HUS2300003I1 - VIII. faktort kódoló gént tartalmazó AAV vektorok - Google Patents
VIII. faktort kódoló gént tartalmazó AAV vektorokInfo
- Publication number
- HUS2300003I1 HUS2300003I1 HUS2300003C HUS2300003C HUS2300003I1 HU S2300003 I1 HUS2300003 I1 HU S2300003I1 HU S2300003 C HUS2300003 C HU S2300003C HU S2300003 C HUS2300003 C HU S2300003C HU S2300003 I1 HUS2300003 I1 HU S2300003I1
- Authority
- HU
- Hungary
- Prior art keywords
- gene encoding
- factor viii
- aav vectors
- encoding factor
- aav
- 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
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/36—Blood coagulation or fibrinolysis factors
- A61K38/37—Factors VIII
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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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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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
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/745—Blood coagulation or fibrinolysis factors
- C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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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
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
-
- 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
- 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
-
- 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
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- 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
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Hematology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Toxicology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361877042P | 2013-09-12 | 2013-09-12 | |
| EP14771729.2A EP3044231B1 (en) | 2013-09-12 | 2014-09-10 | Aav vectors comprising a gene encoding factor viii |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HUS2300003I1 true HUS2300003I1 (hu) | 2023-01-28 |
Family
ID=51585244
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE14771729A HUE050484T2 (hu) | 2013-09-12 | 2014-09-10 | VIII. faktort kódoló gént tartalmazó AAV vektorok |
| HUS2300003C HUS2300003I1 (hu) | 2013-09-12 | 2023-01-11 | VIII. faktort kódoló gént tartalmazó AAV vektorok |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE14771729A HUE050484T2 (hu) | 2013-09-12 | 2014-09-10 | VIII. faktort kódoló gént tartalmazó AAV vektorok |
Country Status (33)
| Country | Link |
|---|---|
| US (5) | US9504762B2 (hu) |
| EP (2) | EP3770169A1 (hu) |
| JP (4) | JP6735672B2 (hu) |
| KR (2) | KR20230006058A (hu) |
| CN (2) | CN112538501A (hu) |
| AR (1) | AR097657A1 (hu) |
| AU (1) | AU2014318890B2 (hu) |
| CA (3) | CA2921232C (hu) |
| CL (1) | CL2016000502A1 (hu) |
| CY (1) | CY1124213T1 (hu) |
| DK (1) | DK3044231T3 (hu) |
| ES (1) | ES2813698T3 (hu) |
| FI (1) | FIC20230007I1 (hu) |
| FR (1) | FR23C1007I1 (hu) |
| HK (1) | HK1226416A1 (hu) |
| HR (1) | HRP20201324T1 (hu) |
| HU (2) | HUE050484T2 (hu) |
| IL (2) | IL244404B (hu) |
| LT (2) | LT3044231T (hu) |
| MX (2) | MX2016003262A (hu) |
| NL (1) | NL301222I2 (hu) |
| NO (1) | NO2023003I1 (hu) |
| NZ (1) | NZ716549A (hu) |
| PE (1) | PE20160769A1 (hu) |
| PH (1) | PH12016500328A1 (hu) |
| PL (1) | PL3044231T3 (hu) |
| PT (1) | PT3044231T (hu) |
| RS (1) | RS61039B1 (hu) |
| RU (1) | RU2710307C2 (hu) |
| SG (1) | SG11201601932UA (hu) |
| SI (1) | SI3044231T1 (hu) |
| TW (1) | TWI632239B (hu) |
| WO (1) | WO2015038625A1 (hu) |
