CN102448501A - 治疗肝硬化和肝纤维化的方法和组合物 - Google Patents
治疗肝硬化和肝纤维化的方法和组合物 Download PDFInfo
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- CN102448501A CN102448501A CN2010800230018A CN201080023001A CN102448501A CN 102448501 A CN102448501 A CN 102448501A CN 2010800230018 A CN2010800230018 A CN 2010800230018A CN 201080023001 A CN201080023001 A CN 201080023001A CN 102448501 A CN102448501 A CN 102448501A
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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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
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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
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/575—Hormones
- C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
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- 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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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N2710/14111—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
- C12N2750/14141—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
- C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
- C12N2770/00011—Details
- C12N2770/22011—Dicistroviridae
- C12N2770/22041—Use of virus, viral particle or viral elements as a vector
- C12N2770/22043—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
- 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
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Immunology (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Toxicology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Mycology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| ESP200900846 | 2009-03-27 | ||
| ES200900846 | 2009-03-27 | ||
| PCT/ES2010/070184 WO2010109053A1 (es) | 2009-03-27 | 2010-03-26 | Métodos y composiciones para el tratamiento de cirrosis y fibrosis hepática |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN102448501A true CN102448501A (zh) | 2012-05-09 |
Family
ID=42269789
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN2010800230018A Pending CN102448501A (zh) | 2009-03-27 | 2010-03-26 | 治疗肝硬化和肝纤维化的方法和组合物 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US20120093775A1 (enExample) |
| EP (1) | EP2412387B1 (enExample) |
| JP (1) | JP2012521750A (enExample) |
| CN (1) | CN102448501A (enExample) |
| AU (1) | AU2010227419A1 (enExample) |
| CA (1) | CA2756596A1 (enExample) |
| MX (1) | MX2011010097A (enExample) |
| WO (1) | WO2010109053A1 (enExample) |
Cited By (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN115656517A (zh) * | 2022-10-24 | 2023-01-31 | 南方医科大学南方医院 | 一种用于肝硬化食管胃静脉曲张的诊断和分层的蛋白标记物及其应用 |
| 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 |
| CN118430796A (zh) * | 2024-07-05 | 2024-08-02 | 杭州生奥信息技术有限公司 | 基于血液生化指标的肝纤维化无创诊断方法和系统 |
| EP4351755A4 (en) * | 2021-06-11 | 2025-04-23 | Spark Therapeutics, Inc. | AAV vector column purification method |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2453019A4 (en) * | 2009-07-10 | 2013-04-17 | Univ Barcelona Autonoma | Gene therapy compositions for the prevention and / or treatment of autoimmune diseases |
| WO2011154308A1 (en) * | 2010-06-08 | 2011-12-15 | Proyecto De Biomedicina Cima, S.L. | New compositions and cell therapy methods for the treatment of cirrhosis |
| WO2014012025A2 (en) * | 2012-07-13 | 2014-01-16 | The Trustees Of The University Of Pennsylvania | Igf-1 proteins and therapeutic uses thereof |
| AU2014354839B2 (en) | 2013-11-26 | 2020-06-04 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Adeno-associated virus vectors for treatment of glycogen storage disease |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CA2969145A1 (en) * | 2014-11-26 | 2016-06-02 | The Regents Of The University Of California | Therapeutic compositions comprising transcription factors and methods of making and using the same |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| WO2016106303A1 (en) | 2014-12-23 | 2016-06-30 | THE UNITED STATES OF AMERICA, as representd by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES | Adeno-associated virus vectors encoding modified g6pc and uses thereof |
