MX2023000453A - Polipeptidos de mybpc3 y usos de los mismos. - Google Patents
Polipeptidos de mybpc3 y usos de los mismos.Info
- Publication number
- MX2023000453A MX2023000453A MX2023000453A MX2023000453A MX2023000453A MX 2023000453 A MX2023000453 A MX 2023000453A MX 2023000453 A MX2023000453 A MX 2023000453A MX 2023000453 A MX2023000453 A MX 2023000453A MX 2023000453 A MX2023000453 A MX 2023000453A
- Authority
- MX
- Mexico
- Prior art keywords
- mybpc3
- polypeptide
- polypeptides
- arrhythmia
- abnormal
- Prior art date
Links
- 229920001184 polypeptide Polymers 0.000 title abstract 3
- 108090000765 processed proteins & peptides Proteins 0.000 title abstract 3
- 102000004196 processed proteins & peptides Human genes 0.000 title abstract 3
- 101150034357 MYBPC3 gene Proteins 0.000 title 1
- 102000013602 Cardiac Myosins Human genes 0.000 abstract 1
- 108010051609 Cardiac Myosins Proteins 0.000 abstract 1
- 206010019280 Heart failures Diseases 0.000 abstract 1
- 102000004912 RYR2 Human genes 0.000 abstract 1
- 108060007241 RYR2 Proteins 0.000 abstract 1
- 230000002159 abnormal effect Effects 0.000 abstract 1
- 206010003119 arrhythmia Diseases 0.000 abstract 1
- 230000006793 arrhythmia Effects 0.000 abstract 1
- 102000023732 binding proteins Human genes 0.000 abstract 1
- 108091008324 binding proteins Proteins 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 108010059725 myosin-binding protein C Proteins 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 229960000856 protein c Drugs 0.000 abstract 1
Classifications
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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
- A61P9/06—Antiarrhythmics
-
- 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
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- A61K38/1719—Muscle proteins, e.g. myosin or actin
-
- 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
-
- 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
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
-
- 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
- C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
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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; 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
- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
-
- 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
- A61K48/005—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
- A61K48/005—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
-
- 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)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Animal Behavior & Ethology (AREA)
- Molecular Biology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Biomedical Technology (AREA)
- Virology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Immunology (AREA)
- Microbiology (AREA)
- Toxicology (AREA)
- Marine Sciences & Fisheries (AREA)
- Cardiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Heart & Thoracic Surgery (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Se proveen en la presente composiciones y métodos para tratar un trastorno asociado con la función anormal de RYR2 (por ejemplo, arritmia o insuficiencia cardiaca); en algunas modalidades, el método comprende administrar a un sujeto en necesidad del mismo una cantidad efectiva de un polipéptido que comprende un dominio C-terminal de la proteína C de unión a la miosina cardiaca (MYBPC3), o un ácido nucleico o un rAAV que codifica para dicho polipéptido.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063049398P | 2020-07-08 | 2020-07-08 | |
| PCT/US2021/040906 WO2022011151A1 (en) | 2020-07-08 | 2021-07-08 | Mybpc3 polypeptides and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2023000453A true MX2023000453A (es) | 2023-02-09 |
Family
ID=79552103
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2023000453A MX2023000453A (es) | 2020-07-08 | 2021-07-08 | Polipeptidos de mybpc3 y usos de los mismos. |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20220023384A1 (es) |
| EP (1) | EP4178603A4 (es) |
| JP (1) | JP2023533751A (es) |
| KR (1) | KR20230038508A (es) |
| CN (1) | CN115803042A (es) |
| AU (1) | AU2021305213A1 (es) |
| BR (1) | BR112022025123A2 (es) |
| CA (1) | CA3186584A1 (es) |
| CL (1) | CL2023000024A1 (es) |
| CO (1) | CO2023000307A2 (es) |
| IL (1) | IL298854A (es) |
| MX (1) | MX2023000453A (es) |
| TW (1) | TW202208402A (es) |
| WO (1) | WO2022011151A1 (es) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112022015979A2 (pt) | 2020-02-13 | 2022-10-11 | Tenaya Therapeutics Inc | Vetores de terapia genética para tratamento de doenças cardíacas |
| WO2023159234A2 (en) * | 2022-02-18 | 2023-08-24 | Arizona Board Of Regents On Behalf Of The University Of Arizona | Methods and compositions for treating or ameliorating cardiac muscle arrhythmias and skeletal muscle tremors |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8933048B2 (en) * | 2011-09-21 | 2015-01-13 | Case Western Reserve University | Methods of treating cardiomyopathy |
| EP2792742A1 (en) * | 2013-04-17 | 2014-10-22 | Universitätsklinikum Hamburg-Eppendorf (UKE) | Gene-therapy vectors for treating cardiomyopathy |
| IT201800004253A1 (it) * | 2018-04-05 | 2019-10-05 | Composizioni e metodi per il trattamento della tachicardia ventricolare polimorfa catecolaminergica dominante ereditaria. |
-
2021
- 2021-07-08 CA CA3186584A patent/CA3186584A1/en active Pending
- 2021-07-08 AU AU2021305213A patent/AU2021305213A1/en active Pending
- 2021-07-08 MX MX2023000453A patent/MX2023000453A/es unknown
- 2021-07-08 IL IL298854A patent/IL298854A/en unknown
- 2021-07-08 JP JP2023501498A patent/JP2023533751A/ja active Pending
- 2021-07-08 US US17/371,017 patent/US20220023384A1/en active Pending
- 2021-07-08 WO PCT/US2021/040906 patent/WO2022011151A1/en active Application Filing
- 2021-07-08 EP EP21837377.7A patent/EP4178603A4/en active Pending
- 2021-07-08 BR BR112022025123A patent/BR112022025123A2/pt unknown
- 2021-07-08 CN CN202180048741.5A patent/CN115803042A/zh active Pending
- 2021-07-08 KR KR1020237004019A patent/KR20230038508A/ko unknown
- 2021-07-08 TW TW110125168A patent/TW202208402A/zh unknown
-
2023
- 2023-01-04 CL CL2023000024A patent/CL2023000024A1/es unknown
- 2023-01-12 CO CONC2023/0000307A patent/CO2023000307A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CN115803042A (zh) | 2023-03-14 |
| TW202208402A (zh) | 2022-03-01 |
| EP4178603A4 (en) | 2024-10-16 |
| CL2023000024A1 (es) | 2023-09-29 |
| IL298854A (en) | 2023-02-01 |
| JP2023533751A (ja) | 2023-08-04 |
| KR20230038508A (ko) | 2023-03-20 |
| AU2021305213A1 (en) | 2023-01-19 |
| BR112022025123A2 (pt) | 2023-01-17 |
| WO2022011151A1 (en) | 2022-01-13 |
| EP4178603A1 (en) | 2023-05-17 |
| US20220023384A1 (en) | 2022-01-27 |
| CA3186584A1 (en) | 2022-01-13 |
| CO2023000307A2 (es) | 2023-01-26 |
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