BR112021024285A2 - Vetores aav com promotor da proteína zero da mielina e usos dos mesmos para tratar doenças associadas a células de schwann como doença de charcot marie tooth - Google Patents
Vetores aav com promotor da proteína zero da mielina e usos dos mesmos para tratar doenças associadas a células de schwann como doença de charcot marie toothInfo
- Publication number
- BR112021024285A2 BR112021024285A2 BR112021024285A BR112021024285A BR112021024285A2 BR 112021024285 A2 BR112021024285 A2 BR 112021024285A2 BR 112021024285 A BR112021024285 A BR 112021024285A BR 112021024285 A BR112021024285 A BR 112021024285A BR 112021024285 A2 BR112021024285 A2 BR 112021024285A2
- Authority
- BR
- Brazil
- Prior art keywords
- schwann cell
- myelin
- associated diseases
- treat
- marie tooth
- Prior art date
Links
- 210000004116 schwann cell Anatomy 0.000 title abstract 6
- 239000013598 vector Substances 0.000 title abstract 4
- 102000006386 Myelin Proteins Human genes 0.000 title abstract 3
- 108010083674 Myelin Proteins Proteins 0.000 title abstract 3
- 201000010099 disease Diseases 0.000 title abstract 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title abstract 3
- 210000005012 myelin Anatomy 0.000 title abstract 3
- 102000004169 proteins and genes Human genes 0.000 title abstract 3
- 108090000623 proteins and genes Proteins 0.000 title abstract 3
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 title 1
- 208000010693 Charcot-Marie-Tooth Disease Diseases 0.000 abstract 2
- 230000002265 prevention Effects 0.000 abstract 2
- 230000003210 demyelinating effect Effects 0.000 abstract 1
- 230000007823 neuropathy Effects 0.000 abstract 1
- 201000001119 neuropathy Diseases 0.000 abstract 1
- 102000040430 polynucleotide Human genes 0.000 abstract 1
- 108091033319 polynucleotide Proteins 0.000 abstract 1
- 239000002157 polynucleotide Substances 0.000 abstract 1
- 239000013603 viral vector Substances 0.000 abstract 1
Classifications
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
- 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
- A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Cell Biology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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 |
|---|---|
| BR112021024285A2 true BR112021024285A2 (pt) | 2022-01-11 |
Family
ID=67385825
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112021024285A BR112021024285A2 (pt) | 2019-06-03 | 2020-06-03 | Vetores aav com promotor da proteína zero da mielina e usos dos mesmos para tratar doenças associadas a células de schwann como doença de charcot marie tooth |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US12582725B2 (https=) |
| EP (1) | EP3976800A1 (https=) |
| JP (1) | JP7692369B2 (https=) |
| KR (1) | KR20220016485A (https=) |
| CN (1) | CN114026242A (https=) |
| AU (1) | AU2020288605A1 (https=) |
| BR (1) | BR112021024285A2 (https=) |
| CA (1) | CA3142211A1 (https=) |
| CL (1) | CL2021003201A1 (https=) |
| CO (1) | CO2021016487A2 (https=) |
| EA (1) | EA202290001A1 (https=) |
| GB (1) | GB201907882D0 (https=) |
| IL (1) | IL288462A (https=) |
| MX (1) | MX2021014789A (https=) |
| SG (1) | SG11202112889VA (https=) |
| WO (1) | WO2020245169A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2022140521A1 (en) * | 2020-12-22 | 2022-06-30 | Wisconsin Alumni Research Foundation | Regulatory elements for schwann cell-specific gene expression |
| EP4413131A1 (en) | 2021-10-07 | 2024-08-14 | Research Institute at Nationwide Children's Hospital | Products and methods for myelin protein zero silencing and treating cmt1b disease |
| WO2023136624A1 (ko) | 2022-01-13 | 2023-07-20 | 주식회사 툴젠 | 슈반 세포 특이적 프로모터 |
| GB202303495D0 (en) * | 2023-03-09 | 2023-04-26 | Ucl Business Ltd | Therapy for charcot-marie-tooth disease |
| WO2025105734A1 (ko) * | 2023-11-15 | 2025-05-22 | 주식회사 카인사이언스 | 탈수초 질환 치료용 펩타이드 및 이의 용도 |
| KR20250126990A (ko) * | 2024-02-16 | 2025-08-26 | 주식회사 엣진 | 샤르코-마리-투스 질환 치료를 위한 징크 핑거 단백질 및 이를 발현시키는 방법 |
| CN118291543A (zh) * | 2024-02-29 | 2024-07-05 | 神拓生物技术(杭州)有限公司 | 一种二型神经纤维瘤基因治疗的慢病毒载体及其应用 |
| KR20260001543A (ko) * | 2024-06-25 | 2026-01-06 | 연세대학교 산학협력단 | 바이러스 벡터를 포함한 난청 치료용 조성물 |
Family Cites Families (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL210751B1 (pl) | 2006-09-12 | 2012-02-29 | Anna Jurewicz | Izolowany fragment białka Nogo-A, jego zastosowanie oraz sposób diagnozowania oraz leczenia stwardnienia rozsianego i polineuropatii zapalno-demielinizacyjnej |
