KR20230002788A - 신피질 레이어 5 글루타메이트성 뉴런에서 유전자 발현을 선택적으로 조절하기 위한 인공 발현 작제물 - Google Patents
신피질 레이어 5 글루타메이트성 뉴런에서 유전자 발현을 선택적으로 조절하기 위한 인공 발현 작제물 Download PDFInfo
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- KR20230002788A KR20230002788A KR1020227040046A KR20227040046A KR20230002788A KR 20230002788 A KR20230002788 A KR 20230002788A KR 1020227040046 A KR1020227040046 A KR 1020227040046A KR 20227040046 A KR20227040046 A KR 20227040046A KR 20230002788 A KR20230002788 A KR 20230002788A
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- C—CHEMISTRY; METALLURGY
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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
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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
- 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
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- 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
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- 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
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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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- 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
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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
- 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
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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
- 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
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- 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)
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- 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)
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 |
|---|---|
| KR20230002788A true KR20230002788A (ko) | 2023-01-05 |
Family
ID=78269916
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020227040046A Pending KR20230002788A (ko) | 2020-04-21 | 2021-04-21 | 신피질 레이어 5 글루타메이트성 뉴런에서 유전자 발현을 선택적으로 조절하기 위한 인공 발현 작제물 |
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)
| 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)
| 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 |
-
2021
- 2021-04-21 AU AU2021261359A patent/AU2021261359A1/en active Pending
- 2021-04-21 EP EP21793682.2A patent/EP4139462A4/en active Pending
- 2021-04-21 US US17/996,910 patent/US20230159952A1/en active Pending
- 2021-04-21 JP JP2022563930A patent/JP7721568B2/ja active Active
- 2021-04-21 KR KR1020227040046A patent/KR20230002788A/ko active Pending
- 2021-04-21 CA CA3175993A patent/CA3175993A1/en active Pending
- 2021-04-21 WO PCT/US2021/028489 patent/WO2021216778A2/en not_active Ceased
-
2025
- 2025-07-30 JP JP2025127056A patent/JP2025163106A/ja active Pending
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 |
| AU2021261359A1 (en) | 2022-12-08 |
| JP2025163106A (ja) | 2025-10-28 |
| EP4139462A4 (en) | 2025-03-12 |
| JP2023522708A (ja) | 2023-05-31 |
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