Modelling neuromuscular disease
We have established a compartmented co-culture system composed of motor neuron, glia and myofibers directly derived from pluripotent stem cells. In this neuromuscular circuit model, motor axons form functional synapses with myofibers, and optogenetic stimulation of motor neurons induces myofiber contraction. We applied this model to the diseases Amyotrophic Lateral Sclerosis and Duchenne Muscular Dystrophy, and in both cases, we observed defects in nerve-muscle connectivity, which could be rescued by candidate drugs.