JP2001517221A5 - - Google Patents

Download PDF

Info

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
Authority
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
Other languages
English (en)
Japanese (ja)
Other versions
JP4289687B2 (ja
JP2001517221A (ja
Filing date
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

Links

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
US5885971A (en) * 1995-03-24 1999-03-23 The Regents Of The University Of California Gene therapy by secretory gland expression
US6531455B1 (en) * 1995-03-24 2003-03-11 The Regents Of The University Of California Delivery of polynucleotides by secretory gland expression
US20020037867A1 (en) * 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES 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
ES2313784T3 (es) * 1998-05-28 2009-03-01 The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Vector aav5 y usos del mismo.
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
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
ATE374251T1 (de) * 1999-02-19 2007-10-15 Engene Inc Zusammensetzungen zur gentherapie von diabetes
US8063022B1 (en) 1999-06-08 2011-11-22 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
US7217699B2 (en) * 2000-01-03 2007-05-15 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
AU2001268373A1 (en) * 2000-06-13 2001-12-24 The Children's Hospital Of Philadelphia Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus
AU2002226028A1 (en) * 2000-11-14 2002-05-27 Board Of Regents, Unversity 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
WO2003013244A1 (en) 2001-08-03 2003-02-20 The Government Of The United State Of America, As Represented By The Secretary Of The Department Of Health And Human Services Oral treatment of hemophilia
DK1567641T3 (da) 2001-08-24 2012-08-27 Uvic Industry Partnerships Inc Proaerolysin indeholdende proteaseaktiveringssekvenser og fremgangsmåder til anvendelse til behandling af prostatacancer.
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
PL3235827T3 (pl) 2003-06-19 2021-07-05 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowania
US8137960B2 (en) 2003-12-04 2012-03-20 The United States Of America As Represented By The Department Of Health And Human Services Bovine adeno-associated viral (BAAV) vector and uses thereof
US20060078542A1 (en) * 2004-02-10 2006-04-13 Mah Cathryn S Gel-based delivery of recombinant adeno-associated virus vectors
US7722596B2 (en) * 2004-02-26 2010-05-25 Osprey Medical, Inc. Regional cardiac tissue treatment
DK1804839T3 (da) 2004-09-22 2012-04-10 St Jude Childrens Res Hospital Forbedret ekspression af faktor ix i genterapivektorer
CA2591544A1 (en) 2004-12-15 2006-06-22 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
ES2634118T3 (es) 2009-02-11 2017-09-26 The University Of North Carolina At Chapel Hill Vectores de virus modificados y métodos para fabricar y utilizar los mismos
FR2952374B1 (fr) * 2009-11-06 2012-11-16 Univ Claude Bernard Lyon Production de facteur ix recombinant dans une lignee cellulaire hepatocytaire humaine
CA2780362C (en) 2009-11-09 2019-12-24 University Of Central Florida Research Foundation, Inc. Administration of plant expressed oral tolerance agents
WO2011071988A1 (en) * 2009-12-08 2011-06-16 Health Research, Inc. Methods and compositions using peroxiredoxin 1 (prx1) as an adjuvant
US9295816B2 (en) * 2009-12-09 2016-03-29 Osprey Medical, Inc. Catheter with distal and proximal ports
PT2529020T (pt) 2010-01-28 2018-07-30 Childrens Hospital Philadelphia Plataforma de fabrico escalável para purificação de vetor viral e vetores virais assim purificados para utilização na terapia génica
WO2012051343A1 (en) * 2010-10-12 2012-04-19 The Children's Hospital Of Philadelphia Methods and compositions for treating hemophilia b
US20140206753A1 (en) * 2011-06-08 2014-07-24 Shire Human Genetic Therapies, Inc. Lipid nanoparticle compositions and methods for mrna delivery
EP2721151B1 (en) 2011-06-20 2017-12-06 Mount Sinai School Of Medicine Anti-tnf-therapy for the mucopolysaccharidoses and other lysosomal disorders
US9211372B2 (en) 2011-08-11 2015-12-15 Osprey Medical, Inc. Systems and methods for limb treatment
RU2705249C2 (ru) * 2013-07-12 2019-11-06 Дзе Чилдрен'З Хоспитал Оф Филадельфия Вектор aav и анализ на нейтрализующие антитела против aav (аденоассоциированного вируса)
CA2939847C (en) 2014-03-21 2023-09-05 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
ES2934848T3 (es) * 2015-12-11 2023-02-27 Univ Pennsylvania Método de purificación escalable para AAV8
SG11201808475XA (en) 2016-04-15 2018-10-30 Baxalta Inc Method and apparatus for providing a pharmacokinetic drug dosing regiment
US10896749B2 (en) 2017-01-27 2021-01-19 Shire Human Genetic Therapies, Inc. Drug monitoring tool
KR102386890B1 (ko) 2017-05-22 2022-04-15 다케다 야쿠힌 고교 가부시키가이샤 B형 혈우병 유전자 요법을 위한 증가된 발현을 가지는 재조합 fix 변이체를 암호화하는 바이러스 벡터
WO2019083171A2 (ko) 2017-10-26 2019-05-02 주식회사 큐로진생명과학 솔루블 vegfr-1 변이체 cdna를 함유하는 raav를 포함하는 황반변성 치료용 조성물
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
BR112021007301A2 (pt) 2018-10-18 2021-07-27 Intellia Therapeutics, Inc. composições e métodos para expressar fator ix

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2625412B2 (ja) * 1982-08-04 1997-07-02 ブリティッシュ・テクノロジー・グループ・リミテッド ヒト第9因子ポリペプチドをコードするcdna
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
ATE465267T1 (de) * 1996-09-06 2010-05-15 Univ Pennsylvania Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen
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
WO1998013070A1 (en) * 1996-09-25 1998-04-02 Cell Genesys, Inc. Non-invasive administration of adeno-associated viral vectors

Similar Documents

Publication Publication Date Title
JP2000509942A5 (enExample)
JP2000509635A5 (enExample)
JP2002501700A5 (enExample)
JP2000509912A5 (enExample)
JP2000509637A5 (enExample)
JP2001513697A5 (enExample)
JP2000510751A5 (enExample)
JP2002512729A5 (enExample)
JP2002502385A5 (enExample)
JP2002508713A5 (enExample)
JP2002500513A5 (enExample)
JP2000509587A5 (enExample)
JP2001517221A5 (enExample)
JP2000508866A5 (enExample)
JP2000510793A5 (enExample)
JP2000508841A5 (enExample)
JP2002501480A5 (enExample)
JP2000508680A5 (enExample)
JP2000509804A5 (enExample)
JP2000509853A5 (enExample)
JP2000509755A5 (enExample)
JP2000510969A5 (enExample)
JP2000510247A5 (enExample)
JP2000508102A5 (enExample)
JP2000508836A5 (enExample)