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

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

Info

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
Authority
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

Links

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
    • 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 (5)

Application Number Priority Date Filing Date Title
AU1094900A AU774706B2 (en) 1998-09-22 1999-09-22 Methods for large-scale production of recombinant AAV vectors
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
NZ51117199A NZ511171A (en) 1998-09-22 1999-09-22 Methods for large-scale production of recombinant AAV vectors
AU2004218673A AU2004218673A1 (en) 1998-09-22 2004-10-07 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

ID=22285008

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 (7)

* 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
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
EP2970966A4 (en) 2013-03-15 2016-10-26 Univ North Carolina Synthetic adeno-associated virus inverted terminal repeats
CN105163764A (en) 2013-03-15 2015-12-16 北卡罗来纳-查佩尔山大学 Methods and compositions for dual glycan binding AAV vectors

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

Similar Documents

Publication Publication Date Title
Hermonat et al. Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells
Romano et al. Gene transfer technology in therapy: current applications and future goals
Kotterman et al. Viral vectors for gene therapy: translational and clinical outlook
Stavropoulos et al. An enhanced packaging system for helper-dependent herpes simplex virus vectors
US5707618A (en) Adenovirus vectors for gene therapy
Enssle et al. Foamy virus reverse transcriptase is expressed independently from the Gag protein
Ponnazhagan et al. Lack of site-specific integration of the recombinant adeno-associated virus 2 genomes in human cells
Gardlík et al. Vectors and delivery systems in gene therapy
Ashktorab et al. Identification of nuclear proteins that specifically interact with adeno-associated virus type 2 inverted terminal repeat hairpin DNA.
Rutledge et al. Adeno-associated virus vector integration junctions.
Bilbao et al. Adenoviral/retroviral vector chimeras: a novel strategy to achieve high-efficiency stable transduction in vivo.
Zhang et al. Lentiviral Vectors for Sustained Transgene Expression in Human Bone Marrow–Derived Stromal Cells
Zádori et al. Analysis of the complete nucleotide sequences of goose and muscovy duck pervoviruses indicates common ancestral origin with adeno-associated virus 2
Oligino et al. Vector systems for gene transfer to joints.
US5478745A (en) Recombinant viral vector system
JPH01191686A (en) Composite plasmid
Mizuuchi et al. Integrative recombination of bacteriophage λ: in vitro study of the intermolecular reaction
Liu et al. Targeted correction of single-base-pair mutations with adeno-associated virus vectors under nonselective conditions
Drolet et al. The region of the herpes simplex virus type 1 LAT gene involved in spontaneous reactivation does not encode a functional protein
WO1996036364A1 (en) Recombinant viral vector system
Burton et al. Replication-defective genomic herpes simplex vectors: design and production
Wang et al. A novel terminal resolution-like site in the adeno-associated virus type 2 genome.
Boyle et al. Construction of recombinant fowlpox viruses carrying multiple vaccine antigens and immunomodulatory molecules
Gilden Interrelationships among RNA tumor viruses and host cells
Dirks et al. A multifunctional vector family for gene expression in mammalian cells

Legal Events

Date Code Title Description
ENP Entry into the national phase in:

Ref country code: AU

Ref document number: 2000 10949

Kind code of ref document: A

Format of ref document f/p: F

AL Designated countries for regional patents

Kind code of ref document: A2

Designated state(s): GH GM KE LS MW SD SL SZ TZ UG ZW AM AZ BY KG KZ MD RU TJ TM AT BE CH CY DE DK ES FI FR GB GR IE IT LU MC NL PT SE BF BJ CF CG CI CM GA GN GW ML MR NE SN TD TG

AK Designated states

Kind code of ref document: A2

Designated state(s): AE AL AM AT AU AZ BA BB BG BR BY CA CH CN CR CU CZ DE DK DM EE ES FI GB GD GE GH GM HR HU ID IL IN IS JP KE KG KP KR KZ LC LK LR LS LT LU LV MD MG MK MN MW MX NO NZ PL PT RO RU SD SE SG SI SK SL TJ TM TR TT TZ UA UG US UZ VN YU ZA ZW

DFPE Request for preliminary examination filed prior to expiration of 19th month from priority date (pct application filed before 20040101)
121 Ep: the epo has been informed by wipo that ep was designated in this application
AK Designated states

Kind code of ref document: A3

Designated state(s): AE AL AM AT AU AZ BA BB BG BR BY CA CH CN CR CU CZ DE DK DM EE ES FI GB GD GE GH GM HR HU ID IL IN IS JP KE KG KP KR KZ LC LK LR LS LT LU LV MD MG MK MN MW MX NO NZ PL PT RO RU SD SE SG SI SK SL TJ TM TR TT TZ UA UG US UZ VN YU ZA ZW

AL Designated countries for regional patents

Kind code of ref document: A3

Designated state(s): GH GM KE LS MW SD SL SZ TZ UG ZW AM AZ BY KG KZ MD RU TJ TM AT BE CH CY DE DK ES FI FR GB GR IE IT LU MC NL PT SE BF BJ CF CG CI CM GA GN GW ML MR NE SN TD TG

ENP Entry into the national phase in:

Ref country code: CA

Ref document number: 2347129

Kind code of ref document: A

Format of ref document f/p: F

Ref document number: 2347129

Country of ref document: CA

WWE Wipo information: entry into national phase

Ref document number: 511171

Country of ref document: NZ

WWE Wipo information: entry into national phase

Ref document number: 1999954648

Country of ref document: EP

Ref document number: 10949/00

Country of ref document: AU

WWP Wipo information: published in national office

Ref document number: 1999954648

Country of ref document: EP

REG Reference to national code

Ref country code: DE

Ref legal event code: 8642

WWG Wipo information: grant in national office

Ref document number: 10949/00

Country of ref document: AU

WWW Wipo information: withdrawn in national office

Ref document number: 1999954648

Country of ref document: EP