JP2021527649A - Ｓｔａｕｆｅｎ１調節剤および関連する方法 - Google Patents

Ｓｔａｕｆｅｎ１調節剤および関連する方法 Download PDF

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JP2021527649A
JP2021527649A JP2020569831A JP2020569831A JP2021527649A JP 2021527649 A JP2021527649 A JP 2021527649A JP 2020569831 A JP2020569831 A JP 2020569831A JP 2020569831 A JP2020569831 A JP 2020569831A JP 2021527649 A JP2021527649 A JP 2021527649A
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seq
nucleotide sequence
staufen1
therapeutic agent
nucleotides
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Japanese (ja)
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JP2021527649A5 (https=
JPWO2019241734A5 (https=
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パルスト、ステファン、エム．
スコールズ、ダニエル、アール．
ポール、シャラン
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University of Utah Research Foundation Inc
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University of Utah Research Foundation Inc
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Priority to JP2024088206A priority Critical patent/JP2024123017A/ja
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2020569831A 2018-06-14 2019-06-14 Ｓｔａｕｆｅｎ１調節剤および関連する方法 Pending JP2021527649A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024088206A JP2024123017A (ja) 2018-06-14 2024-05-30 Ｓｔａｕｆｅｎ１調節剤および関連する方法

Applications Claiming Priority (3)

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US201862685269P 2018-06-14 2018-06-14
US62/685,269 2018-06-14
PCT/US2019/037355 WO2019241734A1 (en) 2018-06-14 2019-06-14 Staufen1 regulating agents and associated methods

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JP2021527649A true JP2021527649A (ja) 2021-10-14
JP2021527649A5 JP2021527649A5 (https=) 2022-06-14
JPWO2019241734A5 JPWO2019241734A5 (https=) 2022-06-14

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JP2024088206A Pending JP2024123017A (ja) 2018-06-14 2024-05-30 Ｓｔａｕｆｅｎ１調節剤および関連する方法

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US (2) US11946046B2 (https=)
EP (1) EP3807291A4 (https=)
JP (2) JP2021527649A (https=)
WO (1) WO2019241734A1 (https=)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4615973A1 (en) * 2022-11-08 2025-09-17 University of Utah Research Foundation Staufen-1 modulating agents, compositions, and methods

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130317087A1 (en) * 2011-02-09 2013-11-28 University Of Rochester Methods and compositions related to staufen 1 binding sites formed by duplexing alu elements

Family Cites Families (9)

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Publication number Priority date Publication date Assignee Title
US20040241845A1 (en) * 1998-05-22 2004-12-02 Luc Desgroseillers Mammalian staufen and use thereof
US20040259247A1 (en) * 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
WO2006039399A2 (en) 2004-09-29 2006-04-13 University Of Rochester Staufen 1 (stau1) - mediated mrna decay
WO2009143371A2 (en) * 2008-05-21 2009-11-26 Intradigm Corporation COMPOSITIONS COMPRISING mTOR SIRNA AND METHODS OF USE THEREOF
EP2753695A1 (en) 2011-09-07 2014-07-16 Fundació Institut d'Investigació Biomèdica de Bellvitge (IDIBELL) Strategies for prevention and/or treatment of diseases based on cd40 silencing
EP3274456B1 (en) * 2015-03-25 2020-06-24 Università Degli Studi Di Trento Rna interference mediated therapy for neurodegenerative diseases
EP3551641A4 (en) 2016-12-08 2021-01-13 University of Utah Research Foundation STAUFEN1 ACTIVE SUBSTANCES AND RELATED PROCEDURES
TW201920672A (zh) * 2017-08-08 2019-06-01 新加坡商波濤生命科學有限公司 寡核苷酸組合物及其使用方法

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130317087A1 (en) * 2011-02-09 2013-11-28 University Of Rochester Methods and compositions related to staufen 1 binding sites formed by duplexing alu elements

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
NEUROLOGY, vol. Vol.86, No.16, Suppl., JPN6023019795, 2016, pages 6 - 396, ISSN: 0005361826 *

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Publication number Publication date
EP3807291A4 (en) 2022-02-23
JP2024123017A (ja) 2024-09-10
US11946046B2 (en) 2024-04-02
US20210238590A1 (en) 2021-08-05
WO2019241734A1 (en) 2019-12-19
US12565650B2 (en) 2026-03-03
EP3807291A1 (en) 2021-04-21
US20240392290A1 (en) 2024-11-28

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