The need for mouse models is clear even when the therapy is not targeted to a particular pathological process: ASO approaches for SOD1-ALS and FUS-ALS have gone forward to the clinic after testing in mice and giving safe, positive outcomes, although the key pathological processes resulting in motor neuron death in vivo are not clear [10, 99, 100]. The gene discussed is SOD1; the disease is amyotrophic lateral sclerosis.