JP7767324B2 - 抑制性新皮質ニューロンにおいて遺伝子発現を選択的に調節する人工発現構築物 - Google Patents

抑制性新皮質ニューロンにおいて遺伝子発現を選択的に調節する人工発現構築物

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JP7767324B2
JP7767324B2 JP2022574447A JP2022574447A JP7767324B2 JP 7767324 B2 JP7767324 B2 JP 7767324B2 JP 2022574447 A JP2022574447 A JP 2022574447A JP 2022574447 A JP2022574447 A JP 2022574447A JP 7767324 B2 JP7767324 B2 JP 7767324B2
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visp
vip
cells
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JP2023529833A (ja
JP2023529833A5 (https=
JPWO2021248085A5 (https=
Inventor
デイグル，ターニャ
グレイバック，ルーカス・ティー
レイン，エドワード・セバスチャン
リーバイ，ボアズ・ピー
ミッチ，ジョン・ケイ
セデノ・コルテス，アドリアナ・エステラ
タシッチ，ボシリカ
ティン，ジョナサン
ウォーカー，ミランダ
ゼン，ホンクイ
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アレン インスティテュート
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • 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
    • 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
    • C12N2510/00—Genetically modified cells
    • C12N2510/02—Cells for production
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Neurology (AREA)
  • Dermatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
JP2022574447A 2020-06-04 2021-06-04 抑制性新皮質ニューロンにおいて遺伝子発現を選択的に調節する人工発現構築物 Active JP7767324B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025181548A JP2026021417A (ja) 2020-06-04 2025-10-28 抑制性新皮質ニューロンにおいて遺伝子発現を選択的に調節する人工発現構築物

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063034794P 2020-06-04 2020-06-04
US63/034,794 2020-06-04
PCT/US2021/036028 WO2021248085A2 (en) 2020-06-04 2021-06-04 Artificial expression constructs for selectively modulating gene expression in inhibitory neocortical neurons

Related Child Applications (1)

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JP2025181548A Division JP2026021417A (ja) 2020-06-04 2025-10-28 抑制性新皮質ニューロンにおいて遺伝子発現を選択的に調節する人工発現構築物

Publications (4)

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JP2023529833A JP2023529833A (ja) 2023-07-12
JP2023529833A5 JP2023529833A5 (https=) 2024-04-23
JPWO2021248085A5 JPWO2021248085A5 (https=) 2024-04-23
JP7767324B2 true JP7767324B2 (ja) 2025-11-11

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JP2025181548A Pending JP2026021417A (ja) 2020-06-04 2025-10-28 抑制性新皮質ニューロンにおいて遺伝子発現を選択的に調節する人工発現構築物

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Country Status (6)

Country Link
US (1) US20230212608A1 (https=)
EP (1) EP4162041A4 (https=)
JP (2) JP7767324B2 (https=)
AU (1) AU2021284465A1 (https=)
CA (1) CA3180017A1 (https=)
WO (1) WO2021248085A2 (https=)

Families Citing this family (1)

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Publication number Priority date Publication date Assignee Title
JP2026509974A (ja) * 2023-02-02 2026-03-26 アレン インスティテュート 脊髄内の細胞の遺伝子発現を調節するための人工発現コンストラクト

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020076614A1 (en) 2018-10-08 2020-04-16 Allen Institute Artificial expression constructs for selectively modulating gene expression in interneurons
WO2020097121A1 (en) 2018-11-05 2020-05-14 Allen Institute Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons

Family Cites Families (8)

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Publication number Priority date Publication date Assignee Title
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
WO2001036623A2 (en) * 1999-11-05 2001-05-25 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
US6812339B1 (en) * 2000-09-08 2004-11-02 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
US10113201B2 (en) * 2013-04-05 2018-10-30 The Wistar Institute Of Anatomy And Biology Methods and compositions for diagnosis of glioblastoma or a subtype thereof
WO2015191780A2 (en) * 2014-06-10 2015-12-17 The General Hospital Corporation Ccctc-binding factor (ctcf) rna interactome
AU2019253700B2 (en) * 2018-04-09 2025-08-14 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020076614A1 (en) 2018-10-08 2020-04-16 Allen Institute Artificial expression constructs for selectively modulating gene expression in interneurons
WO2020097121A1 (en) 2018-11-05 2020-05-14 Allen Institute Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
bioRxiv，2020年04月21日，https://doi.org/10.1101/555318

Also Published As

Publication number Publication date
WO2021248085A2 (en) 2021-12-09
EP4162041A4 (en) 2024-10-16
WO2021248085A3 (en) 2022-01-13
JP2026021417A (ja) 2026-02-10
WO2021248085A4 (en) 2022-03-24
JP2023529833A (ja) 2023-07-12
EP4162041A2 (en) 2023-04-12
AU2021284465A1 (en) 2023-02-02
US20230212608A1 (en) 2023-07-06
CA3180017A1 (en) 2021-12-09

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