Families Citing this family (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| JP6735672B2 (ja) | 2013-09-12 | 2020-08-05 | バイオマリン ファーマシューティカル インコーポレイテッド | アデノ随伴ウイルス第ｖｉｉｉ因子ベクター |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| DK3270944T3 (da) | 2015-03-17 | 2020-01-27 | Univ Brussel Vrije | Optimerede leverspecifikke ekspressionssystemer til FVIII og FIX |
| TWI707951B (zh) * | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| WO2016168728A2 (en) * | 2015-04-16 | 2016-10-20 | Emory University | Recombinant promoters and vectors for protein expression in liver and use thereof |
| MX2018003702A (es) | 2015-09-24 | 2018-04-30 | Biomarin Pharm Inc | Vectores del factor viii del virus adenoasociado, particulas viricas asociadas y formulaciones terapeuticas que las comprenden. |
| CA3002524A1 (en) * | 2015-10-28 | 2017-05-04 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
| PE20231949A1 (es) * | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| TW202302627A (zh) * | 2015-11-13 | 2023-01-16 | 日商武田藥品工業股份有限公司 | 用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體 |
| BR112018009732A8 (pt) | 2015-11-13 | 2019-02-26 | Baxalta GmbH | ?polinucleotídeo, vetor de vírus adeno-associado, partícula de vírus adeno-associado, célula hospedeira, métodos para produzir uma partícula de vírus adeno-associado, para tratar hemofilia a, para transduzir uma célula hospedeira, e, uso de uma partícula de vírus adeno-associado? |
| KR102450833B1 (ko) * | 2016-04-15 | 2022-10-05 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 혈우병 a 치료용 유전자 요법 |
| WO2017205573A1 (en) | 2016-05-25 | 2017-11-30 | Lonza Houston Inc. | Methods for isolating adeno-associated virus using a polydiallyldialkylammonium salt |
| CN109715650B (zh) | 2016-07-26 | 2024-03-08 | 生物马林药物股份有限公司 | 新颖腺相关病毒衣壳蛋白 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| MX2020001402A (es) * | 2017-08-01 | 2021-01-29 | Spark Therapeutics Inc | Metodos de terapia genica del factor viii (fviii). |
| SG11202010830WA (en) | 2018-05-09 | 2020-11-27 | Biomarin Pharm Inc | Methods of treating phenylketonuria |
| SG11202010832YA (en) | 2018-05-14 | 2020-11-27 | Biomarin Pharm Inc | Stable expression of aav vectors in juvenile subjects |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| CA3106590A1 (en) | 2018-07-16 | 2020-01-23 | Baxalta Incorporated | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| WO2020020475A1 (en) * | 2018-07-27 | 2020-01-30 | Zentrum Für Forschungsförderung In Der Pädiatrie Gmbh | Improved concepts for the treatment of genetic disorders with high-capacity plal-generated gold nanoparticles |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| MX2021004213A (es) * | 2018-10-12 | 2021-07-16 | Genzyme Corp | Generacion de pah humana mejorada para el tratamiento de pku grave mediante terapia de sustitucion de genes dirigida al higado. |
| TW202039546A (zh) | 2019-01-16 | 2020-11-01 | 美商巴克斯歐塔公司 | 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體 |
| WO2020168362A1 (en) | 2019-02-15 | 2020-08-20 | Crispr Therapeutics Ag | Gene editing for hemophilia a with improved factor viii expression |
| US20220177545A1 (en) * | 2019-03-13 | 2022-06-09 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing fviii therapeutics |
| JP6831943B1 (ja) | 2019-10-24 | 2021-02-17 | 三ツ星ベルト株式会社 | 摩擦伝動ベルトおよびその製造方法 |
| CN114929735A (zh) * | 2019-11-01 | 2022-08-19 | 自由行疗法有限公司 | 因子viii构建体 |
| US20230023826A1 (en) | 2019-12-12 | 2023-01-26 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| WO2021236908A2 (en) | 2020-05-20 | 2021-11-25 | Biomarin Pharmaceutical Inc. | Use of regulatory proteins for the production of adeno-associated virus |
