JP7721568B2 - 新皮質層５グルタミン酸作動性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

新皮質層５グルタミン酸作動性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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JP7721568B2
JP7721568B2 JP2022563930A JP2022563930A JP7721568B2 JP 7721568 B2 JP7721568 B2 JP 7721568B2 JP 2022563930 A JP2022563930 A JP 2022563930A JP 2022563930 A JP2022563930 A JP 2022563930A JP 7721568 B2 JP7721568 B2 JP 7721568B2
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sequence
artificial
coding sequence
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JP2023522708A5 (enExample
JP2023522708A (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
カルムバック，ブライアン・エドワード
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
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
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    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
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  • Physics & Mathematics (AREA)
  • Virology (AREA)
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  • Neurosurgery (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Prostheses (AREA)
JP2022563930A 2020-04-21 2021-04-21 新皮質層５グルタミン酸作動性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 JP7721568B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025127056A JP2025163106A (ja) 2020-04-21 2025-07-30 新皮質層５グルタミン酸作動性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
US202063013342P 2020-04-21 2020-04-21
US63/013,342 2020-04-21
PCT/US2021/028489 WO2021216778A2 (en) 2020-04-21 2021-04-21 Artificial expression constructs for selectively modulating gene expression in neocortical layer 5 glutamatergic neurons

Related Child Applications (1)

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JP2025127056A Division JP2025163106A (ja) 2020-04-21 2025-07-30 新皮質層５グルタミン酸作動性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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JP2023522708A JP2023522708A (ja) 2023-05-31
JP2023522708A5 JP2023522708A5 (enExample) 2024-02-27
JP7721568B2 true JP7721568B2 (ja) 2025-08-12

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JP2025127056A Pending JP2025163106A (ja) 2020-04-21 2025-07-30 新皮質層５グルタミン酸作動性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 (7)

Country Link
US (1) US20230159952A1 (enExample)
EP (1) EP4139462A4 (enExample)
JP (2) JP7721568B2 (enExample)
KR (1) KR20230002788A (enExample)
AU (1) AU2021261359A1 (enExample)
CA (1) CA3175993A1 (enExample)
WO (1) WO2021216778A2 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3233342A1 (en) * 2021-10-05 2023-04-13 Tanya DAIGLE Artificial expression constructs for modulating gene expression in the cerebellum and a secondary cell type
EP4507785A4 (en) * 2022-04-11 2026-06-03 Broad Inst Inc Activators for directed gene expression in neural cell populations, compositions and associated processes
EP4536835A2 (en) * 2022-06-13 2025-04-16 Allen Institute Artificial expression constructs for modulating gene expression in non-neuronal central nervous system cells
AU2024342927A1 (en) * 2023-09-14 2026-04-09 Allen Institute Artificial expression constructs for modulating gene expression in the basal ganglia
WO2025179180A1 (en) * 2024-02-23 2025-08-28 Allen Institute Artificial expression constructs for modulating gene expression in motor neurons and cerebellar neurons

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9920317B2 (en) * 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
EP3416663B1 (en) * 2016-02-18 2021-04-14 The Penn State Research Foundation Generating gabaergic neurons in brains
US12467064B2 (en) * 2018-10-08 2025-11-11 Allen Institute Artificial expression constructs for selectively modulating gene expression in interneurons
CN120349985A (zh) * 2018-10-12 2025-07-22 建新公司 通过肝导向基因替代疗法治疗严重pku的改良的人pah的产生
CA3142948A1 (en) * 2019-02-15 2020-08-20 Allen Institute Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
GRAYBUCK Lucas T. et al.，Prospective, brain-wide labeling of neuronal subclasses with enhancer-driven AAVs，bioRiv，2019年01月31日，<URL: https://www.biorxiv.org/content/10.1101/525014v2>
Mouse DNA sequence from clone RP23-29H22 on chromosome 4, complete sequence，GenBank, Accession No. AL606962，[retrived on 2025.02.18]，2012年，<URL: https://www.ncbi.nlm.nih.gov/nucleotide/AL606962.22?re port=genbank&log$=nuclalign&blast_rank=1&RID=V8JCSC6G013>

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EP4139462A4 (en) 2025-03-12
JP2025163106A (ja) 2025-10-28
WO2021216778A2 (en) 2021-10-28
AU2021261359A1 (en) 2022-12-08
JP2023522708A (ja) 2023-05-31
CA3175993A1 (en) 2021-10-28
US20230159952A1 (en) 2023-05-25
KR20230002788A (ko) 2023-01-05
EP4139462A2 (en) 2023-03-01
WO2021216778A3 (en) 2021-12-16

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