NIH R01 · 2025
Defining the Mechanisms and Consequences of Nuclear Defects in ALS/FTD
PROJECT SUMMARY A detailed insight into the molecular defects leading to neuronal death in ALS/FTD is still missing, hindering the development of a cure. Defects in the nuclear pore complex (NPC) and nucleocytoplasmic transport (NCT) have been proposed to play a central role in ALS/FTD pathology. An outstanding knowledge gap is what are the mechanisms, consequences, and pathogenic relevance of impaired NCT on neuronal resilience in neurodegenerative diseases and during aging. The objective of this application is to identify at the mechanistic level the novel cellular and molecular pathways that are dysfunctional and cause the failure of the NCT ultimately leading to neurodegeneration. In…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.