WO2002024897A3 - Conditionally replicating viral vectors and their use - Google Patents

Conditionally replicating viral vectors and their use Download PDF

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Publication number
WO2002024897A3
WO2002024897A3 PCT/US2001/029976 US0129976W WO0224897A3 WO 2002024897 A3 WO2002024897 A3 WO 2002024897A3 US 0129976 W US0129976 W US 0129976W WO 0224897 A3 WO0224897 A3 WO 0224897A3
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WO
WIPO (PCT)
Prior art keywords
vectors
improved
methods
host cells
conditionally replicating
Prior art date
Application number
PCT/US2001/029976
Other languages
French (fr)
Other versions
WO2002024897A2 (en
WO2002024897A9 (en
Inventor
Yung-Nien Chang
Xiaobin Lu
Vladimir Slepushkin
Betty Conde
Brian Davis
Qiao Yu
Yanping Yang
Randal Merling
Wei Han
Yajin Ni
Yuexia Li
Boro Dropulic
Original Assignee
Virxsys
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 Virxsys filed Critical Virxsys
Priority to JP2002529492A priority Critical patent/JP2004524813A/en
Priority to CA002422544A priority patent/CA2422544A1/en
Priority to EP01973503A priority patent/EP1356070A2/en
Priority to IL15498701A priority patent/IL154987A0/en
Priority to AU9307501A priority patent/AU9307501A/en
Priority to NZ525283A priority patent/NZ525283A/en
Publication of WO2002024897A2 publication Critical patent/WO2002024897A2/en
Priority to NO20031293A priority patent/NO20031293L/en
Publication of WO2002024897A9 publication Critical patent/WO2002024897A9/en
Publication of WO2002024897A3 publication Critical patent/WO2002024897A3/en

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/108—Plasmid DNA episomal 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/40—Vector systems having a special element relevant for transcription being an insulator
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Virology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Oncology (AREA)
  • Wood Science & Technology (AREA)
  • Communicable Diseases (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • AIDS & HIV (AREA)
  • Biochemistry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The present invention provides improved conditionally replicating vectors that have improved safety against the generation of replication competent vectors or virus. Also disclosed are methods of making, propagating and selectively packaging, modifying, and using such vectors. Included are improved helper constructs, host cells, for use with the improved vectors as well as pharmaceutical compositions and host cells comprising the vectors, the use of vector containing host cells to screen drugs, and methods of using the vectors to determine gene function. The methods also include the prophylatic and therapeutic treatment of disease, especially viral infection, and HIV infection in particular.
PCT/US2001/029976 2000-09-22 2001-09-21 Conditionally replicating viral vectors and their use WO2002024897A2 (en)

Priority Applications (7)

Application Number Priority Date Filing Date Title
JP2002529492A JP2004524813A (en) 2000-09-22 2001-09-21 Vectors replicating with improved conditioning, methods for their production and use
CA002422544A CA2422544A1 (en) 2000-09-22 2001-09-21 Conditionally replicating viral vectors and their use
EP01973503A EP1356070A2 (en) 2000-09-22 2001-09-21 Conditionally replicating viral vectors and their use
IL15498701A IL154987A0 (en) 2000-09-22 2001-09-21 Conditionally replicating viral vectors and their use
AU9307501A AU9307501A (en) 2000-09-22 2001-09-21 Improved conditionally replicating vectors, methods for their production and use
NZ525283A NZ525283A (en) 2000-09-22 2001-09-21 Conditionally replicating vectors, methods for their production and use
NO20031293A NO20031293L (en) 2000-09-22 2003-03-20 Conditionally replicating viral vectors and their use

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US66789300A 2000-09-22 2000-09-22
US09/667,893 2000-09-22

Publications (3)

Publication Number Publication Date
WO2002024897A2 WO2002024897A2 (en) 2002-03-28
WO2002024897A9 WO2002024897A9 (en) 2003-03-20
WO2002024897A3 true WO2002024897A3 (en) 2003-07-31

Family

ID=24680093

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2001/029976 WO2002024897A2 (en) 2000-09-22 2001-09-21 Conditionally replicating viral vectors and their use

Country Status (10)

