“Role of Neurofilaments and Mitochondria in the Pathogenic Cascade of ARSACS : Relevant Biomarkers for Therapeutic Development” – Dr. Heather Durham

In Research in 2016 by ARSACS

The research project has the following  specific objectives :

Aim 1. Determine the effect of sacsin domains

Aim 1.1:  Determine the effect of sacsin domains on IF architecture and IF bundling in ARSACS models.  Aim 1.2: Determine which domains in combination are essential to rescue the ARSACS phenotype.

Aim 2. Develop a peptide drug using neuroprotective sacsin domains. 

Aim 2.1: Optimize the therapeutic polypeptide sequence.

Aim 2.2: Verify intracellular delivery of Sacsin domain polypeptides.

Aim 2.3:  Investigate the dose-response of peptide efficacy in alleviating the phenotype in ARSACS culture models.

Aim 2.4:  Conduct a preliminary investigation of toxicity of Sacs.


Duration: one year
Grant: $100,000

Dr Heather Durham

Dr Heather Durham

Dr. Heather Durham, professor McGill University
Tel:(514)-398-8509; Email: heather.durham@mcgill.ca

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