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 PDF

Info

Publication number
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
Authority
CN
China
Prior art keywords
aav
igf
sequence
liver
virion
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CN2010800230018A
Other languages
English (en)
Chinese (zh)
Inventor
玛丽亚普里菲卡西·福特斯阿隆索
赫苏斯玛丽亚·普列托巴尔图埃彻
卢西亚诺马蒂亚斯·索夫雷维斯
哈拉尔德·佩特里
艾瑞克雅各布斯胡贝图斯·蒂默曼斯
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Proyecto de Biomedicina CIMA SL
Original Assignee
Proyecto de Biomedicina CIMA SL
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Proyecto de Biomedicina CIMA SL filed Critical Proyecto de Biomedicina CIMA SL
Publication of CN102448501A publication Critical patent/CN102448501A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Endocrinology (AREA)
  • Toxicology (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN2010800230018A 2009-03-27 2010-03-26 治疗肝硬化和肝纤维化的方法和组合物 Pending CN102448501A (zh)

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 (https=)
EP (1) EP2412387B1 (https=)
JP (1) JP2012521750A (https=)
CN (1) CN102448501A (https=)
AU (1) AU2010227419A1 (https=)
CA (1) CA2756596A1 (https=)
MX (1) MX2011010097A (https=)
WO (1) WO2010109053A1 (https=)

Cited By (4)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140045923A1 (en) * 2009-07-10 2014-02-13 Universidad Autónoma De Barcelona Gene therapy compositions for preventing and/or treating 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
WO2015081101A1 (en) * 2013-11-26 2015-06-04 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
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN119876138A (zh) 2014-11-14 2025-04-25 沃雅戈治疗公司 调节性多核苷酸
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
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
JP6824169B2 (ja) 2014-12-23 2021-02-03 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
KR102450833B1 (ko) 2016-04-15 2022-10-05 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 혈우병 a 치료용 유전자 요법
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
CA3024449A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
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
CN110770346B (zh) * 2017-03-15 2024-01-12 北卡罗来纳-查佩尔山大学 多倍体腺相关病毒载体及其制备和使用方法
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
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
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
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
US11713468B2 (en) 2017-08-02 2023-08-01 Dna Twopointo Inc. DNA vectors and elements for sustained gene expression in eukaryotic cells
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Ａａｖを送達するための組成物および方法
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
US20200263199A1 (en) 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
WO2019079242A1 (en) 2017-10-16 2019-04-25 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
SG11202011296VA (en) 2018-05-15 2020-12-30 Voyager Therapeutics Inc Compositions and methods for the treatment of parkinson's disease
US12281321B2 (en) 2018-09-28 2025-04-22 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
EP4185608A4 (en) * 2020-07-23 2024-08-21 University of Florida Research Foundation, Inc. INSULIN RECEPTOR-MEDIATED ENHANCEMENT OF GENE TRANSFER
MX2023009554A (es) * 2021-02-15 2023-09-13 Biomarin Pharm Inc Uso de peptidos ricos en histidina como reactivo de transfeccion para la produccion de raav y rbv.
NL2033264B1 (en) * 2022-10-10 2024-04-26 Amarna Holding B V Composition and method for the production of polyomaviral vector particles

