MX2021014248A - Genes ube3a y casetes de expresion y su uso. - Google Patents
Genes ube3a y casetes de expresion y su uso.Info
- Publication number
- MX2021014248A MX2021014248A MX2021014248A MX2021014248A MX2021014248A MX 2021014248 A MX2021014248 A MX 2021014248A MX 2021014248 A MX2021014248 A MX 2021014248A MX 2021014248 A MX2021014248 A MX 2021014248A MX 2021014248 A MX2021014248 A MX 2021014248A
- Authority
- MX
- Mexico
- Prior art keywords
- ube3a
- expression cassettes
- orf
- subject
- same
- Prior art date
Links
Classifications
-
- 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
- C12N9/93—Ligases (6)
-
- 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
- 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
-
- 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
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- 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
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Virology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (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)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962851411P | 2019-05-22 | 2019-05-22 | |
| PCT/US2020/034171 WO2020237130A1 (en) | 2019-05-22 | 2020-05-22 | Ube3a genes and expression cassettes and their use |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2021014248A true MX2021014248A (es) | 2022-01-06 |
Family
ID=73458642
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021014248A MX2021014248A (es) | 2019-05-22 | 2020-05-22 | Genes ube3a y casetes de expresion y su uso. |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US12465655B2 (https=) |
| EP (1) | EP3973059A4 (https=) |
| JP (1) | JP7616668B2 (https=) |
| KR (1) | KR20210158859A (https=) |
| CN (1) | CN114127296B (https=) |
| AU (1) | AU2020279387A1 (https=) |
| CA (1) | CA3139678A1 (https=) |
| EA (1) | EA202193214A1 (https=) |
| IL (1) | IL287943A (https=) |
| MX (1) | MX2021014248A (https=) |
| SG (1) | SG11202112470YA (https=) |
| WO (1) | WO2020237130A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL303239A (en) * | 2020-12-01 | 2023-07-01 | Univ Pennsylvania | Compositions and their uses for the treatment of Engelmann syndrome |
| AU2023285684A1 (en) * | 2022-06-07 | 2025-01-02 | Kicho Inc. | Agents for modulating expression |
| EP4661916A1 (en) * | 2023-02-08 | 2025-12-17 | Ginkgo Bioworks, Inc. | Gene therapy for angelman syndrome |
| WO2025128816A1 (en) * | 2023-12-13 | 2025-06-19 | Kicho Inc. | Methods of modulating expression of ube3a gene |
| WO2025213123A1 (en) * | 2024-04-05 | 2025-10-09 | Encoded Therapeutics, Inc. | Methods and compositions for increasing expression of ube3a |
Family Cites Families (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| WO1998011244A2 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| AU774706B2 (en) | 1998-09-22 | 2004-07-08 | Johns Hopkins University, The | Methods for large-scale production of recombinant AAV vectors |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| ATE318923T1 (de) | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
| WO2014004572A2 (en) * | 2012-06-25 | 2014-01-03 | Isis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| EP2900686B1 (en) | 2012-09-28 | 2020-06-10 | The University of North Carolina At Chapel Hill | Aav vectors targeted to oligodendrocytes |
| MX374532B (es) * | 2013-06-17 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos. |
| CN105745326A (zh) * | 2013-10-24 | 2016-07-06 | 优尼科Ip有限公司 | 用于基因治疗神经疾病的aav-5假型载体 |
| EP4344741A3 (en) | 2014-11-21 | 2024-08-28 | The University of North Carolina at Chapel Hill | Aav vectors targeted to the central nervous system |
| EP3291843B1 (en) | 2015-05-07 | 2023-03-22 | University of South Florida | Modified ube3a gene for a gene therapy approach for angelman syndrome |
| WO2017048466A1 (en) * | 2015-09-15 | 2017-03-23 | The Regents Of The University Of California | Compositions and methods for delivering biotherapeutics |
| LT3374509T (lt) | 2015-11-12 | 2021-03-10 | F. Hoffmann-La Roche Ag | Oligonukleotidai, skirti sukelti iš tėvo paveldėto ube3a raišką |
| BR112019027692A2 (pt) * | 2017-06-28 | 2020-11-24 | University Of South Florida | gene ube3a modificado para uma abordagem terapêutica genética para a síndrome de angelman |
| CN114206393A (zh) * | 2019-03-21 | 2022-03-18 | Ptc医疗公司 | 用于治疗天使综合征的载体和方法 |
