Clotilde Lagier-Tourenne is Associate Professor of Neurology at the Massachusetts General Hospital and Harvard Medical School. She is a member of the Sean M. Healey & AMG Center for ALS at Mass General and an associate member at the Broad Institute. She has served on the NEALS Scientific Advisory Board since 2016. She trained as a medical geneticist in France and at Columbia University. After a postdoctoral fellowship with Dr. Don Cleveland, she became Assistant Professor at UCSD in 2013, and moved to the Massachusetts General Hospital in 2015. She received the Alphonse Laveran Prize, the Milton-Safenowitz Fellowship, the MDA Career Development and Frick Foundation Awards, the 6th International Paulo Gontijo Award in Medicine, the 4th annual Jenny S. Henkel Lectureship, the 2019 Lisa S. Krivickas Lectureship, and the 2017 Grass Foundation – American Neurological Association Award in Neuroscience.
Her group 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.