Several of the spinocerebellar ataxias (SCAs) result from expansion of polyglutamine (polyQ)-encoding regions in different genes. Here, Orr and colleagues examine the clinical features of the the polyQ SCAs, and suggest that understanding the molecular and physiological mechanisms underlying polyQ SCAs can inform therapeutic strategies for these and other polyQ disorders.
- Henry L. Paulson
- Vikram G. Shakkottai
- Harry T. Orr