CA3142948A1 - 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 populations Download PDFInfo
- Publication number
- CA3142948A1 CA3142948A1 CA3142948A CA3142948A CA3142948A1 CA 3142948 A1 CA3142948 A1 CA 3142948A1 CA 3142948 A CA3142948 A CA 3142948A CA 3142948 A CA3142948 A CA 3142948A CA 3142948 A1 CA3142948 A1 CA 3142948A1
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- expression construct
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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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- 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
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- 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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- C12N15/09—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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- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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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- 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
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- 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
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- 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
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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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- 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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- C12N2750/00011—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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- 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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- Veterinary Medicine (AREA)
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- Environmental Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Virology (AREA)
- Neurology (AREA)
- Biodiversity & Conservation Biology (AREA)
- Animal Husbandry (AREA)
- Immunology (AREA)
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- Public Health (AREA)
- Epidemiology (AREA)
- Neurosurgery (AREA)
- Pharmacology & Pharmacy (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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 | |
| US62/806,660 | 2019-02-15 | ||
| US62/806,686 | 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 (1)
| Publication Number | Publication Date |
|---|---|
| CA3142948A1 true CA3142948A1 (en) | 2020-08-20 |
Family
ID=72044145
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3142948A Pending CA3142948A1 (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) | US20220249703A1 (https=) |
| EP (1) | EP3923995A4 (https=) |
| JP (2) | JP7608348B2 (https=) |
| AU (1) | AU2020223367A1 (https=) |
| CA (1) | CA3142948A1 (https=) |
| WO (1) | WO2020168279A2 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2021261359A1 (en) * | 2020-04-21 | 2022-12-08 | Allen Institute | Artificial expression constructs for selectively modulating gene expression in neocortical layer 5 glutamatergic neurons |
| WO2022103859A1 (en) * | 2020-11-10 | 2022-05-19 | Allen Institute | Artificial expression constructs for modulating gene expression in chandelier cells |
| WO2022212585A1 (en) * | 2021-03-30 | 2022-10-06 | Allen Institute | Artificial expression constructs for modulating gene expression in claustrum neurons |
| WO2022246276A2 (en) * | 2021-05-21 | 2022-11-24 | Allen Institute | Artificial expression constructs for modulating gene expression in neurons within the thalamus |
| 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 |
| AU2024308266A1 (en) * | 2023-06-28 | 2025-12-18 | Allen Institute | Artificial expression constructs for modulating gene expression in cardiomyocytes |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| CA2817256A1 (en) * | 2010-11-12 | 2012-05-18 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| AU2013201287B2 (en) * | 2012-03-06 | 2015-05-14 | 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 |
| US9790490B2 (en) * | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| KR102423442B1 (ko) | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| US10973930B2 (en) * | 2016-02-18 | 2021-04-13 | The Penn State Research Foundation | Generating GABAergic neurons in brains |
| WO2020097121A1 (en) * | 2018-11-05 | 2020-05-14 | Allen Institute | Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons |
-
2020
- 2020-02-14 US US17/431,079 patent/US20220249703A1/en active Pending
- 2020-02-14 CA CA3142948A patent/CA3142948A1/en active Pending
- 2020-02-14 WO PCT/US2020/018416 patent/WO2020168279A2/en not_active Ceased
- 2020-02-14 JP JP2021547404A patent/JP7608348B2/ja active Active
- 2020-02-14 EP EP20756018.6A patent/EP3923995A4/en active Pending
- 2020-02-14 AU AU2020223367A patent/AU2020223367A1/en active Pending
-
2024
- 2024-12-17 JP JP2024220548A patent/JP2025041709A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020168279A2 (en) | 2020-08-20 |
| WO2020168279A3 (en) | 2020-09-24 |
| US20220249703A1 (en) | 2022-08-11 |
| JP7608348B2 (ja) | 2025-01-06 |
| EP3923995A2 (en) | 2021-12-22 |
| JP2025041709A (ja) | 2025-03-26 |
| JP2022520437A (ja) | 2022-03-30 |
| EP3923995A4 (en) | 2023-05-17 |
| AU2020223367A1 (en) | 2021-10-07 |
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