BR112018075855A2 - sistema de vetor viral adeno-associado com dupla sobreposição para a expressão abc4a - Google Patents
sistema de vetor viral adeno-associado com dupla sobreposição para a expressão abc4aInfo
- Publication number
- BR112018075855A2 BR112018075855A2 BR112018075855-8A BR112018075855A BR112018075855A2 BR 112018075855 A2 BR112018075855 A2 BR 112018075855A2 BR 112018075855 A BR112018075855 A BR 112018075855A BR 112018075855 A2 BR112018075855 A2 BR 112018075855A2
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- nucleic acid
- sequence
- acid sequence
- end portion
- nucleotides
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- C—CHEMISTRY; METALLURGY
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- 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
- 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
- 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
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
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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
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
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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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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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
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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
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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
- 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
- C12N2750/14171—Demonstrated in vivo effect
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Ophthalmology & Optometry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Cell Biology (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Toxicology (AREA)
- Mycology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Abstract
a presente invenção fornece um sistema de vetores adeno-associados (aav) para expressar uma proteína abca4 humana em uma célula alvo, o sistema vetor aav compreende um primeiro vetor aav compreendendo uma primeira sequência de ácido nucleico e um segundo vetor aav compreendendo uma segunda sequência de ácido nucleico; em que a primeira sequência de ácido nucleico compreende uma porção de extremidade 5' de uma sequência de codificação abca4 (cds) e a segunda sequência de ácido nucleico compreende uma porção de extremidade 3' de uma abca4 cds e a porção de extremidade 5' e a porção de extremidade 3' abrangem todo a abca4 cds; em que a primeira sequência de ácido nucleico compreende uma sequência de nucleotídeos contíguos correspondendo aos nucleotídeos 105 a 3597 da seq id no: 1; em que a segunda sequência de ácido nucleico compreende uma sequência de nucleotídeos contíguos correspondendo aos nucleotídeos 3806 a 6926 da seq id no: 1; em que a primeira sequência de ácido nucleico e a segunda sequência de ácido nucleico compreendem, cada uma, uma região de sequências sobrepostas com a outra; e, em que a região de sobreposição de sequências compreende pelo menos cerca de 20 nucleotídeos contíguos de uma sequência de ácido nucleico correspondente aos nucleotídeos 3598 a 3805 da seq id no: 1. também são fornecidas utilizações dos sistemas de vetores aav na prevenção ou tratamento de doenças .
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GB1610448.1 | 2016-06-15 | ||
| GBGB1610448.1A GB201610448D0 (en) | 2016-06-15 | 2016-06-15 | Adeno-associated viral vector system |
| GB1707261.2 | 2017-05-05 | ||
| GBGB1707261.2A GB201707261D0 (en) | 2017-05-05 | 2017-05-05 | Adeno-associated viral vector system |
| PCT/GB2017/051741 WO2017216560A1 (en) | 2016-06-15 | 2017-06-14 | Dual overlapping adeno-associated viral vector system for expressing abc4a |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112018075855A2 true BR112018075855A2 (pt) | 2019-04-02 |