| WO2008073303A2 (en) | 2006-12-07 | 2008-06-19 | Switchgear Genomics | Transcriptional regulatory elements of biological pathways, tools, and methods |
| US20090215178A1 (en) | 2008-02-22 | 2009-08-27 | Zequn Tang | Methods to enhance the stability and homogeneity of transgene expression in clonal cell lines |
| AU2009271524A1 (en) | 2008-07-16 | 2010-01-21 | The Board Of Trustees Of The Leland Stanford Junior University | Modulation of GM98 (MRF) in remyelination |
| US20120233713A1 (en) | 2009-09-02 | 2012-09-13 | Brian Popko | Methods and systems for inducible ablation of neural cells |
| EP2561073B1 (en) | 2010-04-23 | 2016-08-24 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| EP2713724A4 (en) | 2011-05-26 | 2015-03-11 | Biogen Idec Inc | METHODS OF TREATING MULTIPLE SCLEROSIS AND PRESERVING AND / OR INCREASING MYELINE CONTENT |
| CN107551269A (zh) | 2012-02-29 | 2018-01-09 | 百深公司 | 周围神经的IgG刺激髓鞘再生 |
| WO2013151665A2 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins associated with human disease |
| US9254311B2 (en) | 2012-04-02 | 2016-02-09 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins |
| US9644215B2 (en) | 2013-04-12 | 2017-05-09 | The General Hospital Corporation | AAV1-caspase gene therapy induced pyroptosis for the treatment of tumors |
| WO2015140351A1 (en) | 2014-03-21 | 2015-09-24 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for enhancing myelination |
| EP2933335A1 (en) | 2014-04-18 | 2015-10-21 | Genethon | A method of treating peripheral neuropathies and motor neuron diseases |
| CN114657141B (zh) | 2014-09-12 | 2025-02-25 | 京都府公立大学法人 | 雪旺氏细胞及其制备方法 |
| US20180305689A1 (en) | 2015-04-22 | 2018-10-25 | Mina Therapeutics Limited | Sarna compositions and methods of use |
| JP6981973B2 (ja) | 2015-10-01 | 2021-12-17 | ヒート バイオロジクス，インコーポレイテッド | Ｉ型及びｉｉ型細胞外ドメインを異種キメラタンパク質として連結する組成物及び方法 |
| WO2018056412A1 (ja) | 2016-09-23 | 2018-03-29 | 国立大学法人大阪大学 | シュワン細胞分化促進剤及び末梢神経再生促進剤 |
| KR102622411B1 (ko) | 2016-10-14 | 2024-01-10 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| KR102437228B1 (ko) | 2016-11-14 | 2022-08-30 | 주식회사 툴젠 | 인위적으로 조작된 sc 기능 조절 시스템 |
| WO2018106753A1 (en) | 2016-12-07 | 2018-06-14 | The General Hospital Corporation | Methods and compositions relating the treatment of tumors |
| CN118581154A (zh) | 2017-06-02 | 2024-09-03 | 国家健康与医学研究院 | 用于遗传障碍的基因疗法的基因组编辑手段和结合病毒载体的基因疗法 |
| AU2018345772B2 (en) | 2017-10-02 | 2025-04-17 | Research Institute At Nationwide Children's Hospital | MiRNA detargeting system for tissue specific interference |
| US11926653B2 (en) | 2017-10-20 | 2024-03-12 | Research Institute At Nationwide Children's Hospital | Methods and materials for NT-3 gene therapy |
| IL299762A (en) | 2020-07-13 | 2023-03-01 | Univ Pennsylvania | Useful preparations for the treatment of Charcot-Marietot disease |
-
2019
- 2019-06-03 GB GBGB1907882.3A patent/GB201907882D0/en not_active Ceased
-
2020
- 2020-06-03 MX MX2021014789A patent/MX2021014789A/es unknown
- 2020-06-03 AU AU2020288605A patent/AU2020288605A1/en active Pending
- 2020-06-03 EA EA202290001A patent/EA202290001A1/ru unknown
- 2020-06-03 KR KR1020217042344A patent/KR20220016485A/ko not_active Ceased
- 2020-06-03 BR BR112021024285A patent/BR112021024285A2/pt unknown
- 2020-06-03 WO PCT/EP2020/065312 patent/WO2020245169A1/en not_active Ceased
- 2020-06-03 CN CN202080040867.3A patent/CN114026242A/zh active Pending
- 2020-06-03 JP JP2021571874A patent/JP7692369B2/ja active Active
- 2020-06-03 SG SG11202112889VA patent/SG11202112889VA/en unknown
- 2020-06-03 CA CA3142211A patent/CA3142211A1/en active Pending
- 2020-06-03 US US17/616,006 patent/US12582725B2/en active Active
- 2020-06-03 EP EP20732138.1A patent/EP3976800A1/en active Pending
-
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 |
|---|---|
| CN114026242A (zh) | 2022-02-08 |
| IL288462A (en) | 2022-01-01 |
| CL2021003201A1 (es) | 2023-02-03 |
| US12582725B2 (en) | 2026-03-24 |
| AU2020288605A1 (en) | 2022-02-03 |
| JP2022535412A (ja) | 2022-08-08 |
| JP7692369B2 (ja) | 2025-06-13 |
| CA3142211A1 (en) | 2020-12-10 |
| GB201907882D0 (en) | 2019-07-17 |
| EP3976800A1 (en) | 2022-04-06 |
| EA202290001A1 (ru) | 2022-03-28 |
| US20220323611A1 (en) | 2022-10-13 |
| WO2020245169A1 (en) | 2020-12-10 |
| MX2021014789A (es) | 2022-02-11 |
| SG11202112889VA (en) | 2021-12-30 |
| CO2021016487A2 (es) | 2022-01-17 |
| KR20220016485A (ko) | 2022-02-09 |
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