Delivery of therapeutic agents by gene therapy
AssigneesThe United States of America as represented by the Department of Health and Human Services, Genetic Therapy, Inc.
InventorsEdward H. Oldfield, Zvi Ram, Gerard J. McGarrity
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Abstract
A process for treating a disease or disorder of a host by delivery of a therapeutic agent to the brain of the host, which comprises transducing endothelial cells of blood vessels located in the brain of a host in vivo with a vector including a polynucleotide encoding a therapeutic agent. The vector is administered intravascularly to the host, and the vector produces the therapeutic agent in the endothelial cells.
Claims4
A method for inhibiting the growth of a brain tumor in a subject, comprising:
- administering directly into a carotid artery or vertebral artery in close proximity to the brain tumor, a therapeutically effective amount of a replication-defective retroviral vector comprising a nucleic acid encoding a negative selective marker gene product that converts enzymatically an interacting agent into a cytotoxic agent, thereby selectively transfecting endothelial cells in the vaculature of the brain tumor and the negative selective marker is expressed in the endothelial cells;
- and
- administering the interacting agent to the vasculature of the brain tumor, wherein the negative selective marker gene product converts enzymatically the interacting agent to a cytotoxic agent, thereby destroying the endothelial cells in the vasculature of the brain tumor and inhibiting the growth of the brain tumor.
Dependencies are inferred from the claim text.