MOLECULAR TARGETS AND COMPOUNDS, METHODS TO IDENTIFY THE SAME, USEFUL IN THE TREATMENT OF NEURODEGENERATIVE DISEASES.

摘要

The present invention relates to methods and assays for identifying agents capable of inhibiting the mutant huntingtin protein, inhibiting or reducing polyglutamine-induced protein aggregation, and/or altering huntingtin protein conformation, which inhibition is useful in the prevention, amelioration and / or treatment of neurodegenerative diseases, and protein aggregation diseases more generally. In particular, the present invention provides methods and assays for identifying agents for use in the prevention and / or treatment of Huntingtons disease. The invention provides polypeptide and nucleic acid TARGETs and siRNA sequences based on these TARGETs.