The role of TDP-43 in axonal mRNA trafficking and local translation in healthy and diseased motor neurons

The TDP-43 protein has emerged as an important factor in the ALS disease process. The function of TDP-43 in motor neurons, the nerve cells controlling voluntary muscle movement that are specifically affected in ALS, is still poorly understood. While most efforts focus on the function of this protein in the cell nucleus, the investigators have found that it is also present in the long axonal processes that connect motor neuron cell bodies of the spinal cord with muscles throughout the body. As a novel approach, they propose to investigate the role of TDP-43 in axons and on how mutations in TDP-43 affect this function. Characterizing its function in axons is an important goal to better understand ALS disease mechanisms and identify novel therapeutic approaches.