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
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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
-
- 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
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- 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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- C—CHEMISTRY; METALLURGY
- 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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- C12N15/09—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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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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
- C12N2750/14122—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
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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)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Biochemistry (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)
- Emergency Medicine (AREA)
- Ophthalmology & Optometry (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 | |
| PCT/US2017/020828 WO2017152149A1 (en) | 2016-03-03 | 2017-03-03 | Closed-ended linear duplex dna for non-viral gene transfer |
| EP17760963.3A EP3423110B1 (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)
Families Citing this family (55)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11201703148TA (en) | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| DK3218386T3 (da) | 2014-11-14 | 2021-06-07 | Voyager Therapeutics Inc | Modulatorisk polynukleotid |
| CA3193811A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| RU2752882C2 (ru) | 2016-03-03 | 2021-08-11 | Юниверсити Оф Массачусетс | Линейная дуплексная днк с закрытым концом для невирусного переноса генов |
| AU2017267665C1 (en) | 2016-05-18 | 2023-10-05 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| KR102780287B1 (ko) | 2017-08-09 | 2025-03-14 | 바이오버라티브 테라퓨틱스 인크. | 핵산 분자 및 이의 용도 |
| MX2020002501A (es) * | 2017-09-08 | 2020-09-17 | Generation Bio Co | Formulaciones de nanoparticulas lipidas de vectores de adn libres de capsidos, no virales. |
| KR20200051011A (ko) * | 2017-09-08 | 2020-05-12 | 제너레이션 바이오 컴퍼니 | 변형된 폐쇄-종결된 dna(cedna) |
| IL273448B2 (en) | 2017-09-20 | 2025-05-01 | Univ California | A gene therapy strategy 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 |
| WO2019079362A1 (en) * | 2017-10-16 | 2019-04-25 | Massachusetts Institute Of Technology | HOST-PATHOGENIC INTERACTION OF MYCOBACTERIUM TUBERCULOSIS |
| AU2018378672A1 (en) * | 2017-12-06 | 2020-07-09 | 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 |
| SG11202005271TA (en) | 2018-01-19 | 2020-07-29 | Generation Bio Co | Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors |
| CN111818942A (zh) * | 2018-02-14 | 2020-10-23 | 世代生物公司 | 非病毒dna载体以及其用于产生抗体和融合蛋白的用途 |
| CA3092059A1 (en) * | 2018-02-22 | 2019-08-29 | Generation Bio Co. | Controlled expression of transgenes using close-ended dna (cedna) vectors |
| MA52116A (fr) * | 2018-03-02 | 2021-01-06 | Generation Bio Co | Vecteurs d'adn à extrémité fermée (cedna) pour l'insertion de transgènes au niveau de havres génomiques sécuritaires (gsh) dans des génomes humains et murins |
| CA3092088A1 (en) * | 2018-03-15 | 2019-09-19 | Igtx, Llc | Synthetic dna vectors and methods of use |
| 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 |
| US12319929B2 (en) | 2018-05-15 | 2025-06-03 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of Parkinson's disease |
| WO2019226650A1 (en) | 2018-05-23 | 2019-11-28 | 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 |
| EP3810782A2 (en) * | 2018-06-22 | 2021-04-28 | Asklepios Biopharmaceutical, Inc. | Vectors for gene delivery that persist within cells |
