NeuroscienceJCI InsightPeer-reviewed
Antisense oligonucleotide therapy for KCNT1 encephalopathy
Lisseth Estefania Burbano, Melody Li, Nikola Jancovski…·Steven Petrou
This study demonstrates that antisense oligonucleotide therapy reduces seizures and improves survival in a KCNT1 mouse model of encephalopathy.
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