BR112021018354A2 - Vetor e método para tratar síndrome de angelman - Google Patents
Vetor e método para tratar síndrome de angelmanInfo
- Publication number
- BR112021018354A2 BR112021018354A2 BR112021018354A BR112021018354A BR112021018354A2 BR 112021018354 A2 BR112021018354 A2 BR 112021018354A2 BR 112021018354 A BR112021018354 A BR 112021018354A BR 112021018354 A BR112021018354 A BR 112021018354A BR 112021018354 A2 BR112021018354 A2 BR 112021018354A2
- Authority
- BR
- Brazil
- Prior art keywords
- vector
- angelman syndrome
- treating angelman
- treating
- treatment
- Prior art date
Links
- 208000009575 Angelman syndrome Diseases 0.000 title abstract 4
- 238000000034 method Methods 0.000 title abstract 4
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 230000007812 deficiency Effects 0.000 abstract 1
Classifications
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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
- A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/93—Ligases (6)
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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
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- 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
-
- 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
- A01K2267/0306—Animal model for genetic diseases
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
vetor e método para tratar síndrome de angelman. um aspecto descrito no presente documento refere-se a um vetor de vírus adenoassociado recombinante (raav) e um método para uso do mesmo para o tratamento da síndrome de angelman. outro aspecto aqui descrito é um vetor de ube3a raav e método para uso do mesmo para o tratamento de uma deficiência de ube3a, por exemplo, síndrome de angelman, em seres humanos.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962821442P | 2019-03-21 | 2019-03-21 | |
| PCT/US2020/024030 WO2020191366A1 (en) | 2019-03-21 | 2020-03-20 | Vector and method for treating angelman syndrome |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112021018354A2 true BR112021018354A2 (pt) | 2021-11-23 |
Family
ID=72521277
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112021018354A BR112021018354A2 (pt) | 2019-03-21 | 2020-03-20 | Vetor e método para tratar síndrome de angelman |
Country Status (17)
| Country | Link |
|---|---|
| US (1) | US20220152223A1 (pt) |
| EP (1) | EP3941530A4 (pt) |
| JP (1) | JP2022525564A (pt) |
| KR (1) | KR20210145180A (pt) |
| CN (1) | CN114206393A (pt) |
| AR (1) | AR118481A1 (pt) |
| AU (1) | AU2020240136A1 (pt) |
| BR (1) | BR112021018354A2 (pt) |
| CA (1) | CA3133455A1 (pt) |
| CL (1) | CL2021002427A1 (pt) |
| CO (1) | CO2021013967A2 (pt) |
| EA (1) | EA202192543A1 (pt) |
| IL (1) | IL286476A (pt) |
| MX (1) | MX2021011198A (pt) |
| SG (1) | SG11202109736RA (pt) |
| TW (1) | TW202102672A (pt) |
| WO (1) | WO2020191366A1 (pt) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20210158859A (ko) * | 2019-05-22 | 2021-12-31 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | Ube3a 유전자 및 발현 카세트 및 이들의 용도 |
| WO2022272171A2 (en) * | 2021-06-25 | 2022-12-29 | University Of South Florida | Secreted ube3a for treatment of neurological disorders |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2001092582A1 (en) * | 2000-06-01 | 2001-12-06 | Genaissance Pharmaceuticals, Inc. | Haplotypes of the ube3a gene |
| WO2010017515A2 (en) * | 2008-08-08 | 2010-02-11 | Integrated Diagnostics Inc. | Breast cancer specific markers and methods of use |
| US9546369B2 (en) * | 2010-04-23 | 2017-01-17 | University Of Massachusetts | Multicistronic expression constructs |
| JP5704361B2 (ja) * | 2010-10-27 | 2015-04-22 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| WO2015060722A1 (en) * | 2013-10-24 | 2015-04-30 | Uniqure Ip B.V. | Aav-5 pseudotyped vector for gene therapy for neurological diseases |
| SI3119437T1 (sl) * | 2014-03-21 | 2020-01-31 | Genzyme Corporation | Genska terapija za retinitis pigmentoza |
| WO2016071911A1 (en) * | 2014-11-06 | 2016-05-12 | Yeda Research And Development Co. Ltd. | Treatment of cns inflammatory disorders |
| WO2016179584A1 (en) * | 2015-05-07 | 2016-11-10 | University Of South Florida | Modified ube3a gene for a gene therapy approach for angelman syndrome |
| HRP20231451T1 (hr) * | 2016-02-05 | 2024-03-01 | Emory University | Ubrizgavanje jednolančanog ili samo-komplementarnog adeno-povezanog virusa 9 u cerebrospinalnu tekućinu |
| US20200138921A1 (en) * | 2017-06-23 | 2020-05-07 | The Trustees Of Columbia University In The City Of New York | Methods of preventing and treating diseases characterized by synaptic dysfunction and neurodegeneration including alzheimer's disease |
| CN110869031A (zh) * | 2017-06-28 | 2020-03-06 | 南佛罗里达大学 | 用于天使人综合征的基因治疗方法的修饰的ube3a基因 |
-
2020
- 2020-03-20 MX MX2021011198A patent/MX2021011198A/es unknown
- 2020-03-20 BR BR112021018354A patent/BR112021018354A2/pt unknown
- 2020-03-20 CN CN202080036221.8A patent/CN114206393A/zh active Pending
- 2020-03-20 EA EA202192543A patent/EA202192543A1/ru unknown
- 2020-03-20 SG SG11202109736R patent/SG11202109736RA/en unknown
- 2020-03-20 KR KR1020217033798A patent/KR20210145180A/ko unknown
- 2020-03-20 JP JP2022504038A patent/JP2022525564A/ja active Pending
- 2020-03-20 AU AU2020240136A patent/AU2020240136A1/en active Pending
- 2020-03-20 EP EP20774683.5A patent/EP3941530A4/en active Pending
- 2020-03-20 US US17/439,140 patent/US20220152223A1/en active Pending
- 2020-03-20 CA CA3133455A patent/CA3133455A1/en active Pending
- 2020-03-20 WO PCT/US2020/024030 patent/WO2020191366A1/en active Application Filing
- 2020-03-23 TW TW109109644A patent/TW202102672A/zh unknown
- 2020-03-25 AR ARP200100816A patent/AR118481A1/es unknown
-
2021
- 2021-09-16 CL CL2021002427A patent/CL2021002427A1/es unknown
- 2021-09-19 IL IL286476A patent/IL286476A/en unknown
- 2021-10-19 CO CONC2021/0013967A patent/CO2021013967A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EA202192543A1 (ru) | 2021-12-27 |
| CA3133455A1 (en) | 2020-09-24 |
| IL286476A (en) | 2021-12-01 |
| WO2020191366A1 (en) | 2020-09-24 |
| KR20210145180A (ko) | 2021-12-01 |
| EP3941530A1 (en) | 2022-01-26 |
| CO2021013967A2 (es) | 2022-02-28 |
| US20220152223A1 (en) | 2022-05-19 |
| AR118481A1 (es) | 2021-10-06 |
| CN114206393A (zh) | 2022-03-18 |
| CL2021002427A1 (es) | 2022-07-01 |
| EP3941530A4 (en) | 2022-12-14 |
| TW202102672A (zh) | 2021-01-16 |
| JP2022525564A (ja) | 2022-05-17 |
| MX2021011198A (es) | 2022-03-04 |
| AU2020240136A1 (en) | 2021-09-30 |
| SG11202109736RA (en) | 2021-10-28 |
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