CN112739419A - 用于阿尔茨海默症的基因治疗 - Google Patents
用于阿尔茨海默症的基因治疗 Download PDFInfo
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- CN112739419A CN112739419A CN201980049127.3A CN201980049127A CN112739419A CN 112739419 A CN112739419 A CN 112739419A CN 201980049127 A CN201980049127 A CN 201980049127A CN 112739419 A CN112739419 A CN 112739419A
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- 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
- C07K14/4711—Alzheimer's disease; Amyloid plaque core protein
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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
- 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
- C12N9/6478—Aspartic endopeptidases (3.4.23)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
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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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- 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
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/23—Aspartic endopeptidases (3.4.23)
- C12Y304/23045—Memapsin 1 (3.4.23.45), i.e. beta-secretase 2 or BACE2
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/23—Aspartic endopeptidases (3.4.23)
- C12Y304/23046—Memapsin 2 (3.4.23.46), i.e. beta-secretase 1 or BACE
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
- A61K9/5184—Virus capsids or envelopes enclosing drugs
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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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16111—Cytomegalovirus, e.g. human herpesvirus 5
- C12N2710/16132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16111—Cytomegalovirus, e.g. human herpesvirus 5
- C12N2710/16141—Use of virus, viral particle or viral elements as a vector
- C12N2710/16143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Neurology (AREA)
- Wood Science & Technology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Neurosurgery (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Hospice & Palliative Care (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Psychiatry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Psychology (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Plant Pathology (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862675003P | 2018-05-22 | 2018-05-22 | |
| US62/675,003 | 2018-05-22 | ||
| PCT/US2019/033616 WO2019226832A1 (en) | 2018-05-22 | 2019-05-22 | Gene therapy for alzheimer's disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN112739419A true CN112739419A (zh) | 2021-04-30 |
Family
ID=68617210
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980049127.3A Pending CN112739419A (zh) | 2018-05-22 | 2019-05-22 | 用于阿尔茨海默症的基因治疗 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US12497608B2 (https=) |
| EP (1) | EP3796980A4 (https=) |
| JP (1) | JP7587423B2 (https=) |
| CN (1) | CN112739419A (https=) |
| AU (1) | AU2019272848A1 (https=) |
| CA (1) | CA3100946A1 (https=) |
| IL (1) | IL278813A (https=) |
| WO (1) | WO2019226832A1 (https=) |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN115125244A (zh) * | 2022-05-14 | 2022-09-30 | 吉林大学 | 构建阿尔兹海默症的兔模型方法 |
| CN116024270A (zh) * | 2021-12-27 | 2023-04-28 | 中国科学院昆明动物研究所 | 一种app/psen1双转基因阿尔茨海默病树鼩模型的建立方法 |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3796980A4 (en) | 2018-05-22 | 2022-03-23 | The Brigham & Women's Hospital, Inc. | NEW THERAPY FOR ALZHEIMER'S DISEASE |
| WO2020242892A1 (en) | 2019-05-24 | 2020-12-03 | The Brigham And Women's Hospital, Inc. | Gene therapy for alzheimer's disease |
| IL293285A (en) * | 2019-11-29 | 2022-07-01 | Paros Bio Inc | Gene therapy for neurodegenerative disorders |
| WO2022261413A1 (en) * | 2021-06-10 | 2022-12-15 | The Brigham And Women’S Hospital, Inc. | Cell-based assay for identification of activators of gamma-secretase |
| EP4399311A4 (en) * | 2021-09-10 | 2025-11-26 | Alnylam Pharmaceuticals Inc | APP ARNI COMPOSITIONS AND THEIR METHODS OF USE FOR TREATMENT OR PREVENTION OF DISEASES CHARACTERIZED BY ENLARGED ENDOSOMES |
Citations (4)
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| CN103282374A (zh) * | 2009-05-12 | 2013-09-04 | 加利福尼亚大学董事会 | 用于治疗神经变性病症和阿尔茨海默病并改善正常记忆的方法和组合物 |
| CN103816540A (zh) * | 2012-11-16 | 2014-05-28 | 中国科学院上海生命科学研究院 | 降低β-抑制蛋白1与APH-1蛋白的结合的物质在制备防治神经退行性疾病药物中的应用 |
