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

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CLAIMS

1. The use of an AAV vector encoding a therapeutic protein for the manufacture of a medicament for treating a CNS disorder in a subject by peripheral administration of said AAV vector to said subject, said administration allowing infection of cerebrospinal fluid secretory cells of the brain and subsequent secretion of the therapeutic protein into the cerebrospinal fluid.

2. The use of an AAV vector encoding a therapeutic protein for the manufacture of a medicament for treating a CNS disorder through secretion of said therapeutic protein into the cerebrospinal fluid upon peripheral injection of said vector.

3. The use of an AAV vector for secreting a protein into the cerebrospinal fluid of a subject by peripheral injection of said vector under conditions allowing infection of cerebrospinal fluid secretory cells of the brain, particularly the epithelial cells of the plexus choroids and/or of the ependyma and/or a meningeal membrane).

4. The use of any one of claims 1 to 3, wherein said peripheral injection comprises intraperitoneal (i.p.), intramuscular (i.m.) or intravenous (i.v.) injection.

5. The use of any one of claims 1 to 4, wherein said AAV vector is a human serotype AAV vector, preferably selected from serotypes 1, 2, 3, 4, 5, 6, 7, 8, 9, 10 and 11, more preferably from AAVl, AAV2, AAV6 and AAV9.

6. The use of any one of claims 1 to 5, wherein the AAV vector is a pseudotyped AAV vector.

7. The use of any one of claims 1 to 6, wherein the AAV vector is a single stranded AAV vector.

8. The use of any one of claims 1 to 6, wherein the AAV vector is a double-stranded self-complementary AAV vector.

9. The use of any one of claims 1 to 7, wherein the AAV vector comprises a replication defective AAV genome lacking functional Rep and Cap coding viral sequences.

10. The use of any one of the preceding claims, wherein the therapeutic protein is selected from growth factors, cytokines, hormones, neurotransmitters, enzymes, anti-apoptotic factors, angiogenic factors, and any protein known to be mutated in pathological disorders such as the "survival of motor neuron" protein (SMN).

11. The use of any one of the preceding claims, wherein the CNS disorder is selected from neurodegenerative diseases, neuromuscular diseases, lysosomal diseases, trauma, bone marrow injuries, cancers of the nervous system, demyelinating diseases, autoimmune diseases of the nervous system, neurotoxic syndromes, sleeping disorders.

12. The use of any one of the preceding claims, wherein the vector is a pseudotyped AAV vector comprising an AAV2-derived genome packaged in an AAVl- or AAV9-derived capsid.

13. The use of any one of the preceding claims, wherein expression of the therapeutic protein in the vector is controlled by an ubiquitous, regulated and/or tissue-specific promoter.