MX2018010633A - Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral. - Google Patents
Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.Info
- Publication number
- MX2018010633A MX2018010633A MX2018010633A MX2018010633A MX2018010633A MX 2018010633 A MX2018010633 A MX 2018010633A MX 2018010633 A MX2018010633 A MX 2018010633A MX 2018010633 A MX2018010633 A MX 2018010633A MX 2018010633 A MX2018010633 A MX 2018010633A
- Authority
- MX
- Mexico
- Prior art keywords
- closed
- gene transfer
- viral gene
- duplex dna
- interrupted
- Prior art date
Links
- 108700005077 Viral Genes Proteins 0.000 title 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 3
- 102000039446 nucleic acids Human genes 0.000 abstract 3
- 108020004707 nucleic acids Proteins 0.000 abstract 3
Classifications
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- C—CHEMISTRY; METALLURGY
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- 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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- 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
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- 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
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- 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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- 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
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- C12N2710/00011—Details
- C12N2710/14011—Baculoviridae
- C12N2710/14041—Use of virus, viral particle or viral elements as a vector
- C12N2710/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vectore
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Epidemiology (AREA)
- Obesity (AREA)
- Gastroenterology & Hepatology (AREA)
- Virology (AREA)
- Cell Biology (AREA)
- Pulmonology (AREA)
- Ophthalmology & Optometry (AREA)
- Emergency Medicine (AREA)
- Endocrinology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Crystallography & Structural Chemistry (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662303047P | 2016-03-03 | 2016-03-03 | |
| US201662394720P | 2016-09-14 | 2016-09-14 | |
| US201662406913P | 2016-10-11 | 2016-10-11 | |
| PCT/US2017/020828 WO2017152149A1 (en) | 2016-03-03 | 2017-03-03 | Closed-ended linear duplex dna for non-viral gene transfer |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2018010633A true MX2018010633A (es) | 2019-06-13 |
Family
ID=59743259
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2018010633A MX2018010633A (es) | 2016-03-03 | 2017-03-03 | Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral. |
Country Status (18)
| Country | Link |
|---|---|
| US (3) | US11066679B2 (enExample) |
| EP (2) | EP3423110B1 (enExample) |
| JP (3) | JP6994140B2 (enExample) |
| KR (1) | KR102336362B1 (enExample) |
| CN (2) | CN115287301A (enExample) |
| AU (2) | AU2017228413B2 (enExample) |
| CA (1) | CA3014683A1 (enExample) |
| DK (1) | DK3423110T3 (enExample) |
| ES (1) | ES2898337T3 (enExample) |
| HR (1) | HRP20211697T1 (enExample) |
| IL (2) | IL302398B2 (enExample) |
| LT (1) | LT3423110T (enExample) |
| MX (1) | MX2018010633A (enExample) |
| PT (1) | PT3423110T (enExample) |
| RU (1) | RU2752882C2 (enExample) |
| SG (2) | SG11201806663TA (enExample) |
| WO (1) | WO2017152149A1 (enExample) |
| ZA (1) | ZA201806544B (enExample) |
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| CN107106689A (zh) | 2014-11-05 | 2017-08-29 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| KR102584655B1 (ko) | 2014-11-14 | 2023-10-06 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| PT3423110T (pt) | 2016-03-03 | 2021-11-09 | Univ Massachusetts | Adn em dúplex linear de extremidades fechadas para transferência génica não viral |
| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| JP7374883B2 (ja) | 2017-08-09 | 2023-11-07 | バイオベラティブ セラピューティクス インコーポレイテッド | 核酸分子およびその使用 |
| WO2019051255A1 (en) * | 2017-09-08 | 2019-03-14 | Generation Bio Co. | CLOSED-ENDED DNA (CEDNA) MODIFIED |
