Huntington's and other polyglutamine diseases: many effects of single gene mutations

Despite the passage of more than 15 years since the identification of the first polyglutamine disease-causing mutation, many unanswered questions about the etiologies of these disorders remain. This review discusses the major areas of suspected dysfunction, with an emphasis on recent literature. In addition, it attempts to associate the implicated disease mechanisms with the therapeutic targets likely to be explored in the near- and mid-range future.

Section editor:Graeme Bilde – Novartis Pharma, Basel, Switzerland