CA3219142A1 - Artificial expression constructs for modulating gene expression in neurons within the thalamus - Google Patents

Artificial expression constructs for modulating gene expression in neurons within the thalamus Download PDF

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Publication number
CA3219142A1
CA3219142A1 CA3219142A CA3219142A CA3219142A1 CA 3219142 A1 CA3219142 A1 CA 3219142A1 CA 3219142 A CA3219142 A CA 3219142A CA 3219142 A CA3219142 A CA 3219142A CA 3219142 A1 CA3219142 A1 CA 3219142A1
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Canada
Prior art keywords
ehgt
seq
coding sequence
sequence
heterologous coding
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Pending
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CA3219142A
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English (en)
French (fr)
Inventor
Tanya DAIGLE
Edward Sebastian LEIN
Boaz P. LEVI
John K. Mich
Amanda MITCHELL
Bosiljka Tasic
Jonathan Ting
Natalie WEED
Hongkui Zeng
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Allen Institute
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Allen Institute
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Publication of CA3219142A1 publication Critical patent/CA3219142A1/en
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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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0619—Neurons
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/12—Animals modified by administration of exogenous cells
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
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    • 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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    • 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
    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
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  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
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  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurosurgery (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3219142A 2021-05-21 2022-05-20 Artificial expression constructs for modulating gene expression in neurons within the thalamus Pending CA3219142A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163191832P 2021-05-21 2021-05-21
US63/191,832 2021-05-21
PCT/US2022/030371 WO2022246276A2 (en) 2021-05-21 2022-05-20 Artificial expression constructs for modulating gene expression in neurons within the thalamus

Publications (1)

Publication Number Publication Date
CA3219142A1 true CA3219142A1 (en) 2022-11-24

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ID=84141881

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3219142A Pending CA3219142A1 (en) 2021-05-21 2022-05-20 Artificial expression constructs for modulating gene expression in neurons within the thalamus

Country Status (6)

Country Link
US (1) US20240254514A1 (https=)
EP (1) EP4341410A4 (https=)
JP (1) JP2024519955A (https=)
AU (1) AU2022276538A1 (https=)
CA (1) CA3219142A1 (https=)
WO (1) WO2022246276A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023060112A1 (en) * 2021-10-05 2023-04-13 Allen Institute Artificial expression constructs for modulating gene expression in the cerebellum and a secondary cell type

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3052290A1 (en) * 2017-02-10 2018-08-16 The Rockefeller University Methods for cell-type specific profiling to identify drug targets
US20210395780A1 (en) * 2018-11-05 2021-12-23 Allen Institute Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons
EP3923995A4 (en) * 2019-02-15 2023-05-17 Allen Institute ARTIFICIAL EXPRESSION RECOMBINANTS FOR THE SELECTIVE MODULATION OF GENE EXPRESSION IN SELECTED NEURON CELL POPULATIONS
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

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Publication number Publication date
AU2022276538A1 (en) 2023-11-09
WO2022246276A3 (en) 2022-12-29
JP2024519955A (ja) 2024-05-21
US20240254514A1 (en) 2024-08-01
EP4341410A4 (en) 2026-02-11
WO2022246276A2 (en) 2022-11-24
AU2022276538A9 (en) 2023-11-16
EP4341410A2 (en) 2024-03-27

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