Amyotrophic Lateral Sclerosis (ALS) and frontotemporal dementia (FTD) are devastating diseases in which a protein called TDP-43 ends up in the wrong part of the cell, disrupting its normal role in managing RNA. This loss of TDP-43 function leads to problems with another protein, G3BP1, which is needed for cells to cope with stress. This project will study how a faulty form of G3BP1 behaves in neurons and test a potential therapy designed to fix the RNA error that causes it. The goal is to better understand these disease mechanisms and identify new treatment approaches for ALS and FTD.
