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DE69627532T2 - Helfervirus-freie herstellung von aav - Google Patents

Helfervirus-freie herstellung von aav Download PDF

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Publication number
DE69627532T2
DE69627532T2 DE69627532T DE69627532T DE69627532T2 DE 69627532 T2 DE69627532 T2 DE 69627532T2 DE 69627532 T DE69627532 T DE 69627532T DE 69627532 T DE69627532 T DE 69627532T DE 69627532 T2 DE69627532 T2 DE 69627532T2
Authority
DE
Germany
Prior art keywords
helper virus
aav
free manufacture
virus
manufacture
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 - Lifetime
Application number
DE69627532T
Other languages
English (en)
Other versions
DE69627532D1 (de
Inventor
K Ferrari
Xiao Xiao
J Samulski
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 North Carolina at Chapel Hill
Original Assignee
University of North Carolina at Chapel Hill
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 North Carolina at Chapel Hill filed Critical University of North Carolina at Chapel Hill
Application granted granted Critical
Publication of DE69627532D1 publication Critical patent/DE69627532D1/de
Publication of DE69627532T2 publication Critical patent/DE69627532T2/de
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

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Classifications

    • 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
    • C12N2750/14141Use 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

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
DE69627532T 1995-06-07 1996-06-06 Helfervirus-freie herstellung von aav Expired - Lifetime DE69627532T2 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US48375795A 1995-06-07 1995-06-07
PCT/US1996/009405 WO1996040240A1 (en) 1995-06-07 1996-06-06 Helper virus-free aav production

Publications (2)

Publication Number Publication Date
DE69627532D1 DE69627532D1 (de) 2003-05-22
DE69627532T2 true DE69627532T2 (de) 2004-01-29

Family

ID=23921407

Family Applications (1)

Application Number Title Priority Date Filing Date
DE69627532T Expired - Lifetime DE69627532T2 (de) 1995-06-07 1996-06-06 Helfervirus-freie herstellung von aav

Country Status (7)

Country Link
EP (1) EP0871483B1 (de)
JP (1) JP4108746B2 (de)
AT (1) ATE237352T1 (de)
AU (1) AU707862B2 (de)
CA (1) CA2221293C (de)
DE (1) DE69627532T2 (de)
WO (1) WO1996040240A1 (de)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0850313B8 (de) * 1995-09-08 2009-07-29 Genzyme Corporation Verbesserte aav vektoren für die gentherapie
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
US6221646B1 (en) 1997-07-31 2001-04-24 Chiron Corporation Materials and methods for simplified AAV production
CA2303768C (en) * 1997-09-19 2009-11-24 The Trustees Of The University Of Pennsylvania Methods and vector constructs useful for production of recombinant aav
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
PT1064393E (pt) * 1998-03-20 2005-04-29 Univ Pennsylvania Composicoes e metodos para a producao de virus adeno-associados recombinantes sem auxiliar
MXPA01004169A (es) * 1998-10-27 2002-06-04 Crucell Holland Bv Produccion mejorada de vector de virus adenoasociados.
JP4693244B2 (ja) 1999-03-18 2011-06-01 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 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
US6258595B1 (en) * 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
AU2001257611A1 (en) 2000-04-28 2001-11-12 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
AU2013326503B2 (en) * 2012-10-03 2018-04-19 Glaxosmithkline Biologicals Sa Immunogenic composition
AU2016347887B2 (en) 2015-11-05 2019-11-14 Bamboo Therapeutics, Inc. Modified friedreich ataxia genes and vectors for gene therapy
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
WO2020261178A1 (en) 2019-06-27 2020-12-30 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
IL302608A (en) 2020-11-03 2023-07-01 Pfizer Methods for purification of aav vectors by anion exchange chromatography
JP2024503234A (ja) 2020-12-23 2024-01-25 ファイザー・インク 親和性クロマトグラフィーによるaavベクターの精製方法
US11760788B2 (en) 2021-03-02 2023-09-19 Pathways Neuro Pharma, Inc. Neuroreceptor compositions and methods of use
EP4329820A1 (de) 2021-04-26 2024-03-06 Alexion Pharma International Operations Limited Adeno-assoziierte virale vektorkapside mit verbessertem gewebetropismus
CN113308492B (zh) * 2021-05-31 2023-03-21 北京中因科技有限公司 一种用于复制性aav的检测方法
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5436146A (en) * 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US5173414A (en) * 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
EP0689601B1 (de) * 1993-02-22 2006-10-04 The Rockefeller University Herstellung von helfer-freien retroviren mit hohem titer mittels transienter transfektion

Also Published As

Publication number Publication date
AU6098596A (en) 1996-12-30
EP0871483B1 (de) 2003-04-16
EP0871483A1 (de) 1998-10-21
DE69627532D1 (de) 2003-05-22
EP0871483A4 (de) 2000-01-05
JP4108746B2 (ja) 2008-06-25
CA2221293A1 (en) 1996-12-19
CA2221293C (en) 2012-09-18
AU707862B2 (en) 1999-07-22
WO1996040240A1 (en) 1996-12-19
JPH11510371A (ja) 1999-09-14
ATE237352T1 (de) 2003-05-15

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