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.
Claims15
A composition of matter for delivering a gene to an ocular cell, said composition of matter comprising:
- a liposome having an exterior surface and an internal compartment;
- a plurality of targeting agents comprising blood-retinal barrier and ocular cell membrane targeting agents to provide targeting of said liposome to an ocular cell;
- 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
- a gene selected from the group consisting of opsin protein of rhodopsin gene, cyclic GMP phosophodiesterase α-subunit or β-subunit gene, the alpha subunit of the rod cyclic nucleotide gated channel gene, retinal pigmented epithelium-specific 65 kD protein gene, retinal binding protein 1 gene, ATP binding casette retina gene, peripherin/retinal degeneration slow gene, rod outer segment membrane protein 1 gene, arrestin gene, alpha-transducin gene, rhodopsin kinase gene, guanylate cyclase activator 1A gene, retina specific guanylate cyclase gene, the alpha subunit of the cone cyclic nucleotide gated cation channel gene and cone opsin genes, said gene being located within the internal compartment of said liposome.
A composition of matter for delivering a gene to an ocular cell, said composition of matter comprising:
- a) a receptor-specific liposome comprising:
- a liposome having an exterior surface and an internal compartment;
- a plurality of targeting agents comprising blood-retinal barrier and ocular cell membrane targeting agents to provide targeting of said liposome to an ocular cell;
- a plurality of conjugation agents wherein each targeting agent is connected to said exterior surface of said liposome via at least one of said conjugation agents;
- a gene selected from the group consisting of opsin protein of rhodopsin gene, cyclic GMP phosophodiesterase α-subunit or β-subunit gene, the alpha subunit of the rod cyclic nucleotide gated channel gene, retinal pigmented epithelium-specific 65 kD protein gene, retinal binding protein 1 gene, ATP binding casette retina gene, peripherin/retinal degeneration slow gene, rod outer segment membrane protein 1 gene, arrestin gene, alpha-transducin gene, rhodopsin kinase gene, guanylate cyclase activator 1A gene, retina specific guanylate cyclase gene, the alpha subunit of the cone cyclic nucleotide gated cation channel gene and cone opsin genes, said gene being located within the internal compartment of said liposome;
- b) a pharmaceutically acceptable carrier for said receptor-specific liposome.
Dependencies are inferred from the claim text.