MX2019003745A - Animales no humanos que tienen una expansion de repeticiones de hexanucleotidos en un locus c9orf72. - Google Patents
Animales no humanos que tienen una expansion de repeticiones de hexanucleotidos en un locus c9orf72.Info
- Publication number
- MX2019003745A MX2019003745A MX2019003745A MX2019003745A MX2019003745A MX 2019003745 A MX2019003745 A MX 2019003745A MX 2019003745 A MX2019003745 A MX 2019003745A MX 2019003745 A MX2019003745 A MX 2019003745A MX 2019003745 A MX2019003745 A MX 2019003745A
- Authority
- MX
- Mexico
- Prior art keywords
- hexanucleotide repeat
- repeat expansion
- human animal
- human animals
- c9orf72 locus
- Prior art date
Links
- 241001465754 Metazoa Species 0.000 title 1
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 abstract 5
- 201000011240 Frontotemporal dementia Diseases 0.000 abstract 4
- 241000283984 Rodentia Species 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 208000015122 neurodegenerative disease Diseases 0.000 abstract 1
- 230000000626 neurodegenerative effect Effects 0.000 abstract 1
- 229940124598 therapeutic candidate Drugs 0.000 abstract 1
Classifications
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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
- A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
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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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- 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
- C12N15/62—DNA sequences coding for fusion proteins
- C12N15/625—DNA sequences coding for fusion proteins containing a sequence coding for a signal sequence
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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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0618—Cells of the nervous system
- C12N5/0619—Neurons
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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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0618—Cells of the nervous system
- C12N5/0623—Stem cells
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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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
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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
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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
- A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Cell Biology (AREA)
- Neurology (AREA)
- Environmental Sciences (AREA)
- Neurosurgery (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Mycology (AREA)
- Animal Husbandry (AREA)
- Biodiversity & Conservation Biology (AREA)
- Developmental Biology & Embryology (AREA)
- Medicinal Chemistry (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Investigating Or Analysing Biological Materials (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Se proporciona un modelo de animal no humano (por ejemplo, un roedor) para enfermedades asociadas con una secuencia heteróloga de expansión de repeticiones de hexanucleótidos en C9ORF72, cuyo animal no humano comprende una repetición heteróloga de hexanucleótidos (GGGGCC) en un locus C9ORF72 endógeno. Un animal no humano descrito en la presente descripción que comprende una secuencia heteróloga de expansión de repeticiones de hexanucleótidos que comprende al menos una instancia, por ejemplo, repetición, de una secuencia de hexanucleótidos (GGGGCC) puede exhibir además una característica y/o fenotipo asociado con uno o más trastornos neurodegenerativos (por ejemplo, esclerosis lateral amiotrófica (ALS) y/o demencia frontotemporal (FTD), etc.). También se proporcionan métodos para identificar candidatos terapéuticos que pueden usarse para prevenir, retrasar o tratar uno o más trastornos neurodegenerativos (por ejemplo, esclerosis lateral amiotrófica (ALS, también denominado enfermedad de Lou Gehrig) y demencia frontotemporal (FTD)).
