Generation and validation of a human iPSC-derived TDP-43 knockout model for ALS disease modeling.
Swetha Gurumurthy, Anushka Bhargava, Nguyen PT Huynh, Thomas J. Krzystek, Fernando G. Vieira, Kyle R. Denton
The paper generates and validates a human iPSC-derived TDP-43 knockout model for ALS, showing widespread cryptic exon inclusion and identifying potential therapeutic modulators.
bioRxiv · May 2, 2026 · PreprintNeuroscience