Gene expression-targeted isoflavone therapy

IUBMB Life

Volumn 64, Issue 4, 2012, Pages 307-315

  Wȩgrzyn, Alicja ^a  

^a UNIVERSITY OF GDA SK   (Poland)

Author keywords

genistein; lysosomal storage diseases; mucopolysaccharidosis; neurodegenerative disorders; substrate reduction therapy

Indexed keywords

GENISTEIN; GLYCOSAMINOGLYCAN; ISOFLAVONE;

DRUG EFFICACY; DRUG MECHANISM; GENE EXPRESSION; HUMAN; IN VITRO STUDY; LYSOSOME STORAGE DISEASE; MUCOPOLYSACCHARIDOSIS; NONHUMAN; REVIEW;

ANIMALS; CELLS, CULTURED; CLINICAL TRIALS AS TOPIC; DISEASE MODELS, ANIMAL; GENE EXPRESSION; GENE TARGETING; GENISTEIN; GLYCOSAMINOGLYCANS; HUMANS; ISOFLAVONES; LYSOSOMAL STORAGE DISEASES; MICE; MUCOPOLYSACCHARIDOSES; TRANSLATIONAL MEDICAL RESEARCH;

ANIMALIA; MUS;

EID: 84859083422     PISSN: 15216543     EISSN: 15216551     Source Type: Journal    
DOI: 10.1002/iub.1007     Document Type: Review

References (50)

