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1. CA2694241 - CNS GENE DELIVERY USING PERIPHERAL ADMINISTRATION OF AAV VECTORS

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CLAIMS
1. Use of an adeno-associated virus serotype 9 AAV9 vector or of a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome for peripheral administration encoding a therapeutic protein for the manufacture of a medicament for treating a CNS disorder in a subject, said administration allowing infection of cerebrospinal fluid secretory cells of the brain and subsequent secretion of the therapeutic protein into the cerebrospinal fluid, wherein said peripheral administration is intraperitoneal (i.p.) administration.
2. Use of an adeno-associated virus serotype 9 AAV9 vector or a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome for peripheral administration encoding a therapeutic protein for the manufacture of a medicament for treating a CNS disorder in a subject, said administration allowing infection of cerebrospinal fluid secretory cells of the brain and subsequent secretion of the therapeutic protein into the cerebrospinal fluid, wherein said peripheral administration is intramuscular (i.m.) administration.
3. Use of an adeno-associated virus serotype 9 AAV9 vector or a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome for peripheral administration encoding a therapeutic protein for the manufacture of a medicament for treating a CNS disorder in a subject, said administration allowing infection of cerebrospinal fluid secretory cells of the brain and subsequent secretion of the therapeutic protein into the cerebrospinal fluid, wherein said peripheral administration is intravenous (i.v.) administration.
4. Use of an AAV9 vector or a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome for peripheral administration encoding a therapeutic protein for treating a CNS disorder in a subject, said administration allowing infection of cerebrospinal fluid secretory cells of the brain and subsequent secretion of the
therapeutic protein into the cerebrospinal fluid, wherein said peripheral administration is intraperitoneal (i.p.) administration.
5. Use of an AAV9 vector or a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome for peripheral administration encoding a therapeutic protein for treating a CNS disorder in a subject, said administration allowing infection of cerebrospinal fluid secretory cells of the brain and subsequent secretion of the therapeutic protein into the cerebrospinal fluid, wherein said peripheral administration is intramuscular (i.m.) administration.
6. Use of an AAV9 vector or a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome for peripheral administration encoding a therapeutic protein for treating a CNS disorder in a subject, said administration allowing infection of cerebrospinal fluid secretory cells of the brain and subsequent secretion of the therapeutic protein into the cerebrospinal fluid, wherein said peripheral administration is intravenous (i.v.) administration.
7. Use of an AAV9 vector or a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome and 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, wherein said vector is for peripheral injection, wherein said peripheral injection is intraperitoneal (i.p.) injection and allows infection of cerebrospinal fluid secretory cells of the brain.
8. Use of an AAV9 vector or a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome and 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, wherein said vector is for peripheral injection, wherein said peripheral injection is intramuscular (i.m.) injection and allows infection of cerebrospinal fluid secretory cells of the brain.
9. Use of an AAV9 vector or a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome and 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, wherein said vector is for peripheral injection, wherein said peripheral injection is intravenous (i.v.) injection and allows infection of cerebrospinal fluid secretory cells of the brain.
10. Use of an AAV9 vector or a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome and encoding a therapeutic protein for treating a CNS disorder through secretion of said therapeutic protein into the cerebrospinal fluid, wherein said vector is for peripheral injection, wherein said peripheral injection is intraperitoneal (i.p.) injection and allows infection of cerebrospinal fluid secretory cells of the brain.
11. Use of an AAV9 vector or a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome and encoding a therapeutic protein for treating a CNS disorder through secretion of said therapeutic protein into the cerebrospinal fluid, wherein said vector is for peripheral injection, wherein said peripheral injection is intramuscular (i.m.) injection and allows infection of cerebrospinal fluid secretory cells of the brain.
12. Use of an AAV9 vector or a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome and encoding a therapeutic protein for treating a CNS disorder through secretion of said therapeutic protein into the cerebrospinal fluid, wherein said vector is for peripheral injection, wherein said peripheral injection is intravenous (i.v.) injection and allows infection of cerebrospinal fluid secretory cells of the brain.
13. Use of an AAV9 vector or a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome and encoding a therapeutic protein for treating a CNS disorder through secretion of said therapeutic protein into the cerebrospinal fluid for secreting a protein into the cerebrospinal fluid of a subject, said vector being for peripheral injection and allowing infection of cerebrospinal fluid secretory cells of the brain, wherein said peripheral injection is intraperitoneal (i.p.) injection.
14. Use of an AAV9 vector or a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome and encoding a therapeutic protein for secreting a protein into the cerebrospinal fluid of a subject, said vector being for peripheral injection and allowing infection of cerebrospinal fluid secretory cells of the brain, wherein said peripheral injection is intramuscular (i.m.) injection.
15. Use of an AAV9 vector or a pseudotyped AAV9 vector having an AAV9 capsid, said vector having a double-stranded self-complementary genome and encoding a therapeutic protein for secreting a protein into the cerebrospinal fluid of a subject, said vector being for peripheral injection and allowing infection of cerebrospinal fluid secretory cells of the brain, wherein said peripheral injection is intravenous (i.v.) injection.
16. The use of any one of claims 1 to 15, wherein the cerebrospinal fluid secretory cells of the brain are epithelial cells of the plexus choroids, ependymal, a meningeal membrane, or a combination thereof.
17. The use of any one of claims 1 to 16, wherein the AAV9 vector or the pseudotyped AAV9 vector having said AAV9 capsid comprises a replication defective AAV genome lacking functional Rep and Cap coding viral sequences.
18. The use of any one of claims 1 to 16, wherein the therapeutic protein is a growth factor, a cytokine, hormone, a neurotransmitter, an enzyme, an anti-apoptotic factor, an angiogenic factor, or any protein known to be mutated in a pathological disorder.
19. The use of claim 18, wherein the protein known to be mutated in said pathological disorder is a "survival of motor neuron" protein SMN.
20. The use of any one of claims 1 to 19, wherein the CNS disorder is a neurodegenerative disease, a neuromuscular disease, a lysosomal disease, a trauma, a bone marrow injury, a cancer of the nervous system, a demyelinating disease, an autoimmune disease of the nervous system, a neurotoxic syndrome, or a sleeping disorder.
21. The use of any one of claims 1 to 20, wherein the pseudotyped AAV9 vector having said AAV9 capsid comprises an AAV2-derived genome packaged in an AAV9-derived capsid.
22. The use of any one of claims 1 to 21, wherein expression of the therapeutic protein in the vector is controlled by an ubiquitous promoter, a regulated promoter, a tissuespecific promoter, or a combination thereof
23. An AAV9 vector, wherein the genome of said AAV9 vector encodes the "survival of motor neuron" SMN protein.
24. The AAV9 vector of claim 23, wherein said vector is a pseudotyped AAV9 vector.
25. The AAV9 vector of claim 23 or 24, wherein said vector comprises a doublestranded selfcomplementary genome.