Cns gene delivery using peripheral administration of aav...

Details Cns gene delivery using peripheral administration of aav... Cns gene delivery using peripheral administration of aav...

C - Chemistry, Metallurgy

12

N

C12N 15/864 (2006.01)

Patent

CA 2694241

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.

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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