Importantly for ALS, the preclinical rationale behind ASO knockdown of SOD1 was strong and resulted from a number of years of methodical analyses of RNA transcript and protein levels in ALS fibroblasts, normal rats and monkeys, mSOD1G93A rats and mice, and the correlation with histopathology and behavior.92, 93, 94. The gene discussed is SOD1; the disease is amyotrophic lateral sclerosis.