CN110709511A - 腺相关病毒衣壳变体及其使用方法 - Google Patents
腺相关病毒衣壳变体及其使用方法 Download PDFInfo
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- CN110709511A CN110709511A CN201880035665.2A CN201880035665A CN110709511A CN 110709511 A CN110709511 A CN 110709511A CN 201880035665 A CN201880035665 A CN 201880035665A CN 110709511 A CN110709511 A CN 110709511A
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- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
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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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- 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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- 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
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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
- A61K48/0008—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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- 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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- 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
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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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- 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
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- C12N2800/00—Nucleic acids vectors
- C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
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- 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)
- Virology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Epidemiology (AREA)
- Hospice & Palliative Care (AREA)
- Mycology (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762551133P | 2017-08-28 | 2017-08-28 | |
| US62/551,133 | 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 |
|---|---|
| CN110709511A true CN110709511A (zh) | 2020-01-17 |
Family
ID=65525930
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201880035665.2A Pending CN110709511A (zh) | 2017-08-28 | 2018-08-22 | 腺相关病毒衣壳变体及其使用方法 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US11680249B2 (https=) |
| EP (1) | EP3676373A4 (https=) |
| JP (1) | JP2020534788A (https=) |
| KR (1) | KR20200039617A (https=) |
| CN (1) | CN110709511A (https=) |
| AU (1) | AU2018324477A1 (https=) |
| BR (1) | BR112020003571A2 (https=) |
| CA (1) | CA3059995A1 (https=) |
| MX (1) | MX2020001997A (https=) |
| WO (1) | WO2019046069A1 (https=) |
Cited By (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN113025658A (zh) * | 2021-03-10 | 2021-06-25 | 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 |
| CN113121651A (zh) * | 2021-04-19 | 2021-07-16 | 信念医药科技(上海)有限公司 | 新型低中和抗体腺相关病毒衣壳蛋白 |
| CN113754727A (zh) * | 2020-09-30 | 2021-12-07 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN116249772A (zh) * | 2020-05-29 | 2023-06-09 | 加利福尼亚大学董事会 | 具有变体衣壳的腺相关病毒病毒体及其使用方法 |
| CN116621935A (zh) * | 2022-12-08 | 2023-08-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
Families Citing this family (28)
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| HRP20171334T1 (hr) | 2011-04-22 | 2017-11-17 | The Regents Of The University Of California | Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe |
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| KR102537394B1 (ko) | 2014-03-17 | 2023-05-30 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법 |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| EP3827812B1 (en) | 2016-07-29 | 2025-10-29 | 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 |
| WO2019006182A1 (en) * | 2017-06-30 | 2019-01-03 | The Regents Of The University Of California | VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS AND METHODS OF USE THEREOF |
| JP7221275B2 (ja) | 2017-08-03 | 2023-02-13 | ボイジャー セラピューティクス インコーポレイテッド | Aavを送達するための組成物および方法 |
| 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 |
| US20200263199A1 (en) | 2017-09-29 | 2020-08-20 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
| US12054724B2 (en) | 2018-04-10 | 2024-08-06 | President And Fellows Of Harvard College | AAV vectors encoding clarin-1 or GJB2 and uses thereof |
| WO2020068990A1 (en) | 2018-09-26 | 2020-04-02 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| MX2021011701A (es) * | 2019-03-28 | 2021-12-10 | Massachusetts Gen Hospital | Vectores adenoasociados fabricados por ingenieria (aav) para expresion transgenica. |
| 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 |
| AU2020271108A1 (en) * | 2019-04-11 | 2021-11-11 | California Institute Of Technology | Virus compositions with enhanced specificity in the brain |
| CN114072514A (zh) * | 2019-05-14 | 2022-02-18 | 杜克大学 | 用于治疗atp酶介导的疾病的组合物和方法 |
| IL297391A (en) | 2020-04-20 | 2022-12-01 | Tenaya Therapeutics Inc | An adeno-associated virus with an 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 |
| US12129287B2 (en) | 2020-09-14 | 2024-10-29 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
| WO2022079082A1 (en) | 2020-10-15 | 2022-04-21 | F. Hoffmann-La Roche Ag | Nucleic acid constructs for simultaneous gene activation |
| 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 |
| 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 |
| 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 |
| 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 |
Citations (1)
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| WO2013029030A1 (en) * | 2011-08-24 | 2013-02-28 | The Board Of Trustees Of The Leland Stanford Junior University | New aav capsid proteins for nucleic acid transfer |
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| CN116249772A (zh) * | 2020-05-29 | 2023-06-09 | 加利福尼亚大学董事会 | 具有变体衣壳的腺相关病毒病毒体及其使用方法 |
| CN113754727A (zh) * | 2020-09-30 | 2021-12-07 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN113025658A (zh) * | 2021-03-10 | 2021-06-25 | 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 |
| 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 |
| CN113121651A (zh) * | 2021-04-19 | 2021-07-16 | 信念医药科技(上海)有限公司 | 新型低中和抗体腺相关病毒衣壳蛋白 |
| 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 |
| CN116621935A (zh) * | 2022-12-08 | 2023-08-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN116621935B (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 |
Also Published As
| Publication number | Publication date |
|---|---|
| JP2020534788A (ja) | 2020-12-03 |
| US11680249B2 (en) | 2023-06-20 |
| MX2020001997A (es) | 2020-07-20 |
| BR112020003571A2 (pt) | 2020-08-25 |
| CA3059995A1 (en) | 2019-03-07 |
| KR20200039617A (ko) | 2020-04-16 |
| EP3676373A1 (en) | 2020-07-08 |
| WO2019046069A1 (en) | 2019-03-07 |
| US20200095559A1 (en) | 2020-03-26 |
| EP3676373A4 (en) | 2021-06-09 |
| AU2018324477A1 (en) | 2019-10-17 |
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