Huntington disease With funding from CIRM, the team is moving forward with groundbreaking plans to conduct what they hope will be the first stem cell gene therapy trial for Huntington’s disease. The planned clinical trial involves mesenchymal stem cells being used as delivery agents for BDNF (brain derived neurotrophic factor), a neural growth factor. The Huntington’s team is targeting early phases of HD, before the illness’s full impact takes its toll. Funding from Team KJ and patient advocates supported preliminary biosafety studies that enabled a successful CIRM grant submission. In the pipeline is exciting gene editing research that has the potential to treat the aggressive juvenile form of the disease (JHD). The JHD team is supported by a training grant from the National Institutes for Health, a generous donation from the Phyllis E. Dake Foundation, and other philanthropic contributions.

Learn more about the UC Davis Huntington’s Disease Clinic »