A Humanized Mouse Model of DRPLA Recapitulates Epilepsy and Demonstrates Antisense Oligonucleotide-Mediated Disease Modification
Qian Wu, Inga Erickson, Stephanie Davidson, Sophia Shonka, Velvet L. Smith, Bereket Z. Gidi, Kevin M. Knox, Timothy W. Yu, Vikram Khurana, Jeff Carroll, Joanna A. Korecka, Melissa L. Barker‐Haliski
This study uses a humanized mouse model to show that antisense oligonucleotides reduce seizure activity and mortality in DRPLA.
bioRxiv · Oct 18, 2026 · PreprintNeuroscience