MX2023001419A - Vector viral adenoasociado para la expresion de glut1 y usos de este. - Google Patents
Vector viral adenoasociado para la expresion de glut1 y usos de este.Info
- Publication number
- MX2023001419A MX2023001419A MX2023001419A MX2023001419A MX2023001419A MX 2023001419 A MX2023001419 A MX 2023001419A MX 2023001419 A MX2023001419 A MX 2023001419A MX 2023001419 A MX2023001419 A MX 2023001419A MX 2023001419 A MX2023001419 A MX 2023001419A
- Authority
- MX
- Mexico
- Prior art keywords
- adeno
- viral vector
- associated viral
- glut1
- raav
- Prior art date
Links
- 239000013603 viral vector Substances 0.000 title 1
- 210000000234 capsid Anatomy 0.000 abstract 3
- 210000002845 virion Anatomy 0.000 abstract 3
- 238000000034 method Methods 0.000 abstract 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 abstract 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 abstract 1
- 101100481404 Danio rerio tie1 gene Proteins 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 208000007686 GLUT1 deficiency syndrome Diseases 0.000 abstract 1
- 102000058063 Glucose Transporter Type 1 Human genes 0.000 abstract 1
- 108700006771 Glut1 Deficiency Syndrome Proteins 0.000 abstract 1
- 101100481406 Mus musculus Tie1 gene Proteins 0.000 abstract 1
- 108091006296 SLC2A1 Proteins 0.000 abstract 1
- 108010053096 Vascular Endothelial Growth Factor Receptor-1 Proteins 0.000 abstract 1
- 102100033178 Vascular endothelial growth factor receptor 1 Human genes 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 230000003511 endothelial effect Effects 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 239000000203 mixture Substances 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
- C12N15/86—Viral vectors
-
- 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
- A61K48/0075—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
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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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- 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
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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
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
- A01K2217/077—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out heterozygous knock out animals displaying phenotype
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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
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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
- 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
- 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
- C12N2750/14171—Demonstrated in vivo effect
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Gastroenterology & Hepatology (AREA)
- Neurosurgery (AREA)
- Immunology (AREA)
- Cell Biology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
Abstract
En la presente descripción se proporciona una terapia génica para el síndrome de deficiencia de GLUT1 y trastornos relacionados usando un virión de virus adenoasociado recombinante (rAAV) como un vector para expresar una proteína GLUT1 o variante funcional de esta. El virión de rAAV puede usar un activador específico del endotelio, por ejemplo, un activador FLT-1 o Tie-1. La cápside puede ser una cápside AAV6, AA8, AAV9, AAVrh.74 o AAVrh.10 o una variante funcional de esta. Pueden usarse otros activadores o cápsides. Se proporcionan además métodos de tratamiento, tales como por vía intracerebral y/o intravenosa del virión de rAAV, y otras composiciones y métodos.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063061726P | 2020-08-05 | 2020-08-05 | |
PCT/US2021/044416 WO2022031760A1 (en) | 2020-08-05 | 2021-08-03 | Adeno-associated viral vector for glut1 expression and uses thereof |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2023001419A true MX2023001419A (es) | 2023-05-16 |
Family
ID=80118621
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2023001419A MX2023001419A (es) | 2020-08-05 | 2021-08-03 | Vector viral adenoasociado para la expresion de glut1 y usos de este. |
Country Status (11)
Country | Link |
---|---|
US (1) | US20230272422A1 (es) |
EP (1) | EP4192960A1 (es) |
JP (1) | JP2023536902A (es) |
KR (1) | KR20230043123A (es) |
CN (1) | CN116113700A (es) |
AU (1) | AU2021321412A1 (es) |
BR (1) | BR112023001418A2 (es) |
CA (1) | CA3184233A1 (es) |
IL (1) | IL300185A (es) |
MX (1) | MX2023001419A (es) |
WO (1) | WO2022031760A1 (es) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
KR20230043869A (ko) | 2020-08-07 | 2023-03-31 | 스페이스크래프트 세븐, 엘엘씨 | Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법 |
CN114457045B (zh) * | 2022-02-25 | 2023-07-14 | 中国人民解放军军事科学院军事医学研究院 | 抑制Slc2a1的RNAi腺相关病毒及其制备和应用 |
Family Cites Families (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US8071740B2 (en) * | 2000-11-17 | 2011-12-06 | Vascular Biogenics Ltd. | Promoters exhibiting endothelial cell specificity and methods of using same for regulation of angiogenesis |
US20070161031A1 (en) * | 2005-12-16 | 2007-07-12 | The Board Of Trustees Of The Leland Stanford Junior University | Functional arrays for high throughput characterization of gene expression regulatory elements |
HUE052577T2 (hu) * | 2015-03-10 | 2021-05-28 | Univ Columbia | Rekombináns GLUT1 adeno-asszociált vírusvektor-szerkezetek és kapcsolódó eljárások GLUT1 expresszió helyreállítására |
-
2021
- 2021-08-03 AU AU2021321412A patent/AU2021321412A1/en active Pending
- 2021-08-03 IL IL300185A patent/IL300185A/en unknown
- 2021-08-03 EP EP21854255.3A patent/EP4192960A1/en active Pending
- 2021-08-03 BR BR112023001418A patent/BR112023001418A2/pt unknown
- 2021-08-03 CN CN202180057450.2A patent/CN116113700A/zh active Pending
- 2021-08-03 WO PCT/US2021/044416 patent/WO2022031760A1/en unknown
- 2021-08-03 US US18/019,393 patent/US20230272422A1/en active Pending
- 2021-08-03 KR KR1020237003435A patent/KR20230043123A/ko unknown
- 2021-08-03 JP JP2023507555A patent/JP2023536902A/ja active Pending
- 2021-08-03 CA CA3184233A patent/CA3184233A1/en active Pending
- 2021-08-03 MX MX2023001419A patent/MX2023001419A/es unknown
Also Published As
Publication number | Publication date |
---|---|
KR20230043123A (ko) | 2023-03-30 |
CA3184233A1 (en) | 2022-02-10 |
JP2023536902A (ja) | 2023-08-30 |
WO2022031760A1 (en) | 2022-02-10 |
EP4192960A1 (en) | 2023-06-14 |
CN116113700A (zh) | 2023-05-12 |
IL300185A (en) | 2023-03-01 |
US20230272422A1 (en) | 2023-08-31 |
AU2021321412A1 (en) | 2023-04-06 |
BR112023001418A2 (pt) | 2023-03-07 |
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