Pharmaceutical, cellular and genetic therapies for Huntington's disease

Abstract

HD (Huntington's disease) is a devastating neurodegenerative disorder caused by a polyglutamine expansion in the gene encoding the huntingtin protein. Presently, there is no known cure for HD and existing symptomatic treatments are limited. However, recent advances have identified multiple pathological mechanisms involved in HD, some of which have now become the focus of therapeutic intervention. In this review, we consider progress made towards developing safe and effective pharmaceutical-, cell- and genetic-based therapies, and discuss the extent to which some of these therapies have been successfully translated into clinical trials. These new prospects offer hope for delaying and possibly halting this debilitating disease.