WO2001055361A3 - Recombinant AAV packaging systems - Google Patents

Recombinant AAV packaging systems Download PDF

Info

Publication number
WO2001055361A3
WO2001055361A3 PCT/US2001/002709 US0102709W WO0155361A3 WO 2001055361 A3 WO2001055361 A3 WO 2001055361A3 US 0102709 W US0102709 W US 0102709W WO 0155361 A3 WO0155361 A3 WO 0155361A3
Authority
WO
WIPO (PCT)
Prior art keywords
recombinant aav
vector
particles
herpesvirus
recombinant
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.)
Ceased
Application number
PCT/US2001/002709
Other languages
French (fr)
Other versions
WO2001055361A2 (en
Inventor
Stephen Hardy
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.)
Novartis Vaccines and Diagnostics Inc
Original Assignee
Chiron Corp
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 Chiron Corp filed Critical Chiron Corp
Priority to CA002397532A priority Critical patent/CA2397532A1/en
Priority to AU34597/01A priority patent/AU3459701A/en
Priority to EP01906723A priority patent/EP1257656A2/en
Publication of WO2001055361A2 publication Critical patent/WO2001055361A2/en
Publication of WO2001055361A3 publication Critical patent/WO2001055361A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

Classifications

    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/13043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

Methods and compositions are provided for producing recombinant AAV vector particles; comprising the general steps of (a) introducing into a host cell (i) pfloxAAV a recombinant AAV vector in which two loxP recombination sites are inserted flanking the rep/cap sequence, (ii) a recombinant viral vector encoding plasmid, and (iii) a plasmid encoding herpesvirus, cytomegalovirus, or adenoviral functions, or a herpesvirus, cytomegalovirus, or, adenovirus itself, in order to produce flox AAV particles and recombinant AAV particles; and (b) introducing into a second host cell (i) the recombinant AAV particles of (a), (ii) a vector which directs the expression of Cre, and (iii) a vector which directs the expression of herpesvirus, CMV, or adenovirus helper functions, such that said recombinant AAV vector particles are produced.
PCT/US2001/002709 2000-01-26 2001-01-26 Recombinant aav packaging systems Ceased WO2001055361A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
CA002397532A CA2397532A1 (en) 2000-01-26 2001-01-26 Recombinant aav packaging systems
AU34597/01A AU3459701A (en) 2000-01-26 2001-01-26 Recombinant aav packaging systems
EP01906723A EP1257656A2 (en) 2000-01-26 2001-01-26 Recombinant aav packaging systems

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US17853600P 2000-01-26 2000-01-26
US60/178,536 2000-01-26

Publications (2)

Publication Number Publication Date
WO2001055361A2 WO2001055361A2 (en) 2001-08-02
WO2001055361A3 true WO2001055361A3 (en) 2002-04-25

Family

ID=22652931

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2001/002709 Ceased WO2001055361A2 (en) 2000-01-26 2001-01-26 Recombinant aav packaging systems

Country Status (5)

