Nat. Med. 3: 771-774.
15. Taylor, R. M., and Wolfe, J. H. (1994). Cross-correction of β-glucuronidase deficiency by retroviral vector-mediated gene transfer. Exp. Cell Res. 214: 606-613.
16. Marechal, V., Naffakh, N., Danos, O., and Heard, J. M. (1993). Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically modified bone marrow cells. Blood 84: 1358-1365.
17. Moullier, P., Bohl, D., Heard, J. M., and Danos, O. (1993). Correction of lysosomal storage in the liver and spleen of MPSVII mice by implantation of genetically modified skin fibroblasts. Nat. Genet. 4., 154-159.
18. Naffakh, N, Pinset, C, Montarras, D., Li, Z., Paulin, D, and Danos, O.(1996). Long-term secretion of therapeutic proteins from genetically modified skeletal muscles. Hum. Gene Ther. 7: 11-21.
19. Ponder, K. P., et al. (2002). Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs. Proc. Natl. Acad. Sci. USA 99: 13102-13107.
20. Zhu, J., Kang, W., Wolfe, J. H., and Fraser, N. W. (2000). Significantly increased expression of beta-glucuronidase in the central nervous system of mucopolysacchandosis type VII mice from the latency-associated transcript promoter in a nonpathogenic herpes simplex virus type 1 vector. Mol. Ther. 2: 82-94, doi:10.1006/mthe.2000.0093.
21. Wolfe, J. H., Deshmane, S. L., and Fraser, N. W. (1992). Herpesvirus vector gene transfer and expression of β-glucuronidase in the central nervous system of MPS VII mice. Nat. Genet. 1: 379-384.
22. Ghodsi, A., Stein, C., Derksen, T., Martins, I., Anderson, R. D., and Davidson, B. L. (1999). Systemic hyperosmolality improves β-glucuronidase distribution and pathology in murine MPS VII brain following intraventricular gene transfer. Exp. Neurol. 160: 109-116.
23. Ghodsi, A., Stein, C., Derksen, T., Yang, G., Anderson, R. D., and Davidson, B. L. (1998). Extensive β-glucuronidase activity in murine central nervous system after adenovirus-mediated gene transfer to brain. Hum. Gene Ther. 9: 2331-2340.
24. Ohashi, T., Watabe, K., Uehara, K., Sly, W. S., Vogler, C., and Eto, Y.(1997). Adenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice. Proc. Natl. Acad. Sci. USA 94: 1287-1292.
25. Kosuga, M., et al. (2001). Engraftment of genetically engineered amniotic epithelial cells corrects lysosomal storage in multiple areas of the brain in mucopolysaccharidosis type VII mice. Mol. Ther. 3: 139-148 , doi:10.1006/mthe.2000.0234..
26. Kosuga, M., et al. (2000). Phenotype correction in murine mucopolysaccharidosis type VII by transplantation of human amniotic epithelial cells after adenovirus-mediated gene transfer. Cell Transplant. 9: 687-692.
27. Kosuga, M., et al. (2000). Adenovirus-mediated gene therapy for mucopolysaccharidosis VII: involvement of cross-correction in widespread distribution of the gene products and long-term effects of CTLA-4Ig coexpression. Mol. Ther. 1: 406-413, doi: 10.1006/mthe.2000.0067..
28. Kamata, Y., et al. (2001). Adenovirus-mediated gene therapy for corneal clouding in mice with mucopolysaccharidosis type VII. Mol. Ther. 4: 307-312 , doi:10.1006/mthe.2001.0461..
29. Kamata, Y., et al. (2003). Long-term normalization in the central nervous system, ocular manifestations, and skeletal deformities caused by a single systemic adenovirus injection into neonatal mice with mucopolysaccharidosis VII. Gene Ther. 10: 406-414.
30. Stein, C. S., et al. (2001). In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors. Mol. Ther. 3: 850-856 , doi:10.1006/mthe.2001.0325..
31. Bosch, A., Perret, E., Desmaris, N., Trono, D., and Heard, J. M. (2000). Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer. Hum. Gene Ther. 11: 1139-1150.
32. Sferra, T. J., et al. (2000). Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysaccharidosis type VII mouse. Gene Ther. 11: 507-519.
33. Daly, T. M., Okuyama, T., Vogler, C., Haskins, M. E., Muzyczka, N., and Sands, M. S. (1999). Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged β-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice. Hum. Gene Ther. 10: 85-94.
34. Daly, T. M., Vogler, C., Levy, B., Haskins, M. E., and Sands, M. S.(1999). Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease. Proc. Natl. Acad. Sci. USA 96: 2296-2300.
35. Daly, T. M., Ohlemiler, K. K., Roberts, M. S., Vogler, C., and Sands, M.S. (2001). Prevention of systemic clinical disease in MPSVII mice following AAV-mediated neonatal gene transfer. Gene Ther. 8: 1292-1298.
36. Schultheiss, P. C., Gardner, S. A., Owens, J. M., Wenger, D. A., and Thrall, M. A. (2000). Mucopolysaccharidosis VII in a cat. Vet. Pathol. 37: 502-505.
37. Abreu, S., et al. (1995). Growth plate pathology in feline mucopolysaccharidosis VI. Calcif. Tissue Int. 57: 185-190.
38. Simonaro, C. M., Haskins, M. E., and Schuchman, E. H. (2001). Articular chondrocytes from animals with a dermatan sulfate storage disease undergo a high rate of apoptosis and release nitric oxide and inflammatory cytokines: a possible mechanism underlying degenerative joint disease in the mucopolysaccharidoses. Lab. Invest. 81: 1319-1328.
39. Silveri, P., Kaplan, S., Fallon, D., Bayever, E., and August, S. (1991). Hurler syndrome with special reference to histologic abnormalities of the growth plate. Clin. Orthop. 269: 305-311.
40. Arai, Y., et al. (1997). Adenovirus vector-mediated gene transduction to chondrocyte: in vitro evaluation of therapeutic efficacy of transforming growth factor-β1 and heat shock protein 70 gene transduction. J. Rheumatol. 24: 1787-1795.
41. Watanabe, S., Imagawa, T., Boivin, G. P., Gao, G., Wilson, J., and Hirsch, R. (2000). Adeno-associated virus vector mediates gene transfer and delivery of chondroprotective IL-4 to murine synovium. Mol. Ther. 2: 147-152 , doi:10.1006/mthe.2000.0111..
42. Gouze, E., et al. (2002). In vivo gene delivery to synovium by lentiviral vectors. Mol. Ther. 5: 397-404.
43. Schiller, A. L. (1995). Pathology of Osteoarthritis: Osteoarthritic Disorder (In K. E. Kunettner, and V. M. Goldgerg, Eds.), pp. 95-101. AAOS, Rosemont.
44. Wolfe, J. H., and Sands, M. S. (1996). Murine mucopolysaccharidosis type VII: a model system for somatic gene therapy of the central nervous system. Gene Transfer into Nervous System toward Gene Therapy of Neurological Disorders (in P. Lowenstain, and L. Enquist, Eds.), pp.263-274. Wiley. Essex, UK.