Title:
ANTISENSE NUCLEIC ACID FOR INHIBITING BIOSYNTHESIS OF CHONDROITIN SULFATE
Document Type and Number:
WIPO Patent Application WO/2018/180005
Kind Code:
A1
Abstract:
The present invention provides an antisense oligonucleotide for inhibiting biosynthesis of chondroitin sulfate. This antisense oligonucleotide has at least one modified nucleotide that inhibits expression of chondroitin sulfate N-acetylgalactosaminyltransferase 1 (CSGalNAcT1) gene and/or chondroitin sulfate N-acetylgalactosaminyltransferase 2 (CSGalNAcT2) gene.
Inventors:
OBIKA SATOSHI (JP)
KASAHARA YUYA (JP)
TAKEUCHI KOSEI (JP)
KASAHARA YUYA (JP)
TAKEUCHI KOSEI (JP)
Application Number:
PCT/JP2018/005909
Publication Date:
October 04, 2018
Filing Date:
February 20, 2018
Export Citation:
Assignee:
JAPAN AGENCY FOR MEDICAL RES AND DEVELOPMENT (JP)
NAT INST BIOMEDICAL INNOVATION HEALTH & NUTRITION (JP)
NAT INST BIOMEDICAL INNOVATION HEALTH & NUTRITION (JP)
International Classes:
C12N15/113; A61K31/7115; A61K31/712; A61K31/7125; A61K48/00; A61P25/00; C12N15/09
Domestic Patent References:
| WO2016006697A1 | 2016-01-14 | |||
| WO2013111897A1 | 2013-08-01 | |||
| WO2011052436A1 | 2011-05-05 |
Foreign References:
| JP2015228868A | 2015-12-21 | |||
| JP2007082508A | 2007-04-05 | |||
| JP2009533321A | 2009-09-17 | |||
| JP2017072315A | 2017-04-13 |
Other References:
HOLMBORN K ET AL.: "On the roles and regulation of chondroitin sulfate and heparan sulfate in zebrafish pharyngeal cartilage morphogenesis", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 287, no. 40, 6 August 2012 (2012-08-06) - 28 September 2010 (2010-09-28), pages 33905 - 33916, XP055613122
TAKEUCHI K ET AL.: "Chondroitin sulphate N-acetylgalactosaminyl- transferase-1 inhibits recovery from neural injury", NATURE COMMUNICATIONS, vol. 4, no. 2740, 12 November 2013 (2013-11-12), pages 1 - 11, XP055613124
TAKEUCHI KOSEI: "Amelioration or neuronal injury by the expressional regulation of the glycosaminoglycan", JOURNAL JPN. BIOCHEM. SOC., vol. 87, 2015, pages 744 - 748, XP009517031, ISSN: 0037-1017
TAKEUCHI K ET AL.: "In vivo regulation of chondroitin sulfate gene to recovery from spinal cord injury and brain infarction", JOURNAL OF NEUROCHEMISTRY, vol. 142, no. 1, August 2017 (2017-08-01), pages 118, XP009517032
DZIEDZIC D ET AL.: "Simultaneous siRNA-mediated silencing of pairs of genes coding for enzymes involved in glycosaminoglycan synthesis", ACTA BIOCHIM. POL., vol. 59, no. 2, 14 May 2012 (2012-05-14), pages 293 - 298, XP055613125
ZHANG L ET AL.: "Chondroitin sulfate N-acetylgalactosaminyltransferase-2 contributes to the replication of infectious bursal disease virus via interaction with the capsid protein VP 2", VIRUSES, vol. 7, no. 3, 23 March 2015 (2015-03-23), pages 1474 - 1491, XP055613126
WATANABE, T. ET AL.: "Chondroitin sulfate N-acetylgalactosaminyltransferase-1 is required for normal cartilage development", BIOCHEM. J., vol. 432, 2010, pages 47 - 55
TAKEUCHI, K. ET AL.: "Chondroitin sulphate N-acetylgalactosaminyltransferase-1 inhibits recovery from neural injury", NATURE COMMUNICATION, vol. 4, 2013, pages 2740, XP055613124, doi:10.1038/ncomms3740
ALTSCHUL, S.F. ET AL.: "Basic local alignment search tool", J. MOL. BIOL., vol. 215, 1990, pages 403 - 410, XP002949123, doi:10.1006/jmbi.1990.9999
CHEMBIOCHEM, vol. 13, no. 7, 2012, pages 2513 - 2516
YAMAMOTO, T. ET AL.: "Amido-bridged nucleic acids with small hydrophobic residues enhance hepatic tropism of antisense oligonucleotides in vivo", ORG. BIOMOL. CHEM., vol. 13, 2015, pages 3757 - 3765, XP055286305, doi:10.1039/C5OB00242G
PUNIT, P.S. ET AL.: "Short antisense oligonucleotides with novel 2'-4' conformationally restricted nucleoside analogues show improved potency without increased toxicity in animals", J. MED. CHEM., vol. 52, no. 1, 2009, pages 10 - 13, XP055026023, doi:10.1021/jm801294h
M. ZUKER: "Mfold web server for nucleic acid folding and hybridization prediction", NUCLEIC ACIDS RES., vol. 31, no. 13, 2003, pages 3406 - 15, XP002460708, doi:10.1093/nar/gkg595
