An iPSC model of fragile X syndrome reflects clinical phenotypes and reveals m6A-mediated epi-transcriptomic dysregulation underlying synaptic dysfunction
Lu Lu, Avijite Kumer Sarkar, Lan Dao…·Ziyuan Guo
This study uses iPSC-derived neurons to show that FMRP deficiency in fragile X syndrome leads to m6A-mediated transcriptome dysregulation, causing synaptic dysfunction and suggesting therapeutic targets.