MX2021013913A - Composiciones y metodos para el tratamiento de enfermedades mediadas por atpasa. - Google Patents
Composiciones y metodos para el tratamiento de enfermedades mediadas por atpasa.Info
- Publication number
- MX2021013913A MX2021013913A MX2021013913A MX2021013913A MX2021013913A MX 2021013913 A MX2021013913 A MX 2021013913A MX 2021013913 A MX2021013913 A MX 2021013913A MX 2021013913 A MX2021013913 A MX 2021013913A MX 2021013913 A MX2021013913 A MX 2021013913A
- Authority
- MX
- Mexico
- Prior art keywords
- atpase
- compositions
- treatment
- methods
- mediated diseases
- Prior art date
Links
- 201000010099 disease Diseases 0.000 title abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title abstract 2
- 230000001404 mediated effect Effects 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title abstract 2
- 108091006112 ATPases Proteins 0.000 abstract 1
- 102000057290 Adenosine Triphosphatases Human genes 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 239000013598 vector Substances 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
-
- 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
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
-
- 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
- C12N9/14—Hydrolases (3)
-
- 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
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
-
- 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
- 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
- A61P25/28—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
-
- 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
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
- C12Y306/01—Hydrolases acting on acid anhydrides (3.6) in phosphorus-containing anhydrides (3.6.1)
- C12Y306/0103—Hydrolases acting on acid anhydrides (3.6) in phosphorus-containing anhydrides (3.6.1) m7G(5')pppN diphosphatase (3.6.1.30)
-
- 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
- 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
-
- 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
-
- 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
-
- 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)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Toxicology (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)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
La presente divulgación proporciona casetes de expresión de ácido nucleico, vectores, composiciones y métodos para el tratamiento de enfermedades mediadas por ATPasa en un sujeto.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962847416P | 2019-05-14 | 2019-05-14 | |
PCT/US2020/032978 WO2020232297A1 (en) | 2019-05-14 | 2020-05-14 | Compositions and methods for the treatment of atpase-mediated diseases |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2021013913A true MX2021013913A (es) | 2022-03-17 |
Family
ID=73289760
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2021013913A MX2021013913A (es) | 2019-05-14 | 2020-05-14 | Composiciones y metodos para el tratamiento de enfermedades mediadas por atpasa. |
Country Status (12)
Country | Link |
---|---|
US (2) | US11717560B2 (es) |
EP (1) | EP3969597A4 (es) |
JP (1) | JP2022531809A (es) |
KR (1) | KR20220032005A (es) |
CN (1) | CN114072514A (es) |
AU (1) | AU2020274200A1 (es) |
BR (1) | BR112021022951A2 (es) |
CA (1) | CA3140049A1 (es) |
IL (1) | IL287796A (es) |
MX (1) | MX2021013913A (es) |
SG (1) | SG11202112078TA (es) |
WO (1) | WO2020232297A1 (es) |
Families Citing this family (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
SG11202009452WA (en) | 2018-04-03 | 2020-10-29 | Stridebio Inc | Antibody-evading virus vectors |
AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
