Correction of the UDP-glucuronosyltransferase gene defect in the Gunn rat model of Crigler-Najjar syndrome type I with a chimeric oligonucleotide

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

Volumn 96, Issue 18, 1999, Pages 10349-10354

  Kren, Betsy T ^a   Parashar, Bhupesh ^b   Bandyopadhyay, Paramita ^a   Chowdhury, Namita Roy ^b   Chowdhury, Jayanta Roy ^b   Steer, Clifford J ^a  

^a UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^b ALBERT EINSTEIN COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ASIALOGLYCOPROTEIN RECEPTOR; GLUCURONOSYLTRANSFERASE; LIPOSOME; OLIGONUCLEOTIDE; POLYETHYLENEIMINE;

ANIMAL CELL; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CHIMERA; CONTROLLED STUDY; CRIGLER NAJJAR SYNDROME; DNA REPAIR; ENZYME DEFICIENCY; FRAMESHIFT MUTATION; GENE TARGETING; HYPERBILIRUBINEMIA; MALE; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; RAT;

ANIMALS; BASE SEQUENCE; BILIRUBIN; CHIMERA; CLONING, MOLECULAR; CRIGLER-NAJJAR SYNDROME; DISEASE MODELS, ANIMAL; FRAMESHIFT MUTATION; GENE THERAPY; GLUCURONOSYLTRANSFERASE; GUANOSINE; HUMANS; MOLECULAR SEQUENCE DATA; OLIGONUCLEOTIDES; RATS; RATS, MUTANT STRAINS; SEQUENCE DELETION;

ANIMALIA; RATTUS NORVEGICUS; RODENTIA;

EID: 0033621041     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.96.18.10349     Document Type: Article

References (45)

