Title:
ANTISENSE OLIGONUCLEOTIDE CONTROLLING EXPRESSION AMOUNT OF TDP-43 AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2019/013141
Kind Code:
A1
Abstract:
Provided are: a restraining type antisense oligonucleotide targeting TDP-43 mRNA, said antisense oligonucleotide containing a nucleotide sequence complementary to a sequence consisting of at least 10 consecutive nucleotides in a nucleotide sequence represented by any of SEQ ID NOS: 2 to 4; and an accelerating type antisense oligonucleotide targeting TDP-43 mRNA, said antisense oligonucleotide containing a nucleotide sequence complementary to a sequence consisting of at least 10 consecutive nucleotides in the nucleotide sequence represented by SEQ ID NO: 5.
Inventors:
NAGAI YOSHITAKA (JP)
MAETA KAZUHIRO (JP)
TAKEUCHI TOSHIHIDE (JP)
NEYA MASAHIRO (JP)
FUJIWARA TAKESHI (JP)
MATSUDA SEIJI (JP)
SOBUE GEN (JP)
ISHIGAKI SHINSUKE (JP)
SAHASHI KENTARO (JP)
MAETA KAZUHIRO (JP)
TAKEUCHI TOSHIHIDE (JP)
NEYA MASAHIRO (JP)
FUJIWARA TAKESHI (JP)
MATSUDA SEIJI (JP)
SOBUE GEN (JP)
ISHIGAKI SHINSUKE (JP)
SAHASHI KENTARO (JP)
Application Number:
PCT/JP2018/025792
Publication Date:
January 17, 2019
Filing Date:
July 06, 2018
Export Citation:
Assignee:
UNIV OSAKA (JP)
KNC LABORATORIES CO LTD (JP)
UNIV NAGOYA NAT UNIV CORP (JP)
KNC LABORATORIES CO LTD (JP)
UNIV NAGOYA NAT UNIV CORP (JP)
International Classes:
C12N15/113; A61K31/712; A61K48/00; A61P25/28
Domestic Patent References:
WO2014077693A1 | 2014-05-22 | |||
WO2016088797A1 | 2016-06-09 | |||
WO2016006697A1 | 2016-01-14 | |||
WO2007026485A1 | 2007-03-08 | |||
WO2004080406A2 | 2004-09-23 |
Foreign References:
US20110321179A1 | 2011-12-29 | |||
JP2017071572A | 2017-04-13 | |||
JP2002241393A | 2002-08-28 | |||
JP2000297097A | 2000-10-24 | |||
US3687808A | 1972-08-29 | |||
JP2017134890A | 2017-08-03 |
Other References:
BURATTI, E. ET AL.: "Nuclear factor TDP-43 and proteins promote in vitro and in vivo CFTRexon 9 skipping", EMBO JOURNAL, vol. 20, no. 7, April 2001 (2001-04-01), pages 1774 - 1784, XP055009063, DOI: 10.1093/emboj/20.7.1774
ARIIZUMI, YUKO: "Reduced expression of TDP-43 changes the structures of subcellular organelles", NIIGATA MEDICAL JOURNAL, vol. 125, 2011, pages 482 - 497, XP008174337
SCOTTER, EL ET AL.: "TDP-43 proteinopathy and ALS: Insights into disease mechanisms and therapeutic targets", NEUROTHERAPEUTICS, vol. 12, no. 2, 2015, pages 352 - 363, XP035490053, DOI: 10.1007/s13311-015-0338-x
SUGAI, AKIHIRO ET AL.: "Endogenous TDP-43 overexpression with disrupted autoregulation: Toward an amyotrophic lateral sclerosis model", NIIGATA MEDICAL JOURNAL, vol. 129, 2015, pages 658 - 670, XP009518813
BHARDWAJ, A. ET AL: "Characterizing TDP-43 interaction with its RNA targets", NUCLEIC ACIDS RESEARCH, vol. 41, no. 9, 21 March 2013 (2013-03-21), pages 5062 - 5074, XP055455765, DOI: 10.1093/nar/gkt189
