BR9914643A - Vetores adeno-associados para expressão dofator viii por células alvo - Google Patents

Vetores adeno-associados para expressão dofator viii por células alvo

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Publication number
BR9914643A
BR9914643A BR9914643-6A BR9914643A BR9914643A BR 9914643 A BR9914643 A BR 9914643A BR 9914643 A BR9914643 A BR 9914643A BR 9914643 A BR9914643 A BR 9914643A
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BR
Brazil
Prior art keywords
vectors
adeno
target cells
present
associated vectors
Prior art date
Application number
BR9914643-6A
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English (en)
Inventor
Linda B Couto
Peter C Colosi
Original Assignee
Avigen 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.)
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Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=27379851&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=BR9914643(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Avigen Inc filed Critical Avigen Inc
Publication of BR9914643A publication Critical patent/BR9914643A/pt

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

"VETORES ADENO-ASSOCIADOS PARA EXPRESSãO DOFATOR VIII POR CéLULAS ALVO". A presente invenção fornecevetores virais úteis para a expressão de genes em altos níveis nascélulas humanas. Em particular a presente invenção fornecevetores adeno-associados (AAV) úteis na terapia genética. Estesvetores são capazes de fornecer ácido nucleico contendoconstrutos que resultam na produção de níveis terapêuticoscompletos de Fator VIII biologicamente ativos no indivíduoreceptor in vivo. A presente invenção fornece ainda composiçõesfarmaceuticas compreendendo os tais vetores AAV, assim comométodos para sua produção e o uso dos construtos.
BR9914643-6A 1998-10-20 1999-10-19 Vetores adeno-associados para expressão dofator viii por células alvo BR9914643A (pt)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US10499498P 1998-10-20 1998-10-20
US12597499P 1999-03-24 1999-03-24
US09/364,862 US6221349B1 (en) 1998-10-20 1999-07-30 Adeno-associated vectors for expression of factor VIII by target cells
PCT/US1999/024495 WO2000023116A1 (en) 1998-10-20 1999-10-19 Adeno-associated vectors for expression of factor viii by target cells

Publications (1)

Publication Number Publication Date
BR9914643A true BR9914643A (pt) 2001-12-11

Family

ID=27379851

Family Applications (1)

Application Number Title Priority Date Filing Date
BR9914643-6A BR9914643A (pt) 1998-10-20 1999-10-19 Vetores adeno-associados para expressão dofator viii por células alvo

Country Status (11)

Country Link
US (1) US6221349B1 (pt)
EP (1) EP1124583A4 (pt)
JP (1) JP2002527493A (pt)
KR (1) KR20010082234A (pt)
CN (1) CN1329509A (pt)
AU (1) AU757688B2 (pt)
BR (1) BR9914643A (pt)
CA (1) CA2348068A1 (pt)
IL (1) IL142721A0 (pt)
MX (1) MXPA01004067A (pt)
WO (1) WO2000023116A1 (pt)

