CN111886343A - 使用闭合端dna(cedna)载体控制转基因的表达 - Google Patents
使用闭合端dna(cedna)载体控制转基因的表达 Download PDFInfo
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- CN111886343A CN111886343A CN201980019861.5A CN201980019861A CN111886343A CN 111886343 A CN111886343 A CN 111886343A CN 201980019861 A CN201980019861 A CN 201980019861A CN 111886343 A CN111886343 A CN 111886343A
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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
- A61K48/0083—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 administration regime
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- 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
-
- 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
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
- A61P17/06—Antipsoriatics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
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- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic 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
- 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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- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/14011—Baculoviridae
- C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
- C12N2710/14141—Use of virus, viral particle or viral elements as a vector
- C12N2710/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
- C12N2750/14141—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
- C12N2800/50—Vectors for producing 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0069—Oxidoreductases (1.) acting on single donors with incorporation of molecular oxygen, i.e. oxygenases (1.13)
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Dermatology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Hospice & Palliative Care (AREA)
- Physical Education & Sports Medicine (AREA)
- Rheumatology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Immunology (AREA)
- Psychiatry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862633757P | 2018-02-22 | 2018-02-22 | |
| US201862633795P | 2018-02-22 | 2018-02-22 | |
| US201862633882P | 2018-02-22 | 2018-02-22 | |
| US62/633,882 | 2018-02-22 | ||
| US62/633,795 | 2018-02-22 | ||
| US62/633,757 | 2018-02-22 | ||
| US201862746762P | 2018-10-17 | 2018-10-17 | |
| US62/746,762 | 2018-10-17 | ||
| PCT/US2019/018927 WO2019165050A1 (en) | 2018-02-22 | 2019-02-21 | Controlled expression of transgenes using close-ended dna (cedna) vectors |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN111886343A true CN111886343A (zh) | 2020-11-03 |
Family
ID=67687406
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980019861.5A Pending CN111886343A (zh) | 2018-02-22 | 2019-02-21 | 使用闭合端dna(cedna)载体控制转基因的表达 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20220175970A1 (https=) |
| EP (1) | EP3755803A4 (https=) |
| JP (2) | JP2021513999A (https=) |
| KR (1) | KR20200124250A (https=) |
| CN (1) | CN111886343A (https=) |
| AU (1) | AU2019225937A1 (https=) |
| BR (1) | BR112020017060A2 (https=) |
| CA (1) | CA3092059A1 (https=) |
| IL (1) | IL276658A (https=) |
| MA (1) | MA51915A (https=) |
| MX (1) | MX2020008676A (https=) |
| PH (1) | PH12020551235A1 (https=) |
| SG (1) | SG11202007621TA (https=) |
| WO (1) | WO2019165050A1 (https=) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025114932A1 (en) * | 2023-11-30 | 2025-06-05 | Sanofi | Purification of closed-ended dna molecules |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TW202545984A (zh) | 2017-08-09 | 2025-12-01 | 美商生物化學醫療公司 | 核酸分子及其用途 |
| WO2020033863A1 (en) | 2018-08-09 | 2020-02-13 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| CN113316640A (zh) * | 2018-11-09 | 2021-08-27 | 世代生物公司 | 包含对称的被修饰的反向末端重复序列的被修饰的封闭端dna(cedna) |
| DE102020111571A1 (de) | 2020-03-11 | 2021-09-16 | Immatics US, Inc. | Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren |
| AU2021244559A1 (en) * | 2020-03-24 | 2022-11-17 | Generation Bio Co. | Non-viral DNA vectors and uses thereof for expressing Gaucher therapeutics |
| MX2022011805A (es) * | 2020-03-24 | 2023-01-11 | Generation Bio Co | Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix. |
| JP2023524010A (ja) * | 2020-04-28 | 2023-06-08 | プレジデント アンド フェローズ オブ ハーバード カレッジ | 高効率遺伝子送達系 |
| US20230265440A1 (en) * | 2020-06-26 | 2023-08-24 | The Board Of Trustees Of The Leland Stanford Junior University | Targeting the human ccr5 locus as a safe harbor for the expression of therapeutic proteins |
