CA2251738A1 - Vecteurs viraux a cibles definies - Google Patents

Vecteurs viraux a cibles definies Download PDF

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Publication number
CA2251738A1
CA2251738A1 CA 2251738 CA2251738A CA2251738A1 CA 2251738 A1 CA2251738 A1 CA 2251738A1 CA 2251738 CA2251738 CA 2251738 CA 2251738 A CA2251738 A CA 2251738A CA 2251738 A1 CA2251738 A1 CA 2251738A1
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CA
Canada
Prior art keywords
vector
nucleic acid
cell
aav
viral
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.)
Abandoned
Application number
CA 2251738
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English (en)
Inventor
Michael Mamounas
Gang Yu
Qicheng Yang
Qi-Xiang Li
Jack Barber
Mang Yu
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Immusol Inc
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Individual
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.)
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Publication of CA2251738A1 publication Critical patent/CA2251738A1/fr
Abandoned legal-status Critical Current

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    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2896Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against molecules with a "CD"-designation, not provided for elsewhere
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • 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
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/13011Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041Use of virus, viral particle or viral elements as a vector
    • C12N2740/13043Use 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
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/13011Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041Use of virus, viral particle or viral elements as a vector
    • C12N2740/13045Special targeting system for viral 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/00011Details
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral 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
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/40Vectors comprising a peptide as targeting moiety, e.g. a synthetic peptide, from undefined source
    • 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
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
    • C12N2810/85Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
    • C12N2810/859Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian from immunoglobulins

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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

L'invention se rapporte à des vecteurs viraux dirigés sur des cibles définies, soit des types de cellules sélectionnés, par blocage des sites de liaison des cellules virales de phénotype sauvage et par introduction d'un agent de ciblage dans la particule vectorielle. L'agent de ciblage se lie au type de cellule sélectionné en se fixant à une molécule sur la surface de la cellule, ou en se fixant à un second agent de ciblage qui se lie à la cellule sélectionnée. L'invention se rapporte également à des vecteurs à base de parvovirus, de rétrovirus, et d'herpèsvirus et de virus Ad, ainsi qu'à des banques de vecteurs viraux comportant l'agent de ciblage. L'invention se rapporte encore à des procédés de sélection de vecteurs viraux de recombinaison dans ledites banques, à des ligands polypeptidiques isolés des banques de vecteurs phages ou viraux.
CA 2251738 1996-04-16 1997-04-15 Vecteurs viraux a cibles definies Abandoned CA2251738A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US1549796P 1996-04-16 1996-04-16
US60/015,497 1996-04-16

Publications (1)

Publication Number Publication Date
CA2251738A1 true CA2251738A1 (fr) 1997-10-23

Family

ID=21771753

Family Applications (1)

Application Number Title Priority Date Filing Date
CA 2251738 Abandoned CA2251738A1 (fr) 1996-04-16 1997-04-15 Vecteurs viraux a cibles definies

Country Status (4)

Country Link
EP (1) EP0927044A4 (fr)
AU (1) AU2678097A (fr)
CA (1) CA2251738A1 (fr)
WO (1) WO1997038723A1 (fr)