Families Citing this family (81)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030215422A1 (en) * 1996-09-11 2003-11-20 John A. Chiorini Aav4 vector and uses thereof
US6436392B1 (en) * 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
US6797505B2 (en) * 1998-05-27 2004-09-28 Cell Genesys, Inc. Recombinant AAV vectors for gene therapy of hemophilia A
JP4060531B2 (ja) * 1998-05-28 2008-03-12 アメリカ合衆国 Ａａｖ５ベクターおよびその使用
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
CA2376400A1 (en) * 1999-06-08 2000-12-14 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
US7122335B1 (en) 1999-06-08 2006-10-17 University Of Iowa Research Foundation Compounds and methods to enhance rAAV transduction
US7241447B1 (en) * 1999-10-07 2007-07-10 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
CA2387484A1 (en) * 1999-10-12 2001-04-19 Haim Burstein Adeno-associated virus vectors encoding factor viii and methods of using the same
US6582692B1 (en) * 1999-11-17 2003-06-24 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
CA2400634A1 (en) * 2000-02-17 2001-08-23 Chester Li Genetic modification of the lung as a portal for gene delivery
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
US6593123B1 (en) * 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
US7615537B2 (en) 2000-10-25 2009-11-10 Genzyme Corporation Methods for treating blood coagulation disorders
US20020159978A1 (en) * 2001-02-06 2002-10-31 James Allen Muscle-directed gene transfer by use of recombinant AAV-1 and AAV-6 virions
US20030147853A1 (en) * 2001-03-14 2003-08-07 Mcclelland Alan Recombinant adeno-associated virus-mediated gene transfer via retroductal infusion of virions
WO2002086091A2 (en) * 2001-04-25 2002-10-31 The Regents Of The University Of California Method to treat hemophilia by hepatic gene transfer of factor viii/ix with vesicle vector
EP1395293B1 (en) * 2001-05-14 2009-07-22 Gbp Ip, Llc Lentiviral vectors encoding clotting factors for gene therapy
US8241622B2 (en) * 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
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
CN100488981C (zh) 2001-08-24 2009-05-20 维多利亚大学创新和发展公司 含有蛋白酶激活序列的气单胞菌溶素原及其应用
AU2003223497A1 (en) * 2002-04-05 2003-10-27 Centocor, Inc. Asthma-related anti-il-13 immunoglobulin derived proteins, compositions, methods and uses
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
KR100535325B1 (ko) * 2002-09-24 2005-12-09 한국생명공학연구원 Ａａｖ 유전자 전달체 제조용 헬퍼 플라스미드
US20040092008A1 (en) * 2002-11-12 2004-05-13 Snyder Richard O. Recombinant AAV vectors for gene therapy of hemophilia A
CA2520028A1 (en) * 2003-03-31 2004-10-21 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
US7963956B2 (en) * 2003-04-22 2011-06-21 Antisense Pharma Gmbh Portable equipment for administration of fluids into tissues and tumors by convection enhanced delivery technique
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
US7485291B2 (en) * 2003-06-03 2009-02-03 Cell Genesys, Inc. Compositions and methods for generating multiple polypeptides from a single vector using a virus derived peptide cleavage site, and uses thereof
US7709224B2 (en) * 2003-06-03 2010-05-04 Biosante Pharmaceuticals, Inc. Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
EP1639115A1 (en) * 2003-06-19 2006-03-29 Peter Bromley Protein production system
DK2292779T3 (en) 2003-09-30 2017-02-27 Univ Pennsylvania ADENOASS-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME AND APPLICATIONS THEREOF
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
US20080188431A1 (en) * 2004-09-08 2008-08-07 Chiorini John A Transcytosis of Adeno-Associated Viruses
CN106906241B (zh) 2005-04-07 2020-09-11 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
AU2006233638A1 (en) * 2005-04-14 2006-10-19 Csl Behring Gmbh Modified coagulation Factor VIII with enhanced stability and its derivates
