Genistein reduces lysosomal storage in peripheral tissues of mucopolysaccharide IIIB mice

Molecular Genetics and Metabolism

Volumn 98, Issue 3, 2009, Pages 235-242

  Malinowska, Marcelina ^a,b   Wilkinson, Fiona L ^b   Bennett, William ^b   Langford Smith, Kia J ^b   O'Leary, H Angharad ^b   Jakobkiewicz Banecka, Joanna ^a   Wynn, Rob ^c   Wraith, J Ed ^c   Wegrzyn, Grzegorz ^a   Bigger, Brian W ^b  

^a UNIVERSITY OF GDA SK   (Poland)

^b UNIVERSITY OF MANCHESTER   (United Kingdom)

^c ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

Author keywords

Genistein; Glycosaminoglycan; Lysosomal storage; Mucopolysaccharidosis; Naglu; Sanfilippo syndrome IIIB; Substrate reduction therapy

Indexed keywords

ALPHA N ACETYLGALACTOSAMINIDASE; GENISTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ASTROCYTOSIS; BLOOD BRAIN BARRIER; BRAIN CORTEX; CONTROLLED STUDY; DOSE RESPONSE; DRUG DOSE COMPARISON; DRUG DOSE INCREASE; DRUG EFFECT; DRUG EFFICACY; ENZYME REPLACEMENT; FEMALE; GENE DISRUPTION; GENE TARGETING; IN VIVO STUDY; LIVER PRESERVATION; LYSOSOME; MALE; MAXIMUM TOLERATED DOSE; MORPHOLOGY; MOUSE; NONHUMAN; PRIORITY JOURNAL; SANFILIPPO SYNDROME; SEX RATIO; TREATMENT DURATION; TREATMENT OUTCOME; TREATMENT RESPONSE; WILD TYPE;

ACETYLGLUCOSAMINIDASE; ANIMALS; BLOOD-BRAIN BARRIER; DISEASE MODELS, ANIMAL; FEMALE; GENISTEIN; LIVER; LYSOSOMES; MALE; MICE; MICE, KNOCKOUT; MUCOPOLYSACCHARIDOSIS III;

ANIMALIA; MUS;

EID: 70349820325     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2009.06.013     Document Type: Article

References (29)