| EP3679148A4 (en) * | 2017-09-08 | 2021-06-09 | Generation Bio Co. | LIPID NANOPARTICLE FORMULATIONS FROM NONVIRAL, CAPSID-FREE DNA VECTORS |
| EP3684422B1 (en) | 2017-09-20 | 2023-12-20 | The Regents of the University of California | Connexin-43 gene therapy to restore electrical and cardiac function, and cardiac structure, in arrhythmogenic right ventricular cardiomyopathy |
| US12098385B2 (en) | 2017-09-20 | 2024-09-24 | The Regents Of The University Of California | Gene therapy strategy to restore cardiac electrical and structural function in arrhythmogenic right ventricular cardiomyopathy |
| US11680296B2 (en) * | 2017-10-16 | 2023-06-20 | Massachusetts Institute Of Technology | Mycobacterium tuberculosis host-pathogen interaction |
| US20220290186A1 (en) * | 2017-12-06 | 2022-09-15 | Generation Bio Co. | Gene editing using a modified closed-ended dna (cedna) |
| US20210371877A1 (en) * | 2017-12-18 | 2021-12-02 | Spark Therapeutics, Inc. | Adeno-associated virus (aav) vector lipid nanoparticle compositions and methods of use |
| EP3740571A4 (en) * | 2018-01-19 | 2021-12-08 | Generation Bio Co. | CLOSED-TERM DNA VECTORS OBTAINED FROM CELL-FREE SYNTHESIS AND METHOD FOR OBTAINING CEDNA VECTORS |
| KR20200120649A (ko) * | 2018-02-14 | 2020-10-21 | 제너레이션 바이오 컴퍼니 | 비-바이러스 dna 벡터 및 항체 및 융합 단백질 생산을 위한 이의 용도 |
| BR112020017060A2 (pt) * | 2018-02-22 | 2020-12-15 | Generation Bio Co. | Expressão controlada de transgenes usando vetores de dna de extremidade fechada (cedna) |
| CA3092459A1 (en) * | 2018-03-02 | 2019-09-06 | Generation Bio Co. | Closed-ended dna (cedna) vectors for insertion of transgenes at genomic safe harbors (gsh) in humans and murine genomes |
| CN117757842A (zh) * | 2018-03-15 | 2024-03-26 | 星际治疗有限公司 | 合成dna运载体及使用方法 |
| WO2019191109A1 (en) * | 2018-03-28 | 2019-10-03 | The Board Of Trustees Of The Leland Stanford Junior University | Microrna inhibitors and their use in treating epiretinal membranes |
| MX2020012077A (es) | 2018-05-15 | 2021-03-09 | Voyager Therapeutics Inc | Composiciones y metodos para el tratamiento de la enfermedad de parkinson. |
| EP3796893A1 (en) | 2018-05-23 | 2021-03-31 | Modernatx, Inc. | Delivery of dna |
| WO2019237383A1 (zh) * | 2018-06-16 | 2019-12-19 | 深圳市博奥康生物科技有限公司 | 用于人 tnfsf18 基因编辑的修饰载体、其制备方法及应用 |
| WO2019237391A1 (zh) * | 2018-06-16 | 2019-12-19 | 深圳市博奥康生物科技有限公司 | CRISPR/Cas9 靶向敲除人 TXGP1 基因及其特异性 gRNA |
| WO2019246544A2 (en) * | 2018-06-22 | 2019-12-26 | Asklepios Biopharmaceutical, Inc. | Vectors for gene delivery that persist within cells |
| CN119955796A (zh) | 2018-08-09 | 2025-05-09 | 比奥维拉迪维治疗股份有限公司 | 核酸分子及其用于非病毒基因疗法的用途 |
| WO2020037254A1 (en) * | 2018-08-17 | 2020-02-20 | City Of Hope | Compounds of chemically modified oligonucleotides and methods of use thereof |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| BR112021007102A2 (pt) * | 2018-11-09 | 2021-08-03 | Generation Bio Co. | dna com extremidade fechada modificada (cedna) que compreende repetições terminais invertidas modificadas simétricas |
| AU2020211457A1 (en) * | 2019-01-24 | 2021-09-09 | Generation Bio Co. | Close-ended DNA (ceDNA) and use in methods of reducing gene or nucleic acid therapy related immune response |
| KR20210127935A (ko) * | 2019-02-15 | 2021-10-25 | 제너레이션 바이오 컴퍼니 | 폐쇄형 DNA(ceDNA) 생산에서의 Rep 단백질 활성의 변형 |
| US20220175968A1 (en) * | 2019-03-06 | 2022-06-09 | Generation Bio Co. | Non-active lipid nanoparticles with non-viral, capsid free dna |
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| WO2022023284A1 (en) | 2020-07-27 | 2022-02-03 | Anjarium Biosciences Ag | Compositions of dna molecules, methods of making therefor, and methods of use thereof |
| CA3189673A1 (en) | 2020-08-23 | 2022-03-03 | Ajay MAGHODIA | Modified baculovirus system for improved production of closed-ended dna (cedna) |
| US20230383311A1 (en) * | 2020-09-16 | 2023-11-30 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing fviii therapeutics |
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