AU2021261359A1 - 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

Info

Publication number
AU2021261359A1
AU2021261359A1 AU2021261359A AU2021261359A AU2021261359A1 AU 2021261359 A1 AU2021261359 A1 AU 2021261359A1 AU 2021261359 A AU2021261359 A AU 2021261359A AU 2021261359 A AU2021261359 A AU 2021261359A AU 2021261359 A1 AU2021261359 A1 AU 2021261359A1
Authority
AU
Australia
Prior art keywords
ehgt
wpre3
bghpa
coding sequence
heterologous coding
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
AU2021261359A
Other languages
English (en)
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
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Allen Institute
Original Assignee
Allen Institute
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Allen Institute filed Critical Allen Institute
Publication of AU2021261359A1 publication Critical patent/AU2021261359A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/63—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
    • A61K48/0066—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
    • 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
    • C12N15/63—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
    • 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
    • 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
    • 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
    • C12N2830/008—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Epidemiology (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)
AU2021261359A 2020-04-21 2021-04-21 Artificial expression constructs for selectively modulating gene expression in neocortical layer 5 glutamatergic neurons Pending AU2021261359A1 (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
AU2021261359A1 true AU2021261359A1 (en) 2022-12-08

Family

ID=78269916

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021261359A Pending AU2021261359A1 (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 (https=)
EP (1) EP4139462A4 (https=)
JP (2) JP7721568B2 (https=)
KR (1) KR20230002788A (https=)
AU (1) AU2021261359A1 (https=)
CA (1) CA3175993A1 (https=)
WO (1) WO2021216778A2 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4413142A4 (en) * 2021-10-05 2025-11-05 Allen Inst ARTIFICIAL EXPRESSION CONSTRUCTIONS TO MODULATE GENE EXPRESSION IN THE CEREBELLUM AND IN A SECONDARY CELL TYPE
JP2025520385A (ja) * 2022-06-13 2025-07-03 アレン インスティテュート 中枢神経系の非神経細胞において遺伝子発現を調節するための人工発現コンストラクト
WO2025059552A1 (en) * 2023-09-14 2025-03-20 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
US10973930B2 (en) * 2016-02-18 2021-04-13 The Penn State Research Foundation Generating GABAergic neurons in brains
WO2020076614A1 (en) * 2018-10-08 2020-04-16 Allen Institute Artificial expression constructs for selectively modulating gene expression in interneurons
AU2019357039B2 (en) * 2018-10-12 2025-10-09 Genzyme Corporation Generation of improved human pah for treatment of severe PKU by liver-directed gene replacement therapy
US20220249703A1 (en) * 2019-02-15 2022-08-11 Allen Institute Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations

Also Published As

Publication number Publication date
US20230159952A1 (en) 2023-05-25
JP7721568B2 (ja) 2025-08-12
CA3175993A1 (en) 2021-10-28
WO2021216778A2 (en) 2021-10-28
EP4139462A2 (en) 2023-03-01
WO2021216778A3 (en) 2021-12-16
JP2025163106A (ja) 2025-10-28
EP4139462A4 (en) 2025-03-12
JP2023522708A (ja) 2023-05-31
KR20230002788A (ko) 2023-01-05

Similar Documents

Publication Publication Date Title
JP7721568B2 (ja) 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
US20230117172A1 (en) Artificial expression constructs for selectively modulating gene expression in non-neuronal brain cells
US20250235550A1 (en) Artificial expression constructs for modulating gene expression in the cerebellum and a secondary cell type
JP2026021417A (ja) 抑制性新皮質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
US20240254514A1 (en) Artificial expression constructs for modulating gene expression in neurons within the thalamus
US20240182923A1 (en) Artificial expression constructs for modulating gene expression in claustrum neurons
US20240018543A1 (en) Artificial expression constructs for modulating gene expression in chandelier cells
AU2023293067A1 (en) Artificial expression constructs for modulating gene expression in non-neuronal central nervous system cells
US20250041454A1 (en) Artificial expression constructs for modulating gene expression in neocortical layer 4 or layer 5 intratelencephalic neurons
WO2025179180A1 (en) Artificial expression constructs for modulating gene expression in motor neurons and cerebellar neurons
AU2024342927A1 (en) Artificial expression constructs for modulating gene expression in the basal ganglia
CA3244907A1 (en) ARTIFICIAL EXPRESSION CONSTRUCTIONS TO MODULATE GENE EXPRESSION IN DOPAMINERGIC NEURONS