Functional characterization of the novel ALS susceptibility locus UNC 13A

Amyotrophic lateral sclerosis is an adult-onset, disabling and fatal disease characterized by progressive degeneration of motor neurons in brain and spinal cord. No cure is available for ALS and the median survival is 3 years. The main objective of our research proposal is to initiate a research line that translates recent successes in ALS genetics into functional studies, to identify pathogenic pathways and targets for treatment of patients suffering from this progressive fatal disease.