CN117651775A - 病毒载体产生系统、用于病毒载体产生的工程化细胞及其使用方法 - Google Patents
病毒载体产生系统、用于病毒载体产生的工程化细胞及其使用方法 Download PDFInfo
- Publication number
- CN117651775A CN117651775A CN202280050463.1A CN202280050463A CN117651775A CN 117651775 A CN117651775 A CN 117651775A CN 202280050463 A CN202280050463 A CN 202280050463A CN 117651775 A CN117651775 A CN 117651775A
- Authority
- CN
- China
- Prior art keywords
- nucleic acid
- acid sequence
- viral vector
- vector production
- expression cassette
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Classifications
-
- 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
-
- 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
-
- 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
-
- 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 [RNase]; Deoxyribonucleases [DNase]
-
- 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 [CRISPR]
-
- 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
- C12N2740/15011—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
-
- 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
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15051—Methods of production or purification of viral material
- C12N2740/15052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16051—Methods of production or purification of viral material
- C12N2740/16052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
-
- 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
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
- C12N2750/14043—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
- C12N2750/14051—Methods of production or purification of viral material
- C12N2750/14052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
-
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
-
- 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
- C12N2800/40—Systems of functionally co-operating vectors
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163189771P | 2021-05-18 | 2021-05-18 | |
| US63/189,771 | 2021-05-18 | ||
| PCT/US2022/029601 WO2022245803A2 (en) | 2021-05-18 | 2022-05-17 | Viral vector production systems, engineered cells for viral vector production, and methods of use thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN117651775A true CN117651775A (zh) | 2024-03-05 |
Family
ID=84141898
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202280050463.1A Pending CN117651775A (zh) | 2021-05-18 | 2022-05-17 | 病毒载体产生系统、用于病毒载体产生的工程化细胞及其使用方法 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20240254513A1 (https=) |
| EP (1) | EP4352220A4 (https=) |
| JP (1) | JP2024519090A (https=) |
| KR (1) | KR20240019120A (https=) |
| CN (1) | CN117651775A (https=) |
| AU (1) | AU2022277418A1 (https=) |
| BR (1) | BR112023024205A2 (https=) |
| CA (1) | CA3220476A1 (https=) |
| IL (1) | IL308569A (https=) |
| MX (1) | MX2023013795A (https=) |
| WO (1) | WO2022245803A2 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN119662733B (zh) * | 2024-12-09 | 2025-10-28 | 广州派真生物技术有限公司 | 一种高产rAAV的质粒系统及应用 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9388425B2 (en) * | 2006-10-20 | 2016-07-12 | Trustees Of Boston University | Tunable genetic switch for regulating gene expression |
| WO2008134593A1 (en) * | 2007-04-25 | 2008-11-06 | President And Fellows Of Harvard College | Molecular circuits |
| EP3288594B1 (en) * | 2015-04-27 | 2022-06-29 | The Trustees of The University of Pennsylvania | Dual aav vector system for crispr/cas9 mediated correction of human disease |
| US11767539B2 (en) * | 2017-05-12 | 2023-09-26 | University Of Massachusetts | Viral vector production |
| US11970720B2 (en) * | 2017-08-22 | 2024-04-30 | Salk Institute For Biological Studies | RNA targeting methods and compositions |
-
2022
- 2022-05-17 JP JP2023571907A patent/JP2024519090A/ja active Pending
- 2022-05-17 CN CN202280050463.1A patent/CN117651775A/zh active Pending
- 2022-05-17 WO PCT/US2022/029601 patent/WO2022245803A2/en not_active Ceased
- 2022-05-17 IL IL308569A patent/IL308569A/en unknown
- 2022-05-17 AU AU2022277418A patent/AU2022277418A1/en active Pending
- 2022-05-17 BR BR112023024205A patent/BR112023024205A2/pt unknown
- 2022-05-17 CA CA3220476A patent/CA3220476A1/en active Pending
- 2022-05-17 US US18/561,050 patent/US20240254513A1/en active Pending
- 2022-05-17 KR KR1020237042063A patent/KR20240019120A/ko active Pending
- 2022-05-17 EP EP22805297.3A patent/EP4352220A4/en active Pending
- 2022-05-17 MX MX2023013795A patent/MX2023013795A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AU2022277418A1 (en) | 2023-11-30 |
| EP4352220A2 (en) | 2024-04-17 |
| IL308569A (en) | 2024-01-01 |
| US20240254513A1 (en) | 2024-08-01 |
| BR112023024205A2 (pt) | 2024-01-30 |
| KR20240019120A (ko) | 2024-02-14 |
| CA3220476A1 (en) | 2022-11-24 |
| WO2022245803A3 (en) | 2022-12-29 |
| JP2024519090A (ja) | 2024-05-08 |
| WO2022245803A2 (en) | 2022-11-24 |
| EP4352220A4 (en) | 2025-09-24 |
| MX2023013795A (es) | 2024-02-15 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP6663859B2 (ja) | ハンチントン病の治療化合物 | |
| WO2020132059A1 (en) | Adeno-associated virus (aav) producer cell line and related methods | |
| CN110785495A (zh) | 腺相关病毒载体的生产方法 | |
| US20250019669A1 (en) | Cell lines with improved aav production capacity | |
| WO2015162274A2 (en) | Mirnas enhancing cell productivity | |
| EP4200316B1 (en) | Process for making a recombinant aav library | |
| Osório et al. | Viral vectors expressing a single microRNA-based short-hairpin RNA result in potent gene silencing in vitro and in vivo | |
| CN102311973A (zh) | 一种新型miRNA和编码蛋白基因共表达载体 | |
| WO2022038368A1 (en) | Cell line for use in producing recombinant adenoviruses | |
| Albrecht et al. | Comparison of lentiviral packaging mixes and producer cell lines for RNAi applications | |
| CN117651775A (zh) | 病毒载体产生系统、用于病毒载体产生的工程化细胞及其使用方法 | |
| Borel et al. | Design of AAV vectors for delivery of RNAi | |
| HK40107526A (zh) | 病毒载体产生系统、用於病毒载体产生的工程化细胞及其使用方法 | |
| US20250369017A1 (en) | Improved recombinant adeno-associated virus production | |
| CN105200059B (zh) | 靶向抑制小鼠UCP2基因表达的siRNA及其表达载体的构建 | |
| JP7406257B2 (ja) | 人工マイクロrna前駆体およびそれを含む改良されたマイクロrna発現ベクター | |
| CN108611355A (zh) | X染色体连锁肌小管肌病的基因药物 | |
| EP4589007A1 (en) | Production of viral vectors | |
| CN102703508A (zh) | 靶向GSK3β基因RNA干扰重组慢病毒载体及其构建、筛选方法 | |
| WO2025180495A1 (zh) | Parp特异性抑制剂在提高细胞中病毒包装产量或质量的用途 | |
| JP2017528151A (ja) | 干渉性分子のスクリーニング方法 | |
| HK40096035B (en) | Process for making a recombinant aav library | |
| HK40096035A (en) | Process for making a recombinant aav library | |
| Zhong et al. | A single-step mixing cloning method for assembly of lentiviral short hairpin RNA expression vectors for gene silencing | |
| Sheet | miRNASelect™ pEGP-miR Cloning and Expression Vector |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PB01 | Publication | ||
| PB01 | Publication | ||
| SE01 | Entry into force of request for substantive examination | ||
| SE01 | Entry into force of request for substantive examination | ||
| REG | Reference to a national code |
Ref country code: HK Ref legal event code: DE Ref document number: 40107526 Country of ref document: HK |