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

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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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aav
igf
sequence
liver
virion
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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
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Proyecto de Biomedicina CIMA SL
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    • 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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  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
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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)

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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

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US (2) US20120093775A1 (enExample)
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CN (1) CN102448501A (enExample)
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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
JP6649265B2 (ja) * 2013-11-26 2020-02-19 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 糖原病の処置のためのアデノ随伴ウイルスベクター
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
US20180057839A1 (en) * 2014-11-26 2018-03-01 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
EP3236984B1 (en) 2014-12-23 2020-07-22 The U.S.A. as represented by the Secretary, Department of Health and Human Services Adeno-associated virus vectors encoding modified g6pc and uses thereof
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
PE20181922A1 (es) 2016-04-15 2018-12-11 Univ Pennsylvania Terapia genica para tratar la hemofilia a
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
EP3831281B1 (en) 2016-08-30 2026-02-25 The Regents of The University of California Devices and systems for biomedical targeting and delivery
WO2018170310A1 (en) * 2017-03-15 2018-09-20 The University Of North Carolina At Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using 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
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
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
US11512327B2 (en) 2017-08-03 2022-11-29 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
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
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
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
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
JP2022514863A (ja) * 2018-12-19 2022-02-16 ヴェルサメブ アーゲー タンパク質をコードするｒｎａ
US20230295238A1 (en) * 2020-07-23 2023-09-21 The Scripps Research Institute Insulin receptor-mediated enhancement of gene transfer
WO2022173893A1 (en) * 2021-02-15 2022-08-18 Biomarin Pharmaceutical Inc. USE OF HISTIDINE RICH PEPTIDES AS A TRANSFECTION REAGENT FOR rAAV AND rBV PRODUCTION
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
DE3688920D1 (de) 1985-07-03 1993-09-30 Genencor Int Hybride Polypeptide und Verfahren zu deren Herstellung.
AU598205B2 (en) 1985-08-22 1990-06-21 Gropep Pty Ltd Peptide analogues of mammalian insulin-like growth factor-1
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
EP0346429B1 (en) 1987-12-24 1994-12-28 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
DE69433922T2 (de) 1993-11-09 2005-07-28 Medical College Of Ohio, Toledo Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
US5605793A (en) 1994-02-17 1997-02-25 Affymax Technologies N.V. Methods for in vitro recombination
US6117679A (en) 1994-02-17 2000-09-12 Maxygen, Inc. Methods for generating polynucleotides having desired characteristics by iterative selection and 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
WO1996017947A1 (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
EP0933997A4 (en) * 1996-09-06 2000-08-16 Chiron Corp METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
AU5603998A (en) 1996-12-18 1998-07-15 Targeted Genetics Corporation Recombinase-activatable AAV packaging cassettes for use in the production of AV 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
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
DE69939169D1 (de) 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
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
DE69936104T2 (de) 1998-11-05 2008-01-17 The Trustees Of The University Of Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill 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
US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect 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
PL2657248T3 (pl) 2003-06-19 2017-09-29 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
PT3272872T (pt) 2005-10-20 2020-06-26 Uniqure Ip Bv Vetores aav melhorados produzidos em células de insetos
US20080045609A1 (en) * 2006-03-12 2008-02-21 Devore Dianna L Methods and therapeutics to facilitate liver repair
WO2007148971A2 (en) 2006-06-21 2007-12-27 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells

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
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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

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US20120093775A1 (en) 2012-04-19
EP2412387B1 (en) 2014-11-19
WO2010109053A8 (es) 2011-11-17
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CA2756596A1 (en) 2010-09-30
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