1. M.J. Valstar, G.J. Ruijter, O.P. van Diggelen, B.J. Poorthuis, F.A. Wijburg, Sanfilippo syndrome: A mini-review, J. Inherit. Metab. Dis., 2008.
2. Wynn R.F., Wraith J.E., Mercer J., O'Meara A., Tylee K., Thornley M., Church H.J., and Bigger B.W. Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy. J. Pediatr. 154 (2009) 609-611
3. Wraith J.E., Scarpa M., Beck M., Bodamer O.A., De Meirleir L., Guffon N., Meldgaard Lund A., Malm G., Van der Ploeg A.T., and Zeman J. Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur. J. Pediatr. 167 (2008) 267-277
4. Harmatz P., Giugliani R., Schwartz I.V., Guffon N., Teles E.L., Miranda M.C., Wraith J.E., Beck M., Arash L., Scarpa M., Ketteridge D., Hopwood J.J., Plecko B., Steiner R., Whitley C.B., Kaplan P., Yu Z.F., Swiedler S.J., and Decker C. Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase. Mol. Genet. Metab. 94 (2008) 469-475
5. Vellodi A., Young E., New M., Pot-Mees C., and Hugh-Jones K. Bone marrow transplantation for Sanfilippo disease type B. J. Inherit. Metab. Dis. 15 (1992) 911-918
6. Jeyakumar M., Dwek R.A., Butters T.D., and Platt F.M. Storage solutions: treating lysosomal disorders of the brain. Nat. Rev. Neurosci. 6 (2005) 713-725
7. Keeling K.M., Brooks D.A., Hopwood J.J., Li P., Thompson J.N., and Bedwell D.M. Gentamicin-mediated suppression of Hurler syndrome stop mutations restores a low level of alpha-l-iduronidase activity and reduces lysosomal glycosaminoglycan accumulation. Hum. Mol. Genet. 10 (2001) 291-299
8. Roberts A.L., Thomas B.J., Wilkinson A.S., Fletcher J.M., and Byers S. Inhibition of glycosaminoglycan synthesis using rhodamine B in a mouse model of mucopolysaccharidosis type IIIA. Pediatr. Res. 60 (2006) 309-314
9. Roberts A.L., Rees M.H., Klebe S., Fletcher J.M., and Byers S. Improvement in behaviour after substrate deprivation therapy with rhodamine B in a mouse model of MPS IIIA. Mol. Genet. Metab. 92 (2007) 115-121
10. Berkin A., Szarek W.A., and Kisilevsky R. Synthesis of 4-deoxy-4-fluoro analogues of 2-acetamido-2-deoxy-d-glucose and 2-acetamido-2-deoxy-d-galactose and their effects on cellular glycosaminoglycan biosynthesis. Carbohydr. Res. 326 (2000) 250-263
11. Piotrowska E., Jakobkiewicz-Banecka J., Baranska S., Tylki-Szymanska A., Czartoryska B., Wegrzyn A., and Wegrzyn G. Genistein-mediated inhibition of glycosaminoglycan synthesis as a basis for gene expression-targeted isoflavone therapy for mucopolysaccharidoses. Eur. J. Hum. Genet. 14 (2006) 846-852
12. Akiyama T., Ishida J., Nakagawa S., Ogawara H., Watanabe S., Itoh N., Shibuya M., and Fukami Y. Genistein, a specific inhibitor of tyrosine-specific protein kinases. J. Biol. Chem. 262 (1987) 5592-5595
13. Jakobkiewicz-Banecka J., Piotrowska E., Narajczyk M., Baranska S., and Wegrzyn G. Genistein-mediated inhibition of glycosaminoglycan synthesis, which corrects storage in cells of patients suffering from mucopolysaccharidoses, acts by influencing an epidermal growth factor-dependent pathway. J. Biomed. Sci. 16 (2009) 26
14. Tsai T.H., and blood C.m.o.u.g.i.t. brain and bile of anesthetized rats using microdialysis and its pharmacokinetic application. J. Chromatogr. A 1073 (2005) 317-322
15. Li H.H., Yu W.H., Rozengurt N., Zhao H.Z., Lyons K.M., Anagnostaras S., Fanselow M.S., Suzuki K., Vanier M.T., and Neufeld E.F. Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding alpha-N-acetylglucosaminidase. Proc. Natl. Acad. Sci. USA 96 (1999) 14505-14510
16. Malinowska M., Jakobkiewicz-Banecka J., Kloska A., Tylki-Szymanska A., Czartoryska B., Piotrowska E., Wegrzyn A., and Wegrzyn G. Abnormalities in the hair morphology of patients with some but not all types of mucopolysaccharidoses. Eur. J. Pediatr. 167 (2008) 203-209
17. Michael McClain R., Wolz E., Davidovich A., and Bausch J. Genetic toxicity studies with genistein. Food Chem. Toxicol. 44 (2006) 42-55
18. Michael McClain R., Wolz E., Davidovich A., Pfannkuch F., Edwards J.A., and Bausch J. Acute, subchronic and chronic safety studies with genistein in rats. Food Chem. Toxicol. 44 (2006) 56-80
19. McClain R.M., Wolz E., Davidovich A., Pfannkuch F., and Bausch J. Subchronic and chronic safety studies with genistein in dogs. Food Chem. Toxicol. 43 (2005) 1461-1482
20. Piotrowska E., Jakobkiewicz-Banecka J., Tylki-Szymanska A., Liberek A., Maryniak A., Malinowska M., Czartoryska B., Puk E., Kloska A., Liberek T., Baranska S., Wegrzyn A., and Wegrzyn G. Genistin-rich soy isoflavone extract in substrate reduction therapy for Sanfilippo syndrome: an open-label pilot study in 10 pediatric patients. Curr. Ther. Res. 69 (2008) 166-179
21. Kloska A., Bohdanowicz J., Konopa G., Tylki-Szymnska A., Jakobkiewicz-Banecka J., Czartoryska B., Liberek A., Wegrzyn A., and Wegrzyn G. Changes in hair morphology of mucopolysaccharidosis I patients treated with recombinant human alpha-l-iduronidase (laronidase, Aldurazyme). Am. J. Med. Genet. A 139 (2005) 199-203
22. Di Natale P., Di Domenico C., Gargiulo N., Castaldo S., Gonzalez Y.R.E., Mithbaokar P., De Felice M., Follenzi A., Naldini L., and Villani G.R. Treatment of the mouse model of mucopolysaccharidosis type IIIB with lentiviral-NAGLU vector. Biochem. J. 388 (2005) 639-646
23. Jansson J.O., Ekberg S., Hoath S.B., Beamer W.G., and Frohman L.A. Growth hormone enhances hepatic epidermal growth factor receptor concentration in mice. J. Clin. Invest. 82 (1988) 1871-1876
24. Pisano M.M., and Greene R.M. Epidermal growth factor potentiates the induction of ornithine decarboxylase activity by prostaglandins in embryonic palate mesenchymal cells: effects on cell proliferation and glycosaminoglycan synthesis. Dev. Biol. 122 (1987) 419-431
25. Tirone E., D'Alessandris C., Hascall V.C., Siracusa G., and Salustri A. Hyaluronan synthesis by mouse cumulus cells is regulated by interactions between follicle-stimulating hormone (or epidermal growth factor) and a soluble oocyte factor (or transforming growth factor beta1). J. Biol. Chem. 272 (1997) 4787-4794
26. Zheng Y., Ryazantsev S., Ohmi K., Zhao H.Z., Rozengurt N., Kohn D.B., and Neufeld E.F. Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB. Mol. Genet. Metab. 82 (2004) 286-295
27. Cressant A., Desmaris N., Verot L., Brejot T., Froissart R., Vanier M.T., Maire I., and Heard J.M. Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum. J. Neurosci. 24 (2004) 10229-10239
28. Bigger B.W., Siapati E.K., Mistry A., Waddington S.N., Nivsarkar M.S., Jacobs L., Perrett R., Holder M.V., Ridler C., Kemball-Cook G., Ali R.R., Forbes S.J., Coutelle C., Wright N., Alison M., Thrasher A.J., Bonnet D., and Themis M. Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX. Gene Ther. 13 (2006) 117-126
29. McGlynn R., Dobrenis K., and Walkley S.U. Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders. J. Comp. Neurol. 480 (2004) 415-426