JP2012521750A - 肝硬変及び肝線維症の治療のための方法及び組成物 - Google Patents

肝硬変及び肝線維症の治療のための方法及び組成物 Download PDF

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JP2012521750A
JP2012521750A JP2012501327A JP2012501327A JP2012521750A JP 2012521750 A JP2012521750 A JP 2012521750A JP 2012501327 A JP2012501327 A JP 2012501327A JP 2012501327 A JP2012501327 A JP 2012501327A JP 2012521750 A JP2012521750 A JP 2012521750A
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aav
igf
liver
vector
virion
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JP2012521750A5 (https=
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アロンソ、マリア プリフィカシオン フォルテス
バルトゥエーニャ、ヘスス マリア プリエト
マティーアス ソブレバルス、ルシアノ
ペトリー、ハラルド
ヤコブス フベルトゥス ティンメルマンス、エリク
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Proyecto de Biomedicina CIMA SL
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    • AHUMAN NECESSITIES
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Proteomics, Peptides & Aminoacids (AREA)
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  • 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)
JP2012501327A 2009-03-27 2010-03-26 肝硬変及び肝線維症の治療のための方法及び組成物 Pending JP2012521750A (ja)

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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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JP2012521750A true JP2012521750A (ja) 2012-09-20
JP2012521750A5 JP2012521750A5 (https=) 2013-05-09

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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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020511127A (ja) * 2017-03-15 2020-04-16 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒルThe University Of North Carolina At Chapel Hill ポリプロイド性アデノ随伴ウイルスベクターならびにその作製および使用の方法
US11702676B2 (en) 2017-03-15 2023-07-18 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors for the treatment of disease
JP2024506681A (ja) * 2021-02-15 2024-02-14 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 rAAV及びrBV産生のためのトランスフェクション試薬としてのヒスチジンリッチペプチドの使用

Families Citing this family (37)

* 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
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
EP4351755A4 (en) * 2021-06-11 2025-04-23 Spark Therapeutics, Inc. AAV vector column purification method
NL2033264B1 (en) * 2022-10-10 2024-04-26 Amarna Holding B V Composition and method for the production of polyomaviral vector particles
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
CN116555266B (zh) * 2023-06-09 2024-11-12 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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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 (34)

* 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
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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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 (6)

* Cited by examiner, † Cited by third party
Title
JPN5012010823; VERA M: 'LIVER TRANSDUCTION WITH A SIMIAN VIRUS 40 VECTOR ENCODING INSULIN-LIKE GROWTH FACTOR I REDUCES HEPAT' GENE THERAPY V14 N3, 200702, P203-210 *
JPN5012010824; SOBREVALS L: '89: TREATMENT OF ESTABLISHED CIRRHOSIS USING SV40 VECTOR ENCODING INSULIN-LIKE GROWTH FACTOR I' JOURNAL OF HEPATOLOGY V48, 20080101, P S39, MUNKSGAARD INTERNATIONAL PUBLISHERS *
JPN5012010825; SOBREVALS LUCIANO: 'INSULIN-LIKE GROWTH FACTOR I GENE TRANSFER TO CIRRHOTIC LIVER INDUCES FIBROLYSIS AND REDUCES FIBROGE' HEPATOLOGY V51 N3, 201003, P912-921 *
JPN6014036163; McCarty D. et al.: Gene Therapy Vol.10, 2003, pp.2112-2118 *
JPN6014036166; McCarty D. et al.: Gene Therapy Vol.8, 2001, pp.1248-1254 *
JPN6014036168; McCarty D. M.: Molecular Therapy Vol.16 No.10, 2008, pp.1648-1656 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020511127A (ja) * 2017-03-15 2020-04-16 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒルThe University Of North Carolina At Chapel Hill ポリプロイド性アデノ随伴ウイルスベクターならびにその作製および使用の方法
JP7132934B2 (ja) 2017-03-15 2022-09-07 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル ポリプロイド性アデノ随伴ウイルスベクターならびにその作製および使用の方法
US11702676B2 (en) 2017-03-15 2023-07-18 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors for the treatment of disease
US12584147B2 (en) 2017-03-15 2026-03-24 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors for the treatment of disease
JP2024506681A (ja) * 2021-02-15 2024-02-14 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 rAAV及びrBV産生のためのトランスフェクション試薬としてのヒスチジンリッチペプチドの使用

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