Transgene produces massive overexpression of human β-glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors

Proceedings of the National Academy of Sciences of the United States of America

Volumn 100, Issue 5, 2003, Pages 2669-2673

  Vogler, Carole ^a   Galvin, Nancy ^a   Levy, Beth ^a   Grubb, Jeffery ^a   Jiang, Jinxing ^a   Zhou, Xiao Yan ^a   Sly, William S ^a  

^a SAINT LOUIS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Gene therapy; Lysosomal storage disease; MPS VII; Sly disease; Tumor susceptibility

Indexed keywords

BETA GLUCURONIDASE; LYSOSOME ENZYME;

ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BONE MARROW TRANSPLANTATION; BREAST CARCINOMA; CANCER SUSCEPTIBILITY; CELL TYPE; CUSHING SYNDROME; ENZYME REPLACEMENT; FEMALE; GENE OVEREXPRESSION; GENE THERAPY; HYPOPHYSIS ADENOMA; LYMPHOMA; LYSOSOME STORAGE DISEASE; MALE; MENINGIOMA; MOUSE; MUCOPOLYSACCHARIDOSIS; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; SLY DISEASE; TRANSGENE; TRANSGENIC MOUSE; VIRAL GENE THERAPY;

ANIMALS; CELL LINE; DNA, COMPLEMENTARY; FEMALE; GENE THERAPY; GLUCURONIDASE; IMMUNOHISTOCHEMISTRY; LYSOSOMES; MALE; MICE; MICE, INBRED C57BL; MICE, TRANSGENIC; MICROSCOPY, ELECTRON; MUCOPOLYSACCHARIDOSIS VII; ONCOGENES; SEX FACTORS; TRANSGENES;

ANIMALIA; MUS MUSCULUS;

EID: 0037418258     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.0437941100     Document Type: Article

References (22)

1. Neafeld, E. F. & Muenzer, J. (2001) in The Metabolic and Molecular Basis of Inherited Disease, eds. Scriver, C. R., Beaudet, A. L., Sly, W. S. & Valle, D. (McGraw-Hill, New York), pp. 3421-3452.
2. Sands, M. S., Barker, J. E., Vogler, C., Levy, B., Gwynn, B., Galvin, N., Sly, W. S. & Birkenmeier, E. (1993) Lab. Invest. 68, 676-686.
3. Kakkis, E. D., Muenzer, J., Tiller, G. E., Waber, L., Belmont, J., Passage, M., Izykowski, B., Phillips, J., Doroshow, R., Walot, I., et al. (2001) N. Engl. J. Med. 344, 182-188.
4. Vogler, C., Barker, J., Sands, M. S., Levy, B., Galvin, N. & Sly, W. S. (2001) Pediatr. Dev. Pathol. 4, 421-433.
5. Hahn, C. N., del Pilar Martin, M., Zhou, X. Y., Mann, L. W. & d'Azzo, A. (1998) Proc. Natl. Acad. Sci. USA 95, 4880-14885.
6. Zhou, X. Y., Morreau, H., Rottier, R., Davis, D., Bonten, E., Gillemans, N., Wenger, D., Grosveld, F. G., Doherty, P., Suzuki, K., et al. (1995) Genes Dev. 9, 2623-2634.
7. Daly, T. M., Vogler, C., Levy, B., Haskins, M. E. & Sands, M. S. (1999) Proc. Natl. Acad. Sci. USA 96, 2296-2300.
8. Daly, T. M., Ohlemiller, K. K., Roberts, M. S., Vogler, C. A. & Sands, M. S. (2001) Gene Ther. 8, 1291-1298.
9. Donsante, A., Vogler, C., Muzyczka, N., Crawford, J. M., Barker, J., Flotte, T., Campbell-Thompson, M., Daly, T. & Sands, M. S. (2001) Gene Ther. 8, 1343-1346.
10. Oshima, A., Kyle, J. W., Miller, R. D., Hoffmann, J. W., Powell, P. P., Grubb, J. H., Sly, W. S., Tropak, M., Guise, K. S. & Gravel, R. A. (1987) Proc. Natl. Acad. Sci. USA 84, 685-689.
11. Niwa, H., Yamamura, K. & Miyazaki, J. (1991) Gene 108, 193-200.
12. Sands, M. S., Vogler, C., Torrey, A., Levy, B., Gwynn, B., Grubb, J. & Sly, W. S. (1997) J. Clin. Invest. 99, 1596-1605.
13. Vogler, C., Birkenmeier, E. H., Sly, W. S., Levy, B., Pegors, C., Kyle, J. W. & Beamer, W. G. (1990) Am. J. Pathol. 136, 207-217.
14. Kiernan, J. A. (1990) in Histological and Histochemical Methods: Theory and Practice (Pergamon, New York), pp. 182-186.
15. Kyle, J. W., Birkenmeier, E. H., Gwynn, B., Vogler, C., Hoppe, P. C., Hoffmann, J. W. & Sly, W. S. (1990) Proc. Natl. Acad. Sci. USA 87, 3914-3918.
16. Wu, B. M., Tomatsu, S., Fukuda, S., Sukegawa, K., Orii, T. & Sly, W. S. (1994) J. Biol. Chem. 269, 23681-23688.
17. Ioannou, Y. A., Bishop, D. F. & Desnick, R. J. (1992) J. Cell Biol. 119, 1137-1150.
18. Loser, P., Jennings, G. S., Strauss, M. & Sandig, V. (1998) J. Virol. 72, 180-190.
19. Meikle, P. J., Brooks, D. A., Ravenscroft, E. M., Yan, M., Williams, R. E., Jaunzems, A. E., Chataway, T. K., Karageorgos, L. E., Davey, R. C., Boulter, C. D., et al. (1997) Clin. Chem. 43, 1325-1335.
20. Gonzalez-Noriega, A., Grubb, J. H., Talkad, V. & Sly, W. S. (1980) J Cell Biol. 85, 839-852.
21. Buckley, R. H. (2002) Lancet 360, 1185-1186.
22. Check, E. (2002) Nature 420, 116-117.