Amyotrophic lateral sclerosis (ALS) causes motor neuron degeneration characterized by progressive muscle weakness and eventual death. The translation of animal model work to human ALS studies has been largely unsuccessful owing to the complex biology of this disease. Saravanan Karumbayaram and colleagues expressed mutant forms of superoxide dismutase 1 (SOD1) linked to familial ALS in human embryonic stem cell-derived motor neurons. Neurons expressing mutant SOD1 exhibited abnormalities typical of ALS neurons, such as reduced life span and shorter cell processes. Using human-derived cells as shown here may more accurately model familial ALS, and in turn lead to more effective ALS therapies.
2009
