Non-invasive gene targeting to the brain
AssigneeThe Regents of the University of California
InventorWilliam M. Pardridge
Life of this patent
Estimating the term…
Abstract
Liposomes containing therapeutic genes are conjugated to multiple blood-brain barrier and brain cell membrane targeting agents to provide transport of the encapsulated gene across the blood-brain barrier and brain cell membrane. Once across the blood-brain barrier and brain cell membrane, the encapsulated gene expresses the encoded therapeutic agent within the brain to provide treatment and diagnosis of disease.
Claims26
A receptor-specific liposome for delivering a gene across the blood brain barrier and the brain cell membrane, said liposome comprising:
- a liposome having an exterior surface and an internal compartment;
- a gene comprising a sufficient amount of genetic information to encode an agent, said gene being located within the internal compartment of said liposome;
- a plurality of blood brain barrier and brain cell membrane targeting agents;
- and
- 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.
A method for delivering a gene across the blood brain barrier for expression in the brain, said method comprising the steps of administering to an animal an effective amount of a preparation comprising:
- a) a receptor-specific liposome comprising:
- a liposome having an exterior surface and an internal compartment;
- a gene comprising a sufficient amount of genetic information to encode a pharmaceutical agent, said gene being located within the internal compartment of said liposome;
- a plurality of blood brain barrier and brain 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,
- wherein said receptor-specific liposome crosses said blood brain barrier to provide expression of said gene in said brain.
A composition comprising:
- a) a receptor-specific liposome comprising:
- a liposome having an exterior surface and an internal compartment;
- a gene comprising a sufficient amount of genetic information to encode an agent, said gene being located within the internal compartment of said liposome;
- a plurality of blood brain barrier 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 receptor-specific nanocontainer for delivering a gene to a cell having a specific receptor, said receptor-specific nanocontainer comprising:
- a nanocontainer having an exterior surface and an internal compartment;
- a gene comprising a sufficient amount of genetic information to encode a pharmaceutical agent, said gene being located within the internal compartment of said nanocontainer;
- a plurality of receptor-specific targeting agents which are capable of targeting the receptor located on said cell;
- 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 method for delivering a gene to a cell having a specific receptor, said method comprising the step of administering to an animal an effective amount of a preparation comprising:
- a) a receptor-specific nanocontainer comprising:
- a nanocontainer having an exterior surface and an internal compartment;
- a gene comprising a sufficient amount of genetic information to encode a pharmaceutical agent, said gene being located within the internal compartment of said nanocontainer;
- a plurality of receptor-specific targeting agents which are capable of targeting the receptor located on said cell;
- 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;
- and
- b) a pharmaceutically acceptable carrier for said receptor-specific nanocontainer,
- wherein said receptor-specific nanocontainer is delivered to said cell having having a specific receptor.
Dependencies are inferred from the claim text.