WO1999004026A3 - Lentiviral vectors - Google Patents

Lentiviral vectors Download PDF

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Publication number
WO1999004026A3
WO1999004026A3 PCT/US1998/014996 US9814996W WO9904026A3 WO 1999004026 A3 WO1999004026 A3 WO 1999004026A3 US 9814996 W US9814996 W US 9814996W WO 9904026 A3 WO9904026 A3 WO 9904026A3
Authority
WO
WIPO (PCT)
Prior art keywords
lentiviral
lentiviral vectors
ltr
lentiviral vector
rre
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/US1998/014996
Other languages
French (fr)
Other versions
WO1999004026A2 (en
Inventor
Shin-Tai Chen
Mehdi Gasmi
Jiing Kuan Yee
Douglas J Jolly
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 JP2000503232A priority Critical patent/JP2001510053A/en
Priority to AU85762/98A priority patent/AU8576298A/en
Priority to CA002296319A priority patent/CA2296319A1/en
Priority to EP98936923A priority patent/EP1003894A2/en
Publication of WO1999004026A2 publication Critical patent/WO1999004026A2/en
Publication of WO1999004026A3 publication Critical patent/WO1999004026A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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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
    • 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
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16021—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16051—Methods of production or purification of viral material
    • C12N2740/16052—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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • C12N2830/003—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/60—Vector systems having a special element relevant for transcription from viruses
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Lentiviral vectors are provided comprising a 5' lentiviral LTR, a tRNA binding site, a packaging signal, a promoter operably linked to one or more genes of interest, an origin of second strand DNA synthesis and a 3' lentiviral LTR, wherein the lentiviral vector contains a nuclear transport element that is not RRE. Also provided are expression cassettes and packaging cell lines for producing lentiviral vector particles.
PCT/US1998/014996 1997-07-18 1998-07-20 Lentiviral vectors Ceased WO1999004026A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
JP2000503232A JP2001510053A (en) 1997-07-18 1998-07-20 Lentivirus vector
AU85762/98A AU8576298A (en) 1997-07-18 1998-07-20 Lentiviral vectors
CA002296319A CA2296319A1 (en) 1997-07-18 1998-07-20 Lentiviral vectors
EP98936923A EP1003894A2 (en) 1997-07-18 1998-07-20 Lentiviral vectors

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US5306697P 1997-07-18 1997-07-18
US60/053,066 1997-07-18

Publications (2)

Publication Number Publication Date
WO1999004026A2 WO1999004026A2 (en) 1999-01-28
WO1999004026A3 true WO1999004026A3 (en) 1999-04-08

Family

ID=21981716

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1998/014996 Ceased WO1999004026A2 (en) 1997-07-18 1998-07-20 Lentiviral vectors

Country Status (5)

Country Link
EP (1) EP1003894A2 (en)
JP (1) JP2001510053A (en)
AU (1) AU8576298A (en)
CA (1) CA2296319A1 (en)
WO (1) WO1999004026A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107949424B (en) * 2015-03-20 2022-03-01 天普大学-联邦高等教育系统 TAT-induced CRISPR/endonuclease-based gene editing