POLYMENIDOU, M. ET AL.: "Long pre-mRNA depletion and RNA missplicing contribute to neural vulnerability from loss of TDP-43", NATURE NEUROSCIENCE, vol. 14, no. 4, 27 February 2011 (2011-02-27), pages 459 - 468, XP055666258, ISSN: 1097-6256, DOI: 10.1038/nn.2779
KABASHI, E. ET AL.: "Fus and tardbp but not sodl interact in genetic models of amyotrophic lateral sclerosis", PLOS GENETICS, vol. 7, no. 8, e1002214, 4 August 2011 (2011-08-04), XP009155861, DOI: 10.1371/journal.pgen.1002214
BHANDARE, VV ET AL.: "Identification of possible siRNA molecules for TDP43 mutants causing amyotrophic lateral sclerosis", COMPUT BIOL CHEM., vol. 61, 23 January 2016 (2016-01-23), pages 97 - 108, XP029466612, DOI: 10.1016/j.compbiolchem.2016.01.001
DATABASE Genbank (online) 19 March 2007 (2007-03-19), "Homo sapiens TAR DNA binding protein, mRNA (cDNA clone image:3506121)", XP055666275, Database accession no. BC001487.2
NEUMANN M. ET AL., SCIENCE, vol. 314, no. 5796, 2006, pages 130 - 133
ARAI T. ET AL., BIOCHEM BIOPHYS RES COMMU., vol. 35, no. 3, 2006, pages 602 - 611
LING S.C. ET AL., NEURON, vol. 79, no. 3, 2013, pages 416 - 438
IGUCHI Y. ET AL., J BIOL CHEM, vol. 284, no. 33, 2009, pages 22059 - 22066
KOYAMA A. ET AL., NUCLEIC ACIDS RES., vol. 44, no. 12, 2016, pages 5820 - 5836
AULAS A. ET AL., MOL NEURODEGENER., vol. 7, 2012, pages 54
YU Z. ET AL., J BIOL CHEM., vol. 287, no. 27, 2012, pages 22560 - 22572
LIMBACH ET AL.: "Summary: the modified nucleosides of RNA", NUCLEIC ACIDS RES., vol. 22, 1994, pages 2183 - 2196, XP001152667
MED. CHEM. COMMUN., vol. 5, 2014, pages 1454 - 1471
"Concise Encyclopedia Of Polymer Science And Engineering", 1990, JOHN WILEY & SONS, pages: 858 - 859
ENGLISCH ET AL., ANGEWANDTE CHEMIE, vol. 30, 1991, pages 613
LOAKES, NUCLEIC ACIDS RES., vol. 29, 2001, pages 2437
CLOTILDE LAGIER-TOURENNE ET AL., HUMAN MOLECULAR GENETICS, vol. 19, no. 1, 2010
ARAI, NEUROPATHOLOGY, vol. 34, 2014, pages 578 - 588
"GenBank", Database accession no. BC001487.2
JIANG ET AL., NEURON, 2016
VOLPICELLI-DALEY ET AL., J NEUROSCI., 2016
D'YDEWALLE ET AL., NEURON, 2017
BECKER ET AL., NATURE, 2017
BHARDWAJ AMYERS MPBURATTI EBARALLE FE, NUCLEIC ACIDS RES., vol. 1, no. 9, 2013, pages 5062 - 5074
STAROPOLI ET AL., GENOMICS, 2015
KAPELI ET AL., NAT COMMUN., 2016
SWARUP VPHANEUF DBAREIL CROBERTSON JROULEAU GAKRIZ JJULIEN JP, BRAIN, vol. 134, 2011, pages 2610 - 2626
See also references of EP 3653711A4
ARIIZUMI, YUKO: "Reduced expression of TDP-43 changes the structures of subcellular organelles", NIIGATA MEDICAL JOURNAL, vol. 125, 2011, pages 482 - 497, XP008174337
SCOTTER, EL ET AL.: "TDP-43 proteinopathy and ALS: Insights into disease mechanisms and therapeutic targets", NEUROTHERAPEUTICS, vol. 12, no. 2, 2015, pages 352 - 363, XP035490053, DOI: 10.1007/s13311-015-0338-x
