Claims
- 1. A method of identifying a compound useful in the treatment of a disease characterized by the presence of mutant huntingtin, comprising
(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, wherein an increase in clathrin-mediated endocytosis is an indication that the test compound is useful in treating said disease.
- 2. The method of claim 1, wherein said test compound activates GTP-binding proteins.
- 3. The method of claim 1, wherein said test compound activates protein kinase C.
- 4. The method of claim 3, wherein said activator of protein kinase C is a phorbol ester.
- 5. The method of claim 1, wherein said test compound alters the pH of the extracellular environment.
- 6. The method of claim 1, wherein said test compound alters the concentration of extracellular calcium.
- 7. The method of claim 1, wherein said test compound is a fungal metabolite.
- 8. The method of claim 1, wherein said cells are obtained from a patient who has been diagnosed as having Huntington's Disease.
- 9. The method of claim 8, wherein said cells are neurons.
- 10. The method of claim 8, wherein said cells are fibroblasts.
- 11. The method of claim 1, wherein the method by which said level of clathrin-mediated endocytosis is determined comprises monitoring the rate of uptake of biotinylated transferrin and its receptor.
- 12. A method of determining whether a compound is useful for treatment of a patient who has been diagnosed as having Huntington's Disease, comprising
(a) providing a cell obtained from said patient; (b) contacting the cell with the compound; and (c) determining whether the level of clathrin-mediated endocytosis is increased in the presence of the compound, compared to the level in the absence of the compound, wherein an increase in clathrin-mediated endocytosis is an indication that the compound is useful in treating said patient.
CROSS REFERENCE TO RELATED APPLICATIONS
[0001] This application claims priority from U.S. provisional application Ser. No. 60/030,940, filed Nov. 15, 1996.
STATEMENT AS TO FEDERALLY SPONSORED RESEARCH
[0002] This invention was made with Government support under Contract Numbers NS16367 and NS31579 awarded by the National Institutes of Health. The Government may have certain rights in this invention.
Provisional Applications (1)
|
Number |
Date |
Country |
|
60030940 |
Nov 1996 |
US |