| EP4017523A4 (en) * | 2019-08-22 | 2024-01-17 | The Regents Of The University Of California | UBE3A FOR THE TREATMENT OF ANGELMAN SYNDROME |
-
2020
- 2020-05-22 US US17/612,833 patent/US12465655B2/en active Active
- 2020-05-22 WO PCT/US2020/034171 patent/WO2020237130A1/en not_active Ceased
- 2020-05-22 CN CN202080050918.0A patent/CN114127296B/zh active Active
- 2020-05-22 JP JP2021569494A patent/JP7616668B2/ja active Active
- 2020-05-22 MX MX2021014248A patent/MX2021014248A/es unknown
- 2020-05-22 CA CA3139678A patent/CA3139678A1/en active Pending
- 2020-05-22 KR KR1020217041626A patent/KR20210158859A/ko not_active Withdrawn
- 2020-05-22 AU AU2020279387A patent/AU2020279387A1/en active Pending
- 2020-05-22 EP EP20809370.8A patent/EP3973059A4/en active Pending
- 2020-05-22 EA EA202193214A patent/EA202193214A1/ru unknown
- 2020-05-22 SG SG11202112470YA patent/SG11202112470YA/en unknown
-
2021
- 2021-11-09 IL IL287943A patent/IL287943A/en unknown
-
2025
- 2025-10-15 US US19/359,449 patent/US20260102519A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20220241434A1 (en) | 2022-08-04 |
| CN114127296A (zh) | 2022-03-01 |
| JP2022533448A (ja) | 2022-07-22 |
| EP3973059A1 (en) | 2022-03-30 |
| CA3139678A1 (en) | 2020-11-26 |
| CN114127296B (zh) | 2024-10-25 |
| US20260102519A1 (en) | 2026-04-16 |
| WO2020237130A1 (en) | 2020-11-26 |
| SG11202112470YA (en) | 2021-12-30 |
| EA202193214A1 (ru) | 2022-03-14 |
| KR20210158859A (ko) | 2021-12-31 |
| US12465655B2 (en) | 2025-11-11 |
| IL287943A (en) | 2022-01-01 |
| AU2020279387A1 (en) | 2021-12-09 |
| EP3973059A4 (en) | 2023-10-25 |
| JP7616668B2 (ja) | 2025-01-17 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| MX2021014248A (es) | Genes ube3a y casetes de expresion y su uso. | |
| CO2021005231A2 (es) | Composiciones y métodos para la edición génica de lactato deshidrogenasa (ldha) | |
| CO2022015650A2 (es) | Conjugados de anticuerpo-fármaco que comprenden agonistas de sting | |
| EP4524137A3 (en) | Heterobifunctional compounds as degraders of hpk1 | |
| MX2021011209A (es) | Novedosos inhibidores de molécula pequeña de factores de transcripción tead. | |
| CO2019013609A2 (es) | Vectores de neoantígeno de alfavirus | |
| MX2021001837A (es) | Constructos del receptor de celulas t y usos de los mismos. | |
| CO2017004728A2 (es) | Composiciones y métodos para inhibir la expresión del gen hao1 (hidroxiácido-oxidasa 1 (glicolato-oxidasa) | |
| MX382337B (es) | Terapia génica de globina y uso de la misma para el tratamiento de hemoglobinopatias. | |
| MX2020008268A (es) | Macrociclos como moduladores del regulador de conductancia transmembrana de la fibrosis quistica, composiciones farmaceuticas de estos, su uso en el tratamiento de la fibrosis quistica y procesos para elaborarlos. | |
| ECSP20000655A (es) | AGENTES DE iARN PARA LA INHIBICIÓN DE LA EXPRESIÓN DE ALFA-ENaC Y MÉTODOS DE USO | |
| MX2019005127A (es) | Poblaciones y productos de células madre mesenquimales y sus usos. | |
| MX2020007078A (es) | Composiciones y metodos para la expresion de arns guias crispr que usan el promotor h1. | |
| MX2018016051A (es) | Composiciones y métodos para diagnosticar tipos de cáncer de pulmón usando perfiles de expresión génica. | |
| CL2016001345A1 (es) | Producto farmacéutico que comprende a un inhibidor de la interacción mdm2-p53 y b citarabina y uso para el tratamiento del cáncer. | |
| MX2016013512A (es) | Sistemas y metodos para el uso simultaneo del espectro dentro del espectro usado activamente. | |
| MX2019002699A (es) | Terapia genica para pacientes con anemia de fanconi. | |
| MX2020003954A (es) | Vectores lentivirales para suministro de pklr para el tratamiento de deficiencia de piruvato quinasa. | |
| MX2021007221A (es) | Factor i del complemento y cofactor del factor i del complemento, vectores que realizan codificacion para estos y uso terapeutico. | |
| BR112022006476A2 (pt) | Oligonucleotídeos com análogos de nucleosídeos | |
| AR113450A1 (es) | Vectores adenovirales competentes en la replicación | |
| CO2019008831A2 (es) | Composición química | |
| MX2021003303A (es) | Composiciones y metodos para restaurar la expresion del gen ube3a paterno en sindrome de angelman humano. | |
| MX2016008961A (es) | Matriz de hidrogel que tiene una distribucion no uniforme de celdas que contienen oxigeno. | |
| MX2020006005A (es) | Copolimeros bloque anfifilicos, micelas y metodos para tratar o prevenir insuficiencia cardiaca. |