Chemically oligomerizable TDP-43:a novel chemogenetic tool for studying the pathophysiology of amyotrophic lateral sclerosis

摘要

Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease characterized by the progressive loss of both upper and lower motor neurons.Most ALS cases are sporadic,but approximately 5-10%of patients have a familial background.To date,more than 30 familial ALS-causative genes have been identified (Maurel et al., 2018).