1. Roy Chowdhury, N., Gross, F., Moscioni, A. D., Kram, M., Arias, I. M. & Roy Chowdhury, J. (1987) J. Clin. Invest. 79, 327-334.
2. Bosma, P. J., Seppen, J., Goldhoorn, B., Bakker, C., Oude Elferink, R. P. J., Roy Chowdhury, J., Roy Chowdhury, N. & Jansen, P. L. M. (1994) J. Biol. Chem. 269, 17960-17964.
3. Crigler, J. F. & Najjar, V. A. (1952) Pediatrics 10, 169-172.
4. Arias, I. M. (1962) J. Clin. Invest. 41, 2233-2245.
5. Roy Chowdhury, J., Wolkoff, A. W., Roy Chowdhury, N. & Arias, I. M. (1995) in The Metabolic and Molecular Bases of Inherited Disease, eds. Scriver, C. R., Beaudet, A. L., Sly, W. S. & Valle, D. (McGraw-Hill, New York), pp. 2161-2208.
6. Iyanagi, T., Watanabe, T. & Uchiyama, Y. (1989) J. Biol. Chem. 264, 21302-21307.
7. Roy-Chowdhury, J., Huang, T., Kesari, K., Lederstein, M., Arias, I. M. & Roy-Chowdhury, N. (1991) J. Biol. Chem. 266, 18294-18298.
8. Askari, F. K., Hitomi, Y., Mao, M. & Wilson, J. M. (1996) Gene Ther. 3, 381-388.
9. Li, Q., Murphree, S. S., Willer, S. S., Bolli, R. & French, B. A. (1998) Hum. Gene Ther. 9, 497-505.
10. Takahashi, M., Ilan, Y., Roy Chowdhury, N., Guilda, J., Horwitz, M. & Roy Chowdhury, J. (1996) J. Biol Chem. 271, 26536-26542.
11. Ilan, Y., Prakash, R., Davidson, A., Jona, V., Droguett, G., Horwitz, M. S., Roy Chowdhury, N. & Roy Chowdhury, J. (1997) J. Clin. Invest. 99, 1098-1106.
12. Kmiec, E. B., Cole, A. & Holloman, W. K. (1994) Mol. Cell. Biol. 14, 7163-7172.
13. Yoon, K., Cole-Strauss, A. & Kmiec, E. B. (1996) Proc. Natl. Acad. Sci. USA 93, 2071-2076.
14. Cole-Strauss, A., Yoon, K., Xiang, Y., Byrne, B. C., Rice, M. C., Gryn, J., Holloman, W. K. & Kmiec, E. B. (1996) Science 273, 1386-1389.
15. Kotani, H. & Kmiec, E. B. (1994) Mol. Cell. Biol. 14, 6097-6106.
16. Kotani, H. & Kmiec, E. B. (1994) Mol. Cell. Biol. 14, 1949-1955.
17. Kmiec, E. B., Kren, B. T. & Steer C. J. (1999) in The Development of Human Gene Therapy, ed. Friedmann, T. (Cold Spring Harbor Lab. Press, Plainview, NY), pp. 643-670.
18. Havre, P. A. & Kmiec, E. B. (1998) Mol. Gen. Genet. 258, 580-586.
19. Kren, B. T., Metz, R., Kumar, R. & Steer, C. J. (1999) Sem. Liver Dis. 19, 93-104.
20. Kren, B. T., Cole-Strauss, A., Kmiec, E. B. & Steer, C. J. (1997) Hepatology 25, 1462-1468.
21. Kren, B. T., Bandyopadhyay, P. & Steer, C. J. (1998) Nat. Med. 4, 285-290.
22. Bandyopadhyay, P., Ma, X., Linehan-Stieers, C., Kren, B. T. & Steer, C. J. (1999) J. Biol. Chem. 274, 10163-10172.
23. Fox, I. J., Roy Chowdhury, J., Kaufman, S. S., Goertzen, T. C., Roy Chowdhury, N., Warkentin, P. I., Dorko, K., Sauter, B. V. & Strom, S. C. (1998) N. Engl. J. Med. 338, 1422-1426.
24. Abdallah, B., Hassan, A., Benoist, C., Goula, D., Behr, J. P. & Demeneix, B. A. (1996) Hum. Gene Ther. 7, 1947-1954.
25. Fox, I. J., Roy Chowdhury, N., Gupta, S., Kondapalli, R., Schilsky, M. L., Stockert, R. J. & Roy Chowdhury, J. (1995) Hepatology 21, 837-846.
26. Block, G. D., Locker, J., Bowen, W. C., Petersen, B. E., Katyal, S., Strom, S. C., Riley, T., Howard, T. A. & Michalopoulos, G. K. (1996) J. Cell Biol. 132, 1133-1149.
27. Hara, T., Aramaki, Y., Tsuchiya, S., Hosoi, K. & Okada, A. (1987) Biopharmaceutics. Drug Dispos. 8, 327-339.
28. Ilan, Y., Roy Chowdhury, N., Prakash, R., Jona, V., Attavar, P., Guha, C., Tada, K. & Roy Chowdhury, J. (1997) Transplantation 64, 8-13.
29. Huang, T.-J., Roy Chowdhury, J., Lahiri, P., Yerneni, P. C., Bommineni, V. R., Arias, I. M. & Roy Chowdhury, N. (1992) Hepatology. 16, 756-762.
30. Liu, L., Jang, G.-F., Farnsworth, C. C., Yokoyama, K., Glomset, J. A. & Gelb, M. H. (1995) Methods Enzymol. 250, 189-206.
31. Bandyopadhyay, P., Kren, B. T., Ma, X. & Steer, C. J. (1998) BioTechniques 25, 282-292.
32. Alexeev, V. & Yoon, K. (1998) Nat. Biotech. 16, 1343-1346.
33. Zhang, Z., Eriksson, M., Falk, G., Graff, C., Presnell, S. C., Read, M. S., Nichols, T. C., Blombäck, M. & Anvret, M. (1998) Antisense Nucleic Acid Drug Dev. 8, 531-536.
34. Blanckaert, N. (1980) Biochem. J. 185, 115-128.
35. Fevery, J., Blanckaert, N., Heirwegh, K. P. M., Préaux, A.-M. & Berthelot, P. (1977) J. Clin. Invest. 60, 970-979.
36. Gordon, E. R. & Goresky, C. A. (1980) Can. J. Biochem. 58, 1302-1310.
37. Teckman, J. H., Qu, D. & Perlmutter, D. H. (1996) Hepatology 24, 1504-1516.
38. Rice, M. C., Smith, S. T., Bullrich, F., Havre, P. & Kmeic, E. B. (1997) Proc. Natl. Acad. Sci. USA 94, 7417-7422.
39. Cole-Strauss, A., Gamper H., Holloman, W. K., Muñoz, M., Cheng, N. & Kmiec, E. B. (1999) Nucleic Acids Res. 27, 1323-1330.
40. Yáñez, R. J. & Porter, A. C. G. (1998) Gene Ther. 5, 149-159.
41. Kolodner, R. D. (1995) Trends Biochem. Sci. 20, 397-401.
42. Modrich, P. (1997) J. Biol. Chem. 272, 24727-24730.
43. Genschel, J., Littman, S. J., Drummond, J. T. & Modrich, P. (1998) J. Biol. Chem. 273, 19895-19901.
44. Umar, A., Risinger, J. I., Glaab, W. E., Tindall, K. R., Barrett, J. C. & Kunkel, T. A. (1998) Genetics 148, 1637-1646.
45. Santana, E., Peritz, A. E., Iyer, S., Uitto, J. & Yoon, K. (1998) J. Invest. Dermatol. 111, 1172-1177.