MX2020001997A - Variantes de capside de virus adenoasociado y metodos de uso de estas. - Google Patents
Variantes de capside de virus adenoasociado y metodos de uso de estas.Info
- Publication number
- MX2020001997A MX2020001997A MX2020001997A MX2020001997A MX2020001997A MX 2020001997 A MX2020001997 A MX 2020001997A MX 2020001997 A MX2020001997 A MX 2020001997A MX 2020001997 A MX2020001997 A MX 2020001997A MX 2020001997 A MX2020001997 A MX 2020001997A
- Authority
- MX
- Mexico
- Prior art keywords
- neuronal cell
- present disclosure
- neural stem
- associated virus
- virions
- Prior art date
Links
- 241000702421 Dependoparvovirus Species 0.000 title abstract 4
- 238000000034 method Methods 0.000 title abstract 3
- 210000000234 capsid Anatomy 0.000 title 1
- 210000002569 neuron Anatomy 0.000 abstract 4
- 210000001178 neural stem cell Anatomy 0.000 abstract 3
- 210000002845 virion Anatomy 0.000 abstract 3
- 102000039446 nucleic acids Human genes 0.000 abstract 2
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 108090000565 Capsid Proteins Proteins 0.000 abstract 1
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 1
- 230000004888 barrier function Effects 0.000 abstract 1
- 238000006386 neutralization reaction Methods 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
Classifications
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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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
-
- 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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- 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
- 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
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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
- C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
-
- 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
-
- 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
- C12N2750/14145—Special targeting system for viral vectors
-
- 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
- C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Virology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Hospice & Palliative Care (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Psychiatry (AREA)
- Mycology (AREA)
- General Chemical & Material Sciences (AREA)
- Immunology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (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 (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762551133P | 2017-08-28 | 2017-08-28 | |
| PCT/US2018/047561 WO2019046069A1 (en) | 2017-08-28 | 2018-08-22 | ADENO-ASSOCIATED VIRUS CAPSID VARIANTS AND METHODS OF USE |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2020001997A true MX2020001997A (es) | 2020-07-20 |
Family
ID=65525930
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2020001997A MX2020001997A (es) | 2017-08-28 | 2018-08-22 | Variantes de capside de virus adenoasociado y metodos de uso de estas. |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US11680249B2 (enExample) |
| EP (1) | EP3676373A4 (enExample) |
| JP (1) | JP2020534788A (enExample) |
| KR (1) | KR20200039617A (enExample) |
| CN (1) | CN110709511A (enExample) |
| AU (1) | AU2018324477A1 (enExample) |
| BR (1) | BR112020003571A2 (enExample) |
| CA (1) | CA3059995A1 (enExample) |
| MX (1) | MX2020001997A (enExample) |
| WO (1) | WO2019046069A1 (enExample) |
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| CN105755044A (zh) | 2011-04-22 | 2016-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| EP3003391B1 (en) | 2013-05-31 | 2021-09-22 | The Regents of The University of California | Adeno-associated virus variants and methods of use thereof |
| EP3800191B1 (en) | 2014-03-17 | 2025-08-20 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| AU2017302013B2 (en) | 2016-07-29 | 2022-05-26 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| CA3053154A1 (en) * | 2017-06-30 | 2019-01-03 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsids and methods of use thereof |
