Antisense oligonucleotide therapy for KCNT1 encephalopathy
Lisseth Estefania Burbano, Melody Li, Nikola Jancovski, Paymaan Jafar‐Nejad, Kay Richards, Alicia Sedó, Armand Soriano, Ben Rollo, Linghan Jia, Elena V. Gazina, Sandra Piltz, Fatwa Adikusuma, Paul Quinton Thomas, Helen Kopsidas, Frank Rigo, , Steven Petrou
This study demonstrates that antisense oligonucleotide therapy reduces seizures and improves survival in a KCNT1 mouse model of encephalopathy.
JCI Insight · Sep 29, 2022Peer-reviewedNeuroscience