IL292297A (en) | 2019-10-17 | 2022-06-01 | Stridebio Inc | Adeno-associated viral vectors for the treatment of Niemann-Pick type c disease |
WO2023069425A2 (en) * | 2021-10-18 | 2023-04-27 | Logicbio Therapeutics, Inc. | Gene therapy for the treatment of wilson''s disease |
WO2023230466A1 (en) * | 2022-05-23 | 2023-11-30 | Lutsenka Svetlana | Compositions and methods for treating wilson's disease |
Family Cites Families (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU2138601A (en) * | 1999-12-22 | 2001-07-03 | Aventis Pasteur Limited | Chlamydia antigens and corresponding DNA fragments and uses thereof |
US7473531B1 (en) * | 2003-08-08 | 2009-01-06 | Colora Corporation | Pancreatic cancer targets and uses thereof |
WO2007011682A2 (en) * | 2005-07-15 | 2007-01-25 | Regents Of The University Of California | Methods and reagents for screening new drugs and for treating ion pump associated disorders and diseases |
US20110038852A1 (en) * | 2009-06-10 | 2011-02-17 | 3-V Biosciences, Inc. | Antivirals that target transporters, carriers, and ion channels |
CN107080847A (zh) * | 2011-06-24 | 2017-08-22 | 森彻斯有限公司 | 细胞外靶向药物缀合物 |
US20140350087A9 (en) * | 2012-03-22 | 2014-11-27 | Halozyme, Inc. | Oncovector Nucleic Acid Molecules and Methods of Use |
AU2013243953A1 (en) * | 2012-04-02 | 2014-10-30 | Modernatx, Inc. | Modified polynucleotides for the production of nuclear proteins |
IN2015KN00074A (es) * | 2012-07-06 | 2015-07-31 | Univ Iowa Res Found | |
EP2952898B1 (en) * | 2014-06-06 | 2018-08-08 | Euroimmun Medizinische Labordiagnostika AG | Diagnosis of a neurological disease |
MX2017007965A (es) * | 2014-12-17 | 2018-01-18 | Fundacion Para La Investig Medica Aplicada | Construcciones de acido nucleico y vectores de terapia de genes para uso en el tratamiento de enfermedad de wilson. |
EP3907231A1 (en) * | 2015-07-19 | 2021-11-10 | Yeda Research and Development Co., Ltd. | Selective inhibitors of alpha2-containing isoforms of na,k-atpase and use thereof for reducing intraocular pressure |
WO2019199841A1 (en) * | 2018-04-09 | 2019-10-17 | Cure Ahc, Inc. | Aav-mediated delivery of atp1a3 genes to central nervous system |
KR20210112339A (ko) * | 2019-01-04 | 2021-09-14 | 울트라제닉스 파마수티컬 인코포레이티드 | 윌슨병을 치료하기 위한 유전자 요법 구축물 |
-
2020
- 2020-05-14 AU AU2020274200A patent/AU2020274200A1/en not_active Abandoned
- 2020-05-14 CN CN202080047216.7A patent/CN114072514A/zh active Pending
- 2020-05-14 WO PCT/US2020/032978 patent/WO2020232297A1/en unknown
- 2020-05-14 BR BR112021022951A patent/BR112021022951A2/pt not_active Application Discontinuation
- 2020-05-14 EP EP20805008.8A patent/EP3969597A4/en active Pending
- 2020-05-14 SG SG11202112078TA patent/SG11202112078TA/en unknown
- 2020-05-14 JP JP2021567820A patent/JP2022531809A/ja active Pending
- 2020-05-14 MX MX2021013913A patent/MX2021013913A/es unknown
- 2020-05-14 CA CA3140049A patent/CA3140049A1/en active Pending
- 2020-05-14 KR KR1020217040927A patent/KR20220032005A/ko unknown
-
2021
- 2021-11-02 IL IL287796A patent/IL287796A/en unknown
- 2021-11-11 US US17/524,466 patent/US11717560B2/en active Active
-
2023
- 2023-06-23 US US18/340,028 patent/US20240075111A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
SG11202112078TA (en) | 2021-11-29 |
US20240075111A1 (en) | 2024-03-07 |
EP3969597A4 (en) | 2023-01-25 |
EP3969597A1 (en) | 2022-03-23 |
KR20220032005A (ko) | 2022-03-15 |
JP2022531809A (ja) | 2022-07-11 |
CN114072514A (zh) | 2022-02-18 |
US11717560B2 (en) | 2023-08-08 |
CA3140049A1 (en) | 2020-11-19 |
IL287796A (en) | 2022-01-01 |
WO2020232297A1 (en) | 2020-11-19 |
US20220088152A1 (en) | 2022-03-24 |
AU2020274200A1 (en) | 2021-11-25 |
BR112021022951A2 (pt) | 2022-01-25 |
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