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2012 | 59 | 4 |

Tytuł artykułu

Comparison of siRNA-mediated silencing of glycosaminoglycan synthesis genes and enzyme replacement therapy for mucopolysaccharidosis in cell culture studies

Warianty tytułu

Języki publikacji

EN

Abstrakty

EN
Cytotoxicity of laronidase (Aldurazyme®), employed in enzyme replacement therapy (ERT) for mucopolysaccharidosis type I (MPS I) and various siRNAs, tested previously in studies on substrate reduction therapy (SRT) for mucopolysaccharidoses, was tested. The enzyme did not cause any cytotoxic effects, and the siRNAs did not inhibit growth of most investigated cell lines. However, some cytotoxic effects of some tested siRNAs were observed in one MPS IIIA cell line. The efficacy of a combination of enzyme replacement therapy and siRNA-based substrate deprivation therapy was tested on three MPS I cell lines. Surprisingly, different results were obtained for different cell lines. The decrease of glycosaminoglycan storage in cells treated simultaneously with both methods was: (i) less pronounced than obtained with either of those methods used alone in one cell line, (ii) similar to that observed for enzyme replacement therapy in another cell line, and (iii) stronger than that obtained with either of the methods used alone in the third cell line. Therefore, it appears that the effects of various therapeutic methods may strongly depend on the features of the MPS cell line.

Słowa kluczowe

Wydawca

-

Rocznik

Tom

59

Numer

4

Opis fizyczny

p.697-702,fig.,ref.

Twórcy

autor
  • Department of Molecular Biology, University of Gdansk, Gdańsk, Poland
  • Department of Molecular Biology, University of Gdansk, Gdańsk, Poland
  • Laboratory of Molecular Biology, (affiliated with the University of Gdańsk), Institute of Biochemistry and Biophysics, Polish Academy of Sciences, Gdańsk, Poland
  • Department of Molecular Biology, University of Gdansk, Gdańsk, Poland
  • Department of Neurology, Medical University of Gdańsk, Gdańsk, Poland
autor
  • Department of Microbiology, University of Szczecin, Szczecin, Poland
  • Department of Molecular Biology, University of Gdansk, Gdańsk, Poland

Bibliografia

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  • Dziedzic D, Narajczyk M, Gabig-Cimińska M, Jakóbkiewicz-Banecka J (2012) Simultaneous siRNA-mediated silencing of pairs of genes coding for enzymes involved in glycosaminoglycan synthesis. Acta Biochim Pol 59: 293-298. 
  • Dziedzic D, Wegrzyn G, Jakóbkiewicz-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. 
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  • Jakóbkiewicz-Banecka J, Wegrzyn A, Wegrzyn 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. 
  • Kaidonis X, Liaw WC, Roberts AD, Ly M, Anson D, Byers S (2010) Gene silencing of EXTL2 and EXTL3 as a substrate deprivation therapy for heparan sulfate storing mucopolysaccharidoses. Eur J Hum Genet 18: 194-199. 
  • Kakkis ED, Muenzer J, Tiller GE, Waber L, Belmont J, Passage M, Izykowski B, Phillips J, Doroshow R, Walot I, Hoft R, Neufeld EF (2001) Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med 344: 182-188. 
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  • Peters C, Balthazor M, Shapiro EG, King RJ, Kollman CC, Hegland JD, Henslee-Downey J, Trigg ME, Cowan MJ, Sanders J, Bunin N, Weinstein H, Lenarsky C, Falk P, Harris R, Bowen T, Wiliams TE, Grayson GH, Warkentin P, Sender L, Cool VA, Crittenden M, Packman S, Kaplan P, Lockman LA, Anderson J, Krivit W, Dusenbery K, Wagner J (1996) Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood 87: 4894-4902. 
  • Peters C, Steward CG. (2003) Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. Bone Marrow Transplant 31: 229-239. 
  • Piotrowska E, Jakobkiewicz-Banecka J, Baranska S, Tylki-Szymanska A, Czartoryska B, Wegrzyn A, Wegrzyn G (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. 
  • Piotrowska E, Jakóbkiewicz-Banecka J, Tylki-Szymańska A, Czartoryska B, Węgrzyn A, Węgrzyn G (2008) Correlation between severity of mucopolysaccharidosis and combination of residua enzyme activity and efficiency of glycosaminoglycan synthesis. Acta Paediatr 98: 743-749. 
  • Prydz K, Dalen KT (2000) Synthesis and sorting of proteoglycans. J Cell Sci 113: 193-205. 
  • Rana TM (2007) Illuminating the silence: understanding the structure and function of small RNAs. Nat Rev Mol Cell Biol 8: 23-36. 
  • Snead NM, Rossi JJ (2012) RNA Interference trigger variants: getting the most out of RNA for RNA interference-based therapeutics. Nucleic Acid Ther 22: 139-146. 
  • Uyama T, Kitagawa H, Sugahara K (2007) Biosynthesis of glycosaminoglycans and proteoglycans. In Comprehensive Glycoscience. Vol. 1, pp 79-99. Elsevier. 
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