Ataxia UK is funding a new project led by Dr Filip Lim’s research group at the Autonomous University of Madrid. The project builds upon their previous research into a gene therapy for Friedreich’s ataxia.

In patients with FA the FXN gene, which provides instructions for making the protein frataxin, does not work properly. The gene therapy being investigated involves replacing the faulty FXN gene with a fully functional version using something called a viral ‘vector’; a virus combined with a therapeutic gene.

The researchers want to see how well the vector works in animal models of FA, but first need to develop new models of FA which are cost-effective and quick to produce. In this project, they are going to use the vector they have developed to create new animal models of FA. In this case, the vector will be used for a purpose other than gene therapy, but the researchers will also learn useful information about how effective the vector may be as a therapy.

The information gained should help to speed up their efforts to move their laboratory research into trials in humans.

Posted on 13/08/2019