AAV4 vector and uses thereof
AssigneeThe United States of America, as represented by the Secretary, Department of Health and Human Services
InventorsJohn A. Chiorini, Robert M. Kotin, Beverly Davidson
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Abstract
The present invention relates to AAV4 vectors for methods of delivering nucleic acids to cells. Specifically, the present invention provides methods of delivering nucleic acids to specific regions and cells of the brain, particularly ependymal cells.
Claims12
A method of delivering a nucleic acid to an ependymal cell comprising administering to the ependymal cell an AAV4 particle containing a vector comprising the nucleic acid inserted between a pair of AAV inverted terminal repeats, thereby delivering the nucleic acid to the ependymal cell.
A method of delivering a nucleic acid to a subject comprising administering to an ependymal cell from the subject an AAV4 particle comprising the nucleic acid inserted between a pair of AAV inverted terminal repeats, and returning the ependymal cell to the subject, thereby delivering the nucleic acid to the subject.
A method of delivering a nucleic acid to an ependymal cell in a subject comprising administering to the subject an AAV4 particle comprising the nucleic acid inserted between a pair of AAV inverted terminal repeats, thereby delivering the nucleic acid to an ependymal cell in the subject.
A method of delivering a nucleic acid to an ependymal cell in a subject having antibodies to AAV2 comprising administering to the subject an AAV4 particle comprising the nucleic acid, thereby delivering the nucleic acid to an ependymal cell in the subject.
Dependencies are inferred from the claim text.