CONICET | Buscador de Institutos y Recursos Humanos

Neurodegenerative diseases are characterized byprogressive dysfunction and loss of neurons associated with depositions of pathologicallyaltered proteins. The discovery that aggregated transactive responseDNA-binding protein 43 kDa (TDP-43) is a major component of pathologicalubiquitinated inclusions in amyotrophic lateral sclerosis (ALS) and frontotemporaldementia (FTD) caused seminal progress in understanding the etiology of thesenow so-called ?TDP-43 proteinopathies?. The role of TDP-43 as a neurotoxicitytrigger has been well documented in different in vitro and in vivo experimentalmodels. As such, the investigation of TDP-43 pathomechanisms in various majorneurodegenerative diseases is on the rise. I will present our efforts tounderstand the pathophysiological roles of TDP-43, using inducible transgenicmice that recapitulate key features of the ALS/FTD spectrum.