BR112023023798A2 - Vetores lentivirais e usos dos mesmos - Google Patents
Vetores lentivirais e usos dos mesmosInfo
- Publication number
- BR112023023798A2 BR112023023798A2 BR112023023798A BR112023023798A BR112023023798A2 BR 112023023798 A2 BR112023023798 A2 BR 112023023798A2 BR 112023023798 A BR112023023798 A BR 112023023798A BR 112023023798 A BR112023023798 A BR 112023023798A BR 112023023798 A2 BR112023023798 A2 BR 112023023798A2
- Authority
- BR
- Brazil
- Prior art keywords
- lentiviral vector
- polynucleotide
- lentiviral vectors
- lentiviral
- wpre
- Prior art date
Links
- 239000013598 vector Substances 0.000 title abstract 5
- 102000011755 Phosphoglycerate Kinase Human genes 0.000 abstract 2
- 101001099217 Thermotoga maritima (strain ATCC 43589 / DSM 3109 / JCM 10099 / NBRC 100826 / MSB8) Triosephosphate isomerase Proteins 0.000 abstract 2
- 108091033319 polynucleotide Proteins 0.000 abstract 2
- 239000002157 polynucleotide Substances 0.000 abstract 2
- 102000040430 polynucleotide Human genes 0.000 abstract 2
- 102100033051 40S ribosomal protein S19 Human genes 0.000 abstract 1
- 208000033932 Blackfan-Diamond anemia Diseases 0.000 abstract 1
- 206010053138 Congenital aplastic anaemia Diseases 0.000 abstract 1
- 201000004449 Diamond-Blackfan anemia Diseases 0.000 abstract 1
- 101100530632 Schizosaccharomyces pombe (strain 972 / ATCC 24843) rps1901 gene Proteins 0.000 abstract 1
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 abstract 1
- 241001492404 Woodchuck hepatitis virus Species 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 101150102754 mrps19 gene Proteins 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 230000001124 posttranscriptional effect Effects 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
- 101150064292 rps19 gene Proteins 0.000 abstract 1
- 101150061587 rpsS gene Proteins 0.000 abstract 1
- 230000035897 transcription Effects 0.000 abstract 1
- 238000013518 transcription 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
- C12N15/79—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
-
- 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
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—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
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Zoology (AREA)
- Public Health (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Diabetes (AREA)
- Biophysics (AREA)
- Hematology (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Biochemistry (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)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
vetores lentivirais e usos dos mesmos. a presente invenção fornece um vetor lentiviral, em que o vetor lentiviral compreende um polinucleotídeo, em que o referido polinucleotídeo é caracterizado por compreender uma unidade de transcrição que por sua vez compreende um promotor de fosfoglicerato quinase (pgk) humana, uma sequência de nucleotídeos que codifica a proteína rps19 e um elemento regulador pós-transcricional do vírus da hepatite da marmota (wpre) otimizado. composições e usos do referido vetor lentiviral também são fornecidos no presente documento, particularmente o uso para o tratamento da anemia de diamond-blackfan.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| PCT/ES2021/070343 WO2022238595A1 (es) | 2021-05-13 | 2021-05-13 | Vectores lentivirales y usos de los mismos |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112023023798A2 true BR112023023798A2 (pt) | 2024-01-30 |
Family
ID=76624059
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112023023798A BR112023023798A2 (pt) | 2021-05-13 | 2021-05-13 | Vetores lentivirais e usos dos mesmos |
Country Status (6)
| Country | Link |
|---|---|
| EP (1) | EP4339291A1 (pt) |
| JP (1) | JP2024521989A (pt) |
| CN (1) | CN117441023A (pt) |
| BR (1) | BR112023023798A2 (pt) |
| CA (1) | CA3219359A1 (pt) |
| WO (1) | WO2022238595A1 (pt) |
Family Cites Families (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3076253A1 (en) * | 2017-10-16 | 2019-04-25 | Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicaso.A., M.P. | Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency |
-
2021
- 2021-05-13 BR BR112023023798A patent/BR112023023798A2/pt unknown
- 2021-05-13 WO PCT/ES2021/070343 patent/WO2022238595A1/es active Application Filing
- 2021-05-13 JP JP2023570160A patent/JP2024521989A/ja active Pending
- 2021-05-13 EP EP21734877.0A patent/EP4339291A1/en active Pending
- 2021-05-13 CA CA3219359A patent/CA3219359A1/en active Pending
- 2021-05-13 CN CN202180098198.XA patent/CN117441023A/zh active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CA3219359A1 (en) | 2022-11-17 |
| WO2022238595A1 (es) | 2022-11-17 |
| EP4339291A1 (en) | 2024-03-20 |
| CN117441023A (zh) | 2024-01-23 |
| JP2024521989A (ja) | 2024-06-05 |
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