AAV-mediated delivery of DNA to cells of the nervous system
AssigneesThe Rockefeller University, Yale University
InventorsMichael G. Kaplitt, Matthew J. During
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Abstract
The invention relates to a method of delivering exogenous DNA to a target cell of the mammalian central nervous system using an adeno-associated virus (AAV)-derived vector. Also included in the invention are the AAV-derived vectors containing exogenous DNA which encodes a protein or proteins which prevent or treat nervous system disease, and a method of prevent or treating such disease.
Claims26
A method of delivering to a mammalian nervous system target cell a DNA that is expressed in the target cell for greater than 3 months comprising administering an adenoassociated virus-derived (AAV) vector to the target cell, wherein said vector transduces the target cell;
- and wherein said vector is free of both wildtype and helper virus and has been modified to comprise a DNA which is exogenous to an adeno-associated virus.
An AAV-derived vector which retains only the replication and packaging signals of AAV, and which comprises expressible genes encoding tyrosine hydroxylase and aromatic amino acid decarboxylase.
Dependencies are inferred from the claim text.