Huntingtin-related therapeutics and bioassay

摘要

A method of identifying a compound useful in the treatment of a disease characterized by the presence of mutant huntingtin by: (a) providing a cell which expresses mutant huntingtin; (b) contacting the cell with a test compound; and (c) determining whether the level of clathrin-mediated endocytosis is increased in the presence of the test compound, compared to the level in the absence of the test compound. An increase in clathrin-mediated endocytosis is an indication that the test compound is useful in treating the disease.