SUGAI, AKIHIRO ET AL.: "Endogenous TDP-43 overexpression with disrupted autoregulation: Toward an amyotrophic lateral sclerosis model", NIIGATA MEDICAL JOURNAL, vol. 129, 2015, pages 658 - 670, XP009518813
BHARDWAJ, A. ET AL: "Characterizing TDP-43 interaction with its RNA targets", NUCLEIC ACIDS RESEARCH, vol. 41, no. 9, 21 March 2013 (2013-03-21), pages 5062 - 5074, XP055455765, DOI: 10.1093/nar/gkt189
POLYMENIDOU, M. ET AL.: "Long pre-mRNA depletion and RNA missplicing contribute to neural vulnerability from loss of TDP-43", NATURE NEUROSCIENCE, vol. 14, no. 4, 27 February 2011 (2011-02-27), pages 459 - 468, XP055666258, ISSN: 1097-6256, DOI: 10.1038/nn.2779
KABASHI, E. ET AL.: "Fus and tardbp but not sodl interact in genetic models of amyotrophic lateral sclerosis", PLOS GENETICS, vol. 7, no. 8, e1002214, 4 August 2011 (2011-08-04), XP009155861, DOI: 10.1371/journal.pgen.1002214
BHANDARE, VV ET AL.: "Identification of possible siRNA molecules for TDP43 mutants causing amyotrophic lateral sclerosis", COMPUT BIOL CHEM., vol. 61, 23 January 2016 (2016-01-23), pages 97 - 108, XP029466612, DOI: 10.1016/j.compbiolchem.2016.01.001
DATABASE Genbank (online) 19 March 2007 (2007-03-19), "Homo sapiens TAR DNA binding protein, mRNA (cDNA clone image:3506121)", XP055666275, Database accession no. BC001487.2
NEUMANN M. ET AL., SCIENCE, vol. 314, no. 5796, 2006, pages 130 - 133
ARAI T. ET AL., BIOCHEM BIOPHYS RES COMMU., vol. 35, no. 3, 2006, pages 602 - 611
LING S.C. ET AL., NEURON, vol. 79, no. 3, 2013, pages 416 - 438
IGUCHI Y. ET AL., J BIOL CHEM, vol. 284, no. 33, 2009, pages 22059 - 22066
KOYAMA A. ET AL., NUCLEIC ACIDS RES., vol. 44, no. 12, 2016, pages 5820 - 5836
AULAS A. ET AL., MOL NEURODEGENER., vol. 7, 2012, pages 54
YU Z. ET AL., J BIOL CHEM., vol. 287, no. 27, 2012, pages 22560 - 22572
LIMBACH ET AL.: "Summary: the modified nucleosides of RNA", NUCLEIC ACIDS RES., vol. 22, 1994, pages 2183 - 2196, XP001152667
MED. CHEM. COMMUN., vol. 5, 2014, pages 1454 - 1471
"Concise Encyclopedia Of Polymer Science And Engineering", 1990, JOHN WILEY & SONS, pages: 858 - 859
ENGLISCH ET AL., ANGEWANDTE CHEMIE, vol. 30, 1991, pages 613
LOAKES, NUCLEIC ACIDS RES., vol. 29, 2001, pages 2437
CLOTILDE LAGIER-TOURENNE ET AL., HUMAN MOLECULAR GENETICS, vol. 19, no. 1, 2010
ARAI, NEUROPATHOLOGY, vol. 34, 2014, pages 578 - 588
"GenBank", Database accession no. BC001487.2
JIANG ET AL., NEURON, 2016
VOLPICELLI-DALEY ET AL., J NEUROSCI., 2016
D'YDEWALLE ET AL., NEURON, 2017
BECKER ET AL., NATURE, 2017
BHARDWAJ AMYERS MPBURATTI EBARALLE FE, NUCLEIC ACIDS RES., vol. 1, no. 9, 2013, pages 5062 - 5074
STAROPOLI ET AL., GENOMICS, 2015
KAPELI ET AL., NAT COMMUN., 2016
SWARUP VPHANEUF DBAREIL CROBERTSON JROULEAU GAKRIZ JJULIEN JP, BRAIN, vol. 134, 2011, pages 2610 - 2626
See also references of EP 3653711A4
Attorney, Agent or Firm:
TAKASHIMA, Hajime et al. (JP)
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