The research project has two specific aims: to continue experiments to determine if loss of sacsin functions in NF metabolism plays a central role in pathogenesis of ARSACS and to determine the effect of sacsin domains on the phenotype in our ARSACS culture models and based on those data to develop a therapeutic peptide to move forward in preclinical testing.
Duration: one year
Grant: $100,000
Contact:
Dr Heather Durham
Dr. Heather Durham, professor McGill University
Tel:(514)-398-8509; Email: heather.durham@mcgill.ca