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
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- 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 (OSRAM) |
| EP (1) | EP3676373A4 (OSRAM) |
| JP (1) | JP2020534788A (OSRAM) |
| KR (1) | KR20200039617A (OSRAM) |
| CN (1) | CN110709511A (OSRAM) |
| AU (1) | AU2018324477A1 (OSRAM) |
| BR (1) | BR112020003571A2 (OSRAM) |
| CA (1) | CA3059995A1 (OSRAM) |
| MX (1) | MX2020001997A (OSRAM) |
| WO (1) | WO2019046069A1 (OSRAM) |
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| CN107012171B (zh) | 2011-04-22 | 2021-07-13 | 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 |
| CN105247044B (zh) | 2013-05-31 | 2021-05-07 | 加利福尼亚大学董事会 | 腺相关病毒变体及其使用方法 |
| EP3800191B1 (en) | 2014-03-17 | 2025-08-20 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| EP4700118A3 (en) | 2016-07-29 | 2026-04-22 | The Regents of the University of California | Adeno-associated virus virions with varint capsid and methods of use thereof |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| EP3645052A4 (en) | 2017-06-30 | 2021-07-28 | Regents of the University of California | ADENO-ASSOCIATED VIRUS VIRIONS WITH ASSOCIATED CAPSIDE VARIANTS AND METHODS OF USE |
| TWI832036B (zh) | 2017-08-03 | 2024-02-11 | 美商航海家醫療公司 | 用於aav之遞送之組合物及方法 |
| KR20200039617A (ko) | 2017-08-28 | 2020-04-16 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 아데노-연관 바이러스 캡시드 변이체 및 이의 사용 방법 |
| MX2020003042A (es) | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| WO2019200016A1 (en) | 2018-04-10 | 2019-10-17 | President And Fellows Of Harvard College | Aav vectors encoding clarin-1 or gjb2 and uses thereof |
| SG11202103102WA (en) | 2018-09-26 | 2021-04-29 | California Inst Of Techn | Adeno-associated virus compositions for targeted gene therapy |
| CN113383010B (zh) | 2018-09-28 | 2025-09-09 | 沃雅戈治疗公司 | 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法 |
| AU2020248116B2 (en) * | 2019-03-28 | 2025-12-04 | President And Fellows Of Harvard College | Engineered adeno-associated (AAV) vectors for transgene expression |
| 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 |
| KR20220007056A (ko) * | 2019-04-11 | 2022-01-18 | 캘리포니아 인스티튜트 오브 테크놀로지 | 뇌에서 증진된 특이성을 갖는 바이러스 조성물 |
| JP2022531809A (ja) * | 2019-05-14 | 2022-07-11 | デューク ユニバーシティ | ＡＴＰａｓｅ媒介性疾患の処置のための組成物および方法 |
| KR20230004719A (ko) | 2020-04-20 | 2023-01-06 | 테나야 테라퓨틱스, 인코포레이티드 | 조작된 캡시드를 갖는 아데노-연관 바이러스 |
| JP2023529315A (ja) * | 2020-05-26 | 2023-07-10 | シェイプ セラピューティクス インコーポレイテッド | 機能的ａａｖカプシドのハイスループット工学操作 |
| CA3179597A1 (en) | 2020-05-29 | 2021-12-02 | John G. Flannery | 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 |
| US20240342311A1 (en) * | 2021-07-30 | 2024-10-17 | 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 |
| EP4482850A1 (en) * | 2022-02-22 | 2025-01-01 | The University of North Carolina at Chapel Hill | Chimeric neurotropic aav capsids |
| CN119894920A (zh) * | 2022-07-22 | 2025-04-25 | 上海天泽云泰生物医药有限公司 | 用于靶向神经系统的新型aav衣壳及其用途 |
| CN116789738B (zh) * | 2022-12-08 | 2023-12-19 | 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 |
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2018
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- 2018-08-22 EP EP18850221.5A patent/EP3676373A4/en not_active Withdrawn
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- 2018-08-22 JP JP2019562303A patent/JP2020534788A/ja not_active Withdrawn
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| US20200095559A1 (en) | 2020-03-26 |
| CA3059995A1 (en) | 2019-03-07 |
| KR20200039617A (ko) | 2020-04-16 |
| AU2018324477A1 (en) | 2019-10-17 |
| CN110709511A (zh) | 2020-01-17 |
| BR112020003571A2 (pt) | 2020-08-25 |
| US11680249B2 (en) | 2023-06-20 |
| JP2020534788A (ja) | 2020-12-03 |
| EP3676373A4 (en) | 2021-06-09 |
| WO2019046069A1 (en) | 2019-03-07 |
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