JP2001517221A5 - - Google Patents

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Publication number
JP2001517221A5
JP2001517221A5 JP1998540610A JP54061098A JP2001517221A5 JP 2001517221 A5 JP2001517221 A5 JP 2001517221A5 JP 1998540610 A JP1998540610 A JP 1998540610A JP 54061098 A JP54061098 A JP 54061098A JP 2001517221 A5 JP2001517221 A5 JP 2001517221A5
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JP
Japan
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.)
Granted
Application number
JP1998540610A
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English (en)
Japanese (ja)
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JP2001517221A (ja
JP4289687B2 (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US1998/004790 external-priority patent/WO1998041240A1/en
Publication of JP2001517221A publication Critical patent/JP2001517221A/ja
Publication of JP2001517221A5 publication Critical patent/JP2001517221A5/ja
Application granted granted Critical
Publication of JP4289687B2 publication Critical patent/JP4289687B2/ja
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

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JP54061098A 1997-03-14 1998-03-12 血友病の治療のための遺伝子治療で使用する方法と組成物 Expired - Lifetime JP4289687B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US4071197P 1997-03-14 1997-03-14
US60/040,711 1997-03-14
PCT/US1998/004790 WO1998041240A1 (en) 1997-03-14 1998-03-12 Methods and compositions for use in gene therapy for treatment of hemophilia

Publications (3)

Publication Number Publication Date
JP2001517221A JP2001517221A (ja) 2001-10-02
JP2001517221A5 true JP2001517221A5 (enExample) 2005-11-10
JP4289687B2 JP4289687B2 (ja) 2009-07-01

Family

ID=21912503

Family Applications (1)

Application Number Title Priority Date Filing Date
JP54061098A Expired - Lifetime JP4289687B2 (ja) 1997-03-14 1998-03-12 血友病の治療のための遺伝子治療で使用する方法と組成物

Country Status (9)

Country Link
US (3) US6093392A (enExample)
EP (1) EP1005376B1 (enExample)
JP (1) JP4289687B2 (enExample)
AT (1) ATE486614T1 (enExample)
AU (1) AU745386B2 (enExample)
CA (1) CA2283639A1 (enExample)
DE (1) DE69841982D1 (enExample)
ES (1) ES2355588T3 (enExample)
WO (1) WO1998041240A1 (enExample)

