Novel Chemical Inhibitor against SOD1 Misfolding and Aggregation Protects Neuron-Loss and Ameliorates Disease Symptoms in ALS Mouse Model

Using ELISA-based chemical screening with wild and mutant superoxide dismutase (SOD1) proteins, the authors screened a new small molecule, PRG-A01, which could block the misfolding/aggregation of SOD1 or TDP-43. The drug rescued the cell death induced by mutant SOD1 in human neuroblastoma cell line.