| BR112021002017A2 (pt) | 2018-08-09 | 2021-05-11 | Bioverativ Therapeutics Inc. | moléculas de ácido nucleico e usos das mesmas para terapia genética não viral |
| US20210310000A1 (en) * | 2018-08-17 | 2021-10-07 | City Of Hope | Compounds of chemically modified oligonucleotides and methods of use thereof |
| CA3114621A1 (en) | 2018-09-28 | 2020-04-02 | 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 |
| KR20210119416A (ko) * | 2019-01-24 | 2021-10-05 | 제너레이션 바이오 컴퍼니 | 폐쇄-말단 dna (cedna), 및 유전자 또는 핵산 치료 관련 면역 반응을 감소시키는 방법에서의 이의 용도 |
| WO2020168222A1 (en) * | 2019-02-15 | 2020-08-20 | Generation Bio Co. | Modulation of rep protein activity in closed-ended dna (cedna) production |
| MA55223A (fr) * | 2019-03-06 | 2022-01-12 | Generation Bio Co | Adn à extrémité fermée (cedna) et composés de modulation immunitaire |
| SG11202109426YA (en) * | 2019-03-06 | 2021-09-29 | Generation Bio Co | Non-active lipid nanoparticles with non-viral, capsid free dna |
| EP3938515A4 (en) * | 2019-03-13 | 2022-12-28 | Generation Bio Co. | NON-VIRAL DNA VECTORS AND THEIR USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH) THERAPEUTIC AGENTS |
| KR20210151785A (ko) * | 2019-03-13 | 2021-12-14 | 제너레이션 바이오 컴퍼니 | 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도 |
| GB2617512B (en) * | 2019-04-05 | 2023-12-27 | Earli Inc | Improved methods and compositions for synthetic biomarkers |
| CA3151464A1 (en) | 2019-09-18 | 2021-03-25 | Bruce C. SCHNEPP | Synthetic dna vectors and methods of use |
| EP4110931A4 (en) | 2020-02-25 | 2024-03-27 | University of Massachusetts | Inducible single aav system and uses thereof |
| AU2021241696A1 (en) | 2020-03-27 | 2022-12-01 | Generation Bio Co. | Novel lipids and nanoparticle compositions thereof |
| US20210330759A1 (en) * | 2020-04-22 | 2021-10-28 | The Cleveland Clinic Foundation | Methods and compositions for treating cytokine storm, ards, and acute lung injury using beta-glucocerebrosidase |
| AU2021285812A1 (en) | 2020-06-01 | 2023-01-05 | Modernatx, Inc. | Phenylalanine hydroxylase variants and uses thereof |
| CA3189740A1 (en) | 2020-07-27 | 2022-02-03 | Anjarium Biosciences Ag | Compositions of dna molecules, methods of making therefor, and methods of use thereof |
| CN116194152A (zh) | 2020-08-23 | 2023-05-30 | 比奥维拉迪维治疗股份有限公司 | 用于改进封闭端DNA（ceDNA）的产生的经修饰的杆状病毒系统 |
| AU2021342503A1 (en) * | 2020-09-16 | 2023-04-06 | Generation Bio Co. | Non-viral DNA vectors and uses thereof for expressing FVIII therapeutics |
| KR20240005903A (ko) * | 2021-05-07 | 2024-01-12 | 제너레이션 바이오 컴퍼니 | 백신 전달을 위한 비바이러스성 dna 벡터 |
| CA3218126A1 (en) * | 2021-05-07 | 2022-11-10 | Matthew MANGANIELLO | Lyophilized non-viral dna vector compositions and uses thereof |
| US20240263195A1 (en) | 2021-05-11 | 2024-08-08 | Modernatx, Inc. | Non-viral delivery of dna for prolonged polypeptide expression in vivo |
| WO2023028004A1 (en) * | 2021-08-23 | 2023-03-02 | Avirmax, Inc. | Compositions and methods for transgene expression |
| JP2024534123A (ja) | 2021-08-23 | 2024-09-18 | 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 | バキュロウイルス発現システム |
| CN114525282A (zh) * | 2022-03-04 | 2022-05-24 | 西南大学 | 一种二价核酸适配体及其设计方法 |
| IL322201A (en) * | 2023-02-17 | 2025-09-01 | Flagship Pioneering Innovations Vii Llc | DNA structures containing modified uracil |
| WO2024197242A1 (en) | 2023-03-23 | 2024-09-26 | Carbon Biosciences, Inc. | Protoparvovirus compositions comprising a protoparvovirus variant vp1 capsid polypeptide and related methods |
| WO2024249438A1 (en) * | 2023-05-26 | 2024-12-05 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Copiparvovirus genetic vector platform |
| WO2025212926A1 (en) * | 2024-04-05 | 2025-10-09 | University Of Massachusetts | Aav-mediated rhoa knockdown |
Family Cites Families (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4683195A (en) | 1986-01-30 | 1987-07-28 | Cetus Corporation | Process for amplifying, detecting, and/or-cloning nucleic acid sequences |
