Gestörter Kerntransport und Phasentrennung von RNA-Bindeproteinen

Abstract

Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are fatal neurodegenerative disordes, whose underlying molecular mechanisms are only beginning to emerge. A common molecular hallmark of both diseases is the relocalization of nuclear RNA-binding proteins (RBP) into cytoplasmic aggregates. Defects in nuclear import and aberrant phase separation appear to underlie RBP mislocalization and aggregation and could potentially be targeted in future therapies.