AU2020223367B2 - Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations - Google Patents
Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populationsInfo
- Publication number
- AU2020223367B2 AU2020223367B2 AU2020223367A AU2020223367A AU2020223367B2 AU 2020223367 B2 AU2020223367 B2 AU 2020223367B2 AU 2020223367 A AU2020223367 A AU 2020223367A AU 2020223367 A AU2020223367 A AU 2020223367A AU 2020223367 B2 AU2020223367 B2 AU 2020223367B2
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- ehgt
- neuron
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- cells
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- 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
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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- 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
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- 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
- A01K67/0276—Knock-out vertebrates
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- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
- C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
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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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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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
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
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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
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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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- 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
- A01K2217/00—Genetically modified animals
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- 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
- A01K2217/00—Genetically modified animals
- A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
- A01K2217/054—Animals comprising random inserted nucleic acids (transgenic) inducing loss of function
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- 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
- A01K2217/00—Genetically modified animals
- A01K2217/20—Animal model comprising regulated expression system
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- 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
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- 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
- A01K2227/106—Primate
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- 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
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
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- 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
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0393—Animal model comprising a reporter system for screening tests
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
- C12N2015/8527—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic for producing animal models, e.g. for tests or diseases
- C12N2015/859—Animal models comprising reporter system for screening tests
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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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
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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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- C12N2830/00—Vector systems having a special element relevant for transcription
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- Animal Behavior & Ethology (AREA)
- Neurology (AREA)
- Animal Husbandry (AREA)
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- Epidemiology (AREA)
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- Proteomics, Peptides & Aminoacids (AREA)
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Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962806686P | 2019-02-15 | 2019-02-15 | |
| US201962806660P | 2019-02-15 | 2019-02-15 | |
| US62/806,686 | 2019-02-15 | ||
| US62/806,660 | 2019-02-15 | ||
| US201962874859P | 2019-07-16 | 2019-07-16 | |
| US62/874,859 | 2019-07-16 | ||
| PCT/US2020/018416 WO2020168279A2 (en) | 2019-02-15 | 2020-02-14 | Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2020223367A1 AU2020223367A1 (en) | 2021-10-07 |
| AU2020223367B2 true AU2020223367B2 (en) | 2026-06-11 |
Family
ID=72044145
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2020223367A Active AU2020223367B2 (en) | 2019-02-15 | 2020-02-14 | Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US12702722B2 (enExample) |
| EP (1) | EP3923995A4 (enExample) |
| JP (1) | JP7608348B2 (enExample) |
| AU (1) | AU2020223367B2 (enExample) |
| CA (1) | CA3142948A1 (enExample) |
