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

• Autorzy: Chmielarz I. , Gabig-Ciminska M. , Malinowska M. , Banecka-Majkutewicz Z. , Wegrzyn A. , Jakobkiewicz-Banecka J.
• 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.

• Wydawca: -
• Czasopismo: Acta Biochimica Polonica
• Rocznik: 2012
• Tom: 59
• Numer: 4
• Opis fizyczny: p.697-702,fig.,ref.

Twórcy

autor

Chmielarz I.

• Department of Molecular Biology, University of Gdansk, Gdańsk, Poland

autor

Gabig-Ciminska M.

• 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

autor

Malinowska M.

• Department of Molecular Biology, University of Gdansk, Gdańsk, Poland

autor

Banecka-Majkutewicz Z.

• Department of Neurology, Medical University of Gdańsk, Gdańsk, Poland

autor

Wegrzyn A.

• Department of Microbiology, University of Szczecin, Szczecin, Poland

autor

Jakobkiewicz-Banecka J.

• Department of Molecular Biology, University of Gdansk, Gdańsk, Poland

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