ATE321873T1 - Rekombinante aav-vektoren für die gentherapie von hämophilie a - Google Patents

Rekombinante aav-vektoren für die gentherapie von hämophilie a

Info

Publication number
ATE321873T1
ATE321873T1 AT99925607T AT99925607T ATE321873T1 AT E321873 T1 ATE321873 T1 AT E321873T1 AT 99925607 T AT99925607 T AT 99925607T AT 99925607 T AT99925607 T AT 99925607T AT E321873 T1 ATE321873 T1 AT E321873T1
Authority
AT
Austria
Prior art keywords
hemophilia
gene therapy
factor viii
aav vectors
recombinant aav
Prior art date
Application number
AT99925607T
Other languages
English (en)
Inventor
Richard Snyder
Thomas J Dull
Ryan Mcguinness
Mitchell H Finer
Original Assignee
Cell Genesys 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 Cell Genesys Inc filed Critical Cell Genesys Inc
Application granted granted Critical
Publication of ATE321873T1 publication Critical patent/ATE321873T1/de

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
AT99925607T 1998-05-27 1999-05-27 Rekombinante aav-vektoren für die gentherapie von hämophilie a ATE321873T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US09/084,425 US6797505B2 (en) 1998-05-27 1998-05-27 Recombinant AAV vectors for gene therapy of hemophilia A

Publications (1)

Publication Number Publication Date
ATE321873T1 true ATE321873T1 (de) 2006-04-15

Family

ID=22184892

Family Applications (1)

Application Number Title Priority Date Filing Date
AT99925607T ATE321873T1 (de) 1998-05-27 1999-05-27 Rekombinante aav-vektoren für die gentherapie von hämophilie a

Country Status (8)

Country Link
US (1) US6797505B2 (de)
EP (1) EP1082446B1 (de)
JP (1) JP2002516114A (de)
AT (1) ATE321873T1 (de)
AU (1) AU775423B2 (de)
CA (1) CA2329505A1 (de)
DE (1) DE69930625T2 (de)
WO (1) WO1999061642A1 (de)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
CA2348382C (en) * 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus vectors and methods of making and administering the same
US6759050B1 (en) * 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
JP2003511082A (ja) * 1999-10-12 2003-03-25 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 第ｖｉｉｉ因子をコードするアデノ随伴ウィルスベクターとその使用法
WO2001042444A2 (en) * 1999-12-10 2001-06-14 Ariad Gene Therapeutics, Inc. Methods for expression of genes in primates
JP4085231B2 (ja) 2000-02-28 2008-05-14 株式会社ビークル タンパク質中空ナノ粒子とそれを用いた物質運搬体、ならびに細胞への物質導入方法
EP1306367B1 (de) * 2000-07-19 2007-07-18 Mitsubishi Pharma Corporation Sulphonsäurederivate von hydroxamsäuren und deren verwendung in medizinischen produkten
PT2213730E (pt) 2002-06-18 2016-02-26 Eisai R&D Man Co Ltd Adipócitos de cultura primária para terapia génica
CN1498964A (zh) * 2002-11-07 2004-05-26 本元正阳基因技术股份有限公司 可诱导RNAi途径的用于基因治疗的系列重组腺相关病毒
US20040092008A1 (en) * 2002-11-12 2004-05-13 Snyder Richard O. Recombinant AAV vectors for gene therapy of hemophilia A
EP1636360A4 (de) 2003-06-03 2006-11-08 Cell Genesys Inc Zusammensetzungen und verfahren zur verstärkten expression rekombinanter polypeptide von einem vektor unter verwendung einer peptidspaltstelle
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
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
TWI791433B (zh) 2016-04-15 2023-02-11 賓州大學委員會 治療ａ型血友病之基因治療
US20250352626A1 (en) * 2022-06-10 2025-11-20 University Of Florida Research Foundation, Incorporated Immune tolerance induction to viral capsids

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
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
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
EP1005376B1 (de) * 1997-03-14 2010-11-03 The Children's Hospital of Philadelphia Zusammensetzungen zum einsatz in einer gentherapie zur behandlung von hämophilie
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells

Also Published As

Publication number Publication date
EP1082446B1 (de) 2006-03-29
US20020155580A1 (en) 2002-10-24
DE69930625D1 (de) 2006-05-18
EP1082446A1 (de) 2001-03-14
JP2002516114A (ja) 2002-06-04
AU775423B2 (en) 2004-07-29
US6797505B2 (en) 2004-09-28
CA2329505A1 (en) 1999-12-02
WO1999061642A1 (en) 1999-12-02
AU4185799A (en) 1999-12-13
DE69930625T2 (de) 2007-01-25

Similar Documents

Publication Publication Date Title
ATE321873T1 (de) Rekombinante aav-vektoren für die gentherapie von hämophilie a
EP0998569A4 (de) Polynukleotid kodierend für ein polypeptid mit heparanase-aktivität und dessen expression in transduzierten zellen
ATE136908T1 (de) Peptide und antikörper, die die integrin- ligandbindung inhibieren
DE69535147D1 (de) Überexpression von säugetier- und virusproteinen
WO1999061595A3 (en) Adeno-associated viral vector-mediated expression of factor viii activity
DK0397633T3 (da) Fibronectinbindende protein samt fremstilling deraf
FI855141A7 (fi) Rekombinantti-DNA-vektoreita, jotka kykenevät ilmaisemaan apoaequoriinia.
HUT73962A (en) New bacillus thuringiensis strains and their insecticidal proteins
DK0929564T3 (da) Höjniveau-ekspression af proteiner
ATE423194T1 (de) Von-willebrand-faktor (vwf) spaltendes protease- polypeptid, für das polypeptid codierende nukleinsäure und verwendung des polypeptids
EP0686161A4 (de) Alpha untereinheit des humanen beta-2 integrins
DE69839566D1 (de) Chimäre polypeptide, die das fragment b des shigatoxins sowie peptide von therapeutischem interesse enthalten
IL69469A (en) Polypeptide having human h1-relaxin activity,double-stranded dna fragments coding therefor,vectors containing the dna fragments and methods for preparing the polypeptide,dna fragments and vectors
NO882380L (no) Fibfonektin-bindende protein, samt fremstilling derav.
DE69734141D1 (de) PROTEIN, DAS FÜR MENSCHLICHE Th2-ZELLEN SPEZIFISCH IST, DAFÜR KODIERENDES GEN UND KORRESPONDIERENDE TRANSFORMANTE, REKOMBINANTER VEKTOR UND MONOKLONALER ANTKÖRPER
ATE202570T1 (de) Gene, die für macif proteine kodieren, expressionsvektoren mit diesem genen, und transformantenzellen mit diesem proteinen
NO922421D0 (no) Collagenbindende protein, samt fremstilling derav
DE69328060D1 (de) Angiotensin-II Typ-1 Rezeptor und dessen Herstellung
DE69333284D1 (de) Polypeptide mit serotoninerger (5ht5a) rezeptor-aktivität, für sie kodierende nukleinsäuren und ihre verwendung
ATE294861T1 (de) Polypeptide mit opioider rezeptor-aktvität, für sie kodierende nukleinsäuren und ihre verwendungen
DE69936513D1 (de) Menschliche p51-gene und deren genprodukte
MX9708107A (es) Subunidad alfa de integrina beta 2 humana.

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties