DE60323078D1 - Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine - Google Patents

Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine

Info

Publication number
DE60323078D1
DE60323078D1 DE60323078T DE60323078T DE60323078D1 DE 60323078 D1 DE60323078 D1 DE 60323078D1 DE 60323078 T DE60323078 T DE 60323078T DE 60323078 T DE60323078 T DE 60323078T DE 60323078 D1 DE60323078 D1 DE 60323078D1
Authority
DE
Germany
Prior art keywords
vectors
expression systems
raav
genetic modification
methods
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.)
Expired - Fee Related
Application number
DE60323078T
Other languages
English (en)
Inventor
Kenneth H Warrington
Shaun R Opie
Nicholas Muzyczka
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.)
University of Florida
University of Florida Research Foundation Inc
Original Assignee
University of Florida
University of Florida Research Foundation Inc
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 University of Florida, University of Florida Research Foundation Inc filed Critical University of Florida
Application granted granted Critical
Publication of DE60323078D1 publication Critical patent/DE60323078D1/de
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
    • C12N2810/85Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
    • C12N2810/858Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian from apolipopeptides
DE60323078T 2002-05-01 2003-05-01 Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine Expired - Fee Related DE60323078D1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US37731502P 2002-05-01 2002-05-01
PCT/US2003/013583 WO2004027019A2 (en) 2002-05-01 2003-05-01 Improved raav expression systems for genetic modification of specific capsid proteins

Publications (1)

Publication Number Publication Date
DE60323078D1 true DE60323078D1 (de) 2008-10-02

Family

ID=32030574

Family Applications (1)

Application Number Title Priority Date Filing Date
DE60323078T Expired - Fee Related DE60323078D1 (de) 2002-05-01 2003-05-01 Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine

Country Status (11)

Country Link
US (2) US8802080B2 (de)
EP (1) EP1496944B1 (de)
AT (1) ATE405295T1 (de)
AU (1) AU2003295312B2 (de)
CA (1) CA2483624A1 (de)
DE (1) DE60323078D1 (de)
DK (1) DK1496944T3 (de)
HK (1) HK1071518A1 (de)
NZ (2) NZ536274A (de)
SI (1) SI1496944T1 (de)
WO (1) WO2004027019A2 (de)

Families Citing this family (73)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060093589A1 (en) * 2004-02-19 2006-05-04 Warrington Kenneth H Vp2-modified raav vector compositions and uses therefor
SI1496944T1 (sl) 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
US20040179028A1 (en) * 2003-03-12 2004-09-16 Fuji Photo Film Co., Ltd. Pixel defect correcting method, color mura correcting method and image display device
EP1618201A1 (de) * 2003-05-01 2006-01-25 University of Florida Research Foundation, Inc. Vp2-modifizierte raav-vektoren und verwendungen davon
EP1486567A1 (de) * 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Verbesserter Adeno-assoziierter Virus (AAV)-Vektor für Gentherapie
ES2329636B2 (es) 2006-02-17 2010-07-26 Universitat De Valencia, Estudi General (Participa Con El 70%) Uso del factor pedf para inducir la auto-renovacion de celulas madre.
DK2623605T3 (en) 2007-04-09 2019-03-25 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
JP2011512417A (ja) * 2008-02-20 2011-04-21 ジェンザイム・コーポレーション 血管形成の阻害
AU2009274482A1 (en) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
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
CN102341406B (zh) 2009-03-04 2016-06-08 德国癌症研究中心 组装活化蛋白(aap)及其用于制备基本上由vp3组成的细小病毒颗粒的应用
US11214610B2 (en) 2010-12-01 2022-01-04 H. Lundbeck A/S High-purity production of multi-subunit proteins such as antibodies in transformed microbes such as Pichia pastoris
US9539324B2 (en) 2010-12-01 2017-01-10 Alderbio Holdings, Llc Methods of preventing inflammation and treating pain using anti-NGF compositions
WO2012075340A2 (en) 2010-12-01 2012-06-07 Alderbio Holdings Llc Anti-ngf compositions and use thereof
US9884909B2 (en) 2010-12-01 2018-02-06 Alderbio Holdings Llc Anti-NGF compositions and use thereof
US9067988B2 (en) 2010-12-01 2015-06-30 Alderbio Holdings Llc Methods of preventing or treating pain using anti-NGF antibodies
US9078878B2 (en) 2010-12-01 2015-07-14 Alderbio Holdings Llc Anti-NGF antibodies that selectively inhibit the association of NGF with TrkA, without affecting the association of NGF with p75
EP2500434A1 (de) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsidfreie AAV-Vektoren, Zusammensetzungen und Verfahren zur Vektorherstellung und Genlieferung
US10640785B2 (en) 2011-11-22 2020-05-05 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
CN110606874B (zh) 2013-07-22 2024-04-19 费城儿童医院 用于基因转移到细胞、器官和组织中的变异aav和组合物、方法及用途
CN104592364B (zh) * 2013-10-30 2018-05-01 北京大学 定点突变和定点修饰的腺相关病毒、其制备方法及应用
CN105792914A (zh) * 2013-12-06 2016-07-20 庄信万丰股份有限公司 冷启动催化剂和其在排气系统中的用途
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
CA2941640A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
PE20170260A1 (es) 2014-05-02 2017-04-12 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc
EP3151866B1 (de) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimäre kapside
CN113667696A (zh) * 2014-09-24 2021-11-19 希望之城 用于高效基因组编辑的腺相关病毒载体变异体和其方法
EP3215191A4 (de) 2014-11-05 2018-08-01 Voyager Therapeutics, Inc. Aadc-polynukleotide zur behandlung von morbus parkinson
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
JP6863891B2 (ja) 2014-11-14 2021-04-21 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. 調節性ポリヌクレオチド
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
EP3031820A1 (de) 2014-12-08 2016-06-15 Life Science Inkubator JC Polyomavirus virus-ähnliche Partikeln (VLP) mit einem Targeting-Peptid
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
US10648000B2 (en) 2015-02-16 2020-05-12 University Of Florida Research Foundation, Incorporated rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
KR20230142649A (ko) 2015-05-16 2023-10-11 젠자임 코포레이션 심부 인트론 돌연변이의 유전자 편집
BR112017027956A2 (pt) 2015-06-23 2018-08-28 Childrens Hospital Philadelphia fator ix modificado e composições, métodos e usos para a transferência de gene para células, órgãos e tecidos
BR112018003665A2 (pt) 2015-09-28 2018-09-25 Univ North Carolina Chapel Hill métodos e composições para vetores virais de evasão de anticorpo
US20190071681A1 (en) * 2016-02-26 2019-03-07 University Of Florida Research Foundation, Incorporated Aav heparin mutants that display significantly improved eye and brain transduction
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (de) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. Zusammensetzungen zur behandlung einer erkrankung
CN109476707B (zh) 2016-05-13 2022-12-02 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
BR112018073384A2 (pt) 2016-05-18 2019-03-06 Voyager Therapeutics, Inc. polinucleotídeos moduladores
WO2017201258A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
JP7021191B2 (ja) 2016-08-15 2022-02-16 ジェンザイム・コーポレーション Aavを検出するための方法
EP3506817A4 (de) 2016-08-30 2020-07-22 The Regents of The University of California Verfahren für biomedizinisches targeting und freisetzung sowie vorrichtungen und systeme zur ausführung davon
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
MX2019009720A (es) * 2017-02-15 2019-10-07 Univ North Carolina Chapel Hill Metodos y composiciones para la transferencia de genes a traves de la vasculatura.
US20200047168A1 (en) * 2017-03-13 2020-02-13 Mitsubishi Chemical Corporation Transition metal-carrying zeolite and production method therefor, and nitrogen oxide purification catalyst and method for using 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
CA3054711A1 (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
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
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
PE20201264A1 (es) 2017-09-20 2020-11-19 4D Molecular Therapeutics Inc Variantes de capsides de virus adenoasociados y metodos de uso de estas
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
DK3717636T3 (da) 2017-11-27 2023-05-30 4D Molecular Therapeutics Inc Adeno-associeret-virus-variantcapsider og anvendelse til inhibering af angiogenese
SG11202009854XA (en) * 2018-02-15 2020-11-27 Bjoerklund Tomas Modified viral capsids
CN110691846A (zh) * 2018-05-07 2020-01-14 北卡罗来纳-查佩尔山大学 合理的多倍体腺相关病毒载体及其制造和使用方法
US11821009B2 (en) 2018-05-15 2023-11-21 Cornell University Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
US20210317478A1 (en) * 2018-08-21 2021-10-14 Massachusetts Eye And Ear Infirmary Compositions and methods for modulating transduction efficiency of adeno-associated viruses
JP2022520875A (ja) 2019-02-25 2022-04-01 ノバルティス アーゲー Biettiクリスタリン網膜症を治療するための組成物及び方法
BR112021016501A2 (pt) 2019-02-25 2021-10-26 Novartis Ag Composições e métodos para tratar distrofia cristalina de bietti
US20210082541A1 (en) * 2019-08-31 2021-03-18 Wyatt Technology Corporation Measuring attributes of a viral gene delivery vehicle sample via separation
MX2022004352A (es) 2019-10-17 2022-07-19 Stridebio Inc Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c.
WO2024006433A1 (en) * 2022-06-30 2024-01-04 Carnegie Mellon University Method for production of virus-like particles for gene delivery

