Isogenic patient-derived organoids reveal early neurodevelopmental defects in spinal muscular atrophy initiation

Isogenic patient-derived organoids reveal early neurodevelopmental defects in spinal muscular atrophy initiation

Whether neurodevelopmental defects underlie postnatal neuronal death in neurodegeneration is an intriguing hypothesis only recently explored. Here, we focus on spinal muscular atrophy (SMA), a neuromuscular disorder caused by reduced survival of motor neuron (SMN) protein levels leading to spinal mo...

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Main Authors: Tobias Graß, Zeynep Dokuzluoglu, Felix Buchner, Ines Rosignol, J Thomas, Antonio Caldarelli, Anna Dalinskaya, Jutta Becker, Fabian Rost, Michele Marass, Brunhilde Wirth, Marc Beyer, Lorenzo Bonaguro, Natalia Rodríguez‐Muela
Format: Artigo
Language:English
Published: 2024
Online Access:https://doi.org/10.1016/j.xcrm.2024.101659
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https://doi.org/10.1016/j.xcrm.2024.101659

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