Spinal Muscular Atrophy Organoid Study Points to Early Neurodevelopmental Defects

Scientists researching spinal muscular atrophy (SMA) at DZNE and the Dresden University of Technology have created what they say is the first patient-derived induced pluripotent stem cell model and a spinal cord organoid system. The researchers found SMA organoids showed abnormal development, suggesting early neurodevelopmental problems could contribute to later motor neuron degeneration. While current therapies can increase SMA-affected patient’s survival motor neuron protein levels, the researchers suggested these may not fix all SMA pathologies and that improved therapies could need to target gene regulation.