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US6531123B1 (en) 1997-05-01 2003-03-11 Lung-Ji Chang Lentiviral vectors
WO2000000600A2 (en) * 1997-09-22 2000-01-06 Chang Lung Ji Lentiviral vectors, comprising modified major donor splice sites and major packaging signals
US20030044384A1 (en) 1997-10-09 2003-03-06 Pro-Virus, Inc. Treatment of neoplasms with viruses
US7470426B1 (en) 1997-10-09 2008-12-30 Wellstat Biologics Corporation Treatment of neoplasms with viruses
US7780962B2 (en) 1997-10-09 2010-08-24 Wellstat Biologics Corporation Treatment of neoplasms with RNA viruses
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
AU749059B2 (en) * 1997-12-12 2002-06-20 Cell Genesys, Inc. Therapeutic use of lentiviral vectors
WO1999036511A2 (en) * 1998-01-16 1999-07-22 Chiron Corporation Feline immunodeficiency virus gene therapy vectors
WO2000006760A1 (en) * 1998-07-29 2000-02-10 The Government Of The United States Of America, R Epresented By The Secretary, Department Of Health And Human Services Use of constitutive transport elements for host range control
JP2003530301A (en) 1999-04-15 2003-10-14 ウェルスタット バイオロジクス コーポレイション Treatment of neoplasms with viruses
CA2371216A1 (en) * 1999-04-23 2000-11-02 Centre For Translational Research In Cancer Pseudotyped retroviral vector for gene therapy of cancer
EP1849873B1 (en) * 1999-04-29 2011-10-12 Gbp Ip, Llc Method and means for producing high titer, safe, recombinant lentivirus vectors
US6730297B1 (en) 1999-05-28 2004-05-04 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing lysosomal storage disorders
CA2376790A1 (en) * 1999-06-09 2000-12-14 Syngenix Limited Siv-based packaging-defficient vectors
US8147822B1 (en) 1999-09-17 2012-04-03 Wellstat Biologics Corporation Oncolytic virus
US6656706B2 (en) 1999-12-23 2003-12-02 The United States Of America As Represented By The Department Of Health And Human Services Molecular clones with mutated HIV gag/pol, SIV gag and SIV env genes
AU2001278430A1 (en) * 2000-05-22 2001-12-03 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw Systemic and cardiovascular transduction with lentiviral vectors
AU2001265190A1 (en) * 2000-05-30 2001-12-11 University Of Rochester Siv derived lentiviral vector systems
US7575924B2 (en) 2000-11-13 2009-08-18 Research Development Foundation Methods and compositions relating to improved lentiviral vectors and their applications
US6712612B1 (en) 2000-12-12 2004-03-30 Genecure Llc Safe and stable retroviral helper cell line and related compositions and methods
ATE437221T1 (en) 2001-05-14 2009-08-15 Gbp Ip Llc LENTIVIRAL VECTORS ENCODING CLOTTING FACTORS FOR GENE THERAPY
EP1412493B1 (en) 2001-08-02 2011-10-05 Institut Clayton De La Recherche Methods and compositions relating to improved lentiviral vector production systems
EP1438075A4 (en) 2001-10-02 2006-04-19 Inst Clayton De La Rech Methods and compositions relating to restricted expression lentiviral vectors and their applications
MXPA05009526A (en) 2003-03-07 2005-12-15 Robarts Res Inst Use of myxoma virus for the therapeutic treatment of cancer and chronic viral infection.
US20050196381A1 (en) * 2003-09-09 2005-09-08 Xiaobin Lu Lentivirus vector-based approaches for generating an immune response to HIV in humans
TW200613554A (en) 2004-06-17 2006-05-01 Wyeth Corp Plasmid having three complete transcriptional units and immunogenic compositions for inducing an immune response to HIV
AU2006222500B2 (en) 2005-03-07 2011-09-01 Robarts Research Institute Use of a combination of Myxoma virus and rapamycin for therapeutic treatment
EP3031923A1 (en) 2014-12-11 2016-06-15 Institut Pasteur Lentiviral vector-based japanese encephalitis immunogenic composition
JP2018510160A (en) * 2015-03-20 2018-04-12 ブルーバード バイオ， インコーポレイテッド Vector preparation
US12590321B2 (en) 2016-03-19 2026-03-31 Exuma Biotech Corp. Methods and compositions for genetically modifying and expanding lymphocytes and regulating the activity thereof
US11325948B2 (en) 2016-03-19 2022-05-10 Exuma Biotech Corp. Methods and compositions for genetically modifying lymphocytes to express polypeptides comprising the intracellular domain of MPL
US11111505B2 (en) 2016-03-19 2021-09-07 Exuma Biotech, Corp. Methods and compositions for transducing lymphocytes and regulating the activity thereof
CA3057142A1 (en) * 2017-04-03 2018-10-11 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
EP3648776A4 (en) 2017-07-06 2021-04-07 The Medical College of Wisconsin, Inc. NEW IN VITRO AND IN VIVO ENRICHMENT STRATEGY TARGETING CHS-DERIVED LYMPHOCYTES TRANSDUCED BY A VECTOR FOR THE THERAPY OF DISORDERS
US20210228742A1 (en) 2018-04-27 2021-07-29 Seattle Children's Hospital D/B/A Seattle Children's Research Institute In vivo gene therapy using intraosseous delivery of a lentiviralgene construct
EP3784695B1 (en) 2018-04-27 2023-08-09 The Medical College of Wisconsin, Inc. Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders
US12440572B2 (en) 2018-05-18 2025-10-14 Zhengzhou Gensciences Inc. FVIII fusion protein and use thereof
WO2020059848A1 (en) * 2018-09-20 2020-03-26 国立大学法人東京医科歯科大学 Method for enhancing lentivirus vector production
WO2021055801A1 (en) 2019-09-18 2021-03-25 The Medical College Of Wisconsin, Inc. Improved alpha-galactosidase protein for enzyme replacement therapy (ert) and methods of use

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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107949424B (en) * 2015-03-20 2022-03-01 天普大学-联邦高等教育系统 TAT-induced CRISPR/endonuclease-based gene editing

Also Published As

Publication number Publication date
EP1003894A2 (en) 2000-05-31
CA2296319A1 (en) 1999-01-28
WO1999004026A2 (en) 1999-01-28
AU8576298A (en) 1999-02-10
JP2001510053A (en) 2001-07-31

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