| WO (1) | WO2020168279A2 (enExample) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| CA3142948A1 (en) | 2019-02-15 | 2020-08-20 | Allen Institute | Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations |
| JP7721568B2 (ja) * | 2020-04-21 | 2025-08-12 | アレン インスティテュート | 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 |
| EP4244366A4 (en) * | 2020-11-10 | 2024-12-25 | Allen Institute | Artificial expression constructs for modulating gene expression in chandelier cells |
| US20240182923A1 (en) * | 2021-03-30 | 2024-06-06 | Allen Institute | Artificial expression constructs for modulating gene expression in claustrum neurons |
| AU2022276538A1 (en) * | 2021-05-21 | 2023-11-09 | Allen Institute | Artificial expression constructs for modulating gene expression in neurons within the thalamus |
| 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 |
| EP4735613A1 (en) * | 2023-06-28 | 2026-05-06 | Allen Institute | Artificial expression constructs for modulating gene expression in cardiomyocytes |
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| DE69918090T2 (de) * | 1998-04-24 | 2005-06-16 | University Of Florida, Gainesville | Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie |
| US20070037165A1 (en) | 2000-09-08 | 2007-02-15 | Applera Corporation | Polymorphisms in known genes associated with human disease, methods of detection and uses thereof |
| EP2028268A1 (en) | 2007-08-20 | 2009-02-25 | Université Libre De Bruxelles | Generation of neuronal cells from pluripotent stem cells |
| EP3702460A1 (en) * | 2010-11-12 | 2020-09-02 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| US9284575B2 (en) * | 2012-03-06 | 2016-03-15 | Duke University | Synthetic regulation of gene expression |
| WO2013155222A2 (en) | 2012-04-10 | 2013-10-17 | The Regents Of The University Of California | Brain-specific enhancers for cell-based therapy |
| US9579399B2 (en) | 2012-08-27 | 2017-02-28 | Friedrich Miescher Institute For Biomedical Research | Retinal OFF circuit-specific promoter |
| EP2922972B1 (en) | 2012-11-26 | 2017-04-26 | Ecole Polytechnique Fédérale de Lausanne (EPFL) | Colorectal cancer classification with differential prognosis and personalized therapeutic responses |
| US20150376612A1 (en) | 2014-06-10 | 2015-12-31 | The General Hospital Corporation | CCCTC-Binding Factor (CTCF) RNA Interactome |
| CL2014003590A1 (es) | 2014-12-30 | 2015-07-10 | Univ Chile | Virus aav/xbp1s-ha, método de tratamiento genético y su uso en la optimizacion y mejoramiento de las capacidades cognitivas, de memoria y de aprendizaje. |
| US10682424B2 (en) | 2015-03-31 | 2020-06-16 | New York University | Compositions and method for reducing seizures |
| US9790490B2 (en) * | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| AU2016366549B2 (en) * | 2015-12-11 | 2022-11-10 | California Institute Of Technology | Targeting peptides for directing adeno-associated viruses (AAVs) |
| EP3416663B1 (en) * | 2016-02-18 | 2021-04-14 | The Penn State Research Foundation | Generating gabaergic neurons in brains |
| CN117821509A (zh) | 2017-04-03 | 2024-04-05 | 编码治疗公司 | 组织选择性转基因表达 |
| 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 |
| 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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2020
- 2020-02-14 CA CA3142948A patent/CA3142948A1/en active Pending
- 2020-02-14 US US17/431,079 patent/US12702722B2/en active Active
- 2020-02-14 EP EP20756018.6A patent/EP3923995A4/en active Pending
- 2020-02-14 JP JP2021547404A patent/JP7608348B2/ja active Active
- 2020-02-14 AU AU2020223367A patent/AU2020223367B2/en active Active
- 2020-02-14 WO PCT/US2020/018416 patent/WO2020168279A2/en not_active Ceased
Non-Patent Citations (2)
| Title |
|---|
| DIMIDSCHSTEIN JORDANE, CHEN QIAN, TREMBLAY ROBIN, ROGERS STEPHANIE L, SALDI GIUSEPPE-ANTONIO, GUO LIHUA, XU QING, LIU RUNPENG, LU : "A viral strategy for targeting and manipulating interneurons across vertebrate species", NATURE NEUROSCIENCE, NATURE PUBLISHING GROUP US, NEW YORK, vol. 19, no. 12, 1 December 2016 (2016-12-01), New York, pages 1743 - 1749, XP055872737, ISSN: 1097-6256, DOI: 10.1038/nn.4430 * |
| GRAYBUCK LUCAS T., ADRIANA E. SEDEÑO-CORTÉS, THUC NGHI NGUYEN, MIRANDA WALKER, ERIC SZELENYI, GARRECK LENZ, LA’AKEA SIEVERTS, TAE : "Prospective, brain-wide labeling of neuronal subclasses with enhancer-driven AAVs", BIORXIV, 20 January 2019 (2019-01-20), XP055940913, Retrieved from the Internet <URL:https://web.archive.org/web/20190430183217id_/https://www.biorxiv.org/content/biorxiv/early/2019/01/31/525014.full.pdf> [retrieved on 20220711], DOI: 10.1101/525014 * |
Also Published As
| Publication number | Publication date |
|---|---|
| JP2022520437A (ja) | 2022-03-30 |
| US12702722B2 (en) | 2026-08-11 |
| EP3923995A2 (en) | 2021-12-22 |
| WO2020168279A2 (en) | 2020-08-20 |
| JP7608348B2 (ja) | 2025-01-06 |
| EP3923995A4 (en) | 2023-05-17 |
| AU2020223367A1 (en) | 2021-10-07 |
| WO2020168279A3 (en) | 2020-09-24 |
| JP2025041709A (ja) | 2025-03-26 |
| CA3142948A1 (en) | 2020-08-20 |
| US20220249703A1 (en) | 2022-08-11 |