WO2000017377A3 - Methods for large-scale production of recombinant aav vectors - Google Patents

Methods for large-scale production of recombinant aav vectors Download PDF

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Publication number
WO2000017377A3
WO2000017377A3 PCT/US1999/022052 US9922052W WO0017377A3 WO 2000017377 A3 WO2000017377 A3 WO 2000017377A3 US 9922052 W US9922052 W US 9922052W WO 0017377 A3 WO0017377 A3 WO 0017377A3
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WO
Grant status
Application
Patent type
Prior art keywords
raav
methods
cells
genomes
large
Prior art date
Application number
PCT/US1999/022052
Other languages
French (fr)
Other versions
WO2000017377A2 (en )
Inventor
Barry J Byrne
James E Conway
Gary S Hayward
Nicholas Muzyczka
Sergei Zolotukhin
Original Assignee
Barry J Byrne
James E Conway
Gary S Hayward
Nicholas Muzyczka
Univ Florida
Univ Johns Hopkins
Sergei Zolotukhin
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

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    • 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
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA Viruses
    • C12N2710/00011MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA Viruses dsDNA Viruses
    • C12N2710/16011Herpesviridae
    • C12N2710/16611Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16641Use of virus, viral particle or viral elements as a vector
    • C12N2710/16643Use 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
    • C12N2750/00011MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA Viruses ssDNA Viruses
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/10Plasmid DNA
    • C12N2800/108Plasmid DNA episomal vectors

Abstract

Disclosed are HSV-1 amplicons that supply all necessary helper functions required for rAAV packaging and methods for their use. These HSV-1 amplicons have been shown to be capable of rescuing and replicating all forms of rAAV genomes including rAAV genomes introduced into cells by infection of rAAV virions, rAAV genomes transfected into cells on plasmids or proviral rAAV genomes integrated into cellular chromosomal DNA. Also provided are methods for preparing high-titer rAAV vector compositions suitable for gene therapy and the delivery of exogenous polynucleotides to selected host cells.
PCT/US1999/022052 1998-09-22 1999-09-22 Methods for large-scale production of recombinant aav vectors WO2000017377A3 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
US10150798 true 1998-09-22 1998-09-22
US60/101,507 1998-09-22

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CA 2347129 CA2347129A1 (en) 1998-09-22 1999-09-22 Methods for large-scale production of recombinant aav vectors
EP19990954648 EP1115880A2 (en) 1998-09-22 1999-09-22 Methods for large-scale production of recombinant aav vectors

Publications (2)

Publication Number Publication Date
WO2000017377A2 true WO2000017377A2 (en) 2000-03-30
WO2000017377A3 true true WO2000017377A3 (en) 2000-09-08

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1999/022052 WO2000017377A3 (en) 1998-09-22 1999-09-22 Methods for large-scale production of recombinant aav vectors

Country Status (3)

Country Link
EP (1) EP1115880A2 (en)
CA (1) CA2347129A1 (en)
WO (1) WO2000017377A3 (en)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6627617B1 (en) 1999-10-01 2003-09-30 University Of North Carolina At Chapel Hill Temperature-sensitive regulation of viral vector production
EP1320620B1 (en) * 2000-09-25 2015-01-21 The Regents Of The University Of Michigan Production of viral vectors
WO2005035743A1 (en) * 2003-10-15 2005-04-21 Vector Gene Technology Company Ltd. Method for large-scale production, isolation, purification and the uses of multi-type recombinant adeno-associated virus vectors
ES2634118T3 (en) 2009-02-11 2017-09-26 The University Of North Carolina At Chapel Hill Modified virus vectors and methods for making and using the same
WO2011088081A1 (en) 2010-01-12 2011-07-21 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1995006743A2 (en) * 1993-08-31 1995-03-09 Uab Research Foundation Methods and compositions for the large scale production of recombinant adeno-associated virus
WO1997020935A2 (en) * 1995-12-06 1997-06-12 Cambridge University Technical Services Limited Viral vectors
CN1213699A (en) * 1997-10-08 1999-04-14 中国预防医学科学院病毒学研究院 Prodn. system of novel recombination adenovirus adjoint virus
WO1999020778A1 (en) * 1997-10-17 1999-04-29 Cantab Pharmaceuticals Research Limited Gene delivery vectors and their uses

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1995006743A2 (en) * 1993-08-31 1995-03-09 Uab Research Foundation Methods and compositions for the large scale production of recombinant adeno-associated virus
WO1997020935A2 (en) * 1995-12-06 1997-06-12 Cambridge University Technical Services Limited Viral vectors
CN1213699A (en) * 1997-10-08 1999-04-14 中国预防医学科学院病毒学研究院 Prodn. system of novel recombination adenovirus adjoint virus
WO1999020778A1 (en) * 1997-10-17 1999-04-29 Cantab Pharmaceuticals Research Limited Gene delivery vectors and their uses

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
CONWAY J. E. ET AL.: "High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex virus type I vector expressing AAV-2 Rep and Cap.", GENE THERAPY, vol. 6, no. 6, June 1999 (1999-06-01), pages 986 - 993, XP000906811, ISSN: 0969-7128 *
CONWAY J. E. ET AL.: "Recombinant adeno-associated virus type 2 replication and packaging is entirely supported by a herpes simplex virus type 1 amplicon expressing rep and cap.", JOURNAL OF VIROLOGY, vol. 71, no. 11, 1997, pages 8780 - 8789, XP002136599, ISSN: 0022-538X *
DATABASE WPI Section Ch Week 199933, Derwent World Patents Index; Class B04, AN 1999-386309, XP002136602 *
SHU YUELONG ET AL: "A novel recombinant adeno-associated virus vector packaging system with HSV-1, amplicon providing helper functions.", SCIENCE IN CHINA SERIES C LIFE SCIENCES, vol. 42, no. 5, October 1999 (1999-10-01), pages 465 - 470, XP002136601, ISSN: 1006-9305 *
VINCENT K. A. ET AL.: "ANALYSIS OF RECOMBINANT ADENO-ASSOCIATED VIRUS PACKAGING AND REQUIREMENTS FOR REP AND CAP GENE PRODUCTS", JOURNAL OF VIROLOGY, vol. 71, no. 3, March 1997 (1997-03-01), pages 1897 - 1905, XP000881017, ISSN: 0022-538X *
ZHANG X. ET AL.: "High-titer recombinant adeno-associated virus production from replicating amplicons and herpes vectors deleted for glycoprotein H.", HUMAN GENE THERAPY, vol. 10, no. 15, 10 October 1999 (1999-10-10), pages 2527 - 2537, XP000906820, ISSN: 1043-0342 *

Also Published As

Publication number Publication date Type
EP1115880A2 (en) 2001-07-18 application
CA2347129A1 (en) 2000-03-30 application
WO2000017377A2 (en) 2000-03-30 application

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