WO2005062812A2 - Systeme a base de raav pour la disruption genique de cellules somatiques - Google Patents

Systeme a base de raav pour la disruption genique de cellules somatiques Download PDF

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Publication number
WO2005062812A2
WO2005062812A2 PCT/US2004/042597 US2004042597W WO2005062812A2 WO 2005062812 A2 WO2005062812 A2 WO 2005062812A2 US 2004042597 W US2004042597 W US 2004042597W WO 2005062812 A2 WO2005062812 A2 WO 2005062812A2
Authority
WO
WIPO (PCT)
Prior art keywords
homology arms
locus
desired human
cells
human locus
Prior art date
Application number
PCT/US2004/042597
Other languages
English (en)
Other versions
WO2005062812A3 (fr
Inventor
Bert Vogelstein
Kenneth W. Kinzler
Manu Kohli
Carlo Rago
Original Assignee
The Johns Hopkins University
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 The Johns Hopkins University filed Critical The Johns Hopkins University
Publication of WO2005062812A2 publication Critical patent/WO2005062812A2/fr
Publication of WO2005062812A3 publication Critical patent/WO2005062812A3/fr

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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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT

Abstract

Une nouvelle manifestation donne à penser que des vecteurs viraux adéno-associés recombinants (rAAV) peuvent être utilisés pour le ciblage de gènes spécifiques dans des cellules somatiques humaines. Nous avons développé une procédure de construction de vecteurs rAAV utilisant la PCR de fusion et une étape de clonage unique qui simplifie considérablement le processus d'inactivation. Nous démontrons son utilité par la disruption de gènes à des emplacements spécifiques au sein de cellules humaines du cancer du colon et de cellules épithéliales normales immortalisées. Cette technologie devrait être largement applicable à des études in vitro nécessitant la manipulation du génome humain.
PCT/US2004/042597 2003-12-22 2004-12-21 Systeme a base de raav pour la disruption genique de cellules somatiques WO2005062812A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US53175103P 2003-12-22 2003-12-22
US60/531,751 2003-12-22

Publications (2)

Publication Number Publication Date
WO2005062812A2 true WO2005062812A2 (fr) 2005-07-14
WO2005062812A3 WO2005062812A3 (fr) 2005-09-09

Family

ID=34738691

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2004/042597 WO2005062812A2 (fr) 2003-12-22 2004-12-21 Systeme a base de raav pour la disruption genique de cellules somatiques

Country Status (1)

Country Link
WO (1) WO2005062812A2 (fr)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2814969A4 (fr) * 2012-02-10 2016-02-17 Univ Minnesota Assimiliation de l'adn

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5474935A (en) * 1990-05-23 1995-12-12 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus (AAV)-based eucaryotic vectors
US5948653A (en) * 1997-03-21 1999-09-07 Pati; Sushma Sequence alterations using homologous recombination
US6274354B1 (en) * 1996-09-06 2001-08-14 The Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5474935A (en) * 1990-05-23 1995-12-12 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus (AAV)-based eucaryotic vectors
US6274354B1 (en) * 1996-09-06 2001-08-14 The Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
US5948653A (en) * 1997-03-21 1999-09-07 Pati; Sushma Sequence alterations using homologous recombination

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2814969A4 (fr) * 2012-02-10 2016-02-17 Univ Minnesota Assimiliation de l'adn

Also Published As

Publication number Publication date
WO2005062812A3 (fr) 2005-09-09

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