CA3175993A1 - Artificial expression constructs for selectively modulating gene expression in neocortical layer 5 glutamatergic neurons - Google Patents

Artificial expression constructs for selectively modulating gene expression in neocortical layer 5 glutamatergic neurons Download PDF

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Publication number
CA3175993A1
CA3175993A1 CA3175993A CA3175993A CA3175993A1 CA 3175993 A1 CA3175993 A1 CA 3175993A1 CA 3175993 A CA3175993 A CA 3175993A CA 3175993 A CA3175993 A CA 3175993A CA 3175993 A1 CA3175993 A1 CA 3175993A1
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ehgt
wpre3
bghpa
coding sequence
heterologous coding
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CA3175993A
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French (fr)
Inventor
Tanya DAIGLE
Lucas T. GRAYBUCK
Brian Edward KALMBACH
Edward Sebastian LEIN
Boaz P. LEVI
John K. Mich
Adriana Estela SEDENO CORTES
Bosiljka Tasic
Jonathan Ting
Hongkui Zeng
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Allen Institute
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Allen Institute
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    • 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
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    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • 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
    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • 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)
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  • Neurosurgery (AREA)
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  • 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)
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CA3175993A 2020-04-21 2021-04-21 Artificial expression constructs for selectively modulating gene expression in neocortical layer 5 glutamatergic neurons Pending CA3175993A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3175993A1 true CA3175993A1 (en) 2021-10-28

Family

ID=78269916

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3175993A Pending CA3175993A1 (en) 2020-04-21 2021-04-21 Artificial expression constructs for selectively modulating gene expression in neocortical layer 5 glutamatergic neurons

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

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Publication number Publication date
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
US20230159952A1 (en) 2023-05-25
KR20230002788A (ko) 2023-01-05
EP4139462A2 (en) 2023-03-01
WO2021216778A3 (en) 2021-12-16
JP7721568B2 (ja) 2025-08-12

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