Correction of murine galactosialidosis by bone marrow-derived macrophages overexpressing human protective protein/cathepsin a under control of the colony-stimulating factor-1 receptor promoter

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

Volumn 95, Issue 25, 1998, Pages 14880-14885

  Hahn, Christopher N ^a,b   Del Pilar Martin, Maria ^a   Zhou, Xiao Yan ^a,c   Mann, Linda W ^a   D'Azzo, Alessandra ^a  

^a ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^b HANSON INSTITUTE   (Australia)

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

Author keywords

[No Author keywords available]

Indexed keywords

CARBOXYPEPTIDASE C; COLONY STIMULATING FACTOR RECEPTOR;

ANIMAL CELL; ARTICLE; BONE MARROW TRANSPLANTATION; CELL INFILTRATION; CELL TRANSPLANTATION; CLINICAL FEATURE; GALACTOSIALIDOSIS; GENE OVEREXPRESSION; HEMATOPOIETIC TISSUE; HISTOCHEMISTRY; IMMUNOBLOTTING; IMMUNOCYTOCHEMISTRY; MACROPHAGE; MOUSE; NERVE CELL DEGENERATION; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; TISSUE DISTRIBUTION;

ANIMALS; BONE MARROW TRANSPLANTATION; CARBOXYPEPTIDASE C; CARBOXYPEPTIDASES; GENE THERAPY; HUMANS; LYSOSOMAL STORAGE DISEASES; MACROPHAGES; MICE; MICE, TRANSGENIC; PROMOTER REGIONS (GENETICS); RECEPTOR, MACROPHAGE COLONY-STIMULATING FACTOR; TISSUE THERAPY;

ANIMALIA; ATAXIA; MURINAE; MUS MUSCULUS;

EID: 0032425252     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.95.25.14880     Document Type: Article

References (41)