1. Swarts, K., (2009) Genetic Disorders. Greenhaven Press, Farmington Hills, MI.
2. Barranger, J. A., and, Cabrera-Salazar, M., (2007) Lysosomal Storage Disorders. Springer, Berlin-Heidelberg.
3. Neufeld, E. F., and, Muenzer, J., (2001) Mucopolysaccharidoses. In: The Metabolic and Molecular Bases of Inherited Disease (, Scriver, C. R., Beaudet, A. L., Sly. W. S., and, Valle, D., eds.). pp. 3421-3452, McGraw-Hill Co., New York.
4. WȨgrzyn, G., Jakõbkiewicz-Banecka, J., Narajczyk, M., WiÅniewski, A., Piotrowska, E., et al.(2010) Why are behaviors of children suffering from various neuronopathic types of mucopolysaccharidoses different? Med. Hypoth. 75, 605-609.
5. Wraith, J. E., (2009) Enzyme replacement therapy for the management of the mucopolysaccharidoses. Int. J. Clin. Pharmacol. Ther. 47 (Suppl 1), S63-S65.
6. Beck, M., (2010) Therapy for lysosomal storage disorders. IUBMB Life 62, 33-40.
7. Cotrim, A. P., and, Baum, B. J., (2008) Gene therapy: some history, applications, problems, and prospects. Toxicol. Pathol. 36, 97-103.
8. Schultz, M. L., Tecedor, L., Chang, M., and, Davidson, B. L., (2011) Clarifying lysosomal storage diseases. Trends Neurosci. 34, 401-410.
9. Jakõbkiewicz-Banecka, J., Piotrowska, E., Gabig-Cimińska, M., Borysiewicz, E., Słomińska-Wojewõdzka, M., et al.(2011) Substrate reduction therapies for mucopolysaccharidoses. Curr. Pharm. Biotechnol. 12, 1860-1865.
10. Roberts, A. L., Thomas, B. J., Wilkinson, A. S., Fletcher, J. M., and, Byers, S., (2006) Inhibition of glycosaminoglycan synthesis using a rhodamine B in a mouse model of mucopolysaccharidosis type IIIA. Pediatr. Res. 60, 309-314. (Pubitemid 44267744)
11. Roberts, A. L., Rees, M. H., Klebe, S., Fletcher, J. M., and, Byers, S., (2007) Improvement in behaviour after substrate deprivation therapy with rhodamine B in a mouse model of MPS IIIA. Mol. Genet. Metab. 92, 115-121. (Pubitemid 47361897)
12. Roberts, A. L., Fletcher, J. M., Moore, L., and, Byers, S., (2010) Trans-generational exposure to low levels of rhodamine B does not adversely affect litter size or liver function in murine mucopolysaccharidosis type IIIA. Mol. Genet. Metab. 101, 208-213.
13. Dziedzic, D., WȨgrzyn, G., and, Jakõbkiewicz-Banecka, J., (2010) Impairment of glycosaminoglycan synthesis in mucopolysaccharidosis type IIIA cells by using siRNA: a potential therapeutic approach for Sanfilippo disease. Eur. J. Hum. Genet. 18, 200-205.
14. Kaidonis, X., Liaw, W. C., Roberts, A. D., Ly, M., Anson, D., et al.(2010) Gene silencing of EXTL2 and EXTL3 as a substrate deprivation therapy for heparan sulfate storing mucopolysaccharidoses. Eur. J. Hum. Genet. 18, 194-199.
15. Smith, R. A., Miller, T. M., Yamanaka, K., Monia, B. P., Condon, T. P., et al.(2006) Antisense oligonucleotide therapy for neurodegenerative disease. J. Clin. Invest. 116, 2290-2296. (Pubitemid 44162339)
16. Juliano, R., Alam, M. R., Dixit, V., and, Kang H,.(2008) Mechanism and strategies for effective delivery of antisense and siRNA oligonucleotides. Nucleic Acids Res. 36, 4158-4171. (Pubitemid 351984831)
17. WȨgrzyn, G., WȨgrzyn, A., and, Tylki-Szymańska, A., (2004) A general model for genetic regulation of turnover of glycosaminoglycans suggests a possible procedure for prediction of severity and clinical progress of mucopolysaccharidoses. Med. Hypoth. 62, 986-992. (Pubitemid 38680373)
18. Piotrowska, E., Jakõbkiewicz-Banecka, J., Tylki-Szymańska, A., Czartoryska, B., WȨgrzyn, A., et al.(2009) Correlation between severity of mucopolysaccharidoses and combination of the residual enzyme activity and efficiency of glycosaminoglycan synthesis. Acta Paediatr. 98, 743-749.
19. Pisano, M. M., and, Greene, R. M., (1987) 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, 419-431. (Pubitemid 17157980)
20. Akiyama, T., Ishida, J., Nakagawa, S., Ogawara, H., Watanabe, S., et al.(1987) Genistein, a specific inhibitor of tyrosine-specific protein kinase. J. Biol. Chem. 262, 5592-5595.
21. Tsai, T. H., (2005) Concurrent measurement of unbound genistein in the blood, brain and bile of anesthetized rats using microdialysis and its pharmacokinetic application. J. Chromatogr. A 1073, 317-322.
22. Piotrowska, E., Jakõbkiewicz-Banecka, J., Barańska, S., Tylki-Szymańska, A., Czartoryska, B., et al.(2006) Genistein-mediated inhibition of glycosaminoglycan synthesis as a basis for gene expression-targeted isoflavone therapy for mucopolysaccharidoses. Eur. J. Hum. Genet. 14, 846-852. (Pubitemid 43923370)
23. Jakõbkiewicz-Banecka, J., Piotrowska, E., Narajczyk, M., Barańska, S., and, WȨgrzyn, G., (2009) 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, 26.
24. Arfi, A., Richard, M., Gandolphe, C., and, Scherman, D., (2010) Storage correction in cells of patients suffering from mucopolysaccharidoses types IIIA and VII after treatment with genistein and other isoflavones. J. Inherit. Metab. Dis. 33, 61-67.
25. Kloska, A., Jakõbkiewicz-Banecka, J., Narajczyk, M., Banecka-Majkutewicz, Z., and, WȨgrzyn, G., (2011) Effects of flavonoids on glycosaminoglycan synthesis: implications for substrate reduction therapy in Sanfilippo disease and other mucopolysaccharidoses. Metab. Brain Dis. 26, 1-8.
26. Friso, A., Tomanin, R., Salvalaio, M., and, Scarpa, M., (2010) Genistein reduces glycosaminoglycan levels in a mouse model of mucopolysaccharidosis type II. Br. J. Pharmacol. 159, 1082-1091.
27. Malinowska M., Wilkinson, F. L., Bennett, W., Langford-Smith, K. J., O'Leary, H. A., et al.(2009) Genistein reduces lysosomal storage in peripheral tissues of mucopolysaccharide IIIB mice. Mol. Genet. Metab. 98, 235-242.
