Disease Fact Sheet: Huntington’s Disease (HD)
Clinical Status: To date, there is no effective treatment for HD. Over the past few years, researchers have focused on both the role of neuronal and glial dysfunction in causing neuronal and synaptic dysfunction of HD. As a result, both neural and glial transplantation-based strategies, using progenitor cells produced from human pluripotent stem cells, are under intensive preclinical development. These studies have strongly suggested the therapeutic potential of a cell replacement strategy in HD, whether accomplished by neural or glial progenitor cells. As a result, efforts are now underway in several centers to produce clinically appropriate populations of both neural and glial progenitor cells for intracerebral transplantation in symptomatic HD patients.