A splice-switching antisense oligonucleotide approach for pediatric genetic epilepsies
Haley B. Dame, Hema Kopalle, Belén Miñana, Zin‐Juan Klaft, Jaclyn B. Fahey, David C. McWatters, Vama Rao, Rushil Suresh, Stefan Aigner, Michael F. Hammer, Christopher J. Yuskaitis, Megan Wong, Nicole A. Teaney, Crystal Zhang, Mike Thompson, , Madeleine Julie Oudin
This study identifies splice-switching antisense oligonucleotides that correct splicing defects in SCN8A, reducing seizures and improving survival in a mouse model of genetic epilepsy.
bioRxiv · Oct 22, 2025 · PreprintNeuroscience