MX2020002500A - Adn de extremo cerrado modificado (adnec). - Google Patents
Adn de extremo cerrado modificado (adnec).Info
- Publication number
- MX2020002500A MX2020002500A MX2020002500A MX2020002500A MX2020002500A MX 2020002500 A MX2020002500 A MX 2020002500A MX 2020002500 A MX2020002500 A MX 2020002500A MX 2020002500 A MX2020002500 A MX 2020002500A MX 2020002500 A MX2020002500 A MX 2020002500A
- Authority
- MX
- Mexico
- Prior art keywords
- cedna
- cedna vectors
- linear
- vectors
- ended dna
- Prior art date
Links
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
-
- 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
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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
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
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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
- C12N15/66—General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
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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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- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
- C12N2710/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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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
- C12N2800/00—Nucleic acids 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
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/60—Vectors comprising a special translation-regulating system from viruses
Landscapes
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Cell Biology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
Abstract
La presente invención se refiere a vectores de ADNec que tienen estructura lineal y continua pueden ser producidos en altos rendimientos y usados para la transferencia y expresión efectiva de un transgén. Los vectores de ADNec comprenden un casete de expresión y dos diferentes secuencias de ITR derivadas de genomas de AAV en un orden especificado. Algunos vectores de ADNec proporcionados en la presente comprenden además, elementos cis-reguladores y proporcionan eficiencias de alta expresión de gen. Además se proporciona en la presente, métodos y líneas celulares para la producción confiable y eficiente de vectores de ADN lineales, continuos y libres de cápsido.
Applications Claiming Priority (7)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762556281P | 2017-09-08 | 2017-09-08 | |
US201762556329P | 2017-09-08 | 2017-09-08 | |
US201762556335P | 2017-09-08 | 2017-09-08 | |
US201762556324P | 2017-09-08 | 2017-09-08 | |
US201762556331P | 2017-09-08 | 2017-09-08 | |
US201762556319P | 2017-09-08 | 2017-09-08 | |
PCT/US2018/049996 WO2019051255A1 (en) | 2017-09-08 | 2018-09-07 | CLOSED-ENDED DNA (CEDNA) MODIFIED |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2020002500A true MX2020002500A (es) | 2020-09-17 |
Family
ID=65635235
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2020002500A MX2020002500A (es) | 2017-09-08 | 2018-09-07 | Adn de extremo cerrado modificado (adnec). |
Country Status (14)
Country | Link |
---|---|
US (1) | US20200283794A1 (es) |
EP (1) | EP3678710A4 (es) |
JP (2) | JP2020532981A (es) |
KR (1) | KR20200051011A (es) |
CN (1) | CN111132699A (es) |
AU (1) | AU2018327348A1 (es) |
BR (1) | BR112020004151A2 (es) |
CA (1) | CA3075168A1 (es) |
IL (1) | IL272797A (es) |
MA (1) | MA50100A (es) |
MX (1) | MX2020002500A (es) |
PH (1) | PH12020500465A1 (es) |
SG (1) | SG11202000698SA (es) |
WO (1) | WO2019051255A1 (es) |
Families Citing this family (27)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
WO2015105955A1 (en) | 2014-01-08 | 2015-07-16 | Flodesign Sonics, Inc. | Acoustophoresis device with dual acoustophoretic chamber |
US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
AU2018378672A1 (en) * | 2017-12-06 | 2020-07-09 | Generation Bio Co. | Gene editing using a modified closed-ended dna (ceDNA) |
KR102439221B1 (ko) | 2017-12-14 | 2022-09-01 | 프로디자인 소닉스, 인크. | 음향 트랜스듀서 구동기 및 제어기 |
WO2019161059A1 (en) * | 2018-02-14 | 2019-08-22 | Generation Bio Co. | Non-viral dna vectors and uses thereof for antibody and fusion protein production |
GB201905651D0 (en) | 2019-04-24 | 2019-06-05 | Lightbio Ltd | Nucleic acid constructs and methods for their manufacture |
CA3147414A1 (en) * | 2019-07-17 | 2021-01-21 | Generation Bio Co. | Synthetic production of single-stranded adeno associated viral dna vectors |
US20220228171A1 (en) * | 2019-07-17 | 2022-07-21 | Generation Bio Co. | Compositions and production of nicked closed-ended dna vectors |
CN114929205A (zh) * | 2019-09-06 | 2022-08-19 | 世代生物公司 | 包括末端封闭式dna和可切割脂质的脂质纳米颗粒组合物及其使用方法 |