TAKEUCHI, K. ET AL.: "Chondroitin sulphate N-acetylgal actosaminyltransferase-1 inhibits recovery from neural injury", NATURE COMMUNICATION, vol. 4, 2013, pages 2740
BASSO, D.M. ET AL.: "Basso Mouse Scale for locomotion detects differences in recovery after injury of the spinal cord in five common mouse strains", J NEUROTRAUMA, vol. 23, no. 5, 2006, pages 635 - 59
BASSO, D.M. ET AL.: "A sensitive and reliable locomotor rating scale for open field testing in rats", J NEUROTRAUMA, vol. 12, no. 1, 1995, pages 1 - 21, XP009036806
BASSO, D.M. ET AL.: "Graded histological and locomotor outcomes after spinal cord contusion using the NYU weight-drop device versus transection", EXPERIMENTAL NEUROLOGY, vol. 139, no. 2, 1996, pages 244 - 256
See also references of EP 3604531A4
TAKEUCHI K ET AL.: "Chondroitin sulphate N-acetylgalactosaminyl- transferase-1 inhibits recovery from neural injury", NATURE COMMUNICATIONS, vol. 4, no. 2740, 12 November 2013 (2013-11-12), pages 1 - 11, XP055613124
TAKEUCHI KOSEI: "Amelioration or neuronal injury by the expressional regulation of the glycosaminoglycan", JOURNAL JPN. BIOCHEM. SOC., vol. 87, 2015, pages 744 - 748, XP009517031, ISSN: 0037-1017
TAKEUCHI K ET AL.: "In vivo regulation of chondroitin sulfate gene to recovery from spinal cord injury and brain infarction", JOURNAL OF NEUROCHEMISTRY, vol. 142, no. 1, August 2017 (2017-08-01), pages 118, XP009517032
DZIEDZIC D ET AL.: "Simultaneous siRNA-mediated silencing of pairs of genes coding for enzymes involved in glycosaminoglycan synthesis", ACTA BIOCHIM. POL., vol. 59, no. 2, 14 May 2012 (2012-05-14), pages 293 - 298, XP055613125
ZHANG L ET AL.: "Chondroitin sulfate N-acetylgalactosaminyltransferase-2 contributes to the replication of infectious bursal disease virus via interaction with the capsid protein VP 2", VIRUSES, vol. 7, no. 3, 23 March 2015 (2015-03-23), pages 1474 - 1491, XP055613126
WATANABE, T. ET AL.: "Chondroitin sulfate N-acetylgalactosaminyltransferase-1 is required for normal cartilage development", BIOCHEM. J., vol. 432, 2010, pages 47 - 55
TAKEUCHI, K. ET AL.: "Chondroitin sulphate N-acetylgalactosaminyltransferase-1 inhibits recovery from neural injury", NATURE COMMUNICATION, vol. 4, 2013, pages 2740, XP055613124, doi:10.1038/ncomms3740
ALTSCHUL, S.F. ET AL.: "Basic local alignment search tool", J. MOL. BIOL., vol. 215, 1990, pages 403 - 410, XP002949123, doi:10.1006/jmbi.1990.9999
CHEMBIOCHEM, vol. 13, no. 7, 2012, pages 2513 - 2516
YAMAMOTO, T. ET AL.: "Amido-bridged nucleic acids with small hydrophobic residues enhance hepatic tropism of antisense oligonucleotides in vivo", ORG. BIOMOL. CHEM., vol. 13, 2015, pages 3757 - 3765, XP055286305, doi:10.1039/C5OB00242G
PUNIT, P.S. ET AL.: "Short antisense oligonucleotides with novel 2'-4' conformationally restricted nucleoside analogues show improved potency without increased toxicity in animals", J. MED. CHEM., vol. 52, no. 1, 2009, pages 10 - 13, XP055026023, doi:10.1021/jm801294h
M. ZUKER: "Mfold web server for nucleic acid folding and hybridization prediction", NUCLEIC ACIDS RES., vol. 31, no. 13, 2003, pages 3406 - 15, XP002460708, doi:10.1093/nar/gkg595
TAKEUCHI, K. ET AL.: "Chondroitin sulphate N-acetylgal actosaminyltransferase-1 inhibits recovery from neural injury", NATURE COMMUNICATION, vol. 4, 2013, pages 2740
BASSO, D.M. ET AL.: "Basso Mouse Scale for locomotion detects differences in recovery after injury of the spinal cord in five common mouse strains", J NEUROTRAUMA, vol. 23, no. 5, 2006, pages 635 - 59
BASSO, D.M. ET AL.: "A sensitive and reliable locomotor rating scale for open field testing in rats", J NEUROTRAUMA, vol. 12, no. 1, 1995, pages 1 - 21, XP009036806
BASSO, D.M. ET AL.: "Graded histological and locomotor outcomes after spinal cord contusion using the NYU weight-drop device versus transection", EXPERIMENTAL NEUROLOGY, vol. 139, no. 2, 1996, pages 244 - 256
See also references of EP 3604531A4
Attorney, Agent or Firm:
HIRAKI & ASSOCIATES (JP)
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