| CN116234917A (zh) | 2020-07-27 | 2023-06-06 | 安杰瑞姆生物科学公司 | Dna分子组合物及其制备方法和使用方法 |
| US20220090130A1 (en) * | 2020-08-23 | 2022-03-24 | Bioverativ Therapeutics Inc. | MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA) |
| US20240026374A1 (en) * | 2020-09-16 | 2024-01-25 | Generation Bio Co. | Closed-ended dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) |
| CA3211687A1 (en) * | 2021-03-19 | 2022-09-22 | Ozan ALKAN | Non-viral dna vectors and uses thereof for expressing pfic therapeutics |
| CN121662213B (zh) * | 2026-02-06 | 2026-04-21 | 湖南博极生命科技有限公司 | 基于注意力权重追溯的中药活性成分可解释性分析方法及系统 |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2006130581A2 (en) * | 2005-05-31 | 2006-12-07 | Avigen, Inc. | Methods for delivering genes |
| US20110166207A1 (en) * | 2002-04-09 | 2011-07-07 | Cornell Research Foundation, Inc. | Use of aav integration efficiency element for mediating site-specific integration of a transcription unit |
| WO2015191508A1 (en) * | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| WO2017152149A1 (en) * | 2016-03-03 | 2017-09-08 | University Of Massachusetts | Closed-ended linear duplex dna for non-viral gene transfer |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2010538675A (ja) * | 2007-09-19 | 2010-12-16 | アムステルダム モレキュラー セラピューティクス ビー.ブイ. | タンパク質産生の改善のためのaav複製機構の使用 |
| EP2500434A1 (en) * | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery |
| AU2015311704B2 (en) * | 2014-09-07 | 2021-12-09 | Selecta Biosciences, Inc. | Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses |
| EP3889260A1 (en) * | 2014-12-12 | 2021-10-06 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
-
2019
- 2019-02-21 MA MA051915A patent/MA51915A/fr unknown
- 2019-02-21 BR BR112020017060-7A patent/BR112020017060A2/pt not_active Application Discontinuation
- 2019-02-21 PH PH1/2020/551235A patent/PH12020551235A1/en unknown
- 2019-02-21 MX MX2020008676A patent/MX2020008676A/es unknown
- 2019-02-21 AU AU2019225937A patent/AU2019225937A1/en not_active Abandoned
- 2019-02-21 SG SG11202007621TA patent/SG11202007621TA/en unknown
- 2019-02-21 WO PCT/US2019/018927 patent/WO2019165050A1/en not_active Ceased
- 2019-02-21 KR KR1020207026566A patent/KR20200124250A/ko not_active Ceased
- 2019-02-21 US US16/971,016 patent/US20220175970A1/en active Pending
- 2019-02-21 CA CA3092059A patent/CA3092059A1/en active Pending
- 2019-02-21 EP EP19757005.4A patent/EP3755803A4/en active Pending
- 2019-02-21 CN CN201980019861.5A patent/CN111886343A/zh active Pending
- 2019-02-21 JP JP2020544445A patent/JP2021513999A/ja active Pending
-
2020
- 2020-08-11 IL IL276658A patent/IL276658A/en unknown
-
2023
- 2023-12-08 JP JP2023207938A patent/JP2024028931A/ja active Pending
Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20110166207A1 (en) * | 2002-04-09 | 2011-07-07 | Cornell Research Foundation, Inc. | Use of aav integration efficiency element for mediating site-specific integration of a transcription unit |
| WO2006130581A2 (en) * | 2005-05-31 | 2006-12-07 | Avigen, Inc. | Methods for delivering genes |
| US20090208563A1 (en) * | 2005-05-31 | 2009-08-20 | Linda May Rothblum Watkins | Method for delivering genes |
| WO2015191508A1 (en) * | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| WO2017152149A1 (en) * | 2016-03-03 | 2017-09-08 | University Of Massachusetts | Closed-ended linear duplex dna for non-viral gene transfer |
Non-Patent Citations (1)
| Title |
|---|
| LINA LI ET AL.: "Production and characterization of novel recombinant adeno-associated virus replicative-form genomes: a eukaryotic source of DNA for gene transfer", PLOS ONE, vol. 8, no. 8, pages 69879 * |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025114932A1 (en) * | 2023-11-30 | 2025-06-05 | Sanofi | Purification of closed-ended dna molecules |
Also Published As
| Publication number | Publication date |
|---|---|
| JP2024028931A (ja) | 2024-03-05 |
| BR112020017060A2 (pt) | 2020-12-15 |
| PH12020551235A1 (en) | 2021-04-19 |
| WO2019165050A1 (en) | 2019-08-29 |
| MA51915A (fr) | 2020-12-30 |
| EP3755803A1 (en) | 2020-12-30 |
| CA3092059A1 (en) | 2019-08-29 |
| MX2020008676A (es) | 2020-09-25 |
| US20220175970A1 (en) | 2022-06-09 |
| JP2021513999A (ja) | 2021-06-03 |
| SG11202007621TA (en) | 2020-09-29 |
| EP3755803A4 (en) | 2022-01-19 |
| KR20200124250A (ko) | 2020-11-02 |
| IL276658A (en) | 2020-09-30 |
| RU2020131041A (ru) | 2022-03-22 |
| AU2019225937A1 (en) | 2020-08-13 |
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