In vivo correction with recombinant adenovirus of 4-hydroxyphenylpyruvic acid dioxygenase deficiencies in strain III mice

Human Gene Therapy

Volumn 8, Issue 1, 1997, Pages 65-71

  Kubo, Shuji ^a   Kiwaki, Kohji ^a   Awata, Hisataka ^a   Katoh, Hideki ^b   Kanegae, Yumi ^c   Saito, Izumu ^c   Yamamoto, Tetsuro ^a   Miyazaki, Jun Ichi ^d   Matsuda, Ichiro ^a   Endo, Fumio ^a,e  

^a KUMAMOTO UNIVERSITY   (Japan)

^b CENTRAL INSTITUTE FOR EXPERIMENTAL ANIMALS   (Japan)

^c UNIVERSITY OF TOKYO   (Japan)

^d TOHOKU UNIVERSITY   (Japan)

^e KUMAMOTO UNIVERSITY   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

4 HYDROXYPHENYLPYRUVATE DIOXYGENASE; COMPLEMENTARY DNA; LIVER ENZYME; TYROSINE;

ADENOVIRUS; AMINO ACID BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; ENZYME ACTIVITY; ENZYME DEFICIENCY; EXPRESSION VECTOR; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; IMMUNOBLOTTING; MOUSE; NONHUMAN; NORTHERN BLOTTING; PROMOTER REGION; TYROSINEMIA; VIRUS RECOMBINANT;

EID: 16944366244     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1997.8.1-65     Document Type: Article

References (30)

