MX2021004842A - Adn de extremo cerrado (ceadn) modificado que comprende repeticiones terminales invertidas modificadas simetricas. - Google Patents
Adn de extremo cerrado (ceadn) modificado que comprende repeticiones terminales invertidas modificadas simetricas.Info
- Publication number
- MX2021004842A MX2021004842A MX2021004842A MX2021004842A MX2021004842A MX 2021004842 A MX2021004842 A MX 2021004842A MX 2021004842 A MX2021004842 A MX 2021004842A MX 2021004842 A MX2021004842 A MX 2021004842A MX 2021004842 A MX2021004842 A MX 2021004842A
- Authority
- MX
- Mexico
- Prior art keywords
- modified
- cedna
- inverted terminal
- terminal repeats
- symmetrical
- Prior art date
Links
Classifications
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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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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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
-
- 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
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
-
- 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
-
- 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
-
- 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
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
Landscapes
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Manufacturing & Machinery (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Virology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
Abstract
En la presente se describen vectores de ceADN que tienen una estructura lineal y continua que pueden producirse con altos rendimientos y usarse para la transferencia y expresión eficaces de un transgén. De acuerdo con algunas modalidades, los vectores de ceADN comprenden al menos una secuencia de nucleótidos heteróloga ubicada operativamente entre dos secuencias flanqueantes simétricas de repetición terminal invertida que no son ITR de AAV de tipo silvestre, en donde toda o parte de la secuencia de nucleótidos heteróloga está bajo el control de al menos un interruptor regulador. Algunos vectores de ceADN proporcionados en la presente comprenden además elementos reguladores cis y proporcionan altas eficiencias de expresión génica. También se proporcionan en la presente métodos y estirpes celulares para la producción fiable y eficiente de los vectores de ADN lineal, continuo y sin cápside.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862757892P | 2018-11-09 | 2018-11-09 | |
US201862757872P | 2018-11-09 | 2018-11-09 | |
PCT/US2019/060395 WO2020097417A1 (en) | 2018-11-09 | 2019-11-08 | Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2021004842A true MX2021004842A (es) | 2021-06-08 |
Family
ID=70612486
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2021004842A MX2021004842A (es) | 2018-11-09 | 2019-11-08 | Adn de extremo cerrado (ceadn) modificado que comprende repeticiones terminales invertidas modificadas simetricas. |
Country Status (13)
Country | Link |
---|---|
US (1) | US20210388379A1 (es) |
EP (1) | EP3877528A4 (es) |
JP (1) | JP2022506771A (es) |
KR (1) | KR20210090619A (es) |
CN (1) | CN113316640A (es) |
AU (1) | AU2019376663A1 (es) |
BR (1) | BR112021007102A2 (es) |
CA (1) | CA3119310A1 (es) |
IL (1) | IL282925A (es) |
MA (1) | MA54188A (es) |
MX (1) | MX2021004842A (es) |
SG (1) | SG11202104743WA (es) |
WO (1) | WO2020097417A1 (es) |
Families Citing this family (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA3146966A1 (en) * | 2019-07-17 | 2021-01-21 | Generation Bio Co. | Compositions and production of nicked closed-ended dna vectors |
CA3147414A1 (en) * | 2019-07-17 | 2021-01-21 | Generation Bio Co. | Synthetic production of single-stranded adeno associated viral dna vectors |
EP4189098A1 (en) | 2020-07-27 | 2023-06-07 | Anjarium Biosciences AG | Compositions of dna molecules, methods of making therefor, and methods of use thereof |
CN117802161A (zh) * | 2022-06-30 | 2024-04-02 | 苏州吉恒基因科技有限公司 | 精准重组腺相关病毒载体及其用途 |
CN115896135B (zh) * | 2022-11-02 | 2024-03-01 | 苏州诺洁贝生物技术有限公司 | 优化的pah基因和表达盒及其用途 |
US11993783B1 (en) * | 2023-03-27 | 2024-05-28 | Genecraft Inc. | Nucleic acid molecule comprising asymmetrically modified ITR for improving expression rate of inserted gene, and use thereof |
CN116110602B (zh) * | 2023-04-13 | 2023-06-20 | 云南医无界医疗网络科技有限公司 | 一种应用于医共体的信息处理方法及系统 |
Family Cites Families (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
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 |
JP6831779B2 (ja) * | 2015-06-23 | 2021-02-17 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia | 修飾された第ix因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
ES2898337T3 (es) * | 2016-03-03 | 2022-03-07 | Univ Massachusetts | ADN dúplex lineal de extremos cerrados para la transferencia de genes no víricos |
MX2020005790A (es) * | 2018-01-19 | 2020-10-28 | Generation Bio Co | Vectores de adn de extremo cerrado que pueden obtenerse de síntesis libre de células y un proceso para obtener vectores de ceadn. |
BR112020017060A2 (pt) * | 2018-02-22 | 2020-12-15 | Generation Bio Co. | Expressão controlada de transgenes usando vetores de dna de extremidade fechada (cedna) |
-
2019
- 2019-11-08 WO PCT/US2019/060395 patent/WO2020097417A1/en active Application Filing
- 2019-11-08 KR KR1020217012806A patent/KR20210090619A/ko active Search and Examination
- 2019-11-08 MA MA054188A patent/MA54188A/fr unknown
- 2019-11-08 SG SG11202104743WA patent/SG11202104743WA/en unknown
- 2019-11-08 JP JP2021524359A patent/JP2022506771A/ja active Pending
- 2019-11-08 MX MX2021004842A patent/MX2021004842A/es unknown
- 2019-11-08 AU AU2019376663A patent/AU2019376663A1/en active Pending
- 2019-11-08 US US17/290,787 patent/US20210388379A1/en active Pending
- 2019-11-08 EP EP19881504.5A patent/EP3877528A4/en active Pending
- 2019-11-08 BR BR112021007102-4A patent/BR112021007102A2/pt unknown
- 2019-11-08 CN CN201980073843.5A patent/CN113316640A/zh active Pending
- 2019-11-08 CA CA3119310A patent/CA3119310A1/en active Pending
-
2021
- 2021-05-04 IL IL282925A patent/IL282925A/en unknown
Also Published As
Publication number | Publication date |
---|---|
IL282925A (en) | 2021-06-30 |
JP2022506771A (ja) | 2022-01-17 |
BR112021007102A2 (pt) | 2021-08-03 |
SG11202104743WA (en) | 2021-06-29 |
CN113316640A (zh) | 2021-08-27 |
MA54188A (fr) | 2021-09-15 |
EP3877528A4 (en) | 2022-11-30 |
AU2019376663A1 (en) | 2021-06-24 |
WO2020097417A9 (en) | 2020-06-18 |
EP3877528A1 (en) | 2021-09-15 |
KR20210090619A (ko) | 2021-07-20 |
WO2020097417A1 (en) | 2020-05-14 |
CA3119310A1 (en) | 2020-05-14 |
US20210388379A1 (en) | 2021-12-16 |
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