AU2021284465A1 - Artificial expression constructs for selectively modulating gene expression in inhibitory neocortical neurons - Google Patents

Artificial expression constructs for selectively modulating gene expression in inhibitory neocortical neurons Download PDF

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Publication number
AU2021284465A1
AU2021284465A1 AU2021284465A AU2021284465A AU2021284465A1 AU 2021284465 A1 AU2021284465 A1 AU 2021284465A1 AU 2021284465 A AU2021284465 A AU 2021284465A AU 2021284465 A AU2021284465 A AU 2021284465A AU 2021284465 A1 AU2021284465 A1 AU 2021284465A1
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ehgt
mgt
expression construct
vector
molecule
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Tanya DAIGLE
Lucas T. GRAYBUCK
Edward Sebastian LEIN
Boaz P. LEVI
John K. Mich
Adriana Estela SEDENO CORTES
Bosiljka Tasic
Jonathan Ting
Miranda WALKER
Hongkui Zeng
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Allen Institute
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Allen Institute
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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
    • 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
    • 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
    • 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
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    • 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
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    • 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
    • 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
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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
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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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
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  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
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  • Virology (AREA)
  • Neurology (AREA)
  • Dermatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
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  • 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)
AU2021284465A 2020-06-04 2021-06-04 Artificial expression constructs for selectively modulating gene expression in inhibitory neocortical neurons Pending AU2021284465A1 (en)

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

Publications (1)

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AU2021284465A1 true AU2021284465A1 (en) 2023-02-02

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AU2021284465A Pending AU2021284465A1 (en) 2020-06-04 2021-06-04 Artificial expression constructs for selectively modulating gene expression in inhibitory neocortical neurons

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=)

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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

Family Cites Families (10)

* Cited by examiner, † Cited by third party
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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
JP7695192B2 (ja) 2018-10-08 2025-06-18 アレン インスティテュート 介在ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物
US20210395780A1 (en) * 2018-11-05 2021-12-23 Allen Institute Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons

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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
JP7767324B2 (ja) 2025-11-11
JP2023529833A (ja) 2023-07-12
EP4162041A2 (en) 2023-04-12
US20230212608A1 (en) 2023-07-06
CA3180017A1 (en) 2021-12-09

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