Correction of the galactocerebrosidase deficiency in globoid cell leukodystrophy-cultured cells by SL3-3 retroviral-mediated gene transfer

Biochemical and Biophysical Research Communications

Volumn 218, Issue 3, 1996, Pages 766-771

  Gama Sosa, Miguel A ^a   De Gasperi, Rita ^a   Undevia, Samir ^a   Yeretsian, Joseph ^a   Rouse II, Strutha C ^a   Lyerla, Timothy A ^b   Kolodny, Edwin H ^a  

^a NEW YORK UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b CLARK UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GALACTOSYLCERAMIDASE; GALACTOSYLCERAMIDE; LYSOSOME ENZYME; MYELIN;

ANIMAL CELL; ARTICLE; AUTOSOMAL RECESSIVE INHERITANCE; CELL CULTURE; CONTROLLED STUDY; ENZYME DEFICIENCY; FIBROBLAST; GENE THERAPY; GENE TRANSFER; GLOBOID CELL LEUKODYSTROPHY; HUMAN; HUMAN CELL; LONG TERMINAL REPEAT; MOUSE; NEWBORN; NONHUMAN; PRIORITY JOURNAL; VIRUS RECOMBINANT;

ANIMALIA;

EID: 0005908553     PISSN: 0006291X     EISSN: None     Source Type: Journal    
DOI: 10.1006/bbrc.1996.0136     Document Type: Article

References (28)

1. Suzuki, K., Suzuki, Y., and Suzuki, K. (1995) in The Metabolic Basis of Inherited Disease (Scriver, C. R., Beudet, A. L., Sly, W. S., and Valle, D., Eds.), pp. 2671-2692. MacGraw-Hill, New York.
2. Kolodny, E. H., Raghavan, S., and Krivit, W. (1991) Dev. Neurosci. 13, 232-239.
3. Oehlmann, R., Zlotogora, J., Wenger, D. A., and Knowlton, R. G. (1993) Am J. Hum. Genet. 53, 1250-1255.
4. Chen, Y. Q., Rafi, M. A., deGala, G., and Wenger, D. A. (1993) Hum. Mol. Genet. 2, 1841-1845.
5. Luzi, P., Rafi, M. A., and Wenger, D. A. (1995) Genomics 26, 407-409.
6. Sakai, N., Inui, K., Fujii, N., Fukushima, H., Nishimoto, J., Yanagihara, I., Isegawa, Y., Iwamatsu, A., and Okada, S. (1994) Biochem. Biophys. Res. Commun. 198, 485-491.
7. Chen, Y. Q. and Wenger, D. A. (1993) Biochem. Biophys. Acta 1170, 53-61.
8. Sakai, N., Inui, K., Midorikawa, M., Okuno, Y., Ueda, S., Iwamatsu, A., and Okada, S. (1994) J. Biochem. 116, 615-620.
9. Rafi, M. A., Luzi, P., Chen, Y. Q., and Wenger, D. A. (1995) Hum. Mol. Genet. 4, 1285-1289.
10. Tatsumi, N., Inui, K., Sakai, N., Fukushima, H., Nishimoto, J., Yanagihara, I., Nishigaki, T., Tsukamoto, H., Fu, L., Taniike, M., and Okada, S. (1995) Hum. Mol. Genet. 4, 1865-1868.
11. Suzuki, S. (1994) J. Neuropathol. Exp. Neurol. 53, 359-363.
12. Konola, J. T., Lyerla, T. A., Skiba, M. C., and Raghavan, S. (1988) In Vitro Cell. Dev. Biol. 24, 575-580.
13. Danos, O. and Mulligan, R. C. (1988) Proc. Natl. Acad. Sci. USA 85, 6460-6464.
14. Cepko, C. L., Roberts, B. E., and Mulligan, R. C. (1984) Cell 37, 1053-1062.
15. Gama Sosa, M. A., Rosas, D. H., De Gasperi, R., Morita, E., Hutchison, M. R., and Ruprecht, R. M. (1994) J. Virol. 68, 2662-2670.
16. Wigler, M., Pellicer, A., Silverstein, S., and Axel, R. (1978) Cell 14, 725-731.
17. Wenger, D. A. and Williams, C. (1991) in Techniques in Diagnostic Human Biochemical Genetics (Hommes, F. A., Ed.), pp. 587-617, Wiley-Liss, New York.
18. Shapiro, E. G., Lockman, L. A., Balthazor, M., and Krivit, W. (1995) J. Inher. Metab. Dis. 18, 413-429.
19. Hoogerbrugge, P. M., Poorthuis, B. J. H. M., Roome, A., van de Kamp, J. J. P., Wagemaker, G., and van Bekkum, D. W. (1988) J. Clin. Invest. 81, 1790-1794.
20. Hoogerbrugge, P. M., Suzuki, K., Suzuki, K., Poorthuis, B. J. H. M., Kobayashi, T., Wagemaker, G., and van Bekkum, D. W. (1988) Science 239, 1035-1038.
21. Ichioka, T., Kishimoto, Y., Brennan, S., Santos, G. W., and Yeager, A. M. (1987) Proc. Natl. Acad. Sci. USA 84, 4259-4263.
22. Yeager, A. M., Brennan, S., Tiffany, C., Moser, H. W., and Santos, G. W. (1984) Science 225, 1052-1054.
23. Anderson, W. F. (1992) Science 256, 808-813.
24. Culver, K. W., Anderson, W. F., and Blaese, R. M. (1991) Hum. Gene Ther. 2, 107-109.
25. Miller, A. D. (1992) Nature 357, 455-460.
26. Crystal, R. G. (1995) Science 270, 404-410.
27. Gama Sosa, M. A., De Gasperi, R., Fazely, F., and Ruprecht, R. M. (1989) Biochem. Biophys. Res. Commun. 161, 305-311.
28. Gama Sosa, M. A., De Gasperi, R., Kim, Y.-S., Fazely, F., Sharma, P., and Ruprecht, R. M. (1991) AIDS Res. Hum. Retroviruses 7, 859-860.