New animal models of TDP-43-related neurodegenerative diseases

IGAZ LM

Buenos Aires

Congreso; Reunión Anual de Sociedades Biocientíficas 2019; 2019

Sociedad Argentina de Investigación Clínica (SAIC)

Neurodegenerative diseasesare characterized by progressive dysfunction and loss of neurons associatedwith depositions of pathologically altered proteins. The discovery thataggregated transactive response DNA-binding protein 43 kDa (TDP-43) is a majorcomponent of pathological ubiquitinated inclusions in amyotrophic lateralsclerosis (ALS) and frontotemporal dementia (FTD) caused seminal progress in understandingthe etiology of these now so-called ?TDP-43 proteinopathies?. The role ofTDP-43 as a neurotoxicity trigger has been well documented in different invitro and in vivo experimental models. As such, the investigation of TDP-43pathomechanisms in various major neurodegenerative diseases is on the rise. Iwill present our efforts to understand the pathophysiological roles of TDP-43,using inducible transgenic mice that recapitulate key features of the ALS/FTDspectrum.