MX2022015604A - Expresion transgenica selectiva de tejidos. - Google Patents
Expresion transgenica selectiva de tejidos.Info
- Publication number
- MX2022015604A MX2022015604A MX2022015604A MX2022015604A MX2022015604A MX 2022015604 A MX2022015604 A MX 2022015604A MX 2022015604 A MX2022015604 A MX 2022015604A MX 2022015604 A MX2022015604 A MX 2022015604A MX 2022015604 A MX2022015604 A MX 2022015604A
- Authority
- MX
- Mexico
- Prior art keywords
- transgene
- transgene expression
- selective expression
- tissue selective
- selective transgene
- Prior art date
Links
- 108700019146 Transgenes Proteins 0.000 title abstract 5
- 239000000203 mixture Substances 0.000 abstract 2
- 230000001105 regulatory effect Effects 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
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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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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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
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
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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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- 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
- A61P25/08—Antiepileptics; Anticonvulsants
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- 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
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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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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- 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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- 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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- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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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
- A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
- A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
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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
- A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
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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
- A01K2267/0312—Animal model for Alzheimer's disease
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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
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- 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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- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
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- 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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- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
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- Proteomics, Peptides & Aminoacids (AREA)
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Abstract
En la presente se proporcionan composiciones y métodos para la expresión selectiva de un transgén. Las composiciones y métodos para la expresión selectiva de un transgén comprenden uno o más elementos reguladores humanos, los cuales, cuando son ligados operativamente a un transgén, pueden facilitar la expresión selectiva de un transgén (por ejemplo, expresión selectiva del tipo de células) en una célula blanco en comparación con al menos una o más células no blanco.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762480998P | 2017-04-03 | 2017-04-03 |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2022015604A true MX2022015604A (es) | 2023-02-09 |
Family
ID=63713399
Family Applications (4)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2019011772A MX2019011772A (es) | 2017-04-03 | 2018-04-03 | Expresion transgenica selectiva de tejidos. |
MX2022015604A MX2022015604A (es) | 2017-04-03 | 2019-09-30 | Expresion transgenica selectiva de tejidos. |
MX2022015603A MX2022015603A (es) | 2017-04-03 | 2019-09-30 | Expresion transgenica selectiva de tejidos. |
MX2022015605A MX2022015605A (es) | 2017-04-03 | 2019-09-30 | Expresion transgenica selectiva de tejidos. |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2019011772A MX2019011772A (es) | 2017-04-03 | 2018-04-03 | Expresion transgenica selectiva de tejidos. |
Family Applications After (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2022015603A MX2022015603A (es) | 2017-04-03 | 2019-09-30 | Expresion transgenica selectiva de tejidos. |
MX2022015605A MX2022015605A (es) | 2017-04-03 | 2019-09-30 | Expresion transgenica selectiva de tejidos. |
Country Status (19)
Country | Link |
---|---|
US (6) | US10287608B2 (es) |
EP (2) | EP4403642A3 (es) |
JP (2) | JP7246359B2 (es) |
KR (1) | KR102604159B1 (es) |
CN (2) | CN117821509A (es) |
AU (1) | AU2018250161B2 (es) |
BR (1) | BR112019020777A2 (es) |
CA (1) | CA3058189A1 (es) |
CL (2) | CL2019002805A1 (es) |
CO (1) | CO2019011450A2 (es) |
EA (1) | EA201992358A1 (es) |
ES (1) | ES2981970T3 (es) |
IL (1) | IL269767A (es) |
MX (4) | MX2019011772A (es) |
PL (1) | PL3607073T3 (es) |
SA (1) | SA519410244B1 (es) |
SG (1) | SG11201909203WA (es) |
TW (3) | TWI848486B (es) |
WO (1) | WO2018187363A1 (es) |
Families Citing this family (19)
Publication number | Priority date | Publication date | Assignee | Title |
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GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
CA2963288A1 (en) | 2014-10-03 | 2016-04-07 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
US10196639B2 (en) | 2015-10-09 | 2019-02-05 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
EP3390636B1 (en) | 2015-12-14 | 2021-05-19 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of dravet syndrome |
SG11201909203WA (en) | 2017-04-03 | 2019-11-28 | Encoded Therapeutics Inc | Tissue selective transgene expression |
WO2019040923A1 (en) | 2017-08-25 | 2019-02-28 | Stoke Therapeutics, Inc. | ANTISENSE OLIGOMERS FOR THE TREATMENT OF DISEASE CONDITIONS AND OTHER DISEASES |
MA50942A (fr) | 2017-12-01 | 2020-10-07 | Encoded Therapeutics Inc | Protéines de liaison à l'adn modifiées |
MX2020011695A (es) | 2018-05-04 | 2021-02-26 | Stoke Therapeutics Inc | Métodos y composiciones para el tratamiento de la enfermedad por almacenamiento de éster de colesterilo. |
WO2020068990A1 (en) | 2018-09-26 | 2020-04-02 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
AU2019358806A1 (en) * | 2018-10-08 | 2021-05-20 | Allen Institute | Artificial expression constructs for selectively modulating gene expression in interneurons |
WO2020163102A1 (en) * | 2019-02-05 | 2020-08-13 | The Broad Institute, Inc. | Interneuron-specific therapeutics for normalizing neuronal cell excitability and treating dravet syndrome |
WO2020176426A1 (en) * | 2019-02-25 | 2020-09-03 | University Of Massachusetts | Dna-binding domain transactivators and uses thereof |
BR112021018819A2 (pt) * | 2019-03-22 | 2021-11-23 | Encoded Therapeutics Inc | Multiplexar elementos regulatórios para identificar elementos regulatórios específicos do tipo de célula |
JP7432621B2 (ja) * | 2019-05-29 | 2024-02-16 | エンコーデッド セラピューティクス, インコーポレイテッド | 選択的遺伝子調節のための組成物および方法 |
EP4150092A1 (en) | 2020-05-11 | 2023-03-22 | Stoke Therapeutics, Inc. | Opa1 antisense oligomers for treatment of conditions and diseases |
WO2022011390A1 (en) * | 2020-07-08 | 2022-01-13 | Baylor College Of Medicine | Gene therapy for stxbp1 encephalopathy |
KR20230037586A (ko) * | 2020-07-09 | 2023-03-16 | 가부시키가이샤 모달리스 | Mapt 유전자를 표적으로 하는 알츠하이머병의 치료 방법 |
KR20240099360A (ko) * | 2021-10-28 | 2024-06-28 | 유씨비 바이오파마 에스알엘 | 핵산 구축물, 바이러스 벡터 및 바이러스 입자 |
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