1. Neufeld, E. F. (1991) Annu. Rev. Biochem. 60, 257-280.
2. Scriver, C., Beaudet, A., Sly, W. & Valle, D., eds. (1995) The Metabolic and Molecular Bases of Inherited Disease (McGraw-Hill, New York).
3. Rattazzi, M. C. & Dobrenis, K. (1991) in Treatment of Genetic Diseases, ed. Desnick, R. J. (Churchill Livingstone, New York), PP. 131-152.
4. Haskins, M., Baker, H. J., Birkenmeier, E., Hoogerbrugge, P., Poorthuis, B., Sakiyama, T., Shull, R., Taylor, R., Thrall, M. & Walkley, S. U. (1991) Treatment of Genetic Diseases (Churchill Livingstone, New York).
5. Hoogerbrugge, P. M., Brouwer, O. F., Bordigoni, P., Ringden, O., Kapaun, P., Ortega, J. J., O'Meara, A., Cornu, G., Souillet, G., Frappaz, D., et al. (1995) Lancet 345, 1398-1402.
6. Shull, R. M., Hastings, N. E., Selcer, R. R., Jones, J. B., Smith, J. R., Cullen, W. C. & Constantopoulos, G. (1987) J. Clin. Invest. 79, 435-443.
7. Walkley, S. U., Thrall, M. A., Dobrenis, K., Huang, M., March, P. A., Siegel, D. A. & Wurzelmann, S. (1994) Proc. Natl. Acad. Sci. USA 91, 2970-2974.
8. O'Brien, J. S., Storb, R., Raff, R. F., Harding, J., Appelbaum, F., Morimoto, S., Kishimoto, Y., Graham, T., Ahern-Rindell, A. & O'Brien, S. L. (1990) Clin. Genet. 38, 274-280.
9. Birkenmeier, E. H., Barker, J. E., Vogler, C. A., Kyle, J. W., Sly, W. S., Gwynn, B., Levy, B. & Pegors, C. (1991) Blood 78, 3081.
10. Wolfe, J. H., Deshmane, S. L. & Fraser, N. W. (1992) Nat. Genet. 1, 379-384.
11. Walkley, S., Thrall, M., Dobrenis, K., March, P. A., Siegel, D. A. & Wurzelmann, S. (1993) J. Neuropathol. Exp. Neurol. 52, 315a(abstr.).
12. Crystal, R. (1995) Science 270, 404-410.
13. Marshall, E. (1995) Science 269, 1050-1055.
14. d'Azzo, A., Andria, G., Strisciuglio, P. & Galjaard, H. (1995) in The Metabolic and Molecular Bases of Inherited Disease, eds. Scriver, C., Beaudet, A., Sly, W. & Valle, D. (McGraw-Hill, New York), Vol. 2, pp. 2825-2838.
15. 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.
16. Roberts, W. M., Shapiro, L. H., Ashmun, R. A. & Look, A. T. (1992) Blood 79, 586-593.
17. Galjart, N. J., Gillemans, N., Harris, A., van der Horst, G. T. J., Verheijen, F. W., Galjaard, H. & d'Azzo, A. (1988) Cell 54, 755-764.
18. Marsh, J., Erfle, M. & Wykes, E. (1984) Gene 32, 481-485.
19. Grosveld, F., Blom van Assendelft, G., Greaves, D. R. & Kollias, G. (1987) Cell 51, 975-985.
20. Galjart, N. J., Morreau, H., Willemsen, R., Gillemans, N., Bonten, E. J. & d'Azzo, A. (1991) J. Biol. Chem. 266, 14754-14762.
21. Havenith, C., Askew, D. & Walker, W. (1998) GLIA 22, 348-359.
22. Sapi, E., Flick, M., Rodov, S., Gilmore-Hebert, M., Kelley, M., Rockwell, S. & Kacinski, B. (1996) Cancer Res. 56, 5704-5712.
23. Raivich, G., Gehrmann, J. & Kreutzberg, G. (1991) J. Neurosci. 30, 682-686.
24. Moore, S. C., McCormack, J. M., Armendariz, E., Gatewood, J. & Walker, W. S. (1992) J. Neuroimmunol. 41, 203-214.
25. Chang, Y., Albright, S. & Lee, F. (1994) J. Neuroimmunol. 52, 9-17.
26. Akiyama, H., Nishimura, T., Kondo, H., Ikeda, K., Hayashi, Y. & McGeer, P. L. (1994) Brain Res. 639, 171-174.
27. Rottier, R., Hahn, C., Mann, L., Martin, M., Smeyne, R., Suzuki, K. & d'Azzo, A. (1998) Hum. Mol. Gen. 7, 1787-1794.
28. Paabo, S., Bhat, B. M., Wold, W. S. & Peterson, P. A. (1987) Cell 50, 311-317.
29. Perry, V. H. & Gordon, S. (1991) in International Review of Cytology: A Survey of Cell Biology, eds. Jeon, K. W. & Friedlander, M. (Academic, San Diego), Vol. 125, pp. 203-244.
30. Kennedy, D. & Abkowitz, J. (1997) Blood 90, 986-993.
31. Ziai, M., Sangameswaran, L., Hempstead, J., Danho, W. & Morgan, J. (1988) J. Neurochem. 51, 1771-1776.
32. Kuemmel, T., Schroeder, R. & Stoffel, W. (1997) J. Neuropathol. Exp. Neurol. 56, 171-179.
33. Sherr, C. J., Rettenmier, C. W., Sacca, R., Roussel, M. F., Look, A. T. & Stanley, E. R. (1985) Cell 41, 665-676.
34. Reddy, M. A., Yang, B., Yue, X., Barnett, C. J. K., Ross, I. L., Sweet, M. J., Hume, D. A. & Ostrowski, M. C. (1994) J. Exp. Med. 180, 2309-2319.
35. Jin, D. I., Jameson, S. B., Reddy, M. A., Schenkman, D. & Ostrowski, M. C. (1995) Mol. Cell Biol. 15, 693-703.
36. Krall, W. J., Challita, P. M., Perlmutter, L. S., Skelton, D. C. & Kohn, D. B. (1994) Blood 83, 2737-2748.
37. Hickey, W. F. & Kimura, H. (1988) Science 239, 290-292.
38. Hickey, W. F., Vass, K. & Lassmann, H. (1992) J. Neuropath. Exp. Neurol. 51, 246-256.
39. Sands, M., Vogler, C., Torrey, A., Levy, B., Gwynn, B., Grubb, J., Sly, W. & Birkenmeier, E. (1997) J. Clin. Invest. 99, 1596-1605.
40. Miranda, S., Erlich, S., Freidrich, V., Haskins, M., Gatt, S. & Schuchman, E. (1998) Transplantation 65, 884-892.
41. Freeman, B., Vogler, C., Hofling, A., Nicholes, A. & Sands, M. (1997) Blood 90, 522a (abstr.).