GeneticsbioRxivPreprint
Patient derived model of UBA5-associated encephalopathy identifies defects in neurodevelopment and highlights potential therapies
Helen Chen, Yong‐Dong Wang, Aidan W. Blan…·Heather C. Mefford
The paper develops a patient-derived cortical organoid model of UBA5-associated encephalopathy, identifies neurodevelopmental defects, and highlights potential therapeutic strategies.
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