Families Citing this family (57)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6531455B1 (en) * 1995-03-24 2003-03-11 The Regents Of The University Of California Delivery of polynucleotides by secretory gland expression
US5885971A (en) * 1995-03-24 1999-03-23 The Regents Of The University Of California Gene therapy by secretory gland expression
US20020037867A1 (en) * 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
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
US6797505B2 (en) * 1998-05-27 2004-09-28 Cell Genesys, Inc. Recombinant AAV vectors for gene therapy of hemophilia A
DE69939169D1 (de) * 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
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
CA2364417C (en) * 1999-02-19 2013-11-05 Matthew John During Peroral gene therapy of diabetes and obesity
WO2000074688A1 (en) * 1999-06-08 2000-12-14 The Children's Hospital Of Philadelphia Methods for preventing formation of inhibitory antibodies in the setting of gene therapy
US6610906B1 (en) * 1999-06-09 2003-08-26 The Regents Of The University Of Michigan Nucleotide sequences for gene regulation and methods of use thereof
WO2001049118A1 (en) * 2000-01-03 2001-07-12 Panagenic International, Inc. Compositions comprising genome segments and methods of using the same
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
AU6814901A (en) * 2000-06-01 2001-12-11 Univ North Carolina Methods and compounds for controlled release of recombinant parvovirus vectors
US20020106381A1 (en) * 2000-06-13 2002-08-08 High Katherine A. Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus
WO2002040544A2 (en) * 2000-11-14 2002-05-23 Board Of Regents, University Of Texas Systems Mutant human factor ix with an increased resistance to inhibition by heparin
US6794376B2 (en) * 2000-12-06 2004-09-21 William Beaumont Hospital Methods and compositions for enhancing diffusion of therapeutic agents through tissue
US20020159978A1 (en) * 2001-02-06 2002-10-31 James Allen Muscle-directed gene transfer by use of recombinant AAV-1 and AAV-6 virions
EP1418810A4 (en) 2001-08-03 2006-08-02 Us Gov Health & Human Serv ORAL TREATMENT OF HEMOPHILIA
EP2518142B1 (en) 2001-08-24 2015-07-15 UVic Industry Partnerships Inc. Proaerolysin containing protease activation sequences and methods of use for treatment of prostate cancer
US20030198620A1 (en) * 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
US20040092008A1 (en) * 2002-11-12 2004-05-13 Snyder Richard O. Recombinant AAV vectors for gene therapy of hemophilia A
WO2005017101A2 (en) 2003-05-19 2005-02-24 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH Avian adenoassociated virus (aaav) and uses thereof
PL2657248T3 (pl) 2003-06-19 2017-09-29 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
WO2005056807A2 (en) 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Bovine adeno-associated viral (baav) vector and uses thereof
WO2005077333A2 (en) * 2004-02-10 2005-08-25 University Of Florida Research Foundation, Inc. Gel-based delivery of recombinant adeno-associated virus vectors
US7722596B2 (en) * 2004-02-26 2010-05-25 Osprey Medical, Inc. Regional cardiac tissue treatment
ES2436741T3 (es) 2004-09-22 2014-01-07 St. Jude Children's Research Hospital Expresión mejorada de Factor IX en vectores de terapia génica
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
WO2006089340A2 (en) * 2005-02-23 2006-08-31 V-Kardia Pty Ltd Polynucleotide delivery to cardiac tissue
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
US8152786B2 (en) * 2006-11-07 2012-04-10 Osprey Medical, Inc. Collection catheter and kit
US9050400B2 (en) * 2008-08-12 2015-06-09 Osprey Medical, Inc. Remote sensing catheter system and methods
US20100041984A1 (en) * 2008-08-12 2010-02-18 James Edward Shapland Impedance sensing device and catheter system
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
FR2952374B1 (fr) * 2009-11-06 2012-11-16 Univ Claude Bernard Lyon Production de facteur ix recombinant dans une lignee cellulaire hepatocytaire humaine
US9724400B2 (en) 2009-11-09 2017-08-08 The Trustees Of The University Of Pennsylvania Administration of plant expressed oral tolerance agents
KR20130028044A (ko) * 2009-12-08 2013-03-18 헬스 리서치 인코포레이티드 보조제로서 퍼옥시레독신1 (prx1)을 사용하는 방법 및 조성물
US9295816B2 (en) * 2009-12-09 2016-03-29 Osprey Medical, Inc. Catheter with distal and proximal ports
EP2529020B1 (en) 2010-01-28 2018-04-25 The Children's Hospital of Philadelphia A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
EP2627665B1 (en) 2010-10-12 2015-12-16 The Children's Hospital Of Philadelphia Methods and compositions for treating hemophilia B
KR20220025112A (ko) 2011-06-08 2022-03-03 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
ES2664844T3 (es) 2011-06-20 2018-04-23 Mount Sinai School Of Medicine Tratamiento dirigido contra TNF para las mucopolisacaridosis y otros trastornos lisosómicos
EP2741804B1 (en) 2011-08-11 2017-04-26 Osprey Medical Inc. Systems and methods for limb treatment
WO2015006743A1 (en) * 2013-07-12 2015-01-15 The Children's Hospital Of Philadelphia Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies
US10612041B2 (en) 2014-03-21 2020-04-07 The Board Of Trustees Of The Leland Stanford Junior University Genome editing without nucleases
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
US11015174B2 (en) * 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
CN109804432B (zh) 2016-04-15 2023-07-14 武田药品工业株式会社 提供药代动力学药物给药方案的方法和设备
US10896749B2 (en) 2017-01-27 2021-01-19 Shire Human Genetic Therapies, Inc. Drug monitoring tool
TWI753168B (zh) 2017-05-22 2022-01-21 日商武田藥品工業股份有限公司 用於血友病b基因療法之編碼具有增強表現的重組fix變異體之病毒載體
WO2019083171A2 (ko) 2017-10-26 2019-05-02 주식회사 큐로진생명과학 솔루블 vegfr-1 변이체 cdna를 함유하는 raav를 포함하는 황반변성 치료용 조성물
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
JP7578590B2 (ja) 2018-10-18 2024-11-06 インテリア セラピューティクス,インコーポレーテッド 第ix因子を発現するための組成物及び方法

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1984000560A1 (en) * 1982-08-04 1984-02-16 Nat Res Dev Molecular cloning of the gene for human anti-haemophilic factor ix
US5139941A (en) * 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
NZ244306A (en) * 1991-09-30 1995-07-26 Boehringer Ingelheim Int Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation
US5834266A (en) * 1993-02-12 1998-11-10 President & Fellows Of Harvard College Regulated apoptosis
US6342390B1 (en) * 1994-11-23 2002-01-29 The United States Of America As Represented By The Secretary Of Health And Human Services Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy
US5843742A (en) * 1994-12-16 1998-12-01 Avigen Incorporated Adeno-associated derived vector systems for gene delivery and integration into target cells
US5834306A (en) * 1994-12-23 1998-11-10 Sri International Tissue specific hypoxia regulated therapeutic constructs
US5846528A (en) * 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5858351A (en) * 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5817784A (en) * 1996-08-09 1998-10-06 Amgen Inc. Neurogene
EP1696036B1 (en) * 1996-09-06 2010-04-21 The Trustees of The University of Pennsylvania Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells
US5866552A (en) * 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
JP2001501819A (ja) * 1996-09-25 2001-02-13 セル・ジェネシス・インコーポレイテッド アデノ随伴ウィルスベクターの非侵襲性投与

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