Clotilde Lagier-Tourenne, MD, PhD, performs patient-oriented research to understand the molecular mechanisms driving neurodegeneration in Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) and to develop therapeutic strategies. 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, 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. Lagier-Tourenne 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 linked to C9orf72 expansion.