JP7624410B2 - レット症候群の治療におけるコレステロール 24-ヒドロラーゼのための発現ベクター - Google Patents
レット症候群の治療におけるコレステロール 24-ヒドロラーゼのための発現ベクター Download PDFInfo
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- JP7624410B2 JP7624410B2 JP2021568858A JP2021568858A JP7624410B2 JP 7624410 B2 JP7624410 B2 JP 7624410B2 JP 2021568858 A JP2021568858 A JP 2021568858A JP 2021568858 A JP2021568858 A JP 2021568858A JP 7624410 B2 JP7624410 B2 JP 7624410B2
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- mecp2
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- cyp46a1
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/44—Oxidoreductases (1)
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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
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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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- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
- C12N9/0073—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14) with NADH or NADPH as one donor, and incorporation of one atom of oxygen 1.14.13
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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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
- C12Y114/13—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with NADH or NADPH as one donor, and incorporation of one atom of oxygen (1.14.13)
- C12Y114/13098—Cholesterol 24-hydroxylase (1.14.13.98)
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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
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
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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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- 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
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Microbiology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP19175767.3 | 2019-05-21 | ||
| EP19175767 | 2019-05-21 | ||
| PCT/EP2020/064092 WO2020234363A2 (en) | 2019-05-21 | 2020-05-20 | Expression vector for cholesterol 24-hydrolase in therapy of rett syndrome |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022534202A JP2022534202A (ja) | 2022-07-28 |
| JP2022534202A5 JP2022534202A5 (https=) | 2023-05-31 |
| JP7624410B2 true JP7624410B2 (ja) | 2025-01-30 |
Family
ID=66751865
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021568858A Active JP7624410B2 (ja) | 2019-05-21 | 2020-05-20 | レット症候群の治療におけるコレステロール 24-ヒドロラーゼのための発現ベクター |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20220233654A1 (https=) |
| EP (1) | EP3972631B1 (https=) |
| JP (1) | JP7624410B2 (https=) |
| CN (1) | CN114555813A (https=) |
| WO (1) | WO2020234363A2 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20250262247A1 (en) * | 2022-04-20 | 2025-08-21 | The Regents Of The University Of California | Microglial cells as therapeutic targets in neurodevelopmental disorders |
Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009034127A1 (en) | 2007-09-12 | 2009-03-19 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Use of the cyp46a1 gene for the treatment of alzheimer's disease |
| JP2013544236A (ja) | 2010-10-15 | 2013-12-12 | アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル | ハンチントン病の治療法におけるコレステロール−24−ヒドロラーゼのための発現ベクター |
| WO2018138371A1 (en) | 2017-01-30 | 2018-08-02 | Brainvectis | Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias |
| WO2018226785A1 (en) | 2017-06-06 | 2018-12-13 | University Of Massachusetts | Self-regulating aav vectors for safe expression of mecp2 in rett syndrome |
| JP2019506141A (ja) | 2015-12-11 | 2019-03-07 | カリフォルニア インスティチュート オブ テクノロジー | アデノ随伴ウイルス(aav)を誘導するための標的指向性ペプチド |
Family Cites Families (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US4861719A (en) | 1986-04-25 | 1989-08-29 | Fred Hutchinson Cancer Research Center | DNA constructs for retrovirus packaging cell lines |
| US5278056A (en) | 1988-02-05 | 1994-01-11 | The Trustees Of Columbia University In The City Of New York | Retroviral packaging cell lines and process of using same |
| US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
| AU8200191A (en) | 1990-07-09 | 1992-02-04 | United States of America, as represented by the Secretary, U.S. Department of Commerce, The | High efficiency packaging of mutant adeno-associated virus using amber suppressions |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| DE69233013T2 (de) | 1991-08-20 | 2004-03-04 | The Government Of The United States Of America As Represented By The Secretary Of National Institute Of Health, Office Of Technology Transfer | Adenovirus vermittelter gentransfer in den gastrointestinaltrakt |
| DE69434860T2 (de) | 1993-02-22 | 2007-03-15 | The Rockefeller University | Herstellung von helfer-freien retroviren mit hohem titer mittels transienter transfektion |
| FR2712812B1 (fr) | 1993-11-23 | 1996-02-09 | Centre Nat Rech Scient | Composition pour la production de produits thérapeutiques in vivo. |
| IL116816A (en) | 1995-01-20 | 2003-05-29 | Rhone Poulenc Rorer Sa | Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof |
| US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
| CA2554380C (en) * | 2004-02-17 | 2015-05-19 | The Hospital For Sick Children | Mecp2e1 gene |
| EP3031921B1 (en) * | 2012-12-12 | 2025-03-12 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| MX374532B (es) * | 2013-06-17 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos. |
| WO2014210389A1 (en) * | 2013-06-26 | 2014-12-31 | Rett Syndrome Research Trust | Rett syndrome and treatments therefore |
| ES2739288T3 (es) | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CA2962406A1 (en) * | 2014-09-25 | 2016-03-31 | Cold Spring Harbor Laboratory | Treatment of rett syndrome |
| EP3388520A1 (en) * | 2017-04-11 | 2018-10-17 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical composition for reducing the expression of nkcc1 in a subject in need thereof |
-
2020
- 2020-05-20 JP JP2021568858A patent/JP7624410B2/ja active Active
- 2020-05-20 WO PCT/EP2020/064092 patent/WO2020234363A2/en not_active Ceased
- 2020-05-20 US US17/611,966 patent/US20220233654A1/en active Pending
- 2020-05-20 EP EP20726457.3A patent/EP3972631B1/en active Active
- 2020-05-20 CN CN202080051431.4A patent/CN114555813A/zh active Pending
Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009034127A1 (en) | 2007-09-12 | 2009-03-19 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Use of the cyp46a1 gene for the treatment of alzheimer's disease |
| JP2013544236A (ja) | 2010-10-15 | 2013-12-12 | アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル | ハンチントン病の治療法におけるコレステロール−24−ヒドロラーゼのための発現ベクター |
| JP2019506141A (ja) | 2015-12-11 | 2019-03-07 | カリフォルニア インスティチュート オブ テクノロジー | アデノ随伴ウイルス(aav)を誘導するための標的指向性ペプチド |
| WO2018138371A1 (en) | 2017-01-30 | 2018-08-02 | Brainvectis | Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias |
| WO2018226785A1 (en) | 2017-06-06 | 2018-12-13 | University Of Massachusetts | Self-regulating aav vectors for safe expression of mecp2 in rett syndrome |
Non-Patent Citations (7)
| Title |
|---|
| Brain Research,2017年,Vol.1654,pp.77-84 |
| eLIFE,2016年,Vol.5,e22409 |
| Future Neurology,2015年,Vol.10. No.5,pp.467-483 |
| Lipids,2018年,Vol.53,pp.363-373 |
| Mammalian Genome,2019年02月,Vol.30. No.5,pp.90-110 |
| Nature Genetics,2013年,Vol.45, No.9,pp.1013-1020 |
| The Journal of Neuroscience,2013年,Vol.33. No.34,pp.13612-13620 |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020234363A2 (en) | 2020-11-26 |
| WO2020234363A8 (en) | 2021-10-07 |
| CA3140809A1 (en) | 2020-11-26 |
| EP3972631A2 (en) | 2022-03-30 |
| US20220233654A1 (en) | 2022-07-28 |
| EP3972631B1 (en) | 2024-07-10 |
| CN114555813A (zh) | 2022-05-27 |
| WO2020234363A3 (en) | 2021-11-18 |
| JP2022534202A (ja) | 2022-07-28 |
| EP3972631C0 (en) | 2024-07-10 |
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