Degenerative ataxias: genetics, pathogenesis and animal models.

At present, seven inherited neurodegenerative disorders have been identified to be caused by the polyglutamine-coding CAG triplet expansions in the genes responsible. The long stretch of polyglutamines appears to play a key role in the pathogenesis. Disease models, just recently established with cultured cells and animals, will enable elucidation of common disease mechanisms and treatments.