EA202290001A1 - Векторы aav с промотором миелинового белка ноль и их применение для лечения заболеваний, связанных со шванновскими клетками, таких как болезнь шарко-мари-тута - Google Patents
Векторы aav с промотором миелинового белка ноль и их применение для лечения заболеваний, связанных со шванновскими клетками, таких как болезнь шарко-мари-тутаInfo
- Publication number
- EA202290001A1 EA202290001A1 EA202290001A EA202290001A EA202290001A1 EA 202290001 A1 EA202290001 A1 EA 202290001A1 EA 202290001 A EA202290001 A EA 202290001A EA 202290001 A EA202290001 A EA 202290001A EA 202290001 A1 EA202290001 A1 EA 202290001A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- schwann cells
- treatment
- charcot
- diseases associated
- disease
- Prior art date
Links
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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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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
-
- 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
- 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
-
- 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
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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
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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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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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
- 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
- C07K14/70503—Immunoglobulin superfamily
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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; CARE OF BIRDS, FISHES, INSECTS; 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
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; 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
- A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
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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
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Abstract
В изобретении представлены вирусные векторы для применения в лечении и профилактике заболеваний, связанных со шванновскими клетками, путем специфичной доставки полинуклеотидов в шванновские клетки и достижения специфичной экспрессии в шванновских клетках. Данное изобретение имеет особое применение в лечении и профилактике болезни Шарко-Мари-Тута и других демиелинизирующих нейропатий.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
GBGB1907882.3A GB201907882D0 (en) | 2019-06-03 | 2019-06-03 | Methods |
PCT/EP2020/065312 WO2020245169A1 (en) | 2019-06-03 | 2020-06-03 | Aav vectors with myelin protein zero promoter and uses thereof for treating schwann cell-associated diseases like charcot-marie-tooth disease |
Publications (1)
Publication Number | Publication Date |
---|---|
EA202290001A1 true EA202290001A1 (ru) | 2022-03-28 |
Family
ID=67385825
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EA202290001A EA202290001A1 (ru) | 2019-06-03 | 2020-06-03 | Векторы aav с промотором миелинового белка ноль и их применение для лечения заболеваний, связанных со шванновскими клетками, таких как болезнь шарко-мари-тута |
Country Status (16)
Country | Link |
---|---|
US (1) | US20220323611A1 (ru) |
EP (1) | EP3976800A1 (ru) |
JP (1) | JP2022535412A (ru) |
KR (1) | KR20220016485A (ru) |
CN (1) | CN114026242A (ru) |
AU (1) | AU2020288605A1 (ru) |
BR (1) | BR112021024285A2 (ru) |
CA (1) | CA3142211A1 (ru) |
CL (1) | CL2021003201A1 (ru) |
CO (1) | CO2021016487A2 (ru) |
EA (1) | EA202290001A1 (ru) |
GB (1) | GB201907882D0 (ru) |
IL (1) | IL288462A (ru) |
MX (1) | MX2021014789A (ru) |
SG (1) | SG11202112889VA (ru) |
WO (1) | WO2020245169A1 (ru) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP4267199A1 (en) * | 2020-12-22 | 2023-11-01 | Wisconsin Alumni Research Foundation | Regulatory elements for schwann cell-specific gene expression |
AU2022360382A1 (en) | 2021-10-07 | 2024-05-09 | Research Institute At Nationwide Children's Hospital | Products and methods for myelin protein zero silencing and treating cmt1b disease |
KR20230110194A (ko) * | 2022-01-13 | 2023-07-21 | 주식회사 툴젠 | 슈반 세포 특이적 프로모터 |
Family Cites Families (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9644215B2 (en) * | 2013-04-12 | 2017-05-09 | The General Hospital Corporation | AAV1-caspase gene therapy induced pyroptosis for the treatment of tumors |
US20190336577A1 (en) * | 2016-12-07 | 2019-11-07 | The General Hospital Corporation | Methods and compositions relating the treatment of tumors |
EP3697916A4 (en) * | 2017-10-20 | 2021-08-11 | Research Institute at Nationwide Children's Hospital | METHODS AND MATERIALS FOR NT-3 GENE THERAPY |
-
2019
- 2019-06-03 GB GBGB1907882.3A patent/GB201907882D0/en not_active Ceased
-
2020
- 2020-06-03 AU AU2020288605A patent/AU2020288605A1/en active Pending
- 2020-06-03 MX MX2021014789A patent/MX2021014789A/es unknown
- 2020-06-03 JP JP2021571874A patent/JP2022535412A/ja active Pending
- 2020-06-03 CN CN202080040867.3A patent/CN114026242A/zh active Pending
- 2020-06-03 EP EP20732138.1A patent/EP3976800A1/en active Pending
- 2020-06-03 EA EA202290001A patent/EA202290001A1/ru unknown
- 2020-06-03 KR KR1020217042344A patent/KR20220016485A/ko unknown
- 2020-06-03 US US17/616,006 patent/US20220323611A1/en active Pending
- 2020-06-03 BR BR112021024285A patent/BR112021024285A2/pt unknown
- 2020-06-03 CA CA3142211A patent/CA3142211A1/en active Pending
- 2020-06-03 SG SG11202112889VA patent/SG11202112889VA/en unknown
- 2020-06-03 WO PCT/EP2020/065312 patent/WO2020245169A1/en active Application Filing
-
2021
- 2021-11-28 IL IL288462A patent/IL288462A/en unknown
- 2021-12-01 CL CL2021003201A patent/CL2021003201A1/es unknown
- 2021-12-02 CO CONC2021/0016487A patent/CO2021016487A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
SG11202112889VA (en) | 2021-12-30 |
KR20220016485A (ko) | 2022-02-09 |
CL2021003201A1 (es) | 2023-02-03 |
GB201907882D0 (en) | 2019-07-17 |
CN114026242A (zh) | 2022-02-08 |
IL288462A (en) | 2022-01-01 |
US20220323611A1 (en) | 2022-10-13 |
WO2020245169A1 (en) | 2020-12-10 |
AU2020288605A1 (en) | 2022-02-03 |
CO2021016487A2 (es) | 2022-01-17 |
MX2021014789A (es) | 2022-02-11 |
JP2022535412A (ja) | 2022-08-08 |
EP3976800A1 (en) | 2022-04-06 |
BR112021024285A2 (pt) | 2022-01-11 |
CA3142211A1 (en) | 2020-12-10 |
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