Gene-modified mesenchymal stem cells as a potential treatment for Huntington’s disease: preparing for a planned Phase I clinical trial

Available to view on demand: the UC Davis research team present an update on preclinical development of their cell-gene therapy product and the planned Phase I trial. View webinar on demand Huntington's disease (HD) is an autosomal dominant neurodegenerative disease caused by a CAG triplet expansion in the Huntington gene, which causes progressive neuropsychiatric and motor dysfunction and leads to death. The HD mutation causes selective loss of striatal neurons, leading to more widespread brain degeneration. No current disease-modifying therapies exist, and treatment is strictly palliative. An important mechanism postulated to lead to neuronal death in HD is impaired expression...