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2012 | 59 | 2 | 293-298
Article title

Simultaneous siRNA-mediated silencing of pairs of genes coding for enzymes involved in glycosaminoglycan synthesis

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EN
Abstracts
EN
It has been demonstrated recently that it is possible to decrease expression of genes coding for enzymes involved in synthesis of glycosaminoglycans (GAGs) by using specific siRNAs which interfere with stability of particular mRNAs. This procedure has been proposed as a potential treatment for patients suffering from mucopolysaccharidoses, a group of inherited metabolic diseases caused by dysfunction of enzymes required for GAG degradation, and resultant storage of these compounds in cells of affected persons. Here, we asked if the simultaneous use two species of specific siRNAs aimed at silencing two genes involved in particular steps of GAG synthesis may be more effective than the use of single siRNA. We found that inhibition of GAG synthesis in cells treated with two siRNAs is generally more effective than using single siRNAs. However, the differences were not statistically significant, therefore the potential benefit from the use of two siRNAs over the use of a single siRNA is doubtful in the light of the cost-benefit ratio and possibly stronger side-effects of the putative therapy.
Keywords
EN
GAGs   MPSs  
Year
Volume
59
Issue
2
Pages
293-298
Physical description
Dates
published
2012
received
2011-11-21
revised
2012-02-22
accepted
2012-04-26
(unknown)
2012-05-14
References
  • Akiyama T, Ishida J, Nakagawa S, Ogawara H, Watanabe S, Itoh N, Shibuya M, Fukami Y (1987) Genistein, a specific inhibitor of tyrosine-specific protein kinases. J Biol Chem 262: 5592-5595.
  • Almeida R, Levery SB, Mandel U, Kresse H, Schwientek T, Bennett EP, Clausen H (1999) Cloning and expression of a proteoglycan UDP-galactose:beta-xylose beta14-galactosyltransferase I. A seventh member of the human beta4-galactosyltransferase gene family. J Biol Chem 274: 26165-26171.
  • Bai X, Zhou D, Brown JR, Crawford BE, Hennet T, Esko JD (2001) Biosynthesis of the linkage region of glycosaminoglycans: cloning and activity of galactosyltransferase II the sixth member of the beta 13-galactosyltransferase family (beta 3GalT6). J Biol Chem 276: 48189-48195.
  • Busse M, Feta A, Presto J, Wilén M, Grønning M, Kjellén L, Kusche-Gullberg M (2007) Contribution of EXT1, EXT2 and EXTL3 to heparan sulfate chain elongation. J Biol Chem 282: 32802-3210.
  • Chang CI, Kang HS, Ban C, Kim S, Lee DK (2009) Dual-target gene silencing by using long synthetic siRNA duplexes without triggering antiviral responses. Mol Cells 27: 689-695.
  • Chang HC, Churchwell MI, Delclos KB, Newbold RR, Doerge DR (2000) Mass spectrometric determination of Genistein tissue distribution in diet-exposed Sprague-Dawley rats. J Nutr 130: 1963-1970.
  • Condac E, Dale GL, Bender-Neal D, Ferencz B, Towner R, Hinsdale ME (2009) Xylosyltransferase II is a significant contributor of circulating xylosyltransferase levels and platelets constitute an important source of xylosyltransferase in serum. Glycobiology 19: 829-833.
  • Crowe S (2003) Suppression of chemokine receptor expression by RNA interference allows for inhibition of HIV-1 replication by Martínez et al. AIDS 17 (Suppl 4): S103-S105.
  • Davis ME, Zuckerman JE, Choi CH, Seligson D, Tolcher A, Alabi CA, Yen Y, Heidel JD, Ribas A (2010) Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature 464: 1067-1070.
  • Devi GR (2006) siRNA-based approaches in cancer therapy. Cancer Gene Ther 13: 819-829.
  • 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.
  • Esko JD, Lindahl U (2001) Molecular diversity of heparan sulfate. J Clin Invest 108: 169-173.
  • Götting C, Kuhn J, Kleesiek K (2007) Human xylosyltransferases in health and disease. Cell Mol Life Sci 64: 1498-1517.
  • Götting C, Kuhn J, Zahn R, Brinkmann T, Kleesiek K (2000) Molecular cloning and expression of human UDP-d-Xylose:proteoglycan core protein beta-d-xylosyltransferase and its first isoform XT-II. J Mol Biol 304: 517-528.