Country Link
US (1) US6429001B1 (en)
EP (1) EP1257656A2 (en)
AU (1) AU3459701A (en)
CA (1) CA2397532A1 (en)
WO (1) WO2001055361A2 (en)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1427816A4 (en) * 2001-09-18 2004-12-01 Clontech Lab Inc METHOD FOR PRODUCING ADENOVIRAL VECTORS BASED ON A SITE SPECIFIC RECOMBINASE
US20050080027A1 (en) * 2001-11-30 2005-04-14 Markus Horer Optimized production of viral vectors derived from paroviruses in packaging and production cells by hsv infection or treatment with dna methylation inhibitors
WO2004075861A2 (en) * 2003-02-26 2004-09-10 Children's Hospital, Inc. Recombinant adeno-associated virus production
US20050281786A1 (en) * 2004-06-18 2005-12-22 David Poulsen AAV mediated gene delivery to cochlear cells
WO2008042450A2 (en) * 2006-01-20 2008-04-10 Lawrence Livermore National Security, Llc. Multiplex detection of respiratory pathogens
EP2426142A3 (en) 2006-10-16 2012-06-13 Genelux Corporation Modified vaccinia virus strains for use in a diagnostic and therapeutic method
WO2008150496A2 (en) * 2007-05-31 2008-12-11 Genelux Corporation Assay for sensitivity to chemotherapeutic agents
JP5213075B2 (en) 2007-06-15 2013-06-19 ジェネラックス・コーポレイション Microorganisms for tumor imaging and / or treatment
WO2009120978A2 (en) * 2008-03-27 2009-10-01 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor
CN114262690A (en) 2011-04-15 2022-04-01 吉恩勒克斯公司 Clonal strains of attenuated vaccinia virus and methods of use thereof
WO2013138522A2 (en) 2012-03-16 2013-09-19 Genelux Corporation Methods for assessing effectiveness and monitoring oncolytic virus treatment
WO2013158265A1 (en) 2012-04-20 2013-10-24 Genelux Corporation Imaging methods for oncolytic virus therapy
US20140140959A1 (en) 2012-10-05 2014-05-22 Aladar A. Szalay Energy Absorbing-Based Diagnostic and Therapeutic Methods Employing Nucleic Acid Molecules Encoding Chromophore-Producing Enzymes
US9585971B2 (en) * 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
WO2015103438A2 (en) 2014-01-02 2015-07-09 Genelux Corporation Oncolytic virus adjunct therapy with agents that increase virus infectivity
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
KR102423442B1 (en) 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 Targeting Peptides for Directing Adeno-Associated Viruses
CN109843306A (en) 2016-08-19 2019-06-04 卡琳缪恩股份有限公司 Use the method and composition of self-complementary type recombinant adeno-associated virus treatment illness
CN110325547B (en) 2016-11-07 2023-12-26 德格克斯有限公司 New amyloid beta oligomer-specific binding molecules
KR20250121159A (en) 2016-12-07 2025-08-11 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 IL-1RA CDNAs
GB201800903D0 (en) 2018-01-19 2018-03-07 Oxford Genetics Ltd Vectors
WO2020068990A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
CA3260631A1 (en) 2022-07-08 2024-01-11 Viromissile, Inc. Oncolytic vaccinia viruses and recombinant viruses and methods of use thereof
CN117683797B (en) * 2023-12-04 2024-07-05 广州派真生物技术有限公司 A plasmid system for packaging recombinant adeno-associated virus and its application

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994013788A1 (en) * 1992-12-04 1994-06-23 University Of Pittsburgh Recombinant viral vector system
WO1995014771A1 (en) * 1993-11-24 1995-06-01 GOVERNMENT OF THE UNITED STATES, represented by THE SECRETARY OF THE DEPARTMENT OF HEALTH AND HUMAN SERVICES: National Institutes of Health Vector systems for the generation of adeno-associated virus particles
US5622856A (en) * 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US5658785A (en) * 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5801030A (en) * 1995-09-01 1998-09-01 Genvec, Inc. Methods and vectors for site-specific recombination

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9226537D0 (en) 1992-12-21 1993-02-17 Smithkline Beecham Plc Compounds

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994013788A1 (en) * 1992-12-04 1994-06-23 University Of Pittsburgh Recombinant viral vector system
WO1995014771A1 (en) * 1993-11-24 1995-06-01 GOVERNMENT OF THE UNITED STATES, represented by THE SECRETARY OF THE DEPARTMENT OF HEALTH AND HUMAN SERVICES: National Institutes of Health Vector systems for the generation of adeno-associated virus particles
US5658785A (en) * 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5622856A (en) * 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US5801030A (en) * 1995-09-01 1998-09-01 Genvec, Inc. Methods and vectors for site-specific recombination

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BERGEMANN J ET AL.: "Excision of specific DNA-sequences from integrated retroviral vectors via site-specific recombination", NUCL. ACIDS RES., vol. 23, no. 21, 11 November 1995 (1995-11-11), pages 4451 - 4456, XP000644463 *
CARTER PJ AND SAMULSKI RJ: "Adeno-associated viral vectors as gene delivery vehicles (review)", INT. J. MOL. MED., vol. 6, July 2000 (2000-07-01), pages 17 - 27, XP000943604 *
FERRARI F K ET AL: "New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors", NATURE MEDICINE,US,NATURE PUBLISHING, CO, vol. 3, no. 11, November 1997 (1997-11-01), pages 1295 - 1297, XP002098257, ISSN: 1078-8956 *
TAL J.: "Adeno-associated virus-based vectors in gene therapy", J. BIOMED. SCI., vol. 7, July 2000 (2000-07-01), pages 279 - 291, XP000994813 *