CA3151464A1 (en) * | 2019-09-18 | 2021-03-25 | Bruce C. SCHNEPP | Synthetic dna vectors and methods of use |
WO2021072031A1 (en) * | 2019-10-11 | 2021-04-15 | Insideoutbio, Inc. | Methods and compositions for the manufacture and use of circular dna encoded therapeutics for genetic disorders and other diseases |
WO2021169167A1 (en) * | 2020-02-29 | 2021-09-02 | Nanjing GenScript Biotech Co., Ltd. | Method for treating coronavirus infections |
BR112023001648A2 (pt) | 2020-07-27 | 2023-04-04 | Anjarium Biosciences Ag | Moléculas de dna de fita dupla, veículo de entrega e método para preparar uma molécula de dna com extremidade em grampo |
EP4199971A1 (en) | 2020-08-23 | 2023-06-28 | 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) |
GB202014751D0 (en) * | 2020-09-18 | 2020-11-04 | Lightbio Ltd | Targeting vector |
WO2022232029A2 (en) * | 2021-04-26 | 2022-11-03 | University Of Florida Research Foundation, Incorporated | Synthetic aav vectors for repeated delivery of therapeutic genes |
KR20240011714A (ko) * | 2021-04-27 | 2024-01-26 | 제너레이션 바이오 컴퍼니 | 치료용 항체를 발현하는 비바이러스성 dna 벡터 및 이의 용도 |
WO2022232286A1 (en) * | 2021-04-27 | 2022-11-03 | Generation Bio Co. | Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof |
CN117729934A (zh) * | 2021-05-07 | 2024-03-19 | 世代生物公司 | 用于疫苗递送的非病毒dna载体 |
WO2022236016A1 (en) * | 2021-05-07 | 2022-11-10 | Generation Bio Co. | Lyophilized non-viral dna vector compositions and uses thereof |
WO2023177655A1 (en) | 2022-03-14 | 2023-09-21 | Generation Bio Co. | Heterologous prime boost vaccine compositions and methods of use |
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 |
WO2024040222A1 (en) * | 2022-08-19 | 2024-02-22 | Generation Bio Co. | Cleavable closed-ended dna (cedna) and methods of use thereof |
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 |
Family Cites Families (10)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20050019824A1 (en) * | 1994-03-08 | 2005-01-27 | Human Genome Sciences, Inc. | Fibroblast Growth Factor-10 |
US20050026838A1 (en) * | 1995-06-05 | 2005-02-03 | Human Genome Sciences, Inc. | Fibroblast Growth Factor-13 |
DE10066104A1 (de) * | 2000-09-08 | 2003-01-09 | Medigene Ag | Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung |
US20060166363A1 (en) * | 2004-01-27 | 2006-07-27 | Sergei Zolotukhin | Modified baculovirus expression system for production of pseudotyped rAAV vector |
US9150882B2 (en) * | 2006-01-31 | 2015-10-06 | The Board Of Trustees Of The Leland Stanford Junior University | Self-complementary parvoviral vectors, and methods for making and using the same |
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 |
WO2014003553A1 (en) * | 2012-06-27 | 2014-01-03 | Arthrogen B.V. | Combination for treating an inflammatory disorder |
CN104087613B (zh) * | 2014-06-30 | 2017-08-29 | 中国科学院苏州生物医学工程技术研究所 | 基于aav‑itr的基因表达微载体及其构建方法和应用 |
CN108883100B (zh) * | 2016-01-15 | 2022-11-25 | 美国基因技术国际有限公司 | 用于活化γ-δT细胞的方法和组合物 |
SG11201806663TA (en) * | 2016-03-03 | 2018-09-27 | Univ Massachusetts | Closed-ended linear duplex dna for non-viral gene transfer |
-
2018
- 2018-09-07 KR KR1020207009737A patent/KR20200051011A/ko not_active Application Discontinuation
- 2018-09-07 US US16/644,568 patent/US20200283794A1/en active Pending
- 2018-09-07 MX MX2020002500A patent/MX2020002500A/es unknown
- 2018-09-07 CA CA3075168A patent/CA3075168A1/en active Pending
- 2018-09-07 JP JP2020512843A patent/JP2020532981A/ja active Pending
- 2018-09-07 WO PCT/US2018/049996 patent/WO2019051255A1/en unknown
- 2018-09-07 EP EP18854941.4A patent/EP3678710A4/en active Pending
- 2018-09-07 AU AU2018327348A patent/AU2018327348A1/en active Pending
- 2018-09-07 SG SG11202000698SA patent/SG11202000698SA/en unknown
- 2018-09-07 CN CN201880058155.7A patent/CN111132699A/zh active Pending
- 2018-09-07 MA MA050100A patent/MA50100A/fr unknown
- 2018-09-07 BR BR112020004151-3A patent/BR112020004151A2/pt unknown
-
2020
- 2020-02-20 IL IL272797A patent/IL272797A/en unknown
- 2020-03-06 PH PH12020500465A patent/PH12020500465A1/en unknown
-
2022
- 2022-11-02 JP JP2022176371A patent/JP2022190081A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
SG11202000698SA (en) | 2020-03-30 |
JP2020532981A (ja) | 2020-11-19 |
PH12020500465A1 (en) | 2021-01-25 |
KR20200051011A (ko) | 2020-05-12 |
IL272797A (en) | 2020-04-30 |
EP3678710A4 (en) | 2021-06-09 |
EP3678710A1 (en) | 2020-07-15 |
JP2022190081A (ja) | 2022-12-22 |
CN111132699A (zh) | 2020-05-08 |
AU2018327348A1 (en) | 2020-02-20 |
MA50100A (fr) | 2020-07-15 |
CA3075168A1 (en) | 2019-03-14 |
BR112020004151A2 (pt) | 2020-09-08 |
RU2020109904A (ru) | 2021-10-08 |
WO2019051255A1 (en) | 2019-03-14 |
US20200283794A1 (en) | 2020-09-10 |
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