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, Yanchen Liu, Chunlong Ma, Phyo Han Thwin, Xuyao Chang, George Yoshida, Annie Li, Cenjing Wang, Crace Westerkamp, Lauren Schmitt, Maag Chelsey, Monzon Stephanie, Yu Zhao, , 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.
bioRxiv · Oct 14, 2024 · PreprintNeuroscience