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994029469A2 (en) * 1993-06-07 1994-12-22 Vical Incorporated Plasmids suitable for gene therapy
US6204059B1 (en) * 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5741683A (en) * 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
EP0933997A4 (de) * 1996-09-06 2000-08-16 Chiron Corp Verfahren und zusammensetzungen für die leberspezifische verabreichung von therapeutischen molekülen unter verwendung rekombinanter aav vektoren
US6221646B1 (en) * 1997-07-31 2001-04-24 Chiron Corporation Materials and methods for simplified AAV production
CA2367375A1 (en) 1999-03-15 2000-09-21 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
US6943153B1 (en) * 1999-03-15 2005-09-13 The Regents Of The University Of California Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
US6329181B1 (en) * 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
AU2001292881A1 (en) 2000-09-20 2002-04-02 The Regents Of The University Of California Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
US6962815B2 (en) * 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
US6692815B2 (en) * 2001-06-15 2004-02-17 Vinylex Corporation Extruded plastic lumber and method of manufacture
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
WO2003089612A2 (en) * 2002-04-17 2003-10-30 University Of Florida Research Foundation, Inc. IMPROVED rAAV VECTORS
SI1496944T1 (sl) 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
US7220577B2 (en) * 2002-08-28 2007-05-22 University Of Florida Research Foundation, Inc. Modified AAV
EP1618201A1 (de) 2003-05-01 2006-01-25 University of Florida Research Foundation, Inc. Vp2-modifizierte raav-vektoren und verwendungen davon

Also Published As

Publication number Publication date
AU2003295312B2 (en) 2008-08-14
AU2003295312A1 (en) 2004-04-08
WO2004027019A3 (en) 2004-08-05
EP1496944B1 (de) 2008-08-20
NZ536274A (en) 2008-04-30
NZ561656A (en) 2009-03-31
EP1496944A4 (de) 2006-03-22
US20060088936A1 (en) 2006-04-27
CA2483624A1 (en) 2004-04-01
ATE405295T1 (de) 2008-09-15
US20150005369A1 (en) 2015-01-01
SI1496944T1 (sl) 2009-02-28
DK1496944T3 (da) 2008-12-01
WO2004027019A2 (en) 2004-04-01
US8802080B2 (en) 2014-08-12
EP1496944A2 (de) 2005-01-19
HK1071518A1 (en) 2005-07-22

Similar Documents

Publication Publication Date Title
ATE405295T1 (de) Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine
PH12020550094A1 (en) Adeno-associated virus variant capsids and methods of use thereof
WO2005077333A3 (en) Gel-based delivery of recombinant adeno-associated virus vectors
WO2005005610A3 (en) Mutant adeno-associated virus virions and methods of use thereof
TW202102525A (zh) 重組腺相關病毒載體
DK1783225T3 (da) Strukturprotein fra AAV, dets fremstilling og anvendelse
DE60033293D1 (de) Neue b7-4 moleküle und deren verwendungen
JP2008523813A (ja) キメラベクター
WO2003042362A3 (en) PGC-1β, A NOVEL PGC-1 HOMOLOGUE AND USES THEREFOR
MX2022002961A (es) Metodos para tratar enfermedades neovasculares oculares usando variantes de aav2 que codifican para aflibercept.
DE60031418D1 (de) 15571, ein neues gpcr-ähnliches molekül der secretin-ähnlichen familie und dessen verwendungen
DE60237613D1 (de) Neue pgc-1-isoformen und deren verwendungen
WO2002002148A3 (en) Dual recombinant gene therapy compositions and methods of use
WO2001083781A3 (en) 14094, a novel human trypsin family member and uses thereof
BR112022003206A2 (pt) Métodos para tratar doenças neovasculares oculares usando variantes de aav2 que codificam aflibercepte
DK1664315T3 (da) AAV-vektorer til in vivo genterapi af rheumatoid arthritis
DE60237236D1 (de) Ein mitglied der menschlichen proteinkinasefamilie, und verwendungen dafür
DE60234047D1 (de) 15603, ein mitglied der humanen ionenkanal-familie
WO2023010120A3 (en) Engineering aav vectors with improved cns targeting
WO2003035840A3 (en) 69583 and 85924, novel human protein kinase family members and uses therefor
WO2002016568A3 (en) 46863, a human methyltransferase and uses thereof
DE60136832D1 (de) Atcr-1, eine humane acyltransferase und verwendungen davon
AU2001288284A1 (en) 14189, a human kinase and uses thereof
WO2001075098A3 (en) 33166, a human hydrolase-like molecule and uses thereof
ATE331033T1 (de) Proteinkinase-molekül 14911 und dessen anwendungen

Legal Events

Date Code Title Description
8364 No opposition during term of opposition
8339 Ceased/non-payment of the annual fee