Non-invasive gene targeting to ocular cells
AssigneeThe Regents of the University of California
InventorWilliam M. Pardridge
Life of this patent
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Abstract
Liposomes containing therapeutic genes are conjugated to multiple targeting agents to provide transport of the encapsulated gene across the blood-retinal barrier and the plasma membrane of ocular cells. Once across the blood-retinal barrier and ocular cell membrane, the encapsulated gene expresses the encoded therapeutic agent within the ocular cells to provide diagnosis and/or treatment of disease.
Claims24
A receptor-specific liposome for delivering a therapeutic gene to ocular cells, said liposome comprising:
- a liposome having an exterior surface and an internal compartment;
- a therapeutic gene comprising a sufficient amount of genetic information to encode a therapeutic agent, said therapeutic gene being located within the internal compartment of said nanocontainer;
- a plurality of targeting agents comprising blood-retinal barrier and ocular cell membrane targeting agents;
- and
- a plurality of conjugation agents wherein each targeting agent is connected to the exterior surface of said nanocontainer via at least one of said conjugation agents.
A non-invasive method for delivering a therapeutic gene to ocular cells for expression therein, said method comprising administering to a patient an effective amount of a pharmaceutical preparation comprising:
- a) a receptor-specific liposome comprising:
- a liposome having an exterior surface and an internal compartment;
- a therapeutic gene comprising a sufficient amount of genetic information to encode a therapeutic agent, said therapeutic gene being located within the internal compartment of said liposome;
- a plurality of targeting agents comprising blood-retinal barrier and ocular cell membrane targeting agents;
- a plurality of conjugation agents wherein each targeting agent is connected to the exterior surface of said liposome via at least one of said conjugation agents;
- and
- b) a pharmaceutically acceptable carrier for said receptor-specific liposome.
A pharmaceutical preparation comprising:
- a) a receptor-specific liposome comprising:
- a liposome having an exterior surface and an internal compartment;
- a therapeutic gene comprising a sufficient amount of genetic information to encode a therapeutic agent, said therapeutic gene being located within the internal compartment of said liposome;
- a plurality of targeting agents comprising blood-retinal barrier and ocular cell membrane targeting agents;
- a plurality of conjugation agents wherein each targeting agent is connected to the exterior surface of said liposome via at least one of said conjugation agents;
- and
- b) a pharmaceutically acceptable carrier for said receptor-specific liposome.
Dependencies are inferred from the claim text.