Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues
First Claim
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1. A compound comprising Receptor-Associated Protein (RAP) or RAP polypeptide conjugated to an agent of interest.
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Abstract
This invention provides compounds of conjugates of therapeutic or active agents with RAP or a RAP polypeptide, their pharmaceutical compositions and methods for using the such compounds and compositions in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly diseases of the central nervous system or lysosomal storage diseases.
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13 Claims
- 1. A compound comprising Receptor-Associated Protein (RAP) or RAP polypeptide conjugated to an agent of interest.
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3. A method of delivering a therapeutic or diagnostic/investigational agent into the central nervous system by increasing transport across the blood brain barrier (BBB) in a subject in need thereof, said method comprising:
administering to said subject a compound comprising Receptor Associated Protein (RAP) conjugated to a therapeutic or diagnostic/investigational agent in an amount effective to increase transport across the BBB.
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4. A method of treating a disease or condition in a subject in need thereof, said method comprising:
administering to said subject a compound comprising RAP conjugated to a therapeutic agent in an amount effective to treat said disease. - View Dependent Claims (5, 6, 7)
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8. A method of diagnosing a disease in a subject in need thereof, said method comprising:
administering to said subject a compound comprising RAP conjugated to a diagnostic/investigational agent in an amount effective to diagnose said disease.
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9. A method of delivering a therapeutic enzyme to a lysosome in a cell of a subject, said method comprising:
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(i) administering to said subject a compound comprising RAP or RAP polypeptide conjugated to a therapeutic or diagnostic agent;
(ii) transporting said compound across the cell membrane;
(iii) contacting said compound with an LRP receptor on said cell;
(iv) facilitating entry of said compound into said cell; and
(v) delivering said compound to said lysosome in said cell.
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10. A method of treating lysosomal storage diseases in a subject in need thereof, said method comprising:
administering to said subject a compound comprising RAP or RAP polypeptide conjugated to a therapeutic agent, wherein said compound crosses the cell membrane, enters cells and is delivered to lysosomes in an amount effective to treat said lysosomal storage disease. - View Dependent Claims (11, 12, 13)
Specification