| CN106604988A (zh) * | 2014-07-03 | 2017-04-26 | 特兰西穆内有限公司 | 用于获得整体活化的单核细胞的方法 |
| CN107614008A (zh) * | 2015-03-20 | 2018-01-19 | 蓝鸟生物公司 | 载体制剂 |
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| US5328470A (en) | 1989-03-31 | 1994-07-12 | The Regents Of The University Of Michigan | Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor |
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| US5986054A (en) | 1995-04-28 | 1999-11-16 | The Hospital For Sick Children, Hsc Research And Development Limited Partnership | Genetic sequences and proteins related to alzheimer's disease |
| AU732508B2 (en) | 1996-01-26 | 2001-04-26 | Governing Council Of The University Of Toronto, The | Nucleic acids and proteins related to Alzheimer's disease, and uses therefor |
| AR020107A1 (es) * | 1998-07-09 | 2002-04-10 | Boehringer Ingelheim Pharma | Metodo para identificar una sustancia capaz de reducir o eliminar la actividad de la presenilinasa, las sustancias identificables con dicho metodo, el usode estas ultimas en la elaboracion de un medicamento para el tratamiento de enfermedades neurodegenerativas y las composiciones farmaceuticas que |
| AU4992099A (en) * | 1998-07-16 | 2000-02-07 | Incyte Pharmaceuticals, Inc. | Human presenilin-associated protein |
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| WO2005037226A2 (en) | 2003-10-17 | 2005-04-28 | Georgia Tech Research Corporation | Genetically engineered enteroendocrine cells for treating glucose-related metabolic disorders |
| PL1675956T3 (pl) | 2003-10-21 | 2011-06-30 | Merck Serono Sa | Minimalna sekwencja DNA działająca jako insulator chromatyny i jej zastosowanie do wyrażania białek |
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| EP3796980A4 (en) | 2018-05-22 | 2022-03-23 | The Brigham & Women's Hospital, Inc. | NEW THERAPY FOR ALZHEIMER'S DISEASE |
| WO2020242892A1 (en) | 2019-05-24 | 2020-12-03 | The Brigham And Women's Hospital, Inc. | Gene therapy for alzheimer's disease |
| IL293285A (en) | 2019-11-29 | 2022-07-01 | Paros Bio Inc | Gene therapy for neurodegenerative disorders |
| US20230136245A1 (en) | 2020-01-31 | 2023-05-04 | Paros Bio, Inc. | Gene therapy for neurodegenerative disorders using polynucleotide silencing and replacement |
-
2019
- 2019-05-22 EP EP19808081.4A patent/EP3796980A4/en active Pending
- 2019-05-22 CN CN201980049127.3A patent/CN112739419A/zh active Pending
- 2019-05-22 WO PCT/US2019/033616 patent/WO2019226832A1/en not_active Ceased
- 2019-05-22 US US17/057,001 patent/US12497608B2/en active Active
- 2019-05-22 AU AU2019272848A patent/AU2019272848A1/en not_active Abandoned
- 2019-05-22 JP JP2020565341A patent/JP7587423B2/ja active Active
- 2019-05-22 CA CA3100946A patent/CA3100946A1/en active Pending
-
2020
- 2020-11-18 IL IL278813A patent/IL278813A/en unknown
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN103282374A (zh) * | 2009-05-12 | 2013-09-04 | 加利福尼亚大学董事会 | 用于治疗神经变性病症和阿尔茨海默病并改善正常记忆的方法和组合物 |
| CN103816540A (zh) * | 2012-11-16 | 2014-05-28 | 中国科学院上海生命科学研究院 | 降低β-抑制蛋白1与APH-1蛋白的结合的物质在制备防治神经退行性疾病药物中的应用 |
| CN106604988A (zh) * | 2014-07-03 | 2017-04-26 | 特兰西穆内有限公司 | 用于获得整体活化的单核细胞的方法 |
| CN107614008A (zh) * | 2015-03-20 | 2018-01-19 | 蓝鸟生物公司 | 载体制剂 |
Non-Patent Citations (3)
| Title |
|---|
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| "NCBI reference sequence:NM_007318.2" * |
| TANJEW DITTGEN,ET AL: "Lentivirus-based genetic manipulations of cortical neurons and their optical and electrophysiological monitoring in vivo", PROC NATL ACAD SCI USA, vol. 101, no. 52, XP003003909, DOI: 10.1073/pnas.0407976101 * |
Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN116024270A (zh) * | 2021-12-27 | 2023-04-28 | 中国科学院昆明动物研究所 | 一种app/psen1双转基因阿尔茨海默病树鼩模型的建立方法 |
| CN116024270B (zh) * | 2021-12-27 | 2025-07-22 | 中国科学院昆明动物研究所 | 一种app/psen1双转基因阿尔茨海默病树鼩模型的建立方法 |
| CN115125244A (zh) * | 2022-05-14 | 2022-09-30 | 吉林大学 | 构建阿尔兹海默症的兔模型方法 |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2019226832A1 (en) | 2019-11-28 |
| US12497608B2 (en) | 2025-12-16 |
| JP2021525245A (ja) | 2021-09-24 |
| IL278813A (en) | 2021-01-31 |
| AU2019272848A1 (en) | 2020-12-03 |
| CA3100946A1 (en) | 2019-11-28 |
| JP7587423B2 (ja) | 2024-11-20 |
| EP3796980A4 (en) | 2022-03-23 |
| US20210108186A1 (en) | 2021-04-15 |
| EP3796980A1 (en) | 2021-03-31 |
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