CA3173609A1 - Artificial expression constructs for selectively modulating gene expression in non-neuronal brain cells - Google Patents

Artificial expression constructs for selectively modulating gene expression in non-neuronal brain cells Download PDF

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Publication number
CA3173609A1
CA3173609A1 CA3173609A CA3173609A CA3173609A1 CA 3173609 A1 CA3173609 A1 CA 3173609A1 CA 3173609 A CA3173609 A CA 3173609A CA 3173609 A CA3173609 A CA 3173609A CA 3173609 A1 CA3173609 A1 CA 3173609A1
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Prior art keywords
ehgt
bghpa
minbglobin
syfp2
wpre3
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Pending
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CA3173609A
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English (en)
French (fr)
Inventor
Jonathan Ting
Bosiljka Tasic
Boaz P. LEVI
Tanya DAIGLE
Lucas T. GRAYBUCK
Edward Sebastian LEIN
John K. Mich
Adriana Estela SEDENO CORTES
Hongkui Zeng
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Allen Institute
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Allen Institute
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Publication of CA3173609A1 publication Critical patent/CA3173609A1/en
Pending legal-status Critical Current

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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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6897—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids involving reporter genes operably linked to promoters
    • 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
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3173609A 2020-03-27 2021-03-26 Artificial expression constructs for selectively modulating gene expression in non-neuronal brain cells Pending CA3173609A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063001159P 2020-03-27 2020-03-27
US63/001,159 2020-03-27
PCT/US2021/024525 WO2021195591A2 (en) 2020-03-27 2021-03-26 Artificial expression constructs for selectively modulating gene expression in non-neuronal brain cells

Publications (1)

Publication Number Publication Date
CA3173609A1 true CA3173609A1 (en) 2021-09-30

Family

ID=77890746

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3173609A Pending CA3173609A1 (en) 2020-03-27 2021-03-26 Artificial expression constructs for selectively modulating gene expression in non-neuronal brain cells

Country Status (6)

Country Link
US (1) US20230117172A1 (https=)
EP (1) EP4127182A4 (https=)
JP (2) JP7749577B2 (https=)
AU (1) AU2021242353A1 (https=)
CA (1) CA3173609A1 (https=)
WO (1) WO2021195591A2 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2022217778A1 (en) * 2021-02-02 2023-08-31 Allen Institute Artificial expression constructs for modulating gene expression in gabaergic neurons and astrocytes
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

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6602680B2 (en) * 2000-07-14 2003-08-05 The Regents Of The University Of California Production of gabaergic cells
WO2003093479A1 (en) * 2002-05-01 2003-11-13 University Of Florida Research Foundation, Inc. Improved raav expression systems and methods for enhancing transduction of mammalian neural cells
US20080039413A1 (en) * 2003-10-21 2008-02-14 Morris David W Novel compositions and methods in cancer
CA2722238C (en) * 2008-04-22 2017-11-28 Life Sciences Research Partners Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
WO2013155222A2 (en) * 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
EP4653536A3 (en) * 2017-03-17 2026-02-25 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
WO2019068854A1 (en) * 2017-10-06 2019-04-11 Ospedale San Raffaele S.R.L. GENE THERAPY OF NEURODEGENERATIVE DISEASES USING VAA VECTORS
US11364309B2 (en) * 2017-11-10 2022-06-21 Norwegian University Of Science And Technology (Ntnu) Neuronal enhancers
CA3096407A1 (en) * 2018-04-09 2019-10-17 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons

Also Published As

Publication number Publication date
JP7749577B2 (ja) 2025-10-06
EP4127182A4 (en) 2024-07-31
US20230117172A1 (en) 2023-04-20
JP2023518980A (ja) 2023-05-09
JP2026001073A (ja) 2026-01-06
EP4127182A2 (en) 2023-02-08
AU2021242353A1 (en) 2022-11-03
WO2021195591A3 (en) 2021-11-25
WO2021195591A2 (en) 2021-09-30

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