“Therapeutic Approaches for ARSACS”- Dr Benoit Gentil et Dre Heather Durham

This research focuses on developing a potential gene therapy for ARSACS, a rare inherited neurological disease. Scientists created a smaller version of the faulty protein (called minisacsin) that can fit into a viral vector for delivery to nerve cells. In mouse models, early treatment with this therapy delayed or reduced disease symptoms, including movement problems and retinal changes. The team is now testing whether the therapy can also help after symptoms begin.  Our results so far provide a better understanding of the ARSACS pathogenesis would complete preclinical studies aiming at developing  a gene therapy for ARSACS.

Financement : 100 000$

Durée: un an