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US 10,208,318 B2Granted
CNS gene delivery using peripheral administration of AAV vectors
AssigneesGenethon, Centre National de la Recherche Scientifique (CNRS)
InventorMartine Barkats
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Abstract
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.
Claims11
A method for expressing a protein in choroid plexus epithelial cells of a human subject, said method comprising:
- intravenously or intra-arterially administering to the human subject an effective amount of a double-stranded self-complementary human serotype 9 adeno-associated virus (AAV9) vector comprising a transgene encoding said protein,
- said administration resulting in said vector crossing the blood-brain barrier and expressing said protein in choroid plexus epithelial cells of said human subject,
- wherein said method is performed in the presence of an intact blood-brain barrier.
Dependencies are inferred from the claim text.