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)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (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)
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
ATE454445T1 (de) * 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
US6759050B1 (en) * 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
WO2001027303A1 (en) * 1999-10-12 2001-04-19 The University Of North Carolina At Chapel Hill Adeno-associated virus vectors encoding factor viii and methods of using the same
US20010049144A1 (en) * 1999-12-10 2001-12-06 Victor Rivera Methods for high level 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
CN1215052C (zh) * 2000-07-19 2005-08-17 三菱制药株式会社 异羟肟酸的磺酸衍生物及其医药用途
ATE492630T1 (de) 2002-06-18 2011-01-15 Eisai R&D Man Co Ltd Primäre kultivierte adipozyten zur gentherapie
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
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
US7498024B2 (en) 2003-06-03 2009-03-03 Cell Genesys, Inc. Compositions and methods for enhanced expression of immunoglobulins from a single vector using a peptide cleavage site
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
US11779656B2 (en) 2016-04-15 2023-10-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia A
WO2023240222A2 (en) * 2022-06-10 2023-12-14 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
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
WO1998009524A1 (en) 1996-09-06 1998-03-12 Chiron Corporation Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors
JP4289687B2 (ja) * 1997-03-14 2009-07-01 ザ チルドレンズ ホスピタル オブ フィラデルフィア 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
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

Also Published As

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

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Legal Events

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RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties