Modified adeno-associated virus vectors and delivery thereof into the central nervous system
AssigneeCoave Therapeutics
InventorsPhilippe Moullier, Willem Broekaert
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Abstract
The present invention relates to modified adeno-associated virus (AAV) vectors for use in transducing a cell in the central nervous system (CNS) of a subject, and for use in the prevention or treatment of a CNS disease. In particular, the modified AAV vectors according to the present invention comprise at least one surface-bound saccharide, and are to be administered directly to the CNS but not intracerebroventricularly.
Claims19
A method of treating a central nervous system (CNS) disease in a primate subject, comprising:
- administering a modified adeno-associated virus (AAV) vector directly to an intraparenchymal site of the primate subject, wherein the modified AAV vector comprises at least one surface-bound saccharide;
- wherein administration of the modified AAV vector directly to the intraparenchymal site results in transduction of at least one brain tissue that is distant from the site of administration;
- and wherein the CNS disease is a neurological disease or a disease affecting neurons.
A method for transducing one or more cells within a plurality of brain tissues of a primate subject, comprising:
- administering a modified adeno associated virus (AAV) vector directly to an intraparenchymal site of the primate subject, wherein the modified AAV vector comprises:
- at least one surface-bound saccharide;
- and
- at least one transgene;
- wherein expression of the transgene occurs for at least 1 hour after administration;
- and
- wherein the plurality of brain tissues comprises at least one brain tissue that is distant from the site of administration.
Dependencies are inferred from the claim text.