Families Citing this family (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6465253B1 (en) 1994-09-08 2002-10-15 Genvec, Inc. Vectors and methods for gene transfer to cells
US5846782A (en) * 1995-11-28 1998-12-08 Genvec, Inc. Targeting adenovirus with use of constrained peptide motifs
US6127525A (en) * 1995-02-21 2000-10-03 Cornell Research Foundation, Inc. Chimeric adenoviral coat protein and methods of using same
US5770442A (en) 1995-02-21 1998-06-23 Cornell Research Foundation, Inc. Chimeric adenoviral fiber protein and methods of using same
WO1999019500A1 (fr) * 1997-10-10 1999-04-22 The Government Of The United States Of America, Represented By The Secretary, Department Of Healt H And Human Services Complexe de vecteur viral et de ligand biotinyles pour une fourniture de genes ciblee
US6794128B2 (en) 1998-04-24 2004-09-21 The Regents Of The University Of California Methods of selecting internalizing antibodies
US7244826B1 (en) 1998-04-24 2007-07-17 The Regents Of The University Of California Internalizing ERB2 antibodies
WO1999055365A1 (fr) 1998-04-30 1999-11-04 Cornell Research Foundation, Inc. Vecteurs adenoviraux avec proteines a deux types de genes de fibre
DE19827457C1 (de) * 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
US20030017138A1 (en) 1998-07-08 2003-01-23 Menzo Havenga Chimeric adenoviruses
CA2342396A1 (fr) 1998-09-11 2000-03-23 Genvec, Inc. Adenovirus a ciblage altnernatif
EP1123114B1 (fr) 1998-10-21 2005-12-28 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Particules assimilees a des virus pour l'induction d'auto-anticorps
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
WO2000028004A1 (fr) 1998-11-10 2000-05-18 The University Of North Carolina At Chapel Hill Vecteurs viraux et leurs procedes d'elaboration et d'administration
EP1131457B1 (fr) * 1998-11-19 2010-10-06 Georgetown University Systeme d'apport genique systemique de virus/ligands et de therapie genique
US6929946B1 (en) 1998-11-20 2005-08-16 Crucell Holland B.V. Gene delivery vectors provided with a tissue tropism for smooth muscle cells, and/or endothelial cells
US6913922B1 (en) 1999-05-18 2005-07-05 Crucell Holland B.V. Serotype of adenovirus and uses thereof
US6492169B1 (en) 1999-05-18 2002-12-10 Crucell Holland, B.V. Complementing cell lines
WO2000073478A2 (fr) * 1999-06-01 2000-12-07 University Of Washington Vecteurs adenoviraux recombinants pour l'infection specifique de cellules, l'integration de genomes et l'expression de proteines fibreuses chimeriques
US7094398B1 (en) 1999-06-01 2006-08-22 University Of Washington Recombinant adenoviral vectors expressing chimeric fiber proteins for cell specific infection and genome integration
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
DE19933288A1 (de) * 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
DE19933719A1 (de) * 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
AU2001238079A1 (en) * 2000-02-09 2001-08-20 Med Immune, Inc. Antibody gene therapy with adeno-associated viral vectors
US6762031B2 (en) * 2000-06-16 2004-07-13 University Of Medicine And Dentistry Of New Jersey Targeting viral vectors to specific cells
US7647184B2 (en) 2001-08-27 2010-01-12 Hanall Pharmaceuticals, Co. Ltd High throughput directed evolution by rational mutagenesis
EP2363487A3 (fr) * 2001-12-21 2012-03-21 Medigene AG Bibliothèque de gènes structuraux modifiés ou de particules à capside modifiée utiles pour l'identification de clones viraux ayant un tropisme cellulaire désiré
WO2004083441A2 (fr) * 2003-03-19 2004-09-30 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Bibliotheque peptidique aleatoire s'affichant sur des vecteurs aav
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
CA2591544A1 (fr) 2004-12-15 2006-06-22 The University Of North Carolina At Chapel Hill Vecteurs chimeriques
WO2010093784A2 (fr) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Vecteurs viraux modifiés et procédés de fabrication et d'utilisation de ceux-ci
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
EP3254703B1 (fr) 2011-04-22 2020-02-19 The Regents of The University of California Virions de virus adéno-associé avec capside variant et procédés d'utilisation de ceux-ci
EP3003391B1 (fr) 2013-05-31 2021-09-22 The Regents of The University of California Variants de virus adéno-associés et leurs méthodes d'utilisation
US10000741B2 (en) 2014-03-17 2018-06-19 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
EP3265178A4 (fr) 2015-03-02 2018-09-05 Adverum Biotechnologies, Inc. Compositions et procédés d'administration intravitréenne de polynucléotides à des cônes rétiniens
US10883117B2 (en) 2015-03-24 2021-01-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
KR102511979B1 (ko) 2016-07-29 2023-03-20 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
US11236360B2 (en) 2016-12-09 2022-02-01 Regents Of The University Of Minnesota Adeno-associated viruses engineered for selectable tropism
KR20200039617A (ko) 2017-08-28 2020-04-16 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 아데노-연관 바이러스 캡시드 변이체 및 이의 사용 방법

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) * 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
WO1991018088A1 (fr) * 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Vecteurs eucaryotiques a base de virus adeno-associes (aav)
SE503225C2 (sv) * 1991-10-30 1996-04-22 Leif Lindholm Konsult Ab Virus-antikroppskomplex för införing av virus i mammalieceller
GB9223084D0 (en) * 1992-11-04 1992-12-16 Imp Cancer Res Tech Compounds to target cells
CA2148934C (fr) * 1992-11-09 2005-08-02 French W. Anderson Particules vectorielles ciblables
US5478745A (en) * 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
EP0786010A1 (fr) * 1994-03-04 1997-07-30 University Of Medicine And Dentistry Of New Jersey Transfert genique specifique de types de cellules a l'aide de vecteurs retroviraux contenant des proteines de fusion d'enveloppe d'anticorps et d'enveloppe de type sauvage
EP0840797B1 (fr) * 1995-07-25 2014-09-03 Crucell Holland B.V. Procedes et moyens d'apport cible de genes

Also Published As

Publication number Publication date
EP0927044A4 (fr) 1999-09-08
AU2678097A (en) 1997-11-07
EP0927044A1 (fr) 1999-07-07
WO1997038723A1 (fr) 1997-10-23

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