| US5549910A (en) | 1989-03-31 | 1996-08-27 | The Regents Of The University Of California | Preparation of liposome and lipid complex compositions |
| US5252334A (en) | 1989-09-08 | 1993-10-12 | Cygnus Therapeutic Systems | Solid matrix system for transdermal drug delivery |
| US5316908A (en) | 1990-07-13 | 1994-05-31 | Life Technologies, Inc. | Size markers for electrophoretic analysis of DNA |
| JP3218637B2 (ja) | 1990-07-26 | 2001-10-15 | 大正製薬株式会社 | 安定なリポソーム水懸濁液 |
| JP2958076B2 (ja) | 1990-08-27 | 1999-10-06 | 株式会社ビタミン研究所 | 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法 |
| US5399363A (en) | 1991-01-25 | 1995-03-21 | Eastman Kodak Company | Surface modified anticancer nanoparticles |
| US5543158A (en) | 1993-07-23 | 1996-08-06 | Massachusetts Institute Of Technology | Biodegradable injectable nanoparticles |
| AU689815B2 (en) * | 1993-08-30 | 1998-04-09 | Promega Corporation | Nucleic acid purification compositions and methods |
| US5741516A (en) | 1994-06-20 | 1998-04-21 | Inex Pharmaceuticals Corporation | Sphingosomes for enhanced drug delivery |
| US5795587A (en) | 1995-01-23 | 1998-08-18 | University Of Pittsburgh | Stable lipid-comprising drug delivery complexes and methods for their production |
| US5697899A (en) | 1995-02-07 | 1997-12-16 | Gensia | Feedback controlled drug delivery system |
| IE80468B1 (en) | 1995-04-04 | 1998-07-29 | Elan Corp Plc | Controlled release biodegradable nanoparticles containing insulin |
| US5738868A (en) | 1995-07-18 | 1998-04-14 | Lipogenics Ltd. | Liposome compositions and kits therefor |
| US5656016A (en) | 1996-03-18 | 1997-08-12 | Abbott Laboratories | Sonophoretic drug delivery system |
| US5797898A (en) | 1996-07-02 | 1998-08-25 | Massachusetts Institute Of Technology | Microchip drug delivery devices |
| US5783208A (en) | 1996-07-19 | 1998-07-21 | Theratech, Inc. | Transdermal drug delivery matrix for coadministering estradiol and another steroid |
| US7098320B1 (en) | 1996-07-29 | 2006-08-29 | Nanosphere, Inc. | Nanoparticles having oligonucleotides attached thereto and uses therefor |
| US5779708A (en) | 1996-08-15 | 1998-07-14 | Cyberdent, Inc. | Intraosseous drug delivery device and method |
| IL128780A0 (en) | 1996-09-06 | 2000-01-31 | Univ Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase |
| US6177403B1 (en) | 1996-10-21 | 2001-01-23 | The Trustees Of The University Of Pennsylvania | Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal |
| AU1750400A (en) | 1998-12-04 | 2000-06-26 | Mosaic Technologies, Inc. | Method for the immobilization of oligonucleotides |
| DE10044384A1 (de) | 2000-09-08 | 2002-04-18 | Medigene Ag | Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung |
| US8241622B2 (en) * | 2001-07-13 | 2012-08-14 | University Of Iowa Research Foundation | Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences |
| KR101133799B1 (ko) | 2006-08-18 | 2012-04-24 | 에프. 호프만-라 로슈 아게 | 폴리뉴클레오타이드의 생체내 전달을 위한 다가접합체 |
| CA2927045A1 (en) | 2006-10-03 | 2008-04-10 | Muthiah Manoharan | Lipid containing formulations |
| WO2009073809A2 (en) | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| US8507455B2 (en) | 2007-12-04 | 2013-08-13 | Alnylam Pharmaceuticals, Inc. | Folate conjugates |
| LT2440183T (lt) | 2009-06-10 | 2018-08-10 | Arbutus Biopharma Corporation | Patobulinta lipido kompozicija |
| WO2012068187A1 (en) | 2010-11-19 | 2012-05-24 | Merck Sharp & Dohme Corp. | Poly(amide) polymers for the delivery of oligonucleotides |
| 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 |
| RU2752882C2 (ru) | 2016-03-03 | 2021-08-11 | Юниверсити Оф Массачусетс | Линейная дуплексная днк с закрытым концом для невирусного переноса генов |
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