Protein aggregation regulators
AssigneeThe Regents of the University of CA
InventorsMarc I Diamond, Sonia K Pollitt
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Abstract
Disclosed herein is a high-throughput assay to measure intracellular polyglutamine protein aggregation using fluorescence resonance energy transfer (FRET). Three libraries of over 3000 biologically active small molecules were screened for inhibitory activity, and a lead compound was characterized in detail. Y-27632, an inhibitor of the Rho-associated kinase p160ROCK, diminished polyglutamine protein aggregation at micromolar concentrations, and reduced neurodegeneration in a Drosophila model of polyglutamine disease.
Claims18
A method of modulating aggregation of a first protein in a cell, wherein the first protein aggregates in the cell and is selected from the group consisting of a polyglutamine protein, a tau protein, a synuclein, a superoxide dismutase, a PABPN1, an Aβ peptide, a serpin, a transthyretin, an ataxin, and a prion protein, the method comprising administering a sufficient amount of a protein aggregation regulator to the cell, wherein said protein aggregation regulator comprises a small molecule, such that aggregation of the first protein is modulated.
Dependencies are inferred from the claim text.