  • Götting C, Sollberg S, Kuhn J, Weilke C, Huerkamp C, Brinkmann T, Krieg T, Kleesiek K (1999) Serum xylosyltransferase: a new biochemical marker of the sclerotic process in systemic sclerosis. J Invest Dermatol 112: 919-924.
  • Harmatz P, Giugliani R, Schwartz I, Guffon N, Teles EL, Miranda MC, Wraith JE, Beck M, Arash L, Scarpa M, Yu ZF, Wittes J, Berger KI, Newman MS, Lowe AM, Kakkis E, Swiedler SJ, Group MVPS (2006) Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3 randomized double-blind placebo-controlled multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on open-label extension study. J Pediatr 148: 533-539.
  • Herman-Antosiewicz A, Johnson DE, Singh SV (2006) Sulforaphane causes autophagy to inhibit release of cytochrome C and apoptosis in human prostate cancer cells. Cancer Res 66: 5828-5835.
  • Jakóbkiewicz-Banecka J, Piotrowska E, Narajczyk M, Barańska S, Wegrzyn 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.
  • 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.
  • Kang TH, Lee JH, Noh KH, Han HD, Shin BC, Choi EY, Peng S, Hung CF, Wu TC, Kim TW (2007) Enhancing dendritic cell vaccine potency by combining a BAK/BAX siRNA-mediated antiapoptotic strategy to prolong dendritic cell life with an intracellular strategy to target antigen to lysosomal compartments. Int J Cancer 120: 1696-1703.
  • Kim DW, Kim KO, Shin MJ, Ha JH, Seo SW, Yang J, Lee FY (2009) siRNA-based targeting of antiapoptotic genes can reverse chemoresistance in P-glycoprotein expressing chondrosarcoma cells. Mol Cancer 8: 28.
  • Kitagawa H, Tone Y, Tamura J, Neumann KW, Ogawa T, Oka S, Kawasaki T, Sugahara K (1998) Molecular cloning and expression of glucuronyltransferase I involved in the biosynthesis of the glycosaminoglycan-protein linkage region of proteoglycans. J Biol Chem 273: 6615-6618.
  • Kitagawa H, Uyama T, Sugahara K (2001) Molecular cloning and expression of a human chondroitin synthase. J Biol Chem 276: 38721-38726.
  • Kleene R, Berger EG (1993) The molecular and cell biology of glycosyltransferases. Biochim Biophys Acta 1154: 283-325.
  • Kubiak K, Nawrot B (2009) RNAi in therapy. Biotechnologia 1: 132-151.
  • Kusov Y, Kanda T, Palmenberg A, Sgro JY, Gauss-Müller V (2006) Silencing of hepatitis A virus infection by small interfering RNAs. J Virol 80: 5599-5610.
  • Lambeth LS, Van Hateren NJ, Wilson SA, Nair V (2010) A direct comparison of strategies for combinatorial RNA interference. BMC Mol Biol 11: 77.
  • Li JP (2010) Glucuronyl C5-epimerase an enzyme converting glucuronic acid to iduronic acid in heparan sulfate/heparin biosynthesis. Prog Mol Biol Transl Sci 93: 59-78.
  • Livak KJ, Schmittgen TD (2001) Analysis of relative gene expression data using real-time quantitative PCR and the 2(-Delta Delta C(T)) Method. Methods 25: 402-408.
  • Mcintyre GJ, Yu YH, Tran A, Jaramillo AB, Arndt AJ, Millington ML, Boyd MP, Elliott FA, Shen SW, Murray JM, Applegate TL (2009) Cassette deletion in multiple shRNA lentiviral vectors for HIV-1 and its impact on treatment success. Virol J 6: 184.
  • McSwiggen JA, Seth S (2008) A potential treatment for pandemic influenza using siRNAs targeting conserved regions of influenza A. Expert Opin Biol Ther 8: 299-313.
  • Muenzer J, Wraith JE, Beck M, Giugliani R, Harmatz P, Eng CM, Vellodi A, Martin R, Ramaswami U, Gucsavas-Calikoglu M, Vijayaraghavan S, Wendt S, Puga AC, Ulbrich B, Shinawi M, Cleary M, Piper D, Conway AM, Kimura A (2006) A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet Med 8: 465-473.