Also Published As

Publication number Publication date
AU3459701A (en) 2001-08-07
WO2001055361A2 (en) 2001-08-02
US6429001B1 (en) 2002-08-06
EP1257656A2 (en) 2002-11-20
CA2397532A1 (en) 2001-08-02
US20020058325A1 (en) 2002-05-16

Similar Documents

Publication Publication Date Title
WO2001055361A3 (en) Recombinant AAV packaging systems
CA2418442A1 (en) Novel helper functions for recombinant vector production
WO2003092594A3 (en) Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
CA2303477A1 (en) Chimaeric adenoviruses
AU2220800A (en) Compositions and methods for packaging of alphavirus vectors
AU7335296A (en) Recombinant sendai virus
WO2001083797A3 (en) Polynucleotides for use in recombinant adeno-associated virus virion production
AU2001294624A1 (en) Recombinant adenovirus vectors that are replication-competent in tert-expressing cells
EP1046711A3 (en) Recombinant adeno-associated virus
NO995382L (en) Lentivirus based gene transfer vectors
WO2001055362A3 (en) Hybrid adeno-retroviral vector for the transfection of cells
AU2001220765A1 (en) Mixed buffer system for stabilizing polypeptide formulations
WO2002040665A3 (en) Complementing cell lines
WO1998039464A3 (en) Adenovirus vectors containing heterologous transcription regulatory elements and methods of using same
WO2003061582A3 (en) Generation of recombinant adeno-associated viral vectors by a complete adenovirus-mediated approach
AU4624197A (en) Packaging cell lines for use in facilitating the development of high-capacity adenoviral vectors
AU7630196A (en) Recombinant live vaccine containing feline herpes virus type 1, particularly for treating feline infectious peritonitis
AU6217798A (en) Avian recombinant live vaccine using, as vector, the avian infectious laryngotracheitis virus
EP1132478A3 (en) Helper virus for the preparation of recombinant viral recombinants
AU5551996A (en) An adenovirus helper-virus system
CA2228269A1 (en) High efficiency helper system for aav vector production
AU2002211710A1 (en) Cytomegalovirus intron a fragments
WO1995034651A3 (en) Corticotropin-releasing factor2 receptors
AU657488B2 (en) New recombinant herpes viruses, a vaccine based on these recombinants, their preparation process, genes, vectors and plasmids used in this process
WO2000052187A3 (en) Enhanced system for construction of adenovirus vectors

Legal Events

Date Code Title Description
AK Designated states

Kind code of ref document: A2

Designated state(s): AE AG AL AM AT AU AZ BA BB BG BR BY BZ CA CH CN CR CU CZ DE DK DM DZ 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 MA MD MG MK MN MW MX MZ 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: A2

Designated state(s): GH GM KE LS MW MZ 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 TR BF BJ CF CG CI CM GA GN GW ML MR NE SN TD TG

121 Ep: the epo has been informed by wipo that ep was designated in this application
DFPE Request for preliminary examination filed prior to expiration of 19th month from priority date (pct application filed before 20040101)
WWE Wipo information: entry into national phase

Ref document number: 34597/01

Country of ref document: AU

WWE Wipo information: entry into national phase

Ref document number: 2397532

Country of ref document: CA

ENP Entry into the national phase

Ref country code: JP

Ref document number: 2001 554392

Kind code of ref document: A

Format of ref document f/p: F

WWE Wipo information: entry into national phase

Ref document number: 2001906723

Country of ref document: EP

WWP Wipo information: published in national office

Ref document number: 2001906723

Country of ref document: EP

REG Reference to national code

Ref country code: DE

Ref legal event code: 8642

WWW Wipo information: withdrawn in national office

Ref document number: 2001906723

Country of ref document: EP