Slow-onset inhibition of fumarylacetoacetate hydrolase by phosphinate mimics of the tetrahedral intermediate: Kinetics, crystal structure and pharmacokinetics

Biochemical Journal

Volumn 402, Issue 2, 2007, Pages 251-260

  Bateman, Raynard L ^a,b,d   Ashworth, Justin ^a,e   Witte, John F ^a   Baker, L J ^c   Bhanumoorthy, Pullooru ^c   Timm, David E ^c,f   Hurley, Thomas D ^c   Grompe, Markus ^b   McClard, Ronald W ^a  

^a REED COLLEGE   (United States)

^b OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^c INDIANA UNIVERSITY   (United States)

^d UNIVERSITY OF CALIFORNIA   (United States)

^e UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^f ELI LILLY AND COMPANY   (United States)

Author keywords

Crystal structure; Fumarylacetoacetate; Hepatocyte transplantation; Pharmacokineties; Transition state analogue; Tyrosinaemia

Indexed keywords

FUMARYLACETOACETATE; HEPATOCYTE TRANSPLANTATION; TRANSITION STATE ANALOGUE; TYROSINAEMIA;

CATALYSIS; CRYSTAL STRUCTURE; CRYSTALLIZATION; METABOLITES; PHARMACOKINETICS; PROTEINS;

ENZYME KINETICS;

3 [(3 CARBOXY 3 OXOPROPYL)HYDROXYPHOSPHINYL]ACRYLATE; 4 [(2 CARBOXY ETHYL) HYDROXYPHOSPHINYL] 3 OXOBUTYRATE; ACETOACETIC ACID; FUMARIC ACID; FUMARYLACETOACETASE; FUMARYLACETOACETASE INHIBITOR; HYDROXYBUTYRIC ACID; UNCLASSIFIED DRUG; BIOMIMETIC MATERIAL; HYDROLASE; PHOSPHINIC ACID DERIVATIVE; PROTEINASE INHIBITOR;

ARTICLE; CONTROLLED STUDY; CRYSTAL STRUCTURE; DRUG HALF LIFE; IN VIVO CULTURE; KINETICS; MOUSE; NONHUMAN; PRIORITY JOURNAL; TYROSINEMIA; ANIMAL; BINDING SITE; BLOOD; C57BL MOUSE; CHEMICAL STRUCTURE; CHEMISTRY; DRUG ANTAGONISM; HUMAN; MALE; METABOLISM; PROTEIN TERTIARY STRUCTURE; SYNTHESIS; TIME; X RAY CRYSTALLOGRAPHY;

MUS;

ANIMALS; BINDING SITES; BIOMIMETIC MATERIALS; CRYSTALLOGRAPHY, X-RAY; HUMANS; HYDROLASES; KINETICS; MALE; MICE; MICE, INBRED C57BL; MODELS, MOLECULAR; MOLECULAR STRUCTURE; PHOSPHINIC ACIDS; PROTEASE INHIBITORS; PROTEIN STRUCTURE, TERTIARY; TIME FACTORS;

EID: 33847726934     PISSN: 02646021     EISSN: None     Source Type: Journal    
DOI: 10.1042/BJ20060961     Document Type: Article

References (34)