Country Link
EP (1) EP1356070A2 (en)
JP (1) JP2004524813A (en)
AU (1) AU9307501A (en)
CA (1) CA2422544A1 (en)
CZ (1) CZ2003784A3 (en)
IL (1) IL154987A0 (en)
NO (1) NO20031293L (en)
NZ (1) NZ525283A (en)
RU (1) RU2301260C2 (en)
WO (1) WO2002024897A2 (en)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2002241980B2 (en) 2001-01-25 2008-01-31 Virxsys Corporation Methods and compositions for identifying gene function
US20030026791A1 (en) * 2001-03-27 2003-02-06 Laurent Humeau Conditionally replicating vectors for inhibiting viral infections
US20050196381A1 (en) * 2003-09-09 2005-09-08 Xiaobin Lu Lentivirus vector-based approaches for generating an immune response to HIV in humans
FR2872170B1 (en) * 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse NON-INTERACTIVE AND NON-REPLICATIVE LENTIVIRUS, PREPARATION AND USES
BR112012013664B1 (en) 2009-12-10 2020-11-10 Turnstone Limited Partnership oncolitic rhabdovirus
CA2833870C (en) * 2011-04-22 2020-03-10 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US9364532B2 (en) 2011-06-08 2016-06-14 Children's Hospital Of Eastern Ontario Research Institute Inc. Compositions and methods for glioblastoma treatment
BR112014008225A2 (en) * 2011-10-05 2017-04-11 Molmed Spa viral vector purification systems
RU2488633C1 (en) * 2011-11-16 2013-07-27 Общество с ограниченной ответственностью "Лаборатория медицинской биотехнологии" (ООО "ЛМБТ") Expression plasmid vector for heterological expression of recombinant proteins, high-frequency integration and amplified amplification of expression cassette in cells of mammals, bicistronic messenger riziform, method of production of stable lines of producents of recombinant proteins using specified vector, method for production of recombinant proteins
EP2931880B1 (en) * 2012-12-12 2019-03-20 Turnstone Limited Partnership Compositions and methods for the treatment of brain cancers
WO2014127478A1 (en) 2013-02-21 2014-08-28 Children's Hospital Of Eastern Ontario Research Institute Inc. Vaccine composition
RU2548816C1 (en) * 2013-12-16 2015-04-20 Мамонтова Марина Васильевна Method of extraction of microrna from biological fluids
RU2725286C2 (en) 2016-05-13 2020-06-30 4Д Молекьюлар Терапьютикс Инк. Versions of adenoassociated virus capsids and methods of use thereof
KR20200037353A (en) 2017-08-07 2020-04-08 더 리전트 오브 더 유니버시티 오브 캘리포니아 Platform for creating safe cell therapy products
CN114174513A (en) * 2019-07-23 2022-03-11 牛津生物医学（英国）有限公司 Optimized production of lentiviral vectors
US20230374451A1 (en) * 2020-09-23 2023-11-23 Regents Of The University Of Minnesota Immortalized keratinocytes, lentivirus for keratinocyte immortalization, and methods of use

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997020060A1 (en) * 1995-11-28 1997-06-05 The Johns Hopkins University School Of Medicine Conditionally replicating viral vectors and their use
WO1999041397A1 (en) * 1998-02-17 1999-08-19 Oxford Biomedica (Uk) Limited Anti-viral vectors

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997020060A1 (en) * 1995-11-28 1997-06-05 The Johns Hopkins University School Of Medicine Conditionally replicating viral vectors and their use
WO1999041397A1 (en) * 1998-02-17 1999-08-19 Oxford Biomedica (Uk) Limited Anti-viral vectors

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BLOOD, vol. 98, no. 11 Part 2, 16 November 2001 (2001-11-16), 43rd Annual Meeting of the American Society of Hematology, Part 2;Orlando, Florida, USA; December 07-11, 2001, November 16, 2001, pages 409b, ISSN: 0006-4971 *
DATABASE BIOSIS [online] BIOSCIENCES INFORMATION SERVICE, PHILADELPHIA, PA, US; 16 November 2001 (2001-11-16), DROPULIC BORO ET AL: "Pre-clinical optimization of HIV vectors expressing anti-HIV antisense for phase I clinical trials in HIV-infected patients.", XP002226635, Database accession no. PREV200200157496 *
DROPULIC B ET AL: "A CONDITIONALLY REPLICATING HIV-1 VECTOR INTERFeRES WITH WILD-TYPE HIV-1 REPLICATION AND SPREAD", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA, NATIONAL ACADEMY OF SCIENCE. WASHINGTON, US, vol. 93, no. 20, 1 October 1996 (1996-10-01), pages 11103 - 11108, XP002028427, ISSN: 0027-8424 *
MAUTINO MARIO R ET AL: "Potent inhibition of human immunodeficiency virus type 1 replication by conditionally replicating human immunodeficiency virus-based lentiviral vectors expressing envelope antisense mRNA.", HUMAN GENE THERAPY, vol. 11, no. 14, 20 September 2000 (2000-09-20), pages 2025 - 2037, XP002226634, ISSN: 1043-0342 *

Also Published As

Publication number Publication date
WO2002024897A2 (en) 2002-03-28
RU2301260C2 (en) 2007-06-20
CA2422544A1 (en) 2002-03-28
CZ2003784A3 (en) 2003-11-12
JP2004524813A (en) 2004-08-19
NZ525283A (en) 2008-06-30
NO20031293D0 (en) 2003-03-20
WO2002024897A9 (en) 2003-03-20
EP1356070A2 (en) 2003-10-29
AU9307501A (en) 2002-04-02
NO20031293L (en) 2003-05-14
IL154987A0 (en) 2003-10-31

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