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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ehgt
sst
visp
vip
cells
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JP2023529833A5 (enExample
JP2023529833A (ja
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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)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Dermatology (AREA)
  • Virology (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 (3)

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JP2023529833A JP2023529833A (ja) 2023-07-12
JP2023529833A5 JP2023529833A5 (enExample) 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) US12692515B2 (enExample)
EP (1) EP4162041A4 (enExample)
JP (2) JP7767324B2 (enExample)
AU (1) AU2021284465A1 (enExample)
CA (1) CA3180017A1 (enExample)
WO (1) WO2021248085A2 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4507785A4 (en) * 2022-04-11 2026-06-03 Broad Inst Inc Activators for directed gene expression in neural cell populations, compositions and associated processes
US20260226502A1 (en) * 2023-02-02 2026-08-06 Allen Institute Artificial expression constructs for modulating gene expression in cells within the spinal cord

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 (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5851832A (en) 1991-07-08 1998-12-22 Neurospheres, Ltd. In vitro growth and proliferation of multipotent neural stem cells and their progeny
US5766948A (en) 1993-01-06 1998-06-16 The Regents Of The University Of California Method for production of neuroblasts
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US6001654A (en) 1994-01-28 1999-12-14 California Institute Of Technology Methods for differentiating neural stem cells to neurons or smooth muscle cells using TGT-β super family growth factors
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US6048964A (en) 1995-12-12 2000-04-11 Stryker Corporation Compositions and therapeutic methods using morphogenic proteins and stimulatory factors
US5753506A (en) 1996-05-23 1998-05-19 Cns Stem Cell Technology, Inc. Isolation propagation and directed differentiation of stem cells from embryonic and adult central nervous system of mammals
AU3642601A (en) 1999-11-05 2001-05-30 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
US6395546B1 (en) 2000-02-01 2002-05-28 Neurogeneration, Inc. Generation of dopaminergic neurons from human nervous system stem cells
US6812339B1 (en) * 2000-09-08 2004-11-02 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
EP1430114B1 (en) 2001-09-14 2012-01-18 Stem Cell Therapeutics Inc. Prolactin induced increase in neural stem cell numbers and therapeutical use thereof
US20120308530A1 (en) 2011-06-03 2012-12-06 Beijing Allgens Medical Science & Technology Co., Ltd. Materials Composition and Methods to Control Neural Progenitor and Stem Cell Attachment, Proliferation and Guide Cell Differentiation
WO2014165753A1 (en) * 2013-04-05 2014-10-09 The Wistar Institute Of Anatomy And Biology Methods and compositions for diagnosis of glioblastoma or a subtype thereof
US20150376612A1 (en) * 2014-06-10 2015-12-31 The General Hospital Corporation CCCTC-Binding Factor (CTCF) RNA Interactome
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
KR20260004568A (ko) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
AU2017302551B2 (en) 2016-07-26 2023-04-27 The General Hospital Corporation Variants of CRISPR from Prevotella and Francisella 1 (Cpf1)
EP3781174A4 (en) 2018-04-09 2022-01-26 Allen Institute RESCUE OF VOLTAGE CONTROLLED 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
JP2026021417A (ja) 2026-02-10
EP4162041A2 (en) 2023-04-12
US12692515B2 (en) 2026-07-28
CA3180017A1 (en) 2021-12-09
US20230212608A1 (en) 2023-07-06
WO2021248085A3 (en) 2022-01-13
JP2023529833A (ja) 2023-07-12
AU2021284465A1 (en) 2023-02-02
WO2021248085A4 (en) 2022-03-24
EP4162041A4 (en) 2024-10-16
WO2021248085A2 (en) 2021-12-09

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