Huntington disease – Update on ongoing therapeutic developments and a look toward the future

Huntington's disease (HD) is a genetic condition inherited in an autosomal dominant pattern characterized by neurodegeneration. The root cause of HD is a CAG tract expansion occurring in exon 1 of the huntingtin (HTT) gene. This mutation sets in motion a series of detrimental events that progressively lead to neuronal death and structural changes, mainly within the central nervous system but also affecting other organs [1]. Currently, no disease-modifying treatments for HD exist, but an active research effort focuses on potential therapies, with several programs in various stages of development.