An ALS-associated TARDBP mutation drives cryptic exon inclusion and RNA dysregulation
Qi Zhang, Meng Liu, Xiaojuan Fan, N.Y. Chin, Yue Xu, Jessica Suh, Soukaina Amniouel, James Virga, Brothely Jones, Kaari L. Linask, Jizhong Zou, Christopher Grunseich, Markus Hafner, Lichun Ma, Yihong Ye, Wei Zheng
Human brain organoids show that a TDP-43 mutation causes RNA mis-splicing and neuronal stress in ALS.
bioRxiv · Nov 9, 2025 · PreprintNeuroscience