[NFS-03] Neuroscience Frontier Symposium 03
Leading edge of ALS research

[NFS-03-2] Disruption of RNA metabolism in neurodegenerative diseases and emerging therapeutic strategies

^○Clotilde Lagier-Tourenne^1,2 (1.Sean M. Healey & AMG Center for ALS at the Massachusetts General Hospital, USA, 2.Harvard Medical School, USA)

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease resulting from loss of upper and lower motor neurons. Although the majority of ALS cases are sporadic, 5–10% of patients have a positive family history. The pivotal histopathological finding of sporadic ALS is intraneuronal aggregates of TDP-43. Recent advancement in genetics identified a variety genetic cause of ALS, including C9orf72, TDP-43, FUS and SOD1. This provides a tight link between this disease and frontotemporal lobar degeneration (FTLD) as well as important molecular insight into ALS/FTLD such as dysregulated RNA metabolism, disrupted protein quality control, axonal transport impairment and nuclear transport deficit, among others. In this symposium, cutting-edge research on the pathogenesis and therapy development for ALS and related disorders will be discussed.

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 and is a standing member of the CMND study section of the NIH. 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 and Harvard Medical School 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. She is the recipient of the Healey Family ALS Endowed Chair for Research.