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1. WO1996017053 - VECTORS FOR TISSUE-SPECIFIC REPLICATION

Publication Number WO/1996/017053
Publication Date 06.06.1996
International Application No. PCT/US1995/015455
International Filing Date 28.11.1995
Chapter 2 Demand Filed 26.06.1996
IPC
A61K 48/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C12N 15/861 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
861Adenoviral vectors
CPC
A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 35/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
35Antineoplastic agents
C12N 15/86
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
C12N 2710/10343
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
2710dsDNA Viruses
00011dsDNA Viruses
10011Adenoviridae
10311Mastadenovirus, e.g. human or simian adenoviruses
10341Use of virus, viral particle or viral elements as a vector
10343viral genome or elements thereof as genetic vector
C12N 2830/008
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
2830Vector systems having a special element relevant for transcription
008cell type or tissue specific enhancer/promoter combination
C12N 2830/32
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
2830Vector systems having a special element relevant for transcription
32being an silencer not forming part of the promoter region
Applicants
  • GENETIC THERAPY, INC. [US]/[US] (AllExceptUS)
  • HALLENBECK, Paul, L. [US]/[US] (UsOnly)
  • CHANG, Yung-Nien [US]/[US] (UsOnly)
  • CHIANG, Yawen, L. [US]/[US] (UsOnly)
Inventors
  • HALLENBECK, Paul, L.
  • CHANG, Yung-Nien
  • CHIANG, Yawen, L.
Agents
  • GOLDSTEIN, Jorge, A.
Priority Data
08/348,25828.11.1994US
08/487,99207.06.1995US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) VECTORS FOR TISSUE-SPECIFIC REPLICATION
(FR) VECTEURS DE REPLICATION A SPECIFICITE TISSULAIRE
Abstract
(EN)
The invention generally relates to targeted gene therapy using recombinant vectors and particularly adenovirus vectors. The invention specifically relates to replication-conditional vectors and methods for using them. Such vectors are able to selectively replicate in a target tissue to provide a therapeutic benefit from the presence of the vector $i(per se) or from heterologous gene products expressed from the vector and distributed throughout the tissue. In such vectors, a gene essential for replication is placed under the control of a heterologous tissue-specific transcriptional regulatory sequence. Thus, replication is conditioned on the presence of a factor(s) that induces transcription or the absence of a factor(s) that ihibits transcription of the gene by means of the transcriptional regulatory sequence with this vector; therefore, a target tissue can be selectively treated.
(FR)
La présente invention concerne une thérapie génique ciblée mettant en ÷uvre des vecteurs recombinants, en l'occurrence des vecteurs adénoviraux. L'invention concerne plus particulièrement des vecteurs fonction du mode de réplication ainsi que des procédés permettant de les utiliser. De tels vecteurs sont capables d'une réplication sélective dans un tissu cible apportant ainsi une amélioration thérapeutique imputable au vecteur en tant que tel ou imputable aux produits géniques hétérologues exprimés depuis le vecteur et se distribuant à travers le tissu. Dans ce type de vecteurs, un gène essentiel du processus de réplication est régi par une séquence de régulation de la transcription spécifique d'un tissu hétérologue. Il en résulte que la réplication est conditionnée par la présence d'un ou plusieurs facteurs induisant la transcription ou par l'absence d'un ou plusieurs facteurs bloquant la transcription de ce gène en raison de la séquence de régulation de la transcription accompagnant ce vecteur. Cette spécificité permet de traiter de façon sélective un tissu cible.
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