High-throughput screening for human galactokinase inhibitors

Journal of Biomolecular Screening

Volumn 13, Issue 5, 2008, Pages 415-423

  Wierenga, Klaas J ^a   Lai, Kent ^a   Buchwald, Peter ^a   Tang, Manshu ^a  

^a UNIVERSITY OF MIAMI MILLER SCHOOL OF MEDICINE   (United States)

Author keywords

Classic galactosemia; Galactokinase (GALK); Galactose 1 phosphate; Galactose 1 phosphate uridyltransferase (GALT); GHMP small molecule kinase; High throughput screening; Small molecule inhibitors

Indexed keywords

ENZYME INHIBITOR; GALACTOKINASE; GALACTOKINASE INHIBITOR; GALACTOSE 1 PHOSPHATE URIDYLYLTRANSFERASE;

ARTICLE; DRUG SCREENING; ENZYME ACTIVITY; ENZYME INHIBITION; GALACTOSEMIA; HIGH THROUGHPUT SCREENING; HUMAN; IC 50; PRIORITY JOURNAL;

DRUG EVALUATION, PRECLINICAL; ENZYME INHIBITORS; GALACTOKINASE; HUMANS;

BACTERIA (MICROORGANISMS);

EID: 44849109699     PISSN: 10870571     EISSN: 1552454X     Source Type: Journal    
DOI: 10.1177/1087057108318331     Document Type: Article

References (34)

