Supported projects

M201801 – Gene therapy in Friedreich’s Ataxia

Scientific leaders
Benoît Funalot and Angès Rötig

Friedreich’s Ataxia (FRDA) is the most common ataxia and belongs to mitochondrial diseases. The Necker-Enfants Malades Hospital is Reference Center for mitochondrial diseases and more than 200 patients with FRDA are followed. If treatments with idebenone, an antioxidant or deferiprone, an iron chelating agent can correct the cardiomyopathy present in most patients, there is so far, no therapy for neurological disorders. Our project is to develop a gene therapy of Friedreich’s ataxia by using fibroblasts from patients in which we have detected an iron metabolism anomaly and a mouse model of FRDA.

Doc/post-doc

Funding
DIM Thérapie Génique