| EP3384035A4 (en) | 2015-12-02 | 2019-08-07 | Voyager Therapeutics, Inc. | ASSAYS FOR THE DETECTION OF AAV-NEUTRALIZING ANTIBODIES |
| MX2018012537A (es) | 2016-04-15 | 2019-02-25 | Univ Pennsylvania | Terapia de genes para tratar hemofilia a. |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| KR102427379B1 (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| NZ756587A (en) * | 2017-03-15 | 2025-12-19 | Univ North Carolina Chapel Hill | Polyploid adeno-associated virus vectors and methods of making and using the same |
| JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | 筋萎縮性側索硬化症(als)治療組成物および方法 |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | ハンチントン病治療組成物および方法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CA3070087A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| WO2019028273A1 (en) * | 2017-08-02 | 2019-02-07 | Dna2.0, Inc. Dba Atum | DNA VECTORS AND ELEMENTS FOR SUSTAINED GENE EXPRESSION IN EUKARYOTIC CELLS |
| EP3808849A1 (en) | 2017-08-03 | 2021-04-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| US11549125B2 (en) | 2017-08-09 | 2023-01-10 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Closed-ended, linear, duplex adenoassociated virus DNA, and uses thereof |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| EP3697908A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| KR20210019996A (ko) | 2018-05-15 | 2021-02-23 | 보이저 테라퓨틱스, 인크. | 파킨슨병의 치료를 위한 조성물 및 방법 |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CN111135311B (zh) * | 2018-11-02 | 2023-06-13 | 中国科学院分子细胞科学卓越创新中心 | Ecm1在预防和/或治疗肝纤维化相关疾病中的应用 |
| AU2019407921A1 (en) * | 2018-12-19 | 2021-06-10 | Versameb Ag | RNA encoding a protein |
| US20230295238A1 (en) * | 2020-07-23 | 2023-09-21 | The Scripps Research Institute | Insulin receptor-mediated enhancement of gene transfer |
| CN116997656A (zh) * | 2021-02-15 | 2023-11-03 | 生物马林药物股份有限公司 | 富含组氨酸的肽作为用于rAAV和rBV生产的转染试剂的用途 |
| NL2033264B1 (en) * | 2022-10-10 | 2024-04-26 | Amarna Holding B V | Composition and method for the production of polyomaviral vector particles |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008137490A2 (en) * | 2007-05-01 | 2008-11-13 | Genzyme Corporation | Systemic insulin-like growth factor-1 therapy reduces diabetic peripheral neuropathy and improves renal function in diabetic nephropathy |
Family Cites Families (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| EP0208491B1 (en) | 1985-07-03 | 1993-08-25 | Genencor International, Inc. | Hybrid polypeptides and process for their preparation |
| ATE81779T1 (de) | 1985-08-22 | 1992-11-15 | Gropep Pty Ltd | Peptidanalogedes insulinaehnlichen wachstumsfaktors-1 bei saeugetieren. |
| US4876242A (en) | 1987-09-21 | 1989-10-24 | Merck & Co., Inc. | Human insulin-like growth factor analoges with reduced binding to serum carrier proteins and their production in yeast |
| ATE116335T1 (de) | 1987-12-24 | 1995-01-15 | Gropep Pty Ltd | Peptid-analoge vom insulinähnlichen wachstums- faktor 1 (igf-1) oder faktor 2 (igf-2). |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| US5605793A (en) | 1994-02-17 | 1997-02-25 | Affymax Technologies N.V. | Methods for in vitro recombination |
| US5837458A (en) | 1994-02-17 | 1998-11-17 | Maxygen, Inc. | Methods and compositions for cellular and metabolic engineering |
| US6117679A (en) | 1994-02-17 | 2000-09-12 | Maxygen, Inc. | Methods for generating polynucleotides having desired characteristics by iterative selection and recombination |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| CA2207927A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| WO1998009524A1 (en) * | 1996-09-06 | 1998-03-12 | Chiron Corporation | Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors |
