The review, written with collaborator Dr. Gary Dunbar, discusses studies over the past decade that have used mesenchymal stem cells (MSCs) and MSCs engineered to produce neural growth factors in the brain, for the treatment of Huntington’s disease.

Some of the therapeutics, such as MSCs engineered to secrete brain-derived neurotrophic factor (MSC/BDNF) have been effective in HD mouse models. This agent has been shown to correct movement disorders and characteristic clasping phenotype in HD mice. MSC implantation into the striata has also been shown to restore striatal volume in HD mice. These studies are reviewed and summarized in table format in the manuscript.

The Dunbar and Nolta laboratories are working together, with many national and international collaborators, to bring the proposed therapeutic MSC/BDNF into Phase I clinical trials to treat Huntington’s disease.

This work is supported by CIRM translational grant TR1-01257 and CIRM disease team planning grant DR2A-05415, “MSC engineered to produce BDNF for the treatment of Huntington’s disease.”