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1. US20100130594 - CNS gene delivery using peripheral administration of AAV vectors

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Claims

1. 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.
2. The method of claim 1, wherein said subject suffers from a disorder of the central nervous system selected from spinal muscular atrophy and amyotrophic lateral sclerosis.
3. The method of claim 1, wherein the AAV vector is a pseudotyped AAV vector.
4. The method of claim 1, wherein the AAV vector comprises a replication defective AAV genome lacking functional Rep and Cap coding viral sequences.
5. The method of claim 1, wherein the protein is selected from the group consisting of growth factors, cytokines, hormones, neurotransmitters, enzymes, anti-apoptotic factors, and angiogenic factors.
6. The method of claim 1, wherein the protein is survival of motor neuron (SMN) protein.
7. The method of claim 1, wherein the vector is a pseudotyped AAV vector comprising an AAV2-derived genome packaged in an AAV9-derived capsid.
8. The method of claim 1, wherein expression of the protein in the vector is controlled by a ubiquitous, regulated or tissue-specific promoter.
9. The method of claim 1, further comprising detecting said protein in the choroid plexus epithelial cells of the human subject.
10. The method of claim 1, wherein said vector is administered by intravenous injection.
11. The method of claim 1, wherein said vector is administered by intraarterial injection.