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662402613P | 2016-09-30 | 2016-09-30 | |
US201762452795P | 2017-01-31 | 2017-01-31 | |
PCT/US2017/054551 WO2018064600A1 (en) | 2016-09-30 | 2017-09-29 | Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2019003745A true MX2019003745A (es) | 2019-12-11 |
Family
ID=60201654
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2019003745A MX2019003745A (es) | 2016-09-30 | 2017-09-29 | Animales no humanos que tienen una expansion de repeticiones de hexanucleotidos en un locus c9orf72. |
MX2021003996A MX2021003996A (es) | 2016-09-30 | 2019-03-29 | Animales no humanos que tienen una expansion de repeticiones de hexanucleotidos en un locus c9orf72. |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2021003996A MX2021003996A (es) | 2016-09-30 | 2019-03-29 | Animales no humanos que tienen una expansion de repeticiones de hexanucleotidos en un locus c9orf72. |
Country Status (12)
Country | Link |
---|---|
US (2) | US10781453B2 (es) |
EP (1) | EP3518667A1 (es) |
JP (2) | JP7026678B2 (es) |
KR (2) | KR102527979B1 (es) |
CN (1) | CN109862785B (es) |
AU (1) | AU2017336100B2 (es) |
CA (1) | CA3038548A1 (es) |
IL (2) | IL265669B (es) |
MX (2) | MX2019003745A (es) |
RU (1) | RU2760877C2 (es) |
SG (1) | SG10202102997UA (es) |
WO (1) | WO2018064600A1 (es) |
Families Citing this family (22)
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JP6570447B2 (ja) | 2012-10-15 | 2019-09-04 | アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. | C9orf72発現を調節するための組成物 |
EP2906697A4 (en) | 2012-10-15 | 2016-06-22 | Ionis Pharmaceuticals Inc | METHODS OF MONITORING C9ORF72 EXPRESSION |
CN105637090B (zh) | 2013-10-11 | 2020-10-16 | Ionis制药公司 | 用于调节c9orf72表达的组合物 |
US10280418B2 (en) | 2014-03-18 | 2019-05-07 | Univeristy Of Massachusetts | RAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
ES2784360T3 (es) | 2015-05-29 | 2020-09-24 | Regeneron Pharma | Animales no humanos que tienen una interrupción en un locus C9ORF72 |
WO2017079291A1 (en) | 2015-11-02 | 2017-05-11 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating c90rf72 |
WO2017109757A1 (en) * | 2015-12-23 | 2017-06-29 | Crispr Therapeutics Ag | Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration |
MX2019003745A (es) * | 2016-09-30 | 2019-12-11 | Regeneron Pharma | Animales no humanos que tienen una expansion de repeticiones de hexanucleotidos en un locus c9orf72. |
EP3622073A4 (en) * | 2017-05-09 | 2021-01-06 | University of Massachusetts | METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
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WO2018022480A1 (en) | 2016-07-25 | 2018-02-01 | Mayo Foundation For Medical Education And Research | Treating cancer |
MX2019003745A (es) * | 2016-09-30 | 2019-12-11 | Regeneron Pharma | Animales no humanos que tienen una expansion de repeticiones de hexanucleotidos en un locus c9orf72. |
US20190256868A1 (en) | 2016-10-28 | 2019-08-22 | Genethon | Compositions and methods for the treatment of myotonic dystrophy |
US11427824B2 (en) | 2016-10-28 | 2022-08-30 | Genethon | Compositions and methods for the treatment of myotonic dystrophy |
WO2018165541A1 (en) | 2017-03-10 | 2018-09-13 | The Board Of Regents Of The University Of Texas System | Treatment of fuchs' endothelial corneal dystrophy |
EP3622073A4 (en) | 2017-05-09 | 2021-01-06 | University of Massachusetts | METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
KR20200077529A (ko) | 2017-10-24 | 2020-06-30 | 상가모 테라퓨틱스, 인코포레이티드 | 희귀 질환의 치료를 위한 방법 및 조성물 |
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2017
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US20200370054A1 (en) | 2020-11-26 |
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SG10202102997UA (en) | 2021-04-29 |
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CA3038548A1 (en) | 2018-04-05 |
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KR20190057104A (ko) | 2019-05-27 |
JP7026678B2 (ja) | 2022-02-28 |
RU2019108888A3 (es) | 2021-01-18 |
JP2019530453A (ja) | 2019-10-24 |
CN109862785A (zh) | 2019-06-07 |
KR20210109649A (ko) | 2021-09-06 |
MX2021003996A (es) | 2021-06-23 |
AU2017336100A1 (en) | 2019-05-23 |
RU2760877C2 (ru) | 2021-12-01 |
AU2017336100B2 (en) | 2023-11-30 |
CN109862785B (zh) | 2022-09-06 |
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