Family
ID=59101505
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112018075855-8A BR112018075855A2 (pt) | 2016-06-15 | 2017-06-14 | sistema de vetor viral adeno-associado com dupla sobreposição para a expressão abc4a |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20190309326A1 (pt) |
| EP (1) | EP3472328A1 (pt) |
| JP (1) | JP2019523648A (pt) |
| KR (1) | KR20190020745A (pt) |
| CN (1) | CN109642242A (pt) |
| AU (1) | AU2017286623A1 (pt) |
| BR (1) | BR112018075855A2 (pt) |
| CA (1) | CA3025445A1 (pt) |
| IL (1) | IL263523A (pt) |
| MX (1) | MX2018015629A (pt) |
| RU (1) | RU2765826C2 (pt) |
| SG (1) | SG11201811244SA (pt) |
| WO (1) | WO2017216560A1 (pt) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019195726A1 (en) * | 2018-04-05 | 2019-10-10 | Oxford University Innovation Limited | Compositions and methods for the treatment of stargardt disease |
| BR112021007221A2 (pt) * | 2018-10-15 | 2021-08-10 | Fondazione Telethon | proteínas inteínas e seus usos |
| GB201817469D0 (en) * | 2018-10-26 | 2018-12-12 | Univ Oxford Innovation Ltd | Gene therapy for retinal disease |
| US20220267802A1 (en) * | 2019-07-15 | 2022-08-25 | President And Fellows Of Harvard College | Methods and compositions for gene delivery |
| US20230149565A1 (en) * | 2020-04-01 | 2023-05-18 | University Of Florida Research Foundation, Incorporated | Dual aav-myo7a vectors with improved safety for the treatment of ush1b |
| EP4196140A1 (en) * | 2020-08-14 | 2023-06-21 | Case Western Reserve University | Plasmid vectors and nanoparticles for treating ocular disorders |
| CA3218195A1 (en) * | 2021-05-07 | 2022-11-10 | Robin Ali | Abca4 genome editing |
| WO2023160454A1 (zh) * | 2022-02-25 | 2023-08-31 | 北京中因科技有限公司 | 表达盒组合及其应用 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2410133T3 (es) * | 2005-10-20 | 2013-07-01 | Uniqure Ip B.V. | Vectores de VAA mejorados producidos en células de insecto. |
| US20090214478A1 (en) * | 2008-02-21 | 2009-08-27 | Alberto Auricchio | Method of treating ocular diseases by gene therapy |
| RU2555346C2 (ru) * | 2009-08-07 | 2015-07-10 | Трансген Са | Композиция для лечения инфекции вирусом гепатита в |
| WO2013075008A1 (en) * | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| ES2704677T3 (es) * | 2013-04-18 | 2019-03-19 | Fond Telethon | Suministro eficaz de genes grandes por vectores de AAV duales |
-
2017
- 2017-06-14 MX MX2018015629A patent/MX2018015629A/es unknown
- 2017-06-14 CA CA3025445A patent/CA3025445A1/en not_active Abandoned
- 2017-06-14 US US16/309,428 patent/US20190309326A1/en not_active Abandoned
- 2017-06-14 AU AU2017286623A patent/AU2017286623A1/en not_active Abandoned
- 2017-06-14 RU RU2019100525A patent/RU2765826C2/ru active
- 2017-06-14 JP JP2018565324A patent/JP2019523648A/ja active Pending
- 2017-06-14 WO PCT/GB2017/051741 patent/WO2017216560A1/en unknown
- 2017-06-14 CN CN201780037639.9A patent/CN109642242A/zh active Pending
- 2017-06-14 EP EP17732175.9A patent/EP3472328A1/en not_active Withdrawn
- 2017-06-14 SG SG11201811244SA patent/SG11201811244SA/en unknown
- 2017-06-14 BR BR112018075855-8A patent/BR112018075855A2/pt not_active Application Discontinuation
- 2017-06-14 KR KR1020197001281A patent/KR20190020745A/ko not_active Application Discontinuation
-
2018
- 2018-12-05 IL IL263523A patent/IL263523A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CN109642242A (zh) | 2019-04-16 |
| CA3025445A1 (en) | 2017-12-21 |
| RU2765826C2 (ru) | 2022-02-03 |
| IL263523A (en) | 2019-01-31 |
| EP3472328A1 (en) | 2019-04-24 |
| JP2019523648A (ja) | 2019-08-29 |
| SG11201811244SA (en) | 2019-01-30 |
| MX2018015629A (es) | 2019-09-26 |
| US20190309326A1 (en) | 2019-10-10 |
| WO2017216560A1 (en) | 2017-12-21 |
| AU2017286623A1 (en) | 2018-12-20 |
| KR20190020745A (ko) | 2019-03-04 |
| RU2019100525A3 (pt) | 2020-07-15 |
| RU2019100525A (ru) | 2020-07-15 |
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