MA62371A1 - Acide nucléique à codons optimisés codant pour la protéine du facteur fix - Google Patents
Acide nucléique à codons optimisés codant pour la protéine du facteur fixInfo
- Publication number
- MA62371A1 MA62371A1 MA62371A MA62371A MA62371A1 MA 62371 A1 MA62371 A1 MA 62371A1 MA 62371 A MA62371 A MA 62371A MA 62371 A MA62371 A MA 62371A MA 62371 A1 MA62371 A1 MA 62371A1
- Authority
- MA
- Morocco
- Prior art keywords
- nucleic acid
- acid encoding
- codon
- optimized nucleic
- factor protein
- Prior art date
Links
- 108090000623 proteins and genes Proteins 0.000 title abstract 3
- 102000039446 nucleic acids Human genes 0.000 title abstract 2
- 108020004707 nucleic acids Proteins 0.000 title abstract 2
- 150000007523 nucleic acids Chemical class 0.000 title abstract 2
- 102000004169 proteins and genes Human genes 0.000 title abstract 2
- 102000013831 Coagulation factor IX Human genes 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 108010076282 Factor IX Proteins 0.000 abstract 1
- 241000700605 Viruses Species 0.000 abstract 1
- 229940105774 coagulation factor ix Drugs 0.000 abstract 1
- 238000001415 gene therapy Methods 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
- C12N15/67—General methods for enhancing the expression
-
- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- 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)
- A61K38/482—Serine endopeptidases (3.4.21)
- A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
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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
- 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
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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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)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
- C12N9/644—Coagulation factor IXa (3.4.21.22)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21022—Coagulation factor IXa (3.4.21.22)
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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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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
- 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)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Mycology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
La présente invention concerne les domaines de la génétique, de la thérapie génique et de la biologie moléculaire. Plus particulièrement, la présente invention concerne un acide nucléique isolé à codons optimisés codant pour la protéine fix (facteur ix de coagulation), une cassette d'expression et un vecteur basé sur cette dernière, ainsi qu'un virus recombiné basé sur l'aav5 (virus adéno-associé de sérotype 5) pour augmenter l'expression du gène fix dans des cellules cibles, et son utilisation.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
RU2021105703A RU2021105703A (ru) | 2021-03-05 | Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок фактора свёртывания крови IX, и ее применение | |
PCT/RU2022/050073 WO2022186734A1 (fr) | 2021-03-05 | 2022-03-05 | Acide nucléique à codons optimisés codant pour la protéine du facteur fix |
Publications (1)
Publication Number | Publication Date |
---|---|
MA62371A1 true MA62371A1 (fr) | 2024-03-29 |
Family
ID=83154378
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MA62371A MA62371A1 (fr) | 2021-03-05 | 2022-03-05 | Acide nucléique à codons optimisés codant pour la protéine du facteur fix |
Country Status (19)
Country | Link |
---|---|
US (1) | US20240076691A1 (fr) |
EP (1) | EP4288549A1 (fr) |
JP (1) | JP2024509224A (fr) |
KR (1) | KR20230154067A (fr) |
CN (1) | CN117716038A (fr) |
AR (1) | AR125041A1 (fr) |
AU (1) | AU2022230548A1 (fr) |
BR (1) | BR112023018003A2 (fr) |
CA (1) | CA3212809A1 (fr) |
CL (1) | CL2023002629A1 (fr) |
CO (1) | CO2023011679A2 (fr) |
CR (1) | CR20230429A (fr) |
EC (1) | ECSP23067213A (fr) |
IL (1) | IL305679A (fr) |
MA (1) | MA62371A1 (fr) |
MX (1) | MX2023010350A (fr) |
TW (1) | TW202246505A (fr) |
UY (1) | UY39659A (fr) |
WO (1) | WO2022186734A1 (fr) |
Family Cites Families (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6458563B1 (en) * | 1996-06-26 | 2002-10-01 | Emory University | Modified factor VIII |
GB0911870D0 (en) * | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
US10842853B2 (en) * | 2017-05-22 | 2020-11-24 | Baxalta Incorporated | Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia B |
-
2022
- 2022-03-04 TW TW111108106A patent/TW202246505A/zh unknown
- 2022-03-05 CR CR20230429A patent/CR20230429A/es unknown
- 2022-03-05 MX MX2023010350A patent/MX2023010350A/es unknown
- 2022-03-05 WO PCT/RU2022/050073 patent/WO2022186734A1/fr active Application Filing
- 2022-03-05 IL IL305679A patent/IL305679A/en unknown
- 2022-03-05 BR BR112023018003A patent/BR112023018003A2/pt unknown
- 2022-03-05 AU AU2022230548A patent/AU2022230548A1/en active Pending
- 2022-03-05 JP JP2023554061A patent/JP2024509224A/ja active Pending
- 2022-03-05 EP EP22763678.4A patent/EP4288549A1/fr active Pending
- 2022-03-05 KR KR1020237034078A patent/KR20230154067A/ko unknown
- 2022-03-05 CN CN202280017566.8A patent/CN117716038A/zh active Pending
- 2022-03-05 MA MA62371A patent/MA62371A1/fr unknown
- 2022-03-05 US US18/280,338 patent/US20240076691A1/en active Pending
- 2022-03-05 CA CA3212809A patent/CA3212809A1/fr active Pending
- 2022-03-07 UY UY0001039659A patent/UY39659A/es unknown
- 2022-03-07 AR ARP220100514A patent/AR125041A1/es unknown
-
2023
- 2023-09-04 CO CONC2023/0011679A patent/CO2023011679A2/es unknown
- 2023-09-04 CL CL2023002629A patent/CL2023002629A1/es unknown
- 2023-09-05 EC ECSENADI202367213A patent/ECSP23067213A/es unknown
Also Published As
Publication number | Publication date |
---|---|
TW202246505A (zh) | 2022-12-01 |
WO2022186734A1 (fr) | 2022-09-09 |
ECSP23067213A (es) | 2023-10-31 |
BR112023018003A2 (pt) | 2023-12-26 |
KR20230154067A (ko) | 2023-11-07 |
EP4288549A1 (fr) | 2023-12-13 |
MX2023010350A (es) | 2023-09-22 |
AU2022230548A1 (en) | 2023-09-28 |
US20240076691A1 (en) | 2024-03-07 |
JP2024509224A (ja) | 2024-02-29 |
IL305679A (en) | 2023-11-01 |
CR20230429A (es) | 2024-03-08 |
CO2023011679A2 (es) | 2024-01-15 |
CL2023002629A1 (es) | 2024-04-01 |
UY39659A (es) | 2022-09-30 |
AR125041A1 (es) | 2023-05-31 |
CA3212809A1 (fr) | 2022-09-09 |
CN117716038A (zh) | 2024-03-15 |
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