Antisense oligonucleotide therapy for SCN2A gain-of-function epilepsy
Melody Li, Nikola Jancovski, Paymaan Jafar‐Nejad, Lisseth Estefania Burbano, Ben Rollo, Kay Richards, Lisa Drew, Alicia Sedó, Svenja Pachernegg, Armand Soriano, Linghan Jia, Todd Blackburn, Blaine Russell Roberts, Alex Nemiroff, Kelley Dalby, , Steven Petrou
This study shows that reducing SCN2A function with an antisense oligonucleotide improves survival and behavior in a mouse model of early seizure onset DEE.
bioRxiv · Sep 9, 2020 · PreprintNeuroscience