Isogenic Patient-Derived Organoids Reveal Early Neurodevelopmental Defects in Spinal Muscular Atrophy Initiation

Using the first isogenic patient-derived iPSC model and a spinal cord organoid (SCO) system, the authors showed that SMA SCOs exhibited abnormal morphological development, reduced expression of early neural progenitor markers, and accelerated expression of motor neuron (MN) progenitor and MN markers.