| IL302128A (en) | 2020-11-02 | 2023-06-01 | Biomarin Pharm Inc | A process for the enrichment of adeno-associated virus |
| WO2022264040A1 (en) | 2021-06-14 | 2022-12-22 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| JP2023002483A (ja) * | 2021-06-22 | 2023-01-10 | ファイザー・インク | 昆虫細胞におけるアデノ随伴ウイルスベクターの産生 |
| EP4377466A1 (en) * | 2021-07-28 | 2024-06-05 | BioMarin Pharmaceutical Inc. | Large scale adeno-associated virus production systems |
| WO2023028456A1 (en) * | 2021-08-23 | 2023-03-02 | Bioverativ Therapeutics Inc. | Optimized factor viii genes |
| WO2023036054A2 (en) * | 2021-09-08 | 2023-03-16 | Inspirar Limited | Composition and method for treating hemophilia |
| WO2023135273A2 (en) | 2022-01-14 | 2023-07-20 | Anjarium Biosciences Ag | Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof |
| TW202400796A (zh) * | 2022-03-11 | 2024-01-01 | 美商同源醫藥公司 | 雙向雙重啟動子表現載體及其用途 |
| WO2023205300A2 (en) * | 2022-04-20 | 2023-10-26 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii |
| WO2024064856A1 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of cardiomyopathy with aav gene therapy vectors |
| WO2024064863A2 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors |
| CN115948408A (zh) * | 2022-09-23 | 2023-04-11 | 上海信致医药科技有限公司 | 改进的人凝血因子viii基因表达盒及其应用 |
| WO2024074143A1 (en) * | 2022-10-08 | 2024-04-11 | Lingyi Biotech Co., Ltd. | Construct for enhancing gene expression |
| CN116555266A (zh) * | 2023-06-09 | 2023-08-08 | 呈诺再生医学科技(北京)有限公司 | 肝脏特异性表达的重组启动子和载体及其在肝脏相关疾病治疗中的应用 |
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| IE58011B1 (en) | 1983-05-27 | 1993-06-16 | Texas A & M Univ Sys | Method for producing a recombinant baculovirus expression vector |
| DK518384A (da) | 1984-01-31 | 1985-07-01 | Idaho Res Found | Vektor til fremstilling af et gen-produkt i insektceller, fremgangsmaade til dens fremstilling samt dens anvendelse |
| DE69435108D1 (de) * | 1993-07-13 | 2008-08-14 | Centelion | Defekte adenovirus-vektoren und deren verwendung in der gentherapie |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| JP2001500376A (ja) | 1996-09-06 | 2001-01-16 | カイロン コーポレイション | 組換えａａｖベクターを使用する治療分子の肝臓特異的送達のための方法および組成物 |
| AU5689699A (en) * | 1998-08-24 | 2000-03-14 | Uab Research Foundation | Methods of producing high titer recombinant adeno-associated virus |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| US6221349B1 (en) | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| EP2438931B1 (en) | 2004-09-22 | 2013-11-13 | St. Jude Children's Research Hospital | Improved expression of factor IX in gene therapy vectors |
| CN1286981C (zh) * | 2004-11-30 | 2006-11-29 | 华中科技大学同济医学院附属同济医院 | 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法 |
| EP1739179A1 (en) * | 2005-06-30 | 2007-01-03 | Octapharma AG | Serum-free stable transfection and production of recombinant human proteins in human cell lines |
| US7943374B2 (en) * | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| US7943379B2 (en) | 2008-04-30 | 2011-05-17 | Nationwide Children's Hospital, Inc. | Production of rAAV in vero cells using particular adenovirus helpers |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| CN103215308B (zh) * | 2013-02-01 | 2015-12-23 | 中国科学院苏州生物医学工程技术研究所 | 表达重组人fviii的整合质粒、细胞株及其构建方法和应用 |
| JP6735672B2 (ja) * | 2013-09-12 | 2020-08-05 | バイオマリン ファーマシューティカル インコーポレイテッド | アデノ随伴ウイルス第ｖｉｉｉ因子ベクター |
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