| WO1998011244A2 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| JP2001506132A (ja) | 1996-12-18 | 2001-05-15 | ターゲティッド ジェネティクス コーポレイション | Aavベクターの産生における使用のためのリコンビナーゼ活性化可能aavパッケージングカセット |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6251677B1 (en) | 1997-08-25 | 2001-06-26 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| US6103526A (en) | 1998-10-08 | 2000-08-15 | Protein Sciences Corporation | Spodoptera frugiperda single cell suspension cell line in serum-free media, methods of producing and using |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| US6387368B1 (en) | 1999-02-08 | 2002-05-14 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| ATE318923T1 (de) | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
| 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 |
| WO2003042361A2 (en) | 2001-11-09 | 2003-05-22 | Government Of The United States Of America, Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| AU2003212708A1 (en) | 2002-03-05 | 2003-09-16 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| SI1633772T1 (sl) | 2003-06-19 | 2016-06-30 | Genzyme Corporation | AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen |
| US20060166363A1 (en) | 2004-01-27 | 2006-07-27 | Sergei Zolotukhin | Modified baculovirus expression system for production of pseudotyped rAAV vector |
| EP2311966A3 (en) | 2005-10-20 | 2012-09-05 | Amsterdam Molecular Therapeutics (AMT) B.V. | Improved AAV vectors produced in insect cells |
| US20080045609A1 (en) * | 2006-03-12 | 2008-02-21 | Devore Dianna L | Methods and therapeutics to facilitate liver repair |
| CN103849629B (zh) | 2006-06-21 | 2017-06-09 | 尤尼克尔Ip股份有限公司 | 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体 |
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2010
- 2010-03-26 US US13/260,654 patent/US20120093775A1/en not_active Abandoned
- 2010-03-26 MX MX2011010097A patent/MX2011010097A/es not_active Application Discontinuation
- 2010-03-26 CN CN2010800230018A patent/CN102448501A/zh active Pending
- 2010-03-26 AU AU2010227419A patent/AU2010227419A1/en not_active Abandoned
- 2010-03-26 EP EP10714045.1A patent/EP2412387B1/en active Active
- 2010-03-26 WO PCT/ES2010/070184 patent/WO2010109053A1/es not_active Ceased
- 2010-03-26 CA CA2756596A patent/CA2756596A1/en not_active Abandoned
- 2010-03-26 JP JP2012501327A patent/JP2012521750A/ja active Pending
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2013
- 2013-05-07 US US13/888,748 patent/US20130273006A1/en not_active Abandoned
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008137490A2 (en) * | 2007-05-01 | 2008-11-13 | Genzyme Corporation | Systemic insulin-like growth factor-1 therapy reduces diabetic peripheral neuropathy and improves renal function in diabetic nephropathy |
Non-Patent Citations (4)
| Title |
|---|
| L.SOBREVALS ECT.AL: "89 Treatment of established cirrhosis using SV40 vector endoding insulin-like growth factor I", 《JOURNAL OF HEPATOLOGY》 * |
| M.GABRIELA KRAMER ECT.AL: "In vitro and in vivo comparative study of chimeric liver-specific promoters", 《MOLECULAR THERAPY》 * |
| MARK P CHADWICK: "《modification of retroviral tropsim by display of IGF-I》", 《JOURNAL OF MOLECULAR BIOLOGY》 * |
| 岳凤鸣: "腺相关病毒介导的胰岛素样生长因子1基因治疗糖尿病的相关研究", 《河北医科大学》 * |
Cited By (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4351755A4 (en) * | 2021-06-11 | 2025-04-23 | Spark Therapeutics, Inc. | AAV vector column purification method |
| CN115656517A (zh) * | 2022-10-24 | 2023-01-31 | 南方医科大学南方医院 | 一种用于肝硬化食管胃静脉曲张的诊断和分层的蛋白标记物及其应用 |
| CN115656517B (zh) * | 2022-10-24 | 2023-09-12 | 南方医科大学南方医院 | 一种用于肝硬化食管胃静脉曲张的诊断和分层的蛋白标记物及其应用 |
| 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 |
| CN118430796A (zh) * | 2024-07-05 | 2024-08-02 | 杭州生奥信息技术有限公司 | 基于血液生化指标的肝纤维化无创诊断方法和系统 |
Also Published As
| Publication number | Publication date |
|---|---|
| US20130273006A1 (en) | 2013-10-17 |
| AU2010227419A1 (en) | 2011-10-20 |
| WO2010109053A8 (es) | 2011-11-17 |
| JP2012521750A (ja) | 2012-09-20 |
| WO2010109053A1 (es) | 2010-09-30 |
| US20120093775A1 (en) | 2012-04-19 |
| EP2412387B1 (en) | 2014-11-19 |
| EP2412387A1 (en) | 2012-02-01 |
| CA2756596A1 (en) | 2010-09-30 |
| MX2011010097A (es) | 2011-10-19 |
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