1. AUSUBEL, F.M., BRENT, R., KINGSTON, R.E., MOORE, D.D., SEIDMAN, J.G., SMITH, J.A., and STRUHL, K., eds. (1987). Current Protocols in Molecular Biology. (Wiley & Sons Inc., New York) Suppl. 1.
2. BUCKTHAL, D.J., and ROCHE, P.A. (1987). 4-Hydroxyphenylpyruvate dioxygenase from pig liver. Methods Enzymol. 142, 132-138.
3. ENDO, F., KITANO, A., UEHARA, I., NAGATA, N., MATSUDA, I., SHINKA, T., KUHARA, T., and MATSUMOTO, I. (1983). Four-hydroxyphenylpyruvic acid oxidase deficiency with normal fumarylacetoacetase: A new variant form of hereditary hypertyrosinemia. Pediatr. Res. 17, 92-96.
4. ENDO, F., KATOH, H., YAMAMOTO, S., and MATSUDA, I. (1991). A murine model for type III tyrosinemia: lack of immunologically detectable 4-hydroxyphenylpyruvic acid dioxygenase enzyme protein in a novel mouse strain with hypertyrosinemia. Am. J. Hum. Genet. 48, 704-709.
5. ENDO, F., AWATA, H., TANOUE, A., ISHIGURO, M., EDA, Y., TITANI, K., and MATSUDA, I. (1992). Primary structure deduced from complementary DNA sequence and expression in cultured cells of mammalian 4-hydroxyphenylpyruvic acid dioxygenase. Evidence that the enzyme is a homodimer of identical subunits homologous to rat liver-specific alloantigen. F. J. Biol. Chem. 267, 24235-24240.
6. ENDO, F., AWATA, H., KATOH, H., and MATSUDA, I. (1995). A nonsense mutation in the 4-hydroxyphenylpyruvic acid dioxygenase gene (Hpd) causes skipping of the constitutive exon and hypertyrosinemia in mouse strain III. Genomics 25, 164-169.
7. FANG, B., EISENSMITH, R.C, LI, X.H., FINEGOLD, M.J., SHEDLOVSKY, A., DOVE, W., and WOO, S.L. (1994). Gene therapy for phenylketonuria: phenotypic correction in a genetically deficient mouse model by adenovirus-mediated hepatic gene transfer. Gene Ther. 1, 247-254.
8. FELLMAN, J.H. (1987). 4-Hydroxyphenylpyruvate dioxygenase from avian liver. Methods Enzymol. 142, 148-154.
9. GIARDINI, O., CANTANI, A., KENNEWAY, N.G., and D'EUFEMIA, P. (1983). Chronic tyrosinemia associated with 4-hydroxyphenylpyruvate dioxygenase deficiency with acute intermittent ataxia and without visceral and bone involvement. Pediatr. Res. 17, 25-29.
10. GREBER, U.F., WILLETTS, M., WEBSTER, P., and HELENIUS, A. (1993). Stepwise dismantling of adenovirus 2 during entry into cells. Cell 75, 477-486.
11. HERZ, J., and GERARD, R.D. (1993). Adenovirus-mediated transfer of low density lipoprotein receptor gene acutely accelerates cholesterol clearance in normal mice. Proc. Natl. Acad. Sci. USA 90, 2812-2816.
12. HUARD, J., OCHMULLER, H., ACSADI, G., MASSIE, B., and KARPATI, G. (1995). The route of administration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants. Gene Ther. 2, 107-115.
13. ISHIBASHI, S., BROWN, M.S., GOLDSTEIN, J.L., GERARD, R.D., HAMMER, R.E., and HERZ, J. (1993). Hypercholesterolemia in low density lipoprotein receptor knockout mice and its reversal by adenovirus-mediated gene delivery. J. Clin. Invest. 92, 883-893.
14. KANEGAE, Y., MAKIMURA, M., and SAITO, I. (1994). A simple and efficient method for purification of infectious recombinant adenovirus. Jpn. J. Med. Sci. Biol. 47, 157-166.
15. KASS-EISLER, A., FALCK-PEDERSEN, E., ELFENBEIN, D.H., ALVIRA, M., BUTTRICK, P.M., and LEINWAND, L.A. (1994). The impact of developmental stage, route of administration and the immune system on adenovirus-mediated gene transfer. Gene Ther. 1, 395-402.
16. KATOH, H., ENDO, F., SUZUKI, K., and MATSUDA, I. (1991). Hereditary hypertyrosinemia mouse. Mouse Genome 89, 572.
17. KAY, M.A., LANDEN, C.N., ROTHENBERG, S.R., TAYLOR, L.A., LELAND, F., WIEHLE, S., FANG, B., BELLINGER, D., FINEGOLD, M., THOMPSON, A.R., READ, M., BRINKHOUS, K.M., and WOOL, S.L.C. (1994). In vivo hepatic gene therapy: Complete albeit transient correction of factor IX deficiency in hemophilia B dogs. Proc. Natl. Acad. Sci. USA 91, 2353-2357.
18. ash mice and OTC deficient human hepatocytes with recombinant adenoviruses bearing the CAG promoter. Hum. Gene Ther. 7, 615-624.
19. KOZARSKY, K.F., McKINLEY, D.R., AUSTIN, L.L., RAPER, S.E., STRATFORD-PERRICAUDET, L.D., and WILSON, J.M. (1994). In vivo correction of low density lipoprotein receptor deficiency in the Watanabe heritable hyperlipidemic rabbit with recombinant adenoviruses. J. Biol. Chem. 269, 13695-13702.
20. KREMER, E.J., and PERRICAUDET, M. (1995). Adenovirus and adeno-associated virus mediated gene transfer. Br. Med. Bull. 51, 31-44.
21. LI, Q., KAY, M.A., FINEGOLD, M., STRATFORD-PERRICAUDET, L.D., and WOO, S.L.C. (1993). Assessment of recombinant adenoviral vectors for hepatic gene therapy. Hum. Gene Ther. 4, 403-409.
22. LINDBLAD, B., LINDSTEDT, S., and STEEN, G. (1977). On the enzymic defects in hereditary tyrosinemia. Proc. Natl. Acad. Sci. USA 74, 4641-4645.
23. LINDSTEDT, S., and ODELHOG, B. (1987). 4-Hydroxyphenylpyruvate dioxygenase from pseudomonas. Methods Enzymol. 142, 143-148.
24. MITCHELL, G.A., LAMBERT, M., and TANGUAY, R.M. (1995). Hypertyrosinemia. In The Metabolic and Molecular Basis of Inherited Disease, 7th ed. C.R. Scriver, A.L. Beaudet, W.S. Sly, and D. Valle, eds (McGraw-Hill, New York) pp. 1077-1106.
25. MIYAKE, S., MAKIMURA, M., KANEGAE, Y., HARADA, S., SATO, Y., TAKAMORI, K., TOKUDA, C., and SAITO, I. (1996). Efficient generation of recombinant adenoviruses using adenovirus DNA-terminal protein complex and a cosmid bearing the full-length virus genome. Proc. Natl. Acad. Sci. USA 93, 1320-1324.
26. MIYAZAKI, J., TAKAKI, S., ARAKI, K., TASHIRO, F., TOMINAGA, A., TAKATSU, K., and YAMAMURA, K. (1989). Expression vector system based on the chicken β-actin promoter directs efficient production of interleukin-5. Gene 79, 269-277.
27. MORSY, M.A., ALFORD, E.L., BETT, A., GRAHAM, F.L., and CASKEY, C.T. (1993). Efficient adenoviral-mediated ornithine transcarbamylase expression in deficient mouse and human hepatocytes. J. Clin. Invest. 92, 1580-1586.
28. NIWA, H., YAMAMURA, K., and MIYAZAKI, J. (1991). Efficient selection for high-expression transfectants with a novel eukaryotic vector. Gene 108, 193-199.
29. STRATFORD-PERRICAUDET, L.D., LEVRERO, M., CHASSE, J.F., PERRICAUDET, M., and BRIAND, P. (1990). Evaluation of the transfer and expression in mice of an enzyme-encoding gene using a human adenovirus vector. Hum. Gene Ther. 1, 241-256.
30. UENO, T., MIYAMURA, T., SAITO, I., and MIZUNO, K. (1993). Immortalization of differentiated human hepatocytes by a combination of a viral vector and collagen gel culture. Hum. Cell 6, 126-135.