HRP20211697T1 - Zatvorena linearna dupleks dnk za nevirusni prijenos gena - Google Patents
Zatvorena linearna dupleks dnk za nevirusni prijenos gena Download PDFInfo
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- HRP20211697T1 HRP20211697T1 HRP20211697TT HRP20211697T HRP20211697T1 HR P20211697 T1 HRP20211697 T1 HR P20211697T1 HR P20211697T T HRP20211697T T HR P20211697TT HR P20211697 T HRP20211697 T HR P20211697T HR P20211697 T1 HRP20211697 T1 HR P20211697T1
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- Prior art keywords
- cedna
- nucleic acid
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- 108700005077 Viral Genes Proteins 0.000 title 1
- 108020004707 nucleic acids Proteins 0.000 claims 17
- 102000039446 nucleic acids Human genes 0.000 claims 17
- 150000007523 nucleic acids Chemical class 0.000 claims 17
- 210000004027 cell Anatomy 0.000 claims 13
- 238000000034 method Methods 0.000 claims 7
- 101710150114 Protein rep Proteins 0.000 claims 6
- 101710152114 Replication protein Proteins 0.000 claims 6
- 239000000758 substrate Substances 0.000 claims 6
- 238000005096 rolling process Methods 0.000 claims 5
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 108091081548 Palindromic sequence Proteins 0.000 claims 3
- 239000005547 deoxyribonucleotide Substances 0.000 claims 3
- 125000002637 deoxyribonucleotide group Chemical group 0.000 claims 3
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims 2
- 238000010362 genome editing Methods 0.000 claims 2
- 230000028993 immune response Effects 0.000 claims 2
- 238000000338 in vitro Methods 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 239000013598 vector Substances 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- 101100524317 Adeno-associated virus 2 (isolate Srivastava/1982) Rep40 gene Proteins 0.000 claims 1
- 101100524321 Adeno-associated virus 2 (isolate Srivastava/1982) Rep68 gene Proteins 0.000 claims 1
- 241001203868 Autographa californica Species 0.000 claims 1
- 108091033409 CRISPR Proteins 0.000 claims 1
- 108090000565 Capsid Proteins Proteins 0.000 claims 1
- 102100023321 Ceruloplasmin Human genes 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 241000238631 Hexapoda Species 0.000 claims 1
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 1
- VYPSYNLAJGMNEJ-UHFFFAOYSA-N Silicium dioxide Chemical compound O=[Si]=O VYPSYNLAJGMNEJ-UHFFFAOYSA-N 0.000 claims 1
- 108700019146 Transgenes Proteins 0.000 claims 1
- 230000001580 bacterial effect Effects 0.000 claims 1
- 230000036755 cellular response Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000013604 expression vector Substances 0.000 claims 1
- 230000008348 humoral response Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000004806 packaging method and process Methods 0.000 claims 1
- 239000002245 particle Substances 0.000 claims 1
- 230000004962 physiological condition Effects 0.000 claims 1
- 238000002360 preparation method Methods 0.000 claims 1
- 238000000746 purification Methods 0.000 claims 1
- 230000003362 replicative effect Effects 0.000 claims 1
- 239000011347 resin Substances 0.000 claims 1
- 229920005989 resin Polymers 0.000 claims 1
- 239000000741 silica gel Substances 0.000 claims 1
- 229910002027 silica gel Inorganic materials 0.000 claims 1
- 241000701447 unidentified baculovirus Species 0.000 claims 1
- 241000701366 unidentified nuclear polyhedrosis viruses Species 0.000 claims 1
- 210000005253 yeast cell Anatomy 0.000 claims 1
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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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
-
- 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
- A61K48/005—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
-
- 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
- A61K48/0075—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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
- A61P11/12—Mucolytics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- 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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- C12N15/00—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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- C12N15/102—Mutagenizing nucleic acids
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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
- C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
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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
- C12N15/66—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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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/14011—Baculoviridae
- C12N2710/14041—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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- 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
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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
- C12N2750/14141—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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- 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
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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 (5)
| 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 | |
| EP17760963.3A EP3423110B1 (en) | 2016-03-03 | 2017-03-03 | Closed-ended linear duplex dna for non-viral gene transfer |
| 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 |
|---|---|
| HRP20211697T1 true HRP20211697T1 (hr) | 2022-02-04 |
Family
ID=59743259
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20211697TT HRP20211697T1 (hr) | 2016-03-03 | 2017-03-03 | Zatvorena linearna dupleks dnk za nevirusni prijenos gena |
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) |
Families Citing this family (54)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
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| BR112021007102A2 (pt) * | 2018-11-09 | 2021-08-03 | Generation Bio Co. | dna com extremidade fechada modificada (cedna) que compreende repetições terminais invertidas modificadas simétricas |
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