Article

An isogenic human iPSC model unravels neurodevelopmental abnormalities in SMA

2023-01-03

Abstract excerpt

Whether neurodevelopmental defects underlie the selective neuronal death that characterizes neurodegenerative diseases is becoming an intriguing question. To address it, we focused on the motor neuron (MN) disease Spinal Muscular Atrophy (SMA), caused by reduced levels of the ubiquitous protein SMN. Taking advantage of the first isogenic human induced pluripotent stem cell-derived SMA model that we have generated...

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Literature Corpus work: 5e8f6fc8-8765-54db-b7c8-737f60a7b2b9
DOI: 10.1101/2023.01.02.522499

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