WO2000040741A3 - Lentivirus vector system - Google Patents

Lentivirus vector system Download PDF

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Publication number
WO2000040741A3
WO2000040741A3 PCT/US2000/000390 US0000390W WO0040741A3 WO 2000040741 A3 WO2000040741 A3 WO 2000040741A3 US 0000390 W US0000390 W US 0000390W WO 0040741 A3 WO0040741 A3 WO 0040741A3
Authority
WO
WIPO (PCT)
Prior art keywords
packaging
vector
encapsidation
vectors
transgene
Prior art date
Application number
PCT/US2000/000390
Other languages
French (fr)
Other versions
WO2000040741A9 (en
WO2000040741A2 (en
Inventor
Suresh K Arya
Original Assignee
Us Health
Suresh K Arya
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 Us Health, Suresh K Arya filed Critical Us Health
Priority to AU24953/00A priority Critical patent/AU2495300A/en
Priority to US09/869,588 priority patent/US6790657B1/en
Publication of WO2000040741A2 publication Critical patent/WO2000040741A2/en
Publication of WO2000040741A3 publication Critical patent/WO2000040741A3/en
Publication of WO2000040741A9 publication Critical patent/WO2000040741A9/en
Priority to US10/731,988 priority patent/US7226780B2/en

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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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15051—Methods of production or purification of viral material
    • C12N2740/15052—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
    • 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
    • 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
    • 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
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor

Landscapes

  • 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)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

A method is disclosed for improving encapsidation of transgene RNA using retroviral packaging and transfer vectors. An HIV-2 transfer vector, which includes the transgene, is introduced into a packaging cell that is also transfected with (or stably expresses) an HIV-2 derived packaging vector or a combination of packaging vectors. The packaging vector has mutations in packaging signal sequences that are both upstream and downstream of the 5' splice donor site. It can also be composed of a combination of two or more partial vectors. A transfer vector, which is introduced into the packaging cell line, has a mutation that renders its splice donor site non-functional. Transgene RNA expression and encapsidation from these cells is markedly increased, but with little or no levels of infectious viral RNA encapsidation.
PCT/US2000/000390 1999-01-07 2000-01-06 Lentivirus vector system WO2000040741A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
AU24953/00A AU2495300A (en) 1999-01-07 2000-01-06 Lentivirus vector system
US09/869,588 US6790657B1 (en) 1999-01-07 2000-01-06 Lentivirus vector system
US10/731,988 US7226780B2 (en) 1999-01-07 2003-12-09 Lentivirus vector system

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US11524799P 1999-01-07 1999-01-07
US60/115,247 1999-01-07

Related Child Applications (3)

Application Number Title Priority Date Filing Date
US09/869,588 A-371-Of-International US6790657B1 (en) 1999-01-07 2000-01-06 Lentivirus vector system
US09869588 A-371-Of-International 2000-01-06
US10/731,988 Division US7226780B2 (en) 1999-01-07 2003-12-09 Lentivirus vector system

Publications (3)

Publication Number Publication Date
WO2000040741A2 WO2000040741A2 (en) 2000-07-13
WO2000040741A3 true WO2000040741A3 (en) 2000-11-02
WO2000040741A9 WO2000040741A9 (en) 2001-10-25

Family

ID=22360159

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2000/000390 WO2000040741A2 (en) 1999-01-07 2000-01-06 Lentivirus vector system

Country Status (2)

