Correction of glycogen storage disease type 1a in a mouse model by gene therapy

Journal of Biological Chemistry

Volumn 275, Issue 2, 2000, Pages 828-832

  Zingone, Adriana ^a   Hiraiwa, Hisayuki ^a   Pan, Chi Jiunn ^a   Lin, Baochuan ^a   Chen, Hungwen ^b   Ward, Jerrold M ^c   Chou, Janice Yang ^a  

^a EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT   (United States)

^b INSTITUTE OF BIOLOGICAL CHEMISTRY   (Taiwan)

^c NATIONAL CANCER INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CHOLESTEROL; GLUCOSE; GLUCOSE 6 PHOSPHATASE; GLUCOSE 6 PHOSPHATE; PHOSPHATASE; TRIACYLGLYCEROL; URIC ACID; VIRUS VECTOR;

ANIMAL CELL; ANIMAL TISSUE; ARTICLE; GENE THERAPY; GLUCOSE HOMEOSTASIS; GLYCOGEN STORAGE DISEASE TYPE 1; MOUSE; NEWBORN; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; SURVIVAL RATE; SYMPTOMATOLOGY;

ADENOVIRIDAE; ANIMALS; DISEASE MODELS, ANIMAL; GENE THERAPY; GENETIC VECTORS; GLUCOSE-6-PHOSPHATASE; GLUCOSE-6-PHOSPHATE; GLYCOGEN STORAGE DISEASE TYPE I; HUMANS; LIVER; MICE; MICE, KNOCKOUT; MICROSOMES, LIVER;

ANIMALIA; MURINAE;

EID: 0033979899     PISSN: 00219258     EISSN: None     Source Type: Journal    
DOI: 10.1074/jbc.275.2.828     Document Type: Article

References (24)

1. Chen, Y.-T., and Burchell, A. (1995) in The Molecular and Metabolic Bases of Inherited Disease (Scriver, C. R., Beaudet, A. L., Sly, W. S., and Valle, D., eds) 7th Ed., pp. 935-965, McGraw-Hill Book Co., New York
2. Nordlie, R. C., and Sukalski, K. A. (1985) in The Enzymes of Biological Membranes (Martonosi, A. N., ed) 2nd Ed., pp. 349-398, Plenum Press, New York
3. Pan, C.-J., Lei, K.-J., Annabi, B., Hemrika, W., and Chou, J. Y. (1998) J. Biol. Chem. 273, 6144-6148
4. Arion, W. J., Lange, A. J., Walls, H. E., and Ballas, L. M. (1980) J. Biol. Chem. 255, 10396-10406
5. Lei, K.-J., Chen, H., Pan, C.-J., Ward, J. M., Mosinger, B., Lee, E. J., Westphal, H., and Chou, J. Y. (1996) Nat. Genet. 13, 203-209
6. Hiraiwa, H., Pan, C. -J., Lin, B., Moses, S. W., and Chou, J. Y. (1999) J. Biol. Chem. 274, 5532-5536
7. Chen, Y.-T., Cornblath, M., and Sidbury, J. B. (1984) N. Engl. J. Med. 310, 171-175
8. Emmett, M., and Narins, R. G. (1978) J. Am. Med. Assoc. 239, 1642-1644
9. Chen, Y.-T., and Scheinman, J. I. (1991) J. Inherited Metab. Dis. 14, 80-86
10. Kirschner, B. S., Baker, A. L., and Thorp, F. K. (1991) Gastroenterology 101, 238-241
11. Selby, R., Starzl, T. E., Yunis, E., Todo, S., Tzakis, A. G., Brown, B. I., and Kendall, R. S. (1993) Eur. J. Pediatr. 152, Suppl. 1, S71-S76
12. Engelhardt, J. F., Ye, X., Doranz, B., and Wilson, J. M. (1994) Proc. Natl. Acad. Sci. U. S. A. 91, 6196-6200
13. Yang, Y., Nunes, F. A., Berencsi, K., Gonczol, E., Engelhardt, J. F., and Wilson, J. M. (1994) Nat. Genet. 7, 362-369
14. Mittereder, N., Yei, S., Bachurski, C., Cuppoletti, J., Whitsett, J. A., Tolstoshev, P., and Trapnell, B. C. (1994) Hum. Gene Ther. 5, 719-731
15. Shelly, L. L., Lei, K.-J., Pan, C.-J., Sakata, S. F., Ruppert, S., Schutz, G., and Chou, J. Y. (1993) J. Biol. Chem. 268, 21482-21485
16. Teutsch, H. F. (1981) in Progress in Histochemistry and Cytochemistry: Chemomorphology of Liver Paraenchyma (Graumann, W., Lojda, Z., Pearse, A. G. E., and Schlebler, T. H., eds) Vol. 14, p. 2.2.2., Gustav Fisher Verlag, Stuttgart
17. Teutsch, H. F. (1981) in Progress in Histochemistry and Cytochemistry: Chemomorphology of Liver Paraenchyma (Graumann, W., Lojda, Z., Pearse, A. G. E., and Schlebler, T. H., eds) Vol. 14, p. 2.2.5., Gustav Fisher Verlag, Stuttgart
18. Stratford-Perricaudet, L. D., and Perricaudet, M. (1991) in Gene Therapeutics: Methods and Applications of Direct Gene Transfer (Wolff, J. A., ed) pp. 345-362, Birkhauser Boston Inc., Boston
19. Kozarsky, K. F., and Wilson, J. M. (1993) Curr. Opin. Genet. Dev. 3, 499-503
20. Trapnell, B. C. (1993) Adv. Drug Delivery Rev. 12, 185-199
21. Pan, C.-J., Lei, K.-J., Chen, H., Ward, J. M., and Chou, J. Y. (1998) Arch. Biochem. Biophys. 358, 17-24
22. Lin, B., Hiraiwa, H., Annabi, B., Pan, C.-J., and Chou, J. Y. (1998) J. Biol. Chem. 273, 31656-31670
23. Argaud, D., Kirby, T. L., Newgard, C. B., and Lange, A. J. (1997) J. Biol. Chem. 272, 12854-12861
24. Iynedjian, P. B., Ucla, C., and Mach, B. (1987) J. Biol. Chem. 262, 6032-6038