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
Dr. Heather Durham, professor McGill University
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