Published on January 29, 2014 at 7:46 AM
The project involves the research groups headed by Javier D-az-Nido at the CBMSO, an international expert in gene therapy and Friedreich's ataxia, and Ernest Giralt at IRB, an international authority on the design of transporters, such as nanoparticles, that can carry drugs into the brain and thus overcome the blood-brain barrier. Patients with this ataxia inherit a mutated version of the frataxin gene, which causes neurodegeneration. The project seeks to rescue this defect in cells of the central nervous system.
Jennifer Farmer, executive director of FARA, explains "when you are working on a rare disease, critical resources, such as funding, are quite small so it is imperative that FARA and other global groups with an interest in supporting and advancing research in Friedreich's ataxia work together". "We can't afford to duplicate effort or compete." She goes on to add that "we are proud to give our support to the solid scientific project led by doctors D-az-Nido and Giralt to identify new therapies and at the same time to strike up an alliance with patients in Spain."
Source: Institute for Research in Biomedicine (IRB Barcelona)