Country Link
AU (1) AU2495300A (en)
WO (1) WO2000040741A2 (en)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU4936500A (en) * 1999-05-21 2000-12-12 Oxford Biomedica (Uk) Limited Method for selecting improved vectors
US20040093623A1 (en) * 2000-12-20 2004-05-13 Zeger Debyser Non-human animal disease models
GB0108065D0 (en) * 2001-03-30 2001-05-23 Syngenix Ltd Viral vectors
US7271150B2 (en) 2001-05-14 2007-09-18 United States Of America, Represented By The Secretary, Department Of Health And Human Services Modified growth hormone
MY144532A (en) 2001-08-20 2011-09-30 Lundbeck & Co As H Novel method for down-regulation of amyloid
US8163892B2 (en) 2002-07-08 2012-04-24 Oncolys Biopharma, Inc. Oncolytic virus replicating selectively in tumor cells
WO2006089001A2 (en) * 2005-02-16 2006-08-24 Lentigen Corporation Lentiviral vectors and their use
CN108728495A (en) * 2018-05-31 2018-11-02 深圳市免疫基因治疗研究院 A kind of Sanfilippo A syndromes slow virus carrier, slow virus and its preparation method and application
CN108795986A (en) * 2018-05-31 2018-11-13 深圳市免疫基因治疗研究院 A kind of haemophilia A slow virus carrier, slow virus and its preparation method and application
CN108715868A (en) * 2018-05-31 2018-10-30 深圳市免疫基因治疗研究院 A kind of Gaucher slow virus carriers, slow virus and its preparation method and application
CN108728494A (en) * 2018-05-31 2018-11-02 深圳市免疫基因治疗研究院 A kind of X-SCID slow virus carriers, slow virus and its preparation method and application
CN108707627A (en) * 2018-05-31 2018-10-26 深圳市免疫基因治疗研究院 A kind of MLD slow virus carriers, slow virus and its preparation method and application
CN108715867A (en) * 2018-05-31 2018-10-30 深圳市免疫基因治疗研究院 A kind of Sanfilippo B syndromes slow virus carrier, slow virus and its preparation method and application
CN108795985A (en) * 2018-05-31 2018-11-13 深圳市免疫基因治疗研究院 A kind of mucopolysaccharidosis slow virus carrier, slow virus and its preparation method and application
CN108676815A (en) * 2018-05-31 2018-10-19 深圳市免疫基因治疗研究院 A kind of haemophilia B slow virus carrier, slow virus and its preparation method and application

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5665577A (en) * 1989-02-06 1997-09-09 Dana-Farber Cancer Institute Vectors containing HIV packaging sequences, packaging defective HIV vectors, and uses thereof
WO1997036481A1 (en) * 1996-04-02 1997-10-09 The Regents Of The University Of California Design of novel highly efficient hiv based packaging systems for gene therapy
WO1998051810A1 (en) * 1997-05-13 1998-11-19 University Of North Carolina At Chapel Hill Lentivirus-based gene transfer vectors
WO1999061598A2 (en) * 1998-05-26 1999-12-02 University Of Florida Lentiviral vector compositions and methods of use

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5665577A (en) * 1989-02-06 1997-09-09 Dana-Farber Cancer Institute Vectors containing HIV packaging sequences, packaging defective HIV vectors, and uses thereof
WO1997036481A1 (en) * 1996-04-02 1997-10-09 The Regents Of The University Of California Design of novel highly efficient hiv based packaging systems for gene therapy
WO1998051810A1 (en) * 1997-05-13 1998-11-19 University Of North Carolina At Chapel Hill Lentivirus-based gene transfer vectors
WO1999061598A2 (en) * 1998-05-26 1999-12-02 University Of Florida Lentiviral vector compositions and methods of use

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
ARYA S K ET AL.: "Human immunodeficiency virus type 2 lentivirus vectors for gene transfer: expression and potential for helper virus-free packaging", HUMAN GENE THERAPY, vol. 9, no. 9, 10 June 1998 (1998-06-10), pages 1371 - 1380, XP000923199 *
BORG K T ET AL.: "Involvement of human immunodeficiency virus type-1 splice sites in the cytoplasmic accumulation of viral RNA", VIROLOGY, vol. 236, no. 1, 15 September 1997 (1997-09-15), pages 95 - 103, XP000910228 *
BUCHSCHACHER G L ET AL.: "Development of lentiviral vectors for gene therapy for human diseases", BLOOD, vol. 95, no. 8, 15 April 2000 (2000-04-15), pages 2499 - 2504, XP002141657 *
POESCHLA E ET AL.: "Identification of a human immunodeficiency virus type 2 (HIV-2) encapsidation determinant and transduction of nondividing human cells by HIV-2-based lentivirus vectors", JOURNAL OF VIROLOGY, vol. 72, no. 8, August 1998 (1998-08-01), pages 6527 - 6536, XP002141655 *
SADAIE M R ET AL.: "Towards developing HIV-2 lentivirus-based retroviral vectors for gene therapy: dual gene expression in the context of HIV-2 LTR and Tat", JOURNAL OF MEDICAL VIROLOGY, vol. 54, no. 2, February 1998 (1998-02-01), pages 118 - 128, XP000915147 *

Also Published As

Publication number Publication date
AU2495300A (en) 2000-07-24
WO2000040741A9 (en) 2001-10-25
WO2000040741A2 (en) 2000-07-13

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