MX2023001615A - Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav. - Google Patents
Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav.Info
- Publication number
- MX2023001615A MX2023001615A MX2023001615A MX2023001615A MX2023001615A MX 2023001615 A MX2023001615 A MX 2023001615A MX 2023001615 A MX2023001615 A MX 2023001615A MX 2023001615 A MX2023001615 A MX 2023001615A MX 2023001615 A MX2023001615 A MX 2023001615A
- Authority
- MX
- Mexico
- Prior art keywords
- pkp2
- plakophilin
- gene therapy
- vector
- promoter
- Prior art date
Links
- 239000013598 vector Substances 0.000 title abstract 3
- 108010057275 Plakophilins Proteins 0.000 title abstract 2
- 238000001415 gene therapy Methods 0.000 title abstract 2
- 102000003753 Plakophilins Human genes 0.000 title 1
- 210000000234 capsid Anatomy 0.000 abstract 3
- 102100030348 Plakophilin-2 Human genes 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 101000583179 Homo sapiens Plakophilin-2 Proteins 0.000 abstract 1
- 102000004987 Troponin T Human genes 0.000 abstract 1
- 108090001108 Troponin T Proteins 0.000 abstract 1
- 230000000747 cardiac effect Effects 0.000 abstract 1
- 238000001990 intravenous administration Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 239000013608 rAAV vector Substances 0.000 abstract 1
Classifications
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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
- 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
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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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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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
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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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
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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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
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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
- A01K2267/0306—Animal model for genetic diseases
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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
- A01K2267/035—Animal model for multifactorial diseases
- A01K2267/0375—Animal model for cardiovascular diseases
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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
- 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
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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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
En la presente se proporciona una genoterapia para PKP2 (Placofilina-2), por ejemplo, mediante el uso de un vector de virus adenoasociado (AAV). El promotor del vector puede ser un promotor MHCK7 o un promotor de troponina cardíaca T (HTNNT2). La cápside puede ser una cápside de AAV9 o AAVrh74 o variante funcional de esta. Pueden usarse otros promotores o cápsides. Se proporcionan además métodos de tratamiento, tales como por administración intravenosa, intracoronaria, intracarótida o intracardíaca del vector de rAAV, y otras composiciones y métodos.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063063032P | 2020-08-07 | 2020-08-07 | |
PCT/US2021/045220 WO2022032226A1 (en) | 2020-08-07 | 2021-08-09 | Plakophilin-2 (pkp2) gene therapy using aav vector |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2023001615A true MX2023001615A (es) | 2023-03-08 |
Family
ID=80118582
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2023001615A MX2023001615A (es) | 2020-08-07 | 2021-08-09 | Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav. |
Country Status (11)
Country | Link |
---|---|
US (2) | US11883506B2 (es) |
EP (1) | EP4192487A1 (es) |
JP (1) | JP2023538519A (es) |
KR (1) | KR20230043869A (es) |
CN (1) | CN116194154A (es) |
AU (1) | AU2021320902A1 (es) |
BR (1) | BR112023001852A2 (es) |
CA (1) | CA3185267A1 (es) |
IL (1) | IL300263A (es) |
MX (1) | MX2023001615A (es) |
WO (1) | WO2022032226A1 (es) |
Families Citing this family (7)
Publication number | Priority date | Publication date | Assignee | Title |
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KR20230043869A (ko) | 2020-08-07 | 2023-03-31 | 스페이스크래프트 세븐, 엘엘씨 | Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법 |
US11781156B2 (en) * | 2020-10-09 | 2023-10-10 | Tenaya Therapeutics, Inc. | Plakophillin-2 gene therapy methods and compositions |
IL305959A (en) * | 2021-03-19 | 2023-11-01 | Ucl Business Ltd | Combination of genetic therapy and treatment of right ventricular arrhythmogenic cardiomyopathy |
EP4367251A1 (en) * | 2021-07-08 | 2024-05-15 | Tenaya Therapeutics, Inc. | Optimized expression cassettes for gene therapy |
WO2023200742A2 (en) * | 2022-04-11 | 2023-10-19 | Tenaya Therapeutics, Inc. | Capsids for plakophillin-2 gene therapy |
WO2023200736A2 (en) * | 2022-04-11 | 2023-10-19 | Tenaya Therapeutics, Inc. | Plakophillin-2 gene therapy treatment methods |
WO2024064863A2 (en) * | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors |
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2021
- 2021-08-09 KR KR1020237003609A patent/KR20230043869A/ko unknown
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US20220168446A1 (en) | 2022-06-02 |
CA3185267A1 (en) | 2022-02-10 |
CN116194154A (zh) | 2023-05-30 |
KR20230043869A (ko) | 2023-03-31 |
IL300263A (en) | 2023-03-01 |
EP4192487A1 (en) | 2023-06-14 |
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