Evidence that a particular chaperone can indeed moderate aggregation comes from studies in transfected cells expressing the chaperone and either mutant ataxin-l or mutant ARGln. The finding that the proteasome and chaperones are involved in SCA1 and SBMA pathology opens up intriguing new possibilities for therapeutic interventions in these and other polyglutamine disorders, as well as Alzheimer disease, Parkinson disease, and prion diseases. The invention provides a novel method for treating neurodegenerative disease. There is also a novel method for screening for compounds having chaperone-like activity or having activity to increase proteasome activity. The screening method works in either cultured cells or animal models.
Further studies are underway in transgenic mice and tissue culture cells.