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 treat nervous system disease, and a method of treating such disease.
Claims15
A method for ameliorating a symptom of a central nervous system disorder in a mammal, the method comprising, direct administration of an adeno-associated virus-derived vector to a target cell in the brain of the mammal, said vector comprising a DNA sequence, wherein said DNA sequence is exogenous to an adeno-associated virus and comprises a sequence encoding a therapeutic protein in operable linkage with a promoter sequence, wherein said adeno-associated virus-derived vector is free of both wild-type and helper virus, and wherein the exogenous DNA sequence is expressed in the target cell for greater than 3 months such that the symptom of the central nervous system disorder is ameliorated.
An adeno-associated virus-derived vector comprising only the replication and packaging signals of adeno-associated virus, and further comprising a DNA molecule comprising a DNA sequence encoding tyrosine hydroxylase, a DNA sequence encoding aromatic amino acid decarboxylase, and a promoter sequence.
Dependencies are inferred from the claim text.