1. Kvittingen, E. A. and Bradtkorb, E. (1986) The pre- and post-natal diagnosis of tyrosinemia type I and the detection of the carrier state by assay of fumarylacetoacetase. Scand. J. Clin. Lab. Invest. Suppl. 184, 35-40
2. Mitchell, G., Larochelle, J., Lambert, M., Michaud, J., Grenier, A., Ogier, H., Gauthier, M., Lacroix, J., Vanasse, M., Larbrisseau, A. et al. (1990) Neurologic crises in hereditary tyrosinemia. N. Engl. J. Med. 322, 432-437
3. Grompe, M., al-Dhalimy, M., Finegold, M., Ou, C. N., Burlingame, T., Kennaway, N. G. and Soriano, P. (1993) Loss of fumarylacetoacetate hydrolase is responsible for the neonatal hepatic dysfunction phenotype of lethal albino mice. Genes Dev. 7, 2298-2307
4. Grompe, M., Lindstedt, S., al-Dhalimy, M., Kennaway, N. G., Papaconstantinou, J., Torres-Ramos, C. A., Ou, C. N. and Finegold, M. (1995) Pharmacological correction of neonatal lethal hepatic dysfunction in a murine model of hereditary tyrosinaemia type I. Nat. Genet. 10, 453-460
5. Montini, E., Held, P. K., Noll, M., Morcinek, N., Al-Dhalimy, M., Finegold, M., Yant, S. R., Kay, M. A. and Grompe, M. (2002) In vivo correction of murine tyrosinemia type I by DNA-mediated transposition. Mol. Then 6, 759-769
6. Overturf, K., al-Dhalimy, M., Ou, C. N., Finegold, M., Tanguay, R., Lieber, A., Kay, M. and Grompe, M. (1997) Adenovirus-mediated gene therapy in a mouse model of hereditary tyrosinemia type I. Hum. Gene Ther. 8, 513-521
7. Overturf, K., Al-Dhalimy, M., Tanguay, R., Brantly, M., Ou, C. N., Finegold, M. and Grompe, M. (1996) Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type I. Nat. Genet. 12, 266-273
8. Bateman, R. L., Bhanumoorthy, P., Witte, J. F., McClard, R. W., Grompe, M. and Timm, D. E. (2001) Mechanistic inferences from the crystal structure of fumarylacetoacetate hydrolase with a bound phosphorus-based inhibitor. J. Biol. Chem. 276, 15284-15291
9. Fernandez-Canon, J. M., Baetscher, M. W., Finegold, M., Burlingame, I, Gibson, K. M. and Grompe, M. (2002) Maleylacetoacetate isomerase (MAAI/GSTZ)-deficient mice reveal a glutathione-dependent nonenzymatic bypass in ryrosine catabolism. Mol. Cell. Biol. 22, 4943-4951
10. Knox, W. E. and Edwards, S. W. (1955) Homogentisate oxidase of liver. J. Biol. Chem. 216, 479-487
11. Mahuran, D. J., Angus, R. H., Braun, C. V., Sim, S. S. and Schmidt, Jr, D. E. (1977) Characterization and substrate specificity of fumarylacetoacetate fumarylhydrolase. Can. J. Biochem. 55, 1-8
12. Timm, D. E., Mueller, H. A., Bhanumoorthy, P., Harp, J. M. and Bunick, G. J. (1999) Crystal structure and mechanism of a carbon-carbon bond hydrolase. Structure 7, 1023-1033
13. Hsiang, H. H., Sim, S. S., Mahuran, D. J. and Schmidt, Jr, D. E. (1972) Purification and properties of a diketo acid hydrolase from beef liver. Biochemistry 11, 2098-2102
14. Ames, B. N. (1966) Assay of inorganic phosphate, total phosphate and phosphatases. Methods Enzymol. 8, 115-118
15. Morrison, J. F. and Walsh, C. T. (1988) The behavior and significance of slow-binding enzyme inhibitors. Adv. Enzymol. Relat. Areas Mol. Biol. 61, 201-301
16. Kuzmic, P. (1996) Program DYNAFIT for the analysis of enzyme kinetic data: application to HIV proteinase. Anal. Biochem. 237, 260-273
17. Jones, T. A., Zou, J. Y., Cowan, S. W. and Kjeldgaard, M. (1991) Improved methods for building protein models in electron density maps and the location of errors in these models. Acta Crystallogr. Sect. A Found. Crystallogr. 47, 110-119
