Clotilde Lagier-Tourenne’s laboratory investigates the molecular mechanisms driving neurodegeneration in amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and Huntington’s disease. Mutations and/or cellular mislocalization of several RNA binding proteins have been identified as central components in the pathogenesis of ALS and FTD. Using innovative cellular and genomics techniques, the Lagier-Tourenne’s group explores the regulatory networks between RNA binding proteins and changes in RNA expression that occur in these diseases. The team also develops cellular and animal models to uncover mechanistic insights underlying neuronal death in ALS and FTD patients with C9ORF72 expansion, the most common genetic cause of these conditions. Clotilde has established collaborations with academic and pharmaceutical partners to develop novel approaches to therapy, including RNA-targeting antisense oligonucleotides and immunotherapies for patients with ALS and FTD. In particular, her work in collaboration with IONIS Pharmaceuticals is at the stem of the therapeutic development of antisense oligonucleotides in C9ORF72 disease and the recent initiation by Biogen of a clinical trial in ALS patients.