28. Malinowska, M., Wilkinson, F. L., Langford-Smith, K. J., Langford-Smith, A., Brown, J. R., et al.(2010) Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic disease. PLoS One 5, e14192.
29. Jakõbkiewicz-Banecka. J., WȨgrzyn, A., and, WȨgrzyn, G., (2007) Substrate deprivation therapy: a new hope for patients suffering from neuronopathic forms of inherited lysosomal storage diseases. J. Appl. Genet. 48, 383-388. (Pubitemid 350219938)
30. Piotrowska, E., Jakõbkiewicz-Banecka, J., Tylki-Szymańska, A., Liberek, A., Maryniak, A., et al.(2008) The use of genistein-rich isoflavone extract in substrate reduction therapy for Sanfilippo disease: open-label, pilot study in 10 pediatric patients. Curr. Ther. Res. Clin. Exp. 69, 166-179. (Pubitemid 351834736)
31. Kloska, A., Bohdanowicz, J., Konopa, G., Tylki-Szymańska, A., Jakõbkiewicz-Banecka, J., et al.(2005) Changes in hair morphologyof mucopolysaccharidosis I patients treated with recombinant human α- L -iduronidase (laronidase, Aldurazyme). Am. J. Med. Genet. A 139, 199-203. (Pubitemid 41740590)
32. Malinowska, M., Jakõbkiewicz-Banecka, J., Kloska, A., Tylki-Szymańska, A., and, Czartoryska, B., (2007) Abnormalities in the hair morphology of patients of same but not all types of mucopolysaccharidoses. Eur. J. Pediatr. 167, 203-209.
33. Piotrowska, E., Jakobkiewicz-Banecka, J., Maryniak, A., Tylki-Szymańska, A., Puk, E., et al.(2011) Two-year follow-up of Sanfilippo disease patients treated with a genistein-rich isoflavone extract: assessment of effects on cognitive functions and general status of patients. Med. Sci. Monit. 17, CR196-CR202.
34. Delgadillo, V., del Mar O'Callaghan, M., Artuch, R., Montero, R., and, Pineda, M., (2011) Genistein supplementation in patients affected by Sanfilippo disease. J. Inherit. Metab. Dis. 34, 1039-1044.
35. Malinova, V., WȨgrzyn, G., and, Narajczyk, M., (2012) The use of elevated doses of genistein-rich soy extract in the gene expression-targeted isoflavone therapy (GET IT) for Sanfilippo disease patients. JIMD Rep., in press (DOI: 10.1007/8904-2011-87).
36. de Ruijter, J., Valstar, M. J., Narajczyk, M., WȨgrzyn, G., Kulik, W., et al.(2012) Genistein in Sanfilippo disease: a randomized controlled cross-over trial. Ann. Neurol. 71, 110-120.
37. Marucha, J., Tylki-Szymańska, A., Jakõbkiewicz-Banecka, J., Piotrowska, E., Kloska, A., et al.(2011) Improvement in the range of joint motion in seven patients with mucopolysaccharidosis type II during experimental gene expression-targeted isoflavone therapy (GET IT). Am. J. Med. Genet. A 155, 2257-2262.
38. Piotrowska, E., Jakõbkiewicz-Banecka, J., and, WȨgrzyn, G., (2010) Different amounts of isoflavones in various commercially available soy extracts in the light of gene expression-targeted isoflavone therapy. Phytother. Res. 24 (Suppl 1), S109-S113.
39. Szkudelska, K., and, Nogowski, L., (2007) Genistein-a dietary compound inducing hormonal and metabolic changes. J. Steroid Biochem. Mol. Biol. 105, 37-45. (Pubitemid 47211954)
40. Rusin, A., Krawczyk, Z., Grynkiewicz, G., Gogler, A., Zawisza-Puchałka, J., et al.(2010) Synthetic derivatives of genistein, their properties and possible applications. Acta Biochim. Pol. 57, 23-34.
41. WȨgrzyn, G., Jakõbkiewicz-Banecka, J., Gabig- Cimińska, M., Piotrowska, E., Narajczyk, M., et al.(2010) Genistein: a natural isoflavone with a potential for treatment of genetic diseases. Biochem. Soc. Trans. 38, 695-701.
42. Hamano, K., Hayashi, M., Shioda, K., Fukatsu, R., and, Mizutani, S., (2008) Mechanisms of neurodegeneration in mucopolysacchridoses II and IIIB: analysis of human brain tissue. Acta Neuropathol. 115, 547-559.
43. Villani, G. R., Gargiulo, N., Faraonio, R., Castaldo, S., Gonzalez, Y., et al.(2007) Cytokines, nuerotrophins, and oxidative stress in brain disease from mucopolysaccharidosis IIIB. J. Neurosci. Res. 85, 612-622.
44. Villani, G. R., Di Domenico, C., Musella, A., Cecere, F., Di Napoli, D., et al.(2009) Mucopolysaccharisosis IIIB: oxidative damage and cytotoxic cell involvement in the neuronal pathogenesis. Brain Res. 1279, 99-108.
45. Ohmi, K., Kudo, L. C., Ryazantsev, S., Zhao, H. Z., Karsten, S. L., et al.(2009) Sanfilippo syndrome type B, a lysosomal storage disease, is also a tauopathy. Proc. Natl. Acad. Sci. U.S.A. 106, 8332-8337.
46. Bang, O. H., Hong, H. S., Kim, D. H., Kim, H., Boo, J. H., et al.(2004) Neuroprotective effect of genistein against beta amyloid-induces neurotoxicity. Neurobiol. Dis. 16, 21-28. (Pubitemid 38569792)
47. Liang, H. W., Qiu, S. F., Shen, J., Sun, L. N., and, Wang, J. Y., (2008) Genistein attenuates oxidative stress and neuronal damage following transient global cerebral ischemia in rat hippocampus. Neurosci. Lett. 438, 116-120.
48. Valles, S. L., Dolz-Gaiton, P., Gambini, J., Borras, C., Loret, A. L., et al.(2010) Estradiol or genistein prevent Alzheimer's disease-associated inflammation correlating with an increase PPAR gamma expression cultured astrocytes. Brain Res. 1312, 138-144.
49. Kajta, M., Domin, H., Grynkiewicz, G., and, Lason, W., (2007) Genistein inhibits glutamate-induced apoptotic processes in primary neuronal cell cultures: an involvement of aryl hydrocarbon receptor and estrogen receptor/glycogen synthase kinase-3beta intracellular signaling pathway. Neuroscience 145, 592-604. (Pubitemid 46341646)
50. Chindewa, R., Lapanantasin, S., Sanvarinda, Y., and, Chongthammakun, S., (2007) Genistein effects on synaptophysin expression in rat hippocampal neurons. Ann. Microsc. 7, 95-101.