Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues
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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.
44 Citations
18 Claims
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1-2. -2. (canceled)
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3. A method of delivering a therapeutic or diagnostic/investigational agent into the central nervous system in a subject in need thereof, said method comprising:
administering to said subject a compound comprising a Receptor Associated Protein (RAP) amino acid sequence having at least 80% homology to SEQ ID NO;
1, wherein the RAP amino acid sequence retains binding to a lipoprotein receptor-related protein (LRP) receptor and is conjugated to the therapeutic or diagnostic/investigational agent.- View Dependent Claims (4, 5, 6, 7, 14, 15, 16, 17, 18)
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8-13. -13. (canceled)
Specification