Co-Investigator(s): Heather Durham (Montreal Neurological Institute)
Collaborator(s): John Reid (QurCan), Mohammad Amini (QurCan)
This project aims to develop a new RNA therapy for the rare neurodegenerative disorder ARSACS, using advanced C-TERP nanoparticles to deliver treatment directly to neural cells. Unlike traditional gene therapies that can cause immune reactions and dosage issues, this RNA-based approach offers better control, repeat dosing, and improved delivery to the nervous system. Building on prior success with gene therapy, researchers will compare RNA and DNA delivery methods to determine the most effective strategy for restoring key proteins and protecting nerve cells. Initial studies will use long-term neural cell cultures, paving the way for future testing in animal models and other related conditions. This technology has broad potential for treating rare genetic disorders, and the collaboration with QurCan positions the team for further funding and industry partnerships, with interest already emerging from major pharmaceutical companies.