METHODS FOR IDENTIFYING AGENTS USEFUL FOR MODULATING THE EXPRESSION AND AGGREGATION OF CAG-EXPANDED GENE PRODUCT IN CELLS

摘要

This invention provides a method for modulating the expression of a first gene in a cell wherein the first gene is one containing more than 36 CAG trinucleotide repeats and encoding a protein that form polyglutamine-mediated protein aggregation. Suppression of the first gene is achieved by reducing the expression of SPT4 gene or SUPT4H gene. It can also be achieved by inhibiting the formation of a Spt4/Spt5 complex or a Supt4h/Supt5h complex. Also provided is a method for identifying an agent useful for modulating the expression and aggregation of CAG-expanded gene product, or treating a polyglutamine disease such as Huntington's disease.

主权项

1. A method for identifying a compound useful for modulating a first gene, or for treating a polyglutamine disease, said method comprising:
screening a plurality of test compounds to identify one having an inhibitory activity capable of disrupting formation of an Spt4/Spt5 complex or an Supt4h/Supt5h complex, wherein said first gene contains expanded CAG repeats.