WO2006119432A2 (en) * 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
WO2009130208A1 (en) * 2008-04-22 2009-10-29 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
JP6224459B2 (ja) 2011-02-17 2017-11-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
CN103215308B (zh) * 2013-02-01 2015-12-23 中国科学院苏州生物医学工程技术研究所 表达重组人fviii的整合质粒、细胞株及其构建方法和应用
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
PL3044231T3 (pl) 2013-09-12 2021-01-11 Biomarin Pharmaceutical Inc. Wektory aav zawierające gen kodujący czynnik viii
CN104087613B (zh) * 2014-06-30 2017-08-29 中国科学院苏州生物医学工程技术研究所 基于aav‑itr的基因表达微载体及其构建方法和应用
DK3193911T3 (da) * 2014-07-25 2023-11-27 Csl Behring Gmbh Forbedrede faktor VII-præparater til brug ved behandling af hæmofili A
CA2958141C (en) * 2014-08-13 2024-06-11 The Children's Hospital Of Philadelphia An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders
US10335466B2 (en) 2014-11-05 2019-07-02 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of parkinson's disease
ES2763551T3 (es) * 2015-04-16 2020-05-29 Univ Emory Promotores y vectores recombinantes para la expresión de proteínas en el hígado y uso de los mismos
CN108368521A (zh) * 2015-08-06 2018-08-03 宾夕法尼亚州大学信托人 Glp-1和其在用于治疗代谢疾病的组合物中的用途
PE20181338A1 (es) 2015-09-24 2018-08-21 Biomarin Pharm Inc Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden
AU2016343887B2 (en) 2015-10-28 2023-04-06 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
KR20230008256A (ko) * 2015-11-13 2023-01-13 다케다 야쿠힌 고교 가부시키가이샤 A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터
MX2018005969A (es) * 2015-11-13 2018-11-29 Baxalta Inc Vectores virales que codifican variantes del factor fviii recombinantes con mayor expresión para la terapia génica de hemofilia a.
EP3384034B1 (en) 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
CN109415704B (zh) 2016-02-16 2022-02-25 利兰斯坦福初级大学董事会 对先已存在的人中和抗体有抗性的新型重组腺相关病毒衣壳
EP3210632B1 (en) * 2016-02-23 2022-09-28 EyeServ GmbH Gene therapy for the treatment of a retinal degeneration disease
WO2017155973A1 (en) 2016-03-07 2017-09-14 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
CN109562191A (zh) 2016-04-15 2019-04-02 宾夕法尼亚州大学信托人 用于治疗血友病a的基因疗法
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
WO2018132747A1 (en) 2017-01-13 2018-07-19 University Of Iowa Research Foundation Bocaparvovirus small noncoding rna and uses thereof
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
US11827906B2 (en) 2017-02-28 2023-11-28 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clade f vector and uses therefor
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
KR20200035130A (ko) * 2017-08-09 2020-04-01 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 이의 용도
US11608510B2 (en) 2018-03-30 2023-03-21 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
CN112384624A (zh) * 2018-05-15 2021-02-19 杜克大学 用于对阿克曼菌进行基因操纵的系统和方法
WO2020232044A1 (en) * 2019-05-14 2020-11-19 Biomarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
CN114269940A (zh) * 2019-07-03 2022-04-01 学校法人日本医科大学 用于产生包封核酸的aav中空颗粒的方法
CN111876400B (zh) * 2020-08-06 2022-05-24 昆明理工大学 一种常温裂解酶Sly和编码此酶的多核苷酸
WO2022032140A2 (en) 2020-08-07 2022-02-10 Amicus Therapeutics, Inc. Vesicle targeting proteins and uses of same
US20230287451A1 (en) 2020-08-14 2023-09-14 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
AR123838A1 (es) 2020-10-18 2023-01-18 Univ Pennsylvania Vector mejorado de virus adenoasociado (aav) y usos de este
WO2022094180A1 (en) 2020-10-29 2022-05-05 The Trustees Of The University Of Pennsylvania Aav capsids and compositions containing same
TW202309066A (zh) 2021-04-27 2023-03-01 賓州大學委員會 衍生自豬的腺相關病毒衣殼及其用途
EP4359548A1 (en) 2021-06-23 2024-05-01 Synpromics Ltd. Regulatory nucleic acid sequences
WO2024015966A2 (en) 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
EP4389899A1 (en) 2022-12-22 2024-06-26 Synpromics Ltd. Regulatory nucleic acid sequences

