We are currently considering a new form of funding for Professor Tremblay’s project. Our goal is to develop a mouse model for the research on Friedreich’s Ataxia.

The research project will soon be published in the American crowdfundig site CONSANO. Ataxia Canada will contribute equally to the amount collected.

Access to a mouse model remains a problem. Dr. Hélène Puccio’s model is not adequate for the type of research that Professor Tremblay would like to perform, as it does not contain the human gene frataxin. This mouse model containing the human frataxin gene obtained from a Friedreich’s Ataxia patient. However, the trinucleotide repeat in intron 1 is not long enough to produce a sufficient reduction of frataxin to induce clear disease symptoms. The goal of Dr. Tremblay project is to further reduce the expression of frataxin in the mouse so that it develops clearest symptoms. The presence of typical symptoms of Friedreich’s Ataxia in the improved mouse model would permit to assess more easily and clearly the efficacy of different therapies.

This extremely important project will bring us closer to clinical trials.