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1. WO2009013290 - CNS GENE DELIVERY USING PERIPHERAL ADMINISTRATION OF AAV VECTORS

Publication Number WO/2009/013290
Publication Date 29.01.2009
International Application No. PCT/EP2008/059595
International Filing Date 22.07.2008
IPC
C12N 15/864 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
864Parvoviral vectors
CPC
A61K 48/0075
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
0075characterised by an aspect of the delivery route, e.g. oral, subcutaneous
A61P 25/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
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 2750/14143
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
2750ssDNA Viruses
00011ssDNA Viruses
14011Parvoviridae
14111Dependovirus, e.g. adenoassociated viruses
14141Use of virus, viral particle or viral elements as a vector
14143viral genome or elements thereof as genetic vector
Applicants
  • GENETHON [FR]/[FR] (AllExceptUS)
  • CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE [FR]/[FR] (AllExceptUS)
  • BARKATS, Martine [FR]/[FR] (UsOnly)
Inventors
  • BARKATS, Martine
Agents
  • BECKER, Philippe
Priority Data
07301263.523.07.2007EP
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) CNS GENE DELIVERY USING PERIPHERAL ADMINISTRATION OF AAV VECTORS
(FR) ADMINISTRATION DE GÈNE AU SYSTÈME NERVEUX CENTRAL PAR ADMINISTRATION PÉRIPHÉRIQUE DE VECTEURS AAV
Abstract
(EN)
The present invention relates to compositions and methods for the delivery of therapeutic proteins to the CNS using recombinant AAV vectors. More specifically, the invention relates to compositions and methods for delivering proteins into the cerebrospinal fluid of mammalian subjects through peripheral administration of AAV vectors. The invention may be used to treat various disorders of the central nervous system, including degenerative diseases and motor neuron diseases.
(FR)
La présente invention porte sur des compositions et des procédés pour l'administration de protéines thérapeutiques au système nerveux central à l'aide de vecteurs AAV recombinants. Plus spécifiquement, l'invention porte sur des compositions et des procédés pour administrer des protéines dans le liquide céphalo-rachidien de sujets mammifères par administration périphérique de vecteurs AAV. L'invention peut être utilisée pour traiter divers troubles du système nerveux central, comprenant des maladies dégénératives et des maladies du neurone moteur.
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