1. Isselbacher KJ, Anderson EP, Kurahashi K., Kalckar HM: Congenital galactosemia, a single enzymatic block in galactose metabolism. Science 1956 ; 123: 635-636.
2. Leloir LF: The enzymatic transformation of uridine diphosphate glucose into a galactose derivative. Arch Biochem 1951 ; 33: 186-190.
3. Gitzelmann R.: Estimation of galactose-I-phosphate in erythrocytes: a rapid and simple enzymatic method. Clin Chim Acta 1969 ; 26: 313-316.
4. Gitzelmann R., Curtius HC, Schneller I.: Galactitol and galactose-1-phosphate in the lens of a galactosemic infant. Exp Eye Res 1967 ; 6: 1-3.
5. Lai K., Langley SD, Khwaja FW, Schmitt EW, Elsas LJ: GALT deficiency causes UDP-hexose deficit in human galactosemic cells. Glycobiology 2003 ; 13: 285-294.
6. Segal S., Berry GT: Disorders of galactose metabolism. In Scriver D, Sly W, Valle D (eds): The Metabolic Basis of Inherited Diseases. New York: McGraw-Hill, 1995: 967-1000.
7. Kaye CI, Committee on Genetics, Accurso F., La Franchi S., Lane PA, Hope N.,, et al. Newborn screening fact sheets. Pediatrics 2006 ; 118: e934 - e963.
8. Gitzelmann R., Steinmann B.: Galactosemia: how does long-term treatment change the outcome ? Enzyme 1984 ; 32: 37-46.
9. Waggoner D., Buist NRM, Donnell GN: Long-term prognosis in galactosemia: results of a survey of 350 cases. J Inherited Metab Disord 1990 ; 13: 802-818.
10. Waggoner D., Buist NRM: Long-term complications in treated galactosemia: 175 U.S. cases. Int Pediatr 1993 ; 8: 97-100.
11. Gitzelmann R.: Galactose-1-phosphate in the pathophysiology of galactosemia. Eur J Pediatr 1995 ; 154 (suppl 2). S45 - S49.
12. Holden HM, Thoden JB, Timson DJ, Reece RJ: Galactokinase: structure, function and role in type II galactosemia. Cell Mol Life Sci 2004 ; 61: 2471-2484.
13. Zhou T., Daugherty M., Grishin NV, Osterman AL, Zhang H.: Structure and mechanism of homoserine kinase: prototype for the GHMP kinase super-family. Structure 2000 ; 8: 1247-1257.
14. Gitzelmann R.: Letter: additional findings in galactokinase deficiency. J Pediatr 1975 ; 87 (pt 1). 1007-1008.
15. Gitzelmann R., Wells HJ, Segal S.: Galactose metabolism in a patient with hereditary galactokinase deficiency. Eur J Clin Invest 1974 ; 4: 79-84.
16. Bosch AM, Bakker HD, van Gennip AH, van Kempen JV, Wanders RJ, Wijburg FA: Clinical features of galactokinase deficiency: a review of the literature. J Inherit Metab Disord 2002 ; 25: 629-634.
17. Douglas HC, Hawthorne DC: Enzymatic expression and genetic linkage of genes controlling galactose utilization in saccharomyces. Genetics 1964 ; 49: 837-844.
18. Douglas HC, Hawthorne DC: Regulation of genes controlling synthesis of the galactose pathway enzymes in yeast. Genetics 1966 ; 54: 911-916.
19. Slepak T., Tang M., Addo F., Lai K.: Intracellular galactose-1-phosphate accumulation leads to environmental stress response in yeast model. Mol Genet Metab 2005 ; 86: 360-371.
20. Acosta PB, Gross KC: Hidden sources of galactose in the environment. Eur J Pediatr 1995 ; 154 (suppl 2). S87 - S92.
21. Berry GT, Palmieri M., Gross KC, Acosta PB, Henstenburg JA, Mazur,, et al. The effect of dietary fruits and vegetables on urinary galactitol excretion in galactose-1-phosphate uridyltransferase deficiency. J Inherit Metab Disord 1993 ; 16: 91-100.
22. Bosch AM, Bakker HD, Wenniger-Prick LJ, Wanders RJ, Wijburg FA: High tolerance for oral galactose in classical galactosaemia: dietary implications. Arch Dis Child 2004 ; 89: 1034-1036.
23. Berry GT, Moate PJ, Reynolds RA, Yager CT, Ning C., Boston RC,, et al. The rate of de novo galactose synthesis in patients with galactose-1-phosphate uridyltransferase deficiency. Mol Genet Metab 2004 ; 81: 22-30.
24. Berry GT, Nissim I., Lin Z., Mazur AT, Gibson JB, Segal S.: Endogenous synthesis of galactose in normal men and patients with hereditary galactosaemia. Lancet 1995 ; 346: 1073-1074.
25. Heinrich MR, Howard SM: Galactokinase. Methods Enzymol 1966 ; 9: 407-412.
26. Zhang JH, Chung TD, Oldenburg KR: A simple statistical parameter for use in evaluation and validation of high throughput screening assays. J Biomol Screen 1999 ; 4: 67-73.
27. Seiler KP, George GA, Happ MP, Bodycombe NE, Carrinski HA, Norton S.,, et al. ChemBank: a small-molecule screening and cheminformatics resource database. Nucleic Acids Res 2007.
28. CHEMBANK Web site [Online]. Retrieved from http://chembank.broad.harvard. edu/details.htm?tag=Help.
29. Wu G., Yuan Y., Hodge CN: Determining appropriate substrate conversion for enzymatic assays in high-throughput screening. J Biomol Screen 2003 ; 8: 694-700.
30. Thoden JB, Timson DJ, Reece RJ, Holden HM: Molecular structure of human galactokinase: implications for type II galactosemia. J Biol Chem 2005 ; 280: 9662-9670.
31. Timson DJ, Reece RJ: Functional analysis of disease-causing mutations in human galactokinase. Eur J Biochem 2003 ; 270: 1767-1774.
32. Shoichet BK: Virtual screening of chemical libraries. Nature 2004 ; 432: 862-865.
33. Barberis A., Gunde T., Berset C., Audetat S., Luthi U.: Yeast as a screening tool. Drug Discov Today 2005 ; 2: 187-192.
34. Perkins E., Sun D., Nguyen A., Tulac S., Francesco M., Tavana H.,, et al. Novel inhibitors of poly(ADP-ribose) polymerase/PARP1 and PARP2 identified using a cell-based screen in yeast. Cancer Res 2001 ; 61: 4175-4183.