| EP3808849A1 (en) | 2017-08-03 | 2021-04-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| JP2020534788A (ja) | 2017-08-28 | 2020-12-03 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | アデノ随伴ウイルスカプシド変異体及びその使用方法 |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| WO2019200016A1 (en) | 2018-04-10 | 2019-10-17 | President And Fellows Of Harvard College | Aav vectors encoding clarin-1 or gjb2 and uses thereof |
| CN113966399A (zh) | 2018-09-26 | 2022-01-21 | 加州理工学院 | 用于靶向基因疗法的腺相关病毒组合物 |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| KR102934700B1 (ko) * | 2019-03-28 | 2026-03-09 | 더 제너럴 하스피탈 코포레이션 | 트랜스진 발현을 위한 조작된 아데노-연관 (aav) 벡터 |
| TW202102526A (zh) * | 2019-04-04 | 2021-01-16 | 美商銳進科斯生物股份有限公司 | 重組腺相關病毒及其用途 |
| JP2022526018A (ja) * | 2019-04-11 | 2022-05-20 | カリフォルニア インスティテュート オブ テクノロジー | 脳内への特異性が増強されたウイルス組成物 |
| CA3140049A1 (en) * | 2019-05-14 | 2020-11-19 | Duke University | Compositions and methods for the treatment of atpase-mediated diseases |
| US12552838B2 (en) | 2020-04-20 | 2026-02-17 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| CN116390934A (zh) * | 2020-05-26 | 2023-07-04 | 塑造治疗公司 | 功能性aav衣壳的高通量工程化 |
| US12031147B2 (en) | 2020-05-29 | 2024-07-09 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsids and methods of use thereof |
| US12129287B2 (en) | 2020-09-14 | 2024-10-29 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
| CN113754727B (zh) * | 2020-09-30 | 2022-07-12 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| EP4229204A1 (en) | 2020-10-15 | 2023-08-23 | F. Hoffmann-La Roche AG | Nucleic acid constructs for simultaneous gene activation |
| CN113025658B (zh) * | 2021-03-10 | 2022-04-22 | 华侨大学 | 一种神经干细胞特异性基因的递送载体及其应用 |
| CN113121651B (zh) * | 2021-04-19 | 2023-11-17 | 信念医药科技(上海)有限公司 | 低中和抗体腺相关病毒衣壳蛋白 |
| WO2023010120A2 (en) * | 2021-07-30 | 2023-02-02 | University Of Florida Research Foundation, Incorporated | Engineering aav vectors with improved cns targeting |
| CN114057840B (zh) * | 2021-10-14 | 2022-09-20 | 和元生物技术（上海）股份有限公司 | 包含变异的aav9衣壳蛋白的重组腺相关病毒颗粒 |
| CN119095866A (zh) * | 2022-02-22 | 2024-12-06 | 北卡罗来纳大学查佩尔希尔分校 | 嵌合神经向性aav衣壳 |
| WO2024017387A1 (en) | 2022-07-22 | 2024-01-25 | Shanghai Vitalgen Biopharma Co., Ltd. | Novel aav capsids for targeting nervous system and uses thereof |
| CN116789739B (zh) * | 2022-12-08 | 2024-01-26 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN117012275A (zh) * | 2023-08-11 | 2023-11-07 | 瑞诺元(苏州)生物科技有限公司 | 一种腺相关病毒测序数据的分析方法及系统 |
| WO2025147741A1 (en) * | 2024-01-11 | 2025-07-17 | Children's Medical Research Institute | Aav capsids and vectors for transduction of cells |
| KR20260014488A (ko) * | 2024-07-22 | 2026-01-30 | 한국생명공학연구원 | 변이체 캡시드를 포함하는 아데노-연관 바이러스 및 이의 용도 |
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2018
- 2018-08-22 JP JP2019562303A patent/JP2020534788A/ja not_active Withdrawn
- 2018-08-22 US US16/500,706 patent/US11680249B2/en active Active
- 2018-08-22 KR KR1020197035110A patent/KR20200039617A/ko not_active Withdrawn
- 2018-08-22 BR BR112020003571-8A patent/BR112020003571A2/pt not_active IP Right Cessation
- 2018-08-22 WO PCT/US2018/047561 patent/WO2019046069A1/en not_active Ceased
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- 2018-08-22 AU AU2018324477A patent/AU2018324477A1/en not_active Abandoned
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| AU2018324477A1 (en) | 2019-10-17 |
| KR20200039617A (ko) | 2020-04-16 |
| WO2019046069A1 (en) | 2019-03-07 |
| EP3676373A4 (en) | 2021-06-09 |
| BR112020003571A2 (pt) | 2020-08-25 |
| US11680249B2 (en) | 2023-06-20 |
| CN110709511A (zh) | 2020-01-17 |
| CA3059995A1 (en) | 2019-03-07 |
| US20200095559A1 (en) | 2020-03-26 |
| JP2020534788A (ja) | 2020-12-03 |
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