CA3180017A1 - 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
CA3180017A1
CA3180017A1 CA3180017A CA3180017A CA3180017A1 CA 3180017 A1 CA3180017 A1 CA 3180017A1 CA 3180017 A CA3180017 A CA 3180017A CA 3180017 A CA3180017 A CA 3180017A CA 3180017 A1 CA3180017 A1 CA 3180017A1
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Prior art keywords
ehgt
mgt
expression construct
vector
molecule
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CA3180017A
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English (en)
French (fr)
Inventor
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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Publication of CA3180017A1 publication Critical patent/CA3180017A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • 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)
CA3180017A 2020-06-04 2021-06-04 Artificial expression constructs for selectively modulating gene expression in inhibitory neocortical neurons Pending CA3180017A1 (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)

Publication Number Publication Date
CA3180017A1 true CA3180017A1 (en) 2021-12-09

Family

ID=78831739

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3180017A Pending CA3180017A1 (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=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20250142388A (ko) * 2023-02-02 2025-09-30 알렌 인스티튜트 척수 내의 세포에서 유전자 발현을 조정하기 위한 인공 발현 구축물

Family Cites Families (10)

* 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
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
AU3642601A (en) * 1999-11-05 2001-05-30 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
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
WO2015191780A2 (en) * 2014-06-10 2015-12-17 The General Hospital Corporation Ccctc-binding factor (ctcf) rna interactome
CA3096407A1 (en) 2018-04-09 2019-10-17 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons
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

Also Published As

Publication number Publication date
JP7767324B2 (ja) 2025-11-11
EP4162041A4 (en) 2024-10-16
EP4162041A2 (en) 2023-04-12
JP2026021417A (ja) 2026-02-10
WO2021248085A3 (en) 2022-01-13
WO2021248085A4 (en) 2022-03-24
JP2023529833A (ja) 2023-07-12
WO2021248085A2 (en) 2021-12-09
AU2021284465A1 (en) 2023-02-02
US20230212608A1 (en) 2023-07-06

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