  • Murata M, Bonassar LJ, Wright M, Mankin HJ, Towle CA (2003) A role for the interleukin-1 receptor in the pathway linking static mechanical compression to decreased proteoglycan synthesis in surface articular cartilage. Arch Biochem Biophys 413: 229-235.
  • Neufeld EF, Muenzer J (2001) The mucopolysaccharidoses. In The Metabolic and Molecular Bases of Inherited Disease. Scriver CR et al., eds pp 3421-3452. McGraw-Hill Co New York.
  • Okajima T, Yoshida K, Kondo T, Furukawa K (1999) Human homolog of Caenorhabditis elegans sqv-3 gene is galactosyltransferase I involved in the biosynthesis of the glycosaminoglycan-protein linkage region of proteoglycans. J Biol Chem 274: 22915-22918.
  • Okuda T, Mita S, Yamauchi S, Matsubara T, Yagi F, Yamamori D, Fukuta M, Kuroiwa A, Matsuda Y, Habuchi O (2000) Molecular cloning, expression, and chromosomal mapping of human chondroitin 4-sulfotransferase, whose expression pattern in human tissues is different from that of chondroitin 6-sulfotransferase. J Biochem 128: 763-770.
  • Pacheco B, Malmström A, Maccarana M (2009) Two dermatan sulfate epimerases form iduronic acid domains in dermatan sulfate. J Biol Chem 284: 9788-9795.
  • Piotrowska E, Jakóbkiewicz-Banecka J, Barańska S, Tylki-Szymańska 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.
  • Pönighaus C, Ambrosius M, Casanova JC, Prante C, Kuhn J, Esko JD, Kleesiek K, Götting C (2007) Human xylosyltransferase II is involved in the biosynthesis of the uniform tetrasaccharide linkage region in chondroitin sulfate and heparan sulfate proteoglycans. J Biol Chem 282: 5201-5206.
  • Raoul C, Barker SD, Aebischer P (2006) Viral-based modelling and correction of neurodegenerative diseases by RNA interference. Gene Ther 13: 487-495.
  • Sato T, Gotoh M, Kiyohara K, Akashima T, Iwasaki H, Kameyama A, Mochizuki H, Yada T, Inaba N, Togayachi A, Kudo T, Asada M, Watanabe H, Imamura T, Kimata K, Narimatsu H (2003) Differential roles of two N-acetylgalactosaminyltransferases CSGalNAcT-1 and a novel enzyme CSGalNAcT-2. Initiation and elongation in synthesis of chondroitin sulfate. J Biol Chem 278: 3063-3071.
  • Shworak NW, Liu J, Petros LM, Zhang L, Kobayashi M, Copeland NG, Jenkins NA, Rosenberg RD (1999) Multiple isoforms of heparan sulfate d-glucosaminyl 3-O-sulfotransferase. Isolation characterization and expression of human cdnas and identification of distinct genomic loci. J Biol Chem 274: 5170-5184.
  • Stevenson M (2004) Therapeutic potential of RNA interference. N Engl J Med 351: 1772-1777.
  • Tone Y, Kitagawa H, Imiya K, Oka S, Kawasaki T, Sugahara K (1999) Characterization of recombinant human glucuronyltransferase I involved in the biosynthesis of the glycosaminoglycan-protein linkage region of proteoglycans. FEBS Lett 459: 415-420.
  • Uyama T, Kitagawa H, Tanaka J, Tamura J, Ogawa T, Sugahara K (2003) Molecular cloning and expression of a second chondroitin N-acetylgalactosaminyltransferase involved in the initiation and elongation of chondroitin/dermatan sulfate. J Biol Chem 278: 3072-3078.
  • Voglmeir J, Voglauer R, Wilson IB (2007) XT-II, the second isoform of human peptide-O-xylosyltransferase, displays enzymatic activity. J Biol Chem 282: 5984-5990.
  • Watkins SC, Cullen MJ (1987) A qualitative and quantitative study of the ultrastructure of regenerating muscle fibres in Duchenne muscular dystrophy and polymyositis. J Neurol Sci 82: 181-192.
  • Wilson IB (2004) The never-ending story of peptide O-xylosyltransferase. Cell Mol Life Sci 61: 794-809.
Document Type
Publication order reference
YADDA identifier
bwmeta1.element.bwnjournal-article-abpv59p293kz
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