Amyotrophic lateral sclerosis (ALS) is a neurodegenerative condition with a prevalence of 2-3 per 100,000 people, involving the loss of large motor neurons in the brain and spinal cord. It is characterized by progressive paralysis that leads to death typically in 3-5 years. Familial ALS accounts for 10% of all ALS cases and approximately 25% of these cases are due to mutations in the Cu/Zn superoxide dismutase gene (SOD1). The invention relates to a high throughput assay and methods for detecting inhibitors. The invention also relates to methods of using the inhibitors to treat and prevent disease.
Assay for therapies that inhibit expression of the cytosolic Cu/Zn superoxide dismutase (SOD1) gene