18. Brunger, A. T., Adams, P. D., Clore, G. M., DeLano, W. L., Gros, P., Grosse-Kunstleve, R. W., Jiang, J. S., Kuszewski, J., Nilges, M., Pannu, N. S. et al. (1998) Crystallography & NMR system: a new software suite for macromolecular structure determination. Acta Crystallogr. Sect. D Biol. Crystallogr. 54, 905-921
19. Murshudov, G. M., Vagin, A. A. and Dodson, E. J. (1997) Refinement of macromolecular structures by the maximum-likelihood method. Acta Crystallogr. Sect. D Biol. Crystallogr. 53, 240-255
20. Labelle, Y., Puymirat, J. and Tanguay, R. M. (1993) Localization of cells in the rat brain expressing fumarylacetoacetate hydrolase, the deficient enzyme in hereditary tyrosinemia type 1. Biochim. Biophys. Acta 1180, 250-256
21. Grenier, A. and Lescault, A. (1985) Succinylacetone. In Methods of Enzymatic. Analysis (Bergmeyer, H. V., ed.), 3rd edn, volume 8, pp. 73-79, Verlag Chemie, Weinheim
22. Bartlett, P. A. and Marlowe, C. K. (1983) Phosphonamidates as transition-state analogue inhibitors of thermolysin. Biochemistry 22, 4618-4624
23. Fraser, M. E., Strynadka, N. C., Bartlett, P. A., Hanson, J. E. and James, M. N. (1992) Crystallographic analysis of transition-state mimics bound to penicillopepsin: phosphorus-containing peptide analogues. Biochemistry 31, 5201-5214
24. Hanson, J. E., Kaplan, A. P. and Bartlett, P. A. (1989) Phosphorate analogues of carboxypeptidase A substrates are potent transition-state analogue inhibitors. Biochemistry 28, 6294-6305
25. Phillips, M. A., Kaplan, A. P., Rutter, W. J. and Bartlett, P. A. (1992) Transition-state characterization: a new approach combining inhibitor analogues and variation in enzyme structure. Biochemistry 31, 959-963
26. Melppelder, F. H. and Beck, H. C. (1975) The nuclear magnetic resonance spectra of diethyl Z- and E-2-(methoxycarbonyl)vinylphosphonate. Rec. Trav. Chim. 94, 149-150
27. Castagnetto, J. M., Hennessy, S. W., Roberts, V. A., Getzoff, E. D., Tainer, J. A. and Pique, M. E. (2002) MDB: the Metalloprotein Database and Browser at The Scripps Research Institute. Nucleic Acids Res. 30, 379-382
28. + binding sites. J. Mol. Biol. 256, 228-234
29. Kvittingen, E. A., Rootwelt, H., Berger, R. and Brandtzaeg, P. (1994) Self-induced correction of the genetic defect in tyrosinemia type I. J. Clin. Invest. 94, 1657-1661
30. Lagasse, E., Connors, H., Al-Dhalimy, M., Reitsma, M., Dohse, M., Osborne, L., Wang, X., Finegold, M., Weissman, I. L. and Grompe, M. (2000) Purified hematopoietic stem cells can differentiate into hepatocytes in vivo. Nat. Med. 6, 1229-1234
31. Manning, K., Al-Dhalimy, M., Finegold, M. and Grompe, M. (1999) In vivo suppressor mutations correct a murine model of hereditary tyrosinemia type I. Proc. Natl. Acad, Sci. U.S.A. 96, 11928-11933
32. Endo, F., Kubo, S., Awata, H., Kiwaki, K., Katoh, H., Kanegae, Y., Saito, I., Miyazaki, J., Yamamoto, T., Jakobs, C. et al. (1997) Complete rescue of lethal albino c14CoS mice by null mutation of 4-hydroxyphenylpyruvate dioxygenase and induction of apoptosis of hepatocytes in these mice by in vivo retrieval of the tyrosine catabolic pathway. J. Biol. Chem. 272, 24426-24432
33. Davis, B. M., Humeau, L. and Dropulic, B. (2004) In vivo selection for human and murine hematopoietic cells transduced with a therapeutic MGMT lentiviral vector that inhibits HIV replication. Mol. Ther. 9, 160-172
34. Arbuthnot, P., Carmona, S. and Ely, A, (2005) Exploiting the RNA interference pathway to counter hepatitis B virus replication. Liver Int. 25, 9-15