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
    • 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
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    • 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

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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)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • Medicinal Chemistry (AREA)
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  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
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  • Virology (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)
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

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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

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US (1) US12692515B2 (enExample)
EP (1) EP4162041A4 (enExample)
JP (2) JP7767324B2 (enExample)
AU (1) AU2021284465A1 (enExample)
CA (1) CA3180017A1 (enExample)
WO (1) WO2021248085A2 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4507785A4 (en) * 2022-04-11 2026-06-03 Broad Inst Inc Activators for directed gene expression in neural cell populations, compositions and associated processes
US20260226502A1 (en) * 2023-02-02 2026-08-06 Allen Institute Artificial expression constructs for modulating gene expression in cells within the spinal cord

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US5766948A (en) 1993-01-06 1998-06-16 The Regents Of The University Of California Method for production of neuroblasts
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US6001654A (en) 1994-01-28 1999-12-14 California Institute Of Technology Methods for differentiating neural stem cells to neurons or smooth muscle cells using TGT-β super family growth factors
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US6048964A (en) 1995-12-12 2000-04-11 Stryker Corporation Compositions and therapeutic methods using morphogenic proteins and stimulatory factors
US5753506A (en) 1996-05-23 1998-05-19 Cns Stem Cell Technology, Inc. Isolation propagation and directed differentiation of stem cells from embryonic and adult central nervous system of mammals
AU3642601A (en) 1999-11-05 2001-05-30 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
US6395546B1 (en) 2000-02-01 2002-05-28 Neurogeneration, Inc. Generation of dopaminergic neurons from human nervous system stem cells
US6812339B1 (en) * 2000-09-08 2004-11-02 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
EP1430114B1 (en) 2001-09-14 2012-01-18 Stem Cell Therapeutics Inc. Prolactin induced increase in neural stem cell numbers and therapeutical use thereof
US20120308530A1 (en) 2011-06-03 2012-12-06 Beijing Allgens Medical Science & Technology Co., Ltd. Materials Composition and Methods to Control Neural Progenitor and Stem Cell Attachment, Proliferation and Guide Cell Differentiation
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
US20150376612A1 (en) * 2014-06-10 2015-12-31 The General Hospital Corporation CCCTC-Binding Factor (CTCF) RNA Interactome
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
KR20260004568A (ko) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
AU2017302551B2 (en) 2016-07-26 2023-04-27 The General Hospital Corporation Variants of CRISPR from Prevotella and Francisella 1 (Cpf1)
EP3781174A4 (en) 2018-04-09 2022-01-26 Allen Institute RESCUE OF VOLTAGE CONTROLLED SODIUM CHANNEL FUNCTION IN INHIBITORY NEURONS
US12467064B2 (en) * 2018-10-08 2025-11-11 Allen Institute Artificial expression constructs for selectively modulating gene expression in interneurons
JP7590962B2 (ja) * 2018-11-05 2024-11-27 アレン インスティテュート 皮質興奮性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

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JP2026021417A (ja) 2026-02-10
EP4162041A2 (en) 2023-04-12
US12692515B2 (en) 2026-07-28
CA3180017A1 (en) 2021-12-09
US20230212608A1 (en) 2023-07-06
WO2021248085A3 (en) 2022-01-13
JP7767324B2 (ja) 2025-11-11
JP2023529833A (ja) 2023-07-12
WO2021248085A4 (en) 2022-03-24
EP4162041A4 (en) 2024-10-16
WO2021248085A2 (en) 2021-12-09

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