In conjunction with the National Institutes of Health (NIH), the Foundation awarded a grant to the Pittsburgh Institute for Neurodegenerative Diseases (PIND) at the University of Pittsburgh to study the feasibility, safety, and effectiveness of a gene therapy approach to reduce levels of alpha-synuclein protein in a model of Parkinson’s disease. Excessive alpha-synuclein protein in brain cells is associated with the destruction of neurons and the spread of PD. Scientists at PIND designed a gene to target alpha-synuclein which was then packaged into a virus for delivery to the brain. The study found that this form of gene therapy (i) was safe, (ii) improved motor function, and (iii) preserved dopamine neurons and their dendritic and axonal processes.

Results of this study were featured in the June 15, 2015 publication of the Journal of Clinical Investigation. To read the full press release, click here.

It is hoped and anticipated that this and similar approaches can be translated to human clinical trials in the next few years.