Family Cites Families (62)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4722848A (en) 1982-12-08 1988-02-02 Health Research, Incorporated Method for immunizing animals with synthetically modified vaccinia virus
US4886876A (en) 1983-03-31 1989-12-12 Scripps Clinic And Research Foundation Factor VIII coagulant polypeptides
US4757006A (en) 1983-10-28 1988-07-12 Genetics Institute, Inc. Human factor VIII:C gene and recombinant methods for production
JPH07106156B2 (ja) 1983-10-28 1995-11-15 ジェネティックス、インスティチュ−ト ファクタ−▲ｖｉｉｉ▼および関連生産物の製造
US5045455A (en) 1984-01-12 1991-09-03 Chiron Corporation Factor VIII:C cDNA cloning and expression
US4965199A (en) 1984-04-20 1990-10-23 Genentech, Inc. Preparation of functional human factor VIII in mammalian cells using methotrexate based selection
FI86885C (fi) 1984-04-20 1992-10-26 Genentech Inc Foerfarande foer framstaellning av human rekombinantfaktor viii och nukleinsyrasekvenser och vektorer anvaend daertill
EP0182448A3 (en) 1984-08-24 1987-10-28 Genetics Institute, Inc. Production of factor viii and related products
DE3538181A1 (de) 1985-10-26 1987-04-30 Messer Griesheim Gmbh Vorrichtung zur automatischen begrenzung des fuellstandes kryogener fluessigkeiten
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
FI98829C (fi) 1986-01-27 1997-08-25 Chiron Corp Menetelmä rekombinoidun proteiinikompleksin valmistamiseksi, jolla on humaanitekijä VIII:C-aktiivisuutta
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5426029A (en) 1986-03-31 1995-06-20 T Cell Diagnostics, Inc. Therapeutic and diagnostic methods using leukocyte surface antigens
US5422260A (en) 1986-05-29 1995-06-06 Genetics Institute, Inc. -Legal Affairs Human factor VIII:c muteins
US5451521A (en) 1986-05-29 1995-09-19 Genetics Institute, Inc. Procoagulant proteins
US5219740A (en) 1987-02-13 1993-06-15 Fred Hutchinson Cancer Research Center Retroviral gene transfer into diploid fibroblasts for gene therapy
US5149637A (en) 1987-04-06 1992-09-22 Scripps Clinic & Research Foundation Recombinant Factor VIIIC fragments
WO1988009809A2 (en) 1987-06-12 1988-12-15 Cetus Corporation Cell system for expression of recombinant protein without production of virus
IL86693A (en) 1987-06-12 1994-06-24 Stichting Centraal Lab Proteins that have the activity IIIV of the blood, a process for their preparation that uses cells are produced through genetic engineering and pharmaceutical preparations that contain them
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
DE10399031I1 (de) 1987-08-28 2004-01-29 Health Research Inc Rekombinante Viren.
US5004803A (en) 1988-11-14 1991-04-02 Genetics Institute, Inc. Production of procoagulant proteins
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US5225347A (en) 1989-09-25 1993-07-06 Innovir Laboratories, Inc. Therapeutic ribozyme compositions and expression vectors
HUT64591A (en) 1989-11-17 1994-01-28 Novo Nordisk As Protein complexes with viii/c factor activity and process for their production
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
WO1991018088A1 (en) 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
AU8200191A (en) 1990-07-09 1992-02-04 United States of America, as represented by the Secretary, U.S. Department of Commerce, The High efficiency packaging of mutant adeno-associated virus using amber suppressions
MY109299A (en) 1990-08-15 1996-12-31 Virogenetics Corp Recombinant pox virus encoding flaviviral structural proteins
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
SE468050C (sv) 1991-03-15 1998-04-27 Pharmacia & Upjohn Ab Rekombinant derivat av human faktor VIII
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
ATE185424T1 (de) 1991-07-29 1999-10-15 Serex Inc Differentielle bindungsaffinitäten und darauf beruhende dissoziationstestverfahren
DE69233013T2 (de) 1991-08-20 2004-03-04 The Government Of The United States Of America As Represented By The Secretary Of National Institute Of Health, Office Of Technology Transfer Adenovirus vermittelter gentransfer in den gastrointestinaltrakt
CA2078721A1 (en) 1991-09-24 1993-03-25 Hiroshi Yonemura Process for preparing human coagulation factor viii protein complex
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
CA2106260A1 (en) 1992-09-17 1994-03-18 Robert M. Kotin Human adeno-associated virus integration site dna and uses thereof
US5563045A (en) 1992-11-13 1996-10-08 Genetics Institute, Inc. Chimeric procoagulant proteins
AU5602394A (en) 1992-11-13 1994-06-08 Duke University Chimeric blood coagulation proteins
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
WO1994029471A1 (en) * 1993-06-10 1994-12-22 Genetic Therapy, Inc. Adenoviral vectors for treatment of hemophilia
ES2226029T3 (es) 1993-06-10 2005-03-16 Bayer Corporation Vector y linea celular de mamifero con productividad mejorada.
AU7676894A (en) 1993-08-27 1995-03-21 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Convection-enhanced drug delivery
US5834441A (en) 1993-09-13 1998-11-10 Rhone-Poulenc Rorer Pharmaceuticals Inc. Adeno-associated viral (AAV) liposomes and methods related thereto
DE69433592T2 (de) 1993-11-09 2005-02-10 Targeted Genetics Corp., Seattle Die erzielung hoher titer des rekombinanten aav-vektors
US5693531A (en) 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
AUPM901594A0 (en) 1994-10-26 1994-11-17 Ici Australia Operations Proprietary Limited Apparatus and process for loading upholes with explosives
US5872005A (en) 1994-11-03 1999-02-16 Cell Genesys Inc. Packaging cell lines for adeno-associated viral vectors
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
US5681746A (en) 1994-12-30 1997-10-28 Chiron Viagene, Inc. Retroviral delivery of full length factor VIII
US5677158A (en) 1995-06-07 1997-10-14 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
WO1997003195A1 (en) 1995-07-11 1997-01-30 Chiron Corporation Novel factor viii:c polypeptide analogs with altered protease sites
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
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
EP0933997A4 (en) * 1996-09-06 2000-08-16 Chiron Corp METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS
US6797505B2 (en) * 1998-05-27 2004-09-28 Cell Genesys, Inc. Recombinant AAV vectors for gene therapy of hemophilia A
JP2002516345A (ja) * 1998-05-27 2002-06-04 セル ジェネシス インコーポレイテッド 第ｖｉｉｉ因子活性のアデノ随伴ウイルスベクター媒介性発現

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EP1124583A4 (en) 2002-04-17
CN1329509A (zh) 2002-01-02
US6221349B1 (en) 2001-04-24
CA2348068A1 (en) 2000-04-27
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