Citations (1)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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
JPH0720993B2 (ja) 1985-08-22 1995-03-08 グロペップ プロプライエタリー リミテッド 生長因子
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
US5164370A (en) 1987-12-24 1992-11-17 Gropep Pty. Ltd. Peptide analogues of insulin-like growth factor 1 (igf-1) or factor 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
US6117679A (en) 1994-02-17 2000-09-12 Maxygen, Inc. Methods for generating polynucleotides having desired characteristics by iterative selection and recombination
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
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
AU707866B2 (en) 1994-12-06 1999-07-22 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
US6521225B1 (en) * 1996-09-06 2003-02-18 Chiron Corporation AAV vectors
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services Aav4 vector and uses thereof
CA2269661A1 (en) 1996-12-18 1998-06-25 Targeted Genetics Corporation Recombinase-activatable aav packaging cassettes for use in the production of aav vectors
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
WO1999061643A1 (en) 1998-05-27 1999-12-02 University Of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient
EP1082413B1 (en) 1998-05-28 2008-07-23 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
CA2349838C (en) 1998-11-05 2011-06-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
NZ522840A (en) 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
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
WO2003074714A1 (en) 2002-03-05 2003-09-12 Stichting Voor De Technische Wetenschappen Baculovirus expression system
SI3235827T1 (sl) 2003-06-19 2021-05-31 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
EP2311967B1 (en) 2005-10-20 2017-09-20 UniQure IP 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
KR101589259B1 (ko) 2006-06-21 2016-02-01 유니큐어 아이피 비.브이. 곤충세포 내 ａａｖ의 생산에 유용한 ａａｖ-ｒｅｐ78의 번역을 위한 변형된 개시 코돈을 갖는 벡터

Patent Citations (1)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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
EP2412387A1 (en) 2012-02-01
WO2010109053A1 (es) 2010-09-30
CA2756596A1 (en) 2010-09-30
AU2010227419A1 (en) 2011-10-20
MX2011010097A (es) 2011-10-19
EP2412387B1 (en) 2014-11-19
JP2012521750A (ja) 2012-09-20
WO2010109053A8 (es) 2011-11-17
US20130273006A1 (en) 2013-10-17
US20120093775A1 (en) 2012-04-19

Similar Documents

Publication Publication Date Title
CN102448501A (zh) 治疗肝硬化和肝纤维化的方法和组合物
US20240318149A1 (en) AAV Vectors
JP6836999B2 (ja) アデノ随伴ウイルス変異体及びその使用方法
TWI654301B (zh) 用於基因治療之經修飾的弗里德賴希（ｆｒｉｅｄｒｅｉｃｈ）運動失調基因及載體
RU2745567C2 (ru) Конструкции нуклеиновой кислоты и векторы для генотерапии для применения для лечения болезни вильсона и других состояний
AU2013388083B2 (en) Compositions and methods for treating spinal muscular atrophy
MX2011010116A (es) Metodo de codificacion de video, metodo de decodificacion de video, aparato de codificacion de video y aparato de decodificacion de video.
JP2018505663A (ja) ウィルソン病及び他の状態の処置に使用するための核酸構築物及び遺伝子治療ベクター
KR20230029616A (ko) 종간 호환가능한 아데노-관련 바이러스 조성물 및 이의 사용 방법
KR20190016509A (ko) 최적화된 cln1 유전자 및 발현 카세트 및 그의 용도
JP2021532810A (ja) 糖原病ｉｉｉの処置のためのミニｇｄｅ
WO2021092518A1 (en) Transgene cassettes, aav vectors and aav viral vectors for the expression of human codon-optimized slc6a1
JP2023505851A (ja) ハンター病治療用のアデノ随伴ウイルスベクター
US20240376493A1 (en) Optimized ap4m1 polyneucleotides and expression cassettes and their use
CN108103102B (zh) 一种aav1病毒介导的骨骼肌特异性pck1基因表达载体及其用途
CN119331062A (zh) 靶向骨骼肌的aav衣壳蛋白变体及其用途
WO2023056367A1 (en) Slc13a5 gene therapy vectors and uses thereof
EP4323534A1 (en) Gene therapy for arrhythmogenic right ventricular cardiomyopathy
CN116445492A (zh) 用于x染色体连锁肾上腺脑白质营养不良的基因治疗药物和方法
JP2022530264A (ja) 乳児バッテン病の治療のための髄腔内および静脈内組合せ遺伝子療法
WO2025006957A2 (en) Non-neutralizing aav constructs and methods of generating and using same
Ohashi et al. 1014. Engineering Liver Tissues at the Kidney Capsule in Mice
HK1246355B (en) Adeno-associated virus variants and methods of use thereof

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
C02 Deemed withdrawal of patent application after publication (patent law 2001)
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20120509