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1. US20190153474 - CNS GENE DELIVERY USING PERIPHERAL ADMINISTRATION OF AAV VECTORS

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Claims

1.- 13. (canceled)
14. A method for expressing a transgene in cerebrospinal fluid secretory cells of a subject, said method comprising peripherally administering to said subject an effective amount of a single-stranded adeno-associated virus (AAV) vector comprising an AAV9 capsid and a single-stranded genome comprising said transgene.
15. The method according to claim 14, wherein the AAV vector is a pseudotyped AAV vector.
16. The method according to claim 15, wherein the AAV vector comprises a genome comprising AAV2 inverted terminal repeats, said genome being encapsidated in an AAV9 capsid.
17. The method according to claim 14, wherein the AAV vector has a replication defective AAV genome lacking functional Rep and Cap encoding viral sequences.
18. The method according to claim 14, wherein expression of the transgene is controlled by a ubiquitous promoter.
19. The method according to claim 14, wherein expression of the transgene is controlled by a regulated promoter.
20. The method according to claim 14, wherein expression of the transgene is controlled by a tissue-specific promoter.
21. The method according to claim 14, wherein the AAV vector is administered by intravascular injection.
22. The method according to claim 14, wherein the AAV vector is administered by intravenous injection.
23. The method according to claim 14, wherein the AAV vector is administered by intra-arterial injection.
24. The method according to claim 14, wherein the transgene encodes a therapeutic protein
25. The method according to claim 14, wherein the transgene encodes a therapeutic protein 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).
26. The method according to claim 14, wherein said subject suffers from a central nervous system (CNS) disorder 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.