Optogenetic Modeling of Human Neuromuscular Circuits in Duchenne Muscular Dystrophy with CRISPR and Pharmacological Corrections

Investigators combined transcriptome analysis and modeling of Duchenne muscular dystrophy (DMD) patient-derived neuromuscular circuits with CRISPR-corrected isogenic controls in compartmentalized microdevices and showed that neuromuscular junction volumes and optogenetic motor neuron–stimulated myofiber contraction were compromised in DMD neuromuscular circuits.